To the Editor:

Although biologics provide major therapeutic benefits for dermatologic conditions, they also come with a substantial cost, making them among the most expensive medications available. Medicare and Medicaid spending on biologics for dermatologic conditions increased by 320% from 2012 to 2018, reaching a staggering $10.6 billion in 2018 alone.¹ Biosimilars show promise in reducing health care spending for dermatologic conditions; however, their utilization has been limited due to multiple factors, including delayed market entry from patent thickets, exclusionary formulary contracts, and prescriber skepticism regarding their safety and efficacy.² For instance, a national survey of 1201 US physicians in specialties that are high prescribers of biologics reported that 55% doubted the safety and appropriateness of biosimilars.³

US Food and Drug Administration approval of biosimilars for adalimumab and etanercept offers the potential to reduce health care spending for dermatologic conditions. However, this cost reduction is dependent on utilization rates among dermatologists. In this national cross-sectional review of Medicare data, we predicted the impact of these biosimilars on dermatologic Medicare costs and demonstrated how differing utilization rates among dermatologists can influence potential savings.

To model 2023 utilization and cost reduction from biosimilars, we analyzed Medicare Part D data from 2020 on existing biosimilars, including granulocyte colony–stimulating factors, erythropoiesis-stimulating agents, and tumor necrosis factor α inhibitors.⁴ Methods in line with a 2021 report from the US Department of Health and Human Services⁵ as well as those of Yazdany et al⁶ were used. For each class, we calculated the 2020 distribution of biosimilar and originator drug claims as well as biosimilar cost reduction per 30-day claim. We utilized 2018-2021 annual growth rates for branded adalimumab and etanercept to estimate 30-day claims for 2023 and the cost of these branded agents in the absence of biosimilars. The hypothetical 2023 cost reduction from adalimumab and etanercept biosimilars was estimated by assuming 2020 biosimilar utilization rates and mean cost reduction per claim. This study utilized publicly available or aggregate summary data (not attributable to specific patients) and did not qualify as human subject research; therefore, institutional review board approval was not required.

In 2020, biosimilar utilization proportions ranged from 6.4% (tumor necrosis factor α inhibitors) to 82.7% (granulocyte colony–stimulating factors), with a mean across all classes of 35.7%. On average, the cost per 30-day claim of biosimilars was 66.8% of originator agents (Table 1). In 2021, we identified 57,868 30-day claims for branded adalimumab and etanercept submitted by dermatologists. From 2018 to 2021, 30-day branded adalimumab claims increased by 1.27% annually (cost + 10.62% annually), while claims for branded etanercept decreased by 13.0% annually (cost + 5.68% annually). Assuming these trends, the cost of branded adalimumab and etanercept was estimated to be $539 million in 2023. Applying the aforementioned 35.7% utilization, the introduction of biosimilars in dermatology would yield a cost reduction of approximately $118 million (21.9%). A high utilization rate (82.7%) of biosimilars among dermatologists would increase cost savings to $199 million (36.9%)(Table 2).

We found that the extent of savings from biosimilars was dependent on the utilization rates among dermatologists, with the highest utilization rate almost doubling the total savings of average utilization rates. Given the impact of high utilization and the wide variation observed, understanding the factors that have influenced uptake of biosimilars is important to increasing utilization as these medications become integrated into dermatology. For instance, limited uptake of infliximab initially may have been influenced by concerns about efficacy and increased adverse events.^8,9 In contrast, the high utilization of filgrastim biosimilars (82.7%) may be attributed to its longevity in the market and familiarity to prescribers, as filgrastim was the first biosimilar to be approved in the United States.¹⁰

Promoting reasonable utilization of biosimilars may require prescriber education on their safety and approval processes, which could foster increased utilization and reduce skepticism.⁴ Under the Biologics Price Competition and Innovation Act, the US Food and Drug Administration approves biosimilars only when they exhibit “high similarity” and show no “clinically meaningful differences” compared to the reference biologic, with no added safety risks or reduced efficacy.¹¹ Moreover, a 2023 systematic review of 17 studies found no major difference in efficacy and safety between biosimilars and originators of etanercept, infliximab, and other biologics.¹² Understanding these findings may reassure dermatologists and patients about the reliability and safety of biosimilars.

A limitation of our study is that it solely assesses Medicare data and estimates derived from existing (separate) biologic classes. It also does not account for potential expenditure shifts to newer biologic agents (eg, IL-12/17/23 inhibitors) or changes in manufacturer behavior or promotions. Nevertheless, it indicates notable financial savings from new biosimilar agents in dermatology; along with their compelling efficacy and safety profiles, this could represent a substantial benefit to patients and the health care system.

To the Editor:

Although biologics provide major therapeutic benefits for dermatologic conditions, they also come with a substantial cost, making them among the most expensive medications available. Medicare and Medicaid spending on biologics for dermatologic conditions increased by 320% from 2012 to 2018, reaching a staggering $10.6 billion in 2018 alone.¹ Biosimilars show promise in reducing health care spending for dermatologic conditions; however, their utilization has been limited due to multiple factors, including delayed market entry from patent thickets, exclusionary formulary contracts, and prescriber skepticism regarding their safety and efficacy.² For instance, a national survey of 1201 US physicians in specialties that are high prescribers of biologics reported that 55% doubted the safety and appropriateness of biosimilars.³

US Food and Drug Administration approval of biosimilars for adalimumab and etanercept offers the potential to reduce health care spending for dermatologic conditions. However, this cost reduction is dependent on utilization rates among dermatologists. In this national cross-sectional review of Medicare data, we predicted the impact of these biosimilars on dermatologic Medicare costs and demonstrated how differing utilization rates among dermatologists can influence potential savings.

To model 2023 utilization and cost reduction from biosimilars, we analyzed Medicare Part D data from 2020 on existing biosimilars, including granulocyte colony–stimulating factors, erythropoiesis-stimulating agents, and tumor necrosis factor α inhibitors.⁴ Methods in line with a 2021 report from the US Department of Health and Human Services⁵ as well as those of Yazdany et al⁶ were used. For each class, we calculated the 2020 distribution of biosimilar and originator drug claims as well as biosimilar cost reduction per 30-day claim. We utilized 2018-2021 annual growth rates for branded adalimumab and etanercept to estimate 30-day claims for 2023 and the cost of these branded agents in the absence of biosimilars. The hypothetical 2023 cost reduction from adalimumab and etanercept biosimilars was estimated by assuming 2020 biosimilar utilization rates and mean cost reduction per claim. This study utilized publicly available or aggregate summary data (not attributable to specific patients) and did not qualify as human subject research; therefore, institutional review board approval was not required.

In 2020, biosimilar utilization proportions ranged from 6.4% (tumor necrosis factor α inhibitors) to 82.7% (granulocyte colony–stimulating factors), with a mean across all classes of 35.7%. On average, the cost per 30-day claim of biosimilars was 66.8% of originator agents (Table 1). In 2021, we identified 57,868 30-day claims for branded adalimumab and etanercept submitted by dermatologists. From 2018 to 2021, 30-day branded adalimumab claims increased by 1.27% annually (cost + 10.62% annually), while claims for branded etanercept decreased by 13.0% annually (cost + 5.68% annually). Assuming these trends, the cost of branded adalimumab and etanercept was estimated to be $539 million in 2023. Applying the aforementioned 35.7% utilization, the introduction of biosimilars in dermatology would yield a cost reduction of approximately $118 million (21.9%). A high utilization rate (82.7%) of biosimilars among dermatologists would increase cost savings to $199 million (36.9%)(Table 2).

We found that the extent of savings from biosimilars was dependent on the utilization rates among dermatologists, with the highest utilization rate almost doubling the total savings of average utilization rates. Given the impact of high utilization and the wide variation observed, understanding the factors that have influenced uptake of biosimilars is important to increasing utilization as these medications become integrated into dermatology. For instance, limited uptake of infliximab initially may have been influenced by concerns about efficacy and increased adverse events.^8,9 In contrast, the high utilization of filgrastim biosimilars (82.7%) may be attributed to its longevity in the market and familiarity to prescribers, as filgrastim was the first biosimilar to be approved in the United States.¹⁰

Promoting reasonable utilization of biosimilars may require prescriber education on their safety and approval processes, which could foster increased utilization and reduce skepticism.⁴ Under the Biologics Price Competition and Innovation Act, the US Food and Drug Administration approves biosimilars only when they exhibit “high similarity” and show no “clinically meaningful differences” compared to the reference biologic, with no added safety risks or reduced efficacy.¹¹ Moreover, a 2023 systematic review of 17 studies found no major difference in efficacy and safety between biosimilars and originators of etanercept, infliximab, and other biologics.¹² Understanding these findings may reassure dermatologists and patients about the reliability and safety of biosimilars.

A limitation of our study is that it solely assesses Medicare data and estimates derived from existing (separate) biologic classes. It also does not account for potential expenditure shifts to newer biologic agents (eg, IL-12/17/23 inhibitors) or changes in manufacturer behavior or promotions. Nevertheless, it indicates notable financial savings from new biosimilar agents in dermatology; along with their compelling efficacy and safety profiles, this could represent a substantial benefit to patients and the health care system.

To the Editor:

Although biologics provide major therapeutic benefits for dermatologic conditions, they also come with a substantial cost, making them among the most expensive medications available. Medicare and Medicaid spending on biologics for dermatologic conditions increased by 320% from 2012 to 2018, reaching a staggering $10.6 billion in 2018 alone.¹ Biosimilars show promise in reducing health care spending for dermatologic conditions; however, their utilization has been limited due to multiple factors, including delayed market entry from patent thickets, exclusionary formulary contracts, and prescriber skepticism regarding their safety and efficacy.² For instance, a national survey of 1201 US physicians in specialties that are high prescribers of biologics reported that 55% doubted the safety and appropriateness of biosimilars.³

US Food and Drug Administration approval of biosimilars for adalimumab and etanercept offers the potential to reduce health care spending for dermatologic conditions. However, this cost reduction is dependent on utilization rates among dermatologists. In this national cross-sectional review of Medicare data, we predicted the impact of these biosimilars on dermatologic Medicare costs and demonstrated how differing utilization rates among dermatologists can influence potential savings.

To model 2023 utilization and cost reduction from biosimilars, we analyzed Medicare Part D data from 2020 on existing biosimilars, including granulocyte colony–stimulating factors, erythropoiesis-stimulating agents, and tumor necrosis factor α inhibitors.⁴ Methods in line with a 2021 report from the US Department of Health and Human Services⁵ as well as those of Yazdany et al⁶ were used. For each class, we calculated the 2020 distribution of biosimilar and originator drug claims as well as biosimilar cost reduction per 30-day claim. We utilized 2018-2021 annual growth rates for branded adalimumab and etanercept to estimate 30-day claims for 2023 and the cost of these branded agents in the absence of biosimilars. The hypothetical 2023 cost reduction from adalimumab and etanercept biosimilars was estimated by assuming 2020 biosimilar utilization rates and mean cost reduction per claim. This study utilized publicly available or aggregate summary data (not attributable to specific patients) and did not qualify as human subject research; therefore, institutional review board approval was not required.

In 2020, biosimilar utilization proportions ranged from 6.4% (tumor necrosis factor α inhibitors) to 82.7% (granulocyte colony–stimulating factors), with a mean across all classes of 35.7%. On average, the cost per 30-day claim of biosimilars was 66.8% of originator agents (Table 1). In 2021, we identified 57,868 30-day claims for branded adalimumab and etanercept submitted by dermatologists. From 2018 to 2021, 30-day branded adalimumab claims increased by 1.27% annually (cost + 10.62% annually), while claims for branded etanercept decreased by 13.0% annually (cost + 5.68% annually). Assuming these trends, the cost of branded adalimumab and etanercept was estimated to be $539 million in 2023. Applying the aforementioned 35.7% utilization, the introduction of biosimilars in dermatology would yield a cost reduction of approximately $118 million (21.9%). A high utilization rate (82.7%) of biosimilars among dermatologists would increase cost savings to $199 million (36.9%)(Table 2).

We found that the extent of savings from biosimilars was dependent on the utilization rates among dermatologists, with the highest utilization rate almost doubling the total savings of average utilization rates. Given the impact of high utilization and the wide variation observed, understanding the factors that have influenced uptake of biosimilars is important to increasing utilization as these medications become integrated into dermatology. For instance, limited uptake of infliximab initially may have been influenced by concerns about efficacy and increased adverse events.^8,9 In contrast, the high utilization of filgrastim biosimilars (82.7%) may be attributed to its longevity in the market and familiarity to prescribers, as filgrastim was the first biosimilar to be approved in the United States.¹⁰

Promoting reasonable utilization of biosimilars may require prescriber education on their safety and approval processes, which could foster increased utilization and reduce skepticism.⁴ Under the Biologics Price Competition and Innovation Act, the US Food and Drug Administration approves biosimilars only when they exhibit “high similarity” and show no “clinically meaningful differences” compared to the reference biologic, with no added safety risks or reduced efficacy.¹¹ Moreover, a 2023 systematic review of 17 studies found no major difference in efficacy and safety between biosimilars and originators of etanercept, infliximab, and other biologics.¹² Understanding these findings may reassure dermatologists and patients about the reliability and safety of biosimilars.

A limitation of our study is that it solely assesses Medicare data and estimates derived from existing (separate) biologic classes. It also does not account for potential expenditure shifts to newer biologic agents (eg, IL-12/17/23 inhibitors) or changes in manufacturer behavior or promotions. Nevertheless, it indicates notable financial savings from new biosimilar agents in dermatology; along with their compelling efficacy and safety profiles, this could represent a substantial benefit to patients and the health care system.

To the Editor:

Drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome—a severe cutaneous adverse drug reaction—is characterized by a cutaneous rash and systemic upset in the form of various internal organ and hematologic disturbances. This delayed and idiosyncratic syndrome went by several names, including anticonvulsant hypersensitivity syndrome, before Bocquet et al¹ proposed the term DRESS syndrome.

Phenytoin, a hydantoin derivative used in neurology, was implicated in 41% of cases of DRESS syndrome in a study of 100 patients conducted in southern India.^2,3 While DRESS syndrome is a newer name, the clinical picture of DRESS secondary to phenytoin use remains similar in that it manifests with a morbilliform rash and systemic upset. We sought to describe the clinical and laboratory characteristics of phenytoin-induced DRESS syndrome in this case series.

The analysis included 23 patients with DRESS syndrome secondary to phenytoin use who presented to a tertiary care institution in North India between July 2021 and December 2022, satisfied the European Registry of Severe Cutaneous Adverse Reaction (RegiSCAR) criteria,⁴ and achieved a DRESS diagnostic score of more than 1. The mean age of the patients was 44 years (range, 14–74 years). There was a slight female predominance with a male to female ratio of 0.9:1. More than half of the patients (52.2% [12/23]) presented directly to the dermatology outpatient department; the remaining patients were referred from other departments (47.8% [11/23]). Patients primarily were receiving phenytoin for neurologic indications. Specific reasons included antiseizure prophylaxis following a traffic accident (34.8% [8/23]); epilepsy (26.1% [6/23]); and neoplastic (17.4% [4/23]), vascular (17.4% [4/23]), and infectious (4.3% [1/23]) causes. The mean latency period from drug intake to symptom onset was 29 days (range, 6–62 days), and the mean illness duration was 9 days (range, 1–45 days).

The majority of patients experienced pruritus (91.3% [21/23]) and fever (74.0% [17/23]), and all initially had a rash. Maculopapular morphology was seen in all patients. Erythema multiforme–like (17.4% [4/23]), erythrodermic (17.4% [4/23]), and vesicular (13.0% [3/23]) rashes also were documented (Figure 1). The trunk (100% [23/23]) and extremities (95.7% [22/23]) were involved most often, followed by the palms and soles (56.5% [13/23]). The mean total body surface area affected was 73.65%. Only 7 patients (30.4%) had mucosal ­involvement; nonhemorrhagic cheilitis was the most common manifestation.

To the Editor:

Drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome—a severe cutaneous adverse drug reaction—is characterized by a cutaneous rash and systemic upset in the form of various internal organ and hematologic disturbances. This delayed and idiosyncratic syndrome went by several names, including anticonvulsant hypersensitivity syndrome, before Bocquet et al¹ proposed the term DRESS syndrome.

Phenytoin, a hydantoin derivative used in neurology, was implicated in 41% of cases of DRESS syndrome in a study of 100 patients conducted in southern India.^2,3 While DRESS syndrome is a newer name, the clinical picture of DRESS secondary to phenytoin use remains similar in that it manifests with a morbilliform rash and systemic upset. We sought to describe the clinical and laboratory characteristics of phenytoin-induced DRESS syndrome in this case series.

The analysis included 23 patients with DRESS syndrome secondary to phenytoin use who presented to a tertiary care institution in North India between July 2021 and December 2022, satisfied the European Registry of Severe Cutaneous Adverse Reaction (RegiSCAR) criteria,⁴ and achieved a DRESS diagnostic score of more than 1. The mean age of the patients was 44 years (range, 14–74 years). There was a slight female predominance with a male to female ratio of 0.9:1. More than half of the patients (52.2% [12/23]) presented directly to the dermatology outpatient department; the remaining patients were referred from other departments (47.8% [11/23]). Patients primarily were receiving phenytoin for neurologic indications. Specific reasons included antiseizure prophylaxis following a traffic accident (34.8% [8/23]); epilepsy (26.1% [6/23]); and neoplastic (17.4% [4/23]), vascular (17.4% [4/23]), and infectious (4.3% [1/23]) causes. The mean latency period from drug intake to symptom onset was 29 days (range, 6–62 days), and the mean illness duration was 9 days (range, 1–45 days).

The majority of patients experienced pruritus (91.3% [21/23]) and fever (74.0% [17/23]), and all initially had a rash. Maculopapular morphology was seen in all patients. Erythema multiforme–like (17.4% [4/23]), erythrodermic (17.4% [4/23]), and vesicular (13.0% [3/23]) rashes also were documented (Figure 1). The trunk (100% [23/23]) and extremities (95.7% [22/23]) were involved most often, followed by the palms and soles (56.5% [13/23]). The mean total body surface area affected was 73.65%. Only 7 patients (30.4%) had mucosal ­involvement; nonhemorrhagic cheilitis was the most common manifestation.

To the Editor:

Drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome—a severe cutaneous adverse drug reaction—is characterized by a cutaneous rash and systemic upset in the form of various internal organ and hematologic disturbances. This delayed and idiosyncratic syndrome went by several names, including anticonvulsant hypersensitivity syndrome, before Bocquet et al¹ proposed the term DRESS syndrome.

Phenytoin, a hydantoin derivative used in neurology, was implicated in 41% of cases of DRESS syndrome in a study of 100 patients conducted in southern India.^2,3 While DRESS syndrome is a newer name, the clinical picture of DRESS secondary to phenytoin use remains similar in that it manifests with a morbilliform rash and systemic upset. We sought to describe the clinical and laboratory characteristics of phenytoin-induced DRESS syndrome in this case series.

The analysis included 23 patients with DRESS syndrome secondary to phenytoin use who presented to a tertiary care institution in North India between July 2021 and December 2022, satisfied the European Registry of Severe Cutaneous Adverse Reaction (RegiSCAR) criteria,⁴ and achieved a DRESS diagnostic score of more than 1. The mean age of the patients was 44 years (range, 14–74 years). There was a slight female predominance with a male to female ratio of 0.9:1. More than half of the patients (52.2% [12/23]) presented directly to the dermatology outpatient department; the remaining patients were referred from other departments (47.8% [11/23]). Patients primarily were receiving phenytoin for neurologic indications. Specific reasons included antiseizure prophylaxis following a traffic accident (34.8% [8/23]); epilepsy (26.1% [6/23]); and neoplastic (17.4% [4/23]), vascular (17.4% [4/23]), and infectious (4.3% [1/23]) causes. The mean latency period from drug intake to symptom onset was 29 days (range, 6–62 days), and the mean illness duration was 9 days (range, 1–45 days).

The majority of patients experienced pruritus (91.3% [21/23]) and fever (74.0% [17/23]), and all initially had a rash. Maculopapular morphology was seen in all patients. Erythema multiforme–like (17.4% [4/23]), erythrodermic (17.4% [4/23]), and vesicular (13.0% [3/23]) rashes also were documented (Figure 1). The trunk (100% [23/23]) and extremities (95.7% [22/23]) were involved most often, followed by the palms and soles (56.5% [13/23]). The mean total body surface area affected was 73.65%. Only 7 patients (30.4%) had mucosal ­involvement; nonhemorrhagic cheilitis was the most common manifestation.

SAVANNAH, GEORGIA — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of nerve/muscle specialists.

The cardiac conditions can range from asymptomatic to potentially lethal, Nicholas J. Silvestri, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “It’s really important to know when to do tests and refer to cardiology, and it’s really important to find a cardiologist who can work in concert in taking care of these patients.”
 

Protein Alterations May Disrupt Heart Muscles

In muscular dystrophies, a prevailing theory suggests that alterations to proteins such as dystrophin disrupt structural integrity in both muscle and cardiac cells, he said.

In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to some degree before that, and it’s not going to get better over time,” he said.

Cardiac problems are universal in DMD by age 18, he said. “Men and boys are living longer, so they have the opportunity to develop the cardiac abnormalities that accrue with time.” Conduction abnormalities typically appear first. “In a lot of these boys, you’ll typically see persistent sinus tachycardia. But they can also develop atrial arrhythmias and bundle branch blocks.”

Sudden cardiac death is responsible for mortality in an estimated 15% patients with DMD. “Very sadly, I lost a patient this way just a few months ago,” Silvestri said.
 

ECGs and Echos Are Recommended

Screening is crucial. “Make sure that patients get that referral and get these tests done,” he said. “You need an ECG and echo by diagnosis or age 6. This is usually repeated annually or biannually, typically by the cardiologist you’re working with.”

The good news is that there’s evidence of survival benefits from treatment with angiotensin-converting enzyme inhibitors for dilated cardiomyopathy. “Some cardiac experts feel treatment with angiotensin receptor blockers (ARBs) is equivalent.”

Most boys will get echocardiograms, he said, “but there’s a lot of evidence showing that cardiac MRI is probably preferable for a number of reasons,” including better visualization. But the need for sedation limits access, he said, and cardiac MRI may not be available at some facilities.
 

Worse Outcomes in Becker Muscular Dystrophy (BMD)

Cardiac involvement is more common and more severe in BMD than in DMD. About 50% of deaths in BMD are attributed to malignant arrhythmias or congestive heart failure, he said.

Screening requirements and treatment options in BMD are similar to those in DMD, with the added option of heart transplantation.

Silvestri cautioned that up to 40% of female carriers of dystrophin mutations can develop cardiac dysfunction similar to that seen in DMD and BMD. Cardiac assessments are recommended every 5 years. “It’s important to genotype Mom,” he said, especially in light of the fact that two thirds of DMD cases may be inherited.

“When I send genetic testing on the mother and find her to be a carrier, I send her to a cardiologist so she has the appropriate screening done,” he said.
 

Pacemakers May Be Considered in Type 1 Myotonic Dystrophy

In type 1 myotonic dystrophy, cardiac conduction abnormalities are seen in two thirds of patients, and sudden cardiac death in up to 30% of patients. “When it is diagnosed, patients do need an ECG at that time, as well as annually,” he said.

Holter monitoring or implantable loop recorders may be recommended, and permanent pacing via an implantable cardioverter-defibrillator might be appropriate.

“Based on the literature to date, the exact timing is not is not clear,” Silvestri said. “The electrophysiologists in my area tend to be very aggressive, thankfully, and treat them fairly soon with pacemakers when we see the first sign of trouble.”

Silvestri disclosed consultant/advisory relationships with argenx, Alexion, Amgen, UCB, Immunovant, and Janssen.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of nerve/muscle specialists.

The cardiac conditions can range from asymptomatic to potentially lethal, Nicholas J. Silvestri, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “It’s really important to know when to do tests and refer to cardiology, and it’s really important to find a cardiologist who can work in concert in taking care of these patients.”
 

Protein Alterations May Disrupt Heart Muscles

In muscular dystrophies, a prevailing theory suggests that alterations to proteins such as dystrophin disrupt structural integrity in both muscle and cardiac cells, he said.

In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to some degree before that, and it’s not going to get better over time,” he said.

Cardiac problems are universal in DMD by age 18, he said. “Men and boys are living longer, so they have the opportunity to develop the cardiac abnormalities that accrue with time.” Conduction abnormalities typically appear first. “In a lot of these boys, you’ll typically see persistent sinus tachycardia. But they can also develop atrial arrhythmias and bundle branch blocks.”

Sudden cardiac death is responsible for mortality in an estimated 15% patients with DMD. “Very sadly, I lost a patient this way just a few months ago,” Silvestri said.
 

ECGs and Echos Are Recommended

Screening is crucial. “Make sure that patients get that referral and get these tests done,” he said. “You need an ECG and echo by diagnosis or age 6. This is usually repeated annually or biannually, typically by the cardiologist you’re working with.”

The good news is that there’s evidence of survival benefits from treatment with angiotensin-converting enzyme inhibitors for dilated cardiomyopathy. “Some cardiac experts feel treatment with angiotensin receptor blockers (ARBs) is equivalent.”

Most boys will get echocardiograms, he said, “but there’s a lot of evidence showing that cardiac MRI is probably preferable for a number of reasons,” including better visualization. But the need for sedation limits access, he said, and cardiac MRI may not be available at some facilities.
 

Worse Outcomes in Becker Muscular Dystrophy (BMD)

Cardiac involvement is more common and more severe in BMD than in DMD. About 50% of deaths in BMD are attributed to malignant arrhythmias or congestive heart failure, he said.

Screening requirements and treatment options in BMD are similar to those in DMD, with the added option of heart transplantation.

Silvestri cautioned that up to 40% of female carriers of dystrophin mutations can develop cardiac dysfunction similar to that seen in DMD and BMD. Cardiac assessments are recommended every 5 years. “It’s important to genotype Mom,” he said, especially in light of the fact that two thirds of DMD cases may be inherited.

“When I send genetic testing on the mother and find her to be a carrier, I send her to a cardiologist so she has the appropriate screening done,” he said.
 

Pacemakers May Be Considered in Type 1 Myotonic Dystrophy

In type 1 myotonic dystrophy, cardiac conduction abnormalities are seen in two thirds of patients, and sudden cardiac death in up to 30% of patients. “When it is diagnosed, patients do need an ECG at that time, as well as annually,” he said.

Holter monitoring or implantable loop recorders may be recommended, and permanent pacing via an implantable cardioverter-defibrillator might be appropriate.

“Based on the literature to date, the exact timing is not is not clear,” Silvestri said. “The electrophysiologists in my area tend to be very aggressive, thankfully, and treat them fairly soon with pacemakers when we see the first sign of trouble.”

Silvestri disclosed consultant/advisory relationships with argenx, Alexion, Amgen, UCB, Immunovant, and Janssen.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of nerve/muscle specialists.

The cardiac conditions can range from asymptomatic to potentially lethal, Nicholas J. Silvestri, MD, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, in New York, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024. “It’s really important to know when to do tests and refer to cardiology, and it’s really important to find a cardiologist who can work in concert in taking care of these patients.”
 

Protein Alterations May Disrupt Heart Muscles

In muscular dystrophies, a prevailing theory suggests that alterations to proteins such as dystrophin disrupt structural integrity in both muscle and cardiac cells, he said.

In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to some degree before that, and it’s not going to get better over time,” he said.

Cardiac problems are universal in DMD by age 18, he said. “Men and boys are living longer, so they have the opportunity to develop the cardiac abnormalities that accrue with time.” Conduction abnormalities typically appear first. “In a lot of these boys, you’ll typically see persistent sinus tachycardia. But they can also develop atrial arrhythmias and bundle branch blocks.”

Sudden cardiac death is responsible for mortality in an estimated 15% patients with DMD. “Very sadly, I lost a patient this way just a few months ago,” Silvestri said.
 

ECGs and Echos Are Recommended

Screening is crucial. “Make sure that patients get that referral and get these tests done,” he said. “You need an ECG and echo by diagnosis or age 6. This is usually repeated annually or biannually, typically by the cardiologist you’re working with.”

The good news is that there’s evidence of survival benefits from treatment with angiotensin-converting enzyme inhibitors for dilated cardiomyopathy. “Some cardiac experts feel treatment with angiotensin receptor blockers (ARBs) is equivalent.”

Most boys will get echocardiograms, he said, “but there’s a lot of evidence showing that cardiac MRI is probably preferable for a number of reasons,” including better visualization. But the need for sedation limits access, he said, and cardiac MRI may not be available at some facilities.
 

Worse Outcomes in Becker Muscular Dystrophy (BMD)

Cardiac involvement is more common and more severe in BMD than in DMD. About 50% of deaths in BMD are attributed to malignant arrhythmias or congestive heart failure, he said.

Screening requirements and treatment options in BMD are similar to those in DMD, with the added option of heart transplantation.

Silvestri cautioned that up to 40% of female carriers of dystrophin mutations can develop cardiac dysfunction similar to that seen in DMD and BMD. Cardiac assessments are recommended every 5 years. “It’s important to genotype Mom,” he said, especially in light of the fact that two thirds of DMD cases may be inherited.

“When I send genetic testing on the mother and find her to be a carrier, I send her to a cardiologist so she has the appropriate screening done,” he said.
 

Pacemakers May Be Considered in Type 1 Myotonic Dystrophy

In type 1 myotonic dystrophy, cardiac conduction abnormalities are seen in two thirds of patients, and sudden cardiac death in up to 30% of patients. “When it is diagnosed, patients do need an ECG at that time, as well as annually,” he said.

Holter monitoring or implantable loop recorders may be recommended, and permanent pacing via an implantable cardioverter-defibrillator might be appropriate.

“Based on the literature to date, the exact timing is not is not clear,” Silvestri said. “The electrophysiologists in my area tend to be very aggressive, thankfully, and treat them fairly soon with pacemakers when we see the first sign of trouble.”

Silvestri disclosed consultant/advisory relationships with argenx, Alexion, Amgen, UCB, Immunovant, and Janssen.
 

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Current disease-modifying therapies for amyotrophic lateral sclerosis (ALS) don’t extend lifespans by much, but several drug options are available, nerve specialists learned.

The glutamate blocker riluzole (Rilutek), which became the first ALS drug to receive US Food and Drug Administration (FDA) approval in 1995, continues to be used, Michael D. Weiss, MD, professor of neurology at University of Washington School of Medicine, Seattle, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Weiss highlighted a 2012 Cochrane Library review that examined research and found the drug is “reasonably safe” and prolongs median survival by about 2-3 months. “About 12% develop liver disease. It’s pretty rare that we stop the medicine due to liver toxicity.”
 

Earlier Treatment Could Pay Dividends

A recent study “suggests we might be able to get more bang for our buck from riluzole” by initiating treatment earlier, Weiss said.

Researchers tracked 4778 patients with ALS, including 3446 (72.1%) who took riluzole. Those who took the drug survived a median 2 extra months (22.6 vs 20.2 months; P < .001). The data suggested that delaying riluzole initiation by 1 year (from 6 months to 18 months after diagnosis) reduced the median survival by 1.9 months (from 40.1 to 38.2 months).

There’s “a relatively significant additional benefit” to earlier treatment, Weiss said, although patients will vary on whether they think it’s meaningful. As for limitations, “there’s no clear impact on disease progression, and there’s a need for periodic monitoring of liver function profile.” 

He added that there’s an out-of-pocket co-pay. “Even as a generic, it’s not that cheap. Depending on the source, it could cost anywhere from $1800 to $8400 a year.”
 

Edaravone Could Lack Relevant Benefit

No other ALS treatment appeared until 2017, when the FDA approved the novel antioxidant edaravone (Radicava). In 2022, the agency approved an oral suspension version, but a study published that year suggested there may not be a clinically relevant benefit.

The University of Washington, where Weiss works, offered the drug to 144 patients, according to an analysis. Eighty percent of the patients wanted it, but insurers refused to cover it for more than 20%. The average time to treatment with the drug was 28 days after patients said they wanted it.

That’s a “substantial delay,” Weiss said.

The cost is about $171,000 a year, he said, and assistance is limited for underinsured patients.*

Other Options

As Weiss noted, another drug, AMX0035 (Relyvrio), received FDA approval in 2022, but its manufacturer pulled it from the US/Canada market in April 2024 following poor phase 3 trial findings.

In 2023, the FDA approved another drug, the antisense oligonucleotide tofersen (Qalsody), in patients with ALS associated with a mutation in the superoxide dismutase 1 gene. According to the FDA, reductions in plasma neurofilament light concentration were “reasonably likely to predict a clinical benefit in patients.”

Only 1%-2% of patients with ALS fit the criteria to get the drug, Weiss said. He noted other limitations such as the cost ($180,000 a year), the need for lifelong monthly intrathecal injections, and serious neurological side effects in 7% of patients per a 2022 trial.

Weiss disclosed advisory board (Alexion, Ra [now UCB], argenx, Biogen, Mitsubishi Tanabe Pharma, Amylyx), data safety monitoring board (Sanofi, AI), consulting (Cytokinetics, CSL Behring), and speaker (Soleo) relationships.

*Correction, 10/23/2024: This story originally quoted Weiss as saying the maker of edaravone provides no assistance to underinsured patients. Weiss has clarified that he should have said this coverage is “limited.”

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Current disease-modifying therapies for amyotrophic lateral sclerosis (ALS) don’t extend lifespans by much, but several drug options are available, nerve specialists learned.

The glutamate blocker riluzole (Rilutek), which became the first ALS drug to receive US Food and Drug Administration (FDA) approval in 1995, continues to be used, Michael D. Weiss, MD, professor of neurology at University of Washington School of Medicine, Seattle, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Weiss highlighted a 2012 Cochrane Library review that examined research and found the drug is “reasonably safe” and prolongs median survival by about 2-3 months. “About 12% develop liver disease. It’s pretty rare that we stop the medicine due to liver toxicity.”
 

Earlier Treatment Could Pay Dividends

A recent study “suggests we might be able to get more bang for our buck from riluzole” by initiating treatment earlier, Weiss said.

Researchers tracked 4778 patients with ALS, including 3446 (72.1%) who took riluzole. Those who took the drug survived a median 2 extra months (22.6 vs 20.2 months; P < .001). The data suggested that delaying riluzole initiation by 1 year (from 6 months to 18 months after diagnosis) reduced the median survival by 1.9 months (from 40.1 to 38.2 months).

There’s “a relatively significant additional benefit” to earlier treatment, Weiss said, although patients will vary on whether they think it’s meaningful. As for limitations, “there’s no clear impact on disease progression, and there’s a need for periodic monitoring of liver function profile.” 

He added that there’s an out-of-pocket co-pay. “Even as a generic, it’s not that cheap. Depending on the source, it could cost anywhere from $1800 to $8400 a year.”
 

Edaravone Could Lack Relevant Benefit

No other ALS treatment appeared until 2017, when the FDA approved the novel antioxidant edaravone (Radicava). In 2022, the agency approved an oral suspension version, but a study published that year suggested there may not be a clinically relevant benefit.

The University of Washington, where Weiss works, offered the drug to 144 patients, according to an analysis. Eighty percent of the patients wanted it, but insurers refused to cover it for more than 20%. The average time to treatment with the drug was 28 days after patients said they wanted it.

That’s a “substantial delay,” Weiss said.

The cost is about $171,000 a year, he said, and assistance is limited for underinsured patients.*

Other Options

As Weiss noted, another drug, AMX0035 (Relyvrio), received FDA approval in 2022, but its manufacturer pulled it from the US/Canada market in April 2024 following poor phase 3 trial findings.

In 2023, the FDA approved another drug, the antisense oligonucleotide tofersen (Qalsody), in patients with ALS associated with a mutation in the superoxide dismutase 1 gene. According to the FDA, reductions in plasma neurofilament light concentration were “reasonably likely to predict a clinical benefit in patients.”

Only 1%-2% of patients with ALS fit the criteria to get the drug, Weiss said. He noted other limitations such as the cost ($180,000 a year), the need for lifelong monthly intrathecal injections, and serious neurological side effects in 7% of patients per a 2022 trial.

Weiss disclosed advisory board (Alexion, Ra [now UCB], argenx, Biogen, Mitsubishi Tanabe Pharma, Amylyx), data safety monitoring board (Sanofi, AI), consulting (Cytokinetics, CSL Behring), and speaker (Soleo) relationships.

*Correction, 10/23/2024: This story originally quoted Weiss as saying the maker of edaravone provides no assistance to underinsured patients. Weiss has clarified that he should have said this coverage is “limited.”

A version of this article appeared on Medscape.com.

SAVANNAH, GEORGIA — Current disease-modifying therapies for amyotrophic lateral sclerosis (ALS) don’t extend lifespans by much, but several drug options are available, nerve specialists learned.

The glutamate blocker riluzole (Rilutek), which became the first ALS drug to receive US Food and Drug Administration (FDA) approval in 1995, continues to be used, Michael D. Weiss, MD, professor of neurology at University of Washington School of Medicine, Seattle, said in a presentation at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.

Weiss highlighted a 2012 Cochrane Library review that examined research and found the drug is “reasonably safe” and prolongs median survival by about 2-3 months. “About 12% develop liver disease. It’s pretty rare that we stop the medicine due to liver toxicity.”
 

Earlier Treatment Could Pay Dividends

A recent study “suggests we might be able to get more bang for our buck from riluzole” by initiating treatment earlier, Weiss said.

Researchers tracked 4778 patients with ALS, including 3446 (72.1%) who took riluzole. Those who took the drug survived a median 2 extra months (22.6 vs 20.2 months; P < .001). The data suggested that delaying riluzole initiation by 1 year (from 6 months to 18 months after diagnosis) reduced the median survival by 1.9 months (from 40.1 to 38.2 months).

There’s “a relatively significant additional benefit” to earlier treatment, Weiss said, although patients will vary on whether they think it’s meaningful. As for limitations, “there’s no clear impact on disease progression, and there’s a need for periodic monitoring of liver function profile.” 

He added that there’s an out-of-pocket co-pay. “Even as a generic, it’s not that cheap. Depending on the source, it could cost anywhere from $1800 to $8400 a year.”
 

Edaravone Could Lack Relevant Benefit

No other ALS treatment appeared until 2017, when the FDA approved the novel antioxidant edaravone (Radicava). In 2022, the agency approved an oral suspension version, but a study published that year suggested there may not be a clinically relevant benefit.

The University of Washington, where Weiss works, offered the drug to 144 patients, according to an analysis. Eighty percent of the patients wanted it, but insurers refused to cover it for more than 20%. The average time to treatment with the drug was 28 days after patients said they wanted it.

That’s a “substantial delay,” Weiss said.

The cost is about $171,000 a year, he said, and assistance is limited for underinsured patients.*

Other Options

As Weiss noted, another drug, AMX0035 (Relyvrio), received FDA approval in 2022, but its manufacturer pulled it from the US/Canada market in April 2024 following poor phase 3 trial findings.

In 2023, the FDA approved another drug, the antisense oligonucleotide tofersen (Qalsody), in patients with ALS associated with a mutation in the superoxide dismutase 1 gene. According to the FDA, reductions in plasma neurofilament light concentration were “reasonably likely to predict a clinical benefit in patients.”

Only 1%-2% of patients with ALS fit the criteria to get the drug, Weiss said. He noted other limitations such as the cost ($180,000 a year), the need for lifelong monthly intrathecal injections, and serious neurological side effects in 7% of patients per a 2022 trial.

Weiss disclosed advisory board (Alexion, Ra [now UCB], argenx, Biogen, Mitsubishi Tanabe Pharma, Amylyx), data safety monitoring board (Sanofi, AI), consulting (Cytokinetics, CSL Behring), and speaker (Soleo) relationships.

*Correction, 10/23/2024: This story originally quoted Weiss as saying the maker of edaravone provides no assistance to underinsured patients. Weiss has clarified that he should have said this coverage is “limited.”

A version of this article appeared on Medscape.com.

Like millions of other modern humans, my daughter and I stood in the backyard recently and watched comet C/2023 A3 (Tsuchinshan–ATLAS) with binoculars. It took a few minutes to locate, but once you see it is unmistakable.

It’s got a long (at least in human terms) orbit, roughly 80,000 years. So what was going on here, on our pale blue dot, the last time it graced our skies?

Well, here in Phoenix, the people were ... not here. Nor were they in Arizona, or North America, or pretty much the entire Western Hemisphere.

In fact, Homo sapiens were confined to Africa. The hardier Neanderthals had successfully moved into Eurasia, but our lineage was just starting to migrate there. There’s some evidence that we numbered maybe 10,000-15,000 at that point. Far more people saw the comet that night in the United States than our entire population count last time it swung by.

But we were moving up in the world. Our ancestors at the time had developed the first forms of jewelry, using seashells. There’s evidence that we’d learned to trade with other, distant, communities. We were using spears to put dinner on the table with less risk to ourselves than clubs posed.

And, in what’s now Kenya, in the same time frame, a pair of grieving parents carefully buried their 3-year-old child, wrapped in a covering and gently placed on a pillow.

Sadly, this isn’t a scene we’re unfamiliar with. Possibly the most famous painting of a physician is “The Doctor” (1891) by Luke Fildes, showing a physician trying to treat a seriously ill child while the parents look on helplessly.

Tate, London 2017

Dr. Block has a solo neurology practice in Scottsdale, Arizona.

Like millions of other modern humans, my daughter and I stood in the backyard recently and watched comet C/2023 A3 (Tsuchinshan–ATLAS) with binoculars. It took a few minutes to locate, but once you see it is unmistakable.

It’s got a long (at least in human terms) orbit, roughly 80,000 years. So what was going on here, on our pale blue dot, the last time it graced our skies?

Well, here in Phoenix, the people were ... not here. Nor were they in Arizona, or North America, or pretty much the entire Western Hemisphere.

In fact, Homo sapiens were confined to Africa. The hardier Neanderthals had successfully moved into Eurasia, but our lineage was just starting to migrate there. There’s some evidence that we numbered maybe 10,000-15,000 at that point. Far more people saw the comet that night in the United States than our entire population count last time it swung by.

But we were moving up in the world. Our ancestors at the time had developed the first forms of jewelry, using seashells. There’s evidence that we’d learned to trade with other, distant, communities. We were using spears to put dinner on the table with less risk to ourselves than clubs posed.

And, in what’s now Kenya, in the same time frame, a pair of grieving parents carefully buried their 3-year-old child, wrapped in a covering and gently placed on a pillow.

Sadly, this isn’t a scene we’re unfamiliar with. Possibly the most famous painting of a physician is “The Doctor” (1891) by Luke Fildes, showing a physician trying to treat a seriously ill child while the parents look on helplessly.

Tate, London 2017

Dr. Block has a solo neurology practice in Scottsdale, Arizona.

Like millions of other modern humans, my daughter and I stood in the backyard recently and watched comet C/2023 A3 (Tsuchinshan–ATLAS) with binoculars. It took a few minutes to locate, but once you see it is unmistakable.

It’s got a long (at least in human terms) orbit, roughly 80,000 years. So what was going on here, on our pale blue dot, the last time it graced our skies?

Well, here in Phoenix, the people were ... not here. Nor were they in Arizona, or North America, or pretty much the entire Western Hemisphere.

In fact, Homo sapiens were confined to Africa. The hardier Neanderthals had successfully moved into Eurasia, but our lineage was just starting to migrate there. There’s some evidence that we numbered maybe 10,000-15,000 at that point. Far more people saw the comet that night in the United States than our entire population count last time it swung by.

But we were moving up in the world. Our ancestors at the time had developed the first forms of jewelry, using seashells. There’s evidence that we’d learned to trade with other, distant, communities. We were using spears to put dinner on the table with less risk to ourselves than clubs posed.

And, in what’s now Kenya, in the same time frame, a pair of grieving parents carefully buried their 3-year-old child, wrapped in a covering and gently placed on a pillow.

Sadly, this isn’t a scene we’re unfamiliar with. Possibly the most famous painting of a physician is “The Doctor” (1891) by Luke Fildes, showing a physician trying to treat a seriously ill child while the parents look on helplessly.

Tate, London 2017

Dr. Block has a solo neurology practice in Scottsdale, Arizona.

THE DIAGNOSIS: Ectopic (Accessory) Breast Tissue

Ectopic (accessory) breast tissue (EBT) is a phenomenon caused by failed regression of one or more components of the embryonic mammary ridges— paired ectodermal thickenings that eventually develop into definitive breast tissue including the nipples, areolae, and parenchyma. Ectopic breast tissue is more common in women than men and is believed to be sporadic, although an autosomal-dominant inheritance mechanism with incomplete penetrance has been proposed for some cases.¹ The reported incidence of EBT varies greatly among racial and ethnic groups but is most common in individuals of Asian descent. The incidence across all types of EBT is estimated at 0.25% to 6% in the general population.²

Observed clinical variations of EBT range from simple polythelia (additional nipple[s] without associated parenchyma) to complete polymastia (organized and differentiated accessory breasts). Some types of EBT are rarer than others: One report of gynecologic cancer screenings in 1660 patients found polymastia and polythelia incidences of 0.12% and 5.48%, respectively.³ Of the symptomatic variations, isolated parenchymal EBT without a nipple or areolar complex is the most common and may manifest clinically as unilateral or bilateral tender, mildly erythematous nodules or masses often located in the axillae. Ectopic breast tissue generally is observed along the milk line, a developmental regional designation corresponding to the embryologic mammary ridge and extending linearly from the anterior axilla to the inguinal fold on both sides of the body; however, there have been rare reports of EBT manifesting in areas outside the milk line, such as the face, neck, back, vulva, and extremities.^2,3

Given that the underlying elements of EBT usually are hormone responsive (as with normal breast tissue), the initial symptom onset and subsequent manifestation frequently coincide with pubertal milestones, pregnancy, or lactation. Furthermore, some patients with EBT may experience symptom fluctuations in concordance with monthly menstrual phases. Many cases of EBT are selflimited and resolve within weeks to months after the end of a pregnancy or lactation, but some cases may persist. Continued observation and follow-up are advisable in all patients, as EBT symptoms often recur and the tissue is susceptible to the same disease processes that affect normal breasts, the most concerning of which is malignancy.⁴ Although the true incidence is limited by available data, primary ectopic breast malignancy has been estimated to account for 0.3% to 3.8% of diagnosed breast malignancies.² Cases of malignancy arising from EBT often are of higher grade and poorer prognosis, a finding that may be attributable to diagnostic delays caused by oversight or misdiagnosis of EBT rather than inherent differences in the biologic profile of the tumors.^2,4 Patients with a documented history of EBT may benefit from having their routine breast cancer screenings expanded to include areas with EBT foci.

Potential misdiagnoses for EBT include subcutaneous lipoma, axillary lymphadenopathy, abscess, hidradenitis suppurativa, or malignancy. Features that are suggestive of EBT include symptom association with hormone fluctuations (eg, menstrual phases), absence of fever, and lactescent rather than purulent drainage. Among reported EBT cases, spontaneous lactation rarely is described and, if present, often is associated with a history of prior trauma (eg, core needle biopsy or local abscess formation).⁵ This trauma creates an aberrant connection known as a milk fistula between the underlying parenchyma and the skin surface. Interestingly, our patient denied any history of axillary trauma, but she was noted to be lactating from an apparent milk fistula rather than an organized secretory duct system.

Though a patient history and clinical examination may be sufficient to diagnose EBT cases that are more physically apparent and well correlated with hormone fluctuations, many cases require additional diagnostic studies for confirmation. Of the tools available, ultrasonography generally is considered first-line due to its noninvasive nature, low cost, minimal risk, and high diagnostic value.² Ultrasonography quickly differentiates between abscesses and cystlike processes, which may appear as discrete areas of decreased echogenicity, and breast tissue, which manifests with fibroglandular tissue and lobules of fat.^2,6 Additionally, ultrasonography may demonstrate the secretion of milk through ducts or fistulae, if present. Should examination with ultrasonography prove inconclusive, follow-up studies using conventional radiographic mammography or magnetic resonance imaging may be warranted. Biopsy of EBT foci generally is not indicated unless first-line noninvasive studies fail to yield a conclusive diagnosis; however, biopsy also may be warranted if initial imaging is suggestive of malignancy arising from EBT.²

Management of EBT generally is conservative, and symptoms often resolve without intervention.⁴ Symptomatic relief may be achieved through techniques such as application of warm/cold compresses, avoidance of mechanical stimulation, and use of over-the-counter pain medicine. In cases that are persistent, frequently recurrent, or associated with severe symptoms or that cause considerable cosmetic impact, management with surgical excision and/or liposuction may be warranted.⁷ In our patient, the symptoms were not bothersome enough to warrant surgical intervention, so she was managed conservatively and did not return for follow-up.

THE DIAGNOSIS: Ectopic (Accessory) Breast Tissue

Ectopic (accessory) breast tissue (EBT) is a phenomenon caused by failed regression of one or more components of the embryonic mammary ridges— paired ectodermal thickenings that eventually develop into definitive breast tissue including the nipples, areolae, and parenchyma. Ectopic breast tissue is more common in women than men and is believed to be sporadic, although an autosomal-dominant inheritance mechanism with incomplete penetrance has been proposed for some cases.¹ The reported incidence of EBT varies greatly among racial and ethnic groups but is most common in individuals of Asian descent. The incidence across all types of EBT is estimated at 0.25% to 6% in the general population.²

Observed clinical variations of EBT range from simple polythelia (additional nipple[s] without associated parenchyma) to complete polymastia (organized and differentiated accessory breasts). Some types of EBT are rarer than others: One report of gynecologic cancer screenings in 1660 patients found polymastia and polythelia incidences of 0.12% and 5.48%, respectively.³ Of the symptomatic variations, isolated parenchymal EBT without a nipple or areolar complex is the most common and may manifest clinically as unilateral or bilateral tender, mildly erythematous nodules or masses often located in the axillae. Ectopic breast tissue generally is observed along the milk line, a developmental regional designation corresponding to the embryologic mammary ridge and extending linearly from the anterior axilla to the inguinal fold on both sides of the body; however, there have been rare reports of EBT manifesting in areas outside the milk line, such as the face, neck, back, vulva, and extremities.^2,3

Given that the underlying elements of EBT usually are hormone responsive (as with normal breast tissue), the initial symptom onset and subsequent manifestation frequently coincide with pubertal milestones, pregnancy, or lactation. Furthermore, some patients with EBT may experience symptom fluctuations in concordance with monthly menstrual phases. Many cases of EBT are selflimited and resolve within weeks to months after the end of a pregnancy or lactation, but some cases may persist. Continued observation and follow-up are advisable in all patients, as EBT symptoms often recur and the tissue is susceptible to the same disease processes that affect normal breasts, the most concerning of which is malignancy.⁴ Although the true incidence is limited by available data, primary ectopic breast malignancy has been estimated to account for 0.3% to 3.8% of diagnosed breast malignancies.² Cases of malignancy arising from EBT often are of higher grade and poorer prognosis, a finding that may be attributable to diagnostic delays caused by oversight or misdiagnosis of EBT rather than inherent differences in the biologic profile of the tumors.^2,4 Patients with a documented history of EBT may benefit from having their routine breast cancer screenings expanded to include areas with EBT foci.

Potential misdiagnoses for EBT include subcutaneous lipoma, axillary lymphadenopathy, abscess, hidradenitis suppurativa, or malignancy. Features that are suggestive of EBT include symptom association with hormone fluctuations (eg, menstrual phases), absence of fever, and lactescent rather than purulent drainage. Among reported EBT cases, spontaneous lactation rarely is described and, if present, often is associated with a history of prior trauma (eg, core needle biopsy or local abscess formation).⁵ This trauma creates an aberrant connection known as a milk fistula between the underlying parenchyma and the skin surface. Interestingly, our patient denied any history of axillary trauma, but she was noted to be lactating from an apparent milk fistula rather than an organized secretory duct system.

Though a patient history and clinical examination may be sufficient to diagnose EBT cases that are more physically apparent and well correlated with hormone fluctuations, many cases require additional diagnostic studies for confirmation. Of the tools available, ultrasonography generally is considered first-line due to its noninvasive nature, low cost, minimal risk, and high diagnostic value.² Ultrasonography quickly differentiates between abscesses and cystlike processes, which may appear as discrete areas of decreased echogenicity, and breast tissue, which manifests with fibroglandular tissue and lobules of fat.^2,6 Additionally, ultrasonography may demonstrate the secretion of milk through ducts or fistulae, if present. Should examination with ultrasonography prove inconclusive, follow-up studies using conventional radiographic mammography or magnetic resonance imaging may be warranted. Biopsy of EBT foci generally is not indicated unless first-line noninvasive studies fail to yield a conclusive diagnosis; however, biopsy also may be warranted if initial imaging is suggestive of malignancy arising from EBT.²

Management of EBT generally is conservative, and symptoms often resolve without intervention.⁴ Symptomatic relief may be achieved through techniques such as application of warm/cold compresses, avoidance of mechanical stimulation, and use of over-the-counter pain medicine. In cases that are persistent, frequently recurrent, or associated with severe symptoms or that cause considerable cosmetic impact, management with surgical excision and/or liposuction may be warranted.⁷ In our patient, the symptoms were not bothersome enough to warrant surgical intervention, so she was managed conservatively and did not return for follow-up.

THE DIAGNOSIS: Ectopic (Accessory) Breast Tissue

Ectopic (accessory) breast tissue (EBT) is a phenomenon caused by failed regression of one or more components of the embryonic mammary ridges— paired ectodermal thickenings that eventually develop into definitive breast tissue including the nipples, areolae, and parenchyma. Ectopic breast tissue is more common in women than men and is believed to be sporadic, although an autosomal-dominant inheritance mechanism with incomplete penetrance has been proposed for some cases.¹ The reported incidence of EBT varies greatly among racial and ethnic groups but is most common in individuals of Asian descent. The incidence across all types of EBT is estimated at 0.25% to 6% in the general population.²

Observed clinical variations of EBT range from simple polythelia (additional nipple[s] without associated parenchyma) to complete polymastia (organized and differentiated accessory breasts). Some types of EBT are rarer than others: One report of gynecologic cancer screenings in 1660 patients found polymastia and polythelia incidences of 0.12% and 5.48%, respectively.³ Of the symptomatic variations, isolated parenchymal EBT without a nipple or areolar complex is the most common and may manifest clinically as unilateral or bilateral tender, mildly erythematous nodules or masses often located in the axillae. Ectopic breast tissue generally is observed along the milk line, a developmental regional designation corresponding to the embryologic mammary ridge and extending linearly from the anterior axilla to the inguinal fold on both sides of the body; however, there have been rare reports of EBT manifesting in areas outside the milk line, such as the face, neck, back, vulva, and extremities.^2,3

Given that the underlying elements of EBT usually are hormone responsive (as with normal breast tissue), the initial symptom onset and subsequent manifestation frequently coincide with pubertal milestones, pregnancy, or lactation. Furthermore, some patients with EBT may experience symptom fluctuations in concordance with monthly menstrual phases. Many cases of EBT are selflimited and resolve within weeks to months after the end of a pregnancy or lactation, but some cases may persist. Continued observation and follow-up are advisable in all patients, as EBT symptoms often recur and the tissue is susceptible to the same disease processes that affect normal breasts, the most concerning of which is malignancy.⁴ Although the true incidence is limited by available data, primary ectopic breast malignancy has been estimated to account for 0.3% to 3.8% of diagnosed breast malignancies.² Cases of malignancy arising from EBT often are of higher grade and poorer prognosis, a finding that may be attributable to diagnostic delays caused by oversight or misdiagnosis of EBT rather than inherent differences in the biologic profile of the tumors.^2,4 Patients with a documented history of EBT may benefit from having their routine breast cancer screenings expanded to include areas with EBT foci.

Potential misdiagnoses for EBT include subcutaneous lipoma, axillary lymphadenopathy, abscess, hidradenitis suppurativa, or malignancy. Features that are suggestive of EBT include symptom association with hormone fluctuations (eg, menstrual phases), absence of fever, and lactescent rather than purulent drainage. Among reported EBT cases, spontaneous lactation rarely is described and, if present, often is associated with a history of prior trauma (eg, core needle biopsy or local abscess formation).⁵ This trauma creates an aberrant connection known as a milk fistula between the underlying parenchyma and the skin surface. Interestingly, our patient denied any history of axillary trauma, but she was noted to be lactating from an apparent milk fistula rather than an organized secretory duct system.

Though a patient history and clinical examination may be sufficient to diagnose EBT cases that are more physically apparent and well correlated with hormone fluctuations, many cases require additional diagnostic studies for confirmation. Of the tools available, ultrasonography generally is considered first-line due to its noninvasive nature, low cost, minimal risk, and high diagnostic value.² Ultrasonography quickly differentiates between abscesses and cystlike processes, which may appear as discrete areas of decreased echogenicity, and breast tissue, which manifests with fibroglandular tissue and lobules of fat.^2,6 Additionally, ultrasonography may demonstrate the secretion of milk through ducts or fistulae, if present. Should examination with ultrasonography prove inconclusive, follow-up studies using conventional radiographic mammography or magnetic resonance imaging may be warranted. Biopsy of EBT foci generally is not indicated unless first-line noninvasive studies fail to yield a conclusive diagnosis; however, biopsy also may be warranted if initial imaging is suggestive of malignancy arising from EBT.²

Management of EBT generally is conservative, and symptoms often resolve without intervention.⁴ Symptomatic relief may be achieved through techniques such as application of warm/cold compresses, avoidance of mechanical stimulation, and use of over-the-counter pain medicine. In cases that are persistent, frequently recurrent, or associated with severe symptoms or that cause considerable cosmetic impact, management with surgical excision and/or liposuction may be warranted.⁷ In our patient, the symptoms were not bothersome enough to warrant surgical intervention, so she was managed conservatively and did not return for follow-up.

LOS ANGELES — Embedding a new discharge order set into electronic health records (EHRs) with a preselected 5-day antibiotic course for children aged 2 years or older diagnosed with acute otitis media (AOM) cut antibiotic duration sharply, according to new data presented at the Infectious Disease Week (IDWeek) 2024 Annual Meeting.

“We were effectively able to cut antibiotic use in half by shortening the duration of treatment,” said lead author Joana Dimo, DO, a Pediatric Infectious Diseases fellow at the University of Colorado Denver/Children’s Hospital Colorado.

In the United States, 80% of children will experience otitis media during their lifetime. Untreated ear infections can lead to symptoms ranging from mild ear discharge to life-threatening conditions such as mastoiditis and intracranial abscesses.
 

Most Cases Resolve Without Antibiotics

Ear infections “are the leading reason for antibiotic prescriptions in kids,” Dimo noted, adding that 24% of all pediatric antibiotic prescriptions are for AOM. Amoxicillin is the preferred first-line treatment. “Research supports that 75% of children get better on their own without antibiotics, and when needed, short courses of just 5 days are safe and effective.”

Antibiotics can cause side effects such as diarrhea and rashes. “Each additional day of antibiotics that are not needed leads to more side effects,” Dimo said, as well as contributing to antibiotic resistance.

Dimo’s team implemented new EHR order sets across the University of Colorado/Children’s Hospital Colorado health network’s four emergency departments and four urgent care centers and included 31,929 patients in the study.

Then they conducted a retrospective review of patients 61 days to 18 years old who entered those settings and had confirmed AOM between January 2019 through December 2023, before and after the April 2021 intervention. The researchers also developed a guideline on managing ear infections to support clinicians as part of the intervention in December 2022.
 

Compliance Grew From 3% to 83%

Dimo said they found very few clinicians in their study had been prescribing according to current guidelines. Their results showed a jump from 3% to 83% in providers prescribing 5-day durations of antibiotics for children aged 2 years or older after their intervention.

The intervention did not lead to increased treatment failures or complications, she added. The team looked for diagnostic codes for mastoiditis, subperiosteal abscess, petrositis, labyrinthitis, meningitis, and intracranial abscess, and “none of our patients” developed any of those complications, Dimo said.

Dimo said the overall rate of prescribing, however, increased. Finding out why prescribing rates remained high throughout the study, before and after their intervention, is a question they are investigating in future work, she said.
 

Cost-Effective and Scalable

“The benefit of this strategy to other institutions is that it’s not labor-intensive. It’s cost-effective, and it can result in dramatic changes in antibiotic use,” Dimo said.

“In the outpatient setting, there’s still a lot of antibiotics being given unnecessarily to children with acute otitis media,” said William Schaffner, MD, infectious disease specialist at Vanderbilt University School of Medicine in Nashville, Tennessee, who was not part of the research. “The American Academy of Pediatrics has been working on that for about a decade — to get pediatricians attuned to when you use them. Most of these episodes of acute otitis media — it’s now well-established — are due to viral infections.”

He said that some physicians may still be defaulting to the longer doses — up to 10 days — that they may have learned in medical school or residency.

“The data would indicate that 5 days of treatment — when treatment is appropriate — is, in the vast majority of instances, sufficient,” Schaffner said.

The researchers “were remarkably successful,” he said, adding that another question is ripe for research. “They still have to get to this issue of whether all of these antibiotic starts were necessary.”

Not knowing whether antibiotic prescriptions in this study were warranted is a limitation of the study, Dimo said, as was not being able to track whether patients presented to institutions outside their own for a return visit or for complications.

She said she thinks one of the reasons for such a sharp increase in compliance was that clinicians in their system routinely use order sets, so using the new order sets easily became part of their workflow.

Dimo and Schaffner reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

LOS ANGELES — Embedding a new discharge order set into electronic health records (EHRs) with a preselected 5-day antibiotic course for children aged 2 years or older diagnosed with acute otitis media (AOM) cut antibiotic duration sharply, according to new data presented at the Infectious Disease Week (IDWeek) 2024 Annual Meeting.

“We were effectively able to cut antibiotic use in half by shortening the duration of treatment,” said lead author Joana Dimo, DO, a Pediatric Infectious Diseases fellow at the University of Colorado Denver/Children’s Hospital Colorado.

In the United States, 80% of children will experience otitis media during their lifetime. Untreated ear infections can lead to symptoms ranging from mild ear discharge to life-threatening conditions such as mastoiditis and intracranial abscesses.
 

Most Cases Resolve Without Antibiotics

Ear infections “are the leading reason for antibiotic prescriptions in kids,” Dimo noted, adding that 24% of all pediatric antibiotic prescriptions are for AOM. Amoxicillin is the preferred first-line treatment. “Research supports that 75% of children get better on their own without antibiotics, and when needed, short courses of just 5 days are safe and effective.”

Antibiotics can cause side effects such as diarrhea and rashes. “Each additional day of antibiotics that are not needed leads to more side effects,” Dimo said, as well as contributing to antibiotic resistance.

Dimo’s team implemented new EHR order sets across the University of Colorado/Children’s Hospital Colorado health network’s four emergency departments and four urgent care centers and included 31,929 patients in the study.

Then they conducted a retrospective review of patients 61 days to 18 years old who entered those settings and had confirmed AOM between January 2019 through December 2023, before and after the April 2021 intervention. The researchers also developed a guideline on managing ear infections to support clinicians as part of the intervention in December 2022.
 

Compliance Grew From 3% to 83%

Dimo said they found very few clinicians in their study had been prescribing according to current guidelines. Their results showed a jump from 3% to 83% in providers prescribing 5-day durations of antibiotics for children aged 2 years or older after their intervention.

The intervention did not lead to increased treatment failures or complications, she added. The team looked for diagnostic codes for mastoiditis, subperiosteal abscess, petrositis, labyrinthitis, meningitis, and intracranial abscess, and “none of our patients” developed any of those complications, Dimo said.

Dimo said the overall rate of prescribing, however, increased. Finding out why prescribing rates remained high throughout the study, before and after their intervention, is a question they are investigating in future work, she said.
 

Cost-Effective and Scalable

“The benefit of this strategy to other institutions is that it’s not labor-intensive. It’s cost-effective, and it can result in dramatic changes in antibiotic use,” Dimo said.

“In the outpatient setting, there’s still a lot of antibiotics being given unnecessarily to children with acute otitis media,” said William Schaffner, MD, infectious disease specialist at Vanderbilt University School of Medicine in Nashville, Tennessee, who was not part of the research. “The American Academy of Pediatrics has been working on that for about a decade — to get pediatricians attuned to when you use them. Most of these episodes of acute otitis media — it’s now well-established — are due to viral infections.”

He said that some physicians may still be defaulting to the longer doses — up to 10 days — that they may have learned in medical school or residency.

“The data would indicate that 5 days of treatment — when treatment is appropriate — is, in the vast majority of instances, sufficient,” Schaffner said.

The researchers “were remarkably successful,” he said, adding that another question is ripe for research. “They still have to get to this issue of whether all of these antibiotic starts were necessary.”

Not knowing whether antibiotic prescriptions in this study were warranted is a limitation of the study, Dimo said, as was not being able to track whether patients presented to institutions outside their own for a return visit or for complications.

She said she thinks one of the reasons for such a sharp increase in compliance was that clinicians in their system routinely use order sets, so using the new order sets easily became part of their workflow.

Dimo and Schaffner reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

LOS ANGELES — Embedding a new discharge order set into electronic health records (EHRs) with a preselected 5-day antibiotic course for children aged 2 years or older diagnosed with acute otitis media (AOM) cut antibiotic duration sharply, according to new data presented at the Infectious Disease Week (IDWeek) 2024 Annual Meeting.

“We were effectively able to cut antibiotic use in half by shortening the duration of treatment,” said lead author Joana Dimo, DO, a Pediatric Infectious Diseases fellow at the University of Colorado Denver/Children’s Hospital Colorado.

In the United States, 80% of children will experience otitis media during their lifetime. Untreated ear infections can lead to symptoms ranging from mild ear discharge to life-threatening conditions such as mastoiditis and intracranial abscesses.
 

Most Cases Resolve Without Antibiotics

Ear infections “are the leading reason for antibiotic prescriptions in kids,” Dimo noted, adding that 24% of all pediatric antibiotic prescriptions are for AOM. Amoxicillin is the preferred first-line treatment. “Research supports that 75% of children get better on their own without antibiotics, and when needed, short courses of just 5 days are safe and effective.”

Antibiotics can cause side effects such as diarrhea and rashes. “Each additional day of antibiotics that are not needed leads to more side effects,” Dimo said, as well as contributing to antibiotic resistance.

Dimo’s team implemented new EHR order sets across the University of Colorado/Children’s Hospital Colorado health network’s four emergency departments and four urgent care centers and included 31,929 patients in the study.

Then they conducted a retrospective review of patients 61 days to 18 years old who entered those settings and had confirmed AOM between January 2019 through December 2023, before and after the April 2021 intervention. The researchers also developed a guideline on managing ear infections to support clinicians as part of the intervention in December 2022.
 

Compliance Grew From 3% to 83%

Dimo said they found very few clinicians in their study had been prescribing according to current guidelines. Their results showed a jump from 3% to 83% in providers prescribing 5-day durations of antibiotics for children aged 2 years or older after their intervention.

The intervention did not lead to increased treatment failures or complications, she added. The team looked for diagnostic codes for mastoiditis, subperiosteal abscess, petrositis, labyrinthitis, meningitis, and intracranial abscess, and “none of our patients” developed any of those complications, Dimo said.

Dimo said the overall rate of prescribing, however, increased. Finding out why prescribing rates remained high throughout the study, before and after their intervention, is a question they are investigating in future work, she said.
 

Cost-Effective and Scalable

“The benefit of this strategy to other institutions is that it’s not labor-intensive. It’s cost-effective, and it can result in dramatic changes in antibiotic use,” Dimo said.

“In the outpatient setting, there’s still a lot of antibiotics being given unnecessarily to children with acute otitis media,” said William Schaffner, MD, infectious disease specialist at Vanderbilt University School of Medicine in Nashville, Tennessee, who was not part of the research. “The American Academy of Pediatrics has been working on that for about a decade — to get pediatricians attuned to when you use them. Most of these episodes of acute otitis media — it’s now well-established — are due to viral infections.”

He said that some physicians may still be defaulting to the longer doses — up to 10 days — that they may have learned in medical school or residency.

“The data would indicate that 5 days of treatment — when treatment is appropriate — is, in the vast majority of instances, sufficient,” Schaffner said.

The researchers “were remarkably successful,” he said, adding that another question is ripe for research. “They still have to get to this issue of whether all of these antibiotic starts were necessary.”

Not knowing whether antibiotic prescriptions in this study were warranted is a limitation of the study, Dimo said, as was not being able to track whether patients presented to institutions outside their own for a return visit or for complications.

She said she thinks one of the reasons for such a sharp increase in compliance was that clinicians in their system routinely use order sets, so using the new order sets easily became part of their workflow.

Dimo and Schaffner reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

This transcript has been edited for clarity. 

What do we do if we don’t like patients? We take the Hippocratic Oath as young students in Glasgow. We do that just before our graduation ceremony; we hold our hands up and repeat the Hippocratic Oath: “First, do no harm,” and so on.

What happens if we intensely dislike a patient? Is it possible to offer them the very best care? I was thinking back over a long career. I’ve been a cancer doctor for 40 years and I quite like saying that.

I can only think genuinely over a couple of times in which I’ve acted reflexively when a patient has done something awful. The couple of times it happened, it was just terrible racist comments to junior doctors who were with me. Extraordinarily dreadful things such as, “I don’t want to be touched by ...” or something of that sort.

Without really thinking about it, you react as a normal citizen and say, “That’s absolutely awful. Apologize immediately or leave the consultation room, and never ever come back again.” 

I remember that it happened once in Glasgow and once when I was a young professor in Birmingham, and it’s just an automatic gut reaction. The patient got a fright, and I immediately apologized and groveled around. In that relationship, we hold all the power, don’t we? Rather than being gentle about it, I was genuinely angry because of these ridiculous comments. 

Otherwise, I think most of the doctor-patient relationships are predicated on nonromantic love. I think patients want us to love them as one would a son, mother, father, or daughter, because if we do, then we will do better for them and we’ll pull out all the stops. “Placebo” means “I will please.” I think in the vast majority of cases, at least in our National Health Service (NHS), patients come with trust and a sense of wanting to build that relationship. That may be changing, but not for me. 

What about putting the boot on the other foot? What if the patients don’t like us rather than vice versa? As part of our accreditation appraisal process, from time to time we have to take patient surveys as to whether the patients felt that, after they had been seen in a consultation, they were treated with dignity, the quality of information given was appropriate, and they were treated with kindness. 

It’s an excellent exercise. Without bragging about it, patients objectively, according to these measures, appreciate the service that I give. It’s like getting five-star reviews on Trustpilot, or whatever these things are, that allow you to review car salesmen and so on. I have always had five-star reviews across the board. 

That, again, I thought was just a feature of that relationship, of patients wanting to please. These are patients who had been treated, who were in the outpatient department, who were in the midst of battle. Still, the scores are very high. I speak to my colleagues and that’s not uniformly the case. Patients actually do use these feedback forms, I think in a positive rather than negative way, reflecting back on the way that they were treated.

It has caused some of my colleagues to think quite hard about their personal style and approach to patients. That sense of feedback is important. 

What about losing trust? If that’s at the heart of everything that we do, then what would be an objective measure of losing trust? Again, in our healthcare system, it has been exceedingly unusual for a patient to request a second opinion. Now, that’s changing. The government is trying to change it. Leaders of the NHS are trying to change it so that patients feel assured that they can seek second opinions.

Again, in all the years I’ve been a cancer doctor, it has been incredibly infrequent that somebody has sought a second opinion after I’ve said something. That may be a measure of trust. Again, I’ve lived through an NHS in which seeking second opinions was something of a rarity. 

I’d be really interested to see what you think. In your own sphere of healthcare practice, is it possible for us to look after patients that we don’t like, or should we be honest and say, “I don’t like you. Our relationship has broken down. I want you to be seen by a colleague,” or “I want you to be nursed by somebody else”?

Has that happened? Is that something that you think is common or may become more common? What about when trust breaks down the other way? Can you think of instances in which the relationship, for whatever reason, just didn’t work and the patient had to move on because of that loss of trust and what underpinned it? I’d be really interested to know. 

I seek to be informed rather than the other way around. Can we truly look after patients that we don’t like or can we rise above it as Hippocrates might have done? 

Thanks for listening, as always. For the time being, over and out.

Dr. Kerr, Professor, Nuffield Department of Clinical Laboratory Science, University of Oxford; Professor of Cancer Medicine, Oxford Cancer Centre, Oxford, United Kingdom, disclosed ties with Celleron Therapeutics, Oxford Cancer Biomarkers, Afrox, GlaxoSmithKline, Bayer HealthCare Pharmaceuticals, Genomic Health, Merck Serono, and Roche.

A version of this article appeared on Medscape.com.

This transcript has been edited for clarity. 

What do we do if we don’t like patients? We take the Hippocratic Oath as young students in Glasgow. We do that just before our graduation ceremony; we hold our hands up and repeat the Hippocratic Oath: “First, do no harm,” and so on.

What happens if we intensely dislike a patient? Is it possible to offer them the very best care? I was thinking back over a long career. I’ve been a cancer doctor for 40 years and I quite like saying that.

I can only think genuinely over a couple of times in which I’ve acted reflexively when a patient has done something awful. The couple of times it happened, it was just terrible racist comments to junior doctors who were with me. Extraordinarily dreadful things such as, “I don’t want to be touched by ...” or something of that sort.

Without really thinking about it, you react as a normal citizen and say, “That’s absolutely awful. Apologize immediately or leave the consultation room, and never ever come back again.” 

I remember that it happened once in Glasgow and once when I was a young professor in Birmingham, and it’s just an automatic gut reaction. The patient got a fright, and I immediately apologized and groveled around. In that relationship, we hold all the power, don’t we? Rather than being gentle about it, I was genuinely angry because of these ridiculous comments. 

Otherwise, I think most of the doctor-patient relationships are predicated on nonromantic love. I think patients want us to love them as one would a son, mother, father, or daughter, because if we do, then we will do better for them and we’ll pull out all the stops. “Placebo” means “I will please.” I think in the vast majority of cases, at least in our National Health Service (NHS), patients come with trust and a sense of wanting to build that relationship. That may be changing, but not for me. 

What about putting the boot on the other foot? What if the patients don’t like us rather than vice versa? As part of our accreditation appraisal process, from time to time we have to take patient surveys as to whether the patients felt that, after they had been seen in a consultation, they were treated with dignity, the quality of information given was appropriate, and they were treated with kindness. 

It’s an excellent exercise. Without bragging about it, patients objectively, according to these measures, appreciate the service that I give. It’s like getting five-star reviews on Trustpilot, or whatever these things are, that allow you to review car salesmen and so on. I have always had five-star reviews across the board. 

That, again, I thought was just a feature of that relationship, of patients wanting to please. These are patients who had been treated, who were in the outpatient department, who were in the midst of battle. Still, the scores are very high. I speak to my colleagues and that’s not uniformly the case. Patients actually do use these feedback forms, I think in a positive rather than negative way, reflecting back on the way that they were treated.

It has caused some of my colleagues to think quite hard about their personal style and approach to patients. That sense of feedback is important. 

What about losing trust? If that’s at the heart of everything that we do, then what would be an objective measure of losing trust? Again, in our healthcare system, it has been exceedingly unusual for a patient to request a second opinion. Now, that’s changing. The government is trying to change it. Leaders of the NHS are trying to change it so that patients feel assured that they can seek second opinions.

Again, in all the years I’ve been a cancer doctor, it has been incredibly infrequent that somebody has sought a second opinion after I’ve said something. That may be a measure of trust. Again, I’ve lived through an NHS in which seeking second opinions was something of a rarity. 

I’d be really interested to see what you think. In your own sphere of healthcare practice, is it possible for us to look after patients that we don’t like, or should we be honest and say, “I don’t like you. Our relationship has broken down. I want you to be seen by a colleague,” or “I want you to be nursed by somebody else”?

Has that happened? Is that something that you think is common or may become more common? What about when trust breaks down the other way? Can you think of instances in which the relationship, for whatever reason, just didn’t work and the patient had to move on because of that loss of trust and what underpinned it? I’d be really interested to know. 

I seek to be informed rather than the other way around. Can we truly look after patients that we don’t like or can we rise above it as Hippocrates might have done? 

Thanks for listening, as always. For the time being, over and out.

Dr. Kerr, Professor, Nuffield Department of Clinical Laboratory Science, University of Oxford; Professor of Cancer Medicine, Oxford Cancer Centre, Oxford, United Kingdom, disclosed ties with Celleron Therapeutics, Oxford Cancer Biomarkers, Afrox, GlaxoSmithKline, Bayer HealthCare Pharmaceuticals, Genomic Health, Merck Serono, and Roche.

A version of this article appeared on Medscape.com.

This transcript has been edited for clarity. 

What do we do if we don’t like patients? We take the Hippocratic Oath as young students in Glasgow. We do that just before our graduation ceremony; we hold our hands up and repeat the Hippocratic Oath: “First, do no harm,” and so on.

What happens if we intensely dislike a patient? Is it possible to offer them the very best care? I was thinking back over a long career. I’ve been a cancer doctor for 40 years and I quite like saying that.

I can only think genuinely over a couple of times in which I’ve acted reflexively when a patient has done something awful. The couple of times it happened, it was just terrible racist comments to junior doctors who were with me. Extraordinarily dreadful things such as, “I don’t want to be touched by ...” or something of that sort.

Without really thinking about it, you react as a normal citizen and say, “That’s absolutely awful. Apologize immediately or leave the consultation room, and never ever come back again.” 

I remember that it happened once in Glasgow and once when I was a young professor in Birmingham, and it’s just an automatic gut reaction. The patient got a fright, and I immediately apologized and groveled around. In that relationship, we hold all the power, don’t we? Rather than being gentle about it, I was genuinely angry because of these ridiculous comments. 

Otherwise, I think most of the doctor-patient relationships are predicated on nonromantic love. I think patients want us to love them as one would a son, mother, father, or daughter, because if we do, then we will do better for them and we’ll pull out all the stops. “Placebo” means “I will please.” I think in the vast majority of cases, at least in our National Health Service (NHS), patients come with trust and a sense of wanting to build that relationship. That may be changing, but not for me. 

What about putting the boot on the other foot? What if the patients don’t like us rather than vice versa? As part of our accreditation appraisal process, from time to time we have to take patient surveys as to whether the patients felt that, after they had been seen in a consultation, they were treated with dignity, the quality of information given was appropriate, and they were treated with kindness. 

It’s an excellent exercise. Without bragging about it, patients objectively, according to these measures, appreciate the service that I give. It’s like getting five-star reviews on Trustpilot, or whatever these things are, that allow you to review car salesmen and so on. I have always had five-star reviews across the board. 

That, again, I thought was just a feature of that relationship, of patients wanting to please. These are patients who had been treated, who were in the outpatient department, who were in the midst of battle. Still, the scores are very high. I speak to my colleagues and that’s not uniformly the case. Patients actually do use these feedback forms, I think in a positive rather than negative way, reflecting back on the way that they were treated.

It has caused some of my colleagues to think quite hard about their personal style and approach to patients. That sense of feedback is important. 

What about losing trust? If that’s at the heart of everything that we do, then what would be an objective measure of losing trust? Again, in our healthcare system, it has been exceedingly unusual for a patient to request a second opinion. Now, that’s changing. The government is trying to change it. Leaders of the NHS are trying to change it so that patients feel assured that they can seek second opinions.

Again, in all the years I’ve been a cancer doctor, it has been incredibly infrequent that somebody has sought a second opinion after I’ve said something. That may be a measure of trust. Again, I’ve lived through an NHS in which seeking second opinions was something of a rarity. 

I’d be really interested to see what you think. In your own sphere of healthcare practice, is it possible for us to look after patients that we don’t like, or should we be honest and say, “I don’t like you. Our relationship has broken down. I want you to be seen by a colleague,” or “I want you to be nursed by somebody else”?

Has that happened? Is that something that you think is common or may become more common? What about when trust breaks down the other way? Can you think of instances in which the relationship, for whatever reason, just didn’t work and the patient had to move on because of that loss of trust and what underpinned it? I’d be really interested to know. 

I seek to be informed rather than the other way around. Can we truly look after patients that we don’t like or can we rise above it as Hippocrates might have done? 

Thanks for listening, as always. For the time being, over and out.

Dr. Kerr, Professor, Nuffield Department of Clinical Laboratory Science, University of Oxford; Professor of Cancer Medicine, Oxford Cancer Centre, Oxford, United Kingdom, disclosed ties with Celleron Therapeutics, Oxford Cancer Biomarkers, Afrox, GlaxoSmithKline, Bayer HealthCare Pharmaceuticals, Genomic Health, Merck Serono, and Roche.

A version of this article appeared on Medscape.com.

The American Stroke Association (ASA) has issued a new updated guideline for primary stroke prevention.

The first update in a decade, the 2024 Guideline for the Primary Prevention of Stroke, replaces the 2014 version and is intended to be a resource for clinicians to help them implement a variety of prevention strategies in patients with no previous history of stroke. It aligns with the American Heart Association’s Life’s Essential 8.

“This guideline is an important and timely update from 2014 for multiple reasons. First, there have been groundbreaking clinical trials that have been published with new medications to not only treat the target disease [including] diabetes/obesity and high cholesterol], but also lower the risk of stroke and heart disease,” said chair of the guideline writing group, Cheryl D. Bushnell, MD, MHS, FAHA, and vice chair of the research, Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, North Carolina.

It was published online on October 21 in Stroke.
 

Up to 80% of Strokes Preventable

Estimates show that every year in the United States, more than 500,000 individuals have a first stroke. However, the guideline authors noted that up to 80% of strokes may be preventable. As a result, they called for better primary stroke prevention that includes improved screening and lifestyle changes.

This includes adoption of the Mediterranean diet, which has been shown to significantly reduce stroke risk, especially when supplemented with consumption of nuts and olive oil.

The guideline recommendations also emphasize the need for physical activity, which is “essential” for cardiovascular health and stroke reduction. The authors underscored this point and provided a new recommendation to screen for sedentary behavior and advise patients to avoid inactivity and engage in regular moderate to vigorous physical activity.

Another new recommendation is based on “robust” data that glucagon-like peptide 1 receptor agonists (GLP-1s) significantly improve the management of type 2 diabetes, weight loss, and lower the risk for cardiovascular disease. As a result, guideline authors called for the use of GLP-1s in patients with diabetes and high cardiovascular risk or established cardiovascular disease.

“The glucagon-like peptide receptor agonists have been shown to not only drastically reduce blood sugars in patients with diabetes, but they also lead to significant weight loss in these patients, which has many downstream benefits. Together, this reduces the risk of stroke and other complications of diabetes,” said Bushnell. 

She also noted that another drug class introduced since the 2014 guidelines were published, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, have proven to be highly effective in lowering low-density lipoprotein cholesterol. These medications have also been shown to reduce the risk for stroke.
 

At Least Two Meds Needed to Reduce BP

Effective blood pressure (BP) management is crucial for stroke prevention. Randomized controlled trials show that a single antihypertensive medication helps only about 30% of participants reach their BP target.

Most patients only achieve the desired BP target with two to three medications. In line with these data, the committee recommends using at least two antihypertensives for primary stroke prevention in most patients who require BP-lowering medications for hypertension.

In patients with antiphospholipid syndrome or systemic lupus erythematosus and no history of stroke or unprovoked venous thromboembolism, the authors recommended antiplatelet therapy to prevent stroke. They added that patients with antiphospholipid syndrome who have had a prior unprovoked venous thrombosis will likely benefit from vitamin K antagonist therapy (target international normalized ratio, 2-3) over direct oral anticoagulants.
 

Emphasis on Women’s Health

Preventing pregnancy-related stroke is achieved primarily by managing hypertension, the guideline authors noted. They recommended treating verified systolic BP over 160 mm Hg or diastolic BP over 110 mm Hg during pregnancy and up to 6 weeks postpartum to lower the risk for fatal maternal intracerebral hemorrhage.

They noted that adverse pregnancy outcomes are also common and linked to chronic hypertension, which increases stroke risk later in life. Therefore, they recommended screening for these outcomes to assess and manage vascular risk factors. The guideline includes a screening tool to help with this in clinical practice.

Endometriosis, premature ovarian failure (before age 40 years), and early-onset menopause (before age 45 years) are all associated with increased stroke risk. As a result, the guideline authors said screening for all three of these conditions is a “reasonable step in the evaluation and management of vascular risk factors in these individuals to reduce stroke risk.”

Finally, the guideline authors addressed primary stroke prevention in transgender individuals, noting that transgender women undergoing estrogen therapy for gender affirmation are at increased risk. They emphasized that evaluating and modifying risk factors could be beneficial for reducing stroke risk in this patient population.
 

Challenges Lie Ahead

Now that the guideline has been published, the challenge lies in determining how best to implement “its screening recommendations in primary care and other practices when these clinicians are already pushed to see as many patients as possible,” Bushnell said.

Development of screening tools that can be easily incorporated into the clinic visit or the electronic health record, as well as additional personnel to provide counseling, are probably needed to disseminate them, she added. 

Bushnell also emphasized that the guideline includes a strong focus on social determinants of health and related social needs. 

“We worked hard to use inclusive language and to consider populations historically excluded from research. In acknowledging that social determinants of health including access to healthcare, access to education, economic stability, neighborhood and geographic location, and social and community context have a tremendous influence on stroke risk, we describe how these factors are closely tied to the prevalence and management of many medical risks like obesity, hypertension, and diabetes.

“Our recommendations offer practical steps for screening and addressing essential health-related social needs, including access to nutritious food, stable housing, and reliable transportation, within clinical practice. By considering these factors more comprehensively, we believe we can make meaningful strides toward reducing the disparities in stroke risk,” said Bushnell. 
 

A version of this article appeared on Medscape.com.

The American Stroke Association (ASA) has issued a new updated guideline for primary stroke prevention.

The first update in a decade, the 2024 Guideline for the Primary Prevention of Stroke, replaces the 2014 version and is intended to be a resource for clinicians to help them implement a variety of prevention strategies in patients with no previous history of stroke. It aligns with the American Heart Association’s Life’s Essential 8.

“This guideline is an important and timely update from 2014 for multiple reasons. First, there have been groundbreaking clinical trials that have been published with new medications to not only treat the target disease [including] diabetes/obesity and high cholesterol], but also lower the risk of stroke and heart disease,” said chair of the guideline writing group, Cheryl D. Bushnell, MD, MHS, FAHA, and vice chair of the research, Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, North Carolina.

It was published online on October 21 in Stroke.
 

Up to 80% of Strokes Preventable

Estimates show that every year in the United States, more than 500,000 individuals have a first stroke. However, the guideline authors noted that up to 80% of strokes may be preventable. As a result, they called for better primary stroke prevention that includes improved screening and lifestyle changes.

This includes adoption of the Mediterranean diet, which has been shown to significantly reduce stroke risk, especially when supplemented with consumption of nuts and olive oil.

The guideline recommendations also emphasize the need for physical activity, which is “essential” for cardiovascular health and stroke reduction. The authors underscored this point and provided a new recommendation to screen for sedentary behavior and advise patients to avoid inactivity and engage in regular moderate to vigorous physical activity.

Another new recommendation is based on “robust” data that glucagon-like peptide 1 receptor agonists (GLP-1s) significantly improve the management of type 2 diabetes, weight loss, and lower the risk for cardiovascular disease. As a result, guideline authors called for the use of GLP-1s in patients with diabetes and high cardiovascular risk or established cardiovascular disease.

“The glucagon-like peptide receptor agonists have been shown to not only drastically reduce blood sugars in patients with diabetes, but they also lead to significant weight loss in these patients, which has many downstream benefits. Together, this reduces the risk of stroke and other complications of diabetes,” said Bushnell. 

She also noted that another drug class introduced since the 2014 guidelines were published, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, have proven to be highly effective in lowering low-density lipoprotein cholesterol. These medications have also been shown to reduce the risk for stroke.
 

At Least Two Meds Needed to Reduce BP

Effective blood pressure (BP) management is crucial for stroke prevention. Randomized controlled trials show that a single antihypertensive medication helps only about 30% of participants reach their BP target.

Most patients only achieve the desired BP target with two to three medications. In line with these data, the committee recommends using at least two antihypertensives for primary stroke prevention in most patients who require BP-lowering medications for hypertension.

In patients with antiphospholipid syndrome or systemic lupus erythematosus and no history of stroke or unprovoked venous thromboembolism, the authors recommended antiplatelet therapy to prevent stroke. They added that patients with antiphospholipid syndrome who have had a prior unprovoked venous thrombosis will likely benefit from vitamin K antagonist therapy (target international normalized ratio, 2-3) over direct oral anticoagulants.
 

Emphasis on Women’s Health

Preventing pregnancy-related stroke is achieved primarily by managing hypertension, the guideline authors noted. They recommended treating verified systolic BP over 160 mm Hg or diastolic BP over 110 mm Hg during pregnancy and up to 6 weeks postpartum to lower the risk for fatal maternal intracerebral hemorrhage.

They noted that adverse pregnancy outcomes are also common and linked to chronic hypertension, which increases stroke risk later in life. Therefore, they recommended screening for these outcomes to assess and manage vascular risk factors. The guideline includes a screening tool to help with this in clinical practice.

Endometriosis, premature ovarian failure (before age 40 years), and early-onset menopause (before age 45 years) are all associated with increased stroke risk. As a result, the guideline authors said screening for all three of these conditions is a “reasonable step in the evaluation and management of vascular risk factors in these individuals to reduce stroke risk.”

Finally, the guideline authors addressed primary stroke prevention in transgender individuals, noting that transgender women undergoing estrogen therapy for gender affirmation are at increased risk. They emphasized that evaluating and modifying risk factors could be beneficial for reducing stroke risk in this patient population.
 

Challenges Lie Ahead

Now that the guideline has been published, the challenge lies in determining how best to implement “its screening recommendations in primary care and other practices when these clinicians are already pushed to see as many patients as possible,” Bushnell said.

Development of screening tools that can be easily incorporated into the clinic visit or the electronic health record, as well as additional personnel to provide counseling, are probably needed to disseminate them, she added. 

Bushnell also emphasized that the guideline includes a strong focus on social determinants of health and related social needs. 

“We worked hard to use inclusive language and to consider populations historically excluded from research. In acknowledging that social determinants of health including access to healthcare, access to education, economic stability, neighborhood and geographic location, and social and community context have a tremendous influence on stroke risk, we describe how these factors are closely tied to the prevalence and management of many medical risks like obesity, hypertension, and diabetes.

“Our recommendations offer practical steps for screening and addressing essential health-related social needs, including access to nutritious food, stable housing, and reliable transportation, within clinical practice. By considering these factors more comprehensively, we believe we can make meaningful strides toward reducing the disparities in stroke risk,” said Bushnell. 
 

A version of this article appeared on Medscape.com.

The American Stroke Association (ASA) has issued a new updated guideline for primary stroke prevention.

The first update in a decade, the 2024 Guideline for the Primary Prevention of Stroke, replaces the 2014 version and is intended to be a resource for clinicians to help them implement a variety of prevention strategies in patients with no previous history of stroke. It aligns with the American Heart Association’s Life’s Essential 8.

“This guideline is an important and timely update from 2014 for multiple reasons. First, there have been groundbreaking clinical trials that have been published with new medications to not only treat the target disease [including] diabetes/obesity and high cholesterol], but also lower the risk of stroke and heart disease,” said chair of the guideline writing group, Cheryl D. Bushnell, MD, MHS, FAHA, and vice chair of the research, Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, North Carolina.

It was published online on October 21 in Stroke.
 

Up to 80% of Strokes Preventable

Estimates show that every year in the United States, more than 500,000 individuals have a first stroke. However, the guideline authors noted that up to 80% of strokes may be preventable. As a result, they called for better primary stroke prevention that includes improved screening and lifestyle changes.

This includes adoption of the Mediterranean diet, which has been shown to significantly reduce stroke risk, especially when supplemented with consumption of nuts and olive oil.

The guideline recommendations also emphasize the need for physical activity, which is “essential” for cardiovascular health and stroke reduction. The authors underscored this point and provided a new recommendation to screen for sedentary behavior and advise patients to avoid inactivity and engage in regular moderate to vigorous physical activity.

Another new recommendation is based on “robust” data that glucagon-like peptide 1 receptor agonists (GLP-1s) significantly improve the management of type 2 diabetes, weight loss, and lower the risk for cardiovascular disease. As a result, guideline authors called for the use of GLP-1s in patients with diabetes and high cardiovascular risk or established cardiovascular disease.

“The glucagon-like peptide receptor agonists have been shown to not only drastically reduce blood sugars in patients with diabetes, but they also lead to significant weight loss in these patients, which has many downstream benefits. Together, this reduces the risk of stroke and other complications of diabetes,” said Bushnell. 

She also noted that another drug class introduced since the 2014 guidelines were published, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, have proven to be highly effective in lowering low-density lipoprotein cholesterol. These medications have also been shown to reduce the risk for stroke.
 

At Least Two Meds Needed to Reduce BP

Effective blood pressure (BP) management is crucial for stroke prevention. Randomized controlled trials show that a single antihypertensive medication helps only about 30% of participants reach their BP target.

Most patients only achieve the desired BP target with two to three medications. In line with these data, the committee recommends using at least two antihypertensives for primary stroke prevention in most patients who require BP-lowering medications for hypertension.

In patients with antiphospholipid syndrome or systemic lupus erythematosus and no history of stroke or unprovoked venous thromboembolism, the authors recommended antiplatelet therapy to prevent stroke. They added that patients with antiphospholipid syndrome who have had a prior unprovoked venous thrombosis will likely benefit from vitamin K antagonist therapy (target international normalized ratio, 2-3) over direct oral anticoagulants.
 

Emphasis on Women’s Health

Preventing pregnancy-related stroke is achieved primarily by managing hypertension, the guideline authors noted. They recommended treating verified systolic BP over 160 mm Hg or diastolic BP over 110 mm Hg during pregnancy and up to 6 weeks postpartum to lower the risk for fatal maternal intracerebral hemorrhage.

They noted that adverse pregnancy outcomes are also common and linked to chronic hypertension, which increases stroke risk later in life. Therefore, they recommended screening for these outcomes to assess and manage vascular risk factors. The guideline includes a screening tool to help with this in clinical practice.

Endometriosis, premature ovarian failure (before age 40 years), and early-onset menopause (before age 45 years) are all associated with increased stroke risk. As a result, the guideline authors said screening for all three of these conditions is a “reasonable step in the evaluation and management of vascular risk factors in these individuals to reduce stroke risk.”

Finally, the guideline authors addressed primary stroke prevention in transgender individuals, noting that transgender women undergoing estrogen therapy for gender affirmation are at increased risk. They emphasized that evaluating and modifying risk factors could be beneficial for reducing stroke risk in this patient population.
 

Challenges Lie Ahead

Now that the guideline has been published, the challenge lies in determining how best to implement “its screening recommendations in primary care and other practices when these clinicians are already pushed to see as many patients as possible,” Bushnell said.

Development of screening tools that can be easily incorporated into the clinic visit or the electronic health record, as well as additional personnel to provide counseling, are probably needed to disseminate them, she added. 

Bushnell also emphasized that the guideline includes a strong focus on social determinants of health and related social needs. 

“We worked hard to use inclusive language and to consider populations historically excluded from research. In acknowledging that social determinants of health including access to healthcare, access to education, economic stability, neighborhood and geographic location, and social and community context have a tremendous influence on stroke risk, we describe how these factors are closely tied to the prevalence and management of many medical risks like obesity, hypertension, and diabetes.

“Our recommendations offer practical steps for screening and addressing essential health-related social needs, including access to nutritious food, stable housing, and reliable transportation, within clinical practice. By considering these factors more comprehensively, we believe we can make meaningful strides toward reducing the disparities in stroke risk,” said Bushnell. 
 

A version of this article appeared on Medscape.com.

A new study provides more evidence that glucagon-like peptide 1 receptor agonists (GLP-1 RAs) used to treat diabetes and obesity could be repurposed for opioid use disorder (OUD) and alcohol use disorder (AUD).

Researchers found that patients with OUD or AUD who were taking semaglutide (Ozempic, Novo Nordisk) or similar medications for diabetes or weight-related conditions had a 40% lower rate of opioid overdose and a 50% lower rate of alcohol intoxication than their peers with OUD or AUD who were not taking these medications.

Their real-world study of more than 1 million adults with a history of OUD or AUD provide “foundational” estimates of the association between glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 RA prescriptions and opioid overdose/alcohol intoxication “and introduce the idea that GLP-1 RA and other related drugs should be investigated as a novel pharmacotherapy treatment option for individuals with OUD or AUD,” wrote the investigators, led by Fares Qeadan, PhD, Parkinson School of Health Sciences and Public Health, Loyola University Chicago, Maywood, Illinois.

The study was published online in the journal Addiction.
 

Protective Effect?

As previously reported by Medscape Medical News, earlier studies have pointed to a link between weight loss drugs and reduced overdose risk in people with OUD and decreased alcohol intake in people with AUD.

Until now, most studies on GLP-1 RAs and GIP agonists like tirzepatide (Mounjaro) to treat substance use disorders consisted of animal studies and small-scale clinical trials, investigators noted.

This new retrospective cohort study analyzed de-identified electronic health record data from the Oracle Health Real-World Data.

Participants, all aged 18 years or older, included 503,747 patients with a history of OUD, of whom 8103 had a GLP-1 RA or GIP prescription, and 817,309 patients with a history of AUD, of whom 5621 had a GLP-1 RA or GIP prescription.

Patients with OUD who were prescribed GLP-1 RAs had a 40% lower rate of opioid overdose than those without such prescriptions (adjusted incidence rate ratio [aIRR], 0.60; 95% CI, 0.43-0.83), the study team found.

In addition, patients with AUD and a GLP-1 RA prescription exhibited a 50% lower rate of alcohol intoxication (aIRR, 0.50; 95% CI, 0.40-0.63).

The protective effect of GLP-1 RA on opioid overdose and alcohol intoxication was maintained across patients with comorbid conditions, such as type 2 diabetes and obesity.

“Future research should focus on prospective clinical trials to validate these findings, explore the underlying mechanisms, and determine the long-term efficacy and safety of GIP/GLP-1 RA medications in diverse populations,” Qeadan and colleagues concluded.

“Additionally, the study highlights the importance of interdisciplinary research in understanding the neurobiological links between metabolic disorders and problematic substance use, potentially leading to more effective treatment strategies within healthcare systems,” they added.
 

Questions Remain

In a statement from the UK nonprofit Science Media Centre, Matt Field, DPhil, professor of psychology, The University of Sheffield, in England, noted that the findings “add to those from other studies, particularly animal research, which suggest that this and similar drugs might one day be prescribed to help people with addiction.”

However, “a note of caution is that the outcomes are very extreme instances of substance intoxication,” added Field, who wasn’t involved in the study.

“Those outcomes are very different from the outcomes used when researchers test new treatments for addiction, in which case we might look at whether the treatment helps people to stop taking the substance altogether (complete abstinence), or if it helps people to reduce the amount of substance they consume, or how often they consume it. Those things could not be measured in this study,” he continued.

“This leaves open the possibility that while Ozempic may — for reasons currently unknown — prevent people from taking so much alcohol or heroin that they overdose and end up in hospital, it may not actually help them to reduce their substance use, or to abstain altogether,” Field said.

The study had no specific funding. The study authors and Field declared no relevant conflicts of interest.
 

A version of this article first appeared on Medscape.com.

A new study provides more evidence that glucagon-like peptide 1 receptor agonists (GLP-1 RAs) used to treat diabetes and obesity could be repurposed for opioid use disorder (OUD) and alcohol use disorder (AUD).

Researchers found that patients with OUD or AUD who were taking semaglutide (Ozempic, Novo Nordisk) or similar medications for diabetes or weight-related conditions had a 40% lower rate of opioid overdose and a 50% lower rate of alcohol intoxication than their peers with OUD or AUD who were not taking these medications.

Their real-world study of more than 1 million adults with a history of OUD or AUD provide “foundational” estimates of the association between glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 RA prescriptions and opioid overdose/alcohol intoxication “and introduce the idea that GLP-1 RA and other related drugs should be investigated as a novel pharmacotherapy treatment option for individuals with OUD or AUD,” wrote the investigators, led by Fares Qeadan, PhD, Parkinson School of Health Sciences and Public Health, Loyola University Chicago, Maywood, Illinois.

The study was published online in the journal Addiction.
 

Protective Effect?

As previously reported by Medscape Medical News, earlier studies have pointed to a link between weight loss drugs and reduced overdose risk in people with OUD and decreased alcohol intake in people with AUD.

Until now, most studies on GLP-1 RAs and GIP agonists like tirzepatide (Mounjaro) to treat substance use disorders consisted of animal studies and small-scale clinical trials, investigators noted.

This new retrospective cohort study analyzed de-identified electronic health record data from the Oracle Health Real-World Data.

Participants, all aged 18 years or older, included 503,747 patients with a history of OUD, of whom 8103 had a GLP-1 RA or GIP prescription, and 817,309 patients with a history of AUD, of whom 5621 had a GLP-1 RA or GIP prescription.

Patients with OUD who were prescribed GLP-1 RAs had a 40% lower rate of opioid overdose than those without such prescriptions (adjusted incidence rate ratio [aIRR], 0.60; 95% CI, 0.43-0.83), the study team found.

In addition, patients with AUD and a GLP-1 RA prescription exhibited a 50% lower rate of alcohol intoxication (aIRR, 0.50; 95% CI, 0.40-0.63).

The protective effect of GLP-1 RA on opioid overdose and alcohol intoxication was maintained across patients with comorbid conditions, such as type 2 diabetes and obesity.

“Future research should focus on prospective clinical trials to validate these findings, explore the underlying mechanisms, and determine the long-term efficacy and safety of GIP/GLP-1 RA medications in diverse populations,” Qeadan and colleagues concluded.

“Additionally, the study highlights the importance of interdisciplinary research in understanding the neurobiological links between metabolic disorders and problematic substance use, potentially leading to more effective treatment strategies within healthcare systems,” they added.
 

Questions Remain

In a statement from the UK nonprofit Science Media Centre, Matt Field, DPhil, professor of psychology, The University of Sheffield, in England, noted that the findings “add to those from other studies, particularly animal research, which suggest that this and similar drugs might one day be prescribed to help people with addiction.”

However, “a note of caution is that the outcomes are very extreme instances of substance intoxication,” added Field, who wasn’t involved in the study.

“Those outcomes are very different from the outcomes used when researchers test new treatments for addiction, in which case we might look at whether the treatment helps people to stop taking the substance altogether (complete abstinence), or if it helps people to reduce the amount of substance they consume, or how often they consume it. Those things could not be measured in this study,” he continued.

“This leaves open the possibility that while Ozempic may — for reasons currently unknown — prevent people from taking so much alcohol or heroin that they overdose and end up in hospital, it may not actually help them to reduce their substance use, or to abstain altogether,” Field said.

The study had no specific funding. The study authors and Field declared no relevant conflicts of interest.
 

A version of this article first appeared on Medscape.com.

A new study provides more evidence that glucagon-like peptide 1 receptor agonists (GLP-1 RAs) used to treat diabetes and obesity could be repurposed for opioid use disorder (OUD) and alcohol use disorder (AUD).

Researchers found that patients with OUD or AUD who were taking semaglutide (Ozempic, Novo Nordisk) or similar medications for diabetes or weight-related conditions had a 40% lower rate of opioid overdose and a 50% lower rate of alcohol intoxication than their peers with OUD or AUD who were not taking these medications.

Their real-world study of more than 1 million adults with a history of OUD or AUD provide “foundational” estimates of the association between glucose-dependent insulinotropic polypeptide (GIP)/GLP-1 RA prescriptions and opioid overdose/alcohol intoxication “and introduce the idea that GLP-1 RA and other related drugs should be investigated as a novel pharmacotherapy treatment option for individuals with OUD or AUD,” wrote the investigators, led by Fares Qeadan, PhD, Parkinson School of Health Sciences and Public Health, Loyola University Chicago, Maywood, Illinois.

The study was published online in the journal Addiction.
 

Protective Effect?

As previously reported by Medscape Medical News, earlier studies have pointed to a link between weight loss drugs and reduced overdose risk in people with OUD and decreased alcohol intake in people with AUD.

Until now, most studies on GLP-1 RAs and GIP agonists like tirzepatide (Mounjaro) to treat substance use disorders consisted of animal studies and small-scale clinical trials, investigators noted.

This new retrospective cohort study analyzed de-identified electronic health record data from the Oracle Health Real-World Data.

Participants, all aged 18 years or older, included 503,747 patients with a history of OUD, of whom 8103 had a GLP-1 RA or GIP prescription, and 817,309 patients with a history of AUD, of whom 5621 had a GLP-1 RA or GIP prescription.

Patients with OUD who were prescribed GLP-1 RAs had a 40% lower rate of opioid overdose than those without such prescriptions (adjusted incidence rate ratio [aIRR], 0.60; 95% CI, 0.43-0.83), the study team found.

In addition, patients with AUD and a GLP-1 RA prescription exhibited a 50% lower rate of alcohol intoxication (aIRR, 0.50; 95% CI, 0.40-0.63).

The protective effect of GLP-1 RA on opioid overdose and alcohol intoxication was maintained across patients with comorbid conditions, such as type 2 diabetes and obesity.

“Future research should focus on prospective clinical trials to validate these findings, explore the underlying mechanisms, and determine the long-term efficacy and safety of GIP/GLP-1 RA medications in diverse populations,” Qeadan and colleagues concluded.

“Additionally, the study highlights the importance of interdisciplinary research in understanding the neurobiological links between metabolic disorders and problematic substance use, potentially leading to more effective treatment strategies within healthcare systems,” they added.
 

Questions Remain

In a statement from the UK nonprofit Science Media Centre, Matt Field, DPhil, professor of psychology, The University of Sheffield, in England, noted that the findings “add to those from other studies, particularly animal research, which suggest that this and similar drugs might one day be prescribed to help people with addiction.”

However, “a note of caution is that the outcomes are very extreme instances of substance intoxication,” added Field, who wasn’t involved in the study.

“Those outcomes are very different from the outcomes used when researchers test new treatments for addiction, in which case we might look at whether the treatment helps people to stop taking the substance altogether (complete abstinence), or if it helps people to reduce the amount of substance they consume, or how often they consume it. Those things could not be measured in this study,” he continued.

“This leaves open the possibility that while Ozempic may — for reasons currently unknown — prevent people from taking so much alcohol or heroin that they overdose and end up in hospital, it may not actually help them to reduce their substance use, or to abstain altogether,” Field said.

The study had no specific funding. The study authors and Field declared no relevant conflicts of interest.
 

A version of this article first appeared on Medscape.com.