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Also today, disease-modifying therapies and stem cell transplants both reduce disease progression in MS, the American Academy of Pediatrics guidelines on hemangioma should empower primary care clinicians, and a treat-to-target approach for CVD risk factors decreased atherosclerosis in patients with rheumatoid arthritis.
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Also today, disease-modifying therapies and stem cell transplants both reduce disease progression in MS, the American Academy of Pediatrics guidelines on hemangioma should empower primary care clinicians, and a treat-to-target approach for CVD risk factors decreased atherosclerosis in patients with rheumatoid arthritis.
Amazon Alexa
Apple Podcasts
Google Podcasts
Spotify
Also today, disease-modifying therapies and stem cell transplants both reduce disease progression in MS, the American Academy of Pediatrics guidelines on hemangioma should empower primary care clinicians, and a treat-to-target approach for CVD risk factors decreased atherosclerosis in patients with rheumatoid arthritis.
Amazon Alexa
Apple Podcasts
Google Podcasts
Spotify
Frailty may affect the expression of dementia
according to research published online ahead of print Jan. 17 in Lancet Neurology. Data suggest that frailty reduces the threshold for Alzheimer’s disease pathology to cause cognitive decline. Frailty also may contribute to other mechanisms that cause dementia, such as inflammation and immunosenescence, said the investigators.
“While more research is needed, given that frailty is potentially reversible, it is possible that helping people to maintain function and independence in later life could reduce both dementia risk and the severity of debilitating symptoms common in this disease,” said Professor Kenneth Rockwood, MD, of the Nova Scotia Health Authority and Dalhousie University in Halifax, N.S., in a press release.
More susceptible to dementia?
The presence of amyloid plaques and neurofibrillary tangles is not a sufficient condition for the clinical expression of dementia. Some patients with a high degree of Alzheimer’s disease pathology have no apparent cognitive decline. Other factors therefore may modify the relationship between pathology and dementia.
Most people who develop Alzheimer’s disease dementia are older than 65 years, and many of these patients are frail. Frailty is understood as a decreased physiologic reserve and an increased risk for adverse health outcomes. Dr. Rockwood and his colleagues hypothesized that frailty moderates the clinical expression of dementia in relation to Alzheimer’s disease pathology.
To test their hypothesis, the investigators performed a cross-sectional analysis of data from the Rush Memory and Aging Project, which collects clinical and pathologic data from adults older than 59 years without dementia at baseline who live in Illinois. Since 1997, participants have undergone annual clinical and neuropsychological evaluations, and the cohort has been followed for 21 years. For their analysis, Dr. Rockwood and his colleagues included participants without dementia or with Alzheimer’s dementia at their last clinical assessment. Eligible participants had died, and complete autopsy data were available for them.
The researchers measured Alzheimer’s disease pathology using a summary measure of neurofibrillary tangles and neuritic and diffuse plaques. Clinical diagnoses of Alzheimer’s dementia were based on clinician consensus. Dr. Rockwood and his colleagues retrospectively created a 41-item frailty index from variables (e.g., symptoms, signs, comorbidities, and function) that were obtained at each clinical evaluation.
Logistic regression and moderation modeling allowed the investigators to evaluate relationships between Alzheimer’s disease pathology, frailty, and Alzheimer’s dementia. Dr. Rockwood and hus colleagues adjusted all analyses for age, sex, and education.
In all, 456 participants were included in the analysis. The sample’s mean age at death was 89.7 years, and 69% of participants were women. At participants’ last clinical assessment, 242 (53%) had possible or probable Alzheimer’s dementia.
The sample’s mean frailty index was 0.42. The median frailty index was 0.41, a value similar to the threshold commonly used to distinguish between moderate and severe frailty. People with high frailty index scores (i.e., 0.41 or greater) were older, had lower Mini-Mental State Examination scores, were more likely to have a diagnosis of dementia, and had a higher Braak stage than those with moderate or low frailty index scores.
Significant interaction between frailty and Alzheimer’s disease
After the investigators adjusted for age, sex, and education, frailty (odds ratio, 1.76) and Alzheimer’s disease pathology (OR, 4.81) were independently associated with Alzheimer’s dementia. When the investigators added frailty to the model for the relationship between Alzheimer’s disease pathology and Alzheimer’s dementia, the model fit improved. They found a significant interaction between frailty and Alzheimer’s disease pathology (OR, 0.73). People with a low amount of frailty were better able to tolerate Alzheimer’s disease pathology, and people with higher amounts of frailty were more likely to have more Alzheimer’s disease pathology and clinical dementia.
One of the study’s limitations is that it is a secondary analysis, according to Dr. Rockwood and his colleagues. In addition, frailty was measured close to participants’ time of death, and the measurements may thus reflect terminal decline. Participant deaths resulting from causes other than those related to dementia might have confounded the results. Finally, the sample came entirely from people living in retirement homes in Illinois, which might have introduced bias. Future research should use a population-based sample, said the authors.
Frailty could be a basis for risk stratification and could inform the management and treatment of older adults, said Dr. Rockwood and his colleagues. The study results have “the potential to improve our understanding of disease expression, explain failures in pharmacologic treatment, and aid in the development of more appropriate therapeutic targets, approaches, and measurements of success,” they concluded.
The study had no source of funding. The authors reported receiving fees and grants from DGI Clinical, GlaxoSmithKline, Pfizer, and Sanofi. Authors also received support from governmental bodies such as the National Institutes of Health and the Canadian Institutes of Health Research.
SOURCE: Wallace LMK et al. Lancet Neurol. 2019;18:177-84.
The results of the study by Rockwood and colleagues confirm the strong links between frailty and Alzheimer’s disease and other dementias, said Francesco Panza, MD, PhD, of the University of Bari (Italy) Aldo Moro, and his colleagues in an accompanying editorial.
Frailty is primary or preclinical when it is not directly associated with a specific disease or when the patient has no substantial disability. Frailty is considered secondary or clinical when it is associated with known comorbidities (e.g., cardiovascular disease or depression). “This distinction is central in identifying frailty phenotypes with the potential to predict and prevent dementia, using novel models of risk that introduce modifiable factors,” wrote Dr. Panza and his colleagues.
“In light of current knowledge on the cognitive frailty phenotype, secondary preventive strategies for cognitive impairment and physical frailty can be suggested,” they added. “For instance, individualized multidomain interventions can target physical, nutritional, cognitive, and psychological domains that might delay the progression to overt dementia and secondary occurrence of adverse health-related outcomes, such as disability, hospitalization, and mortality.”
Dr. Panza, Madia Lozupone, MD, PhD , and Giancarlo Logroscino, MD, PhD , are affiliated with the neurodegenerative disease unit in the department of basic medicine, neuroscience, and sense organs at the University of Bari (Italy) Aldo Moro. The above remarks come from an editorial that these authors wrote to accompany the study by Rockwood et al. The authors declared no competing interests.
The results of the study by Rockwood and colleagues confirm the strong links between frailty and Alzheimer’s disease and other dementias, said Francesco Panza, MD, PhD, of the University of Bari (Italy) Aldo Moro, and his colleagues in an accompanying editorial.
Frailty is primary or preclinical when it is not directly associated with a specific disease or when the patient has no substantial disability. Frailty is considered secondary or clinical when it is associated with known comorbidities (e.g., cardiovascular disease or depression). “This distinction is central in identifying frailty phenotypes with the potential to predict and prevent dementia, using novel models of risk that introduce modifiable factors,” wrote Dr. Panza and his colleagues.
“In light of current knowledge on the cognitive frailty phenotype, secondary preventive strategies for cognitive impairment and physical frailty can be suggested,” they added. “For instance, individualized multidomain interventions can target physical, nutritional, cognitive, and psychological domains that might delay the progression to overt dementia and secondary occurrence of adverse health-related outcomes, such as disability, hospitalization, and mortality.”
Dr. Panza, Madia Lozupone, MD, PhD , and Giancarlo Logroscino, MD, PhD , are affiliated with the neurodegenerative disease unit in the department of basic medicine, neuroscience, and sense organs at the University of Bari (Italy) Aldo Moro. The above remarks come from an editorial that these authors wrote to accompany the study by Rockwood et al. The authors declared no competing interests.
The results of the study by Rockwood and colleagues confirm the strong links between frailty and Alzheimer’s disease and other dementias, said Francesco Panza, MD, PhD, of the University of Bari (Italy) Aldo Moro, and his colleagues in an accompanying editorial.
Frailty is primary or preclinical when it is not directly associated with a specific disease or when the patient has no substantial disability. Frailty is considered secondary or clinical when it is associated with known comorbidities (e.g., cardiovascular disease or depression). “This distinction is central in identifying frailty phenotypes with the potential to predict and prevent dementia, using novel models of risk that introduce modifiable factors,” wrote Dr. Panza and his colleagues.
“In light of current knowledge on the cognitive frailty phenotype, secondary preventive strategies for cognitive impairment and physical frailty can be suggested,” they added. “For instance, individualized multidomain interventions can target physical, nutritional, cognitive, and psychological domains that might delay the progression to overt dementia and secondary occurrence of adverse health-related outcomes, such as disability, hospitalization, and mortality.”
Dr. Panza, Madia Lozupone, MD, PhD , and Giancarlo Logroscino, MD, PhD , are affiliated with the neurodegenerative disease unit in the department of basic medicine, neuroscience, and sense organs at the University of Bari (Italy) Aldo Moro. The above remarks come from an editorial that these authors wrote to accompany the study by Rockwood et al. The authors declared no competing interests.
according to research published online ahead of print Jan. 17 in Lancet Neurology. Data suggest that frailty reduces the threshold for Alzheimer’s disease pathology to cause cognitive decline. Frailty also may contribute to other mechanisms that cause dementia, such as inflammation and immunosenescence, said the investigators.
“While more research is needed, given that frailty is potentially reversible, it is possible that helping people to maintain function and independence in later life could reduce both dementia risk and the severity of debilitating symptoms common in this disease,” said Professor Kenneth Rockwood, MD, of the Nova Scotia Health Authority and Dalhousie University in Halifax, N.S., in a press release.
More susceptible to dementia?
The presence of amyloid plaques and neurofibrillary tangles is not a sufficient condition for the clinical expression of dementia. Some patients with a high degree of Alzheimer’s disease pathology have no apparent cognitive decline. Other factors therefore may modify the relationship between pathology and dementia.
Most people who develop Alzheimer’s disease dementia are older than 65 years, and many of these patients are frail. Frailty is understood as a decreased physiologic reserve and an increased risk for adverse health outcomes. Dr. Rockwood and his colleagues hypothesized that frailty moderates the clinical expression of dementia in relation to Alzheimer’s disease pathology.
To test their hypothesis, the investigators performed a cross-sectional analysis of data from the Rush Memory and Aging Project, which collects clinical and pathologic data from adults older than 59 years without dementia at baseline who live in Illinois. Since 1997, participants have undergone annual clinical and neuropsychological evaluations, and the cohort has been followed for 21 years. For their analysis, Dr. Rockwood and his colleagues included participants without dementia or with Alzheimer’s dementia at their last clinical assessment. Eligible participants had died, and complete autopsy data were available for them.
The researchers measured Alzheimer’s disease pathology using a summary measure of neurofibrillary tangles and neuritic and diffuse plaques. Clinical diagnoses of Alzheimer’s dementia were based on clinician consensus. Dr. Rockwood and his colleagues retrospectively created a 41-item frailty index from variables (e.g., symptoms, signs, comorbidities, and function) that were obtained at each clinical evaluation.
Logistic regression and moderation modeling allowed the investigators to evaluate relationships between Alzheimer’s disease pathology, frailty, and Alzheimer’s dementia. Dr. Rockwood and hus colleagues adjusted all analyses for age, sex, and education.
In all, 456 participants were included in the analysis. The sample’s mean age at death was 89.7 years, and 69% of participants were women. At participants’ last clinical assessment, 242 (53%) had possible or probable Alzheimer’s dementia.
The sample’s mean frailty index was 0.42. The median frailty index was 0.41, a value similar to the threshold commonly used to distinguish between moderate and severe frailty. People with high frailty index scores (i.e., 0.41 or greater) were older, had lower Mini-Mental State Examination scores, were more likely to have a diagnosis of dementia, and had a higher Braak stage than those with moderate or low frailty index scores.
Significant interaction between frailty and Alzheimer’s disease
After the investigators adjusted for age, sex, and education, frailty (odds ratio, 1.76) and Alzheimer’s disease pathology (OR, 4.81) were independently associated with Alzheimer’s dementia. When the investigators added frailty to the model for the relationship between Alzheimer’s disease pathology and Alzheimer’s dementia, the model fit improved. They found a significant interaction between frailty and Alzheimer’s disease pathology (OR, 0.73). People with a low amount of frailty were better able to tolerate Alzheimer’s disease pathology, and people with higher amounts of frailty were more likely to have more Alzheimer’s disease pathology and clinical dementia.
One of the study’s limitations is that it is a secondary analysis, according to Dr. Rockwood and his colleagues. In addition, frailty was measured close to participants’ time of death, and the measurements may thus reflect terminal decline. Participant deaths resulting from causes other than those related to dementia might have confounded the results. Finally, the sample came entirely from people living in retirement homes in Illinois, which might have introduced bias. Future research should use a population-based sample, said the authors.
Frailty could be a basis for risk stratification and could inform the management and treatment of older adults, said Dr. Rockwood and his colleagues. The study results have “the potential to improve our understanding of disease expression, explain failures in pharmacologic treatment, and aid in the development of more appropriate therapeutic targets, approaches, and measurements of success,” they concluded.
The study had no source of funding. The authors reported receiving fees and grants from DGI Clinical, GlaxoSmithKline, Pfizer, and Sanofi. Authors also received support from governmental bodies such as the National Institutes of Health and the Canadian Institutes of Health Research.
SOURCE: Wallace LMK et al. Lancet Neurol. 2019;18:177-84.
according to research published online ahead of print Jan. 17 in Lancet Neurology. Data suggest that frailty reduces the threshold for Alzheimer’s disease pathology to cause cognitive decline. Frailty also may contribute to other mechanisms that cause dementia, such as inflammation and immunosenescence, said the investigators.
“While more research is needed, given that frailty is potentially reversible, it is possible that helping people to maintain function and independence in later life could reduce both dementia risk and the severity of debilitating symptoms common in this disease,” said Professor Kenneth Rockwood, MD, of the Nova Scotia Health Authority and Dalhousie University in Halifax, N.S., in a press release.
More susceptible to dementia?
The presence of amyloid plaques and neurofibrillary tangles is not a sufficient condition for the clinical expression of dementia. Some patients with a high degree of Alzheimer’s disease pathology have no apparent cognitive decline. Other factors therefore may modify the relationship between pathology and dementia.
Most people who develop Alzheimer’s disease dementia are older than 65 years, and many of these patients are frail. Frailty is understood as a decreased physiologic reserve and an increased risk for adverse health outcomes. Dr. Rockwood and his colleagues hypothesized that frailty moderates the clinical expression of dementia in relation to Alzheimer’s disease pathology.
To test their hypothesis, the investigators performed a cross-sectional analysis of data from the Rush Memory and Aging Project, which collects clinical and pathologic data from adults older than 59 years without dementia at baseline who live in Illinois. Since 1997, participants have undergone annual clinical and neuropsychological evaluations, and the cohort has been followed for 21 years. For their analysis, Dr. Rockwood and his colleagues included participants without dementia or with Alzheimer’s dementia at their last clinical assessment. Eligible participants had died, and complete autopsy data were available for them.
The researchers measured Alzheimer’s disease pathology using a summary measure of neurofibrillary tangles and neuritic and diffuse plaques. Clinical diagnoses of Alzheimer’s dementia were based on clinician consensus. Dr. Rockwood and his colleagues retrospectively created a 41-item frailty index from variables (e.g., symptoms, signs, comorbidities, and function) that were obtained at each clinical evaluation.
Logistic regression and moderation modeling allowed the investigators to evaluate relationships between Alzheimer’s disease pathology, frailty, and Alzheimer’s dementia. Dr. Rockwood and hus colleagues adjusted all analyses for age, sex, and education.
In all, 456 participants were included in the analysis. The sample’s mean age at death was 89.7 years, and 69% of participants were women. At participants’ last clinical assessment, 242 (53%) had possible or probable Alzheimer’s dementia.
The sample’s mean frailty index was 0.42. The median frailty index was 0.41, a value similar to the threshold commonly used to distinguish between moderate and severe frailty. People with high frailty index scores (i.e., 0.41 or greater) were older, had lower Mini-Mental State Examination scores, were more likely to have a diagnosis of dementia, and had a higher Braak stage than those with moderate or low frailty index scores.
Significant interaction between frailty and Alzheimer’s disease
After the investigators adjusted for age, sex, and education, frailty (odds ratio, 1.76) and Alzheimer’s disease pathology (OR, 4.81) were independently associated with Alzheimer’s dementia. When the investigators added frailty to the model for the relationship between Alzheimer’s disease pathology and Alzheimer’s dementia, the model fit improved. They found a significant interaction between frailty and Alzheimer’s disease pathology (OR, 0.73). People with a low amount of frailty were better able to tolerate Alzheimer’s disease pathology, and people with higher amounts of frailty were more likely to have more Alzheimer’s disease pathology and clinical dementia.
One of the study’s limitations is that it is a secondary analysis, according to Dr. Rockwood and his colleagues. In addition, frailty was measured close to participants’ time of death, and the measurements may thus reflect terminal decline. Participant deaths resulting from causes other than those related to dementia might have confounded the results. Finally, the sample came entirely from people living in retirement homes in Illinois, which might have introduced bias. Future research should use a population-based sample, said the authors.
Frailty could be a basis for risk stratification and could inform the management and treatment of older adults, said Dr. Rockwood and his colleagues. The study results have “the potential to improve our understanding of disease expression, explain failures in pharmacologic treatment, and aid in the development of more appropriate therapeutic targets, approaches, and measurements of success,” they concluded.
The study had no source of funding. The authors reported receiving fees and grants from DGI Clinical, GlaxoSmithKline, Pfizer, and Sanofi. Authors also received support from governmental bodies such as the National Institutes of Health and the Canadian Institutes of Health Research.
SOURCE: Wallace LMK et al. Lancet Neurol. 2019;18:177-84.
FROM LANCET NEUROLOGY
Key clinical point: Frailty modifies the association between Alzheimer’s disease pathology and Alzheimer dementia.
Major finding: Frailty index score (odds ratio, 1.76) is independently associated with dementia status.
Study details: A cross-sectional analysis of 456 deceased participants in the Rush Memory and Aging Project.
Disclosures: The study had no outside funding.
Source: Wallace LMK et al. Lancet Neurol. 2019;18:177-84.
GoFundMe CEO: ‘Gigantic gaps’ in health system showing up in crowdfunding
Scrolling through the GoFundMe website reveals seemingly an endless number of people who need help or community support. A common theme: the cost of health care.
It didn’t start out this way. Back in 2010, when the crowdfunding website began, it suggested fundraisers for “ideas and dreams,” “wedding donations and honeymoon registry” or “special occasions.” A spokeswoman said the bulk of collection efforts from the first year were “related to charities and foundations.” A category for medical needs existed, but it was farther down the list.
In the 9 years since, campaigns to pay for health care have reaped the most cash. Of the $5 billion the company says it has raised, about a third has been for medical expenses from more than 250,000 medical campaigns conducted annually.
Take, for instance, the 25-year-old California woman who had a stroke and “needs financial support for rehabilitation, home nursing, medical equipment, and uncovered medical expenses.” Or the Tennessee couple who want to get pregnant, but whose insurance doesn’t cover the $20,000 worth of “medications, surgeries, scans, lab monitoring, and appointments [that] will need to be paid for upfront and out-of-pocket” for in vitro fertilization.
The prominence of the medical category is the symptom of a broken system, according to CEO Rob Solomon, 51, who has a long tech résumé as an executive at places like Groupon and Yahoo. He said he never realized how hard it was for some people to pay their bills: “I needed to understand the gigantic gaps in the system.”
This year, Time Magazine named Mr. Solomon one of the 50 most influential people in health care.
“We didn’t build the platform to focus on medical expenses,” Mr. Solomon said. But it turned out, he said, to be one of those “categories of need” with which many people struggle.
Mr. Solomon talked to Kaiser Health News’ Rachel Bluth about his company’s role in financing health care and what it says about the system when so many people rely on the kindness of strangers to get treatment. The conversation has been edited for length and clarity.
Q: KHN and other news outlets have reported that hospitals often advise patients to crowdfund their transplants. It’s become almost institutionalized to use GoFundMe. How do you feel about that?
It saddens me that this is a reality. Every single day on GoFundMe we see the huge challenges people face. Their stories are heartbreaking.
Some progress has been made here and there with the Affordable Care Act, and it’s under fire, but there’s ever-widening gaps in coverage for treatment, for prescriptions, for everything related to health care costs. Even patients who have insurance and supposedly decent insurance [come up short]. We’ve become an indispensable institution, indispensable technology, and indispensable platform for anyone who finds themselves needing help because there just isn’t adequate coverage or assistance.
I would love nothing more than for “medical” to not be a category on GoFundMe. The reality is, though, that access to health care is connected to the ability to pay for it. If you can’t do that, people die. People suffer. We feel good that our platform is there when people need it.
Q: Did anyone expect medical funding would become such a big part of GoFundMe?
I don’t think anyone anticipated it. What we realized early on is that medical need is a gigantic category.
A lot of insurance doesn’t cover clinical trials and research and things like that, where people need access to leading-edge potential treatments. We strive to fill these gaps until the institutions that are supposed to handle this handle it properly. There has to be a renaissance, a dramatic change in public policy, in how the government focuses on this and how the health care companies solve this.
This is very interesting. In the places like the United Kingdom, Canada, and other European countries that have some form of universal or government-sponsored health coverage, medical [costs] are still the largest category. So it’s not just medical bills for treatment. There’s travel and accommodations for families who have to support people when they fall ill.
Q: What have you learned that you didn’t know before?
I guess what I realized [when I came] to this job is that I had no notion of how severe the problem is. You read about the debate about single-payer health care and all the issues, the partisan politics. What I really learned is the health care system in the United States is really broken. Way too many people fall through the cracks.
The government is supposed to be there, and sometimes they are. The health care companies are supposed to be there, and sometimes they are. But for literally millions of people they’re not. The only thing you can really do is rely on the kindness of friends and family and community. That’s where GoFundMe comes in.
I was not ready for that at all when I started at the company. When you live and breathe it every day and you see the need that exists, when you realize there are many people with rare diseases but they aren’t diseases a drug company can make money from, they’re just left with nothing.
Q: But what does this say about the system?
The system is terrible. It needs to be rethought and retooled. Politicians are failing us. Health care companies are failing us. Those are realities. I don’t want to mince words here. We are facing a huge potential tragedy. We provide relief for a lot of people. But there are people who are not getting relief from us or from the institutions that are supposed to be there. We shouldn’t be the solution to a complex set of systemic problems. They should be solved by the government working properly, and by health care companies working with their constituents. We firmly believe that access to comprehensive health care is a right and things have to be fixed at the local, state, and federal levels of government to make this a reality.
Q: Do you ever worry that medical fundraising on your site is taking away from other causes or other things that need to be funded?
We have billions being raised on our platform on an annual basis. Everything from medical, memorial, and emergency to people funding Little League teams and community projects.
Another thing that’s happened in the last few years is we’ve really become the “take action button.” Whenever there’s a news cycle on something where people want to help, they create GoFundMe campaigns. This government shutdown, for example: We have over a thousand campaigns right now for people who have been affected by it – they’re raising money for people to pay rent, mortgages, car payments while the government isn’t.
Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Scrolling through the GoFundMe website reveals seemingly an endless number of people who need help or community support. A common theme: the cost of health care.
It didn’t start out this way. Back in 2010, when the crowdfunding website began, it suggested fundraisers for “ideas and dreams,” “wedding donations and honeymoon registry” or “special occasions.” A spokeswoman said the bulk of collection efforts from the first year were “related to charities and foundations.” A category for medical needs existed, but it was farther down the list.
In the 9 years since, campaigns to pay for health care have reaped the most cash. Of the $5 billion the company says it has raised, about a third has been for medical expenses from more than 250,000 medical campaigns conducted annually.
Take, for instance, the 25-year-old California woman who had a stroke and “needs financial support for rehabilitation, home nursing, medical equipment, and uncovered medical expenses.” Or the Tennessee couple who want to get pregnant, but whose insurance doesn’t cover the $20,000 worth of “medications, surgeries, scans, lab monitoring, and appointments [that] will need to be paid for upfront and out-of-pocket” for in vitro fertilization.
The prominence of the medical category is the symptom of a broken system, according to CEO Rob Solomon, 51, who has a long tech résumé as an executive at places like Groupon and Yahoo. He said he never realized how hard it was for some people to pay their bills: “I needed to understand the gigantic gaps in the system.”
This year, Time Magazine named Mr. Solomon one of the 50 most influential people in health care.
“We didn’t build the platform to focus on medical expenses,” Mr. Solomon said. But it turned out, he said, to be one of those “categories of need” with which many people struggle.
Mr. Solomon talked to Kaiser Health News’ Rachel Bluth about his company’s role in financing health care and what it says about the system when so many people rely on the kindness of strangers to get treatment. The conversation has been edited for length and clarity.
Q: KHN and other news outlets have reported that hospitals often advise patients to crowdfund their transplants. It’s become almost institutionalized to use GoFundMe. How do you feel about that?
It saddens me that this is a reality. Every single day on GoFundMe we see the huge challenges people face. Their stories are heartbreaking.
Some progress has been made here and there with the Affordable Care Act, and it’s under fire, but there’s ever-widening gaps in coverage for treatment, for prescriptions, for everything related to health care costs. Even patients who have insurance and supposedly decent insurance [come up short]. We’ve become an indispensable institution, indispensable technology, and indispensable platform for anyone who finds themselves needing help because there just isn’t adequate coverage or assistance.
I would love nothing more than for “medical” to not be a category on GoFundMe. The reality is, though, that access to health care is connected to the ability to pay for it. If you can’t do that, people die. People suffer. We feel good that our platform is there when people need it.
Q: Did anyone expect medical funding would become such a big part of GoFundMe?
I don’t think anyone anticipated it. What we realized early on is that medical need is a gigantic category.
A lot of insurance doesn’t cover clinical trials and research and things like that, where people need access to leading-edge potential treatments. We strive to fill these gaps until the institutions that are supposed to handle this handle it properly. There has to be a renaissance, a dramatic change in public policy, in how the government focuses on this and how the health care companies solve this.
This is very interesting. In the places like the United Kingdom, Canada, and other European countries that have some form of universal or government-sponsored health coverage, medical [costs] are still the largest category. So it’s not just medical bills for treatment. There’s travel and accommodations for families who have to support people when they fall ill.
Q: What have you learned that you didn’t know before?
I guess what I realized [when I came] to this job is that I had no notion of how severe the problem is. You read about the debate about single-payer health care and all the issues, the partisan politics. What I really learned is the health care system in the United States is really broken. Way too many people fall through the cracks.
The government is supposed to be there, and sometimes they are. The health care companies are supposed to be there, and sometimes they are. But for literally millions of people they’re not. The only thing you can really do is rely on the kindness of friends and family and community. That’s where GoFundMe comes in.
I was not ready for that at all when I started at the company. When you live and breathe it every day and you see the need that exists, when you realize there are many people with rare diseases but they aren’t diseases a drug company can make money from, they’re just left with nothing.
Q: But what does this say about the system?
The system is terrible. It needs to be rethought and retooled. Politicians are failing us. Health care companies are failing us. Those are realities. I don’t want to mince words here. We are facing a huge potential tragedy. We provide relief for a lot of people. But there are people who are not getting relief from us or from the institutions that are supposed to be there. We shouldn’t be the solution to a complex set of systemic problems. They should be solved by the government working properly, and by health care companies working with their constituents. We firmly believe that access to comprehensive health care is a right and things have to be fixed at the local, state, and federal levels of government to make this a reality.
Q: Do you ever worry that medical fundraising on your site is taking away from other causes or other things that need to be funded?
We have billions being raised on our platform on an annual basis. Everything from medical, memorial, and emergency to people funding Little League teams and community projects.
Another thing that’s happened in the last few years is we’ve really become the “take action button.” Whenever there’s a news cycle on something where people want to help, they create GoFundMe campaigns. This government shutdown, for example: We have over a thousand campaigns right now for people who have been affected by it – they’re raising money for people to pay rent, mortgages, car payments while the government isn’t.
Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Scrolling through the GoFundMe website reveals seemingly an endless number of people who need help or community support. A common theme: the cost of health care.
It didn’t start out this way. Back in 2010, when the crowdfunding website began, it suggested fundraisers for “ideas and dreams,” “wedding donations and honeymoon registry” or “special occasions.” A spokeswoman said the bulk of collection efforts from the first year were “related to charities and foundations.” A category for medical needs existed, but it was farther down the list.
In the 9 years since, campaigns to pay for health care have reaped the most cash. Of the $5 billion the company says it has raised, about a third has been for medical expenses from more than 250,000 medical campaigns conducted annually.
Take, for instance, the 25-year-old California woman who had a stroke and “needs financial support for rehabilitation, home nursing, medical equipment, and uncovered medical expenses.” Or the Tennessee couple who want to get pregnant, but whose insurance doesn’t cover the $20,000 worth of “medications, surgeries, scans, lab monitoring, and appointments [that] will need to be paid for upfront and out-of-pocket” for in vitro fertilization.
The prominence of the medical category is the symptom of a broken system, according to CEO Rob Solomon, 51, who has a long tech résumé as an executive at places like Groupon and Yahoo. He said he never realized how hard it was for some people to pay their bills: “I needed to understand the gigantic gaps in the system.”
This year, Time Magazine named Mr. Solomon one of the 50 most influential people in health care.
“We didn’t build the platform to focus on medical expenses,” Mr. Solomon said. But it turned out, he said, to be one of those “categories of need” with which many people struggle.
Mr. Solomon talked to Kaiser Health News’ Rachel Bluth about his company’s role in financing health care and what it says about the system when so many people rely on the kindness of strangers to get treatment. The conversation has been edited for length and clarity.
Q: KHN and other news outlets have reported that hospitals often advise patients to crowdfund their transplants. It’s become almost institutionalized to use GoFundMe. How do you feel about that?
It saddens me that this is a reality. Every single day on GoFundMe we see the huge challenges people face. Their stories are heartbreaking.
Some progress has been made here and there with the Affordable Care Act, and it’s under fire, but there’s ever-widening gaps in coverage for treatment, for prescriptions, for everything related to health care costs. Even patients who have insurance and supposedly decent insurance [come up short]. We’ve become an indispensable institution, indispensable technology, and indispensable platform for anyone who finds themselves needing help because there just isn’t adequate coverage or assistance.
I would love nothing more than for “medical” to not be a category on GoFundMe. The reality is, though, that access to health care is connected to the ability to pay for it. If you can’t do that, people die. People suffer. We feel good that our platform is there when people need it.
Q: Did anyone expect medical funding would become such a big part of GoFundMe?
I don’t think anyone anticipated it. What we realized early on is that medical need is a gigantic category.
A lot of insurance doesn’t cover clinical trials and research and things like that, where people need access to leading-edge potential treatments. We strive to fill these gaps until the institutions that are supposed to handle this handle it properly. There has to be a renaissance, a dramatic change in public policy, in how the government focuses on this and how the health care companies solve this.
This is very interesting. In the places like the United Kingdom, Canada, and other European countries that have some form of universal or government-sponsored health coverage, medical [costs] are still the largest category. So it’s not just medical bills for treatment. There’s travel and accommodations for families who have to support people when they fall ill.
Q: What have you learned that you didn’t know before?
I guess what I realized [when I came] to this job is that I had no notion of how severe the problem is. You read about the debate about single-payer health care and all the issues, the partisan politics. What I really learned is the health care system in the United States is really broken. Way too many people fall through the cracks.
The government is supposed to be there, and sometimes they are. The health care companies are supposed to be there, and sometimes they are. But for literally millions of people they’re not. The only thing you can really do is rely on the kindness of friends and family and community. That’s where GoFundMe comes in.
I was not ready for that at all when I started at the company. When you live and breathe it every day and you see the need that exists, when you realize there are many people with rare diseases but they aren’t diseases a drug company can make money from, they’re just left with nothing.
Q: But what does this say about the system?
The system is terrible. It needs to be rethought and retooled. Politicians are failing us. Health care companies are failing us. Those are realities. I don’t want to mince words here. We are facing a huge potential tragedy. We provide relief for a lot of people. But there are people who are not getting relief from us or from the institutions that are supposed to be there. We shouldn’t be the solution to a complex set of systemic problems. They should be solved by the government working properly, and by health care companies working with their constituents. We firmly believe that access to comprehensive health care is a right and things have to be fixed at the local, state, and federal levels of government to make this a reality.
Q: Do you ever worry that medical fundraising on your site is taking away from other causes or other things that need to be funded?
We have billions being raised on our platform on an annual basis. Everything from medical, memorial, and emergency to people funding Little League teams and community projects.
Another thing that’s happened in the last few years is we’ve really become the “take action button.” Whenever there’s a news cycle on something where people want to help, they create GoFundMe campaigns. This government shutdown, for example: We have over a thousand campaigns right now for people who have been affected by it – they’re raising money for people to pay rent, mortgages, car payments while the government isn’t.
Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Mandatory reporting laws
Question: You are moonlighting in the emergency department and have just finished treating a 5-year-old boy with an apparent Colles’ fracture, who was accompanied by his mother with bruises on her face. Her exam revealed additional bruises over her abdominal wall. The mother said they accidentally tripped and fell down the stairs, and spontaneously denied any acts of violence in the family.
Given this scenario, which of the following is best?
A. You suspect both child and spousal abuse, but lack sufficient evidence to report the incident.
B. Failure to report based on reasonable suspicion alone may amount to a criminal offense punishable by possible imprisonment.
C. You may face a potential malpractice lawsuit if subsequent injuries caused by abuse could have been prevented had you reported.
D. Mandatory reporting laws apply not only to abuse of children and spouses, but also of the elderly and other vulnerable adults.
E. All are correct except A.
Answer: E. All doctors, especially those working in emergency departments, treat injuries on a regular basis. Accidents probably account for the majority of these injuries, but the most pernicious are those caused by willful abuse or neglect. Such conduct, believed to be widespread and underrecognized, victimizes children, women, the elderly, and other vulnerable groups.
Mandatory reporting laws arose from the need to identify and prevent these activities that cause serious harm and loss of lives. Physicians and other health care workers are in a prime position to diagnose or raise the suspicion of abuse and neglect. This article focuses on laws that mandate physician reporting of such behavior. Not addressed are other reportable situations such as certain infectious diseases, gunshot wounds, threats to third parties, and so on.
Child abuse
The best-known example of a mandatory reporting law relates to child abuse, which is broadly defined as when a parent or caretaker emotionally, physically, or sexually abuses, neglects, or abandons a child. Child abuse laws are intended to protect children from serious harm without abridging parental discipline of their children.
Cases of child abuse are pervasive; four or five children are tragically killed by abuse or neglect every day, and each year, some 6 million children are reported as victims of child abuse. Henry Kempe’s studies on the “battered child syndrome” in 1962 served to underscore the physician’s role in exposing child maltreatment, and 1973 saw the enactment of the Child Abuse Prevention and Treatment Act, which set standards for mandatory reporting as a condition for federal funding.
All U.S. states have statutes identifying persons who are required to report suspected child maltreatment to an appropriate agency, such as child protective services. Reasonable suspicion, without need for proof, is sufficient to trigger the mandatory reporting duty. A summary of the general reporting requirements, as well as each state’s key statutory features, are available at Child Welfare Information Gateway.1
Bruises, fractures, and burns are recurring examples of injuries resulting from child abuse, but there are many others, including severe emotional harm, which is an important consequence. Clues to abuse include a child’s fearful and anxious demeanor, wearing clothes to hide injuries, and inappropriate sexual conduct.2 The perpetrators and/or complicit parties typically blame an innocent home accident for the victim’s injuries to mislead the health care provider.
Elder abuse
Elder abuse is broadly construed to include physical, sexual, and psychological abuse, as well as financial exploitation and caregiver neglect.3 It is a serious problem in the United States, estimated in 2008 to affect 1 in 10 elders. The figure is likely an underestimate, because many elderly victims are afraid or unwilling to lodge a complaint against the abuser whom they love and may depend upon.4
The law, which protects the “elderly” (e.g., those aged 62 years or older in Hawaii), may also be extended to other younger vulnerable adults, who because of an impairment, are unable to 1) communicate or make responsible decisions to manage one’s own care or resources, 2) carry out or arrange for essential activities of daily living, or 3) protect one’s self from abuse.5
The law mandates reporting where there is reason to believe abuse has occurred or the vulnerable adult is in danger of abuse if immediate action is not taken. Reporting statutes for elder abuse vary somewhat on the identity of mandated reporters (health care providers are always included), the victim’s mental capacity, dwelling place (home or in an assisted-living facility), and type of purported activity that warrants reporting.
Domestic violence
As defined by the National Coalition Against Domestic Violence, “Domestic violence is the willful intimidation, physical assault, battery, sexual assault, and/or other abusive behavior as part of a systematic pattern of power and control perpetrated by one intimate partner against another. ... The frequency and severity of domestic violence can vary dramatically; however, the one constant component of domestic violence is one partner’s consistent efforts to maintain power and control over the other.”6 Domestic violence is said to have reached epidemic proportions, with one in four women experiencing it at some point in her life.
Virtually all states mandate the reporting of domestic violence by health care providers if there is a reasonable suspicion that observed patient injuries are the result of physical abuse.7 California, for example, requires the provider to call local law enforcement as soon as possible or to send in a written report within 48 hours.
There may be exceptions to required reporting, as when an adult victim withholds consent but accepts victim referral services. State laws encourage but do not always require that the health care provider inform the patient about the report, but federal law dictates otherwise unless this puts the patient at risk. Hawaii’s domestic violence laws were originally enacted to deter spousal abuse, but they now also protect other household members.8
Any individual who assumes a duty or responsibility pursuant to all of these reporting laws is immunized from criminal or civil liability. On the other hand, a mandated reporter who knowingly fails to report an incident or who willfully prevents another person from reporting such an incident commits a criminal offence.
In the case of a physician, there is the added risk of a malpractice lawsuit based on “violation of statute” (breach of a legal duty), should another injury occur down the road that was arguably preventable by his or her failure to report.
Experts generally believe that mandatory reporting laws are important in identifying child maltreatment. However, it has been asserted that despite a 5-decade history of mandatory reporting, no clear endpoints attest to the efficacy of this approach, and it is argued that no data exist to demonstrate that incremental increases in reporting have contributed to child safety.
Particularly challenging are attempts at impact comparisons between states with different policies. A number of countries, including the United Kingdom, do not have mandatory reporting laws and regulate reporting by professional societies.9
In addition, some critics of mandatory reporting raise concerns surrounding law enforcement showing up at the victim’s house to question the family about abuse, or to make an arrest or issue warnings. They posit that when the behavior of an abuser is under scrutiny, this can paradoxically create a potentially more dangerous environment for the patient-victim, whom the perpetrator now considers to have betrayed his or her trust. Others bemoan that revealing patient confidences violates the physician’s ethical code.
However, the intolerable incidence of violence against the vulnerable has properly made mandatory reporting the law of the land. Although the criminal penalty is currently light for failure to report, there is a move toward increasing its severity. Hawaii, for example, recently introduced Senate Bill 2477 that makes nonreporting by those required to do so a Class C felony punishable by up to 5 years in prison. The offense currently is a petty misdemeanor punishable by up to 30 days in jail.
Dr. Tan is emeritus professor of medicine and former adjunct professor of law at the University of Hawaii, Honolulu. This article is meant to be educational and does not constitute medical, ethical, or legal advice. For additional information, readers may contact the author at siang@hawaii.edu.
References
1. Child Welfare Information Gateway (2016). Mandatory reporters of child abuse and neglect. Washington, D.C.: U.S. Department of Health and Human Services, Children’s Bureau. Available at www.childwelfare.gov; email: info@childwelfare.gov; phone: 800-394-3366.
2. Available at www.childwelfare.gov/topics/can.
3. Available at www.justice.gov/elderjustice/elder-justice-statutes-0.
4. Available at www.cdc.gov/violenceprevention/elderabuse/index.html.
5. Hawaii Revised Statutes, Sec. 346-222, 346-224, 346-250, 412:3-114.5.
6. Available at ncadv.org.
7. Ann Emerg Med. 2002 Jan;39(1):56-60.
8. Hawaii Revised Statutes, Sec. 709-906.
9. Pediatrics. 2017 Apr;139(4). pii: e20163511.
Question: You are moonlighting in the emergency department and have just finished treating a 5-year-old boy with an apparent Colles’ fracture, who was accompanied by his mother with bruises on her face. Her exam revealed additional bruises over her abdominal wall. The mother said they accidentally tripped and fell down the stairs, and spontaneously denied any acts of violence in the family.
Given this scenario, which of the following is best?
A. You suspect both child and spousal abuse, but lack sufficient evidence to report the incident.
B. Failure to report based on reasonable suspicion alone may amount to a criminal offense punishable by possible imprisonment.
C. You may face a potential malpractice lawsuit if subsequent injuries caused by abuse could have been prevented had you reported.
D. Mandatory reporting laws apply not only to abuse of children and spouses, but also of the elderly and other vulnerable adults.
E. All are correct except A.
Answer: E. All doctors, especially those working in emergency departments, treat injuries on a regular basis. Accidents probably account for the majority of these injuries, but the most pernicious are those caused by willful abuse or neglect. Such conduct, believed to be widespread and underrecognized, victimizes children, women, the elderly, and other vulnerable groups.
Mandatory reporting laws arose from the need to identify and prevent these activities that cause serious harm and loss of lives. Physicians and other health care workers are in a prime position to diagnose or raise the suspicion of abuse and neglect. This article focuses on laws that mandate physician reporting of such behavior. Not addressed are other reportable situations such as certain infectious diseases, gunshot wounds, threats to third parties, and so on.
Child abuse
The best-known example of a mandatory reporting law relates to child abuse, which is broadly defined as when a parent or caretaker emotionally, physically, or sexually abuses, neglects, or abandons a child. Child abuse laws are intended to protect children from serious harm without abridging parental discipline of their children.
Cases of child abuse are pervasive; four or five children are tragically killed by abuse or neglect every day, and each year, some 6 million children are reported as victims of child abuse. Henry Kempe’s studies on the “battered child syndrome” in 1962 served to underscore the physician’s role in exposing child maltreatment, and 1973 saw the enactment of the Child Abuse Prevention and Treatment Act, which set standards for mandatory reporting as a condition for federal funding.
All U.S. states have statutes identifying persons who are required to report suspected child maltreatment to an appropriate agency, such as child protective services. Reasonable suspicion, without need for proof, is sufficient to trigger the mandatory reporting duty. A summary of the general reporting requirements, as well as each state’s key statutory features, are available at Child Welfare Information Gateway.1
Bruises, fractures, and burns are recurring examples of injuries resulting from child abuse, but there are many others, including severe emotional harm, which is an important consequence. Clues to abuse include a child’s fearful and anxious demeanor, wearing clothes to hide injuries, and inappropriate sexual conduct.2 The perpetrators and/or complicit parties typically blame an innocent home accident for the victim’s injuries to mislead the health care provider.
Elder abuse
Elder abuse is broadly construed to include physical, sexual, and psychological abuse, as well as financial exploitation and caregiver neglect.3 It is a serious problem in the United States, estimated in 2008 to affect 1 in 10 elders. The figure is likely an underestimate, because many elderly victims are afraid or unwilling to lodge a complaint against the abuser whom they love and may depend upon.4
The law, which protects the “elderly” (e.g., those aged 62 years or older in Hawaii), may also be extended to other younger vulnerable adults, who because of an impairment, are unable to 1) communicate or make responsible decisions to manage one’s own care or resources, 2) carry out or arrange for essential activities of daily living, or 3) protect one’s self from abuse.5
The law mandates reporting where there is reason to believe abuse has occurred or the vulnerable adult is in danger of abuse if immediate action is not taken. Reporting statutes for elder abuse vary somewhat on the identity of mandated reporters (health care providers are always included), the victim’s mental capacity, dwelling place (home or in an assisted-living facility), and type of purported activity that warrants reporting.
Domestic violence
As defined by the National Coalition Against Domestic Violence, “Domestic violence is the willful intimidation, physical assault, battery, sexual assault, and/or other abusive behavior as part of a systematic pattern of power and control perpetrated by one intimate partner against another. ... The frequency and severity of domestic violence can vary dramatically; however, the one constant component of domestic violence is one partner’s consistent efforts to maintain power and control over the other.”6 Domestic violence is said to have reached epidemic proportions, with one in four women experiencing it at some point in her life.
Virtually all states mandate the reporting of domestic violence by health care providers if there is a reasonable suspicion that observed patient injuries are the result of physical abuse.7 California, for example, requires the provider to call local law enforcement as soon as possible or to send in a written report within 48 hours.
There may be exceptions to required reporting, as when an adult victim withholds consent but accepts victim referral services. State laws encourage but do not always require that the health care provider inform the patient about the report, but federal law dictates otherwise unless this puts the patient at risk. Hawaii’s domestic violence laws were originally enacted to deter spousal abuse, but they now also protect other household members.8
Any individual who assumes a duty or responsibility pursuant to all of these reporting laws is immunized from criminal or civil liability. On the other hand, a mandated reporter who knowingly fails to report an incident or who willfully prevents another person from reporting such an incident commits a criminal offence.
In the case of a physician, there is the added risk of a malpractice lawsuit based on “violation of statute” (breach of a legal duty), should another injury occur down the road that was arguably preventable by his or her failure to report.
Experts generally believe that mandatory reporting laws are important in identifying child maltreatment. However, it has been asserted that despite a 5-decade history of mandatory reporting, no clear endpoints attest to the efficacy of this approach, and it is argued that no data exist to demonstrate that incremental increases in reporting have contributed to child safety.
Particularly challenging are attempts at impact comparisons between states with different policies. A number of countries, including the United Kingdom, do not have mandatory reporting laws and regulate reporting by professional societies.9
In addition, some critics of mandatory reporting raise concerns surrounding law enforcement showing up at the victim’s house to question the family about abuse, or to make an arrest or issue warnings. They posit that when the behavior of an abuser is under scrutiny, this can paradoxically create a potentially more dangerous environment for the patient-victim, whom the perpetrator now considers to have betrayed his or her trust. Others bemoan that revealing patient confidences violates the physician’s ethical code.
However, the intolerable incidence of violence against the vulnerable has properly made mandatory reporting the law of the land. Although the criminal penalty is currently light for failure to report, there is a move toward increasing its severity. Hawaii, for example, recently introduced Senate Bill 2477 that makes nonreporting by those required to do so a Class C felony punishable by up to 5 years in prison. The offense currently is a petty misdemeanor punishable by up to 30 days in jail.
Dr. Tan is emeritus professor of medicine and former adjunct professor of law at the University of Hawaii, Honolulu. This article is meant to be educational and does not constitute medical, ethical, or legal advice. For additional information, readers may contact the author at siang@hawaii.edu.
References
1. Child Welfare Information Gateway (2016). Mandatory reporters of child abuse and neglect. Washington, D.C.: U.S. Department of Health and Human Services, Children’s Bureau. Available at www.childwelfare.gov; email: info@childwelfare.gov; phone: 800-394-3366.
2. Available at www.childwelfare.gov/topics/can.
3. Available at www.justice.gov/elderjustice/elder-justice-statutes-0.
4. Available at www.cdc.gov/violenceprevention/elderabuse/index.html.
5. Hawaii Revised Statutes, Sec. 346-222, 346-224, 346-250, 412:3-114.5.
6. Available at ncadv.org.
7. Ann Emerg Med. 2002 Jan;39(1):56-60.
8. Hawaii Revised Statutes, Sec. 709-906.
9. Pediatrics. 2017 Apr;139(4). pii: e20163511.
Question: You are moonlighting in the emergency department and have just finished treating a 5-year-old boy with an apparent Colles’ fracture, who was accompanied by his mother with bruises on her face. Her exam revealed additional bruises over her abdominal wall. The mother said they accidentally tripped and fell down the stairs, and spontaneously denied any acts of violence in the family.
Given this scenario, which of the following is best?
A. You suspect both child and spousal abuse, but lack sufficient evidence to report the incident.
B. Failure to report based on reasonable suspicion alone may amount to a criminal offense punishable by possible imprisonment.
C. You may face a potential malpractice lawsuit if subsequent injuries caused by abuse could have been prevented had you reported.
D. Mandatory reporting laws apply not only to abuse of children and spouses, but also of the elderly and other vulnerable adults.
E. All are correct except A.
Answer: E. All doctors, especially those working in emergency departments, treat injuries on a regular basis. Accidents probably account for the majority of these injuries, but the most pernicious are those caused by willful abuse or neglect. Such conduct, believed to be widespread and underrecognized, victimizes children, women, the elderly, and other vulnerable groups.
Mandatory reporting laws arose from the need to identify and prevent these activities that cause serious harm and loss of lives. Physicians and other health care workers are in a prime position to diagnose or raise the suspicion of abuse and neglect. This article focuses on laws that mandate physician reporting of such behavior. Not addressed are other reportable situations such as certain infectious diseases, gunshot wounds, threats to third parties, and so on.
Child abuse
The best-known example of a mandatory reporting law relates to child abuse, which is broadly defined as when a parent or caretaker emotionally, physically, or sexually abuses, neglects, or abandons a child. Child abuse laws are intended to protect children from serious harm without abridging parental discipline of their children.
Cases of child abuse are pervasive; four or five children are tragically killed by abuse or neglect every day, and each year, some 6 million children are reported as victims of child abuse. Henry Kempe’s studies on the “battered child syndrome” in 1962 served to underscore the physician’s role in exposing child maltreatment, and 1973 saw the enactment of the Child Abuse Prevention and Treatment Act, which set standards for mandatory reporting as a condition for federal funding.
All U.S. states have statutes identifying persons who are required to report suspected child maltreatment to an appropriate agency, such as child protective services. Reasonable suspicion, without need for proof, is sufficient to trigger the mandatory reporting duty. A summary of the general reporting requirements, as well as each state’s key statutory features, are available at Child Welfare Information Gateway.1
Bruises, fractures, and burns are recurring examples of injuries resulting from child abuse, but there are many others, including severe emotional harm, which is an important consequence. Clues to abuse include a child’s fearful and anxious demeanor, wearing clothes to hide injuries, and inappropriate sexual conduct.2 The perpetrators and/or complicit parties typically blame an innocent home accident for the victim’s injuries to mislead the health care provider.
Elder abuse
Elder abuse is broadly construed to include physical, sexual, and psychological abuse, as well as financial exploitation and caregiver neglect.3 It is a serious problem in the United States, estimated in 2008 to affect 1 in 10 elders. The figure is likely an underestimate, because many elderly victims are afraid or unwilling to lodge a complaint against the abuser whom they love and may depend upon.4
The law, which protects the “elderly” (e.g., those aged 62 years or older in Hawaii), may also be extended to other younger vulnerable adults, who because of an impairment, are unable to 1) communicate or make responsible decisions to manage one’s own care or resources, 2) carry out or arrange for essential activities of daily living, or 3) protect one’s self from abuse.5
The law mandates reporting where there is reason to believe abuse has occurred or the vulnerable adult is in danger of abuse if immediate action is not taken. Reporting statutes for elder abuse vary somewhat on the identity of mandated reporters (health care providers are always included), the victim’s mental capacity, dwelling place (home or in an assisted-living facility), and type of purported activity that warrants reporting.
Domestic violence
As defined by the National Coalition Against Domestic Violence, “Domestic violence is the willful intimidation, physical assault, battery, sexual assault, and/or other abusive behavior as part of a systematic pattern of power and control perpetrated by one intimate partner against another. ... The frequency and severity of domestic violence can vary dramatically; however, the one constant component of domestic violence is one partner’s consistent efforts to maintain power and control over the other.”6 Domestic violence is said to have reached epidemic proportions, with one in four women experiencing it at some point in her life.
Virtually all states mandate the reporting of domestic violence by health care providers if there is a reasonable suspicion that observed patient injuries are the result of physical abuse.7 California, for example, requires the provider to call local law enforcement as soon as possible or to send in a written report within 48 hours.
There may be exceptions to required reporting, as when an adult victim withholds consent but accepts victim referral services. State laws encourage but do not always require that the health care provider inform the patient about the report, but federal law dictates otherwise unless this puts the patient at risk. Hawaii’s domestic violence laws were originally enacted to deter spousal abuse, but they now also protect other household members.8
Any individual who assumes a duty or responsibility pursuant to all of these reporting laws is immunized from criminal or civil liability. On the other hand, a mandated reporter who knowingly fails to report an incident or who willfully prevents another person from reporting such an incident commits a criminal offence.
In the case of a physician, there is the added risk of a malpractice lawsuit based on “violation of statute” (breach of a legal duty), should another injury occur down the road that was arguably preventable by his or her failure to report.
Experts generally believe that mandatory reporting laws are important in identifying child maltreatment. However, it has been asserted that despite a 5-decade history of mandatory reporting, no clear endpoints attest to the efficacy of this approach, and it is argued that no data exist to demonstrate that incremental increases in reporting have contributed to child safety.
Particularly challenging are attempts at impact comparisons between states with different policies. A number of countries, including the United Kingdom, do not have mandatory reporting laws and regulate reporting by professional societies.9
In addition, some critics of mandatory reporting raise concerns surrounding law enforcement showing up at the victim’s house to question the family about abuse, or to make an arrest or issue warnings. They posit that when the behavior of an abuser is under scrutiny, this can paradoxically create a potentially more dangerous environment for the patient-victim, whom the perpetrator now considers to have betrayed his or her trust. Others bemoan that revealing patient confidences violates the physician’s ethical code.
However, the intolerable incidence of violence against the vulnerable has properly made mandatory reporting the law of the land. Although the criminal penalty is currently light for failure to report, there is a move toward increasing its severity. Hawaii, for example, recently introduced Senate Bill 2477 that makes nonreporting by those required to do so a Class C felony punishable by up to 5 years in prison. The offense currently is a petty misdemeanor punishable by up to 30 days in jail.
Dr. Tan is emeritus professor of medicine and former adjunct professor of law at the University of Hawaii, Honolulu. This article is meant to be educational and does not constitute medical, ethical, or legal advice. For additional information, readers may contact the author at siang@hawaii.edu.
References
1. Child Welfare Information Gateway (2016). Mandatory reporters of child abuse and neglect. Washington, D.C.: U.S. Department of Health and Human Services, Children’s Bureau. Available at www.childwelfare.gov; email: info@childwelfare.gov; phone: 800-394-3366.
2. Available at www.childwelfare.gov/topics/can.
3. Available at www.justice.gov/elderjustice/elder-justice-statutes-0.
4. Available at www.cdc.gov/violenceprevention/elderabuse/index.html.
5. Hawaii Revised Statutes, Sec. 346-222, 346-224, 346-250, 412:3-114.5.
6. Available at ncadv.org.
7. Ann Emerg Med. 2002 Jan;39(1):56-60.
8. Hawaii Revised Statutes, Sec. 709-906.
9. Pediatrics. 2017 Apr;139(4). pii: e20163511.
Obesity paradox applies to post-stroke mortality
CHICAGO – Overweight and obese military veterans who experienced an in-hospital stroke had a lower 30-day and 1-year all-cause mortality than did those who were normal weight in a large national study, Lauren Costa reported at the American Heart Association scientific sessions.
Underweight patients had a significantly increased mortality risk, added Ms. Costa of the VA Boston Healthcare System.
It’s yet another instance of what is known as the obesity paradox, which has also been described in patients with heart failure, acute coronary syndrome, MI, chronic obstructive pulmonary disease, and other conditions.
Ms. Costa presented a retrospective study of 26,267 patients in the Veterans Health Administration database who had a first stroke in-hospital during 2002-2012. There were subsequently 14,166 deaths, including 2,473 within the first 30 days and 5,854 in the first year post stroke.
Each patient’s body mass index was calculated based on the average of all BMI measurements obtained 1-24 months prior to the stroke. The analysis of the relationship between BMI and poststroke mortality included extensive statistical adjustment for potential confounders, including age, sex, smoking, cancer, dementia, peripheral artery disease, diabetes, coronary heart disease, atrial fibrillation, chronic kidney disease, use of statins, and antihypertensive therapy.
Breaking down the study population into eight BMI categories, Ms. Costa found that the adjusted risk of 30-day all-cause mortality post stroke was reduced by 22%-38% in patients in the overweight or obese groupings, compared with the reference population with a normal-weight BMI of 22.5 to less than 25 kg/m2.
One-year, all-cause mortality showed the same pattern of BMI-based significant differences.
Of deaths within 30 days post stroke, 34% were stroke-related. In an analysis restricted to that group, the evidence of an obesity paradox was attenuated. Indeed, the only BMI group with an adjusted 30-day stroke-related mortality significantly different from the normal-weight reference group were patients with Class III obesity, defined as a BMI of 40 or more. Their risk was reduced by 45%.
The obesity paradox remains a controversial issue among epidemiologists. The increased mortality associated with being underweight among patients with diseases where the obesity paradox has been documented is widely thought to be caused by frailty and/or an underlying illness not adjusted for in analyses. But the mechanism for the reduced mortality risk in overweight and obese patients seen in the VA stroke study and other studies remains unknown despite much speculation.
Ms. Costa reported having no financial conflicts regarding her study, which was supported by the Department of Veterans Affairs.
SOURCE: Costa L. Circulation. 2018;138(suppl 1): Abstract 14288.
CHICAGO – Overweight and obese military veterans who experienced an in-hospital stroke had a lower 30-day and 1-year all-cause mortality than did those who were normal weight in a large national study, Lauren Costa reported at the American Heart Association scientific sessions.
Underweight patients had a significantly increased mortality risk, added Ms. Costa of the VA Boston Healthcare System.
It’s yet another instance of what is known as the obesity paradox, which has also been described in patients with heart failure, acute coronary syndrome, MI, chronic obstructive pulmonary disease, and other conditions.
Ms. Costa presented a retrospective study of 26,267 patients in the Veterans Health Administration database who had a first stroke in-hospital during 2002-2012. There were subsequently 14,166 deaths, including 2,473 within the first 30 days and 5,854 in the first year post stroke.
Each patient’s body mass index was calculated based on the average of all BMI measurements obtained 1-24 months prior to the stroke. The analysis of the relationship between BMI and poststroke mortality included extensive statistical adjustment for potential confounders, including age, sex, smoking, cancer, dementia, peripheral artery disease, diabetes, coronary heart disease, atrial fibrillation, chronic kidney disease, use of statins, and antihypertensive therapy.
Breaking down the study population into eight BMI categories, Ms. Costa found that the adjusted risk of 30-day all-cause mortality post stroke was reduced by 22%-38% in patients in the overweight or obese groupings, compared with the reference population with a normal-weight BMI of 22.5 to less than 25 kg/m2.
One-year, all-cause mortality showed the same pattern of BMI-based significant differences.
Of deaths within 30 days post stroke, 34% were stroke-related. In an analysis restricted to that group, the evidence of an obesity paradox was attenuated. Indeed, the only BMI group with an adjusted 30-day stroke-related mortality significantly different from the normal-weight reference group were patients with Class III obesity, defined as a BMI of 40 or more. Their risk was reduced by 45%.
The obesity paradox remains a controversial issue among epidemiologists. The increased mortality associated with being underweight among patients with diseases where the obesity paradox has been documented is widely thought to be caused by frailty and/or an underlying illness not adjusted for in analyses. But the mechanism for the reduced mortality risk in overweight and obese patients seen in the VA stroke study and other studies remains unknown despite much speculation.
Ms. Costa reported having no financial conflicts regarding her study, which was supported by the Department of Veterans Affairs.
SOURCE: Costa L. Circulation. 2018;138(suppl 1): Abstract 14288.
CHICAGO – Overweight and obese military veterans who experienced an in-hospital stroke had a lower 30-day and 1-year all-cause mortality than did those who were normal weight in a large national study, Lauren Costa reported at the American Heart Association scientific sessions.
Underweight patients had a significantly increased mortality risk, added Ms. Costa of the VA Boston Healthcare System.
It’s yet another instance of what is known as the obesity paradox, which has also been described in patients with heart failure, acute coronary syndrome, MI, chronic obstructive pulmonary disease, and other conditions.
Ms. Costa presented a retrospective study of 26,267 patients in the Veterans Health Administration database who had a first stroke in-hospital during 2002-2012. There were subsequently 14,166 deaths, including 2,473 within the first 30 days and 5,854 in the first year post stroke.
Each patient’s body mass index was calculated based on the average of all BMI measurements obtained 1-24 months prior to the stroke. The analysis of the relationship between BMI and poststroke mortality included extensive statistical adjustment for potential confounders, including age, sex, smoking, cancer, dementia, peripheral artery disease, diabetes, coronary heart disease, atrial fibrillation, chronic kidney disease, use of statins, and antihypertensive therapy.
Breaking down the study population into eight BMI categories, Ms. Costa found that the adjusted risk of 30-day all-cause mortality post stroke was reduced by 22%-38% in patients in the overweight or obese groupings, compared with the reference population with a normal-weight BMI of 22.5 to less than 25 kg/m2.
One-year, all-cause mortality showed the same pattern of BMI-based significant differences.
Of deaths within 30 days post stroke, 34% were stroke-related. In an analysis restricted to that group, the evidence of an obesity paradox was attenuated. Indeed, the only BMI group with an adjusted 30-day stroke-related mortality significantly different from the normal-weight reference group were patients with Class III obesity, defined as a BMI of 40 or more. Their risk was reduced by 45%.
The obesity paradox remains a controversial issue among epidemiologists. The increased mortality associated with being underweight among patients with diseases where the obesity paradox has been documented is widely thought to be caused by frailty and/or an underlying illness not adjusted for in analyses. But the mechanism for the reduced mortality risk in overweight and obese patients seen in the VA stroke study and other studies remains unknown despite much speculation.
Ms. Costa reported having no financial conflicts regarding her study, which was supported by the Department of Veterans Affairs.
SOURCE: Costa L. Circulation. 2018;138(suppl 1): Abstract 14288.
REPORTING FROM THE AHA SCIENTIFIC SESSIONS
Key clinical point: Heavier stroke patients have lower 30-day and 1-year all-cause mortality.
Major finding: The 30-day stroke-related mortality rate after in-hospital stroke was reduced by 45% in VA patients with Class III obesity.
Study details: This retrospective study looked at the relationship between body mass index and post-stroke mortality in more than 26,000 veterans who had an inpatient stroke, with extensive adjustments made for potential confounders.
Disclosures: The presenter reported having no financial conflicts regarding the study, which was sponsored by the Department of Veterans Affairs.
Source: Costa L. Circulation. 2018;138(suppl 1): Abstract 14288.
Carbs vs. fats for CVD
Also today, from MDedge Pediatrics, a consult on influenza and pneumonia, brodalumab beats ustekinumab to PASI 100, and a novel agent cuts LDL in patients who are intolerant to statins.
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Also today, from MDedge Pediatrics, a consult on influenza and pneumonia, brodalumab beats ustekinumab to PASI 100, and a novel agent cuts LDL in patients who are intolerant to statins.
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Also today, from MDedge Pediatrics, a consult on influenza and pneumonia, brodalumab beats ustekinumab to PASI 100, and a novel agent cuts LDL in patients who are intolerant to statins.
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FDA approves generic version of vigabatrin
The drug is approved for the adjunctive treatment of focal seizures in patients aged 10 years and older who have not had an adequate response to other therapies.
The approval was granted to Teva Pharmaceuticals.
An FDA announcement noted that the agency has prioritized the approval of generic versions of drugs to improve access to treatments and to lower drug costs. Vigabatrin had been included on an FDA list of off-patent, off-exclusivity branded drugs without approved generics. The approval of generic vigabatrin “demonstrates that there is an open pathway to approving products like this one,” said FDA Commissioner Scott Gottlieb, MD.
The label for vigabatrin tablets includes a boxed warning for permanent vision loss. The generic vigabatrin tablets are part of a single shared-system Risk Evaluation and Mitigation Strategy (REMS) program with other drug products containing vigabatrin.
The most common side effects associated with vigabatrin tablets include dizziness, fatigue, sleepiness, involuntary eye movement, tremor, blurred vision, memory impairment, weight gain, joint pain, upper respiratory tract infection, aggression, double vision, abnormal coordination, and a confused state. Serious side effects associated with vigabatrin tablets include permanent vision loss and risk of suicidal thoughts or actions.
The drug is approved for the adjunctive treatment of focal seizures in patients aged 10 years and older who have not had an adequate response to other therapies.
The approval was granted to Teva Pharmaceuticals.
An FDA announcement noted that the agency has prioritized the approval of generic versions of drugs to improve access to treatments and to lower drug costs. Vigabatrin had been included on an FDA list of off-patent, off-exclusivity branded drugs without approved generics. The approval of generic vigabatrin “demonstrates that there is an open pathway to approving products like this one,” said FDA Commissioner Scott Gottlieb, MD.
The label for vigabatrin tablets includes a boxed warning for permanent vision loss. The generic vigabatrin tablets are part of a single shared-system Risk Evaluation and Mitigation Strategy (REMS) program with other drug products containing vigabatrin.
The most common side effects associated with vigabatrin tablets include dizziness, fatigue, sleepiness, involuntary eye movement, tremor, blurred vision, memory impairment, weight gain, joint pain, upper respiratory tract infection, aggression, double vision, abnormal coordination, and a confused state. Serious side effects associated with vigabatrin tablets include permanent vision loss and risk of suicidal thoughts or actions.
The drug is approved for the adjunctive treatment of focal seizures in patients aged 10 years and older who have not had an adequate response to other therapies.
The approval was granted to Teva Pharmaceuticals.
An FDA announcement noted that the agency has prioritized the approval of generic versions of drugs to improve access to treatments and to lower drug costs. Vigabatrin had been included on an FDA list of off-patent, off-exclusivity branded drugs without approved generics. The approval of generic vigabatrin “demonstrates that there is an open pathway to approving products like this one,” said FDA Commissioner Scott Gottlieb, MD.
The label for vigabatrin tablets includes a boxed warning for permanent vision loss. The generic vigabatrin tablets are part of a single shared-system Risk Evaluation and Mitigation Strategy (REMS) program with other drug products containing vigabatrin.
The most common side effects associated with vigabatrin tablets include dizziness, fatigue, sleepiness, involuntary eye movement, tremor, blurred vision, memory impairment, weight gain, joint pain, upper respiratory tract infection, aggression, double vision, abnormal coordination, and a confused state. Serious side effects associated with vigabatrin tablets include permanent vision loss and risk of suicidal thoughts or actions.
Prescription puppy, China’s commode hospital, Hall of Fame diuretics
Prescription puppies
I’d like a script for a golden retriever who loves fetch and has a super sniffer, please. Turns out, man’s best friend can be especially friendly for patients with type 1 diabetes mellitus.
Dogs have been trained to detect seizures, lead the blind, and even call 9-1-1, and now they are increasingly being used as glycemia monitors for people with diabetes. Obviously, people don’t get a prescription for their helpful furry friends, but one can dream about an all-puppy pharmacy, right?
A recently published study took a look at how glycemia-alert dogs improve the quality of life of people living with type 1 diabetes, and assessed the reliability of the dogs to respond to hypo- and hyperglycemic episodes.
Researchers concluded that, overall, a trained pup’s response to these episodes is more sensitive than previously thought. In addition, researchers also found that 100% of the study participants were extremely good dogs.
Really selective hearing loss
A woman in China is experiencing something straight out of a movie – she woke up one morning and couldn’t hear men’s voices anymore. Some might say this is a tragic loss. Others might contend that she is living the dream.
The woman was diagnosed with reverse-slope hearing loss, a very rare type of hearing loss that makes the patient unable able to hear low-frequency sounds, such as a man’s voice. Only 1 in every 12,000 people with hearing loss has this kind, and it is most often caused by genetics.
In this case, the woman’s stress and extreme fatigue appear to be contributing factors to her newfound superpower. Her doctor expects her to make a full recovery, but I’d milk it for as long as possible: “What’s that? Something about the dishes, honey? Sorry I can’t hear what you’re saying!”
The epitome of crappy design
Here at MDedge News, we approve of all scatologically related humor, and the Guangxi International Zhuangyi Hospital in Nanning, China, certainly fits the bill. In an ode to the digestive system, the Chinese have built a hospital that bears an undeniable resemblance to a toilet.
The building is huge, spread out over 42 acres. The patients are contained in the multistory tank, while the medical departments line the outside of the massive bowl.
According to a local citizen, the hospital was designed this way so patients could go from the main section to the departments without carrying an umbrella. We hope it’s because they have an excellent gastroenterology department they want to show off.
Leaking the news: Hall of Fame edition
Can you name the ultimate prize for a life-saving achievement in medicine? You’re right, it is induction into the National Inventors Hall of Fame!
And that’s just what’s about to happen to the inventors of thiazide diuretics. The NIHF just announced its class of 2019, and it includes pharmacologists John Baer and Karl H. Beyer Jr. and organic chemists Frederick Novello and James Sprague, who developed Diuril (chlorothiazide) while at Merck Sharp & Dohme in the 1950s. (Is it just us, or do you get the feeling that Don Draper and the rest of the Sterling Cooper crew must have handled the Diuril account?)
The honor is, unfortunately, posthumous for all four men, but they were around for the Lasker Foundation Special Public Health Award they received in 1975.
We here at LOTME can’t top either of these commendations, but there’s at least one hypertensive on the staff who will be thinking of these men and their achievement when he experiences his next forced diuresis.
Prescription puppies
I’d like a script for a golden retriever who loves fetch and has a super sniffer, please. Turns out, man’s best friend can be especially friendly for patients with type 1 diabetes mellitus.
Dogs have been trained to detect seizures, lead the blind, and even call 9-1-1, and now they are increasingly being used as glycemia monitors for people with diabetes. Obviously, people don’t get a prescription for their helpful furry friends, but one can dream about an all-puppy pharmacy, right?
A recently published study took a look at how glycemia-alert dogs improve the quality of life of people living with type 1 diabetes, and assessed the reliability of the dogs to respond to hypo- and hyperglycemic episodes.
Researchers concluded that, overall, a trained pup’s response to these episodes is more sensitive than previously thought. In addition, researchers also found that 100% of the study participants were extremely good dogs.
Really selective hearing loss
A woman in China is experiencing something straight out of a movie – she woke up one morning and couldn’t hear men’s voices anymore. Some might say this is a tragic loss. Others might contend that she is living the dream.
The woman was diagnosed with reverse-slope hearing loss, a very rare type of hearing loss that makes the patient unable able to hear low-frequency sounds, such as a man’s voice. Only 1 in every 12,000 people with hearing loss has this kind, and it is most often caused by genetics.
In this case, the woman’s stress and extreme fatigue appear to be contributing factors to her newfound superpower. Her doctor expects her to make a full recovery, but I’d milk it for as long as possible: “What’s that? Something about the dishes, honey? Sorry I can’t hear what you’re saying!”
The epitome of crappy design
Here at MDedge News, we approve of all scatologically related humor, and the Guangxi International Zhuangyi Hospital in Nanning, China, certainly fits the bill. In an ode to the digestive system, the Chinese have built a hospital that bears an undeniable resemblance to a toilet.
The building is huge, spread out over 42 acres. The patients are contained in the multistory tank, while the medical departments line the outside of the massive bowl.
According to a local citizen, the hospital was designed this way so patients could go from the main section to the departments without carrying an umbrella. We hope it’s because they have an excellent gastroenterology department they want to show off.
Leaking the news: Hall of Fame edition
Can you name the ultimate prize for a life-saving achievement in medicine? You’re right, it is induction into the National Inventors Hall of Fame!
And that’s just what’s about to happen to the inventors of thiazide diuretics. The NIHF just announced its class of 2019, and it includes pharmacologists John Baer and Karl H. Beyer Jr. and organic chemists Frederick Novello and James Sprague, who developed Diuril (chlorothiazide) while at Merck Sharp & Dohme in the 1950s. (Is it just us, or do you get the feeling that Don Draper and the rest of the Sterling Cooper crew must have handled the Diuril account?)
The honor is, unfortunately, posthumous for all four men, but they were around for the Lasker Foundation Special Public Health Award they received in 1975.
We here at LOTME can’t top either of these commendations, but there’s at least one hypertensive on the staff who will be thinking of these men and their achievement when he experiences his next forced diuresis.
Prescription puppies
I’d like a script for a golden retriever who loves fetch and has a super sniffer, please. Turns out, man’s best friend can be especially friendly for patients with type 1 diabetes mellitus.
Dogs have been trained to detect seizures, lead the blind, and even call 9-1-1, and now they are increasingly being used as glycemia monitors for people with diabetes. Obviously, people don’t get a prescription for their helpful furry friends, but one can dream about an all-puppy pharmacy, right?
A recently published study took a look at how glycemia-alert dogs improve the quality of life of people living with type 1 diabetes, and assessed the reliability of the dogs to respond to hypo- and hyperglycemic episodes.
Researchers concluded that, overall, a trained pup’s response to these episodes is more sensitive than previously thought. In addition, researchers also found that 100% of the study participants were extremely good dogs.
Really selective hearing loss
A woman in China is experiencing something straight out of a movie – she woke up one morning and couldn’t hear men’s voices anymore. Some might say this is a tragic loss. Others might contend that she is living the dream.
The woman was diagnosed with reverse-slope hearing loss, a very rare type of hearing loss that makes the patient unable able to hear low-frequency sounds, such as a man’s voice. Only 1 in every 12,000 people with hearing loss has this kind, and it is most often caused by genetics.
In this case, the woman’s stress and extreme fatigue appear to be contributing factors to her newfound superpower. Her doctor expects her to make a full recovery, but I’d milk it for as long as possible: “What’s that? Something about the dishes, honey? Sorry I can’t hear what you’re saying!”
The epitome of crappy design
Here at MDedge News, we approve of all scatologically related humor, and the Guangxi International Zhuangyi Hospital in Nanning, China, certainly fits the bill. In an ode to the digestive system, the Chinese have built a hospital that bears an undeniable resemblance to a toilet.
The building is huge, spread out over 42 acres. The patients are contained in the multistory tank, while the medical departments line the outside of the massive bowl.
According to a local citizen, the hospital was designed this way so patients could go from the main section to the departments without carrying an umbrella. We hope it’s because they have an excellent gastroenterology department they want to show off.
Leaking the news: Hall of Fame edition
Can you name the ultimate prize for a life-saving achievement in medicine? You’re right, it is induction into the National Inventors Hall of Fame!
And that’s just what’s about to happen to the inventors of thiazide diuretics. The NIHF just announced its class of 2019, and it includes pharmacologists John Baer and Karl H. Beyer Jr. and organic chemists Frederick Novello and James Sprague, who developed Diuril (chlorothiazide) while at Merck Sharp & Dohme in the 1950s. (Is it just us, or do you get the feeling that Don Draper and the rest of the Sterling Cooper crew must have handled the Diuril account?)
The honor is, unfortunately, posthumous for all four men, but they were around for the Lasker Foundation Special Public Health Award they received in 1975.
We here at LOTME can’t top either of these commendations, but there’s at least one hypertensive on the staff who will be thinking of these men and their achievement when he experiences his next forced diuresis.
Courts stop contraceptive mandate
Too much sleep and too little sleep are linked to atherosclerosis, there is no drop in gout prevalence, but there isn’t an increase either, and back pain persists in one in five patients.
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Too much sleep and too little sleep are linked to atherosclerosis, there is no drop in gout prevalence, but there isn’t an increase either, and back pain persists in one in five patients.
Amazon Alexa
Apple Podcasts
Google Podcasts
Spotify
Too much sleep and too little sleep are linked to atherosclerosis, there is no drop in gout prevalence, but there isn’t an increase either, and back pain persists in one in five patients.
Amazon Alexa
Apple Podcasts
Google Podcasts
Spotify
DMTs, stem cell transplants both reduce disease progression in MS
Disease-modifying therapies give patients with relapsing-remitting multiple sclerosis a lower risk of developing secondary progressive disease that may only be topped in specific patients with highly active disease by the use of nonmyeloablative hematopoietic stem cell transplantation, according to findings from two studies published online Jan. 15 in JAMA.
The first study found that interferon-beta, glatiramer acetate (Copaxone), fingolimod (Gilenya), natalizumab (Tysabri), and alemtuzumab (Lemtrada) are associated with a lower risk of conversion to secondary progressive MS, compared with no treatment. Initial treatment with the newer therapies provided a greater risk reduction, compared with initial treatment with interferon-beta or glatiramer acetate.
The second study, described as “the first randomized trial of HSCT [nonmyeloablative hematopoietic stem cell transplantation] in patients with relapsing-remitting MS,” suggests that HSCT prolongs the time to disease progression, compared with disease-modifying therapies (DMTs). It also suggests that HSCT can lead to clinical improvement.
DMTs reduced risk of conversion to secondary progressive MS
Few previous studies have examined the association between DMTs and the risk of conversion from relapsing-remitting MS to secondary progressive MS. Those that have analyzed this association have not used a validated definition of secondary progressive MS. J. William L. Brown, MD, of the University of Cambridge, England, and his colleagues used a validated definition of secondary progressive MS that was published in 2016 to investigate how DMTs affect the rate of conversion, compared with no treatment. The researchers also compared the risk reduction provided by fingolimod, alemtuzumab, or natalizumab with that provided by interferon-beta or glatiramer acetate.
Dr. Brown and his colleagues analyzed prospectively collected clinical data from an international observational cohort study called MSBase. Eligible participants had relapsing-remitting MS, the complete MSBase minimum data set, at least one Expanded Disability Status Scale (EDSS) score recorded within 6 months before baseline, and at least two EDSS scores recorded after baseline. Participants initiated a DMT or began clinical monitoring during 1988-2012. The population had a minimum follow-up duration of 4 years. Patients who stopped their initial therapy within 6 months and those participating in clinical trials were excluded.
The primary outcome was conversion to secondary progressive MS. Dr. Brown and his colleagues defined this outcome as an EDSS increase of 1 point for participants with a baseline EDSS score of 5.5 or less and as an increase of 0.5 points for participants with a baseline EDSS score higher than 5.5. This increase had to occur in the absence of relapses and be confirmed at a subsequent visit 3 or fewer months later. In addition, the increased EDSS score had to be 4 or more.
After excluding ineligible participants, the investigators matched 1,555 patients from 68 centers in 21 countries. Each therapy analyzed was associated with reduced risk of converting to secondary progressive MS, compared with no treatment. The hazard ratios for conversion were 0.71 for interferon-beta or glatiramer acetate, 0.37 for fingolimod, 0.61 for natalizumab, and 0.52 for alemtuzumab, compared with no treatment.
Treatment with interferon-beta or glatiramer acetate within 5 years of disease onset was associated with a reduced risk of conversion (HR, 0.77), compared with treatment later than 5 years after disease onset. Similarly, patients who escalated treatment from interferon-beta or glatiramer acetate to any of the other three DMTs within 5 years of disease onset had a significantly lower risk of conversion (HR, 0.76) than did those who escalated later. Furthermore, initial treatment with fingolimod, alemtuzumab, or natalizumab was associated with a significantly reduced risk of conversion (HR, 0.66), compared with initial treatment with interferon-beta or glatiramer acetate.
One of the study’s limitations is its observational design, which precludes the determination of causality, Dr. Brown and his colleagues said. In addition, functional score subcomponents of the EDSS were unavailable, which prevented the researchers from using the definition of secondary progressive MS with the best combination of sensitivity, specificity, and accuracy. Some analyses were limited by small numbers of patients, and the study did not evaluate the risks associated with DMTs. Nevertheless, “these findings, considered along with these therapies’ risks, may help inform decisions about DMT selection,” the authors concluded.
Financial support for this study was provided by the National Health and Medical Research Council of Australia and the University of Melbourne. Dr. Brown received a Next Generation Fellowship funded by the Grand Charity of the Freemasons and an MSBase 2017 Fellowship. Alemtuzumab studies conducted in Cambridge were supported by the National Institute for Health Research Cambridge Biomedical Research Centre and the MS Society UK.
HSCT delayed disease progression
In a previous case series, Richard K. Burt, MD, of Northwestern University in Chicago, and his colleagues found that patients with relapsing-remitting MS who underwent nonmyeloablative HSCT had neurologic improvement and a 70% likelihood of having a 4-year period of disease remission. Dr. Burt and his colleagues undertook the MS international stem cell transplant trial to compare the effects of nonmyeloablative HSCT with those of continued DMT treatment on disease progression in participants with highly active relapsing-remitting MS.
The researchers enrolled 110 participants at four international centers into their open-label trial. Eligible participants had two or more clinical relapses or one relapse and at least one gadolinium-enhancing lesion at a separate time within the previous 12 months, despite DMT treatment. The investigators also required participants to have an EDSS score between 2.0 and 6.0. Patients with primary or secondary progressive MS were excluded.
Dr. Burt and his colleagues randomized participants to receive HSCT or an approved DMT that was more effective or in a different class than the one they were receiving at baseline. Ocrelizumab (Ocrevus) was not administered during the study because it had not yet been approved. The investigators excluded alemtuzumab because of its association with persistent lymphopenia and autoimmune disorders. After 1 year of treatment, patients receiving a DMT who had disability progression could cross over to the HSCT arm. Patients randomized to HSCT stopped taking their usual DMT.
Time to disease progression was the study’s primary endpoint. The investigators defined disease progression as an increase in EDSS score of at least 1 point on two evaluations 6 months apart after at least 1 year of treatment. The increase was required to result from MS. The neurologist who recorded participants’ EDSS evaluations was blinded to treatment group assignment.
The researchers randomized 55 patients to each study arm. Approximately 66% of participants were women, and the sample’s mean age was 36 years. There were no significant baseline differences between groups on demographic, clinical, or imaging characteristics. Three patients in the HSCT group were withdrawn from the study, and four in the DMT group were lost to follow-up after seeking HSCT at outside facilities.
Three patients in the HSCT group and 34 patients in the DMT group had disease progression. Mean follow-up duration was 2.8 years. The investigators could not calculate the median time to progression in the HSCT group because too few events occurred. Median time to progression was 24 months in the DMT group (HR, 0.07). During the first year, mean EDSS scores decreased (indicating improvement) from 3.38 to 2.36 in the HSCT group. Mean EDSS scores increased from 3.31 to 3.98 in the DMT group. No participants died, and no patients who received HSCT-developed nonhematopoietic grade 4 toxicities.
“To our knowledge, this is the first randomized trial of HSCT in patients with relapsing-remitting MS,” Dr. Burt and his colleagues said. Although observational studies have found similar EDSS improvements following HSCT, “this degree of improvement has not been demonstrated in pharmaceutical trials even with more intensive DMT such as alemtuzumab,” they concluded.
The Danhakl Family Foundation, the Cumming Foundation, the McNamara Purcell Foundation, Morgan Stanley, and the National Institute for Health Research Sheffield Clinical Research Facility provided financial support for this study. No pharmaceutical companies supported the study.
SOURCEs: Brown JWL et al. JAMA. 2019;321(2):175-87. doi: 10.1001/jama.2018.20588.; and Burt RK et al. JAMA. 2019;321(2):165-74. doi: 10.1001/jama.2018.18743.
The study by Brown et al. provides evidence that DMTs slow the appearance of persistent disabilities in patients with multiple sclerosis (MS), Harold Atkins, MD, wrote in an accompanying editorial (JAMA. 2019 Jan 15;321[2]:153-4). Although disease-modifying therapies (DMTs) may suppress clinical signs of disease activity for long periods in some patients, these therapies slow MS rather than halt it. DMTs require long-term administration and may cause intolerable side effects that impair patients’ quality of life. These therapies also may result in complications such as severe depression or progressive multifocal leukoencephalopathy.
“The study by Burt et al. ... provides a rigorous indication that HSCT [hematopoietic stem cell transplantation] can be an effective treatment for selected patients with MS,” Dr. Atkins said. Treating physicians, however, have concerns about this procedure, which is resource-intensive and “requires specialized medical and nursing expertise and dedicated hospital infrastructure to minimize its risks.” Many patients in the study had moderate to severe acute toxicity following treatment, and patient selection thus requires caution.
An important limitation of the study is that participants did not have access to alemtuzumab or ocrelizumab, which arguably are the most effective DMTs, Dr. Atkins said. The study began in 2005, when fewer DMTs were available. “The inclusion of patients who were less than optimally treated in the DMT group needs to be considered when interpreting the results of this study,” Dr. Atkins said.
Furthermore, Burt and colleagues studied patients with highly active MS, but “only a small proportion of the MS patient population exhibits this degree of activity,” he added. The results therefore may not be generalizable. Nevertheless, “even with the limitations of the trial, the results support a role for HSCT delivered at centers that are experienced in the clinical care of patients with highly active MS,” Dr. Atkins concluded.
Dr. Atkins is affiliated with the Ottawa Hospital Blood and Marrow Transplant Program at the University of Ottawa in Ontario. He reported no conflicts of interest.
The study by Brown et al. provides evidence that DMTs slow the appearance of persistent disabilities in patients with multiple sclerosis (MS), Harold Atkins, MD, wrote in an accompanying editorial (JAMA. 2019 Jan 15;321[2]:153-4). Although disease-modifying therapies (DMTs) may suppress clinical signs of disease activity for long periods in some patients, these therapies slow MS rather than halt it. DMTs require long-term administration and may cause intolerable side effects that impair patients’ quality of life. These therapies also may result in complications such as severe depression or progressive multifocal leukoencephalopathy.
“The study by Burt et al. ... provides a rigorous indication that HSCT [hematopoietic stem cell transplantation] can be an effective treatment for selected patients with MS,” Dr. Atkins said. Treating physicians, however, have concerns about this procedure, which is resource-intensive and “requires specialized medical and nursing expertise and dedicated hospital infrastructure to minimize its risks.” Many patients in the study had moderate to severe acute toxicity following treatment, and patient selection thus requires caution.
An important limitation of the study is that participants did not have access to alemtuzumab or ocrelizumab, which arguably are the most effective DMTs, Dr. Atkins said. The study began in 2005, when fewer DMTs were available. “The inclusion of patients who were less than optimally treated in the DMT group needs to be considered when interpreting the results of this study,” Dr. Atkins said.
Furthermore, Burt and colleagues studied patients with highly active MS, but “only a small proportion of the MS patient population exhibits this degree of activity,” he added. The results therefore may not be generalizable. Nevertheless, “even with the limitations of the trial, the results support a role for HSCT delivered at centers that are experienced in the clinical care of patients with highly active MS,” Dr. Atkins concluded.
Dr. Atkins is affiliated with the Ottawa Hospital Blood and Marrow Transplant Program at the University of Ottawa in Ontario. He reported no conflicts of interest.
The study by Brown et al. provides evidence that DMTs slow the appearance of persistent disabilities in patients with multiple sclerosis (MS), Harold Atkins, MD, wrote in an accompanying editorial (JAMA. 2019 Jan 15;321[2]:153-4). Although disease-modifying therapies (DMTs) may suppress clinical signs of disease activity for long periods in some patients, these therapies slow MS rather than halt it. DMTs require long-term administration and may cause intolerable side effects that impair patients’ quality of life. These therapies also may result in complications such as severe depression or progressive multifocal leukoencephalopathy.
“The study by Burt et al. ... provides a rigorous indication that HSCT [hematopoietic stem cell transplantation] can be an effective treatment for selected patients with MS,” Dr. Atkins said. Treating physicians, however, have concerns about this procedure, which is resource-intensive and “requires specialized medical and nursing expertise and dedicated hospital infrastructure to minimize its risks.” Many patients in the study had moderate to severe acute toxicity following treatment, and patient selection thus requires caution.
An important limitation of the study is that participants did not have access to alemtuzumab or ocrelizumab, which arguably are the most effective DMTs, Dr. Atkins said. The study began in 2005, when fewer DMTs were available. “The inclusion of patients who were less than optimally treated in the DMT group needs to be considered when interpreting the results of this study,” Dr. Atkins said.
Furthermore, Burt and colleagues studied patients with highly active MS, but “only a small proportion of the MS patient population exhibits this degree of activity,” he added. The results therefore may not be generalizable. Nevertheless, “even with the limitations of the trial, the results support a role for HSCT delivered at centers that are experienced in the clinical care of patients with highly active MS,” Dr. Atkins concluded.
Dr. Atkins is affiliated with the Ottawa Hospital Blood and Marrow Transplant Program at the University of Ottawa in Ontario. He reported no conflicts of interest.
Disease-modifying therapies give patients with relapsing-remitting multiple sclerosis a lower risk of developing secondary progressive disease that may only be topped in specific patients with highly active disease by the use of nonmyeloablative hematopoietic stem cell transplantation, according to findings from two studies published online Jan. 15 in JAMA.
The first study found that interferon-beta, glatiramer acetate (Copaxone), fingolimod (Gilenya), natalizumab (Tysabri), and alemtuzumab (Lemtrada) are associated with a lower risk of conversion to secondary progressive MS, compared with no treatment. Initial treatment with the newer therapies provided a greater risk reduction, compared with initial treatment with interferon-beta or glatiramer acetate.
The second study, described as “the first randomized trial of HSCT [nonmyeloablative hematopoietic stem cell transplantation] in patients with relapsing-remitting MS,” suggests that HSCT prolongs the time to disease progression, compared with disease-modifying therapies (DMTs). It also suggests that HSCT can lead to clinical improvement.
DMTs reduced risk of conversion to secondary progressive MS
Few previous studies have examined the association between DMTs and the risk of conversion from relapsing-remitting MS to secondary progressive MS. Those that have analyzed this association have not used a validated definition of secondary progressive MS. J. William L. Brown, MD, of the University of Cambridge, England, and his colleagues used a validated definition of secondary progressive MS that was published in 2016 to investigate how DMTs affect the rate of conversion, compared with no treatment. The researchers also compared the risk reduction provided by fingolimod, alemtuzumab, or natalizumab with that provided by interferon-beta or glatiramer acetate.
Dr. Brown and his colleagues analyzed prospectively collected clinical data from an international observational cohort study called MSBase. Eligible participants had relapsing-remitting MS, the complete MSBase minimum data set, at least one Expanded Disability Status Scale (EDSS) score recorded within 6 months before baseline, and at least two EDSS scores recorded after baseline. Participants initiated a DMT or began clinical monitoring during 1988-2012. The population had a minimum follow-up duration of 4 years. Patients who stopped their initial therapy within 6 months and those participating in clinical trials were excluded.
The primary outcome was conversion to secondary progressive MS. Dr. Brown and his colleagues defined this outcome as an EDSS increase of 1 point for participants with a baseline EDSS score of 5.5 or less and as an increase of 0.5 points for participants with a baseline EDSS score higher than 5.5. This increase had to occur in the absence of relapses and be confirmed at a subsequent visit 3 or fewer months later. In addition, the increased EDSS score had to be 4 or more.
After excluding ineligible participants, the investigators matched 1,555 patients from 68 centers in 21 countries. Each therapy analyzed was associated with reduced risk of converting to secondary progressive MS, compared with no treatment. The hazard ratios for conversion were 0.71 for interferon-beta or glatiramer acetate, 0.37 for fingolimod, 0.61 for natalizumab, and 0.52 for alemtuzumab, compared with no treatment.
Treatment with interferon-beta or glatiramer acetate within 5 years of disease onset was associated with a reduced risk of conversion (HR, 0.77), compared with treatment later than 5 years after disease onset. Similarly, patients who escalated treatment from interferon-beta or glatiramer acetate to any of the other three DMTs within 5 years of disease onset had a significantly lower risk of conversion (HR, 0.76) than did those who escalated later. Furthermore, initial treatment with fingolimod, alemtuzumab, or natalizumab was associated with a significantly reduced risk of conversion (HR, 0.66), compared with initial treatment with interferon-beta or glatiramer acetate.
One of the study’s limitations is its observational design, which precludes the determination of causality, Dr. Brown and his colleagues said. In addition, functional score subcomponents of the EDSS were unavailable, which prevented the researchers from using the definition of secondary progressive MS with the best combination of sensitivity, specificity, and accuracy. Some analyses were limited by small numbers of patients, and the study did not evaluate the risks associated with DMTs. Nevertheless, “these findings, considered along with these therapies’ risks, may help inform decisions about DMT selection,” the authors concluded.
Financial support for this study was provided by the National Health and Medical Research Council of Australia and the University of Melbourne. Dr. Brown received a Next Generation Fellowship funded by the Grand Charity of the Freemasons and an MSBase 2017 Fellowship. Alemtuzumab studies conducted in Cambridge were supported by the National Institute for Health Research Cambridge Biomedical Research Centre and the MS Society UK.
HSCT delayed disease progression
In a previous case series, Richard K. Burt, MD, of Northwestern University in Chicago, and his colleagues found that patients with relapsing-remitting MS who underwent nonmyeloablative HSCT had neurologic improvement and a 70% likelihood of having a 4-year period of disease remission. Dr. Burt and his colleagues undertook the MS international stem cell transplant trial to compare the effects of nonmyeloablative HSCT with those of continued DMT treatment on disease progression in participants with highly active relapsing-remitting MS.
The researchers enrolled 110 participants at four international centers into their open-label trial. Eligible participants had two or more clinical relapses or one relapse and at least one gadolinium-enhancing lesion at a separate time within the previous 12 months, despite DMT treatment. The investigators also required participants to have an EDSS score between 2.0 and 6.0. Patients with primary or secondary progressive MS were excluded.
Dr. Burt and his colleagues randomized participants to receive HSCT or an approved DMT that was more effective or in a different class than the one they were receiving at baseline. Ocrelizumab (Ocrevus) was not administered during the study because it had not yet been approved. The investigators excluded alemtuzumab because of its association with persistent lymphopenia and autoimmune disorders. After 1 year of treatment, patients receiving a DMT who had disability progression could cross over to the HSCT arm. Patients randomized to HSCT stopped taking their usual DMT.
Time to disease progression was the study’s primary endpoint. The investigators defined disease progression as an increase in EDSS score of at least 1 point on two evaluations 6 months apart after at least 1 year of treatment. The increase was required to result from MS. The neurologist who recorded participants’ EDSS evaluations was blinded to treatment group assignment.
The researchers randomized 55 patients to each study arm. Approximately 66% of participants were women, and the sample’s mean age was 36 years. There were no significant baseline differences between groups on demographic, clinical, or imaging characteristics. Three patients in the HSCT group were withdrawn from the study, and four in the DMT group were lost to follow-up after seeking HSCT at outside facilities.
Three patients in the HSCT group and 34 patients in the DMT group had disease progression. Mean follow-up duration was 2.8 years. The investigators could not calculate the median time to progression in the HSCT group because too few events occurred. Median time to progression was 24 months in the DMT group (HR, 0.07). During the first year, mean EDSS scores decreased (indicating improvement) from 3.38 to 2.36 in the HSCT group. Mean EDSS scores increased from 3.31 to 3.98 in the DMT group. No participants died, and no patients who received HSCT-developed nonhematopoietic grade 4 toxicities.
“To our knowledge, this is the first randomized trial of HSCT in patients with relapsing-remitting MS,” Dr. Burt and his colleagues said. Although observational studies have found similar EDSS improvements following HSCT, “this degree of improvement has not been demonstrated in pharmaceutical trials even with more intensive DMT such as alemtuzumab,” they concluded.
The Danhakl Family Foundation, the Cumming Foundation, the McNamara Purcell Foundation, Morgan Stanley, and the National Institute for Health Research Sheffield Clinical Research Facility provided financial support for this study. No pharmaceutical companies supported the study.
SOURCEs: Brown JWL et al. JAMA. 2019;321(2):175-87. doi: 10.1001/jama.2018.20588.; and Burt RK et al. JAMA. 2019;321(2):165-74. doi: 10.1001/jama.2018.18743.
Disease-modifying therapies give patients with relapsing-remitting multiple sclerosis a lower risk of developing secondary progressive disease that may only be topped in specific patients with highly active disease by the use of nonmyeloablative hematopoietic stem cell transplantation, according to findings from two studies published online Jan. 15 in JAMA.
The first study found that interferon-beta, glatiramer acetate (Copaxone), fingolimod (Gilenya), natalizumab (Tysabri), and alemtuzumab (Lemtrada) are associated with a lower risk of conversion to secondary progressive MS, compared with no treatment. Initial treatment with the newer therapies provided a greater risk reduction, compared with initial treatment with interferon-beta or glatiramer acetate.
The second study, described as “the first randomized trial of HSCT [nonmyeloablative hematopoietic stem cell transplantation] in patients with relapsing-remitting MS,” suggests that HSCT prolongs the time to disease progression, compared with disease-modifying therapies (DMTs). It also suggests that HSCT can lead to clinical improvement.
DMTs reduced risk of conversion to secondary progressive MS
Few previous studies have examined the association between DMTs and the risk of conversion from relapsing-remitting MS to secondary progressive MS. Those that have analyzed this association have not used a validated definition of secondary progressive MS. J. William L. Brown, MD, of the University of Cambridge, England, and his colleagues used a validated definition of secondary progressive MS that was published in 2016 to investigate how DMTs affect the rate of conversion, compared with no treatment. The researchers also compared the risk reduction provided by fingolimod, alemtuzumab, or natalizumab with that provided by interferon-beta or glatiramer acetate.
Dr. Brown and his colleagues analyzed prospectively collected clinical data from an international observational cohort study called MSBase. Eligible participants had relapsing-remitting MS, the complete MSBase minimum data set, at least one Expanded Disability Status Scale (EDSS) score recorded within 6 months before baseline, and at least two EDSS scores recorded after baseline. Participants initiated a DMT or began clinical monitoring during 1988-2012. The population had a minimum follow-up duration of 4 years. Patients who stopped their initial therapy within 6 months and those participating in clinical trials were excluded.
The primary outcome was conversion to secondary progressive MS. Dr. Brown and his colleagues defined this outcome as an EDSS increase of 1 point for participants with a baseline EDSS score of 5.5 or less and as an increase of 0.5 points for participants with a baseline EDSS score higher than 5.5. This increase had to occur in the absence of relapses and be confirmed at a subsequent visit 3 or fewer months later. In addition, the increased EDSS score had to be 4 or more.
After excluding ineligible participants, the investigators matched 1,555 patients from 68 centers in 21 countries. Each therapy analyzed was associated with reduced risk of converting to secondary progressive MS, compared with no treatment. The hazard ratios for conversion were 0.71 for interferon-beta or glatiramer acetate, 0.37 for fingolimod, 0.61 for natalizumab, and 0.52 for alemtuzumab, compared with no treatment.
Treatment with interferon-beta or glatiramer acetate within 5 years of disease onset was associated with a reduced risk of conversion (HR, 0.77), compared with treatment later than 5 years after disease onset. Similarly, patients who escalated treatment from interferon-beta or glatiramer acetate to any of the other three DMTs within 5 years of disease onset had a significantly lower risk of conversion (HR, 0.76) than did those who escalated later. Furthermore, initial treatment with fingolimod, alemtuzumab, or natalizumab was associated with a significantly reduced risk of conversion (HR, 0.66), compared with initial treatment with interferon-beta or glatiramer acetate.
One of the study’s limitations is its observational design, which precludes the determination of causality, Dr. Brown and his colleagues said. In addition, functional score subcomponents of the EDSS were unavailable, which prevented the researchers from using the definition of secondary progressive MS with the best combination of sensitivity, specificity, and accuracy. Some analyses were limited by small numbers of patients, and the study did not evaluate the risks associated with DMTs. Nevertheless, “these findings, considered along with these therapies’ risks, may help inform decisions about DMT selection,” the authors concluded.
Financial support for this study was provided by the National Health and Medical Research Council of Australia and the University of Melbourne. Dr. Brown received a Next Generation Fellowship funded by the Grand Charity of the Freemasons and an MSBase 2017 Fellowship. Alemtuzumab studies conducted in Cambridge were supported by the National Institute for Health Research Cambridge Biomedical Research Centre and the MS Society UK.
HSCT delayed disease progression
In a previous case series, Richard K. Burt, MD, of Northwestern University in Chicago, and his colleagues found that patients with relapsing-remitting MS who underwent nonmyeloablative HSCT had neurologic improvement and a 70% likelihood of having a 4-year period of disease remission. Dr. Burt and his colleagues undertook the MS international stem cell transplant trial to compare the effects of nonmyeloablative HSCT with those of continued DMT treatment on disease progression in participants with highly active relapsing-remitting MS.
The researchers enrolled 110 participants at four international centers into their open-label trial. Eligible participants had two or more clinical relapses or one relapse and at least one gadolinium-enhancing lesion at a separate time within the previous 12 months, despite DMT treatment. The investigators also required participants to have an EDSS score between 2.0 and 6.0. Patients with primary or secondary progressive MS were excluded.
Dr. Burt and his colleagues randomized participants to receive HSCT or an approved DMT that was more effective or in a different class than the one they were receiving at baseline. Ocrelizumab (Ocrevus) was not administered during the study because it had not yet been approved. The investigators excluded alemtuzumab because of its association with persistent lymphopenia and autoimmune disorders. After 1 year of treatment, patients receiving a DMT who had disability progression could cross over to the HSCT arm. Patients randomized to HSCT stopped taking their usual DMT.
Time to disease progression was the study’s primary endpoint. The investigators defined disease progression as an increase in EDSS score of at least 1 point on two evaluations 6 months apart after at least 1 year of treatment. The increase was required to result from MS. The neurologist who recorded participants’ EDSS evaluations was blinded to treatment group assignment.
The researchers randomized 55 patients to each study arm. Approximately 66% of participants were women, and the sample’s mean age was 36 years. There were no significant baseline differences between groups on demographic, clinical, or imaging characteristics. Three patients in the HSCT group were withdrawn from the study, and four in the DMT group were lost to follow-up after seeking HSCT at outside facilities.
Three patients in the HSCT group and 34 patients in the DMT group had disease progression. Mean follow-up duration was 2.8 years. The investigators could not calculate the median time to progression in the HSCT group because too few events occurred. Median time to progression was 24 months in the DMT group (HR, 0.07). During the first year, mean EDSS scores decreased (indicating improvement) from 3.38 to 2.36 in the HSCT group. Mean EDSS scores increased from 3.31 to 3.98 in the DMT group. No participants died, and no patients who received HSCT-developed nonhematopoietic grade 4 toxicities.
“To our knowledge, this is the first randomized trial of HSCT in patients with relapsing-remitting MS,” Dr. Burt and his colleagues said. Although observational studies have found similar EDSS improvements following HSCT, “this degree of improvement has not been demonstrated in pharmaceutical trials even with more intensive DMT such as alemtuzumab,” they concluded.
The Danhakl Family Foundation, the Cumming Foundation, the McNamara Purcell Foundation, Morgan Stanley, and the National Institute for Health Research Sheffield Clinical Research Facility provided financial support for this study. No pharmaceutical companies supported the study.
SOURCEs: Brown JWL et al. JAMA. 2019;321(2):175-87. doi: 10.1001/jama.2018.20588.; and Burt RK et al. JAMA. 2019;321(2):165-74. doi: 10.1001/jama.2018.18743.
FROM JAMA