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Abuse of the Safety-Net 340B Drug Pricing Program: Why Should Physicians Care?
The 340B Drug Pricing Program began as a noble endeavor, a lifeline designed to help safety-net providers deliver affordable care to America’s most vulnerable populations. However, over the years, this well-intentioned program has strayed from its original purpose, becoming a lucrative space where profits often outweigh patients. Loopholes, lax oversight, and unchecked expansion have allowed some powerful players, such as certain disproportionate share hospitals and their “child sites” as well as for-profit pharmacies, to exploit the system. What was once a program to uplift underserved communities now risks becoming a case study in how good intentions can go astray without accountability.
What exactly is this “340B program” that has captured headlines and the interest of legislatures around the country? What ensures that pharmaceutical manufacturers continue to participate in this program? How lucrative is it? How have underserved populations benefited and how is that measured?
The 340B Drug Pricing Program was established in 1992 under the Public Health Service Act. Its primary goal is to enable covered entities (such as hospitals and clinics serving low-income and uninsured patients) to purchase outpatient drugs from pharmaceutical manufacturers at significantly reduced prices in order to support their care of the low-income and underserved populations. Drug makers are required to participate in this program as a condition of their participation in Medicaid and Medicare Part B and offer these steep discounts to covered entities if they want their medications to be available to 38% of patients nationwide.
The hospitals that make up 78% of the program’s spending are known as disproportionate share hospitals (DSHs). These hospitals must be nonprofit and have at least an 11.75% “disproportionate” share of low-income Medicare or Medicaid inpatients. The other types of non-hospital entities qualifying for 340B pricing are known as initial “federal grantees.” Some examples include federally qualified health centers (FQHC), Ryan White HIV/AIDS program grantees, and other types of specialized clinics, such as hemophilia treatment centers. It needs to be noted up front that it is not these initial non-hospital federal grantees that need more oversight or reform, since according to the Health Resources and Services Administration (HRSA) 2023 report they make up only 22% of all program spending. It is the large, predominantly DSH health systems that are profiting immensely through exponential growth of their clinics and contract pharmacies. However, these health systems have not been able to show exactly who are their eligible patients and how they have been benefiting them.
When the 340B program was established to offer financial relief to hospitals and clinics taking care of the uninsured, it allowed them to save 20%-50% on drug purchases, which could be reinvested in patient care services. It was hoped that savings from the program could be used to provide free or low-cost medications, free vaccines, and other essential health services, essentially allowing safety-net providers to serve their communities despite financial constraints. The initial grantees are fulfilling that mission, but there are concerns regarding DSHs. (See the Coalition of State Rheumatology Organization’s 340B explanatory statement and policy position for more.)
Why Should Independent Practice Physicians Care About This?
Independent doctors should care about the lack of oversight in the 340B program because it affects healthcare costs, patient assistance, market competition, and access to affordable care for underserved and uninsured patients.
It also plays a strong hand in the healthcare consolidation that continues to threaten private physician practices. These acquisitions threaten the viability of independent practices in a variety of specialties across the United States, including rheumatology. HRSA allows 340B-covered entities to register their off-campus outpatient facilities, or child sites, under their 340B designation. Covered entities can acquire drugs at the 340B price, while imposing markups on the reimbursement they submit to private insurance. The additional revenue these covered entities can pocket provides them with a cash flow advantage that physician practices and outpatient clinics will never be able to actualize. This uneven playing field may make rheumatology practices more susceptible to hospital acquisitions. In fact, between 2016 and 2022, large 340B hospitals were responsible for approximately 80% of hospital acquisitions.
Perhaps the most important reason that we should all be concerned about the trajectory of this well-meaning program is that we have seen patients with hospital debt being sued by DSHs who receive 340B discounts so that they can take care of the low-income patients they are suing. We have seen Medicaid patients be turned away from a DSH clinic after being discharged from that hospital, because the hospital had reached its disproportionate share (11.75%) of inpatient Medicare and Medicaid patients. While not illegal, that type of behavior by covered entities is WRONG! Oversight and reform are needed if the 340B program is going to live up to its purpose and not be just another well-intentioned program not fulfilling its mission.
Areas of Concern
There has been controversy regarding the limited oversight of the 340B program by HRSA, leading to abuse of the program. There are deep concerns regarding a lack of transparency in how savings from the program are being used, and there are concerns about the challenges associated with accurate tracking and reporting of 340B discounts, possibly leading to the duplication of discounts for both Medicaid and 340B. For example, a “duplicate discount” occurs if a manufacturer sells medications to a DSH at the 340B price and later pays a Medicaid rebate on the same drug. The extent of duplicate discounts in the 340B program is unknown. However, an audit of 1,536 cases conducted by HRSA between 2012 and 2019 found 429 instances of noncompliance related to duplicate discounts, which is nearly 30% of cases.
DSHs and their contracted pharmacies have been accused of exploiting the program by increasing the number of contract pharmacies and expanding the number of offsite outpatient clinics to maximize profits. As of mid-2024, the number of 340B contract pharmacies, counted by Drug Channels Institute (DCI), numbered 32,883 unique locations. According to DCI, the top five pharmacies in the program happen also to be among the top pharmacy revenue generators and are “for-profit.” They are CVS, Walgreens, Walmart, Express Scripts, and Optum RX. Additionally, a study in JAMA Health Forum showed that, from 2011 to 2019, contract pharmacies in areas with the lowest income decreased by 5.6% while those in the most affluent neighborhoods grew by 5%.
There also has been tremendous growth in the number of covered entities in the 340B program, which grew from just over 8,100 in 2000 to 50,000 in 2020. Before 2004, DSHs made up less than 10% of these entities, but by 2020, they accounted for over 60%. Another study shows that DSHs are expanding their offsite outpatient clinics (“child clinics”) into the affluent neighborhoods serving commercially insured patients who are not low income, to capture the high commercial reimbursements for medications they acquired at steeply discounted prices. This clearly is diverting care away from the intended beneficiaries of the 340B program.
Furthermore, DSHs have been acquiring specialty practices that prescribe some of the most expensive drugs, in order to take advantage of commercial reimbursement for medications that were acquired at the 340B discount price. Independent oncology practices have complained specifically about this happening in their area, where in some cases the DSHs have “stolen” their patients to profit off of the 340B pricing margins. This has the unintended consequence of increasing government spending, according to a study in the New England Journal of Medicine that showed price markups at 340B eligible hospitals were 6.59 times as high as those in independent physician practices after accounting for drug, patient, and geographic factors.
Legal Challenges and Legislation
On May 21, 2024, the US Court of Appeals for the DC Circuit issued a unanimous decision in favor of drug manufacturers, finding that certain manufacturer restrictions on the use of contract pharmacies under the 340B drug pricing program are permissible. The court’s decision follows a lower court (3rd Circuit) ruling which concluded that the 340B statute does not require manufacturers to deliver 340B drugs to an “unlimited number of contract pharmacies.” We’re still awaiting a decision from the 7th Circuit Court on a similar issue. If the 7th Circuit agrees with the government, creating a split decision, there is an increase in the likelihood that the Supreme Court would take up the case.
Johnson & Johnson has also sued the federal government for blocking their proposed use of a rebate model for DSHs that purchase through 340B two of its medications, Stelara and Xarelto, whose maximum fair price was negotiated through the Inflation Reduction Act’s Medicare Drug Price Negotiation Program. J&J states this would ensure that the claims are actually acquired and dispensed by a covered 340B entity, as well as ensuring there are no duplicate discounts as statutorily required by the IRA. When initially proposed, HRSA threatened to remove J&J’s access to Medicare and Medicaid if it pursued this change. J&J’s suit challenges that decision.
However, seven states (Arkansas, Kansas, Louisiana, Minnesota, Missouri, Mississippi, and West Virginia) have been active on this issue, passing laws to prevent manufacturers from limiting contract pharmacies’ ability to acquire 340B-discounted drugs. The model legislation also bans restrictions on the “number, location, ownership, or type of 340B contract pharmacy.”
It should also be noted that there are states that are looking for ways to encourage certain independent private practice specialties (such as gastroenterology and rheumatology) to see Medicaid patients, as well as increase testing for sexually transmitted diseases, by offering the possibility of obtaining 340B pricing in their clinics.
Shifting our focus to Congress, six bipartisan Senators, known as the Group of 6, are working to modernize the 340B program, which hasn’t been updated since the original law in 1992. In 2024, legislation was introduced (see here and here) to reform a number of the features of the 340B drug discount program, including transparency, contract pharmacy requirements, and federal agency oversight.
Who’s Guarding the Hen House?
The Government Accountability Office and the Office of Inspector General over the last 5-10 years have asked HRSA to better define an “eligible” patient, to have more specifics concerning hospital eligibility criteria, and to have better oversight of the program to avoid duplicate discounts. HRSA has said that it doesn’t have the ability or the funding to achieve some of these goals. Consequently, little has been done on any of these fronts, creating frustration among pharmaceutical manufacturers and those calling for more oversight of the program to ensure that eligible patients are receiving the benefit of 340B pricing. Again, these frustrations are not pointed at the initial federally qualified centers or “grantees.”
HRSA now audits 200 covered entities a year, which is less than 2% of entities participating in the 340B program. HRSA expects the 340B entities themselves to have an oversight committee in place to ensure compliance with program requirements.
So essentially, the fox is guarding the hen house?
Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at rhnews@mdedge.com.
The 340B Drug Pricing Program began as a noble endeavor, a lifeline designed to help safety-net providers deliver affordable care to America’s most vulnerable populations. However, over the years, this well-intentioned program has strayed from its original purpose, becoming a lucrative space where profits often outweigh patients. Loopholes, lax oversight, and unchecked expansion have allowed some powerful players, such as certain disproportionate share hospitals and their “child sites” as well as for-profit pharmacies, to exploit the system. What was once a program to uplift underserved communities now risks becoming a case study in how good intentions can go astray without accountability.
What exactly is this “340B program” that has captured headlines and the interest of legislatures around the country? What ensures that pharmaceutical manufacturers continue to participate in this program? How lucrative is it? How have underserved populations benefited and how is that measured?
The 340B Drug Pricing Program was established in 1992 under the Public Health Service Act. Its primary goal is to enable covered entities (such as hospitals and clinics serving low-income and uninsured patients) to purchase outpatient drugs from pharmaceutical manufacturers at significantly reduced prices in order to support their care of the low-income and underserved populations. Drug makers are required to participate in this program as a condition of their participation in Medicaid and Medicare Part B and offer these steep discounts to covered entities if they want their medications to be available to 38% of patients nationwide.
The hospitals that make up 78% of the program’s spending are known as disproportionate share hospitals (DSHs). These hospitals must be nonprofit and have at least an 11.75% “disproportionate” share of low-income Medicare or Medicaid inpatients. The other types of non-hospital entities qualifying for 340B pricing are known as initial “federal grantees.” Some examples include federally qualified health centers (FQHC), Ryan White HIV/AIDS program grantees, and other types of specialized clinics, such as hemophilia treatment centers. It needs to be noted up front that it is not these initial non-hospital federal grantees that need more oversight or reform, since according to the Health Resources and Services Administration (HRSA) 2023 report they make up only 22% of all program spending. It is the large, predominantly DSH health systems that are profiting immensely through exponential growth of their clinics and contract pharmacies. However, these health systems have not been able to show exactly who are their eligible patients and how they have been benefiting them.
When the 340B program was established to offer financial relief to hospitals and clinics taking care of the uninsured, it allowed them to save 20%-50% on drug purchases, which could be reinvested in patient care services. It was hoped that savings from the program could be used to provide free or low-cost medications, free vaccines, and other essential health services, essentially allowing safety-net providers to serve their communities despite financial constraints. The initial grantees are fulfilling that mission, but there are concerns regarding DSHs. (See the Coalition of State Rheumatology Organization’s 340B explanatory statement and policy position for more.)
Why Should Independent Practice Physicians Care About This?
Independent doctors should care about the lack of oversight in the 340B program because it affects healthcare costs, patient assistance, market competition, and access to affordable care for underserved and uninsured patients.
It also plays a strong hand in the healthcare consolidation that continues to threaten private physician practices. These acquisitions threaten the viability of independent practices in a variety of specialties across the United States, including rheumatology. HRSA allows 340B-covered entities to register their off-campus outpatient facilities, or child sites, under their 340B designation. Covered entities can acquire drugs at the 340B price, while imposing markups on the reimbursement they submit to private insurance. The additional revenue these covered entities can pocket provides them with a cash flow advantage that physician practices and outpatient clinics will never be able to actualize. This uneven playing field may make rheumatology practices more susceptible to hospital acquisitions. In fact, between 2016 and 2022, large 340B hospitals were responsible for approximately 80% of hospital acquisitions.
Perhaps the most important reason that we should all be concerned about the trajectory of this well-meaning program is that we have seen patients with hospital debt being sued by DSHs who receive 340B discounts so that they can take care of the low-income patients they are suing. We have seen Medicaid patients be turned away from a DSH clinic after being discharged from that hospital, because the hospital had reached its disproportionate share (11.75%) of inpatient Medicare and Medicaid patients. While not illegal, that type of behavior by covered entities is WRONG! Oversight and reform are needed if the 340B program is going to live up to its purpose and not be just another well-intentioned program not fulfilling its mission.
Areas of Concern
There has been controversy regarding the limited oversight of the 340B program by HRSA, leading to abuse of the program. There are deep concerns regarding a lack of transparency in how savings from the program are being used, and there are concerns about the challenges associated with accurate tracking and reporting of 340B discounts, possibly leading to the duplication of discounts for both Medicaid and 340B. For example, a “duplicate discount” occurs if a manufacturer sells medications to a DSH at the 340B price and later pays a Medicaid rebate on the same drug. The extent of duplicate discounts in the 340B program is unknown. However, an audit of 1,536 cases conducted by HRSA between 2012 and 2019 found 429 instances of noncompliance related to duplicate discounts, which is nearly 30% of cases.
DSHs and their contracted pharmacies have been accused of exploiting the program by increasing the number of contract pharmacies and expanding the number of offsite outpatient clinics to maximize profits. As of mid-2024, the number of 340B contract pharmacies, counted by Drug Channels Institute (DCI), numbered 32,883 unique locations. According to DCI, the top five pharmacies in the program happen also to be among the top pharmacy revenue generators and are “for-profit.” They are CVS, Walgreens, Walmart, Express Scripts, and Optum RX. Additionally, a study in JAMA Health Forum showed that, from 2011 to 2019, contract pharmacies in areas with the lowest income decreased by 5.6% while those in the most affluent neighborhoods grew by 5%.
There also has been tremendous growth in the number of covered entities in the 340B program, which grew from just over 8,100 in 2000 to 50,000 in 2020. Before 2004, DSHs made up less than 10% of these entities, but by 2020, they accounted for over 60%. Another study shows that DSHs are expanding their offsite outpatient clinics (“child clinics”) into the affluent neighborhoods serving commercially insured patients who are not low income, to capture the high commercial reimbursements for medications they acquired at steeply discounted prices. This clearly is diverting care away from the intended beneficiaries of the 340B program.
Furthermore, DSHs have been acquiring specialty practices that prescribe some of the most expensive drugs, in order to take advantage of commercial reimbursement for medications that were acquired at the 340B discount price. Independent oncology practices have complained specifically about this happening in their area, where in some cases the DSHs have “stolen” their patients to profit off of the 340B pricing margins. This has the unintended consequence of increasing government spending, according to a study in the New England Journal of Medicine that showed price markups at 340B eligible hospitals were 6.59 times as high as those in independent physician practices after accounting for drug, patient, and geographic factors.
Legal Challenges and Legislation
On May 21, 2024, the US Court of Appeals for the DC Circuit issued a unanimous decision in favor of drug manufacturers, finding that certain manufacturer restrictions on the use of contract pharmacies under the 340B drug pricing program are permissible. The court’s decision follows a lower court (3rd Circuit) ruling which concluded that the 340B statute does not require manufacturers to deliver 340B drugs to an “unlimited number of contract pharmacies.” We’re still awaiting a decision from the 7th Circuit Court on a similar issue. If the 7th Circuit agrees with the government, creating a split decision, there is an increase in the likelihood that the Supreme Court would take up the case.
Johnson & Johnson has also sued the federal government for blocking their proposed use of a rebate model for DSHs that purchase through 340B two of its medications, Stelara and Xarelto, whose maximum fair price was negotiated through the Inflation Reduction Act’s Medicare Drug Price Negotiation Program. J&J states this would ensure that the claims are actually acquired and dispensed by a covered 340B entity, as well as ensuring there are no duplicate discounts as statutorily required by the IRA. When initially proposed, HRSA threatened to remove J&J’s access to Medicare and Medicaid if it pursued this change. J&J’s suit challenges that decision.
However, seven states (Arkansas, Kansas, Louisiana, Minnesota, Missouri, Mississippi, and West Virginia) have been active on this issue, passing laws to prevent manufacturers from limiting contract pharmacies’ ability to acquire 340B-discounted drugs. The model legislation also bans restrictions on the “number, location, ownership, or type of 340B contract pharmacy.”
It should also be noted that there are states that are looking for ways to encourage certain independent private practice specialties (such as gastroenterology and rheumatology) to see Medicaid patients, as well as increase testing for sexually transmitted diseases, by offering the possibility of obtaining 340B pricing in their clinics.
Shifting our focus to Congress, six bipartisan Senators, known as the Group of 6, are working to modernize the 340B program, which hasn’t been updated since the original law in 1992. In 2024, legislation was introduced (see here and here) to reform a number of the features of the 340B drug discount program, including transparency, contract pharmacy requirements, and federal agency oversight.
Who’s Guarding the Hen House?
The Government Accountability Office and the Office of Inspector General over the last 5-10 years have asked HRSA to better define an “eligible” patient, to have more specifics concerning hospital eligibility criteria, and to have better oversight of the program to avoid duplicate discounts. HRSA has said that it doesn’t have the ability or the funding to achieve some of these goals. Consequently, little has been done on any of these fronts, creating frustration among pharmaceutical manufacturers and those calling for more oversight of the program to ensure that eligible patients are receiving the benefit of 340B pricing. Again, these frustrations are not pointed at the initial federally qualified centers or “grantees.”
HRSA now audits 200 covered entities a year, which is less than 2% of entities participating in the 340B program. HRSA expects the 340B entities themselves to have an oversight committee in place to ensure compliance with program requirements.
So essentially, the fox is guarding the hen house?
Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at rhnews@mdedge.com.
The 340B Drug Pricing Program began as a noble endeavor, a lifeline designed to help safety-net providers deliver affordable care to America’s most vulnerable populations. However, over the years, this well-intentioned program has strayed from its original purpose, becoming a lucrative space where profits often outweigh patients. Loopholes, lax oversight, and unchecked expansion have allowed some powerful players, such as certain disproportionate share hospitals and their “child sites” as well as for-profit pharmacies, to exploit the system. What was once a program to uplift underserved communities now risks becoming a case study in how good intentions can go astray without accountability.
What exactly is this “340B program” that has captured headlines and the interest of legislatures around the country? What ensures that pharmaceutical manufacturers continue to participate in this program? How lucrative is it? How have underserved populations benefited and how is that measured?
The 340B Drug Pricing Program was established in 1992 under the Public Health Service Act. Its primary goal is to enable covered entities (such as hospitals and clinics serving low-income and uninsured patients) to purchase outpatient drugs from pharmaceutical manufacturers at significantly reduced prices in order to support their care of the low-income and underserved populations. Drug makers are required to participate in this program as a condition of their participation in Medicaid and Medicare Part B and offer these steep discounts to covered entities if they want their medications to be available to 38% of patients nationwide.
The hospitals that make up 78% of the program’s spending are known as disproportionate share hospitals (DSHs). These hospitals must be nonprofit and have at least an 11.75% “disproportionate” share of low-income Medicare or Medicaid inpatients. The other types of non-hospital entities qualifying for 340B pricing are known as initial “federal grantees.” Some examples include federally qualified health centers (FQHC), Ryan White HIV/AIDS program grantees, and other types of specialized clinics, such as hemophilia treatment centers. It needs to be noted up front that it is not these initial non-hospital federal grantees that need more oversight or reform, since according to the Health Resources and Services Administration (HRSA) 2023 report they make up only 22% of all program spending. It is the large, predominantly DSH health systems that are profiting immensely through exponential growth of their clinics and contract pharmacies. However, these health systems have not been able to show exactly who are their eligible patients and how they have been benefiting them.
When the 340B program was established to offer financial relief to hospitals and clinics taking care of the uninsured, it allowed them to save 20%-50% on drug purchases, which could be reinvested in patient care services. It was hoped that savings from the program could be used to provide free or low-cost medications, free vaccines, and other essential health services, essentially allowing safety-net providers to serve their communities despite financial constraints. The initial grantees are fulfilling that mission, but there are concerns regarding DSHs. (See the Coalition of State Rheumatology Organization’s 340B explanatory statement and policy position for more.)
Why Should Independent Practice Physicians Care About This?
Independent doctors should care about the lack of oversight in the 340B program because it affects healthcare costs, patient assistance, market competition, and access to affordable care for underserved and uninsured patients.
It also plays a strong hand in the healthcare consolidation that continues to threaten private physician practices. These acquisitions threaten the viability of independent practices in a variety of specialties across the United States, including rheumatology. HRSA allows 340B-covered entities to register their off-campus outpatient facilities, or child sites, under their 340B designation. Covered entities can acquire drugs at the 340B price, while imposing markups on the reimbursement they submit to private insurance. The additional revenue these covered entities can pocket provides them with a cash flow advantage that physician practices and outpatient clinics will never be able to actualize. This uneven playing field may make rheumatology practices more susceptible to hospital acquisitions. In fact, between 2016 and 2022, large 340B hospitals were responsible for approximately 80% of hospital acquisitions.
Perhaps the most important reason that we should all be concerned about the trajectory of this well-meaning program is that we have seen patients with hospital debt being sued by DSHs who receive 340B discounts so that they can take care of the low-income patients they are suing. We have seen Medicaid patients be turned away from a DSH clinic after being discharged from that hospital, because the hospital had reached its disproportionate share (11.75%) of inpatient Medicare and Medicaid patients. While not illegal, that type of behavior by covered entities is WRONG! Oversight and reform are needed if the 340B program is going to live up to its purpose and not be just another well-intentioned program not fulfilling its mission.
Areas of Concern
There has been controversy regarding the limited oversight of the 340B program by HRSA, leading to abuse of the program. There are deep concerns regarding a lack of transparency in how savings from the program are being used, and there are concerns about the challenges associated with accurate tracking and reporting of 340B discounts, possibly leading to the duplication of discounts for both Medicaid and 340B. For example, a “duplicate discount” occurs if a manufacturer sells medications to a DSH at the 340B price and later pays a Medicaid rebate on the same drug. The extent of duplicate discounts in the 340B program is unknown. However, an audit of 1,536 cases conducted by HRSA between 2012 and 2019 found 429 instances of noncompliance related to duplicate discounts, which is nearly 30% of cases.
DSHs and their contracted pharmacies have been accused of exploiting the program by increasing the number of contract pharmacies and expanding the number of offsite outpatient clinics to maximize profits. As of mid-2024, the number of 340B contract pharmacies, counted by Drug Channels Institute (DCI), numbered 32,883 unique locations. According to DCI, the top five pharmacies in the program happen also to be among the top pharmacy revenue generators and are “for-profit.” They are CVS, Walgreens, Walmart, Express Scripts, and Optum RX. Additionally, a study in JAMA Health Forum showed that, from 2011 to 2019, contract pharmacies in areas with the lowest income decreased by 5.6% while those in the most affluent neighborhoods grew by 5%.
There also has been tremendous growth in the number of covered entities in the 340B program, which grew from just over 8,100 in 2000 to 50,000 in 2020. Before 2004, DSHs made up less than 10% of these entities, but by 2020, they accounted for over 60%. Another study shows that DSHs are expanding their offsite outpatient clinics (“child clinics”) into the affluent neighborhoods serving commercially insured patients who are not low income, to capture the high commercial reimbursements for medications they acquired at steeply discounted prices. This clearly is diverting care away from the intended beneficiaries of the 340B program.
Furthermore, DSHs have been acquiring specialty practices that prescribe some of the most expensive drugs, in order to take advantage of commercial reimbursement for medications that were acquired at the 340B discount price. Independent oncology practices have complained specifically about this happening in their area, where in some cases the DSHs have “stolen” their patients to profit off of the 340B pricing margins. This has the unintended consequence of increasing government spending, according to a study in the New England Journal of Medicine that showed price markups at 340B eligible hospitals were 6.59 times as high as those in independent physician practices after accounting for drug, patient, and geographic factors.
Legal Challenges and Legislation
On May 21, 2024, the US Court of Appeals for the DC Circuit issued a unanimous decision in favor of drug manufacturers, finding that certain manufacturer restrictions on the use of contract pharmacies under the 340B drug pricing program are permissible. The court’s decision follows a lower court (3rd Circuit) ruling which concluded that the 340B statute does not require manufacturers to deliver 340B drugs to an “unlimited number of contract pharmacies.” We’re still awaiting a decision from the 7th Circuit Court on a similar issue. If the 7th Circuit agrees with the government, creating a split decision, there is an increase in the likelihood that the Supreme Court would take up the case.
Johnson & Johnson has also sued the federal government for blocking their proposed use of a rebate model for DSHs that purchase through 340B two of its medications, Stelara and Xarelto, whose maximum fair price was negotiated through the Inflation Reduction Act’s Medicare Drug Price Negotiation Program. J&J states this would ensure that the claims are actually acquired and dispensed by a covered 340B entity, as well as ensuring there are no duplicate discounts as statutorily required by the IRA. When initially proposed, HRSA threatened to remove J&J’s access to Medicare and Medicaid if it pursued this change. J&J’s suit challenges that decision.
However, seven states (Arkansas, Kansas, Louisiana, Minnesota, Missouri, Mississippi, and West Virginia) have been active on this issue, passing laws to prevent manufacturers from limiting contract pharmacies’ ability to acquire 340B-discounted drugs. The model legislation also bans restrictions on the “number, location, ownership, or type of 340B contract pharmacy.”
It should also be noted that there are states that are looking for ways to encourage certain independent private practice specialties (such as gastroenterology and rheumatology) to see Medicaid patients, as well as increase testing for sexually transmitted diseases, by offering the possibility of obtaining 340B pricing in their clinics.
Shifting our focus to Congress, six bipartisan Senators, known as the Group of 6, are working to modernize the 340B program, which hasn’t been updated since the original law in 1992. In 2024, legislation was introduced (see here and here) to reform a number of the features of the 340B drug discount program, including transparency, contract pharmacy requirements, and federal agency oversight.
Who’s Guarding the Hen House?
The Government Accountability Office and the Office of Inspector General over the last 5-10 years have asked HRSA to better define an “eligible” patient, to have more specifics concerning hospital eligibility criteria, and to have better oversight of the program to avoid duplicate discounts. HRSA has said that it doesn’t have the ability or the funding to achieve some of these goals. Consequently, little has been done on any of these fronts, creating frustration among pharmaceutical manufacturers and those calling for more oversight of the program to ensure that eligible patients are receiving the benefit of 340B pricing. Again, these frustrations are not pointed at the initial federally qualified centers or “grantees.”
HRSA now audits 200 covered entities a year, which is less than 2% of entities participating in the 340B program. HRSA expects the 340B entities themselves to have an oversight committee in place to ensure compliance with program requirements.
So essentially, the fox is guarding the hen house?
Dr. Feldman is a rheumatologist in private practice with The Rheumatology Group in New Orleans. She is the CSRO’s vice president of advocacy and government affairs and its immediate past president, as well as past chair of the Alliance for Safe Biologic Medicines and a past member of the American College of Rheumatology insurance subcommittee. You can reach her at rhnews@mdedge.com.
Promising New Data for Drugs Both Novel and Established in Several Common GI Conditions
I’ve just returned from the annual meeting of the American College of Gastroenterology (ACG), which was held in Philadelphia, Pennsylvania.
which will be split into two parts. These studies are not presented in any order of priority because I think they all have clinical applications, some of which are more immediate. Where possible, I’ll also provide you with some teaching points that occurred to me when I viewed the data in these presentations.
Repetitive Use Injuries Among Endoscopists
First is a study that assessed musculoskeletal injuries that occurred while performing endoscopy, which is a large part of what we do as gastroenterologists. I’ve personally experienced virtually all these injuries during my 45-plus years in the field. These findings provide a useful perspective of what we can do to potentially avoid such injuries going forward.
This study comes to us from researchers at the University of Utah, who looked at an author-developed and validated questionnaire called QuickDash (Disability of Arm, Shoulder, Hand). Endoscopists were evaluated by occupational therapists in their department, who looked at strength testing as well as a series of provocative tests to identify injuries.
Thirty-five endoscopists were enrolled. Overall, 34% reported experiencing pain and 17% reported numbness. In the previous week, 48% had been bothered by pain and 11% by tingling, and 17% reported limitations on what they could do at work.
Additionally, 17% experienced interrupted sleep. I think that finding is particularly important, because when you have fragmented sleep it lowers sensory thresholds. Essentially, this means that the day after interrupted sleep, you respond with a heightened sensory response.
Physical testing showed reduction in grip strength in approximately half of participants, including both right and left grip. More than 70% had at least one abnormal positive provocative test.
There were a couple factors associated with an increased risk for adverse outcomes. Those who performed 20 or more procedures a week were at higher risk of pain (P = .007). I recognize that some of you probably perform 20 or more endoscopies in a single day.
Negative outcomes were also more likely to occur among physicians performing biliary endoscopy. Performing endoscopic retrograde cholangiopancreatography during 20%-60% of the week resulted in greater likelihood of experiencing decreased bilateral pinch strength.
The bottom line is that there’s an extremely high prevalence of repetitive use injury among endoscopists. Whether you’re just starting out, mid-career, or more senior, you need to be aware of this.
Kudos to the ACG who put together hands-on sessions across 2 days with extended ergonomics training provided by physical therapists with expertise in this area. These sessions were standing room only every time I visited them.
During the meeting, the ACG also announced the publication of a new book, Ergonomics for Endoscopy: Optimal Preparation, Performance, and Recovery, that is available on the ACG’s website. I had the privilege to meet the two authors, survey the book, and briefly discuss it with them. It’s phenomenal, and something that I think every endoscopist can benefit from.
So, in summary, don’t put aside these concerns. It’s your career, and the better we can do in preventing these injuries, I think the better you’ll feel going forward.
On-Demand Vonoprazan for Heartburn
The second study of note dealt with vonoprazan, a potassium-competitive acid blocker.
The question surrounding vonoprazan is whether it has clinical value as an on-demand option rather than simply as maintenance therapy administered via daily dosing. Previous results have suggested that it may have a more rapid effect when it’s taken on demand.
This post hoc analysis of a randomized trial evaluated a 4-week run-in period during which patients received vonoprazan 20 mg daily, followed by a 6-week period after which patients switched to on-demand therapy. To be eligible, during the run-in period patients had to be 80% compliant with the study drug and report no heartburn during the last 7 days of treatment. If so, they were then randomized to receive vonoprazan at 10 mg, 20 mg, or 40 mg, or placebo.
Eligible patients reported 16% heartburn-free days during the screening period. During the run-in period, heartburn-free days increased to 83% among those taking vonoprazan 20 mg daily. When this was stopped and patients transitioned to on-demand vonoprazan, they still had a very high rate of heartburn-free days, ranging from 71% to 75%. Over 90% in the treatment group had their symptoms improved within 2 hours, with improvement noted as early as within 1 hour.
We know that proton pump inhibitors don’t produce this effect, which presents a challenge for us. This study suggests there may be a very strong role for on-demand therapy in patients who have reflux, which certainly showed in terms of responsiveness during the 4-week run-in period.
Rifaximin Monotherapy Reduces Risk for Overt Hepatic Encephalopathy Recurrence
The next trial looked at rifaximin monotherapy for prevention of relapse of overt hepatic encephalopathy.
Rifaximin has been added to the baseline primary treatment of lactulose, a combination that has been analyzed in pivotal studies. However, lactulose is a difficult drug to titrate. We typically ask patients to let us know if they have Bristol Stool Scale scores ≥ 6. This results in an inordinate number of calls back to the clinic or office. Diarrhea is a particular problem in these patients.
This study, which was presented by Dr Jasmohan S. Bajaj from Virginia Commonwealth University, analyzed data from two randomized trials: a phase 3, double-blind trial and a phase 4 open-label trial. Both studies were conducted in patients with Child-Pugh classification A and B cirrhosis, whose overt hepatic encephalopathy was graded with a Conn score ≤ 1. Researchers only looked at those patients who received rifaximin 550 mg twice daily without lactulose, or lactulose titrated to a target of two to three soft stools a day plus placebo. The primary endpoint, which was used in both trials, was time to first breakthrough overt hepatic encephalopathy episode, measured as a Conn score ≥ 2.
There were 125 patients in the rifaximin group and 145 patients in the lactulose group. Patients in both groups had mean age of 57 years and a median Model for End-Stage Liver Disease score of 12.
Treatment with rifaximin produced significantly striking results. In the rifaximin group, the risk for breakthrough overt hepatic encephalopathy episodes was reduced by 60%, with a number needed to treat of 4, which is extremely powerful. Regarding mortality reduction, the number needed to treat with rifaximin alone was 19.
The take-away message here is that it’s difficult to use lactulose. We frequently have to stop it. These results provide reassurance that rifaximin has a dramatic effect even when used by itself. The recommended first-line therapy is still to begin with lactulose, but this study provides us with very strong data regarding the use of rifaximin alone.
Apraglutide’s Efficacy in Short-Bowel Syndrome
The fourth study I’d like to highlight adds to the growing data around a long-acting glucagon-like peptide 2 analog, apraglutide.
Results from the phase 3, double-blind STARS trial were first presented at Digestive Disease Week earlier in the year. They showed that apraglutide had efficacy and contributed to a reduction in the risk for needing parenteral support, maintenance of weight, and fluid requirements in patients with short-bowel syndrome and intestinal failure. However, questions remained regarding whether there were variations in response based on patient demographics and short-bowel syndrome–specific characteristics.
Researchers looked at a subgroup of patients from the STARS trial. The primary endpoint remained the same as in the main trial: relative change from baseline in actual parenteral support weekly volume at week 24. However, rather than measure it in the overall population, they did so according to geographic region, gender, age, body weight, race and ethnicity, and short-bowel syndrome characteristics, including differences in parenteral support volume, length of the remnant small intestine, and time from short-bowel syndrome diagnosis.
Across all these demographic and disease characteristic categories, there was absolutely no difference in the primary endpoint.
Apraglutide seems to be inordinately promising. This is a once-weekly treatment, as opposed to liraglutide, which is problematic because it has to be given daily.
From what I understand, apraglutide has been offered a fast-track status by the US Food and Drug Administration. Again, from what I’ve heard, it will be evaluated upon submission beginning sometime in the first quarter of 2025, which may mean that apraglutide could be available as early as 2026.
This would be a big deal for patients with short-bowel syndrome who would have a once-weekly treatment option as opposed to daily treatment and its accompanying problems of compliance and relatively reduced response.
Biologics for Treating Immune Checkpoint Inhibitor Colitis
The final study I want to discuss in this presentation dealt with adverse responses to immune checkpoint inhibitor (ICI) therapy. We see this increasingly in our clinics as ICIs become more widely used. They are wonderful drugs, but ICI-induced colitis can occur in up to 30% of patients.
The oncologic approach is to try and stay the course, while gastroenterologists are tasked with treating the colitis so that these patients can maintain their cancer treatment. Steroid therapy is the primary first-line treatment against ICI colitis, but the use of biologic therapy with infliximab or vedolizumab has been associated with favorable outcomes as well.
ICI colitis is graded on a scale. Whereas grade 1 ICI colitis indicates increased stool frequency of less than four a day and the absence of symptoms, grade 2 indicates a progression to a stool frequency of four to six times a day, the appearance of blood or mucus in the stool, and symptoms like abdominal pain.
Researchers sought to answer the question of which treatment is better for patients with moderate to severe ICI colitis: infliximab or vedolizumab? They performed a database analysis of patients at their institution who received at least one dose of these biologics. The endpoint was sustained clinical response (ie, without a recurrent colitis episode), as well as patients achieving improvement to grade 1 ICI colitis.
The data for infliximab and vedolizumab were quite good. Sustained clinical response was noted in 91% of patients receiving infliximab and 86% receiving vedolizumab. There was no difference in infection risk between the groups.
There’s a teaching point in the study’s other key finding, which is that following biologic initiation, steroids were more rapidly discontinued with vedolizumab vs infliximab (median, 25 days vs 56 days, respectively). Therefore, vedolizumab may have the added benefit of patients being able to get off steroids more quickly. That’s the take-home message: Vedolizumab may be better. We certainly are comfortable with both biologics, but patients getting off steroids would be better.
There are two additional teaching points I’d like to convey.
First, don’t forget to perform a biopsy, because there are patients who may have cytomegalovirus colitis and we don’t want to miss it. A biopsy may also reveal whether they have a macroscopically normal rectosigmoid. So, you should biopsy to look for microscopic changes. As 98% of ICI colitis cases involve the left colon, you can get by with just using a flexible sigmoidoscopy.
Second, don’t forget to check for celiac disease. Patients taking ICIs may develop celiac disease as a side effect of treatment. So, I always order a celiac profile as well.
These are, in my opinion, five of the top studies from ACG 2024, with the remaining studies to be discussed in my next video. They all provide opportunities to help us improve our patients’ health.
Dr. Johnson is professor of medicine and chief of gastroenterology at Eastern Virginia Medical School in Norfolk, Virginia, and a past president of the American College of Gastroenterology. He reported serving in an advisory position with ISOTHRIVE. This transcript has been edited for clarity.
A version of this article appeared on Medscape.com.
I’ve just returned from the annual meeting of the American College of Gastroenterology (ACG), which was held in Philadelphia, Pennsylvania.
which will be split into two parts. These studies are not presented in any order of priority because I think they all have clinical applications, some of which are more immediate. Where possible, I’ll also provide you with some teaching points that occurred to me when I viewed the data in these presentations.
Repetitive Use Injuries Among Endoscopists
First is a study that assessed musculoskeletal injuries that occurred while performing endoscopy, which is a large part of what we do as gastroenterologists. I’ve personally experienced virtually all these injuries during my 45-plus years in the field. These findings provide a useful perspective of what we can do to potentially avoid such injuries going forward.
This study comes to us from researchers at the University of Utah, who looked at an author-developed and validated questionnaire called QuickDash (Disability of Arm, Shoulder, Hand). Endoscopists were evaluated by occupational therapists in their department, who looked at strength testing as well as a series of provocative tests to identify injuries.
Thirty-five endoscopists were enrolled. Overall, 34% reported experiencing pain and 17% reported numbness. In the previous week, 48% had been bothered by pain and 11% by tingling, and 17% reported limitations on what they could do at work.
Additionally, 17% experienced interrupted sleep. I think that finding is particularly important, because when you have fragmented sleep it lowers sensory thresholds. Essentially, this means that the day after interrupted sleep, you respond with a heightened sensory response.
Physical testing showed reduction in grip strength in approximately half of participants, including both right and left grip. More than 70% had at least one abnormal positive provocative test.
There were a couple factors associated with an increased risk for adverse outcomes. Those who performed 20 or more procedures a week were at higher risk of pain (P = .007). I recognize that some of you probably perform 20 or more endoscopies in a single day.
Negative outcomes were also more likely to occur among physicians performing biliary endoscopy. Performing endoscopic retrograde cholangiopancreatography during 20%-60% of the week resulted in greater likelihood of experiencing decreased bilateral pinch strength.
The bottom line is that there’s an extremely high prevalence of repetitive use injury among endoscopists. Whether you’re just starting out, mid-career, or more senior, you need to be aware of this.
Kudos to the ACG who put together hands-on sessions across 2 days with extended ergonomics training provided by physical therapists with expertise in this area. These sessions were standing room only every time I visited them.
During the meeting, the ACG also announced the publication of a new book, Ergonomics for Endoscopy: Optimal Preparation, Performance, and Recovery, that is available on the ACG’s website. I had the privilege to meet the two authors, survey the book, and briefly discuss it with them. It’s phenomenal, and something that I think every endoscopist can benefit from.
So, in summary, don’t put aside these concerns. It’s your career, and the better we can do in preventing these injuries, I think the better you’ll feel going forward.
On-Demand Vonoprazan for Heartburn
The second study of note dealt with vonoprazan, a potassium-competitive acid blocker.
The question surrounding vonoprazan is whether it has clinical value as an on-demand option rather than simply as maintenance therapy administered via daily dosing. Previous results have suggested that it may have a more rapid effect when it’s taken on demand.
This post hoc analysis of a randomized trial evaluated a 4-week run-in period during which patients received vonoprazan 20 mg daily, followed by a 6-week period after which patients switched to on-demand therapy. To be eligible, during the run-in period patients had to be 80% compliant with the study drug and report no heartburn during the last 7 days of treatment. If so, they were then randomized to receive vonoprazan at 10 mg, 20 mg, or 40 mg, or placebo.
Eligible patients reported 16% heartburn-free days during the screening period. During the run-in period, heartburn-free days increased to 83% among those taking vonoprazan 20 mg daily. When this was stopped and patients transitioned to on-demand vonoprazan, they still had a very high rate of heartburn-free days, ranging from 71% to 75%. Over 90% in the treatment group had their symptoms improved within 2 hours, with improvement noted as early as within 1 hour.
We know that proton pump inhibitors don’t produce this effect, which presents a challenge for us. This study suggests there may be a very strong role for on-demand therapy in patients who have reflux, which certainly showed in terms of responsiveness during the 4-week run-in period.
Rifaximin Monotherapy Reduces Risk for Overt Hepatic Encephalopathy Recurrence
The next trial looked at rifaximin monotherapy for prevention of relapse of overt hepatic encephalopathy.
Rifaximin has been added to the baseline primary treatment of lactulose, a combination that has been analyzed in pivotal studies. However, lactulose is a difficult drug to titrate. We typically ask patients to let us know if they have Bristol Stool Scale scores ≥ 6. This results in an inordinate number of calls back to the clinic or office. Diarrhea is a particular problem in these patients.
This study, which was presented by Dr Jasmohan S. Bajaj from Virginia Commonwealth University, analyzed data from two randomized trials: a phase 3, double-blind trial and a phase 4 open-label trial. Both studies were conducted in patients with Child-Pugh classification A and B cirrhosis, whose overt hepatic encephalopathy was graded with a Conn score ≤ 1. Researchers only looked at those patients who received rifaximin 550 mg twice daily without lactulose, or lactulose titrated to a target of two to three soft stools a day plus placebo. The primary endpoint, which was used in both trials, was time to first breakthrough overt hepatic encephalopathy episode, measured as a Conn score ≥ 2.
There were 125 patients in the rifaximin group and 145 patients in the lactulose group. Patients in both groups had mean age of 57 years and a median Model for End-Stage Liver Disease score of 12.
Treatment with rifaximin produced significantly striking results. In the rifaximin group, the risk for breakthrough overt hepatic encephalopathy episodes was reduced by 60%, with a number needed to treat of 4, which is extremely powerful. Regarding mortality reduction, the number needed to treat with rifaximin alone was 19.
The take-away message here is that it’s difficult to use lactulose. We frequently have to stop it. These results provide reassurance that rifaximin has a dramatic effect even when used by itself. The recommended first-line therapy is still to begin with lactulose, but this study provides us with very strong data regarding the use of rifaximin alone.
Apraglutide’s Efficacy in Short-Bowel Syndrome
The fourth study I’d like to highlight adds to the growing data around a long-acting glucagon-like peptide 2 analog, apraglutide.
Results from the phase 3, double-blind STARS trial were first presented at Digestive Disease Week earlier in the year. They showed that apraglutide had efficacy and contributed to a reduction in the risk for needing parenteral support, maintenance of weight, and fluid requirements in patients with short-bowel syndrome and intestinal failure. However, questions remained regarding whether there were variations in response based on patient demographics and short-bowel syndrome–specific characteristics.
Researchers looked at a subgroup of patients from the STARS trial. The primary endpoint remained the same as in the main trial: relative change from baseline in actual parenteral support weekly volume at week 24. However, rather than measure it in the overall population, they did so according to geographic region, gender, age, body weight, race and ethnicity, and short-bowel syndrome characteristics, including differences in parenteral support volume, length of the remnant small intestine, and time from short-bowel syndrome diagnosis.
Across all these demographic and disease characteristic categories, there was absolutely no difference in the primary endpoint.
Apraglutide seems to be inordinately promising. This is a once-weekly treatment, as opposed to liraglutide, which is problematic because it has to be given daily.
From what I understand, apraglutide has been offered a fast-track status by the US Food and Drug Administration. Again, from what I’ve heard, it will be evaluated upon submission beginning sometime in the first quarter of 2025, which may mean that apraglutide could be available as early as 2026.
This would be a big deal for patients with short-bowel syndrome who would have a once-weekly treatment option as opposed to daily treatment and its accompanying problems of compliance and relatively reduced response.
Biologics for Treating Immune Checkpoint Inhibitor Colitis
The final study I want to discuss in this presentation dealt with adverse responses to immune checkpoint inhibitor (ICI) therapy. We see this increasingly in our clinics as ICIs become more widely used. They are wonderful drugs, but ICI-induced colitis can occur in up to 30% of patients.
The oncologic approach is to try and stay the course, while gastroenterologists are tasked with treating the colitis so that these patients can maintain their cancer treatment. Steroid therapy is the primary first-line treatment against ICI colitis, but the use of biologic therapy with infliximab or vedolizumab has been associated with favorable outcomes as well.
ICI colitis is graded on a scale. Whereas grade 1 ICI colitis indicates increased stool frequency of less than four a day and the absence of symptoms, grade 2 indicates a progression to a stool frequency of four to six times a day, the appearance of blood or mucus in the stool, and symptoms like abdominal pain.
Researchers sought to answer the question of which treatment is better for patients with moderate to severe ICI colitis: infliximab or vedolizumab? They performed a database analysis of patients at their institution who received at least one dose of these biologics. The endpoint was sustained clinical response (ie, without a recurrent colitis episode), as well as patients achieving improvement to grade 1 ICI colitis.
The data for infliximab and vedolizumab were quite good. Sustained clinical response was noted in 91% of patients receiving infliximab and 86% receiving vedolizumab. There was no difference in infection risk between the groups.
There’s a teaching point in the study’s other key finding, which is that following biologic initiation, steroids were more rapidly discontinued with vedolizumab vs infliximab (median, 25 days vs 56 days, respectively). Therefore, vedolizumab may have the added benefit of patients being able to get off steroids more quickly. That’s the take-home message: Vedolizumab may be better. We certainly are comfortable with both biologics, but patients getting off steroids would be better.
There are two additional teaching points I’d like to convey.
First, don’t forget to perform a biopsy, because there are patients who may have cytomegalovirus colitis and we don’t want to miss it. A biopsy may also reveal whether they have a macroscopically normal rectosigmoid. So, you should biopsy to look for microscopic changes. As 98% of ICI colitis cases involve the left colon, you can get by with just using a flexible sigmoidoscopy.
Second, don’t forget to check for celiac disease. Patients taking ICIs may develop celiac disease as a side effect of treatment. So, I always order a celiac profile as well.
These are, in my opinion, five of the top studies from ACG 2024, with the remaining studies to be discussed in my next video. They all provide opportunities to help us improve our patients’ health.
Dr. Johnson is professor of medicine and chief of gastroenterology at Eastern Virginia Medical School in Norfolk, Virginia, and a past president of the American College of Gastroenterology. He reported serving in an advisory position with ISOTHRIVE. This transcript has been edited for clarity.
A version of this article appeared on Medscape.com.
I’ve just returned from the annual meeting of the American College of Gastroenterology (ACG), which was held in Philadelphia, Pennsylvania.
which will be split into two parts. These studies are not presented in any order of priority because I think they all have clinical applications, some of which are more immediate. Where possible, I’ll also provide you with some teaching points that occurred to me when I viewed the data in these presentations.
Repetitive Use Injuries Among Endoscopists
First is a study that assessed musculoskeletal injuries that occurred while performing endoscopy, which is a large part of what we do as gastroenterologists. I’ve personally experienced virtually all these injuries during my 45-plus years in the field. These findings provide a useful perspective of what we can do to potentially avoid such injuries going forward.
This study comes to us from researchers at the University of Utah, who looked at an author-developed and validated questionnaire called QuickDash (Disability of Arm, Shoulder, Hand). Endoscopists were evaluated by occupational therapists in their department, who looked at strength testing as well as a series of provocative tests to identify injuries.
Thirty-five endoscopists were enrolled. Overall, 34% reported experiencing pain and 17% reported numbness. In the previous week, 48% had been bothered by pain and 11% by tingling, and 17% reported limitations on what they could do at work.
Additionally, 17% experienced interrupted sleep. I think that finding is particularly important, because when you have fragmented sleep it lowers sensory thresholds. Essentially, this means that the day after interrupted sleep, you respond with a heightened sensory response.
Physical testing showed reduction in grip strength in approximately half of participants, including both right and left grip. More than 70% had at least one abnormal positive provocative test.
There were a couple factors associated with an increased risk for adverse outcomes. Those who performed 20 or more procedures a week were at higher risk of pain (P = .007). I recognize that some of you probably perform 20 or more endoscopies in a single day.
Negative outcomes were also more likely to occur among physicians performing biliary endoscopy. Performing endoscopic retrograde cholangiopancreatography during 20%-60% of the week resulted in greater likelihood of experiencing decreased bilateral pinch strength.
The bottom line is that there’s an extremely high prevalence of repetitive use injury among endoscopists. Whether you’re just starting out, mid-career, or more senior, you need to be aware of this.
Kudos to the ACG who put together hands-on sessions across 2 days with extended ergonomics training provided by physical therapists with expertise in this area. These sessions were standing room only every time I visited them.
During the meeting, the ACG also announced the publication of a new book, Ergonomics for Endoscopy: Optimal Preparation, Performance, and Recovery, that is available on the ACG’s website. I had the privilege to meet the two authors, survey the book, and briefly discuss it with them. It’s phenomenal, and something that I think every endoscopist can benefit from.
So, in summary, don’t put aside these concerns. It’s your career, and the better we can do in preventing these injuries, I think the better you’ll feel going forward.
On-Demand Vonoprazan for Heartburn
The second study of note dealt with vonoprazan, a potassium-competitive acid blocker.
The question surrounding vonoprazan is whether it has clinical value as an on-demand option rather than simply as maintenance therapy administered via daily dosing. Previous results have suggested that it may have a more rapid effect when it’s taken on demand.
This post hoc analysis of a randomized trial evaluated a 4-week run-in period during which patients received vonoprazan 20 mg daily, followed by a 6-week period after which patients switched to on-demand therapy. To be eligible, during the run-in period patients had to be 80% compliant with the study drug and report no heartburn during the last 7 days of treatment. If so, they were then randomized to receive vonoprazan at 10 mg, 20 mg, or 40 mg, or placebo.
Eligible patients reported 16% heartburn-free days during the screening period. During the run-in period, heartburn-free days increased to 83% among those taking vonoprazan 20 mg daily. When this was stopped and patients transitioned to on-demand vonoprazan, they still had a very high rate of heartburn-free days, ranging from 71% to 75%. Over 90% in the treatment group had their symptoms improved within 2 hours, with improvement noted as early as within 1 hour.
We know that proton pump inhibitors don’t produce this effect, which presents a challenge for us. This study suggests there may be a very strong role for on-demand therapy in patients who have reflux, which certainly showed in terms of responsiveness during the 4-week run-in period.
Rifaximin Monotherapy Reduces Risk for Overt Hepatic Encephalopathy Recurrence
The next trial looked at rifaximin monotherapy for prevention of relapse of overt hepatic encephalopathy.
Rifaximin has been added to the baseline primary treatment of lactulose, a combination that has been analyzed in pivotal studies. However, lactulose is a difficult drug to titrate. We typically ask patients to let us know if they have Bristol Stool Scale scores ≥ 6. This results in an inordinate number of calls back to the clinic or office. Diarrhea is a particular problem in these patients.
This study, which was presented by Dr Jasmohan S. Bajaj from Virginia Commonwealth University, analyzed data from two randomized trials: a phase 3, double-blind trial and a phase 4 open-label trial. Both studies were conducted in patients with Child-Pugh classification A and B cirrhosis, whose overt hepatic encephalopathy was graded with a Conn score ≤ 1. Researchers only looked at those patients who received rifaximin 550 mg twice daily without lactulose, or lactulose titrated to a target of two to three soft stools a day plus placebo. The primary endpoint, which was used in both trials, was time to first breakthrough overt hepatic encephalopathy episode, measured as a Conn score ≥ 2.
There were 125 patients in the rifaximin group and 145 patients in the lactulose group. Patients in both groups had mean age of 57 years and a median Model for End-Stage Liver Disease score of 12.
Treatment with rifaximin produced significantly striking results. In the rifaximin group, the risk for breakthrough overt hepatic encephalopathy episodes was reduced by 60%, with a number needed to treat of 4, which is extremely powerful. Regarding mortality reduction, the number needed to treat with rifaximin alone was 19.
The take-away message here is that it’s difficult to use lactulose. We frequently have to stop it. These results provide reassurance that rifaximin has a dramatic effect even when used by itself. The recommended first-line therapy is still to begin with lactulose, but this study provides us with very strong data regarding the use of rifaximin alone.
Apraglutide’s Efficacy in Short-Bowel Syndrome
The fourth study I’d like to highlight adds to the growing data around a long-acting glucagon-like peptide 2 analog, apraglutide.
Results from the phase 3, double-blind STARS trial were first presented at Digestive Disease Week earlier in the year. They showed that apraglutide had efficacy and contributed to a reduction in the risk for needing parenteral support, maintenance of weight, and fluid requirements in patients with short-bowel syndrome and intestinal failure. However, questions remained regarding whether there were variations in response based on patient demographics and short-bowel syndrome–specific characteristics.
Researchers looked at a subgroup of patients from the STARS trial. The primary endpoint remained the same as in the main trial: relative change from baseline in actual parenteral support weekly volume at week 24. However, rather than measure it in the overall population, they did so according to geographic region, gender, age, body weight, race and ethnicity, and short-bowel syndrome characteristics, including differences in parenteral support volume, length of the remnant small intestine, and time from short-bowel syndrome diagnosis.
Across all these demographic and disease characteristic categories, there was absolutely no difference in the primary endpoint.
Apraglutide seems to be inordinately promising. This is a once-weekly treatment, as opposed to liraglutide, which is problematic because it has to be given daily.
From what I understand, apraglutide has been offered a fast-track status by the US Food and Drug Administration. Again, from what I’ve heard, it will be evaluated upon submission beginning sometime in the first quarter of 2025, which may mean that apraglutide could be available as early as 2026.
This would be a big deal for patients with short-bowel syndrome who would have a once-weekly treatment option as opposed to daily treatment and its accompanying problems of compliance and relatively reduced response.
Biologics for Treating Immune Checkpoint Inhibitor Colitis
The final study I want to discuss in this presentation dealt with adverse responses to immune checkpoint inhibitor (ICI) therapy. We see this increasingly in our clinics as ICIs become more widely used. They are wonderful drugs, but ICI-induced colitis can occur in up to 30% of patients.
The oncologic approach is to try and stay the course, while gastroenterologists are tasked with treating the colitis so that these patients can maintain their cancer treatment. Steroid therapy is the primary first-line treatment against ICI colitis, but the use of biologic therapy with infliximab or vedolizumab has been associated with favorable outcomes as well.
ICI colitis is graded on a scale. Whereas grade 1 ICI colitis indicates increased stool frequency of less than four a day and the absence of symptoms, grade 2 indicates a progression to a stool frequency of four to six times a day, the appearance of blood or mucus in the stool, and symptoms like abdominal pain.
Researchers sought to answer the question of which treatment is better for patients with moderate to severe ICI colitis: infliximab or vedolizumab? They performed a database analysis of patients at their institution who received at least one dose of these biologics. The endpoint was sustained clinical response (ie, without a recurrent colitis episode), as well as patients achieving improvement to grade 1 ICI colitis.
The data for infliximab and vedolizumab were quite good. Sustained clinical response was noted in 91% of patients receiving infliximab and 86% receiving vedolizumab. There was no difference in infection risk between the groups.
There’s a teaching point in the study’s other key finding, which is that following biologic initiation, steroids were more rapidly discontinued with vedolizumab vs infliximab (median, 25 days vs 56 days, respectively). Therefore, vedolizumab may have the added benefit of patients being able to get off steroids more quickly. That’s the take-home message: Vedolizumab may be better. We certainly are comfortable with both biologics, but patients getting off steroids would be better.
There are two additional teaching points I’d like to convey.
First, don’t forget to perform a biopsy, because there are patients who may have cytomegalovirus colitis and we don’t want to miss it. A biopsy may also reveal whether they have a macroscopically normal rectosigmoid. So, you should biopsy to look for microscopic changes. As 98% of ICI colitis cases involve the left colon, you can get by with just using a flexible sigmoidoscopy.
Second, don’t forget to check for celiac disease. Patients taking ICIs may develop celiac disease as a side effect of treatment. So, I always order a celiac profile as well.
These are, in my opinion, five of the top studies from ACG 2024, with the remaining studies to be discussed in my next video. They all provide opportunities to help us improve our patients’ health.
Dr. Johnson is professor of medicine and chief of gastroenterology at Eastern Virginia Medical School in Norfolk, Virginia, and a past president of the American College of Gastroenterology. He reported serving in an advisory position with ISOTHRIVE. This transcript has been edited for clarity.
A version of this article appeared on Medscape.com.
The Most Common Chronic Liver Disease in the World
This transcript has been edited for clarity.
Matthew F. Watto, MD: Welcome back to The Curbsiders. I’m Dr. Matthew Frank Watto, here with my great friend and America’s primary care physician, Dr. Paul Nelson Williams. Paul, what is MASLD?
Paul N. Williams, MD:
Watto: We talked about a really stripped-down way of testing people for MASLD. If we see mildly elevated liver enzymes, what should we be testing, and how does alcohol factor in?
Williams: Before you can make a definitive diagnosis of MASLD, you need to rule out other causes of liver inflammation — things that would cause a patient’s transaminases to increase. Alcohol is synergistic with everything that can harm the liver.
A great place to start is to gauge someone’s alcohol intake to make sure it isn’t causing hepatic inflammation. The phosphatidyl ethanol level is a serologic test to determine chronic, heavy alcohol use. It’s a new kid on the block. I’ve seen it mostly ordered by hepatologists. It is a way of determining whether someone has had fairly consistent alcohol use up to 4 weeks after the fact. The cutoff for a positive test is 20 ng/mL.
Dr Tapper frames the test this way. He isn’t using the test to catch someone in a lie about their alcohol use. He tells patients that he orders this test for all patients with liver inflammation, because alcohol is a common cause. The test helps him better understand the factors that might be affecting the patient’s liver function.
If the test comes back positive, you can have a conversation about that, and if it’s not positive, you move on to the next possible cause. Other fairly common causes of liver inflammation are relatively easy to address.
Watto: Instead of ordering ceruloplasmin or alpha-1 antitrypsin tests, for example, the first thing Dr Tapper recommends is checking for hepatitis B and C. We can cure hepatitis C. We can’t cure hepatitis B, but it’s important to know if the patient has it. Primary care physicians should be comfortable ordering these tests.
Really high ALT levels (eg, in the 200s) don’t usually happen from steatotic liver disease. In those cases, we would send an expanded panel that might include tests for autoimmune hepatitis-ANA, anti–smooth muscle antibody, and IgG levels. Otherwise, most of these patients don’t need much more testing.
What is a FIB4 score and how does that factor in?
Williams: The FIB4 score estimates the degree of fibrosis based on the ALT and AST levels, platelet count, and the patient’s age. These data are plugged into a formula. If the FIB4 score is low (meaning not much fibrosis is present), you can stop there and do your counseling about lifestyle changes and address the reversible factors.
If the FIB4 score is above a certain threshold (1.3 in young adults and 2.0 in older adults), you need to find a more concrete way to determine the degree of fibrosis, typically through imaging.
Elastography can be done either with ultrasound or MRI. Ultrasound is typically ordered, but Dr Tapper recommends doing MRI on patients with a BMI > 40. Those patients are probably better served by doing MRI to determine the degree of liver fibrosis.
Watto: Patients with low FIB4 scores probably don’t need elastography but those with high FIB4 scores do. For the interpretation of ultrasound-based elastography results, Dr Tapper gave us the “rule of 5s”.
Elastography results are reported in kilopascal (kPa) units. A finding of 5 kPa or less is normal. Forty percent of those with a result of 10 kPa might have advanced liver disease. Above 15 kPa, the likelihood of cirrhosis is high, becoming very likely at 25 kPa. Finally, with a result of > 25 kPa, portal hypertension is likely, and you might need to have a conversation about starting the patient on medicine to prevent variceal bleeding.
We are moving toward more noninvasive testing and avoiding biopsies. We have cutoff values for MRI-based elastography as well. Both of these tests can help stage the liver.
What can we tell people about diet?
Williams: Weight loss is helpful. You can reverse fibrosis with weight loss. You can truly help your liver and bring it closer to its healthy baseline with weight loss. A loss of 7.5% body weight can reduce steatohepatitis, and with around 10% of body weight loss, you can actually resolve fibrosis, which is remarkable.
We all know that weight loss can be very therapeutic for many conditions. It’s just very hard to achieve. As primary care doctors, we should use what we have in our armamentarium to achieve that goal. Often, that will include certain medications.
Watto: I like giving patients the 10% number because if they weigh 220 pounds, they need to lose 22 pounds. If they weigh 300 pounds, it’s 30 pounds. Most people who weigh 300 pounds think they need to lose 100 pounds to have any sort of health benefit, but it’s much less than that. So, I do find that helpful.
But now a new drug has been approved. It’s a thyroid memetic called resmetirom. It was from the MAESTRO-NASH trial. Without weight loss, it helped to reverse fibrosis.
This is going to be used more and more in the future. It’s still being worked out exactly where the place is for that drug, so much so that Dr Tapper, as a liver expert, hadn’t even had the chance to prescribe it yet. Of course, it was very recently approved.
Dr. Tapper is one of our most celebrated guests, so check out the full podcast here.
A version of this article appeared on Medscape.com.
This transcript has been edited for clarity.
Matthew F. Watto, MD: Welcome back to The Curbsiders. I’m Dr. Matthew Frank Watto, here with my great friend and America’s primary care physician, Dr. Paul Nelson Williams. Paul, what is MASLD?
Paul N. Williams, MD:
Watto: We talked about a really stripped-down way of testing people for MASLD. If we see mildly elevated liver enzymes, what should we be testing, and how does alcohol factor in?
Williams: Before you can make a definitive diagnosis of MASLD, you need to rule out other causes of liver inflammation — things that would cause a patient’s transaminases to increase. Alcohol is synergistic with everything that can harm the liver.
A great place to start is to gauge someone’s alcohol intake to make sure it isn’t causing hepatic inflammation. The phosphatidyl ethanol level is a serologic test to determine chronic, heavy alcohol use. It’s a new kid on the block. I’ve seen it mostly ordered by hepatologists. It is a way of determining whether someone has had fairly consistent alcohol use up to 4 weeks after the fact. The cutoff for a positive test is 20 ng/mL.
Dr Tapper frames the test this way. He isn’t using the test to catch someone in a lie about their alcohol use. He tells patients that he orders this test for all patients with liver inflammation, because alcohol is a common cause. The test helps him better understand the factors that might be affecting the patient’s liver function.
If the test comes back positive, you can have a conversation about that, and if it’s not positive, you move on to the next possible cause. Other fairly common causes of liver inflammation are relatively easy to address.
Watto: Instead of ordering ceruloplasmin or alpha-1 antitrypsin tests, for example, the first thing Dr Tapper recommends is checking for hepatitis B and C. We can cure hepatitis C. We can’t cure hepatitis B, but it’s important to know if the patient has it. Primary care physicians should be comfortable ordering these tests.
Really high ALT levels (eg, in the 200s) don’t usually happen from steatotic liver disease. In those cases, we would send an expanded panel that might include tests for autoimmune hepatitis-ANA, anti–smooth muscle antibody, and IgG levels. Otherwise, most of these patients don’t need much more testing.
What is a FIB4 score and how does that factor in?
Williams: The FIB4 score estimates the degree of fibrosis based on the ALT and AST levels, platelet count, and the patient’s age. These data are plugged into a formula. If the FIB4 score is low (meaning not much fibrosis is present), you can stop there and do your counseling about lifestyle changes and address the reversible factors.
If the FIB4 score is above a certain threshold (1.3 in young adults and 2.0 in older adults), you need to find a more concrete way to determine the degree of fibrosis, typically through imaging.
Elastography can be done either with ultrasound or MRI. Ultrasound is typically ordered, but Dr Tapper recommends doing MRI on patients with a BMI > 40. Those patients are probably better served by doing MRI to determine the degree of liver fibrosis.
Watto: Patients with low FIB4 scores probably don’t need elastography but those with high FIB4 scores do. For the interpretation of ultrasound-based elastography results, Dr Tapper gave us the “rule of 5s”.
Elastography results are reported in kilopascal (kPa) units. A finding of 5 kPa or less is normal. Forty percent of those with a result of 10 kPa might have advanced liver disease. Above 15 kPa, the likelihood of cirrhosis is high, becoming very likely at 25 kPa. Finally, with a result of > 25 kPa, portal hypertension is likely, and you might need to have a conversation about starting the patient on medicine to prevent variceal bleeding.
We are moving toward more noninvasive testing and avoiding biopsies. We have cutoff values for MRI-based elastography as well. Both of these tests can help stage the liver.
What can we tell people about diet?
Williams: Weight loss is helpful. You can reverse fibrosis with weight loss. You can truly help your liver and bring it closer to its healthy baseline with weight loss. A loss of 7.5% body weight can reduce steatohepatitis, and with around 10% of body weight loss, you can actually resolve fibrosis, which is remarkable.
We all know that weight loss can be very therapeutic for many conditions. It’s just very hard to achieve. As primary care doctors, we should use what we have in our armamentarium to achieve that goal. Often, that will include certain medications.
Watto: I like giving patients the 10% number because if they weigh 220 pounds, they need to lose 22 pounds. If they weigh 300 pounds, it’s 30 pounds. Most people who weigh 300 pounds think they need to lose 100 pounds to have any sort of health benefit, but it’s much less than that. So, I do find that helpful.
But now a new drug has been approved. It’s a thyroid memetic called resmetirom. It was from the MAESTRO-NASH trial. Without weight loss, it helped to reverse fibrosis.
This is going to be used more and more in the future. It’s still being worked out exactly where the place is for that drug, so much so that Dr Tapper, as a liver expert, hadn’t even had the chance to prescribe it yet. Of course, it was very recently approved.
Dr. Tapper is one of our most celebrated guests, so check out the full podcast here.
A version of this article appeared on Medscape.com.
This transcript has been edited for clarity.
Matthew F. Watto, MD: Welcome back to The Curbsiders. I’m Dr. Matthew Frank Watto, here with my great friend and America’s primary care physician, Dr. Paul Nelson Williams. Paul, what is MASLD?
Paul N. Williams, MD:
Watto: We talked about a really stripped-down way of testing people for MASLD. If we see mildly elevated liver enzymes, what should we be testing, and how does alcohol factor in?
Williams: Before you can make a definitive diagnosis of MASLD, you need to rule out other causes of liver inflammation — things that would cause a patient’s transaminases to increase. Alcohol is synergistic with everything that can harm the liver.
A great place to start is to gauge someone’s alcohol intake to make sure it isn’t causing hepatic inflammation. The phosphatidyl ethanol level is a serologic test to determine chronic, heavy alcohol use. It’s a new kid on the block. I’ve seen it mostly ordered by hepatologists. It is a way of determining whether someone has had fairly consistent alcohol use up to 4 weeks after the fact. The cutoff for a positive test is 20 ng/mL.
Dr Tapper frames the test this way. He isn’t using the test to catch someone in a lie about their alcohol use. He tells patients that he orders this test for all patients with liver inflammation, because alcohol is a common cause. The test helps him better understand the factors that might be affecting the patient’s liver function.
If the test comes back positive, you can have a conversation about that, and if it’s not positive, you move on to the next possible cause. Other fairly common causes of liver inflammation are relatively easy to address.
Watto: Instead of ordering ceruloplasmin or alpha-1 antitrypsin tests, for example, the first thing Dr Tapper recommends is checking for hepatitis B and C. We can cure hepatitis C. We can’t cure hepatitis B, but it’s important to know if the patient has it. Primary care physicians should be comfortable ordering these tests.
Really high ALT levels (eg, in the 200s) don’t usually happen from steatotic liver disease. In those cases, we would send an expanded panel that might include tests for autoimmune hepatitis-ANA, anti–smooth muscle antibody, and IgG levels. Otherwise, most of these patients don’t need much more testing.
What is a FIB4 score and how does that factor in?
Williams: The FIB4 score estimates the degree of fibrosis based on the ALT and AST levels, platelet count, and the patient’s age. These data are plugged into a formula. If the FIB4 score is low (meaning not much fibrosis is present), you can stop there and do your counseling about lifestyle changes and address the reversible factors.
If the FIB4 score is above a certain threshold (1.3 in young adults and 2.0 in older adults), you need to find a more concrete way to determine the degree of fibrosis, typically through imaging.
Elastography can be done either with ultrasound or MRI. Ultrasound is typically ordered, but Dr Tapper recommends doing MRI on patients with a BMI > 40. Those patients are probably better served by doing MRI to determine the degree of liver fibrosis.
Watto: Patients with low FIB4 scores probably don’t need elastography but those with high FIB4 scores do. For the interpretation of ultrasound-based elastography results, Dr Tapper gave us the “rule of 5s”.
Elastography results are reported in kilopascal (kPa) units. A finding of 5 kPa or less is normal. Forty percent of those with a result of 10 kPa might have advanced liver disease. Above 15 kPa, the likelihood of cirrhosis is high, becoming very likely at 25 kPa. Finally, with a result of > 25 kPa, portal hypertension is likely, and you might need to have a conversation about starting the patient on medicine to prevent variceal bleeding.
We are moving toward more noninvasive testing and avoiding biopsies. We have cutoff values for MRI-based elastography as well. Both of these tests can help stage the liver.
What can we tell people about diet?
Williams: Weight loss is helpful. You can reverse fibrosis with weight loss. You can truly help your liver and bring it closer to its healthy baseline with weight loss. A loss of 7.5% body weight can reduce steatohepatitis, and with around 10% of body weight loss, you can actually resolve fibrosis, which is remarkable.
We all know that weight loss can be very therapeutic for many conditions. It’s just very hard to achieve. As primary care doctors, we should use what we have in our armamentarium to achieve that goal. Often, that will include certain medications.
Watto: I like giving patients the 10% number because if they weigh 220 pounds, they need to lose 22 pounds. If they weigh 300 pounds, it’s 30 pounds. Most people who weigh 300 pounds think they need to lose 100 pounds to have any sort of health benefit, but it’s much less than that. So, I do find that helpful.
But now a new drug has been approved. It’s a thyroid memetic called resmetirom. It was from the MAESTRO-NASH trial. Without weight loss, it helped to reverse fibrosis.
This is going to be used more and more in the future. It’s still being worked out exactly where the place is for that drug, so much so that Dr Tapper, as a liver expert, hadn’t even had the chance to prescribe it yet. Of course, it was very recently approved.
Dr. Tapper is one of our most celebrated guests, so check out the full podcast here.
A version of this article appeared on Medscape.com.
Solo Vs McDoctors Inc.
STAT News recently ran a series on UnitedHealthcare (UHC) and its growing physician empire. This includes the corporation pressuring its employed physicians to see more patients, work weekends, upcode visits, add in diagnoses that will increase reimbursement, yadda, yadda, yadda.
For legal disclaimer purposes, I’m not saying UHC did any of this, nor am I saying they didn’t. But the series on STAT is worth reading.
Reading the articles brings back memories of the last time I was an employed physician, 24 years ago. I didn’t have people telling me to upcode visits, but I do remember hearing terms such as “dollars per physician per square foot” bandied about concerning my performance. At least back then no one was going to yell at me about a 1-star online review from a disgruntled patient.
After a little over 2 years I’d had enough and went solo.
I have no desire at this point to go back to that. I certainly make a lot less money than my employed counterparts, but I also have time and a degree of peace, which are worth something.
I’m not paying for anyone else’s overhead. I don’t slack off, but at least I know what I’m working for, and where the money is going when I write out a check. I can work my schedule around having to take my dog to the vet, or pick a kid up at the airport, or whatever.
I can spend more time with the patients who need it. Isn’t that part of why I’m here?
Wearing Hawaiian shirts and shorts to the office everyday is also a plus (at least I think so).
It surprises me that more physicians aren’t willing to go into solo or small group practice. The big advantage is freedom, only needing to pay the overhead and your salary, and cover for others when needed.
The downside is financial. Like our hunting and gathering ancestors, you eat what you kill. If there’s a shortfall in cash flow, I’m the one who doesn’t get paid. It’s always good to have a line of credit available to fall back on in a pinch.
I can see why it’s daunting. Coming out of training you have loans to pay off. You may have a young family, and your first mortgage. You sure don’t want to take out another loan to start a private practice. The security of a guaranteed paycheck and no start-up costs is attractive. I was there, too, and I also took the first job I was offered back then.
There’s also the fear of suddenly working without a net for the first time in your career. It’s reassuring to get some added experience while being able to bounce a challenging case off another doctor. (I still do that, too, and always will.)
But no one tells me to upcode visits or add diagnostic codes just to get more money. Patients don’t call in panicked that they have an ICD-10 code for a condition no one told them they had.
At the end of the day I can tell the guy in the mirror that I’m doing my best.
Medicine has changed a lot over time ... but being a doctor hasn’t. The spark that led us all here is still there, somewhere, I hope. Go back and read Neighbor Rosicky by Willa Cather, and The Doctor Stories by William Carlos Williams.
In an age when technology is moving us forward, I think the practice of medicine should move backward, away from McDoctors Inc. A small, even solo, medical practice isn’t incompatible with the shiny toys of 2024 medicine. You can make good patient care happen with both.
I freely admit that it’s not for everyone.
But I wish more people would see it as a realistic option, and take the road less traveled.
Dr. Block has a solo neurology practice in Scottsdale, Arizona.
STAT News recently ran a series on UnitedHealthcare (UHC) and its growing physician empire. This includes the corporation pressuring its employed physicians to see more patients, work weekends, upcode visits, add in diagnoses that will increase reimbursement, yadda, yadda, yadda.
For legal disclaimer purposes, I’m not saying UHC did any of this, nor am I saying they didn’t. But the series on STAT is worth reading.
Reading the articles brings back memories of the last time I was an employed physician, 24 years ago. I didn’t have people telling me to upcode visits, but I do remember hearing terms such as “dollars per physician per square foot” bandied about concerning my performance. At least back then no one was going to yell at me about a 1-star online review from a disgruntled patient.
After a little over 2 years I’d had enough and went solo.
I have no desire at this point to go back to that. I certainly make a lot less money than my employed counterparts, but I also have time and a degree of peace, which are worth something.
I’m not paying for anyone else’s overhead. I don’t slack off, but at least I know what I’m working for, and where the money is going when I write out a check. I can work my schedule around having to take my dog to the vet, or pick a kid up at the airport, or whatever.
I can spend more time with the patients who need it. Isn’t that part of why I’m here?
Wearing Hawaiian shirts and shorts to the office everyday is also a plus (at least I think so).
It surprises me that more physicians aren’t willing to go into solo or small group practice. The big advantage is freedom, only needing to pay the overhead and your salary, and cover for others when needed.
The downside is financial. Like our hunting and gathering ancestors, you eat what you kill. If there’s a shortfall in cash flow, I’m the one who doesn’t get paid. It’s always good to have a line of credit available to fall back on in a pinch.
I can see why it’s daunting. Coming out of training you have loans to pay off. You may have a young family, and your first mortgage. You sure don’t want to take out another loan to start a private practice. The security of a guaranteed paycheck and no start-up costs is attractive. I was there, too, and I also took the first job I was offered back then.
There’s also the fear of suddenly working without a net for the first time in your career. It’s reassuring to get some added experience while being able to bounce a challenging case off another doctor. (I still do that, too, and always will.)
But no one tells me to upcode visits or add diagnostic codes just to get more money. Patients don’t call in panicked that they have an ICD-10 code for a condition no one told them they had.
At the end of the day I can tell the guy in the mirror that I’m doing my best.
Medicine has changed a lot over time ... but being a doctor hasn’t. The spark that led us all here is still there, somewhere, I hope. Go back and read Neighbor Rosicky by Willa Cather, and The Doctor Stories by William Carlos Williams.
In an age when technology is moving us forward, I think the practice of medicine should move backward, away from McDoctors Inc. A small, even solo, medical practice isn’t incompatible with the shiny toys of 2024 medicine. You can make good patient care happen with both.
I freely admit that it’s not for everyone.
But I wish more people would see it as a realistic option, and take the road less traveled.
Dr. Block has a solo neurology practice in Scottsdale, Arizona.
STAT News recently ran a series on UnitedHealthcare (UHC) and its growing physician empire. This includes the corporation pressuring its employed physicians to see more patients, work weekends, upcode visits, add in diagnoses that will increase reimbursement, yadda, yadda, yadda.
For legal disclaimer purposes, I’m not saying UHC did any of this, nor am I saying they didn’t. But the series on STAT is worth reading.
Reading the articles brings back memories of the last time I was an employed physician, 24 years ago. I didn’t have people telling me to upcode visits, but I do remember hearing terms such as “dollars per physician per square foot” bandied about concerning my performance. At least back then no one was going to yell at me about a 1-star online review from a disgruntled patient.
After a little over 2 years I’d had enough and went solo.
I have no desire at this point to go back to that. I certainly make a lot less money than my employed counterparts, but I also have time and a degree of peace, which are worth something.
I’m not paying for anyone else’s overhead. I don’t slack off, but at least I know what I’m working for, and where the money is going when I write out a check. I can work my schedule around having to take my dog to the vet, or pick a kid up at the airport, or whatever.
I can spend more time with the patients who need it. Isn’t that part of why I’m here?
Wearing Hawaiian shirts and shorts to the office everyday is also a plus (at least I think so).
It surprises me that more physicians aren’t willing to go into solo or small group practice. The big advantage is freedom, only needing to pay the overhead and your salary, and cover for others when needed.
The downside is financial. Like our hunting and gathering ancestors, you eat what you kill. If there’s a shortfall in cash flow, I’m the one who doesn’t get paid. It’s always good to have a line of credit available to fall back on in a pinch.
I can see why it’s daunting. Coming out of training you have loans to pay off. You may have a young family, and your first mortgage. You sure don’t want to take out another loan to start a private practice. The security of a guaranteed paycheck and no start-up costs is attractive. I was there, too, and I also took the first job I was offered back then.
There’s also the fear of suddenly working without a net for the first time in your career. It’s reassuring to get some added experience while being able to bounce a challenging case off another doctor. (I still do that, too, and always will.)
But no one tells me to upcode visits or add diagnostic codes just to get more money. Patients don’t call in panicked that they have an ICD-10 code for a condition no one told them they had.
At the end of the day I can tell the guy in the mirror that I’m doing my best.
Medicine has changed a lot over time ... but being a doctor hasn’t. The spark that led us all here is still there, somewhere, I hope. Go back and read Neighbor Rosicky by Willa Cather, and The Doctor Stories by William Carlos Williams.
In an age when technology is moving us forward, I think the practice of medicine should move backward, away from McDoctors Inc. A small, even solo, medical practice isn’t incompatible with the shiny toys of 2024 medicine. You can make good patient care happen with both.
I freely admit that it’s not for everyone.
But I wish more people would see it as a realistic option, and take the road less traveled.
Dr. Block has a solo neurology practice in Scottsdale, Arizona.
Nutrition and Medical Education
How comfortable are you giving nutritional advice to your patients? When you offer it are you basing your advice on something you learned during medical school or your training? Was it included in a course devoted to nutrition? Did you learn it later as part of continuing medical education course (CME)? Or was it just something you just picked up from your experience seeing patients (osmosis)? It is very unlikely that a significant portion, or any part for that matter, of your medical training was devoted to nutrition. It certainly wasn’t during my training.
I recently read an interview with Emily M. Broad Leib, JD, faculty director of the Harvard School Center for Health Law and Policy Innovation, Cambridge, Massachusetts, who would like to correct that deficiency. She feels doctors need to know more about food and that acquiring that knowledge should be a significant component of their formal training.
In the interview, Leib said that “roughly 86% of physicians report they do not feel adequately trained to answer basic questions on diet or nutrition.” She also notes that while “72% of entering medical students report they believe food is important to health” less than 50% retained this belief after graduation.
Leib and associates feel they have recently reached a milestone in their efforts to include nutrition in the mainstream of medical education this fall by publishing a paper that demonstrates “consensus on doctor-approved nutritional standard for medical schools and residency programs.”
36 Recommended Competencies
Curious about what these nutrition experts chose to include in medical training, I decided to drill down into the list of 36 consensus-driven competencies they had agreed upon.
It was an interesting voyage into a forest of redundancies, many of which can be boiled down to having the student demonstrate that he/she understands that what we eat is important to our health and that there is a complex web of relationships connecting our society to the food consume.
Some of the recommended competencies I found make perfect sense. For example the student/trainee should be able to take a diet and food history and be able to interpret lab values and anthropometric measurements and be able to discuss the patient’s weight and diet with sensitivity while keeping in mind his/her own biases about food.
Some other recommendations are more problematic, for example, “performs a comprehensive nutrition-focused physical examination” or “demonstrates knowledge of how to create culinary nutrition SMART [Specific, Measurable, Achievable, Relevant, and Time-Bound] goals for personal use and for patient care” or “provides brief counseling interventions to help patients decrease visceral adiposity or reduce the risk of metabolic syndrome.” Including competencies like these demonstrates a lack of understanding of the time restraints and realities of a primary care physician’s life and training.
Instead of simply reinforcing the prospective physician’s preexisting assumption that food and health are entwined and discussing when and how to consult a nutrition expert, these 36 competencies seem to be an attempt to create fast-tracked part-time dietitians and nutrition advocates out of medical students and trainees who already believe that nutrition is important for health but also have a very full plate of clinical responsibilities ahead of them.
The study that Leib quotes — that 72% of medical students believed food was important in health while after graduation only 50% of agreed — doesn’t necessarily mean that professors are preaching that food was unimportant. It is more likely by the end of medical school the students have seen that food must share the spotlight with numerous other factors that influence their patients’ health.
‘A More Appropriate Focus’
In my experience, diet and lifestyle counseling done well is extremely time consuming and best done by people for whom that is their specialty. A more appropriate focus for a list of nutritional competencies for physicians in training would be for the student to achieve an understanding of when and how to consult a dietitian and then how to support and evaluate the dietitian’s recommendations to the patient.
Finally, I don’t think we can ignore a serious public relations problem that hangs like a cloud over the nutrition advocacy community. It is the same one that casts a shadow on the medical community as well. It is a common perception among the lay public that nutritionists (and physicians) are always changing their recommendations when it comes to food. What is believable? Just think about eggs, red wine, or introducing peanuts to infants, to name just a few. And what about the food pyramids that seem to have been rebuilt every several years? The problem is compounded when some “credentialed” nutritionists and physicians continue to make dietary pronouncements with only a shred of evidence or poorly documented anecdotal observations.
The first of the 36 competencies I reviewed reads: “Provide evidence-based, culturally sensitive nutrition and food recommendations for the prevention and treatment of disease.” When it comes to nutrition the “evidence” can be tough to come by. The natural experiments in which individuals and populations had extremely limited access to a certain nutrients (eg, scurvy) don’t occur very often. Animal studies don’t always extrapolate to humans. And, observational studies concerning diet often have co-factors that are difficult to control and must run over time courses that can tax even the most patient researchers.
I certainly applaud Leib and associates for promoting their primary goal of including more about of the relationship between food and health in the medical school and trainee curriculum. But I must voice a caution to be careful to keep it truly evidence-based and in a format that acknowledges the realities of the life and education of a primary care provider.
The best nutritional advice I ever received in my training was from an older pediatric professor who suggested that a healthy diet consisted of everything in moderation.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
How comfortable are you giving nutritional advice to your patients? When you offer it are you basing your advice on something you learned during medical school or your training? Was it included in a course devoted to nutrition? Did you learn it later as part of continuing medical education course (CME)? Or was it just something you just picked up from your experience seeing patients (osmosis)? It is very unlikely that a significant portion, or any part for that matter, of your medical training was devoted to nutrition. It certainly wasn’t during my training.
I recently read an interview with Emily M. Broad Leib, JD, faculty director of the Harvard School Center for Health Law and Policy Innovation, Cambridge, Massachusetts, who would like to correct that deficiency. She feels doctors need to know more about food and that acquiring that knowledge should be a significant component of their formal training.
In the interview, Leib said that “roughly 86% of physicians report they do not feel adequately trained to answer basic questions on diet or nutrition.” She also notes that while “72% of entering medical students report they believe food is important to health” less than 50% retained this belief after graduation.
Leib and associates feel they have recently reached a milestone in their efforts to include nutrition in the mainstream of medical education this fall by publishing a paper that demonstrates “consensus on doctor-approved nutritional standard for medical schools and residency programs.”
36 Recommended Competencies
Curious about what these nutrition experts chose to include in medical training, I decided to drill down into the list of 36 consensus-driven competencies they had agreed upon.
It was an interesting voyage into a forest of redundancies, many of which can be boiled down to having the student demonstrate that he/she understands that what we eat is important to our health and that there is a complex web of relationships connecting our society to the food consume.
Some of the recommended competencies I found make perfect sense. For example the student/trainee should be able to take a diet and food history and be able to interpret lab values and anthropometric measurements and be able to discuss the patient’s weight and diet with sensitivity while keeping in mind his/her own biases about food.
Some other recommendations are more problematic, for example, “performs a comprehensive nutrition-focused physical examination” or “demonstrates knowledge of how to create culinary nutrition SMART [Specific, Measurable, Achievable, Relevant, and Time-Bound] goals for personal use and for patient care” or “provides brief counseling interventions to help patients decrease visceral adiposity or reduce the risk of metabolic syndrome.” Including competencies like these demonstrates a lack of understanding of the time restraints and realities of a primary care physician’s life and training.
Instead of simply reinforcing the prospective physician’s preexisting assumption that food and health are entwined and discussing when and how to consult a nutrition expert, these 36 competencies seem to be an attempt to create fast-tracked part-time dietitians and nutrition advocates out of medical students and trainees who already believe that nutrition is important for health but also have a very full plate of clinical responsibilities ahead of them.
The study that Leib quotes — that 72% of medical students believed food was important in health while after graduation only 50% of agreed — doesn’t necessarily mean that professors are preaching that food was unimportant. It is more likely by the end of medical school the students have seen that food must share the spotlight with numerous other factors that influence their patients’ health.
‘A More Appropriate Focus’
In my experience, diet and lifestyle counseling done well is extremely time consuming and best done by people for whom that is their specialty. A more appropriate focus for a list of nutritional competencies for physicians in training would be for the student to achieve an understanding of when and how to consult a dietitian and then how to support and evaluate the dietitian’s recommendations to the patient.
Finally, I don’t think we can ignore a serious public relations problem that hangs like a cloud over the nutrition advocacy community. It is the same one that casts a shadow on the medical community as well. It is a common perception among the lay public that nutritionists (and physicians) are always changing their recommendations when it comes to food. What is believable? Just think about eggs, red wine, or introducing peanuts to infants, to name just a few. And what about the food pyramids that seem to have been rebuilt every several years? The problem is compounded when some “credentialed” nutritionists and physicians continue to make dietary pronouncements with only a shred of evidence or poorly documented anecdotal observations.
The first of the 36 competencies I reviewed reads: “Provide evidence-based, culturally sensitive nutrition and food recommendations for the prevention and treatment of disease.” When it comes to nutrition the “evidence” can be tough to come by. The natural experiments in which individuals and populations had extremely limited access to a certain nutrients (eg, scurvy) don’t occur very often. Animal studies don’t always extrapolate to humans. And, observational studies concerning diet often have co-factors that are difficult to control and must run over time courses that can tax even the most patient researchers.
I certainly applaud Leib and associates for promoting their primary goal of including more about of the relationship between food and health in the medical school and trainee curriculum. But I must voice a caution to be careful to keep it truly evidence-based and in a format that acknowledges the realities of the life and education of a primary care provider.
The best nutritional advice I ever received in my training was from an older pediatric professor who suggested that a healthy diet consisted of everything in moderation.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
How comfortable are you giving nutritional advice to your patients? When you offer it are you basing your advice on something you learned during medical school or your training? Was it included in a course devoted to nutrition? Did you learn it later as part of continuing medical education course (CME)? Or was it just something you just picked up from your experience seeing patients (osmosis)? It is very unlikely that a significant portion, or any part for that matter, of your medical training was devoted to nutrition. It certainly wasn’t during my training.
I recently read an interview with Emily M. Broad Leib, JD, faculty director of the Harvard School Center for Health Law and Policy Innovation, Cambridge, Massachusetts, who would like to correct that deficiency. She feels doctors need to know more about food and that acquiring that knowledge should be a significant component of their formal training.
In the interview, Leib said that “roughly 86% of physicians report they do not feel adequately trained to answer basic questions on diet or nutrition.” She also notes that while “72% of entering medical students report they believe food is important to health” less than 50% retained this belief after graduation.
Leib and associates feel they have recently reached a milestone in their efforts to include nutrition in the mainstream of medical education this fall by publishing a paper that demonstrates “consensus on doctor-approved nutritional standard for medical schools and residency programs.”
36 Recommended Competencies
Curious about what these nutrition experts chose to include in medical training, I decided to drill down into the list of 36 consensus-driven competencies they had agreed upon.
It was an interesting voyage into a forest of redundancies, many of which can be boiled down to having the student demonstrate that he/she understands that what we eat is important to our health and that there is a complex web of relationships connecting our society to the food consume.
Some of the recommended competencies I found make perfect sense. For example the student/trainee should be able to take a diet and food history and be able to interpret lab values and anthropometric measurements and be able to discuss the patient’s weight and diet with sensitivity while keeping in mind his/her own biases about food.
Some other recommendations are more problematic, for example, “performs a comprehensive nutrition-focused physical examination” or “demonstrates knowledge of how to create culinary nutrition SMART [Specific, Measurable, Achievable, Relevant, and Time-Bound] goals for personal use and for patient care” or “provides brief counseling interventions to help patients decrease visceral adiposity or reduce the risk of metabolic syndrome.” Including competencies like these demonstrates a lack of understanding of the time restraints and realities of a primary care physician’s life and training.
Instead of simply reinforcing the prospective physician’s preexisting assumption that food and health are entwined and discussing when and how to consult a nutrition expert, these 36 competencies seem to be an attempt to create fast-tracked part-time dietitians and nutrition advocates out of medical students and trainees who already believe that nutrition is important for health but also have a very full plate of clinical responsibilities ahead of them.
The study that Leib quotes — that 72% of medical students believed food was important in health while after graduation only 50% of agreed — doesn’t necessarily mean that professors are preaching that food was unimportant. It is more likely by the end of medical school the students have seen that food must share the spotlight with numerous other factors that influence their patients’ health.
‘A More Appropriate Focus’
In my experience, diet and lifestyle counseling done well is extremely time consuming and best done by people for whom that is their specialty. A more appropriate focus for a list of nutritional competencies for physicians in training would be for the student to achieve an understanding of when and how to consult a dietitian and then how to support and evaluate the dietitian’s recommendations to the patient.
Finally, I don’t think we can ignore a serious public relations problem that hangs like a cloud over the nutrition advocacy community. It is the same one that casts a shadow on the medical community as well. It is a common perception among the lay public that nutritionists (and physicians) are always changing their recommendations when it comes to food. What is believable? Just think about eggs, red wine, or introducing peanuts to infants, to name just a few. And what about the food pyramids that seem to have been rebuilt every several years? The problem is compounded when some “credentialed” nutritionists and physicians continue to make dietary pronouncements with only a shred of evidence or poorly documented anecdotal observations.
The first of the 36 competencies I reviewed reads: “Provide evidence-based, culturally sensitive nutrition and food recommendations for the prevention and treatment of disease.” When it comes to nutrition the “evidence” can be tough to come by. The natural experiments in which individuals and populations had extremely limited access to a certain nutrients (eg, scurvy) don’t occur very often. Animal studies don’t always extrapolate to humans. And, observational studies concerning diet often have co-factors that are difficult to control and must run over time courses that can tax even the most patient researchers.
I certainly applaud Leib and associates for promoting their primary goal of including more about of the relationship between food and health in the medical school and trainee curriculum. But I must voice a caution to be careful to keep it truly evidence-based and in a format that acknowledges the realities of the life and education of a primary care provider.
The best nutritional advice I ever received in my training was from an older pediatric professor who suggested that a healthy diet consisted of everything in moderation.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
On Second Thought: Aspirin for Primary Prevention — What We Really Know
This transcript has been edited for clarity.
Our recommendations vis-à-vis aspirin have evolved at a dizzying pace. The young’uns watching us right now don’t know what things were like in the 1980s. The Reagan era was a wild, heady time where nuclear war was imminent and we didn’t prescribe aspirin to patients.
That only started in 1988, which was a banner year in human history. Not because a number of doves were incinerated by the lighting of the Olympic torch at the Seoul Olympics — look it up if you don’t know what I’m talking about — but because 1988 saw the publication of the ISIS-2 trial, which first showed a mortality benefit to prescribing aspirin post–myocardial infarction (MI).
Giving patients aspirin during or after a heart attack is not controversial. It’s one of the few things in this business that isn’t, but that’s secondary prevention — treating somebody after they develop a disease. Primary prevention, treating them before they have their incident event, is a very different ballgame. Here, things are messy.
For one thing, the doses used have been very inconsistent. We should point out that the reason for 81 mg of aspirin is very arbitrary and is rooted in the old apothecary system of weights and measurements. A standard dose of aspirin was 5 grains, where 20 grains made 1 scruple, 3 scruples made 1 dram, 8 drams made 1 oz, and 12 oz made 1 lb - because screw you, metric system. Therefore, 5 grains was 325 mg of aspirin, and 1 quarter of the standard dose became 81 mg if you rounded out the decimal.
People have tried all kinds of dosing structures with aspirin prophylaxis. The Physicians’ Health Study used a full-dose aspirin, 325 mg every 2 days, while the Hypertension Optimal Treatment (HOT) trial tested 75 mg daily and the Women’s Health Study tested 100 mg, but every other day.
Ironically, almost no one has studied 81 mg every day, which is weird if you think about it. The bigger problem here is not the variability of doses used, but the discrepancy when you look at older vs newer studies.
Older studies, like the Physicians’ Health Study, did show a benefit, at least in the subgroup of patients over age 50 years, which is probably where the “everybody over 50 should be taking an aspirin” idea comes from, at least as near as I can tell.
More recent studies, like the Women’s Health Study, ASPREE, or ASPIRE, didn’t show a benefit. I know what you’re thinking: Newer stuff is always better. That’s why you should never trust anybody over age 40 years. The context of primary prevention studies has changed. In the ‘80s and ‘90s, people smoked more and we didn’t have the same medications that we have today. We talked about all this in the beta-blocker video to explain why beta-blockers don’t seem to have a benefit post MI.
We have a similar issue here. The magnitude of the benefit with aspirin primary prevention has decreased because we’re all just healthier overall. So, yay! Progress! Here’s where the numbers matter. No one is saying that aspirin doesn’t help. It does.
If we look at the 2019 meta-analysis published in JAMA, there is a cardiovascular benefit. The numbers bear that out. I know you’re all here for the math, so here we go. Aspirin reduced the composite cardiovascular endpoint from 65.2 to 60.2 events per 10,000 patient-years; or to put it more meaningfully in absolute risk reduction terms, because that’s my jam, an absolute risk reduction of 0.41%, which means a number needed to treat of 241, which is okay-ish. It’s not super-great, but it may be justifiable for something that costs next to nothing.
The tradeoff is bleeding. Major bleeding increased from 16.4 to 23.1 bleeds per 10,000 patient-years, or an absolute risk increase of 0.47%, which is a number needed to harm of 210. That’s the problem. Aspirin does prevent heart disease. The benefit is small, for sure, but the real problem is that it’s outweighed by the risk of bleeding, so you’re not really coming out ahead.
The real tragedy here is that the public is locked into this idea of everyone over age 50 years should be taking an aspirin. Even today, even though guidelines have recommended against aspirin for primary prevention for some time, data from the National Health Interview Survey sample found that nearly one in three older adults take aspirin for primary prevention when they shouldn’t be. That’s a large number of people. That’s millions of Americans — and Canadians, but nobody cares about us. It’s fine.
That’s the point. We’re not debunking aspirin. It does work. The benefits are just really small in a primary prevention population and offset by the admittedly also really small risks of bleeding. It’s a tradeoff that doesn’t really work in your favor.
But that’s aspirin for cardiovascular disease. When it comes to cancer or DVT prophylaxis, that’s another really interesting story. We might have to save that for another time. Do I know how to tease a sequel or what?
Labos, a cardiologist at Kirkland Medical Center, Montreal, Quebec, Canada, has disclosed no relevant financial relationships.
A version of this article appeared on Medscape.com.
This transcript has been edited for clarity.
Our recommendations vis-à-vis aspirin have evolved at a dizzying pace. The young’uns watching us right now don’t know what things were like in the 1980s. The Reagan era was a wild, heady time where nuclear war was imminent and we didn’t prescribe aspirin to patients.
That only started in 1988, which was a banner year in human history. Not because a number of doves were incinerated by the lighting of the Olympic torch at the Seoul Olympics — look it up if you don’t know what I’m talking about — but because 1988 saw the publication of the ISIS-2 trial, which first showed a mortality benefit to prescribing aspirin post–myocardial infarction (MI).
Giving patients aspirin during or after a heart attack is not controversial. It’s one of the few things in this business that isn’t, but that’s secondary prevention — treating somebody after they develop a disease. Primary prevention, treating them before they have their incident event, is a very different ballgame. Here, things are messy.
For one thing, the doses used have been very inconsistent. We should point out that the reason for 81 mg of aspirin is very arbitrary and is rooted in the old apothecary system of weights and measurements. A standard dose of aspirin was 5 grains, where 20 grains made 1 scruple, 3 scruples made 1 dram, 8 drams made 1 oz, and 12 oz made 1 lb - because screw you, metric system. Therefore, 5 grains was 325 mg of aspirin, and 1 quarter of the standard dose became 81 mg if you rounded out the decimal.
People have tried all kinds of dosing structures with aspirin prophylaxis. The Physicians’ Health Study used a full-dose aspirin, 325 mg every 2 days, while the Hypertension Optimal Treatment (HOT) trial tested 75 mg daily and the Women’s Health Study tested 100 mg, but every other day.
Ironically, almost no one has studied 81 mg every day, which is weird if you think about it. The bigger problem here is not the variability of doses used, but the discrepancy when you look at older vs newer studies.
Older studies, like the Physicians’ Health Study, did show a benefit, at least in the subgroup of patients over age 50 years, which is probably where the “everybody over 50 should be taking an aspirin” idea comes from, at least as near as I can tell.
More recent studies, like the Women’s Health Study, ASPREE, or ASPIRE, didn’t show a benefit. I know what you’re thinking: Newer stuff is always better. That’s why you should never trust anybody over age 40 years. The context of primary prevention studies has changed. In the ‘80s and ‘90s, people smoked more and we didn’t have the same medications that we have today. We talked about all this in the beta-blocker video to explain why beta-blockers don’t seem to have a benefit post MI.
We have a similar issue here. The magnitude of the benefit with aspirin primary prevention has decreased because we’re all just healthier overall. So, yay! Progress! Here’s where the numbers matter. No one is saying that aspirin doesn’t help. It does.
If we look at the 2019 meta-analysis published in JAMA, there is a cardiovascular benefit. The numbers bear that out. I know you’re all here for the math, so here we go. Aspirin reduced the composite cardiovascular endpoint from 65.2 to 60.2 events per 10,000 patient-years; or to put it more meaningfully in absolute risk reduction terms, because that’s my jam, an absolute risk reduction of 0.41%, which means a number needed to treat of 241, which is okay-ish. It’s not super-great, but it may be justifiable for something that costs next to nothing.
The tradeoff is bleeding. Major bleeding increased from 16.4 to 23.1 bleeds per 10,000 patient-years, or an absolute risk increase of 0.47%, which is a number needed to harm of 210. That’s the problem. Aspirin does prevent heart disease. The benefit is small, for sure, but the real problem is that it’s outweighed by the risk of bleeding, so you’re not really coming out ahead.
The real tragedy here is that the public is locked into this idea of everyone over age 50 years should be taking an aspirin. Even today, even though guidelines have recommended against aspirin for primary prevention for some time, data from the National Health Interview Survey sample found that nearly one in three older adults take aspirin for primary prevention when they shouldn’t be. That’s a large number of people. That’s millions of Americans — and Canadians, but nobody cares about us. It’s fine.
That’s the point. We’re not debunking aspirin. It does work. The benefits are just really small in a primary prevention population and offset by the admittedly also really small risks of bleeding. It’s a tradeoff that doesn’t really work in your favor.
But that’s aspirin for cardiovascular disease. When it comes to cancer or DVT prophylaxis, that’s another really interesting story. We might have to save that for another time. Do I know how to tease a sequel or what?
Labos, a cardiologist at Kirkland Medical Center, Montreal, Quebec, Canada, has disclosed no relevant financial relationships.
A version of this article appeared on Medscape.com.
This transcript has been edited for clarity.
Our recommendations vis-à-vis aspirin have evolved at a dizzying pace. The young’uns watching us right now don’t know what things were like in the 1980s. The Reagan era was a wild, heady time where nuclear war was imminent and we didn’t prescribe aspirin to patients.
That only started in 1988, which was a banner year in human history. Not because a number of doves were incinerated by the lighting of the Olympic torch at the Seoul Olympics — look it up if you don’t know what I’m talking about — but because 1988 saw the publication of the ISIS-2 trial, which first showed a mortality benefit to prescribing aspirin post–myocardial infarction (MI).
Giving patients aspirin during or after a heart attack is not controversial. It’s one of the few things in this business that isn’t, but that’s secondary prevention — treating somebody after they develop a disease. Primary prevention, treating them before they have their incident event, is a very different ballgame. Here, things are messy.
For one thing, the doses used have been very inconsistent. We should point out that the reason for 81 mg of aspirin is very arbitrary and is rooted in the old apothecary system of weights and measurements. A standard dose of aspirin was 5 grains, where 20 grains made 1 scruple, 3 scruples made 1 dram, 8 drams made 1 oz, and 12 oz made 1 lb - because screw you, metric system. Therefore, 5 grains was 325 mg of aspirin, and 1 quarter of the standard dose became 81 mg if you rounded out the decimal.
People have tried all kinds of dosing structures with aspirin prophylaxis. The Physicians’ Health Study used a full-dose aspirin, 325 mg every 2 days, while the Hypertension Optimal Treatment (HOT) trial tested 75 mg daily and the Women’s Health Study tested 100 mg, but every other day.
Ironically, almost no one has studied 81 mg every day, which is weird if you think about it. The bigger problem here is not the variability of doses used, but the discrepancy when you look at older vs newer studies.
Older studies, like the Physicians’ Health Study, did show a benefit, at least in the subgroup of patients over age 50 years, which is probably where the “everybody over 50 should be taking an aspirin” idea comes from, at least as near as I can tell.
More recent studies, like the Women’s Health Study, ASPREE, or ASPIRE, didn’t show a benefit. I know what you’re thinking: Newer stuff is always better. That’s why you should never trust anybody over age 40 years. The context of primary prevention studies has changed. In the ‘80s and ‘90s, people smoked more and we didn’t have the same medications that we have today. We talked about all this in the beta-blocker video to explain why beta-blockers don’t seem to have a benefit post MI.
We have a similar issue here. The magnitude of the benefit with aspirin primary prevention has decreased because we’re all just healthier overall. So, yay! Progress! Here’s where the numbers matter. No one is saying that aspirin doesn’t help. It does.
If we look at the 2019 meta-analysis published in JAMA, there is a cardiovascular benefit. The numbers bear that out. I know you’re all here for the math, so here we go. Aspirin reduced the composite cardiovascular endpoint from 65.2 to 60.2 events per 10,000 patient-years; or to put it more meaningfully in absolute risk reduction terms, because that’s my jam, an absolute risk reduction of 0.41%, which means a number needed to treat of 241, which is okay-ish. It’s not super-great, but it may be justifiable for something that costs next to nothing.
The tradeoff is bleeding. Major bleeding increased from 16.4 to 23.1 bleeds per 10,000 patient-years, or an absolute risk increase of 0.47%, which is a number needed to harm of 210. That’s the problem. Aspirin does prevent heart disease. The benefit is small, for sure, but the real problem is that it’s outweighed by the risk of bleeding, so you’re not really coming out ahead.
The real tragedy here is that the public is locked into this idea of everyone over age 50 years should be taking an aspirin. Even today, even though guidelines have recommended against aspirin for primary prevention for some time, data from the National Health Interview Survey sample found that nearly one in three older adults take aspirin for primary prevention when they shouldn’t be. That’s a large number of people. That’s millions of Americans — and Canadians, but nobody cares about us. It’s fine.
That’s the point. We’re not debunking aspirin. It does work. The benefits are just really small in a primary prevention population and offset by the admittedly also really small risks of bleeding. It’s a tradeoff that doesn’t really work in your favor.
But that’s aspirin for cardiovascular disease. When it comes to cancer or DVT prophylaxis, that’s another really interesting story. We might have to save that for another time. Do I know how to tease a sequel or what?
Labos, a cardiologist at Kirkland Medical Center, Montreal, Quebec, Canada, has disclosed no relevant financial relationships.
A version of this article appeared on Medscape.com.
Screening Options for Rare Malignancies
Dear colleagues,
As gastroenterologists and endoscopists, we spend significant time preventing and diagnosing GI malignancies.
For instance, is it worthwhile screening for pancreatic cancer, and, if so, how should this be done? Likewise, diagnosing cholangiocarcinoma is challenging; how best should one evaluate for this in higher risk populations, such as primary sclerosing cholangitis? And what about the costs, financial and otherwise, associated with screening?
In this issue of Perspectives, Dr. Darshan Kothari and Dr. Daniel Bernstein discuss their approach to pancreatic cancer screening, including who is eligible, the preferred screening modalities, and the barriers to screening. In the accompanying perspective, Dr. Aparna Goel and Dr. Judah Kupferman focus on cholangiocarcinoma screening, identifying high-risk populations and discussing some of the concerns with screening, necessitating shared decision-making.
We welcome your thoughts on this issue. Share with us on X at @AGA_GIHN.
Gyanprakash A. Ketwaroo, MD, MSc, is associate professor of medicine, Yale University, New Haven, and chief of endoscopy at West Haven VA Medical Center, both in Connecticut. He is an associate editor for GI & Hepatology News.
An Approach to Pancreatic Cancer Screening
BY DANIEL A. BERNSTEIN, MD, AND DARSHAN KOTHARI, MD
Pancreatic cancer carries a dismal prognosis, now accounting for the third-most cancer-related mortality in the United States. A small proportion of patients are diagnosed at a local stage of disease, with over half found to have metastatic disease at presentation. Given the low overall incidence and lifetime risk in the general population, population-based screening is not justified.
About 10% of cases of pancreas cancer are associated with germ-line mutations and/or with a strong family history of pancreatic cancer. Several academic societies and expert committees now recommend regular screening for pancreatic cancer in patients who are considered high-risk individuals, as they carry a fivefold relative risk for pancreatic cancer. Moreover, studies suggest that screening has the potential to identify early-stage resectable disease and decrease mortality in this patient population.
Patients who benefit from pancreatic cancer screening are those who carry an increased lifetime risk (in excess of 5%) of pancreatic cancer. High-risk individuals include those with germ-line mutations and/or those with a family history of pancreatic cancer in first-degree relatives. Consensus guidelines by the International Cancer of the Pancreas Screening Consortium and the American Society for Gastrointestinal Endoscopy provide medical centers with detailed recommendations on who and when to start screening.
High-risk individuals fall into three categories:
- Patients with high-risk germline mutations including: familial atypical multiple mole melanoma syndrome (CDKN2A), hereditary breast and ovarian cancer syndromes (BRCA1, BRCA2, and PALB2), Peutz-Jeghers syndrome (STK11), and hereditary pancreatitis (PRSS1 and SPINK1)
- Patients with low- to moderate-risk germ-line mutations with at least one first-degree relative with pancreatic cancer: Lynch Syndrome (particularly MLH1 mutation), ataxia-telangiectasia (ATM), or Li-Fraumeni syndrome (p53)
- Patients with one first-degree relative with pancreatic cancer who in turn has one first-degree relative with pancreatic cancer (eg, a patient’s mother and maternal aunt or a patient’s father and patient’s sister)
Consistent with established guidelines, we recommend screening for high-risk patients beginning at age 50, or 10 years before the youngest age at which pancreas cancer was diagnosed in an affected relative. Screening is recommended earlier in patients with particularly high risk: at age 40 for patients with CDKN2A and STKI11 mutations and age 40 for patients with PRSS1 mutation or 20 years after the first attack of acute pancreatitis. For patients with a strong family history of pancreas cancer, we recommend comprehensive evaluation by a certified genetic counselor at a high-volume cancer center.
In practice, patients at our institution who are identified as high risk based on the above criteria are referred for an initial consultation at our pancreas center. In most cases, this should occur no sooner than 5 years prior to the recommended starting age for screening. All patients who are identified as high risk should be screened annually for diabetes given the growing evidence base supporting an association between new-onset diabetes and pancreatic cancer.
After an initial visit and discussion of the risks and benefits of screening, most screening protocols start with a baseline endoscopic ultrasound (EUS) and contrast-enhanced magnetic resonance abdomen with magnetic resonance cholangiopancreatography (MRI/MRCP), which will be repeated annually or sooner as the clinical condition warrants. A sooner-interval EUS should be considered for patients already undergoing screening who are newly found to have diabetes.
At our institution, we start with an in-person clinic evaluation followed by EUS. Thereafter, patients undergo MRI/MRCP (synchronized with a same-day clinic visit) alternating with EUS every 6 months to ensure patients are seen twice a year, though there is no specific data to support this approach. Non-diabetics also undergo yearly diabetes screening which will trigger an EUS if patients become diabetic.
We engage in shared decision-making with our high-risk individuals undergoing pancreatic cancer screening and at each visit we review their concurrent medical conditions and suitability to continue screening. We consider discontinuing screening after age 75, at the onset of any life-limiting illness, or after a discussion of risks and benefits if comorbidities lead to a substantial deterioration in a patient’s overall health status.
While a growing body of evidence exists to support the application of pancreatic cancer screening in high-risk individuals, this preventive service remains underutilized. Recent analysis of the screening cohort at our institution showed a demographically homogeneous group of mostly highly educated, high-income White females. These findings are consistent with the patient cohorts described in other pancreatic cancer screening programs and represent only a fraction of people who would qualify for pancreatic cancer screening.
A survey of patients undergoing screening at our institution identified cost, travel, and time associated with pancreatic cancer screening to be frequent challenges to participation. Further studies are needed to fully explore the barriers and psychological burden of pancreas cancer screening in high-risk individuals, and to identify ways to enrich the cohort of patients undergoing screening. This may involve novel methods to identify family members of patients with a new diagnosis of pancreas cancer and increasing health literacy around pancreatic cancer screening among patients and providers.
Pancreatic cancer screening has the potential to identify early-stage disease in patients who are at high risk because of germ-line mutations and/or family history. We recommend that patients engage in pancreatic cancer screening at high-volume centers with well-supported oncology, genetics, and research infrastructure.
Dr. Bernstein is a gastroenterology fellow at Duke University School of Medicine, Durham, North Carolina. Dr. Kothari is an associate professor of medicine in gastroenterology and hepatology at Duke University School of Medicine.
Screening for Cholangiocarcinoma
BY JUDAH KUPFERMAN, MD, AND APARNA GOEL, MD
Cholangiocarcinoma is a rare but aggressive cancer of the bile ducts that poses many diagnostic challenges. Approximately 3% of gastrointestinal cancers are attributed to cholangiocarcinoma, and while the annual incidence of disease in the United States is about 1.26 per 100,000 people, the incidence of intrahepatic disease has been rising considerably.1,2 Screening for cholangiocarcinoma is reserved for high-risk individuals — such as those with primary sclerosing cholangitis (PSC), secondary sclerosing cholangitis (SSC), and biliary tract disorders such as choledochal cysts or Caroli’s disease. The goal is to balance the benefits of early diagnosis with the costs and risks associated with screening, particularly given the limitations of available tools like MRI with cholangiopancreatography (MRCP), which has a sensitivity of 70%-85%. In general, we recommend annual cholangiocarcinoma screening for high-risk individuals with MRI and MRCP as well as with cancer antigen (CA) 19-9. .
Screening in Patients with Primary Sclerosing Cholangitis
The lifetime risk of cholangiocarcinoma in patients with PSC is 10%-15% with an annual risk of 0.5%-1.5%. In our experience, this is often the most feared complication for PSC patients, even more so than the risk of liver transplantation. We recommend annual MRI with MRCP in addition to CA 19-9 for patients with PSC in the first decade of their diagnosis, as most cancers are diagnosed during this period. If a patient’s imaging has remained stable for over a decade and there is minimal hepatic fibrosis, we discuss the option of reducing screening frequency to every 2 years to minimize costs and exposure to MRI contrast risks.
If MRI reveals a concerning new large duct stricture, we will evaluate this with an endoscopic retrograde cholangiopancreatography (ERCP), as differentiating benign and malignant strictures is quite challenging with MRI. We generally recommend ERCP with brush cytology and fluorescence in situ hybridization to improve diagnostic yield. Depending on imaging findings and location of the new large duct stricture, we may consider cholangioscopy during ERCP for direct visualization of the bile duct and directed tissue biopsies. Unfortunately, even in young, asymptomatic patients who undergo regular screening, cholangiocarcinoma is frequently diagnosed at an advanced stage.
Screening in Patients with Secondary Sclerosing Cholangitis
Patients with SSC may develop cholangiocarcinoma because of chronic inflammatory and fibrotic processes, such as IgG4-associated cholangiopathy, sarcoidosis, ischemic cholangiopathy, cystic fibrosis, recurrent pyogenic cholangitis, severe sepsis (as recently seen from SARS-CoV-2), surgical complications, or other etiologies. When the condition is reversible, such as with IgG4-associated cholangiopathy, cancer screening may not be necessary. However, when irreversible damage occurs, the cancer risk increases, though it varies by disease type and severity. In most cases, we recommend routine screening for cholangiocarcinoma with MRI and CA 19-9 in this population.
Screening in Patients with Biliary Tract Disorders
Biliary tract disorders such as choledochal cysts and Caroli’s disease also harbor an increased risk of cholangiocarcinoma. Choledochal cysts are congenital cystic dilations of the bile duct that have a 10%-30% lifetime risk of malignant transformation to cholangiocarcinoma. Surgical intervention to remove the cyst is often recommended because of this high risk. However, some patients may be unable or unwilling to undergo this surgery or they may have residual cysts. We recommend ongoing screening with MRI and CA 19-9 for these patients. Similarly, Caroli’s disease is a congenital disease associated with intrahepatic and extrahepatic bile duct cysts and associated with a 5%-15% lifetime risk of cholangiocarcinoma. MRI with MRCP and CA 19-9 should be performed routinely for patients with Caroli’s disease and syndrome.
Risks and Challenges in Cholangiocarcinoma Screening
While MRI with MRCP is the gold standard for cholangiocarcinoma screening, its limitations must be carefully considered. One growing concern is the potential for gadolinium retention in the brain, bones, or skin following repeated MRI scans. Though the long-term effects of gadolinium retention are not fully understood, we factor this into screening decisions, particularly for younger patients who may undergo decades of regular imaging.
MRI is not always feasible for certain patients, including those with metal implants, on hemodialysis, or with severe allergic reactions. In such cases, CT or ultrasound may serve as alternatives, though with lower sensitivity for detecting cholangiocarcinoma. Additionally, claustrophobia during MRI can be addressed with sedation, but this underscores the importance of shared decision-making.
From our perspective, cholangiocarcinoma screening in high-risk patients is crucial but not without challenges. Our current screening methods, while essential, are far from perfect, often missing early cancers or leading to unnecessary interventions. Because of these limitations, the window for treatment of localized disease can easily be missed. In our practice, we tailor screening strategies to each patient’s specific needs, weighing the potential benefits against the risks, costs, and the inherent uncertainty of early detection tools. We believe it is essential to involve patients in this decision-making process to provide a balanced, individualized approach that considers both clinical evidence and the personal preferences of each person.
Dr. Kupferman is a gastroenterology fellow at Stanford University School of Medicine in California. Dr. Goel is a transplant hepatologist and a clinical associate professor in gastroenterology & hepatology at Stanford.
References
1. Vithayathil M and Khan SA. J Hepatol. 2022 Dec. doi: 10.1016/j.jhep.2022.07.022.
2. Patel N and Benipal B. Cureus. 2019 Jan. doi: 10.7759/cureus.3962.
Dear colleagues,
As gastroenterologists and endoscopists, we spend significant time preventing and diagnosing GI malignancies.
For instance, is it worthwhile screening for pancreatic cancer, and, if so, how should this be done? Likewise, diagnosing cholangiocarcinoma is challenging; how best should one evaluate for this in higher risk populations, such as primary sclerosing cholangitis? And what about the costs, financial and otherwise, associated with screening?
In this issue of Perspectives, Dr. Darshan Kothari and Dr. Daniel Bernstein discuss their approach to pancreatic cancer screening, including who is eligible, the preferred screening modalities, and the barriers to screening. In the accompanying perspective, Dr. Aparna Goel and Dr. Judah Kupferman focus on cholangiocarcinoma screening, identifying high-risk populations and discussing some of the concerns with screening, necessitating shared decision-making.
We welcome your thoughts on this issue. Share with us on X at @AGA_GIHN.
Gyanprakash A. Ketwaroo, MD, MSc, is associate professor of medicine, Yale University, New Haven, and chief of endoscopy at West Haven VA Medical Center, both in Connecticut. He is an associate editor for GI & Hepatology News.
An Approach to Pancreatic Cancer Screening
BY DANIEL A. BERNSTEIN, MD, AND DARSHAN KOTHARI, MD
Pancreatic cancer carries a dismal prognosis, now accounting for the third-most cancer-related mortality in the United States. A small proportion of patients are diagnosed at a local stage of disease, with over half found to have metastatic disease at presentation. Given the low overall incidence and lifetime risk in the general population, population-based screening is not justified.
About 10% of cases of pancreas cancer are associated with germ-line mutations and/or with a strong family history of pancreatic cancer. Several academic societies and expert committees now recommend regular screening for pancreatic cancer in patients who are considered high-risk individuals, as they carry a fivefold relative risk for pancreatic cancer. Moreover, studies suggest that screening has the potential to identify early-stage resectable disease and decrease mortality in this patient population.
Patients who benefit from pancreatic cancer screening are those who carry an increased lifetime risk (in excess of 5%) of pancreatic cancer. High-risk individuals include those with germ-line mutations and/or those with a family history of pancreatic cancer in first-degree relatives. Consensus guidelines by the International Cancer of the Pancreas Screening Consortium and the American Society for Gastrointestinal Endoscopy provide medical centers with detailed recommendations on who and when to start screening.
High-risk individuals fall into three categories:
- Patients with high-risk germline mutations including: familial atypical multiple mole melanoma syndrome (CDKN2A), hereditary breast and ovarian cancer syndromes (BRCA1, BRCA2, and PALB2), Peutz-Jeghers syndrome (STK11), and hereditary pancreatitis (PRSS1 and SPINK1)
- Patients with low- to moderate-risk germ-line mutations with at least one first-degree relative with pancreatic cancer: Lynch Syndrome (particularly MLH1 mutation), ataxia-telangiectasia (ATM), or Li-Fraumeni syndrome (p53)
- Patients with one first-degree relative with pancreatic cancer who in turn has one first-degree relative with pancreatic cancer (eg, a patient’s mother and maternal aunt or a patient’s father and patient’s sister)
Consistent with established guidelines, we recommend screening for high-risk patients beginning at age 50, or 10 years before the youngest age at which pancreas cancer was diagnosed in an affected relative. Screening is recommended earlier in patients with particularly high risk: at age 40 for patients with CDKN2A and STKI11 mutations and age 40 for patients with PRSS1 mutation or 20 years after the first attack of acute pancreatitis. For patients with a strong family history of pancreas cancer, we recommend comprehensive evaluation by a certified genetic counselor at a high-volume cancer center.
In practice, patients at our institution who are identified as high risk based on the above criteria are referred for an initial consultation at our pancreas center. In most cases, this should occur no sooner than 5 years prior to the recommended starting age for screening. All patients who are identified as high risk should be screened annually for diabetes given the growing evidence base supporting an association between new-onset diabetes and pancreatic cancer.
After an initial visit and discussion of the risks and benefits of screening, most screening protocols start with a baseline endoscopic ultrasound (EUS) and contrast-enhanced magnetic resonance abdomen with magnetic resonance cholangiopancreatography (MRI/MRCP), which will be repeated annually or sooner as the clinical condition warrants. A sooner-interval EUS should be considered for patients already undergoing screening who are newly found to have diabetes.
At our institution, we start with an in-person clinic evaluation followed by EUS. Thereafter, patients undergo MRI/MRCP (synchronized with a same-day clinic visit) alternating with EUS every 6 months to ensure patients are seen twice a year, though there is no specific data to support this approach. Non-diabetics also undergo yearly diabetes screening which will trigger an EUS if patients become diabetic.
We engage in shared decision-making with our high-risk individuals undergoing pancreatic cancer screening and at each visit we review their concurrent medical conditions and suitability to continue screening. We consider discontinuing screening after age 75, at the onset of any life-limiting illness, or after a discussion of risks and benefits if comorbidities lead to a substantial deterioration in a patient’s overall health status.
While a growing body of evidence exists to support the application of pancreatic cancer screening in high-risk individuals, this preventive service remains underutilized. Recent analysis of the screening cohort at our institution showed a demographically homogeneous group of mostly highly educated, high-income White females. These findings are consistent with the patient cohorts described in other pancreatic cancer screening programs and represent only a fraction of people who would qualify for pancreatic cancer screening.
A survey of patients undergoing screening at our institution identified cost, travel, and time associated with pancreatic cancer screening to be frequent challenges to participation. Further studies are needed to fully explore the barriers and psychological burden of pancreas cancer screening in high-risk individuals, and to identify ways to enrich the cohort of patients undergoing screening. This may involve novel methods to identify family members of patients with a new diagnosis of pancreas cancer and increasing health literacy around pancreatic cancer screening among patients and providers.
Pancreatic cancer screening has the potential to identify early-stage disease in patients who are at high risk because of germ-line mutations and/or family history. We recommend that patients engage in pancreatic cancer screening at high-volume centers with well-supported oncology, genetics, and research infrastructure.
Dr. Bernstein is a gastroenterology fellow at Duke University School of Medicine, Durham, North Carolina. Dr. Kothari is an associate professor of medicine in gastroenterology and hepatology at Duke University School of Medicine.
Screening for Cholangiocarcinoma
BY JUDAH KUPFERMAN, MD, AND APARNA GOEL, MD
Cholangiocarcinoma is a rare but aggressive cancer of the bile ducts that poses many diagnostic challenges. Approximately 3% of gastrointestinal cancers are attributed to cholangiocarcinoma, and while the annual incidence of disease in the United States is about 1.26 per 100,000 people, the incidence of intrahepatic disease has been rising considerably.1,2 Screening for cholangiocarcinoma is reserved for high-risk individuals — such as those with primary sclerosing cholangitis (PSC), secondary sclerosing cholangitis (SSC), and biliary tract disorders such as choledochal cysts or Caroli’s disease. The goal is to balance the benefits of early diagnosis with the costs and risks associated with screening, particularly given the limitations of available tools like MRI with cholangiopancreatography (MRCP), which has a sensitivity of 70%-85%. In general, we recommend annual cholangiocarcinoma screening for high-risk individuals with MRI and MRCP as well as with cancer antigen (CA) 19-9. .
Screening in Patients with Primary Sclerosing Cholangitis
The lifetime risk of cholangiocarcinoma in patients with PSC is 10%-15% with an annual risk of 0.5%-1.5%. In our experience, this is often the most feared complication for PSC patients, even more so than the risk of liver transplantation. We recommend annual MRI with MRCP in addition to CA 19-9 for patients with PSC in the first decade of their diagnosis, as most cancers are diagnosed during this period. If a patient’s imaging has remained stable for over a decade and there is minimal hepatic fibrosis, we discuss the option of reducing screening frequency to every 2 years to minimize costs and exposure to MRI contrast risks.
If MRI reveals a concerning new large duct stricture, we will evaluate this with an endoscopic retrograde cholangiopancreatography (ERCP), as differentiating benign and malignant strictures is quite challenging with MRI. We generally recommend ERCP with brush cytology and fluorescence in situ hybridization to improve diagnostic yield. Depending on imaging findings and location of the new large duct stricture, we may consider cholangioscopy during ERCP for direct visualization of the bile duct and directed tissue biopsies. Unfortunately, even in young, asymptomatic patients who undergo regular screening, cholangiocarcinoma is frequently diagnosed at an advanced stage.
Screening in Patients with Secondary Sclerosing Cholangitis
Patients with SSC may develop cholangiocarcinoma because of chronic inflammatory and fibrotic processes, such as IgG4-associated cholangiopathy, sarcoidosis, ischemic cholangiopathy, cystic fibrosis, recurrent pyogenic cholangitis, severe sepsis (as recently seen from SARS-CoV-2), surgical complications, or other etiologies. When the condition is reversible, such as with IgG4-associated cholangiopathy, cancer screening may not be necessary. However, when irreversible damage occurs, the cancer risk increases, though it varies by disease type and severity. In most cases, we recommend routine screening for cholangiocarcinoma with MRI and CA 19-9 in this population.
Screening in Patients with Biliary Tract Disorders
Biliary tract disorders such as choledochal cysts and Caroli’s disease also harbor an increased risk of cholangiocarcinoma. Choledochal cysts are congenital cystic dilations of the bile duct that have a 10%-30% lifetime risk of malignant transformation to cholangiocarcinoma. Surgical intervention to remove the cyst is often recommended because of this high risk. However, some patients may be unable or unwilling to undergo this surgery or they may have residual cysts. We recommend ongoing screening with MRI and CA 19-9 for these patients. Similarly, Caroli’s disease is a congenital disease associated with intrahepatic and extrahepatic bile duct cysts and associated with a 5%-15% lifetime risk of cholangiocarcinoma. MRI with MRCP and CA 19-9 should be performed routinely for patients with Caroli’s disease and syndrome.
Risks and Challenges in Cholangiocarcinoma Screening
While MRI with MRCP is the gold standard for cholangiocarcinoma screening, its limitations must be carefully considered. One growing concern is the potential for gadolinium retention in the brain, bones, or skin following repeated MRI scans. Though the long-term effects of gadolinium retention are not fully understood, we factor this into screening decisions, particularly for younger patients who may undergo decades of regular imaging.
MRI is not always feasible for certain patients, including those with metal implants, on hemodialysis, or with severe allergic reactions. In such cases, CT or ultrasound may serve as alternatives, though with lower sensitivity for detecting cholangiocarcinoma. Additionally, claustrophobia during MRI can be addressed with sedation, but this underscores the importance of shared decision-making.
From our perspective, cholangiocarcinoma screening in high-risk patients is crucial but not without challenges. Our current screening methods, while essential, are far from perfect, often missing early cancers or leading to unnecessary interventions. Because of these limitations, the window for treatment of localized disease can easily be missed. In our practice, we tailor screening strategies to each patient’s specific needs, weighing the potential benefits against the risks, costs, and the inherent uncertainty of early detection tools. We believe it is essential to involve patients in this decision-making process to provide a balanced, individualized approach that considers both clinical evidence and the personal preferences of each person.
Dr. Kupferman is a gastroenterology fellow at Stanford University School of Medicine in California. Dr. Goel is a transplant hepatologist and a clinical associate professor in gastroenterology & hepatology at Stanford.
References
1. Vithayathil M and Khan SA. J Hepatol. 2022 Dec. doi: 10.1016/j.jhep.2022.07.022.
2. Patel N and Benipal B. Cureus. 2019 Jan. doi: 10.7759/cureus.3962.
Dear colleagues,
As gastroenterologists and endoscopists, we spend significant time preventing and diagnosing GI malignancies.
For instance, is it worthwhile screening for pancreatic cancer, and, if so, how should this be done? Likewise, diagnosing cholangiocarcinoma is challenging; how best should one evaluate for this in higher risk populations, such as primary sclerosing cholangitis? And what about the costs, financial and otherwise, associated with screening?
In this issue of Perspectives, Dr. Darshan Kothari and Dr. Daniel Bernstein discuss their approach to pancreatic cancer screening, including who is eligible, the preferred screening modalities, and the barriers to screening. In the accompanying perspective, Dr. Aparna Goel and Dr. Judah Kupferman focus on cholangiocarcinoma screening, identifying high-risk populations and discussing some of the concerns with screening, necessitating shared decision-making.
We welcome your thoughts on this issue. Share with us on X at @AGA_GIHN.
Gyanprakash A. Ketwaroo, MD, MSc, is associate professor of medicine, Yale University, New Haven, and chief of endoscopy at West Haven VA Medical Center, both in Connecticut. He is an associate editor for GI & Hepatology News.
An Approach to Pancreatic Cancer Screening
BY DANIEL A. BERNSTEIN, MD, AND DARSHAN KOTHARI, MD
Pancreatic cancer carries a dismal prognosis, now accounting for the third-most cancer-related mortality in the United States. A small proportion of patients are diagnosed at a local stage of disease, with over half found to have metastatic disease at presentation. Given the low overall incidence and lifetime risk in the general population, population-based screening is not justified.
About 10% of cases of pancreas cancer are associated with germ-line mutations and/or with a strong family history of pancreatic cancer. Several academic societies and expert committees now recommend regular screening for pancreatic cancer in patients who are considered high-risk individuals, as they carry a fivefold relative risk for pancreatic cancer. Moreover, studies suggest that screening has the potential to identify early-stage resectable disease and decrease mortality in this patient population.
Patients who benefit from pancreatic cancer screening are those who carry an increased lifetime risk (in excess of 5%) of pancreatic cancer. High-risk individuals include those with germ-line mutations and/or those with a family history of pancreatic cancer in first-degree relatives. Consensus guidelines by the International Cancer of the Pancreas Screening Consortium and the American Society for Gastrointestinal Endoscopy provide medical centers with detailed recommendations on who and when to start screening.
High-risk individuals fall into three categories:
- Patients with high-risk germline mutations including: familial atypical multiple mole melanoma syndrome (CDKN2A), hereditary breast and ovarian cancer syndromes (BRCA1, BRCA2, and PALB2), Peutz-Jeghers syndrome (STK11), and hereditary pancreatitis (PRSS1 and SPINK1)
- Patients with low- to moderate-risk germ-line mutations with at least one first-degree relative with pancreatic cancer: Lynch Syndrome (particularly MLH1 mutation), ataxia-telangiectasia (ATM), or Li-Fraumeni syndrome (p53)
- Patients with one first-degree relative with pancreatic cancer who in turn has one first-degree relative with pancreatic cancer (eg, a patient’s mother and maternal aunt or a patient’s father and patient’s sister)
Consistent with established guidelines, we recommend screening for high-risk patients beginning at age 50, or 10 years before the youngest age at which pancreas cancer was diagnosed in an affected relative. Screening is recommended earlier in patients with particularly high risk: at age 40 for patients with CDKN2A and STKI11 mutations and age 40 for patients with PRSS1 mutation or 20 years after the first attack of acute pancreatitis. For patients with a strong family history of pancreas cancer, we recommend comprehensive evaluation by a certified genetic counselor at a high-volume cancer center.
In practice, patients at our institution who are identified as high risk based on the above criteria are referred for an initial consultation at our pancreas center. In most cases, this should occur no sooner than 5 years prior to the recommended starting age for screening. All patients who are identified as high risk should be screened annually for diabetes given the growing evidence base supporting an association between new-onset diabetes and pancreatic cancer.
After an initial visit and discussion of the risks and benefits of screening, most screening protocols start with a baseline endoscopic ultrasound (EUS) and contrast-enhanced magnetic resonance abdomen with magnetic resonance cholangiopancreatography (MRI/MRCP), which will be repeated annually or sooner as the clinical condition warrants. A sooner-interval EUS should be considered for patients already undergoing screening who are newly found to have diabetes.
At our institution, we start with an in-person clinic evaluation followed by EUS. Thereafter, patients undergo MRI/MRCP (synchronized with a same-day clinic visit) alternating with EUS every 6 months to ensure patients are seen twice a year, though there is no specific data to support this approach. Non-diabetics also undergo yearly diabetes screening which will trigger an EUS if patients become diabetic.
We engage in shared decision-making with our high-risk individuals undergoing pancreatic cancer screening and at each visit we review their concurrent medical conditions and suitability to continue screening. We consider discontinuing screening after age 75, at the onset of any life-limiting illness, or after a discussion of risks and benefits if comorbidities lead to a substantial deterioration in a patient’s overall health status.
While a growing body of evidence exists to support the application of pancreatic cancer screening in high-risk individuals, this preventive service remains underutilized. Recent analysis of the screening cohort at our institution showed a demographically homogeneous group of mostly highly educated, high-income White females. These findings are consistent with the patient cohorts described in other pancreatic cancer screening programs and represent only a fraction of people who would qualify for pancreatic cancer screening.
A survey of patients undergoing screening at our institution identified cost, travel, and time associated with pancreatic cancer screening to be frequent challenges to participation. Further studies are needed to fully explore the barriers and psychological burden of pancreas cancer screening in high-risk individuals, and to identify ways to enrich the cohort of patients undergoing screening. This may involve novel methods to identify family members of patients with a new diagnosis of pancreas cancer and increasing health literacy around pancreatic cancer screening among patients and providers.
Pancreatic cancer screening has the potential to identify early-stage disease in patients who are at high risk because of germ-line mutations and/or family history. We recommend that patients engage in pancreatic cancer screening at high-volume centers with well-supported oncology, genetics, and research infrastructure.
Dr. Bernstein is a gastroenterology fellow at Duke University School of Medicine, Durham, North Carolina. Dr. Kothari is an associate professor of medicine in gastroenterology and hepatology at Duke University School of Medicine.
Screening for Cholangiocarcinoma
BY JUDAH KUPFERMAN, MD, AND APARNA GOEL, MD
Cholangiocarcinoma is a rare but aggressive cancer of the bile ducts that poses many diagnostic challenges. Approximately 3% of gastrointestinal cancers are attributed to cholangiocarcinoma, and while the annual incidence of disease in the United States is about 1.26 per 100,000 people, the incidence of intrahepatic disease has been rising considerably.1,2 Screening for cholangiocarcinoma is reserved for high-risk individuals — such as those with primary sclerosing cholangitis (PSC), secondary sclerosing cholangitis (SSC), and biliary tract disorders such as choledochal cysts or Caroli’s disease. The goal is to balance the benefits of early diagnosis with the costs and risks associated with screening, particularly given the limitations of available tools like MRI with cholangiopancreatography (MRCP), which has a sensitivity of 70%-85%. In general, we recommend annual cholangiocarcinoma screening for high-risk individuals with MRI and MRCP as well as with cancer antigen (CA) 19-9. .
Screening in Patients with Primary Sclerosing Cholangitis
The lifetime risk of cholangiocarcinoma in patients with PSC is 10%-15% with an annual risk of 0.5%-1.5%. In our experience, this is often the most feared complication for PSC patients, even more so than the risk of liver transplantation. We recommend annual MRI with MRCP in addition to CA 19-9 for patients with PSC in the first decade of their diagnosis, as most cancers are diagnosed during this period. If a patient’s imaging has remained stable for over a decade and there is minimal hepatic fibrosis, we discuss the option of reducing screening frequency to every 2 years to minimize costs and exposure to MRI contrast risks.
If MRI reveals a concerning new large duct stricture, we will evaluate this with an endoscopic retrograde cholangiopancreatography (ERCP), as differentiating benign and malignant strictures is quite challenging with MRI. We generally recommend ERCP with brush cytology and fluorescence in situ hybridization to improve diagnostic yield. Depending on imaging findings and location of the new large duct stricture, we may consider cholangioscopy during ERCP for direct visualization of the bile duct and directed tissue biopsies. Unfortunately, even in young, asymptomatic patients who undergo regular screening, cholangiocarcinoma is frequently diagnosed at an advanced stage.
Screening in Patients with Secondary Sclerosing Cholangitis
Patients with SSC may develop cholangiocarcinoma because of chronic inflammatory and fibrotic processes, such as IgG4-associated cholangiopathy, sarcoidosis, ischemic cholangiopathy, cystic fibrosis, recurrent pyogenic cholangitis, severe sepsis (as recently seen from SARS-CoV-2), surgical complications, or other etiologies. When the condition is reversible, such as with IgG4-associated cholangiopathy, cancer screening may not be necessary. However, when irreversible damage occurs, the cancer risk increases, though it varies by disease type and severity. In most cases, we recommend routine screening for cholangiocarcinoma with MRI and CA 19-9 in this population.
Screening in Patients with Biliary Tract Disorders
Biliary tract disorders such as choledochal cysts and Caroli’s disease also harbor an increased risk of cholangiocarcinoma. Choledochal cysts are congenital cystic dilations of the bile duct that have a 10%-30% lifetime risk of malignant transformation to cholangiocarcinoma. Surgical intervention to remove the cyst is often recommended because of this high risk. However, some patients may be unable or unwilling to undergo this surgery or they may have residual cysts. We recommend ongoing screening with MRI and CA 19-9 for these patients. Similarly, Caroli’s disease is a congenital disease associated with intrahepatic and extrahepatic bile duct cysts and associated with a 5%-15% lifetime risk of cholangiocarcinoma. MRI with MRCP and CA 19-9 should be performed routinely for patients with Caroli’s disease and syndrome.
Risks and Challenges in Cholangiocarcinoma Screening
While MRI with MRCP is the gold standard for cholangiocarcinoma screening, its limitations must be carefully considered. One growing concern is the potential for gadolinium retention in the brain, bones, or skin following repeated MRI scans. Though the long-term effects of gadolinium retention are not fully understood, we factor this into screening decisions, particularly for younger patients who may undergo decades of regular imaging.
MRI is not always feasible for certain patients, including those with metal implants, on hemodialysis, or with severe allergic reactions. In such cases, CT or ultrasound may serve as alternatives, though with lower sensitivity for detecting cholangiocarcinoma. Additionally, claustrophobia during MRI can be addressed with sedation, but this underscores the importance of shared decision-making.
From our perspective, cholangiocarcinoma screening in high-risk patients is crucial but not without challenges. Our current screening methods, while essential, are far from perfect, often missing early cancers or leading to unnecessary interventions. Because of these limitations, the window for treatment of localized disease can easily be missed. In our practice, we tailor screening strategies to each patient’s specific needs, weighing the potential benefits against the risks, costs, and the inherent uncertainty of early detection tools. We believe it is essential to involve patients in this decision-making process to provide a balanced, individualized approach that considers both clinical evidence and the personal preferences of each person.
Dr. Kupferman is a gastroenterology fellow at Stanford University School of Medicine in California. Dr. Goel is a transplant hepatologist and a clinical associate professor in gastroenterology & hepatology at Stanford.
References
1. Vithayathil M and Khan SA. J Hepatol. 2022 Dec. doi: 10.1016/j.jhep.2022.07.022.
2. Patel N and Benipal B. Cureus. 2019 Jan. doi: 10.7759/cureus.3962.
Obesity: A Social Vulnerability
Sometime in the last year or 2 I wrote that, despite my considerable reservations, I had finally come to the conclusion that the American Medical Association’s decision to designate obesity as a disease was appropriate. My rationalization was that the disease label would open more opportunities for funding obesity treatments. However, the explosive growth and popularity of glucagon-like peptide 1 (GLP-1) agonists over the last year has had me rethinking my decision to suppress my long-held reservations about the disease designation.
So, if it’s not a disease, then what should we call it? How do we explain its surge in high-income countries that began in the 1980s? While there are still some folks who see obesity as a character flaw, I think you and I as healthcare providers have difficulty explaining the increase prevalence of obesity as either global breakdown of willpower or a widespread genetic shift as the result of burst of radiation from solar flares.
However, if we want to continue our search and finger-pointing we need to have a better definition of exactly what obesity is. If we’re going to continue calling it a disease we have done a pretty sloppy job of creating diagnostic criteria. To be honest, we aren’t doing such a hot job with “long COVID” either.
A recent article in the New York Times makes it clear that I’m not the only physician who is feeling uncomfortable with this lack of diagnostic specificity.
We know that using body mass index (BMI) as a criteria is imprecise. There are healthy individuals with elevated BMIs and there are others who are carrying an unhealthy amount of fat who have normal BMIs. And, there are individuals who have what might appear to be an excess amount of fat who are fit and healthy by other criteria.
Some investigators feel that a set of measurements that includes a waist and/or hip measurement may be a more accurate way of determining visceral adipose tissue. However, this body roundness index (BRI) currently relies on a tape measurement. Until the technique can be preformed by an inexpensive and readily available scanner, the BRI cannot be considered a practical tool for determining obesity.
Dr. Francisco Rubino, the chair of metabolic and bariatric surgery at Kings College in London, England, has been quoted as saying that, “if one defines a disease inaccurately, everything that stems from that – from diagnosis to treatment to policies – will be distorted and biased.”
Denmark has been forced to relabel obesity as a risk factor because the disease designation was stressing the financial viability of their healthcare system as more and more patients were being prescribe GLP-1 agonists, sometimes off label. A rationing strategy was resulting in suboptimal treatment of a significant portion of the obese population.
Spearheaded by Dr. Rubino, a Lancet Commission composed of physicians has tasked itself to define an “evidence-based diagnosis for obesity. Instead of relying on a single metric such as the BMI or BRI, diagnosing “clinical obesity” would involve a broad array of observations including a history, physical examination, standard laboratory and additional testing, “naming signs and symptoms, organ by organ, tissue by tissue, with plausible mechanisms for each one.” In other words, treating each patient as an individual using evidence-based criteria to make a diagnosis. While likely to be time consuming, this strategy feels like a more scientific approach. I suspect once clinical obesity is more rigorously defined it could be divided into several subtypes. For example, there would be a few conditions that were genetic; Prader-Willi syndrome being the best known.
However, I think the Lancet Commission’s strategy will find that the majority of individuals who make up this half-century global surge have become clinically obese because they have been unable to adapt to the obeseogenic forces in our society, which include diet, autocentricity, and attractive sedentary forms of entertainment, to name just three.
In some cases these unfortunate individuals are more vulnerable because there were born into an economically disadvantaged situation. In other scenarios a lack of foresight and/or political will may have left individuals with no other choice but to rely on automobiles to get around. Still others may find themselves living in a nutritional desert because all of the grocery stores have closed.
I recently encountered a descriptor in a story about the Federal Emergency Management Agency which could easily be adapted to describe this large and growing subtype of individuals with clinical obesity. “Social vulnerability” is measure of how well a community can withstand external stressors that impact human health. For example, the emergency management folks are thinking in terms of natural disaster such as hurricanes, floods, and tornadoes and are asking how well a given community can meet the challenges one would create.
But, the term social vulnerability can easily be applied to individuals living in a society in which unhealthy food is abundant, an infrastructure that discourages or outright prevents non-motorized travel, and the temptation of sedentary entertainment options is unavoidable. Fortunately, not every citizen living in an obesogenic society becomes obese. What factors have protected the non-obese individuals from these obeseogenic stressors? What are the characteristics of the unfortunate “vulnerables” living in the same society who end up being obese?
It is time to shift our focus away from a poorly defined disease model to one in which we begin looking at our society to find out why we have so many socially vulnerable individuals. The toll of obesity as it is currently defined is many order of magnitudes greater than any natural disaster. We have become communities that can no longer withstand the its obesogenic stressors many of which we have created and/or allowed to accumulate over the last century.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
Sometime in the last year or 2 I wrote that, despite my considerable reservations, I had finally come to the conclusion that the American Medical Association’s decision to designate obesity as a disease was appropriate. My rationalization was that the disease label would open more opportunities for funding obesity treatments. However, the explosive growth and popularity of glucagon-like peptide 1 (GLP-1) agonists over the last year has had me rethinking my decision to suppress my long-held reservations about the disease designation.
So, if it’s not a disease, then what should we call it? How do we explain its surge in high-income countries that began in the 1980s? While there are still some folks who see obesity as a character flaw, I think you and I as healthcare providers have difficulty explaining the increase prevalence of obesity as either global breakdown of willpower or a widespread genetic shift as the result of burst of radiation from solar flares.
However, if we want to continue our search and finger-pointing we need to have a better definition of exactly what obesity is. If we’re going to continue calling it a disease we have done a pretty sloppy job of creating diagnostic criteria. To be honest, we aren’t doing such a hot job with “long COVID” either.
A recent article in the New York Times makes it clear that I’m not the only physician who is feeling uncomfortable with this lack of diagnostic specificity.
We know that using body mass index (BMI) as a criteria is imprecise. There are healthy individuals with elevated BMIs and there are others who are carrying an unhealthy amount of fat who have normal BMIs. And, there are individuals who have what might appear to be an excess amount of fat who are fit and healthy by other criteria.
Some investigators feel that a set of measurements that includes a waist and/or hip measurement may be a more accurate way of determining visceral adipose tissue. However, this body roundness index (BRI) currently relies on a tape measurement. Until the technique can be preformed by an inexpensive and readily available scanner, the BRI cannot be considered a practical tool for determining obesity.
Dr. Francisco Rubino, the chair of metabolic and bariatric surgery at Kings College in London, England, has been quoted as saying that, “if one defines a disease inaccurately, everything that stems from that – from diagnosis to treatment to policies – will be distorted and biased.”
Denmark has been forced to relabel obesity as a risk factor because the disease designation was stressing the financial viability of their healthcare system as more and more patients were being prescribe GLP-1 agonists, sometimes off label. A rationing strategy was resulting in suboptimal treatment of a significant portion of the obese population.
Spearheaded by Dr. Rubino, a Lancet Commission composed of physicians has tasked itself to define an “evidence-based diagnosis for obesity. Instead of relying on a single metric such as the BMI or BRI, diagnosing “clinical obesity” would involve a broad array of observations including a history, physical examination, standard laboratory and additional testing, “naming signs and symptoms, organ by organ, tissue by tissue, with plausible mechanisms for each one.” In other words, treating each patient as an individual using evidence-based criteria to make a diagnosis. While likely to be time consuming, this strategy feels like a more scientific approach. I suspect once clinical obesity is more rigorously defined it could be divided into several subtypes. For example, there would be a few conditions that were genetic; Prader-Willi syndrome being the best known.
However, I think the Lancet Commission’s strategy will find that the majority of individuals who make up this half-century global surge have become clinically obese because they have been unable to adapt to the obeseogenic forces in our society, which include diet, autocentricity, and attractive sedentary forms of entertainment, to name just three.
In some cases these unfortunate individuals are more vulnerable because there were born into an economically disadvantaged situation. In other scenarios a lack of foresight and/or political will may have left individuals with no other choice but to rely on automobiles to get around. Still others may find themselves living in a nutritional desert because all of the grocery stores have closed.
I recently encountered a descriptor in a story about the Federal Emergency Management Agency which could easily be adapted to describe this large and growing subtype of individuals with clinical obesity. “Social vulnerability” is measure of how well a community can withstand external stressors that impact human health. For example, the emergency management folks are thinking in terms of natural disaster such as hurricanes, floods, and tornadoes and are asking how well a given community can meet the challenges one would create.
But, the term social vulnerability can easily be applied to individuals living in a society in which unhealthy food is abundant, an infrastructure that discourages or outright prevents non-motorized travel, and the temptation of sedentary entertainment options is unavoidable. Fortunately, not every citizen living in an obesogenic society becomes obese. What factors have protected the non-obese individuals from these obeseogenic stressors? What are the characteristics of the unfortunate “vulnerables” living in the same society who end up being obese?
It is time to shift our focus away from a poorly defined disease model to one in which we begin looking at our society to find out why we have so many socially vulnerable individuals. The toll of obesity as it is currently defined is many order of magnitudes greater than any natural disaster. We have become communities that can no longer withstand the its obesogenic stressors many of which we have created and/or allowed to accumulate over the last century.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
Sometime in the last year or 2 I wrote that, despite my considerable reservations, I had finally come to the conclusion that the American Medical Association’s decision to designate obesity as a disease was appropriate. My rationalization was that the disease label would open more opportunities for funding obesity treatments. However, the explosive growth and popularity of glucagon-like peptide 1 (GLP-1) agonists over the last year has had me rethinking my decision to suppress my long-held reservations about the disease designation.
So, if it’s not a disease, then what should we call it? How do we explain its surge in high-income countries that began in the 1980s? While there are still some folks who see obesity as a character flaw, I think you and I as healthcare providers have difficulty explaining the increase prevalence of obesity as either global breakdown of willpower or a widespread genetic shift as the result of burst of radiation from solar flares.
However, if we want to continue our search and finger-pointing we need to have a better definition of exactly what obesity is. If we’re going to continue calling it a disease we have done a pretty sloppy job of creating diagnostic criteria. To be honest, we aren’t doing such a hot job with “long COVID” either.
A recent article in the New York Times makes it clear that I’m not the only physician who is feeling uncomfortable with this lack of diagnostic specificity.
We know that using body mass index (BMI) as a criteria is imprecise. There are healthy individuals with elevated BMIs and there are others who are carrying an unhealthy amount of fat who have normal BMIs. And, there are individuals who have what might appear to be an excess amount of fat who are fit and healthy by other criteria.
Some investigators feel that a set of measurements that includes a waist and/or hip measurement may be a more accurate way of determining visceral adipose tissue. However, this body roundness index (BRI) currently relies on a tape measurement. Until the technique can be preformed by an inexpensive and readily available scanner, the BRI cannot be considered a practical tool for determining obesity.
Dr. Francisco Rubino, the chair of metabolic and bariatric surgery at Kings College in London, England, has been quoted as saying that, “if one defines a disease inaccurately, everything that stems from that – from diagnosis to treatment to policies – will be distorted and biased.”
Denmark has been forced to relabel obesity as a risk factor because the disease designation was stressing the financial viability of their healthcare system as more and more patients were being prescribe GLP-1 agonists, sometimes off label. A rationing strategy was resulting in suboptimal treatment of a significant portion of the obese population.
Spearheaded by Dr. Rubino, a Lancet Commission composed of physicians has tasked itself to define an “evidence-based diagnosis for obesity. Instead of relying on a single metric such as the BMI or BRI, diagnosing “clinical obesity” would involve a broad array of observations including a history, physical examination, standard laboratory and additional testing, “naming signs and symptoms, organ by organ, tissue by tissue, with plausible mechanisms for each one.” In other words, treating each patient as an individual using evidence-based criteria to make a diagnosis. While likely to be time consuming, this strategy feels like a more scientific approach. I suspect once clinical obesity is more rigorously defined it could be divided into several subtypes. For example, there would be a few conditions that were genetic; Prader-Willi syndrome being the best known.
However, I think the Lancet Commission’s strategy will find that the majority of individuals who make up this half-century global surge have become clinically obese because they have been unable to adapt to the obeseogenic forces in our society, which include diet, autocentricity, and attractive sedentary forms of entertainment, to name just three.
In some cases these unfortunate individuals are more vulnerable because there were born into an economically disadvantaged situation. In other scenarios a lack of foresight and/or political will may have left individuals with no other choice but to rely on automobiles to get around. Still others may find themselves living in a nutritional desert because all of the grocery stores have closed.
I recently encountered a descriptor in a story about the Federal Emergency Management Agency which could easily be adapted to describe this large and growing subtype of individuals with clinical obesity. “Social vulnerability” is measure of how well a community can withstand external stressors that impact human health. For example, the emergency management folks are thinking in terms of natural disaster such as hurricanes, floods, and tornadoes and are asking how well a given community can meet the challenges one would create.
But, the term social vulnerability can easily be applied to individuals living in a society in which unhealthy food is abundant, an infrastructure that discourages or outright prevents non-motorized travel, and the temptation of sedentary entertainment options is unavoidable. Fortunately, not every citizen living in an obesogenic society becomes obese. What factors have protected the non-obese individuals from these obeseogenic stressors? What are the characteristics of the unfortunate “vulnerables” living in the same society who end up being obese?
It is time to shift our focus away from a poorly defined disease model to one in which we begin looking at our society to find out why we have so many socially vulnerable individuals. The toll of obesity as it is currently defined is many order of magnitudes greater than any natural disaster. We have become communities that can no longer withstand the its obesogenic stressors many of which we have created and/or allowed to accumulate over the last century.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
Preventing Pediatric Migraine
I suspect you all have some experience with childhood migraine. It can mean a painful several hours for the patient, arriving often without warning, with recurrences spaced months or sometimes even years apart. It may be accompanied by vomiting, which in some cases overshadows the severity of the headache. It can result in lost days from school and ruin family activities. It can occur so infrequently that the family can’t recall accurately when the last episode happened. In some ways it is a different animal than the adult version.
Most of the pediatric patients with migraine I have seen have experienced attacks that were occurring so infrequently that the families and I seldom discussed medication as an option. Back then imipramine was the only choice. However, currently there are more than a half dozen medications and combinations that have been tried. Recently a review of 45 clinical trials of these medications was published in JAMA Network Open.
I will let you review for yourself the details of these Iranian investigators’ network meta-analysis, but the bottom line is that some medications were associated with a reduction in migraine frequency. Others were associated with headache intensity. “However, no treatments were associated with significant improvements in quality of life or reduction of the duration of migraine attacks.”
Obviously, this paper illustrates clearly that we have not yet discovered the medicinal magic bullet for pediatric migraine prophylaxis. This doesn’t surprise me. After listening to scores of families tell their migraine stories, it became apparent to me that there was often a pattern in which the child’s headache had arrived after a period of acute sleep deprivation. For example, a trip to an amusement park in which travel or excitement may have resulted in the child going to bed later and/or getting up earlier. By afternoon the child’s reserves of something (currently unknown) were depleted to a point that the headache and/or vomiting struck.
Because these episodes were often so infrequent, separated by months, that taking a history demonstrating a recurring pattern could take considerable patience on the part of the family and the provider, even for a physician like myself who believes that better sleep is the answer for everything. However, once I could convince a family of the connection between the sleep deprivation and the headaches, they could often recall other episodes in the past that substantiated my explanation.
In some cases there was no obvious history of acute sleep deprivation, or at least it was so subtle that even a history taker with a sleep obsession couldn’t detect it. However, in these cases I could usually elicit a history of chronic sleep deprivation. For example, falling asleep instantly on automobile rides, difficulty with waking in the morning, or unhealthy bedtime routines. With this underlying vulnerability of chronic sleep deprivation, a slightly more exciting or vigorous day was all that was necessary to trigger the headache.
For those of you who don’t share my contention that childhood migraine is usually the result of sleep deprivation, consider the similarity between an epileptic seizure, which can be triggered by fatigue. Both events are usually followed by a deep sleep from which the child wakes refreshed and symptom free.
I think it is interesting that this recent meta-analysis could find no benefit in the quality of life for any of the medications. The explanation may be that the child with migraine already had a somewhat diminished quality of life as a result of the sleep deprivation, either acute or chronic.
When speaking with parents of migraine sufferers, I would tell them that once the headache had started there was little I had to offer to forestall the inevitable pain and vomiting. Certainly not in the form of an oral medication. While many adults will have an aura that warns them of the headache onset, I have found that most children don’t describe an aura. It may be they simply lack the ability to express it. Occasionally an observant parent may detect pallor or a behavior change that indicates a migraine is beginning. On rare occasions a parent may be able to abort the attack by quickly getting the child to a quiet, dark, and calm environment.
Although this recent meta-analysis review of treatment options is discouraging, it may be providing a clue to effective prophylaxis. Some of the medications that decrease the frequency of the attacks may be doing so because they improve the patient’s sleep patterns. Those that decrease the intensity of the pain are probably working on pain pathway that is not specific to migraine.
Continuing a search for a prophylactic medication is a worthy goal, particularly for those patients in which their migraines are debilitating. However, based on my experience, enhanced by my bias, the safest and most effective prophylaxis results from increasing the family’s awareness of the role that sleep deprivation plays in the illness. Even when the family buys into the message and attempts to avoid situations that will tax their vulnerable children, parents will need to accept that sometimes stuff happens even though siblings and peers may be able to tolerate the situation. Spontaneous activities can converge on a day when for whatever reason the migraine-prone child is overtired and the headache and vomiting will erupt.
A lifestyle change is always preferable to a pharmacological intervention. However, that doesn’t mean it is always easy to achieve.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
I suspect you all have some experience with childhood migraine. It can mean a painful several hours for the patient, arriving often without warning, with recurrences spaced months or sometimes even years apart. It may be accompanied by vomiting, which in some cases overshadows the severity of the headache. It can result in lost days from school and ruin family activities. It can occur so infrequently that the family can’t recall accurately when the last episode happened. In some ways it is a different animal than the adult version.
Most of the pediatric patients with migraine I have seen have experienced attacks that were occurring so infrequently that the families and I seldom discussed medication as an option. Back then imipramine was the only choice. However, currently there are more than a half dozen medications and combinations that have been tried. Recently a review of 45 clinical trials of these medications was published in JAMA Network Open.
I will let you review for yourself the details of these Iranian investigators’ network meta-analysis, but the bottom line is that some medications were associated with a reduction in migraine frequency. Others were associated with headache intensity. “However, no treatments were associated with significant improvements in quality of life or reduction of the duration of migraine attacks.”
Obviously, this paper illustrates clearly that we have not yet discovered the medicinal magic bullet for pediatric migraine prophylaxis. This doesn’t surprise me. After listening to scores of families tell their migraine stories, it became apparent to me that there was often a pattern in which the child’s headache had arrived after a period of acute sleep deprivation. For example, a trip to an amusement park in which travel or excitement may have resulted in the child going to bed later and/or getting up earlier. By afternoon the child’s reserves of something (currently unknown) were depleted to a point that the headache and/or vomiting struck.
Because these episodes were often so infrequent, separated by months, that taking a history demonstrating a recurring pattern could take considerable patience on the part of the family and the provider, even for a physician like myself who believes that better sleep is the answer for everything. However, once I could convince a family of the connection between the sleep deprivation and the headaches, they could often recall other episodes in the past that substantiated my explanation.
In some cases there was no obvious history of acute sleep deprivation, or at least it was so subtle that even a history taker with a sleep obsession couldn’t detect it. However, in these cases I could usually elicit a history of chronic sleep deprivation. For example, falling asleep instantly on automobile rides, difficulty with waking in the morning, or unhealthy bedtime routines. With this underlying vulnerability of chronic sleep deprivation, a slightly more exciting or vigorous day was all that was necessary to trigger the headache.
For those of you who don’t share my contention that childhood migraine is usually the result of sleep deprivation, consider the similarity between an epileptic seizure, which can be triggered by fatigue. Both events are usually followed by a deep sleep from which the child wakes refreshed and symptom free.
I think it is interesting that this recent meta-analysis could find no benefit in the quality of life for any of the medications. The explanation may be that the child with migraine already had a somewhat diminished quality of life as a result of the sleep deprivation, either acute or chronic.
When speaking with parents of migraine sufferers, I would tell them that once the headache had started there was little I had to offer to forestall the inevitable pain and vomiting. Certainly not in the form of an oral medication. While many adults will have an aura that warns them of the headache onset, I have found that most children don’t describe an aura. It may be they simply lack the ability to express it. Occasionally an observant parent may detect pallor or a behavior change that indicates a migraine is beginning. On rare occasions a parent may be able to abort the attack by quickly getting the child to a quiet, dark, and calm environment.
Although this recent meta-analysis review of treatment options is discouraging, it may be providing a clue to effective prophylaxis. Some of the medications that decrease the frequency of the attacks may be doing so because they improve the patient’s sleep patterns. Those that decrease the intensity of the pain are probably working on pain pathway that is not specific to migraine.
Continuing a search for a prophylactic medication is a worthy goal, particularly for those patients in which their migraines are debilitating. However, based on my experience, enhanced by my bias, the safest and most effective prophylaxis results from increasing the family’s awareness of the role that sleep deprivation plays in the illness. Even when the family buys into the message and attempts to avoid situations that will tax their vulnerable children, parents will need to accept that sometimes stuff happens even though siblings and peers may be able to tolerate the situation. Spontaneous activities can converge on a day when for whatever reason the migraine-prone child is overtired and the headache and vomiting will erupt.
A lifestyle change is always preferable to a pharmacological intervention. However, that doesn’t mean it is always easy to achieve.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
I suspect you all have some experience with childhood migraine. It can mean a painful several hours for the patient, arriving often without warning, with recurrences spaced months or sometimes even years apart. It may be accompanied by vomiting, which in some cases overshadows the severity of the headache. It can result in lost days from school and ruin family activities. It can occur so infrequently that the family can’t recall accurately when the last episode happened. In some ways it is a different animal than the adult version.
Most of the pediatric patients with migraine I have seen have experienced attacks that were occurring so infrequently that the families and I seldom discussed medication as an option. Back then imipramine was the only choice. However, currently there are more than a half dozen medications and combinations that have been tried. Recently a review of 45 clinical trials of these medications was published in JAMA Network Open.
I will let you review for yourself the details of these Iranian investigators’ network meta-analysis, but the bottom line is that some medications were associated with a reduction in migraine frequency. Others were associated with headache intensity. “However, no treatments were associated with significant improvements in quality of life or reduction of the duration of migraine attacks.”
Obviously, this paper illustrates clearly that we have not yet discovered the medicinal magic bullet for pediatric migraine prophylaxis. This doesn’t surprise me. After listening to scores of families tell their migraine stories, it became apparent to me that there was often a pattern in which the child’s headache had arrived after a period of acute sleep deprivation. For example, a trip to an amusement park in which travel or excitement may have resulted in the child going to bed later and/or getting up earlier. By afternoon the child’s reserves of something (currently unknown) were depleted to a point that the headache and/or vomiting struck.
Because these episodes were often so infrequent, separated by months, that taking a history demonstrating a recurring pattern could take considerable patience on the part of the family and the provider, even for a physician like myself who believes that better sleep is the answer for everything. However, once I could convince a family of the connection between the sleep deprivation and the headaches, they could often recall other episodes in the past that substantiated my explanation.
In some cases there was no obvious history of acute sleep deprivation, or at least it was so subtle that even a history taker with a sleep obsession couldn’t detect it. However, in these cases I could usually elicit a history of chronic sleep deprivation. For example, falling asleep instantly on automobile rides, difficulty with waking in the morning, or unhealthy bedtime routines. With this underlying vulnerability of chronic sleep deprivation, a slightly more exciting or vigorous day was all that was necessary to trigger the headache.
For those of you who don’t share my contention that childhood migraine is usually the result of sleep deprivation, consider the similarity between an epileptic seizure, which can be triggered by fatigue. Both events are usually followed by a deep sleep from which the child wakes refreshed and symptom free.
I think it is interesting that this recent meta-analysis could find no benefit in the quality of life for any of the medications. The explanation may be that the child with migraine already had a somewhat diminished quality of life as a result of the sleep deprivation, either acute or chronic.
When speaking with parents of migraine sufferers, I would tell them that once the headache had started there was little I had to offer to forestall the inevitable pain and vomiting. Certainly not in the form of an oral medication. While many adults will have an aura that warns them of the headache onset, I have found that most children don’t describe an aura. It may be they simply lack the ability to express it. Occasionally an observant parent may detect pallor or a behavior change that indicates a migraine is beginning. On rare occasions a parent may be able to abort the attack by quickly getting the child to a quiet, dark, and calm environment.
Although this recent meta-analysis review of treatment options is discouraging, it may be providing a clue to effective prophylaxis. Some of the medications that decrease the frequency of the attacks may be doing so because they improve the patient’s sleep patterns. Those that decrease the intensity of the pain are probably working on pain pathway that is not specific to migraine.
Continuing a search for a prophylactic medication is a worthy goal, particularly for those patients in which their migraines are debilitating. However, based on my experience, enhanced by my bias, the safest and most effective prophylaxis results from increasing the family’s awareness of the role that sleep deprivation plays in the illness. Even when the family buys into the message and attempts to avoid situations that will tax their vulnerable children, parents will need to accept that sometimes stuff happens even though siblings and peers may be able to tolerate the situation. Spontaneous activities can converge on a day when for whatever reason the migraine-prone child is overtired and the headache and vomiting will erupt.
A lifestyle change is always preferable to a pharmacological intervention. However, that doesn’t mean it is always easy to achieve.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at pdnews@mdedge.com.
Just Call It ‘Chronic Rhinitis’ and Reach for These Treatments
This transcript has been edited for clarity.
Matthew F. Watto, MD: I’m here with my great friend and America’s primary care physician, Dr. Paul Nelson Williams. Paul, are you ready to talk about rhinitis?
Paul N. Williams, MD: I’m excited. It’s always the season to talk about rhinitis.
Watto: We had a great guest for this podcast, Rhinitis and Environmental Allergies with Dr. Olajumoke Fadugba from Penn Medicine. She’s an allergist and immunologist. One of her pet peeves is when people just call everything “allergic rhinitis” because we should be calling it “chronic rhinitis,” if it’s chronic. That’s an umbrella term, and there are many buckets underneath it that people could fall into.
When you’re taking a history, you have to figure out whether it’s perennial (meaning it happens year round) because certain things can cause that. Cat dander is around all the time, so people with cats might have sinus symptoms all year. Dust mites are another one, and it’s pretty hard to avoid those. Those are some perennial allergens.
Then there is allergic vs nonallergic rhinitis, which is something I hadn’t really put too much thought into.
Williams: I didn’t realize exactly how nuanced it got. Nonallergic rhinitis can still be seasonal because changes in temperature and humidity can trigger the rhinitis. And it matters what medications you use for what.
Watto: Here are some ways you can try to figure out if rhinitis is allergic or nonallergic. Ask the patient if they have itchy eyes and are sneezing a lot. That can be more of an allergic rhinitis, but both allergic and nonallergic rhinitis have the congestion, the rhinorrhea, so you can’t figure it out based on that alone.
Dr. Fadugba said that one clue that it might be nonallergic rhinitis is the age of onset. If the symptoms are later in onset (older age), then 30%-40% of rhinitis is nonallergic. If the patient has never had allergies and now all of a sudden they have new chronic sinus symptoms, it’s probably nonallergic rhinitis. It’s a diagnosis of exclusion.
I guess they need allergy testing?
Williams: If you want to make a definitive diagnosis, you need to rule it out. I suspect that you might be able to get away with some empirical treatment. If they get better, you can feel like a winner because getting booked in for allergy testing can be a little bit of a challenge.
Watto: The main treatment difference is that the oral antihistamines do not really seem to work for nonallergic rhinitis, but they can help with allergic rhinitis. Weirdly, the nasal antihistamines and nasal steroids do seem to work for both allergic and nonallergic rhinitis.
I don’t understand the mechanism there, but if you think someone might have nonallergic rhinitis, I wouldn’t go with the oral antihistamines as your first-line treatment. I would go with a nasal spray; you pretty much can’t go wrong with either an antihistamine or a steroid nasal spray.
Williams: We typically start with the nasal sprays. That’s kind of first-line for almost everybody, allergic or nonallergic. You’re probably going to start with an intranasal steroid, and then it’s kind of dealer’s choice what the patient can tolerate and afford. Sometimes you can get them covered by insurance, at least in my experience.
I will say that this is one of the medications — like nicotine patches and other things — where we as doctors don’t really counsel patients on how to use it appropriately. So with our expert, we revisited the idea of the patient pointing the nasal spray laterally, toward their ear basically, and not spraying toward their brain. There should not be a slurping sound afterward, because “if you taste it, you waste it,” as the allergists and immunologists say. It’s supposed to sit up there and not be swallowed immediately.
If your patient is sensitive to the floral flavor of some of the fluticasones (which I don’t mind so much as a user myself), then you can try mometasone or the other formulations. They are all roughly equivalent.
Speaking of medications, which medications can cause rhinitis? Any meds we commonly use in primary care?
Williams: Apparently the combined hormonal oral contraceptives can do it. Also the phosphodiesterase 5 (PDE-5) inhibitors. Drugs that cause vasodilation can also do it. Some of the antihypertensives. I’ve seen beta-blockers and angiotensin-converting enzyme (ACE) inhibitors listed specifically, and some of the medications for benign prostatic hyperplasia (BPH). So there are a couple of medications that you can think about as a potential cause of rhinitis, although my suspicion is not going to be as high as for some of the other causes.
Watto: We mentioned medication treatments for patients who are really bothered by rhinorrhea, and maybe they are already on a steroid or an antihistamine.
You can try nasal ipratropium for people that have really prominent rhinorrhea. Dr. Fadugba said that can work well, and it’s usually taken three or four times a day. I’ve had good success prescribing it for my patients. Another one that I have never prescribed, but that Dr. Fadugba said is available over the counter, is intranasal cromolyn — a mast cell stabilizer. She said it can be beneficial.
Let’s say I had a cat allergy and I was going to visit Paul. I could use the intranasal cromolyn ahead of time to reduce rhinitis when I’m around the cats.
Paul, what about montelukast? I never know what to do with that one.
Williams: I’ve seen it prescribed as a last-ditch attempt to fix chronic rhinitis. Dr. Fadugba said she only ever prescribes it for patients who have rhinitis symptoms and asthma and never just for chronic rhinitis because it doesn’t work. And also, there have been some new black-box warnings from the US Food and Drug Administration (FDA). So unless there’s a solid indication for it, montelukast is not something you should just prescribe to try to see if it will work. That’s probably not the right approach for this.
But if the patient has challenging control asthma, and as a component, challenging nasal symptoms as well, it might be a reasonable medication to try.
Watto: And finally, Paul, how does climate change possibly have anything to do with rhinitis?
Williams: I feel like I’m just seeing more and more of the stuff every year. I don’t know if I’m more sensitive to it or because I’m having more symptoms myself, but it turns out the prevalence actually is going up.
We’re seeing more of it in part because it’s getting hotter outside, which is in turn worsening the production of allergens and increasing the allergen exposure and the severity of the symptoms that go along with it. More people are having more severe disease because the world is changing as a result of the stuff that we do. So fix that. But also be mindful and expect to see even more of these problems as you move forward in your careers.
Watto: Dr. Fadugba gave us so many great tips. You can listen to the full podcast episode here.
Dr. Watto, Clinical Assistant Professor, Department of Medicine, Perelman School of Medicine at University of Pennsylvania; Internist, Department of Medicine, Hospital Medicine Section, Pennsylvania Hospital, Philadelphia, has disclosed no relevant financial relationships. Dr. Williams, Associate Professor of Clinical Medicine, Department of General Internal Medicine, Lewis Katz School of Medicine; Staff Physician, Department of General Internal Medicine, Temple Internal Medicine Associates, Philadelphia, disclosed ties with The Curbsiders.
A version of this article first appeared on Medscape.com.
This transcript has been edited for clarity.
Matthew F. Watto, MD: I’m here with my great friend and America’s primary care physician, Dr. Paul Nelson Williams. Paul, are you ready to talk about rhinitis?
Paul N. Williams, MD: I’m excited. It’s always the season to talk about rhinitis.
Watto: We had a great guest for this podcast, Rhinitis and Environmental Allergies with Dr. Olajumoke Fadugba from Penn Medicine. She’s an allergist and immunologist. One of her pet peeves is when people just call everything “allergic rhinitis” because we should be calling it “chronic rhinitis,” if it’s chronic. That’s an umbrella term, and there are many buckets underneath it that people could fall into.
When you’re taking a history, you have to figure out whether it’s perennial (meaning it happens year round) because certain things can cause that. Cat dander is around all the time, so people with cats might have sinus symptoms all year. Dust mites are another one, and it’s pretty hard to avoid those. Those are some perennial allergens.
Then there is allergic vs nonallergic rhinitis, which is something I hadn’t really put too much thought into.
Williams: I didn’t realize exactly how nuanced it got. Nonallergic rhinitis can still be seasonal because changes in temperature and humidity can trigger the rhinitis. And it matters what medications you use for what.
Watto: Here are some ways you can try to figure out if rhinitis is allergic or nonallergic. Ask the patient if they have itchy eyes and are sneezing a lot. That can be more of an allergic rhinitis, but both allergic and nonallergic rhinitis have the congestion, the rhinorrhea, so you can’t figure it out based on that alone.
Dr. Fadugba said that one clue that it might be nonallergic rhinitis is the age of onset. If the symptoms are later in onset (older age), then 30%-40% of rhinitis is nonallergic. If the patient has never had allergies and now all of a sudden they have new chronic sinus symptoms, it’s probably nonallergic rhinitis. It’s a diagnosis of exclusion.
I guess they need allergy testing?
Williams: If you want to make a definitive diagnosis, you need to rule it out. I suspect that you might be able to get away with some empirical treatment. If they get better, you can feel like a winner because getting booked in for allergy testing can be a little bit of a challenge.
Watto: The main treatment difference is that the oral antihistamines do not really seem to work for nonallergic rhinitis, but they can help with allergic rhinitis. Weirdly, the nasal antihistamines and nasal steroids do seem to work for both allergic and nonallergic rhinitis.
I don’t understand the mechanism there, but if you think someone might have nonallergic rhinitis, I wouldn’t go with the oral antihistamines as your first-line treatment. I would go with a nasal spray; you pretty much can’t go wrong with either an antihistamine or a steroid nasal spray.
Williams: We typically start with the nasal sprays. That’s kind of first-line for almost everybody, allergic or nonallergic. You’re probably going to start with an intranasal steroid, and then it’s kind of dealer’s choice what the patient can tolerate and afford. Sometimes you can get them covered by insurance, at least in my experience.
I will say that this is one of the medications — like nicotine patches and other things — where we as doctors don’t really counsel patients on how to use it appropriately. So with our expert, we revisited the idea of the patient pointing the nasal spray laterally, toward their ear basically, and not spraying toward their brain. There should not be a slurping sound afterward, because “if you taste it, you waste it,” as the allergists and immunologists say. It’s supposed to sit up there and not be swallowed immediately.
If your patient is sensitive to the floral flavor of some of the fluticasones (which I don’t mind so much as a user myself), then you can try mometasone or the other formulations. They are all roughly equivalent.
Speaking of medications, which medications can cause rhinitis? Any meds we commonly use in primary care?
Williams: Apparently the combined hormonal oral contraceptives can do it. Also the phosphodiesterase 5 (PDE-5) inhibitors. Drugs that cause vasodilation can also do it. Some of the antihypertensives. I’ve seen beta-blockers and angiotensin-converting enzyme (ACE) inhibitors listed specifically, and some of the medications for benign prostatic hyperplasia (BPH). So there are a couple of medications that you can think about as a potential cause of rhinitis, although my suspicion is not going to be as high as for some of the other causes.
Watto: We mentioned medication treatments for patients who are really bothered by rhinorrhea, and maybe they are already on a steroid or an antihistamine.
You can try nasal ipratropium for people that have really prominent rhinorrhea. Dr. Fadugba said that can work well, and it’s usually taken three or four times a day. I’ve had good success prescribing it for my patients. Another one that I have never prescribed, but that Dr. Fadugba said is available over the counter, is intranasal cromolyn — a mast cell stabilizer. She said it can be beneficial.
Let’s say I had a cat allergy and I was going to visit Paul. I could use the intranasal cromolyn ahead of time to reduce rhinitis when I’m around the cats.
Paul, what about montelukast? I never know what to do with that one.
Williams: I’ve seen it prescribed as a last-ditch attempt to fix chronic rhinitis. Dr. Fadugba said she only ever prescribes it for patients who have rhinitis symptoms and asthma and never just for chronic rhinitis because it doesn’t work. And also, there have been some new black-box warnings from the US Food and Drug Administration (FDA). So unless there’s a solid indication for it, montelukast is not something you should just prescribe to try to see if it will work. That’s probably not the right approach for this.
But if the patient has challenging control asthma, and as a component, challenging nasal symptoms as well, it might be a reasonable medication to try.
Watto: And finally, Paul, how does climate change possibly have anything to do with rhinitis?
Williams: I feel like I’m just seeing more and more of the stuff every year. I don’t know if I’m more sensitive to it or because I’m having more symptoms myself, but it turns out the prevalence actually is going up.
We’re seeing more of it in part because it’s getting hotter outside, which is in turn worsening the production of allergens and increasing the allergen exposure and the severity of the symptoms that go along with it. More people are having more severe disease because the world is changing as a result of the stuff that we do. So fix that. But also be mindful and expect to see even more of these problems as you move forward in your careers.
Watto: Dr. Fadugba gave us so many great tips. You can listen to the full podcast episode here.
Dr. Watto, Clinical Assistant Professor, Department of Medicine, Perelman School of Medicine at University of Pennsylvania; Internist, Department of Medicine, Hospital Medicine Section, Pennsylvania Hospital, Philadelphia, has disclosed no relevant financial relationships. Dr. Williams, Associate Professor of Clinical Medicine, Department of General Internal Medicine, Lewis Katz School of Medicine; Staff Physician, Department of General Internal Medicine, Temple Internal Medicine Associates, Philadelphia, disclosed ties with The Curbsiders.
A version of this article first appeared on Medscape.com.
This transcript has been edited for clarity.
Matthew F. Watto, MD: I’m here with my great friend and America’s primary care physician, Dr. Paul Nelson Williams. Paul, are you ready to talk about rhinitis?
Paul N. Williams, MD: I’m excited. It’s always the season to talk about rhinitis.
Watto: We had a great guest for this podcast, Rhinitis and Environmental Allergies with Dr. Olajumoke Fadugba from Penn Medicine. She’s an allergist and immunologist. One of her pet peeves is when people just call everything “allergic rhinitis” because we should be calling it “chronic rhinitis,” if it’s chronic. That’s an umbrella term, and there are many buckets underneath it that people could fall into.
When you’re taking a history, you have to figure out whether it’s perennial (meaning it happens year round) because certain things can cause that. Cat dander is around all the time, so people with cats might have sinus symptoms all year. Dust mites are another one, and it’s pretty hard to avoid those. Those are some perennial allergens.
Then there is allergic vs nonallergic rhinitis, which is something I hadn’t really put too much thought into.
Williams: I didn’t realize exactly how nuanced it got. Nonallergic rhinitis can still be seasonal because changes in temperature and humidity can trigger the rhinitis. And it matters what medications you use for what.
Watto: Here are some ways you can try to figure out if rhinitis is allergic or nonallergic. Ask the patient if they have itchy eyes and are sneezing a lot. That can be more of an allergic rhinitis, but both allergic and nonallergic rhinitis have the congestion, the rhinorrhea, so you can’t figure it out based on that alone.
Dr. Fadugba said that one clue that it might be nonallergic rhinitis is the age of onset. If the symptoms are later in onset (older age), then 30%-40% of rhinitis is nonallergic. If the patient has never had allergies and now all of a sudden they have new chronic sinus symptoms, it’s probably nonallergic rhinitis. It’s a diagnosis of exclusion.
I guess they need allergy testing?
Williams: If you want to make a definitive diagnosis, you need to rule it out. I suspect that you might be able to get away with some empirical treatment. If they get better, you can feel like a winner because getting booked in for allergy testing can be a little bit of a challenge.
Watto: The main treatment difference is that the oral antihistamines do not really seem to work for nonallergic rhinitis, but they can help with allergic rhinitis. Weirdly, the nasal antihistamines and nasal steroids do seem to work for both allergic and nonallergic rhinitis.
I don’t understand the mechanism there, but if you think someone might have nonallergic rhinitis, I wouldn’t go with the oral antihistamines as your first-line treatment. I would go with a nasal spray; you pretty much can’t go wrong with either an antihistamine or a steroid nasal spray.
Williams: We typically start with the nasal sprays. That’s kind of first-line for almost everybody, allergic or nonallergic. You’re probably going to start with an intranasal steroid, and then it’s kind of dealer’s choice what the patient can tolerate and afford. Sometimes you can get them covered by insurance, at least in my experience.
I will say that this is one of the medications — like nicotine patches and other things — where we as doctors don’t really counsel patients on how to use it appropriately. So with our expert, we revisited the idea of the patient pointing the nasal spray laterally, toward their ear basically, and not spraying toward their brain. There should not be a slurping sound afterward, because “if you taste it, you waste it,” as the allergists and immunologists say. It’s supposed to sit up there and not be swallowed immediately.
If your patient is sensitive to the floral flavor of some of the fluticasones (which I don’t mind so much as a user myself), then you can try mometasone or the other formulations. They are all roughly equivalent.
Speaking of medications, which medications can cause rhinitis? Any meds we commonly use in primary care?
Williams: Apparently the combined hormonal oral contraceptives can do it. Also the phosphodiesterase 5 (PDE-5) inhibitors. Drugs that cause vasodilation can also do it. Some of the antihypertensives. I’ve seen beta-blockers and angiotensin-converting enzyme (ACE) inhibitors listed specifically, and some of the medications for benign prostatic hyperplasia (BPH). So there are a couple of medications that you can think about as a potential cause of rhinitis, although my suspicion is not going to be as high as for some of the other causes.
Watto: We mentioned medication treatments for patients who are really bothered by rhinorrhea, and maybe they are already on a steroid or an antihistamine.
You can try nasal ipratropium for people that have really prominent rhinorrhea. Dr. Fadugba said that can work well, and it’s usually taken three or four times a day. I’ve had good success prescribing it for my patients. Another one that I have never prescribed, but that Dr. Fadugba said is available over the counter, is intranasal cromolyn — a mast cell stabilizer. She said it can be beneficial.
Let’s say I had a cat allergy and I was going to visit Paul. I could use the intranasal cromolyn ahead of time to reduce rhinitis when I’m around the cats.
Paul, what about montelukast? I never know what to do with that one.
Williams: I’ve seen it prescribed as a last-ditch attempt to fix chronic rhinitis. Dr. Fadugba said she only ever prescribes it for patients who have rhinitis symptoms and asthma and never just for chronic rhinitis because it doesn’t work. And also, there have been some new black-box warnings from the US Food and Drug Administration (FDA). So unless there’s a solid indication for it, montelukast is not something you should just prescribe to try to see if it will work. That’s probably not the right approach for this.
But if the patient has challenging control asthma, and as a component, challenging nasal symptoms as well, it might be a reasonable medication to try.
Watto: And finally, Paul, how does climate change possibly have anything to do with rhinitis?
Williams: I feel like I’m just seeing more and more of the stuff every year. I don’t know if I’m more sensitive to it or because I’m having more symptoms myself, but it turns out the prevalence actually is going up.
We’re seeing more of it in part because it’s getting hotter outside, which is in turn worsening the production of allergens and increasing the allergen exposure and the severity of the symptoms that go along with it. More people are having more severe disease because the world is changing as a result of the stuff that we do. So fix that. But also be mindful and expect to see even more of these problems as you move forward in your careers.
Watto: Dr. Fadugba gave us so many great tips. You can listen to the full podcast episode here.
Dr. Watto, Clinical Assistant Professor, Department of Medicine, Perelman School of Medicine at University of Pennsylvania; Internist, Department of Medicine, Hospital Medicine Section, Pennsylvania Hospital, Philadelphia, has disclosed no relevant financial relationships. Dr. Williams, Associate Professor of Clinical Medicine, Department of General Internal Medicine, Lewis Katz School of Medicine; Staff Physician, Department of General Internal Medicine, Temple Internal Medicine Associates, Philadelphia, disclosed ties with The Curbsiders.
A version of this article first appeared on Medscape.com.