Neurology Reviews

Breastfeeding by women with multiple sclerosis (MS) or neuromyelitis optica spectrum disorder (NMOSD) who are taking monoclonal antibodies (mAbs) appears to be safe for infants, new research confirmed.

Registry data showed no differences in health or development in the first 3 years of life among infants exposed to natalizumab, ocrelizumab, rituximab, or ofatumumab, compared with unexposed infants.

“Most monoclonal antibody medications for multiple sclerosis are not currently approved for use while a mother is breastfeeding,” even though the disease can develop during a person’s reproductive years, study investigator Kerstin Hellwig, MD, with Ruhr University in Bochum, Germany, said in a news release.

“Our data show infants exposed to these medications through breastfeeding experienced no negative effects on health or development within the first 3 years of life,” Dr. Hellwig said.

The findings were released ahead of the study’s scheduled presentation at the annual meeting of the American Academy of Neurology.
 

Registry Data and Analysis

Using the German MS and Pregnancy Registry, researchers identified 183 infants born to mothers taking mAbs while breastfeeding — 180 with a diagnosis of MS and three with a diagnosis of NMOSD. The infants were matched to 183 unexposed infants (control group).

Exposure to mAbs during lactation started a median of 19 days postpartum and lasted for a median of 172 days. The most commonly used mAb during lactation was natalizumab (125 women), followed by ocrelizumab (34 women), rituximab (11 women), and ofatumumab (10 women).

Among the entire infant cohort, two were first exposed to natalizumab and then ocrelizumab; one was exposed to rituximab and then ocrelizumab; three had been previously breastfed on glatiramer acetate and two on interferons.

The primary outcomes were hospitalizations, antibiotic use, developmental delay, and weight during the first 3 years of life in mAb-exposed versus unexposed infants.

In adjusted regression analyses, mAb exposure during breastfeeding was not significantly associated with annual hospitalization (rate ratio [RR], 1.23; P = .473), annual systemic antibiotic use (RR, 1.55; P = .093), developmental delay (odds ratio, 1.16; P = .716), or weight.

A limitation of the study was that only about a third of the infants were followed for the full 3 years. Therefore, Dr. Hellwig said, the results for the third year of life are less meaningful than for years 1 and 2.
 

‘Reassuring’ Data

Reached for comment, Edith L. Graham, MD, Department of Neurology, Multiple Sclerosis and Neuroimmunology, Northwestern University, Chicago, Illinois, noted that this is the largest group of breastfed infants exposed to mAbs used to treat MS and said the data provide “reassuring infant outcomes with no increase in hospitalization, antibiotic use, or developmental delay.”

Dr. Graham noted that recent publications have reported more on the use of anti-CD20 mAbs (ocrelizumab/rituximab/ofatumumab) while breastfeeding, “and this study adds data for patients on natalizumab.”

“It will be important to know how infusion timing after birth impacts transfer of monoclonal antibodies depending on the milk stage as it transitions from colostrum to mature milk in the first month postpartum,” Dr. Graham said.

“While infection rates of infants are reassuring, data on allergies in the exposed infants would be interesting to look at as well,” she added. “While these infusions are not orally bioavailable, we do not know the full extent of impact on the neonatal gut microbiome.”

In addition, Dr. Graham said it would be important to know whether drugs administered monthly, such as natalizumab and ofatumumab, accumulate in the breast milk at higher levels than medications such as ocrelizumab and rituximab, which are administered twice a year.

The German MS and pregnancy registry was partly supported by the Innovation Fund of the Federal Joint Committee, Almirall Hermal GmbH, Biogen GmbH Germany, Hexal AG, Merck Serono GmbH, Novartis Pharma GmbH, Roche Deutschland GmbH, Sanofi Genzyme, and Teva GmbH. Dr. Hellwig and Dr. Graham had no relevant disclosures.

A version of this article appeared on Medscape.com.

Breastfeeding by women with multiple sclerosis (MS) or neuromyelitis optica spectrum disorder (NMOSD) who are taking monoclonal antibodies (mAbs) appears to be safe for infants, new research confirmed.

Registry data showed no differences in health or development in the first 3 years of life among infants exposed to natalizumab, ocrelizumab, rituximab, or ofatumumab, compared with unexposed infants.

“Most monoclonal antibody medications for multiple sclerosis are not currently approved for use while a mother is breastfeeding,” even though the disease can develop during a person’s reproductive years, study investigator Kerstin Hellwig, MD, with Ruhr University in Bochum, Germany, said in a news release.

“Our data show infants exposed to these medications through breastfeeding experienced no negative effects on health or development within the first 3 years of life,” Dr. Hellwig said.

The findings were released ahead of the study’s scheduled presentation at the annual meeting of the American Academy of Neurology.
 

Registry Data and Analysis

Using the German MS and Pregnancy Registry, researchers identified 183 infants born to mothers taking mAbs while breastfeeding — 180 with a diagnosis of MS and three with a diagnosis of NMOSD. The infants were matched to 183 unexposed infants (control group).

Exposure to mAbs during lactation started a median of 19 days postpartum and lasted for a median of 172 days. The most commonly used mAb during lactation was natalizumab (125 women), followed by ocrelizumab (34 women), rituximab (11 women), and ofatumumab (10 women).

Among the entire infant cohort, two were first exposed to natalizumab and then ocrelizumab; one was exposed to rituximab and then ocrelizumab; three had been previously breastfed on glatiramer acetate and two on interferons.

The primary outcomes were hospitalizations, antibiotic use, developmental delay, and weight during the first 3 years of life in mAb-exposed versus unexposed infants.

In adjusted regression analyses, mAb exposure during breastfeeding was not significantly associated with annual hospitalization (rate ratio [RR], 1.23; P = .473), annual systemic antibiotic use (RR, 1.55; P = .093), developmental delay (odds ratio, 1.16; P = .716), or weight.

A limitation of the study was that only about a third of the infants were followed for the full 3 years. Therefore, Dr. Hellwig said, the results for the third year of life are less meaningful than for years 1 and 2.
 

‘Reassuring’ Data

Reached for comment, Edith L. Graham, MD, Department of Neurology, Multiple Sclerosis and Neuroimmunology, Northwestern University, Chicago, Illinois, noted that this is the largest group of breastfed infants exposed to mAbs used to treat MS and said the data provide “reassuring infant outcomes with no increase in hospitalization, antibiotic use, or developmental delay.”

Dr. Graham noted that recent publications have reported more on the use of anti-CD20 mAbs (ocrelizumab/rituximab/ofatumumab) while breastfeeding, “and this study adds data for patients on natalizumab.”

“It will be important to know how infusion timing after birth impacts transfer of monoclonal antibodies depending on the milk stage as it transitions from colostrum to mature milk in the first month postpartum,” Dr. Graham said.

“While infection rates of infants are reassuring, data on allergies in the exposed infants would be interesting to look at as well,” she added. “While these infusions are not orally bioavailable, we do not know the full extent of impact on the neonatal gut microbiome.”

In addition, Dr. Graham said it would be important to know whether drugs administered monthly, such as natalizumab and ofatumumab, accumulate in the breast milk at higher levels than medications such as ocrelizumab and rituximab, which are administered twice a year.

The German MS and pregnancy registry was partly supported by the Innovation Fund of the Federal Joint Committee, Almirall Hermal GmbH, Biogen GmbH Germany, Hexal AG, Merck Serono GmbH, Novartis Pharma GmbH, Roche Deutschland GmbH, Sanofi Genzyme, and Teva GmbH. Dr. Hellwig and Dr. Graham had no relevant disclosures.

A version of this article appeared on Medscape.com.

Breastfeeding by women with multiple sclerosis (MS) or neuromyelitis optica spectrum disorder (NMOSD) who are taking monoclonal antibodies (mAbs) appears to be safe for infants, new research confirmed.

Registry data showed no differences in health or development in the first 3 years of life among infants exposed to natalizumab, ocrelizumab, rituximab, or ofatumumab, compared with unexposed infants.

“Most monoclonal antibody medications for multiple sclerosis are not currently approved for use while a mother is breastfeeding,” even though the disease can develop during a person’s reproductive years, study investigator Kerstin Hellwig, MD, with Ruhr University in Bochum, Germany, said in a news release.

“Our data show infants exposed to these medications through breastfeeding experienced no negative effects on health or development within the first 3 years of life,” Dr. Hellwig said.

The findings were released ahead of the study’s scheduled presentation at the annual meeting of the American Academy of Neurology.
 

Registry Data and Analysis

Using the German MS and Pregnancy Registry, researchers identified 183 infants born to mothers taking mAbs while breastfeeding — 180 with a diagnosis of MS and three with a diagnosis of NMOSD. The infants were matched to 183 unexposed infants (control group).

Exposure to mAbs during lactation started a median of 19 days postpartum and lasted for a median of 172 days. The most commonly used mAb during lactation was natalizumab (125 women), followed by ocrelizumab (34 women), rituximab (11 women), and ofatumumab (10 women).

Among the entire infant cohort, two were first exposed to natalizumab and then ocrelizumab; one was exposed to rituximab and then ocrelizumab; three had been previously breastfed on glatiramer acetate and two on interferons.

The primary outcomes were hospitalizations, antibiotic use, developmental delay, and weight during the first 3 years of life in mAb-exposed versus unexposed infants.

In adjusted regression analyses, mAb exposure during breastfeeding was not significantly associated with annual hospitalization (rate ratio [RR], 1.23; P = .473), annual systemic antibiotic use (RR, 1.55; P = .093), developmental delay (odds ratio, 1.16; P = .716), or weight.

A limitation of the study was that only about a third of the infants were followed for the full 3 years. Therefore, Dr. Hellwig said, the results for the third year of life are less meaningful than for years 1 and 2.
 

‘Reassuring’ Data

Reached for comment, Edith L. Graham, MD, Department of Neurology, Multiple Sclerosis and Neuroimmunology, Northwestern University, Chicago, Illinois, noted that this is the largest group of breastfed infants exposed to mAbs used to treat MS and said the data provide “reassuring infant outcomes with no increase in hospitalization, antibiotic use, or developmental delay.”

Dr. Graham noted that recent publications have reported more on the use of anti-CD20 mAbs (ocrelizumab/rituximab/ofatumumab) while breastfeeding, “and this study adds data for patients on natalizumab.”

“It will be important to know how infusion timing after birth impacts transfer of monoclonal antibodies depending on the milk stage as it transitions from colostrum to mature milk in the first month postpartum,” Dr. Graham said.

“While infection rates of infants are reassuring, data on allergies in the exposed infants would be interesting to look at as well,” she added. “While these infusions are not orally bioavailable, we do not know the full extent of impact on the neonatal gut microbiome.”

In addition, Dr. Graham said it would be important to know whether drugs administered monthly, such as natalizumab and ofatumumab, accumulate in the breast milk at higher levels than medications such as ocrelizumab and rituximab, which are administered twice a year.

The German MS and pregnancy registry was partly supported by the Innovation Fund of the Federal Joint Committee, Almirall Hermal GmbH, Biogen GmbH Germany, Hexal AG, Merck Serono GmbH, Novartis Pharma GmbH, Roche Deutschland GmbH, Sanofi Genzyme, and Teva GmbH. Dr. Hellwig and Dr. Graham had no relevant disclosures.

A version of this article appeared on Medscape.com.

An article in the March 20 JAMA Network Open looked into the use of AI for responding to patient emails. Basically, they found that this led to a reduction in physician burden, but didn’t save any time.

1. Not sure that’s worth the trouble.

2. Unless the AI is simply responding with something like “message received, thank you” I don’t think this is a good idea.

Yeah, we’re all stretched for time, I understand that. From the starting gun each morning we’re racing between patients, phone calls, incoming test results, staff questions, drug reps, sample closets, dictations, and a million other things.

But AI needs to be a lot better before I let it handle patient questions and concerns.

Someday, yeah, maybe it can do this, like 2-1B, the surgical droid that replaced Luke’s hand in “The Empire Strikes Back.” But we’re not even close to that. Just because a log-in screen says “Jumping to Hyperspace” doesn’t mean you’re on the Millennium Falcon.

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

An article in the March 20 JAMA Network Open looked into the use of AI for responding to patient emails. Basically, they found that this led to a reduction in physician burden, but didn’t save any time.

1. Not sure that’s worth the trouble.

2. Unless the AI is simply responding with something like “message received, thank you” I don’t think this is a good idea.

Yeah, we’re all stretched for time, I understand that. From the starting gun each morning we’re racing between patients, phone calls, incoming test results, staff questions, drug reps, sample closets, dictations, and a million other things.

But AI needs to be a lot better before I let it handle patient questions and concerns.

Someday, yeah, maybe it can do this, like 2-1B, the surgical droid that replaced Luke’s hand in “The Empire Strikes Back.” But we’re not even close to that. Just because a log-in screen says “Jumping to Hyperspace” doesn’t mean you’re on the Millennium Falcon.

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

An article in the March 20 JAMA Network Open looked into the use of AI for responding to patient emails. Basically, they found that this led to a reduction in physician burden, but didn’t save any time.

1. Not sure that’s worth the trouble.

2. Unless the AI is simply responding with something like “message received, thank you” I don’t think this is a good idea.

Yeah, we’re all stretched for time, I understand that. From the starting gun each morning we’re racing between patients, phone calls, incoming test results, staff questions, drug reps, sample closets, dictations, and a million other things.

But AI needs to be a lot better before I let it handle patient questions and concerns.

Someday, yeah, maybe it can do this, like 2-1B, the surgical droid that replaced Luke’s hand in “The Empire Strikes Back.” But we’re not even close to that. Just because a log-in screen says “Jumping to Hyperspace” doesn’t mean you’re on the Millennium Falcon.

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

An estimated 6.9 million older adults are living with Alzheimer’s disease (AD) in the United States, and another 200,000 people under age 65 have younger-onset AD, new data showed.

Findings from the annual report from the Alzheimer’s Association showed little change in AD prevalence since 2023, but study authors predicted the number of people over 65 with AD will nearly double by 2050.

The report also included sobering statistics on AD-related mortality — which increased 141% between 2001 and 2021 — and described “dementia neurology deserts” that will leave some states with less than 10 neurologists per 10,000 people with dementia as early as 2025. The shortages extend to other specialties, clinical professionals, and direct care workers, the report authors wrote.

“Dementia healthcare is a complex maze composed of primary care providers, specialists, social services, medication management, and caregiver support,” Sam Fazio, PhD, senior director, psychosocial research and quality care, Alzheimer’s Association, said in a press release.

“As the number of individuals living with Alzheimer’s continues to grow, ensuring patients, their caregivers, and families have a clear understanding of how to navigate dementia care resources is critical to improving health outcomes,” Dr. Fazio added.

The “2024 Alzheimer’s Disease Facts and Figures” study and accompanying report “Mapping a Better Future for Dementia Care Navigation” were published online on March 20 by the Alzheimer’s Association and will appear in the May issue of Alzheimer’s & Dementia.
 

Significant Increase in Mortality

The number of people over 65 with AD rose slightly in 2024 to 6.9 million from 6.7 million in 2023. The number of younger-onset AD cases remained roughly the same.

States and counties in the eastern and southeastern United States have the highest percentage of people over 65 with AD, with the District of Columbia reporting 16.8% and New York, Florida, and Mississippi between 12.5% and 12.7%. Alaska has the lowest with 8.8%.

Based on an analysis of death certificate data, the number of deaths from AD increased 141% between 2000 and 2021, while deaths from heart disease — the number-one cause of death — decreased 2.1%. Among people aged 70, 61% of those with AD are expected to die before age 80 compared with 30% of those without AD.

The cost of health and long-term care for people with AD has also risen, the data suggested, with a projected total for 2024 of $360 billion, a $15 billion increase since 2023. That figure does not include unpaid caregiving by family and friends, which the report valued at nearly $350 billion.

With the prevalence of AD expected to rise — the report projected 11.2 million by 2040 and 12.7 million by 2050 — mortality, morbidity, and healthcare costs will only continue to go up. Without new treatments and advancements in care, study authors estimated the cost will reach $1 trillion in 2050.

The report also waded into the issue of workforce deficits. Between 2012 and 2022, the number of direct care workers in the United States increased from 3.2 million to 4.8 million. Study authors estimated more than 1 million additional direct care workers will be needed before 2031.

There is a shortage of clinicians as well, especially for geriatricians, specially trained family physicians, or board-certified internists who can screen for, detect, and diagnose possible dementia. The National Center for Health Workforce Analysis (NCHWA) determined shortages in that specialty began a decade ago, and the projected need for geriatricians is expected to far exceed the supply in every region of the United States by 2050.

The NCHWA also projected a shortfall of neurologists by 2025. The report listed 20 US states as “dementia neurology deserts,” meaning they’re projected to have fewer than 10 neurologists per 10,000 people with dementia in 2025.

Several factors may contribute to the scarcity of specialists. In addition to an aging population, contributors include lower pay for geriatricians and neurologists compared with other specialists, an inadequate number of clinician educators with relevant specialties on faculties of health professional schools, and limited incentives to choose these specialties.
 

Underestimating a ‘Serious Problem’

The report “probably underestimates” the “serious problem with dementia specialty care in the United States,” David S. Knopman, MD, professor of neurology, Mayo Clinic, Rochester, Minnesota, told this news organization.

Given the complexity of managing treatments for AD, such as the monoclonal antibody lecanemab, or those for dementia with Lewy bodies, “my sense is that very few geriatricians are likely to take an active role in dementia care,” said Dr. Knopman.

Very few neurologists have specialty training in dementia diagnosis and care, he added, and neurologists who do specialize in dementia are generally located exclusively in tertiary medical centers.

“While neurologists are more likely to be able to diagnose dementia subtypes compared to geriatricians or general internists or family physicians, non-specialty neurologists are also unlikely to have the expertise to manage lecanemab therapy or to deal with diagnosis and management of dementia subtypes,” Dr. Knopman said.

“Filling the pipeline with new trainees is going to take a long, long time,” he added.

As it stands, most dementia diagnoses are not made by specialists. The report cited a study of Medicare beneficiaries that found 85% of people living with dementia were diagnosed by providers such as primary care physicians (PCPs).
 

Barriers to Care

Although screening is now a reimbursable service by Medicare, PCPs experience numerous barriers to detecting cognitive impairment and diagnosing dementia. Routinely used cognitive assessments are time-consuming and labor-intensive, making them challenging to use in a busy clinical setting.

“Even if dementia is diagnosed, providers sometimes wait to disclose this information to the patient due to diagnostic uncertainty, time constraints, stigma, and fear of causing emotional distress,” the authors wrote.

A previous survey by the Alzheimer’s Association uncovered a high degree of uncertainty and discomfort among PCPs in making a dementia diagnosis. While almost a third reported referring patients to specialists, 55% said there were not enough geriatricians and other specialists in their area to meet the demand.

In tackling the theme of dementia care navigation, the report included a survey of 1204 nonphysician healthcare workers, including nurses, physician assistants, and social workers.

About 60% believed the US healthcare system isn’t effectively helping patients and families navigate the system and that training in dementia care navigation is lacking and not standardized. Respondents also said nonmedical professionals are best suited to help people with dementia and their caregivers navigate care.

Respondents identified a range of barriers that make navigating dementia care difficult for patients and families. More than three in four (77%) identified a lack of community-based resources as a barrier. And 70% called out restrictions in current payment models as a barrier, with 41% saying this was the greatest barrier.
 

Alternative Model

In July, the Centers for Medicare & Medicaid Services will launch a pilot model in dementia care management, the Guiding an Improved Dementia Experience. The program will test a monthly per-patient payment model as a fee-for-service replacement.

Healthcare providers who participate in the program will deliver supportive services to people living with dementia and provide access to a care navigator to help patients and caregivers access services and support.

“There is growing momentum in this country to enhance dementia care navigation,” Dr. Fazio said in the release. “Dementia care navigation programs have shown they can be a huge benefit to people living with dementia and their caregivers.”

These programs are unfortunately not widespread across the country, but the Alzheimer’s Association hopes this report “will be a catalyst for change,” Dr. Fazio added.

A separate survey of dementia caregivers found they would overwhelmingly welcome navigator support. The vast majority (97%) said they would find navigation services helpful.

Such services may also go a long way to alleviating stresses involved in dementia caregiving, a top stressor being care coordination, the report noted. Seven in 10 caregiver survey respondents (70%) reported coordinating care is stressful. More than half (53%) said navigating healthcare is difficult, and two-thirds (66%) said they have difficulty finding resources and supports.

Around-the-clock support in addition to care coordination and help understanding their care recipient’s condition are among the top services dementia caregiver respondents cited as being most helpful.

Dr. Knopman reported no relevant conflicts of interest.
 

A version of this article appeared on Medscape.com.

An estimated 6.9 million older adults are living with Alzheimer’s disease (AD) in the United States, and another 200,000 people under age 65 have younger-onset AD, new data showed.

Findings from the annual report from the Alzheimer’s Association showed little change in AD prevalence since 2023, but study authors predicted the number of people over 65 with AD will nearly double by 2050.

The report also included sobering statistics on AD-related mortality — which increased 141% between 2001 and 2021 — and described “dementia neurology deserts” that will leave some states with less than 10 neurologists per 10,000 people with dementia as early as 2025. The shortages extend to other specialties, clinical professionals, and direct care workers, the report authors wrote.

“Dementia healthcare is a complex maze composed of primary care providers, specialists, social services, medication management, and caregiver support,” Sam Fazio, PhD, senior director, psychosocial research and quality care, Alzheimer’s Association, said in a press release.

“As the number of individuals living with Alzheimer’s continues to grow, ensuring patients, their caregivers, and families have a clear understanding of how to navigate dementia care resources is critical to improving health outcomes,” Dr. Fazio added.

The “2024 Alzheimer’s Disease Facts and Figures” study and accompanying report “Mapping a Better Future for Dementia Care Navigation” were published online on March 20 by the Alzheimer’s Association and will appear in the May issue of Alzheimer’s & Dementia.
 

Significant Increase in Mortality

The number of people over 65 with AD rose slightly in 2024 to 6.9 million from 6.7 million in 2023. The number of younger-onset AD cases remained roughly the same.

States and counties in the eastern and southeastern United States have the highest percentage of people over 65 with AD, with the District of Columbia reporting 16.8% and New York, Florida, and Mississippi between 12.5% and 12.7%. Alaska has the lowest with 8.8%.

Based on an analysis of death certificate data, the number of deaths from AD increased 141% between 2000 and 2021, while deaths from heart disease — the number-one cause of death — decreased 2.1%. Among people aged 70, 61% of those with AD are expected to die before age 80 compared with 30% of those without AD.

The cost of health and long-term care for people with AD has also risen, the data suggested, with a projected total for 2024 of $360 billion, a $15 billion increase since 2023. That figure does not include unpaid caregiving by family and friends, which the report valued at nearly $350 billion.

With the prevalence of AD expected to rise — the report projected 11.2 million by 2040 and 12.7 million by 2050 — mortality, morbidity, and healthcare costs will only continue to go up. Without new treatments and advancements in care, study authors estimated the cost will reach $1 trillion in 2050.

The report also waded into the issue of workforce deficits. Between 2012 and 2022, the number of direct care workers in the United States increased from 3.2 million to 4.8 million. Study authors estimated more than 1 million additional direct care workers will be needed before 2031.

There is a shortage of clinicians as well, especially for geriatricians, specially trained family physicians, or board-certified internists who can screen for, detect, and diagnose possible dementia. The National Center for Health Workforce Analysis (NCHWA) determined shortages in that specialty began a decade ago, and the projected need for geriatricians is expected to far exceed the supply in every region of the United States by 2050.

The NCHWA also projected a shortfall of neurologists by 2025. The report listed 20 US states as “dementia neurology deserts,” meaning they’re projected to have fewer than 10 neurologists per 10,000 people with dementia in 2025.

Several factors may contribute to the scarcity of specialists. In addition to an aging population, contributors include lower pay for geriatricians and neurologists compared with other specialists, an inadequate number of clinician educators with relevant specialties on faculties of health professional schools, and limited incentives to choose these specialties.
 

Underestimating a ‘Serious Problem’

The report “probably underestimates” the “serious problem with dementia specialty care in the United States,” David S. Knopman, MD, professor of neurology, Mayo Clinic, Rochester, Minnesota, told this news organization.

Given the complexity of managing treatments for AD, such as the monoclonal antibody lecanemab, or those for dementia with Lewy bodies, “my sense is that very few geriatricians are likely to take an active role in dementia care,” said Dr. Knopman.

Very few neurologists have specialty training in dementia diagnosis and care, he added, and neurologists who do specialize in dementia are generally located exclusively in tertiary medical centers.

“While neurologists are more likely to be able to diagnose dementia subtypes compared to geriatricians or general internists or family physicians, non-specialty neurologists are also unlikely to have the expertise to manage lecanemab therapy or to deal with diagnosis and management of dementia subtypes,” Dr. Knopman said.

“Filling the pipeline with new trainees is going to take a long, long time,” he added.

As it stands, most dementia diagnoses are not made by specialists. The report cited a study of Medicare beneficiaries that found 85% of people living with dementia were diagnosed by providers such as primary care physicians (PCPs).
 

Barriers to Care

Although screening is now a reimbursable service by Medicare, PCPs experience numerous barriers to detecting cognitive impairment and diagnosing dementia. Routinely used cognitive assessments are time-consuming and labor-intensive, making them challenging to use in a busy clinical setting.

“Even if dementia is diagnosed, providers sometimes wait to disclose this information to the patient due to diagnostic uncertainty, time constraints, stigma, and fear of causing emotional distress,” the authors wrote.

A previous survey by the Alzheimer’s Association uncovered a high degree of uncertainty and discomfort among PCPs in making a dementia diagnosis. While almost a third reported referring patients to specialists, 55% said there were not enough geriatricians and other specialists in their area to meet the demand.

In tackling the theme of dementia care navigation, the report included a survey of 1204 nonphysician healthcare workers, including nurses, physician assistants, and social workers.

About 60% believed the US healthcare system isn’t effectively helping patients and families navigate the system and that training in dementia care navigation is lacking and not standardized. Respondents also said nonmedical professionals are best suited to help people with dementia and their caregivers navigate care.

Respondents identified a range of barriers that make navigating dementia care difficult for patients and families. More than three in four (77%) identified a lack of community-based resources as a barrier. And 70% called out restrictions in current payment models as a barrier, with 41% saying this was the greatest barrier.
 

Alternative Model

In July, the Centers for Medicare & Medicaid Services will launch a pilot model in dementia care management, the Guiding an Improved Dementia Experience. The program will test a monthly per-patient payment model as a fee-for-service replacement.

Healthcare providers who participate in the program will deliver supportive services to people living with dementia and provide access to a care navigator to help patients and caregivers access services and support.

“There is growing momentum in this country to enhance dementia care navigation,” Dr. Fazio said in the release. “Dementia care navigation programs have shown they can be a huge benefit to people living with dementia and their caregivers.”

These programs are unfortunately not widespread across the country, but the Alzheimer’s Association hopes this report “will be a catalyst for change,” Dr. Fazio added.

A separate survey of dementia caregivers found they would overwhelmingly welcome navigator support. The vast majority (97%) said they would find navigation services helpful.

Such services may also go a long way to alleviating stresses involved in dementia caregiving, a top stressor being care coordination, the report noted. Seven in 10 caregiver survey respondents (70%) reported coordinating care is stressful. More than half (53%) said navigating healthcare is difficult, and two-thirds (66%) said they have difficulty finding resources and supports.

Around-the-clock support in addition to care coordination and help understanding their care recipient’s condition are among the top services dementia caregiver respondents cited as being most helpful.

Dr. Knopman reported no relevant conflicts of interest.
 

A version of this article appeared on Medscape.com.

An estimated 6.9 million older adults are living with Alzheimer’s disease (AD) in the United States, and another 200,000 people under age 65 have younger-onset AD, new data showed.

Findings from the annual report from the Alzheimer’s Association showed little change in AD prevalence since 2023, but study authors predicted the number of people over 65 with AD will nearly double by 2050.

The report also included sobering statistics on AD-related mortality — which increased 141% between 2001 and 2021 — and described “dementia neurology deserts” that will leave some states with less than 10 neurologists per 10,000 people with dementia as early as 2025. The shortages extend to other specialties, clinical professionals, and direct care workers, the report authors wrote.

“Dementia healthcare is a complex maze composed of primary care providers, specialists, social services, medication management, and caregiver support,” Sam Fazio, PhD, senior director, psychosocial research and quality care, Alzheimer’s Association, said in a press release.

“As the number of individuals living with Alzheimer’s continues to grow, ensuring patients, their caregivers, and families have a clear understanding of how to navigate dementia care resources is critical to improving health outcomes,” Dr. Fazio added.

The “2024 Alzheimer’s Disease Facts and Figures” study and accompanying report “Mapping a Better Future for Dementia Care Navigation” were published online on March 20 by the Alzheimer’s Association and will appear in the May issue of Alzheimer’s & Dementia.
 

Significant Increase in Mortality

The number of people over 65 with AD rose slightly in 2024 to 6.9 million from 6.7 million in 2023. The number of younger-onset AD cases remained roughly the same.

States and counties in the eastern and southeastern United States have the highest percentage of people over 65 with AD, with the District of Columbia reporting 16.8% and New York, Florida, and Mississippi between 12.5% and 12.7%. Alaska has the lowest with 8.8%.

Based on an analysis of death certificate data, the number of deaths from AD increased 141% between 2000 and 2021, while deaths from heart disease — the number-one cause of death — decreased 2.1%. Among people aged 70, 61% of those with AD are expected to die before age 80 compared with 30% of those without AD.

The cost of health and long-term care for people with AD has also risen, the data suggested, with a projected total for 2024 of $360 billion, a $15 billion increase since 2023. That figure does not include unpaid caregiving by family and friends, which the report valued at nearly $350 billion.

With the prevalence of AD expected to rise — the report projected 11.2 million by 2040 and 12.7 million by 2050 — mortality, morbidity, and healthcare costs will only continue to go up. Without new treatments and advancements in care, study authors estimated the cost will reach $1 trillion in 2050.

The report also waded into the issue of workforce deficits. Between 2012 and 2022, the number of direct care workers in the United States increased from 3.2 million to 4.8 million. Study authors estimated more than 1 million additional direct care workers will be needed before 2031.

There is a shortage of clinicians as well, especially for geriatricians, specially trained family physicians, or board-certified internists who can screen for, detect, and diagnose possible dementia. The National Center for Health Workforce Analysis (NCHWA) determined shortages in that specialty began a decade ago, and the projected need for geriatricians is expected to far exceed the supply in every region of the United States by 2050.

The NCHWA also projected a shortfall of neurologists by 2025. The report listed 20 US states as “dementia neurology deserts,” meaning they’re projected to have fewer than 10 neurologists per 10,000 people with dementia in 2025.

Several factors may contribute to the scarcity of specialists. In addition to an aging population, contributors include lower pay for geriatricians and neurologists compared with other specialists, an inadequate number of clinician educators with relevant specialties on faculties of health professional schools, and limited incentives to choose these specialties.
 

Underestimating a ‘Serious Problem’

The report “probably underestimates” the “serious problem with dementia specialty care in the United States,” David S. Knopman, MD, professor of neurology, Mayo Clinic, Rochester, Minnesota, told this news organization.

Given the complexity of managing treatments for AD, such as the monoclonal antibody lecanemab, or those for dementia with Lewy bodies, “my sense is that very few geriatricians are likely to take an active role in dementia care,” said Dr. Knopman.

Very few neurologists have specialty training in dementia diagnosis and care, he added, and neurologists who do specialize in dementia are generally located exclusively in tertiary medical centers.

“While neurologists are more likely to be able to diagnose dementia subtypes compared to geriatricians or general internists or family physicians, non-specialty neurologists are also unlikely to have the expertise to manage lecanemab therapy or to deal with diagnosis and management of dementia subtypes,” Dr. Knopman said.

“Filling the pipeline with new trainees is going to take a long, long time,” he added.

As it stands, most dementia diagnoses are not made by specialists. The report cited a study of Medicare beneficiaries that found 85% of people living with dementia were diagnosed by providers such as primary care physicians (PCPs).
 

Barriers to Care

Although screening is now a reimbursable service by Medicare, PCPs experience numerous barriers to detecting cognitive impairment and diagnosing dementia. Routinely used cognitive assessments are time-consuming and labor-intensive, making them challenging to use in a busy clinical setting.

“Even if dementia is diagnosed, providers sometimes wait to disclose this information to the patient due to diagnostic uncertainty, time constraints, stigma, and fear of causing emotional distress,” the authors wrote.

A previous survey by the Alzheimer’s Association uncovered a high degree of uncertainty and discomfort among PCPs in making a dementia diagnosis. While almost a third reported referring patients to specialists, 55% said there were not enough geriatricians and other specialists in their area to meet the demand.

In tackling the theme of dementia care navigation, the report included a survey of 1204 nonphysician healthcare workers, including nurses, physician assistants, and social workers.

About 60% believed the US healthcare system isn’t effectively helping patients and families navigate the system and that training in dementia care navigation is lacking and not standardized. Respondents also said nonmedical professionals are best suited to help people with dementia and their caregivers navigate care.

Respondents identified a range of barriers that make navigating dementia care difficult for patients and families. More than three in four (77%) identified a lack of community-based resources as a barrier. And 70% called out restrictions in current payment models as a barrier, with 41% saying this was the greatest barrier.
 

Alternative Model

In July, the Centers for Medicare & Medicaid Services will launch a pilot model in dementia care management, the Guiding an Improved Dementia Experience. The program will test a monthly per-patient payment model as a fee-for-service replacement.

Healthcare providers who participate in the program will deliver supportive services to people living with dementia and provide access to a care navigator to help patients and caregivers access services and support.

“There is growing momentum in this country to enhance dementia care navigation,” Dr. Fazio said in the release. “Dementia care navigation programs have shown they can be a huge benefit to people living with dementia and their caregivers.”

These programs are unfortunately not widespread across the country, but the Alzheimer’s Association hopes this report “will be a catalyst for change,” Dr. Fazio added.

A separate survey of dementia caregivers found they would overwhelmingly welcome navigator support. The vast majority (97%) said they would find navigation services helpful.

Such services may also go a long way to alleviating stresses involved in dementia caregiving, a top stressor being care coordination, the report noted. Seven in 10 caregiver survey respondents (70%) reported coordinating care is stressful. More than half (53%) said navigating healthcare is difficult, and two-thirds (66%) said they have difficulty finding resources and supports.

Around-the-clock support in addition to care coordination and help understanding their care recipient’s condition are among the top services dementia caregiver respondents cited as being most helpful.

Dr. Knopman reported no relevant conflicts of interest.
 

A version of this article appeared on Medscape.com.

A simple skin biopsy test is able to detect an abnormal form of alpha-synuclein with high accuracy in individuals with neurodegenerative disorders such as Parkinson’s disease (PD).

Researchers are hopeful that the test — which identified phosphorylated alpha-synuclein (P-SYN) with 95.5% accuracy in the blinded, multicenter trial — will accelerate not just early identification of synucleinopathies but also drug development.

Synucleinopathies include PD, dementia with Lewy bodies (DLB), multiple system atrophy (MSA), and pure autonomic failure (PAF).

“Each year, there are nearly 200,000 people in the U.S. who face a diagnosis of Parkinson’s disease, dementia with Lewy bodies, and related disorders,” study investigator Christopher H. Gibbons, MD, professor of neurology at Harvard Medical School in Boston, said in a press release.

He explained that patients often experience delays in diagnosis or are misdiagnosed due to the complexity of synucleinopathies.

“With a simple, minimally invasive skin biopsy test, this blinded, multicenter study demonstrated how we can more objectively identify the underlying pathology of synucleinopathies and offer better diagnostic answers and care for patients.”

The findings were published online on March 20 in JAMA.
 

An Urgent Priority

Affecting an estimated 2.5 million people in the United States, synucleinopathies are progressive neurodegenerative diseases with varying prognoses, so identifying a reliable diagnostic biomarker is an “urgent unmet priority,” the researchers noted.

The disorders share some symptoms such as tremors and cognitive changes, and all are characterized by P-SYN, an abnormal protein found in the cutaneous nerve fibers.

The study included 428 adults aged 40-99 years (mean age, 70 years) recruited from 30 academic and community-based neurology practices across the United States, with 277 diagnosed with PD, DLB, MSA, or PAF. It also included a control group of 120 participants with no symptoms suggestive of synucleinopathy.

Investigators used the commercially available Syn-One Test, developed in 2019 by CND Life Sciences, to analyze levels of P-SYN via 3-mm punch skin biopsies from each participant.

The test detected P-SYN in 95.5% of study participants overall, including 89 of 96 (92.7%) with PD, 54 of 55 (98.2%) with MSA, 48 of 50 (96%) with DLB, 22 of 22 (100%) with PAF, and 4 of 120 (3.3%) of the controls with no synucleinopathy.

The investigators said it is possible that some of the controls who tested positive had a subclinical form of synucleinopathy, which would explain the false positives.

Study limitations include clinical consensus diagnostic criteria without video or autopsy confirmation, a lack of genetic testing on participants (some genetic forms of PD do not have alpha-synuclein deposition), and the fact that controls were younger than those in disease groups.

“Further research is needed in unselected clinical populations to externally validate the findings and fully characterize the potential role of skin biopsy detection of P-SYN in clinical care,” the authors wrote.

Syn-One is not approved by the US Food and Drug Administration as a diagnostic test for PD but is available as a pathologic assay that determines whether a tissue sample contains phosphorylated alpha-synuclein and can be billed through Medicare.

The study was funded by the National Institutes of Health. Dr. Gibbons reported having stock options in CND Life Sciences outside the submitted work. Other disclosures are noted in the original article.

A version of this article appeared on Medscape.com.

A simple skin biopsy test is able to detect an abnormal form of alpha-synuclein with high accuracy in individuals with neurodegenerative disorders such as Parkinson’s disease (PD).

Researchers are hopeful that the test — which identified phosphorylated alpha-synuclein (P-SYN) with 95.5% accuracy in the blinded, multicenter trial — will accelerate not just early identification of synucleinopathies but also drug development.

Synucleinopathies include PD, dementia with Lewy bodies (DLB), multiple system atrophy (MSA), and pure autonomic failure (PAF).

“Each year, there are nearly 200,000 people in the U.S. who face a diagnosis of Parkinson’s disease, dementia with Lewy bodies, and related disorders,” study investigator Christopher H. Gibbons, MD, professor of neurology at Harvard Medical School in Boston, said in a press release.

He explained that patients often experience delays in diagnosis or are misdiagnosed due to the complexity of synucleinopathies.

“With a simple, minimally invasive skin biopsy test, this blinded, multicenter study demonstrated how we can more objectively identify the underlying pathology of synucleinopathies and offer better diagnostic answers and care for patients.”

The findings were published online on March 20 in JAMA.
 

An Urgent Priority

Affecting an estimated 2.5 million people in the United States, synucleinopathies are progressive neurodegenerative diseases with varying prognoses, so identifying a reliable diagnostic biomarker is an “urgent unmet priority,” the researchers noted.

The disorders share some symptoms such as tremors and cognitive changes, and all are characterized by P-SYN, an abnormal protein found in the cutaneous nerve fibers.

The study included 428 adults aged 40-99 years (mean age, 70 years) recruited from 30 academic and community-based neurology practices across the United States, with 277 diagnosed with PD, DLB, MSA, or PAF. It also included a control group of 120 participants with no symptoms suggestive of synucleinopathy.

Investigators used the commercially available Syn-One Test, developed in 2019 by CND Life Sciences, to analyze levels of P-SYN via 3-mm punch skin biopsies from each participant.

The test detected P-SYN in 95.5% of study participants overall, including 89 of 96 (92.7%) with PD, 54 of 55 (98.2%) with MSA, 48 of 50 (96%) with DLB, 22 of 22 (100%) with PAF, and 4 of 120 (3.3%) of the controls with no synucleinopathy.

The investigators said it is possible that some of the controls who tested positive had a subclinical form of synucleinopathy, which would explain the false positives.

Study limitations include clinical consensus diagnostic criteria without video or autopsy confirmation, a lack of genetic testing on participants (some genetic forms of PD do not have alpha-synuclein deposition), and the fact that controls were younger than those in disease groups.

“Further research is needed in unselected clinical populations to externally validate the findings and fully characterize the potential role of skin biopsy detection of P-SYN in clinical care,” the authors wrote.

Syn-One is not approved by the US Food and Drug Administration as a diagnostic test for PD but is available as a pathologic assay that determines whether a tissue sample contains phosphorylated alpha-synuclein and can be billed through Medicare.

The study was funded by the National Institutes of Health. Dr. Gibbons reported having stock options in CND Life Sciences outside the submitted work. Other disclosures are noted in the original article.

A version of this article appeared on Medscape.com.

A simple skin biopsy test is able to detect an abnormal form of alpha-synuclein with high accuracy in individuals with neurodegenerative disorders such as Parkinson’s disease (PD).

Researchers are hopeful that the test — which identified phosphorylated alpha-synuclein (P-SYN) with 95.5% accuracy in the blinded, multicenter trial — will accelerate not just early identification of synucleinopathies but also drug development.

Synucleinopathies include PD, dementia with Lewy bodies (DLB), multiple system atrophy (MSA), and pure autonomic failure (PAF).

“Each year, there are nearly 200,000 people in the U.S. who face a diagnosis of Parkinson’s disease, dementia with Lewy bodies, and related disorders,” study investigator Christopher H. Gibbons, MD, professor of neurology at Harvard Medical School in Boston, said in a press release.

He explained that patients often experience delays in diagnosis or are misdiagnosed due to the complexity of synucleinopathies.

“With a simple, minimally invasive skin biopsy test, this blinded, multicenter study demonstrated how we can more objectively identify the underlying pathology of synucleinopathies and offer better diagnostic answers and care for patients.”

The findings were published online on March 20 in JAMA.
 

An Urgent Priority

Affecting an estimated 2.5 million people in the United States, synucleinopathies are progressive neurodegenerative diseases with varying prognoses, so identifying a reliable diagnostic biomarker is an “urgent unmet priority,” the researchers noted.

The disorders share some symptoms such as tremors and cognitive changes, and all are characterized by P-SYN, an abnormal protein found in the cutaneous nerve fibers.

The study included 428 adults aged 40-99 years (mean age, 70 years) recruited from 30 academic and community-based neurology practices across the United States, with 277 diagnosed with PD, DLB, MSA, or PAF. It also included a control group of 120 participants with no symptoms suggestive of synucleinopathy.

Investigators used the commercially available Syn-One Test, developed in 2019 by CND Life Sciences, to analyze levels of P-SYN via 3-mm punch skin biopsies from each participant.

The test detected P-SYN in 95.5% of study participants overall, including 89 of 96 (92.7%) with PD, 54 of 55 (98.2%) with MSA, 48 of 50 (96%) with DLB, 22 of 22 (100%) with PAF, and 4 of 120 (3.3%) of the controls with no synucleinopathy.

The investigators said it is possible that some of the controls who tested positive had a subclinical form of synucleinopathy, which would explain the false positives.

Study limitations include clinical consensus diagnostic criteria without video or autopsy confirmation, a lack of genetic testing on participants (some genetic forms of PD do not have alpha-synuclein deposition), and the fact that controls were younger than those in disease groups.

“Further research is needed in unselected clinical populations to externally validate the findings and fully characterize the potential role of skin biopsy detection of P-SYN in clinical care,” the authors wrote.

Syn-One is not approved by the US Food and Drug Administration as a diagnostic test for PD but is available as a pathologic assay that determines whether a tissue sample contains phosphorylated alpha-synuclein and can be billed through Medicare.

The study was funded by the National Institutes of Health. Dr. Gibbons reported having stock options in CND Life Sciences outside the submitted work. Other disclosures are noted in the original article.

A version of this article appeared on Medscape.com.

TOPLINE:

Hospitalization for influenza is linked to a greater risk for subsequent neurologic disorders including migraine, stroke, or epilepsy than is hospitalization for COVID-19, results of a large study show.

METHODOLOGY:

• Researchers used healthcare claims data to compare 77,300 people hospitalized with COVID-19 with 77,300 hospitalized with influenza. The study did not include individuals with long COVID.
• In the final sample of 154,500 participants, the mean age was 51 years, and more than half (58%) were female.
• Investigators followed participants from both cohorts for a year to find out how many of them had medical care for six of the most common neurologic disorders: migraine, epilepsy, stroke, neuropathy, movement disorders, and dementia.
• If participants had one of these neurologic disorders prior to the original hospitalization, the primary outcome involved subsequent healthcare encounters for the neurologic diagnosis.

TAKEAWAY:

• Participants hospitalized with COVID-19 versus influenza were significantly less likely to require care in the following year for migraine (2% vs 3.2%), epilepsy (1.6% vs 2.1%), neuropathy (1.9% vs 3.6%), movement disorders (1.5% vs 2.5%), stroke (2% vs 2.4%), and dementia (2% vs 2.3%) (all P < .001).
• After adjusting for age, sex, and other health conditions, researchers found that people hospitalized with COVID-19 had a 35% lower risk of receiving care for migraine, a 22% lower risk of receiving care for epilepsy, and a 44% lower risk of receiving care for neuropathy than those with influenza. They also had a 36% lower risk of receiving care for movement disorders, a 10% lower risk for stroke (all P < .001), as well as a 7% lower risk for dementia (P = .0007).
• In participants who did not have a preexisting neurologic condition at the time of hospitalization for either COVID-19 or influenza, 2.8% hospitalized with COVID-19 developed one in the next year compared with 5% of those hospitalized with influenza.

IN PRACTICE:

“While the results were not what we expected to find, they are reassuring in that we found being hospitalized with COVID did not lead to more care for common neurologic conditions when compared to being hospitalized with influenza,” study investigator Brian C. Callaghan, MD, of University of Michigan, Ann Arbor, said in a press release.

SOURCE:

Adam de Havenon, MD, of Yale University in New Haven, Connecticut, led the study, which was published online on March 20 in Neurology.

LIMITATIONS:

The study relied on ICD codes in health claims databases, which could introduce misclassification bias. Also, by selecting only individuals who had associated hospital-based care, there may have been a selection bias based on disease severity.

DISCLOSURES:

The study was funded by the American Academy of Neurology. Dr. De Havenon reported receiving consultant fees from Integra and Novo Nordisk and royalty fees from UpToDate and has equity in Titin KM and Certus. Dr. Callaghan has consulted for DynaMed and the Vaccine Injury Compensation Program. Other disclosures were noted in the original article.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Hospitalization for influenza is linked to a greater risk for subsequent neurologic disorders including migraine, stroke, or epilepsy than is hospitalization for COVID-19, results of a large study show.

METHODOLOGY:

• Researchers used healthcare claims data to compare 77,300 people hospitalized with COVID-19 with 77,300 hospitalized with influenza. The study did not include individuals with long COVID.
• In the final sample of 154,500 participants, the mean age was 51 years, and more than half (58%) were female.
• Investigators followed participants from both cohorts for a year to find out how many of them had medical care for six of the most common neurologic disorders: migraine, epilepsy, stroke, neuropathy, movement disorders, and dementia.
• If participants had one of these neurologic disorders prior to the original hospitalization, the primary outcome involved subsequent healthcare encounters for the neurologic diagnosis.

TAKEAWAY:

• Participants hospitalized with COVID-19 versus influenza were significantly less likely to require care in the following year for migraine (2% vs 3.2%), epilepsy (1.6% vs 2.1%), neuropathy (1.9% vs 3.6%), movement disorders (1.5% vs 2.5%), stroke (2% vs 2.4%), and dementia (2% vs 2.3%) (all P < .001).
• After adjusting for age, sex, and other health conditions, researchers found that people hospitalized with COVID-19 had a 35% lower risk of receiving care for migraine, a 22% lower risk of receiving care for epilepsy, and a 44% lower risk of receiving care for neuropathy than those with influenza. They also had a 36% lower risk of receiving care for movement disorders, a 10% lower risk for stroke (all P < .001), as well as a 7% lower risk for dementia (P = .0007).
• In participants who did not have a preexisting neurologic condition at the time of hospitalization for either COVID-19 or influenza, 2.8% hospitalized with COVID-19 developed one in the next year compared with 5% of those hospitalized with influenza.

IN PRACTICE:

“While the results were not what we expected to find, they are reassuring in that we found being hospitalized with COVID did not lead to more care for common neurologic conditions when compared to being hospitalized with influenza,” study investigator Brian C. Callaghan, MD, of University of Michigan, Ann Arbor, said in a press release.

SOURCE:

Adam de Havenon, MD, of Yale University in New Haven, Connecticut, led the study, which was published online on March 20 in Neurology.

LIMITATIONS:

The study relied on ICD codes in health claims databases, which could introduce misclassification bias. Also, by selecting only individuals who had associated hospital-based care, there may have been a selection bias based on disease severity.

DISCLOSURES:

The study was funded by the American Academy of Neurology. Dr. De Havenon reported receiving consultant fees from Integra and Novo Nordisk and royalty fees from UpToDate and has equity in Titin KM and Certus. Dr. Callaghan has consulted for DynaMed and the Vaccine Injury Compensation Program. Other disclosures were noted in the original article.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Hospitalization for influenza is linked to a greater risk for subsequent neurologic disorders including migraine, stroke, or epilepsy than is hospitalization for COVID-19, results of a large study show.

METHODOLOGY:

• Researchers used healthcare claims data to compare 77,300 people hospitalized with COVID-19 with 77,300 hospitalized with influenza. The study did not include individuals with long COVID.
• In the final sample of 154,500 participants, the mean age was 51 years, and more than half (58%) were female.
• Investigators followed participants from both cohorts for a year to find out how many of them had medical care for six of the most common neurologic disorders: migraine, epilepsy, stroke, neuropathy, movement disorders, and dementia.
• If participants had one of these neurologic disorders prior to the original hospitalization, the primary outcome involved subsequent healthcare encounters for the neurologic diagnosis.

TAKEAWAY:

• Participants hospitalized with COVID-19 versus influenza were significantly less likely to require care in the following year for migraine (2% vs 3.2%), epilepsy (1.6% vs 2.1%), neuropathy (1.9% vs 3.6%), movement disorders (1.5% vs 2.5%), stroke (2% vs 2.4%), and dementia (2% vs 2.3%) (all P < .001).
• After adjusting for age, sex, and other health conditions, researchers found that people hospitalized with COVID-19 had a 35% lower risk of receiving care for migraine, a 22% lower risk of receiving care for epilepsy, and a 44% lower risk of receiving care for neuropathy than those with influenza. They also had a 36% lower risk of receiving care for movement disorders, a 10% lower risk for stroke (all P < .001), as well as a 7% lower risk for dementia (P = .0007).
• In participants who did not have a preexisting neurologic condition at the time of hospitalization for either COVID-19 or influenza, 2.8% hospitalized with COVID-19 developed one in the next year compared with 5% of those hospitalized with influenza.

IN PRACTICE:

“While the results were not what we expected to find, they are reassuring in that we found being hospitalized with COVID did not lead to more care for common neurologic conditions when compared to being hospitalized with influenza,” study investigator Brian C. Callaghan, MD, of University of Michigan, Ann Arbor, said in a press release.

SOURCE:

Adam de Havenon, MD, of Yale University in New Haven, Connecticut, led the study, which was published online on March 20 in Neurology.

LIMITATIONS:

The study relied on ICD codes in health claims databases, which could introduce misclassification bias. Also, by selecting only individuals who had associated hospital-based care, there may have been a selection bias based on disease severity.

DISCLOSURES:

The study was funded by the American Academy of Neurology. Dr. De Havenon reported receiving consultant fees from Integra and Novo Nordisk and royalty fees from UpToDate and has equity in Titin KM and Certus. Dr. Callaghan has consulted for DynaMed and the Vaccine Injury Compensation Program. Other disclosures were noted in the original article.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Naturally occurring glucose fluctuations affect cognitive function in people with type 1 diabetes, according to a new study. It matters less whether glucose is considerably higher or lower than the patient’s usual glucose level. Rather, cognition is slower when the glucose was atypical for that particular individual, with variations between different individuals.

METHODOLOGY:

• The investigators used continuous glucose monitoring (CGM) digital sensors and smartphone-based cognitive tests (cognitive ecological momentary assessment [EMA]) to collect repeated, high-frequency glucose and cognitive data. Glucose data were collected every 5 minutes; cognitive data were collected three times daily for 15 days as participants went about their daily lives.
• The study included 200 participants (mean [standard deviation] age, 47.5 [15.6] years; 53.5% female; 86% White; mean A1c, 7.5 mmol/mol [1.3]).
• Using CGM and EMA, the researchers obtained “intensive” longitudinal measurements of glucose as well as cognition (processing speed and sustained attention).
• Hierarchical Bayesian modeling estimated dynamic, within-person associations between glucose and cognition, and data-driven lasso regression identified identify clinical characteristics that predicted differences from person to person in cognitive vulnerability to glucose fluctuations.

TAKEAWAY:

• Cognitive performance was reduced both at low and high glucose levels, “reflecting vulnerability to glucose fluctuations.”
• Large glucose fluctuations were associated with slower as well as less accurate processing speed, although slight glucose elevations (relative to the individual’s own means) were associated with faster processing speed, regardless of the absolute level (eg, euglycemic vs hyperglycemic) of those means.
• By contrast, glucose fluctuations were unrelated to sustained attention.
• The researchers identified seven clinical characteristics that predicted individual differences in cognitive vulnerability to glucose fluctuations: Older age, time in hypoglycemia, lifetime severe hypoglycemic events, microvascular complications, glucose variability, fatigue, and larger neck circumference.

IN PRACTICE:

“Our results demonstrate that people can differ a lot from one another in how their brains are impacted by glucose,” co-senior author Laura Germine, PhD, director of the Laboratory for Brain and Cognitive Health Technology, McLean Hospital, Boston, said in a news release. “We found that minimizing glucose fluctuations in daily life is important for optimizing processing speed, and this is especially true for people who are older or have other diabetes-related health conditions.”

SOURCE:

Zoë Hawks, PhD, research investigator, McLean Hospital, Boston, was the lead and corresponding author on the study. It was published online on March 18 in Digital Medicine.

LIMITATIONS:

The researchers required 24-hour access to a smartphone with reliable Internet access, which might have biased sampling toward people of higher economic status. Moreover, the present sample was predominantly White and non-Hispanic, so findings may not be generalizable to other populations.

DISCLOSURES:

The research was supported by grants from the National Institutes of Health, the Brain and Behavior Research Foundation, and the Alzheimer’s Association. Dr. Hawks received consulting fees from Blueprint Health. The other authors’ disclosures were listed in the original paper.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Naturally occurring glucose fluctuations affect cognitive function in people with type 1 diabetes, according to a new study. It matters less whether glucose is considerably higher or lower than the patient’s usual glucose level. Rather, cognition is slower when the glucose was atypical for that particular individual, with variations between different individuals.

METHODOLOGY:

• The investigators used continuous glucose monitoring (CGM) digital sensors and smartphone-based cognitive tests (cognitive ecological momentary assessment [EMA]) to collect repeated, high-frequency glucose and cognitive data. Glucose data were collected every 5 minutes; cognitive data were collected three times daily for 15 days as participants went about their daily lives.
• The study included 200 participants (mean [standard deviation] age, 47.5 [15.6] years; 53.5% female; 86% White; mean A1c, 7.5 mmol/mol [1.3]).
• Using CGM and EMA, the researchers obtained “intensive” longitudinal measurements of glucose as well as cognition (processing speed and sustained attention).
• Hierarchical Bayesian modeling estimated dynamic, within-person associations between glucose and cognition, and data-driven lasso regression identified identify clinical characteristics that predicted differences from person to person in cognitive vulnerability to glucose fluctuations.

TAKEAWAY:

• Cognitive performance was reduced both at low and high glucose levels, “reflecting vulnerability to glucose fluctuations.”
• Large glucose fluctuations were associated with slower as well as less accurate processing speed, although slight glucose elevations (relative to the individual’s own means) were associated with faster processing speed, regardless of the absolute level (eg, euglycemic vs hyperglycemic) of those means.
• By contrast, glucose fluctuations were unrelated to sustained attention.
• The researchers identified seven clinical characteristics that predicted individual differences in cognitive vulnerability to glucose fluctuations: Older age, time in hypoglycemia, lifetime severe hypoglycemic events, microvascular complications, glucose variability, fatigue, and larger neck circumference.

IN PRACTICE:

“Our results demonstrate that people can differ a lot from one another in how their brains are impacted by glucose,” co-senior author Laura Germine, PhD, director of the Laboratory for Brain and Cognitive Health Technology, McLean Hospital, Boston, said in a news release. “We found that minimizing glucose fluctuations in daily life is important for optimizing processing speed, and this is especially true for people who are older or have other diabetes-related health conditions.”

SOURCE:

Zoë Hawks, PhD, research investigator, McLean Hospital, Boston, was the lead and corresponding author on the study. It was published online on March 18 in Digital Medicine.

LIMITATIONS:

The researchers required 24-hour access to a smartphone with reliable Internet access, which might have biased sampling toward people of higher economic status. Moreover, the present sample was predominantly White and non-Hispanic, so findings may not be generalizable to other populations.

DISCLOSURES:

The research was supported by grants from the National Institutes of Health, the Brain and Behavior Research Foundation, and the Alzheimer’s Association. Dr. Hawks received consulting fees from Blueprint Health. The other authors’ disclosures were listed in the original paper.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Naturally occurring glucose fluctuations affect cognitive function in people with type 1 diabetes, according to a new study. It matters less whether glucose is considerably higher or lower than the patient’s usual glucose level. Rather, cognition is slower when the glucose was atypical for that particular individual, with variations between different individuals.

METHODOLOGY:

• The investigators used continuous glucose monitoring (CGM) digital sensors and smartphone-based cognitive tests (cognitive ecological momentary assessment [EMA]) to collect repeated, high-frequency glucose and cognitive data. Glucose data were collected every 5 minutes; cognitive data were collected three times daily for 15 days as participants went about their daily lives.
• The study included 200 participants (mean [standard deviation] age, 47.5 [15.6] years; 53.5% female; 86% White; mean A1c, 7.5 mmol/mol [1.3]).
• Using CGM and EMA, the researchers obtained “intensive” longitudinal measurements of glucose as well as cognition (processing speed and sustained attention).
• Hierarchical Bayesian modeling estimated dynamic, within-person associations between glucose and cognition, and data-driven lasso regression identified identify clinical characteristics that predicted differences from person to person in cognitive vulnerability to glucose fluctuations.

TAKEAWAY:

• Cognitive performance was reduced both at low and high glucose levels, “reflecting vulnerability to glucose fluctuations.”
• Large glucose fluctuations were associated with slower as well as less accurate processing speed, although slight glucose elevations (relative to the individual’s own means) were associated with faster processing speed, regardless of the absolute level (eg, euglycemic vs hyperglycemic) of those means.
• By contrast, glucose fluctuations were unrelated to sustained attention.
• The researchers identified seven clinical characteristics that predicted individual differences in cognitive vulnerability to glucose fluctuations: Older age, time in hypoglycemia, lifetime severe hypoglycemic events, microvascular complications, glucose variability, fatigue, and larger neck circumference.

IN PRACTICE:

“Our results demonstrate that people can differ a lot from one another in how their brains are impacted by glucose,” co-senior author Laura Germine, PhD, director of the Laboratory for Brain and Cognitive Health Technology, McLean Hospital, Boston, said in a news release. “We found that minimizing glucose fluctuations in daily life is important for optimizing processing speed, and this is especially true for people who are older or have other diabetes-related health conditions.”

SOURCE:

Zoë Hawks, PhD, research investigator, McLean Hospital, Boston, was the lead and corresponding author on the study. It was published online on March 18 in Digital Medicine.

LIMITATIONS:

The researchers required 24-hour access to a smartphone with reliable Internet access, which might have biased sampling toward people of higher economic status. Moreover, the present sample was predominantly White and non-Hispanic, so findings may not be generalizable to other populations.

DISCLOSURES:

The research was supported by grants from the National Institutes of Health, the Brain and Behavior Research Foundation, and the Alzheimer’s Association. Dr. Hawks received consulting fees from Blueprint Health. The other authors’ disclosures were listed in the original paper.
 

A version of this article appeared on Medscape.com.

Symptoms of sleep apnea, including snorting, gasping, or paused breathing during sleep, are associated with a significantly greater risk for problems with cognitive and memory problems, results from a large study showed.

Data from a representative sample of US adults show that those who reported sleep apnea symptoms were about 50% more likely to also report cognitive issues versus their counterparts without such symptoms.

“For clinicians, these findings suggest a potential benefit of considering sleep apnea as a possible contributing or exacerbating factor in individuals experiencing memory or cognitive problems. This could prompt further evaluation for sleep apnea, particularly in at-risk individuals,” said study investigator Dominique Low, MD, MPH, Department of Neurology, Boston Medical Center.

The findings were released ahead of the study’s scheduled presentation at the annual meeting of the American Academy of Neurology.
 

Need to Raise Awareness

The findings are based on 4257 adults who participated in the 2017-2018 National Health and Nutrition Examination Survey and completed questionnaires covering sleep, memory, cognition, and decision-making abilities.

Those who reported snorting, gasping, or breathing pauses during sleep were categorized as experiencing sleep apnea symptoms. Those who reported memory trouble, periods of confusion, difficulty concentrating, or decision-making problems were classified as having memory or cognitive symptoms.

Overall, 1079 participants reported symptoms of sleep apnea. Compared with people without sleep apnea, those with symptoms were more likely to have cognitive problems (33% vs 20%) and have greater odds of having memory or cognitive symptoms, even after adjusting for age, gender, race, and education (adjusted odds ratio, 2.02; P < .001).

“While the study did not establish a cause-and-effect relationship, the findings suggest the importance of raising awareness about the potential link between sleep and cognitive function. Early identification and treatment may improve overall health and potentially lead to a better quality of life,” Dr. Low said.

Limitations of the study include self-reported data on sleep apnea symptoms and cognitive issues sourced from one survey.
 

Consistent Data

Reached for comment, Matthew Pase, PhD, with the Turner Institute for Brain and Mental Health, Monash University, Melbourne, Australia, said the results are similar to earlier work that found a link between obstructive sleep apnea and cognition.

For example, in a recent study, the presence of mild to severe OSA, identified using overnight polysomnography in five community-based cohorts with more than 5900 adults, was associated with poorer cognitive test performance, Dr. Pase said.

“These and other results underscore the importance of healthy sleep for optimal brain health. Future research is needed to test if treating OSA and other sleep disorders can reduce the risk of cognitive impairment,” Dr. Pase said.

Yet, in its latest statement on the topic, the US Preventive Services Task Force concluded there remains insufficient evidence to weigh the balance of benefits and harms of screening for OSA among asymptomatic adults and those with unrecognized symptoms.

The study had no specific funding. Dr. Low and Dr. Pase had no relevant disclosures.
 

A version of this article appeared on Medscape.com.

Symptoms of sleep apnea, including snorting, gasping, or paused breathing during sleep, are associated with a significantly greater risk for problems with cognitive and memory problems, results from a large study showed.

Data from a representative sample of US adults show that those who reported sleep apnea symptoms were about 50% more likely to also report cognitive issues versus their counterparts without such symptoms.

“For clinicians, these findings suggest a potential benefit of considering sleep apnea as a possible contributing or exacerbating factor in individuals experiencing memory or cognitive problems. This could prompt further evaluation for sleep apnea, particularly in at-risk individuals,” said study investigator Dominique Low, MD, MPH, Department of Neurology, Boston Medical Center.

The findings were released ahead of the study’s scheduled presentation at the annual meeting of the American Academy of Neurology.
 

Need to Raise Awareness

The findings are based on 4257 adults who participated in the 2017-2018 National Health and Nutrition Examination Survey and completed questionnaires covering sleep, memory, cognition, and decision-making abilities.

Those who reported snorting, gasping, or breathing pauses during sleep were categorized as experiencing sleep apnea symptoms. Those who reported memory trouble, periods of confusion, difficulty concentrating, or decision-making problems were classified as having memory or cognitive symptoms.

Overall, 1079 participants reported symptoms of sleep apnea. Compared with people without sleep apnea, those with symptoms were more likely to have cognitive problems (33% vs 20%) and have greater odds of having memory or cognitive symptoms, even after adjusting for age, gender, race, and education (adjusted odds ratio, 2.02; P < .001).

“While the study did not establish a cause-and-effect relationship, the findings suggest the importance of raising awareness about the potential link between sleep and cognitive function. Early identification and treatment may improve overall health and potentially lead to a better quality of life,” Dr. Low said.

Limitations of the study include self-reported data on sleep apnea symptoms and cognitive issues sourced from one survey.
 

Consistent Data

Reached for comment, Matthew Pase, PhD, with the Turner Institute for Brain and Mental Health, Monash University, Melbourne, Australia, said the results are similar to earlier work that found a link between obstructive sleep apnea and cognition.

For example, in a recent study, the presence of mild to severe OSA, identified using overnight polysomnography in five community-based cohorts with more than 5900 adults, was associated with poorer cognitive test performance, Dr. Pase said.

“These and other results underscore the importance of healthy sleep for optimal brain health. Future research is needed to test if treating OSA and other sleep disorders can reduce the risk of cognitive impairment,” Dr. Pase said.

Yet, in its latest statement on the topic, the US Preventive Services Task Force concluded there remains insufficient evidence to weigh the balance of benefits and harms of screening for OSA among asymptomatic adults and those with unrecognized symptoms.

The study had no specific funding. Dr. Low and Dr. Pase had no relevant disclosures.
 

A version of this article appeared on Medscape.com.

Symptoms of sleep apnea, including snorting, gasping, or paused breathing during sleep, are associated with a significantly greater risk for problems with cognitive and memory problems, results from a large study showed.

Data from a representative sample of US adults show that those who reported sleep apnea symptoms were about 50% more likely to also report cognitive issues versus their counterparts without such symptoms.

“For clinicians, these findings suggest a potential benefit of considering sleep apnea as a possible contributing or exacerbating factor in individuals experiencing memory or cognitive problems. This could prompt further evaluation for sleep apnea, particularly in at-risk individuals,” said study investigator Dominique Low, MD, MPH, Department of Neurology, Boston Medical Center.

The findings were released ahead of the study’s scheduled presentation at the annual meeting of the American Academy of Neurology.
 

Need to Raise Awareness

The findings are based on 4257 adults who participated in the 2017-2018 National Health and Nutrition Examination Survey and completed questionnaires covering sleep, memory, cognition, and decision-making abilities.

Those who reported snorting, gasping, or breathing pauses during sleep were categorized as experiencing sleep apnea symptoms. Those who reported memory trouble, periods of confusion, difficulty concentrating, or decision-making problems were classified as having memory or cognitive symptoms.

Overall, 1079 participants reported symptoms of sleep apnea. Compared with people without sleep apnea, those with symptoms were more likely to have cognitive problems (33% vs 20%) and have greater odds of having memory or cognitive symptoms, even after adjusting for age, gender, race, and education (adjusted odds ratio, 2.02; P < .001).

“While the study did not establish a cause-and-effect relationship, the findings suggest the importance of raising awareness about the potential link between sleep and cognitive function. Early identification and treatment may improve overall health and potentially lead to a better quality of life,” Dr. Low said.

Limitations of the study include self-reported data on sleep apnea symptoms and cognitive issues sourced from one survey.
 

Consistent Data

Reached for comment, Matthew Pase, PhD, with the Turner Institute for Brain and Mental Health, Monash University, Melbourne, Australia, said the results are similar to earlier work that found a link between obstructive sleep apnea and cognition.

For example, in a recent study, the presence of mild to severe OSA, identified using overnight polysomnography in five community-based cohorts with more than 5900 adults, was associated with poorer cognitive test performance, Dr. Pase said.

“These and other results underscore the importance of healthy sleep for optimal brain health. Future research is needed to test if treating OSA and other sleep disorders can reduce the risk of cognitive impairment,” Dr. Pase said.

Yet, in its latest statement on the topic, the US Preventive Services Task Force concluded there remains insufficient evidence to weigh the balance of benefits and harms of screening for OSA among asymptomatic adults and those with unrecognized symptoms.

The study had no specific funding. Dr. Low and Dr. Pase had no relevant disclosures.
 

A version of this article appeared on Medscape.com.

Most survivors of childhood cancer don’t meet surveillance guidelines that recommend screening for adult cancers or other long-term adverse effects of treatment, according to a new study.

Among childhood cancer survivors in Ontario, Canada, who faced an elevated risk due to chemotherapy or radiation treatments, 53% followed screening recommendations for cardiomyopathy, 13% met colorectal cancer screening guidelines, and 6% adhered to breast cancer screening guidelines.

“Although over 80% of children newly diagnosed with cancer will become long-term survivors, as many as four out of five of these survivors will develop a serious or life-threatening late effect of their cancer therapy by age 45,” lead author Jennifer Shuldiner, PhD, MPH, a scientist at Women’s College Hospital Institute for Health Systems Solutions and Virtual Care in Toronto, told this news organization.

For instance, the risk for colorectal cancer in childhood cancer survivors is two to three times higher than it is among the general population, and the risk for breast cancer is similar between those who underwent chest radiation and those with a BRCA mutation. As many as 50% of those who received anthracycline chemotherapy or radiation involving the heart later develop cardiotoxicity.

The North American Children’s Oncology Group has published long-term follow-up guidelines for survivors of childhood cancer, yet many survivors don’t follow them because of lack of awareness or other barriers, said Dr. Shuldiner.

“Prior research has shown that many survivors do not complete these recommended tests,” she said. “With better knowledge of this at-risk population, we can design, test, and implement appropriate interventions and supports to tackle the issues.”

The study was published online on March 11 in CMAJ. 
 

Changes in Adherence 

The researchers conducted a retrospective population-based cohort study analyzing Ontario healthcare administrative data for adult survivors of childhood cancer diagnosed between 1986 and 2014 who faced an elevated risk for therapy-related colorectal cancer, breast cancer, or cardiomyopathy. The research team then assessed long-term adherence to the North American Children’s Oncology Group guidelines and predictors of adherence.

Among 3241 survivors, 3205 (99%) were at elevated risk for cardiomyopathy, 327 (10%) were at elevated risk for colorectal cancer, and 234 (7%) were at elevated risk for breast cancer. In addition, 2806 (87%) were at risk for one late effect, 345 (11%) were at risk for two late effects, and 90 (3%) were at risk for three late effects.

Overall, 53%, 13%, and 6% were adherent to their recommended surveillance for cardiomyopathy, colorectal cancer, and breast cancer, respectively. Over time, adherence increased for colorectal cancer and cardiomyopathy but decreased for breast cancer.

In addition, patients who were older at diagnosis were more likely to follow screening guidelines for colorectal and breast cancers, whereas those who were younger at diagnosis were more likely to follow screening guidelines for cardiomyopathy.

During a median follow-up of 7.8 years, the proportion of time spent adherent was 43% for cardiomyopathy, 14% for colorectal cancer, and 10% for breast cancer.

Survivors who attended a long-term follow-up clinic in the previous year had low adherence rates as well, though they were higher than in the rest of the cohort. In this group, the proportion of time that was spent adherent was 71% for cardiomyopathy, 27% for colorectal cancer, and 15% for breast cancer.

Shuldiner and colleagues are launching a research trial to determine whether a provincial support system can help childhood cancer survivors receive the recommended surveillance. The support system provides information about screening recommendations to survivors as well as reminders and sends key information to their family doctors.

“We now understand that childhood cancer survivors need help to complete the recommended tests,” said Dr. Shuldiner. “If the trial is successful, we hope it will be implemented in Ontario.” 
 

Survivorship Care Plans 

Low screening rates may result from a lack of awareness about screening recommendations and the negative long-term effects of cancer treatments, the study authors wrote. Cancer survivors, caregivers, family physicians, specialists, and survivor support groups can share the responsibility of spreading awareness and adhering to guidelines, they noted. In some cases, a survivorship care plan (SCP) may help.

“SCPs are intended to improve adherence by providing follow-up information and facilitating the transition from cancer treatment to survivorship and from pediatric to adult care,” Adam Yan, MD, a staff oncologist and oncology informatics lead at the Hospital for Sick Children in Toronto, told this news organization.

Dr. Yan, who wasn’t involved with this study, has researched surveillance adherence for secondary cancers and cardiac dysfunction among childhood cancer survivors. He and his colleagues found that screening rates were typically low among survivors who faced high risks for cardiac dysfunction and breast, colorectal, or skin cancers.

However, having a survivorship care plan seemed to help, and survivors treated after 1990 were more likely to have an SCP.

“SCP possession by high-risk survivors was associated with increased breast, skin, and cardiac surveillance,” he said. “It is uncertain whether SCP possession leads to adherence or whether SCP possession is a marker of survivors who are focused on their health and thus likely to adhere to preventive health practices, including surveillance.”

The study was funded by the Canadian Institutes of Health Research and ICES, which receives support from the Ontario Ministry of Health and the Ministry of Long-Term Care. Dr. Shuldiner received a Canadian Institutes of Health Research Health System Impact Postdoctoral Fellowship in support of the work. Dr. Yan disclosed no relevant financial relationships. 
 

A version of this article appeared on Medscape.com.

Most survivors of childhood cancer don’t meet surveillance guidelines that recommend screening for adult cancers or other long-term adverse effects of treatment, according to a new study.

Among childhood cancer survivors in Ontario, Canada, who faced an elevated risk due to chemotherapy or radiation treatments, 53% followed screening recommendations for cardiomyopathy, 13% met colorectal cancer screening guidelines, and 6% adhered to breast cancer screening guidelines.

“Although over 80% of children newly diagnosed with cancer will become long-term survivors, as many as four out of five of these survivors will develop a serious or life-threatening late effect of their cancer therapy by age 45,” lead author Jennifer Shuldiner, PhD, MPH, a scientist at Women’s College Hospital Institute for Health Systems Solutions and Virtual Care in Toronto, told this news organization.

For instance, the risk for colorectal cancer in childhood cancer survivors is two to three times higher than it is among the general population, and the risk for breast cancer is similar between those who underwent chest radiation and those with a BRCA mutation. As many as 50% of those who received anthracycline chemotherapy or radiation involving the heart later develop cardiotoxicity.

The North American Children’s Oncology Group has published long-term follow-up guidelines for survivors of childhood cancer, yet many survivors don’t follow them because of lack of awareness or other barriers, said Dr. Shuldiner.

“Prior research has shown that many survivors do not complete these recommended tests,” she said. “With better knowledge of this at-risk population, we can design, test, and implement appropriate interventions and supports to tackle the issues.”

The study was published online on March 11 in CMAJ. 
 

Changes in Adherence 

The researchers conducted a retrospective population-based cohort study analyzing Ontario healthcare administrative data for adult survivors of childhood cancer diagnosed between 1986 and 2014 who faced an elevated risk for therapy-related colorectal cancer, breast cancer, or cardiomyopathy. The research team then assessed long-term adherence to the North American Children’s Oncology Group guidelines and predictors of adherence.

Among 3241 survivors, 3205 (99%) were at elevated risk for cardiomyopathy, 327 (10%) were at elevated risk for colorectal cancer, and 234 (7%) were at elevated risk for breast cancer. In addition, 2806 (87%) were at risk for one late effect, 345 (11%) were at risk for two late effects, and 90 (3%) were at risk for three late effects.

Overall, 53%, 13%, and 6% were adherent to their recommended surveillance for cardiomyopathy, colorectal cancer, and breast cancer, respectively. Over time, adherence increased for colorectal cancer and cardiomyopathy but decreased for breast cancer.

In addition, patients who were older at diagnosis were more likely to follow screening guidelines for colorectal and breast cancers, whereas those who were younger at diagnosis were more likely to follow screening guidelines for cardiomyopathy.

During a median follow-up of 7.8 years, the proportion of time spent adherent was 43% for cardiomyopathy, 14% for colorectal cancer, and 10% for breast cancer.

Survivors who attended a long-term follow-up clinic in the previous year had low adherence rates as well, though they were higher than in the rest of the cohort. In this group, the proportion of time that was spent adherent was 71% for cardiomyopathy, 27% for colorectal cancer, and 15% for breast cancer.

Shuldiner and colleagues are launching a research trial to determine whether a provincial support system can help childhood cancer survivors receive the recommended surveillance. The support system provides information about screening recommendations to survivors as well as reminders and sends key information to their family doctors.

“We now understand that childhood cancer survivors need help to complete the recommended tests,” said Dr. Shuldiner. “If the trial is successful, we hope it will be implemented in Ontario.” 
 

Survivorship Care Plans 

Low screening rates may result from a lack of awareness about screening recommendations and the negative long-term effects of cancer treatments, the study authors wrote. Cancer survivors, caregivers, family physicians, specialists, and survivor support groups can share the responsibility of spreading awareness and adhering to guidelines, they noted. In some cases, a survivorship care plan (SCP) may help.

“SCPs are intended to improve adherence by providing follow-up information and facilitating the transition from cancer treatment to survivorship and from pediatric to adult care,” Adam Yan, MD, a staff oncologist and oncology informatics lead at the Hospital for Sick Children in Toronto, told this news organization.

Dr. Yan, who wasn’t involved with this study, has researched surveillance adherence for secondary cancers and cardiac dysfunction among childhood cancer survivors. He and his colleagues found that screening rates were typically low among survivors who faced high risks for cardiac dysfunction and breast, colorectal, or skin cancers.

However, having a survivorship care plan seemed to help, and survivors treated after 1990 were more likely to have an SCP.

“SCP possession by high-risk survivors was associated with increased breast, skin, and cardiac surveillance,” he said. “It is uncertain whether SCP possession leads to adherence or whether SCP possession is a marker of survivors who are focused on their health and thus likely to adhere to preventive health practices, including surveillance.”

The study was funded by the Canadian Institutes of Health Research and ICES, which receives support from the Ontario Ministry of Health and the Ministry of Long-Term Care. Dr. Shuldiner received a Canadian Institutes of Health Research Health System Impact Postdoctoral Fellowship in support of the work. Dr. Yan disclosed no relevant financial relationships. 
 

A version of this article appeared on Medscape.com.

Most survivors of childhood cancer don’t meet surveillance guidelines that recommend screening for adult cancers or other long-term adverse effects of treatment, according to a new study.

Among childhood cancer survivors in Ontario, Canada, who faced an elevated risk due to chemotherapy or radiation treatments, 53% followed screening recommendations for cardiomyopathy, 13% met colorectal cancer screening guidelines, and 6% adhered to breast cancer screening guidelines.

“Although over 80% of children newly diagnosed with cancer will become long-term survivors, as many as four out of five of these survivors will develop a serious or life-threatening late effect of their cancer therapy by age 45,” lead author Jennifer Shuldiner, PhD, MPH, a scientist at Women’s College Hospital Institute for Health Systems Solutions and Virtual Care in Toronto, told this news organization.

For instance, the risk for colorectal cancer in childhood cancer survivors is two to three times higher than it is among the general population, and the risk for breast cancer is similar between those who underwent chest radiation and those with a BRCA mutation. As many as 50% of those who received anthracycline chemotherapy or radiation involving the heart later develop cardiotoxicity.

The North American Children’s Oncology Group has published long-term follow-up guidelines for survivors of childhood cancer, yet many survivors don’t follow them because of lack of awareness or other barriers, said Dr. Shuldiner.

“Prior research has shown that many survivors do not complete these recommended tests,” she said. “With better knowledge of this at-risk population, we can design, test, and implement appropriate interventions and supports to tackle the issues.”

The study was published online on March 11 in CMAJ. 
 

Changes in Adherence 

The researchers conducted a retrospective population-based cohort study analyzing Ontario healthcare administrative data for adult survivors of childhood cancer diagnosed between 1986 and 2014 who faced an elevated risk for therapy-related colorectal cancer, breast cancer, or cardiomyopathy. The research team then assessed long-term adherence to the North American Children’s Oncology Group guidelines and predictors of adherence.

Among 3241 survivors, 3205 (99%) were at elevated risk for cardiomyopathy, 327 (10%) were at elevated risk for colorectal cancer, and 234 (7%) were at elevated risk for breast cancer. In addition, 2806 (87%) were at risk for one late effect, 345 (11%) were at risk for two late effects, and 90 (3%) were at risk for three late effects.

Overall, 53%, 13%, and 6% were adherent to their recommended surveillance for cardiomyopathy, colorectal cancer, and breast cancer, respectively. Over time, adherence increased for colorectal cancer and cardiomyopathy but decreased for breast cancer.

In addition, patients who were older at diagnosis were more likely to follow screening guidelines for colorectal and breast cancers, whereas those who were younger at diagnosis were more likely to follow screening guidelines for cardiomyopathy.

During a median follow-up of 7.8 years, the proportion of time spent adherent was 43% for cardiomyopathy, 14% for colorectal cancer, and 10% for breast cancer.

Survivors who attended a long-term follow-up clinic in the previous year had low adherence rates as well, though they were higher than in the rest of the cohort. In this group, the proportion of time that was spent adherent was 71% for cardiomyopathy, 27% for colorectal cancer, and 15% for breast cancer.

Shuldiner and colleagues are launching a research trial to determine whether a provincial support system can help childhood cancer survivors receive the recommended surveillance. The support system provides information about screening recommendations to survivors as well as reminders and sends key information to their family doctors.

“We now understand that childhood cancer survivors need help to complete the recommended tests,” said Dr. Shuldiner. “If the trial is successful, we hope it will be implemented in Ontario.” 
 

Survivorship Care Plans 

Low screening rates may result from a lack of awareness about screening recommendations and the negative long-term effects of cancer treatments, the study authors wrote. Cancer survivors, caregivers, family physicians, specialists, and survivor support groups can share the responsibility of spreading awareness and adhering to guidelines, they noted. In some cases, a survivorship care plan (SCP) may help.

“SCPs are intended to improve adherence by providing follow-up information and facilitating the transition from cancer treatment to survivorship and from pediatric to adult care,” Adam Yan, MD, a staff oncologist and oncology informatics lead at the Hospital for Sick Children in Toronto, told this news organization.

Dr. Yan, who wasn’t involved with this study, has researched surveillance adherence for secondary cancers and cardiac dysfunction among childhood cancer survivors. He and his colleagues found that screening rates were typically low among survivors who faced high risks for cardiac dysfunction and breast, colorectal, or skin cancers.

However, having a survivorship care plan seemed to help, and survivors treated after 1990 were more likely to have an SCP.

“SCP possession by high-risk survivors was associated with increased breast, skin, and cardiac surveillance,” he said. “It is uncertain whether SCP possession leads to adherence or whether SCP possession is a marker of survivors who are focused on their health and thus likely to adhere to preventive health practices, including surveillance.”

The study was funded by the Canadian Institutes of Health Research and ICES, which receives support from the Ontario Ministry of Health and the Ministry of Long-Term Care. Dr. Shuldiner received a Canadian Institutes of Health Research Health System Impact Postdoctoral Fellowship in support of the work. Dr. Yan disclosed no relevant financial relationships. 
 

A version of this article appeared on Medscape.com.

Living in a disadvantaged neighborhood is associated with accelerated brain aging and a higher risk for early dementia, regardless of income level or education, new research suggested.

Analysis of two datasets revealed that people living in the most disadvantaged neighborhoods had a more than 20% higher risk for dementia than those in other areas and measurably poorer brain health as early as age 45, regardless of their own personal income and education.

“If you want to prevent dementia and you’re not asking someone about their neighborhood, you’re missing information that’s important to know,” lead author Aaron Reuben, PhD, postdoctoral scholar in neuropsychology and environmental health at Duke University, Durham, North Carolina, said in a news release.

The study was published online in Alzheimer’s & Dementia.

Higher Risk in Men

Few interventions exist to halt or delay the progression of Alzheimer’s disease and related dementias (ADRD), which has increasingly led to a focus on primary prevention.

Although previous research pointed to a link between socioeconomically disadvantaged neighborhoods and a greater risk for cognitive deficits, mild cognitive impairment, dementia, and poor brain health, the timeline for the emergence of that risk is unknown.

To fill in the gaps, investigators studied data on all 1.4 million New Zealand residents, dividing neighborhoods into quintiles based on level of disadvantage (assessed by the New Zealand Index of Deprivation) to see whether dementia diagnoses followed neighborhood socioeconomic gradients.

After adjusting for covariates, they found that overall, those living in disadvantaged areas were slightly more likely to develop dementia across the 20-year study period (adjusted hazard ratio [HR], 1.09; 95% CI, 1.08-1.10).

The more disadvantaged the neighborhood, the higher the dementia risk, with a 43% higher risk for ADRD among those in the highest quintile than among those in the lowest quintile (HR, 1.43; 95% CI, 1.36-1.49).

The effect was larger in men than in women and in younger vs older individuals, with the youngest age group showing 21% greater risk in women and 26% greater risk in men vs the oldest age group.

Dementia Prevention Starts Early

Researchers then turned to the Dunedin Study, a cohort of 938 New Zealanders (50% female) followed from birth to age 45 to track their psychological, social, and physiological health with brain scans, memory tests, and cognitive self-assessments.

The analysis suggested that by age 45, those living in more disadvantaged neighborhoods across adulthood had accumulated a significantly greater number of midlife risk factors for later ADRD.

They also had worse structural brain integrity, with each standard deviation increase in neighborhood disadvantage resulting in a thinner cortex, greater white matter hyperintensities volume, and older brain age.

Those living in poorer areas had lower cognitive test scores, reported more issues with everyday cognitive function, and showed a greater reduction in IQ from childhood to midlife. Analysis of brain scans also revealed mean brain ages 2.98 years older than those living in the least disadvantaged areas (P = .001).

Limitations included the study’s observational design, which could not establish causation, and the fact that the researchers did not have access to individual-level socioeconomic information for the entire population. Additionally, brain-integrity measures in the Dunedin Study were largely cross-sectional.

“If you want to truly prevent dementia, you’ve got to start early because 20 years before anyone will get a diagnosis, we’re seeing dementia’s emergence,” Dr. Reuben said. “And it could be even earlier.”

Funding for the study was provided by the National Institutes for Health; UK Medical Research Council; Health Research Council of New Zealand; Brain Research New Zealand; New Zealand Ministry of Business, Innovation, & Employment; and the Duke University and the University of North Carolina Alzheimer’s Disease Research Center. The authors declared no relevant financial relationships.

A version of this article appeared on Medscape.com.

Living in a disadvantaged neighborhood is associated with accelerated brain aging and a higher risk for early dementia, regardless of income level or education, new research suggested.

Analysis of two datasets revealed that people living in the most disadvantaged neighborhoods had a more than 20% higher risk for dementia than those in other areas and measurably poorer brain health as early as age 45, regardless of their own personal income and education.

“If you want to prevent dementia and you’re not asking someone about their neighborhood, you’re missing information that’s important to know,” lead author Aaron Reuben, PhD, postdoctoral scholar in neuropsychology and environmental health at Duke University, Durham, North Carolina, said in a news release.

The study was published online in Alzheimer’s & Dementia.

Higher Risk in Men

Few interventions exist to halt or delay the progression of Alzheimer’s disease and related dementias (ADRD), which has increasingly led to a focus on primary prevention.

Although previous research pointed to a link between socioeconomically disadvantaged neighborhoods and a greater risk for cognitive deficits, mild cognitive impairment, dementia, and poor brain health, the timeline for the emergence of that risk is unknown.

To fill in the gaps, investigators studied data on all 1.4 million New Zealand residents, dividing neighborhoods into quintiles based on level of disadvantage (assessed by the New Zealand Index of Deprivation) to see whether dementia diagnoses followed neighborhood socioeconomic gradients.

After adjusting for covariates, they found that overall, those living in disadvantaged areas were slightly more likely to develop dementia across the 20-year study period (adjusted hazard ratio [HR], 1.09; 95% CI, 1.08-1.10).

The more disadvantaged the neighborhood, the higher the dementia risk, with a 43% higher risk for ADRD among those in the highest quintile than among those in the lowest quintile (HR, 1.43; 95% CI, 1.36-1.49).

The effect was larger in men than in women and in younger vs older individuals, with the youngest age group showing 21% greater risk in women and 26% greater risk in men vs the oldest age group.

Dementia Prevention Starts Early

Researchers then turned to the Dunedin Study, a cohort of 938 New Zealanders (50% female) followed from birth to age 45 to track their psychological, social, and physiological health with brain scans, memory tests, and cognitive self-assessments.

The analysis suggested that by age 45, those living in more disadvantaged neighborhoods across adulthood had accumulated a significantly greater number of midlife risk factors for later ADRD.

They also had worse structural brain integrity, with each standard deviation increase in neighborhood disadvantage resulting in a thinner cortex, greater white matter hyperintensities volume, and older brain age.

Those living in poorer areas had lower cognitive test scores, reported more issues with everyday cognitive function, and showed a greater reduction in IQ from childhood to midlife. Analysis of brain scans also revealed mean brain ages 2.98 years older than those living in the least disadvantaged areas (P = .001).

Limitations included the study’s observational design, which could not establish causation, and the fact that the researchers did not have access to individual-level socioeconomic information for the entire population. Additionally, brain-integrity measures in the Dunedin Study were largely cross-sectional.

“If you want to truly prevent dementia, you’ve got to start early because 20 years before anyone will get a diagnosis, we’re seeing dementia’s emergence,” Dr. Reuben said. “And it could be even earlier.”

Funding for the study was provided by the National Institutes for Health; UK Medical Research Council; Health Research Council of New Zealand; Brain Research New Zealand; New Zealand Ministry of Business, Innovation, & Employment; and the Duke University and the University of North Carolina Alzheimer’s Disease Research Center. The authors declared no relevant financial relationships.

A version of this article appeared on Medscape.com.

Living in a disadvantaged neighborhood is associated with accelerated brain aging and a higher risk for early dementia, regardless of income level or education, new research suggested.

Analysis of two datasets revealed that people living in the most disadvantaged neighborhoods had a more than 20% higher risk for dementia than those in other areas and measurably poorer brain health as early as age 45, regardless of their own personal income and education.

“If you want to prevent dementia and you’re not asking someone about their neighborhood, you’re missing information that’s important to know,” lead author Aaron Reuben, PhD, postdoctoral scholar in neuropsychology and environmental health at Duke University, Durham, North Carolina, said in a news release.

The study was published online in Alzheimer’s & Dementia.

Higher Risk in Men

Few interventions exist to halt or delay the progression of Alzheimer’s disease and related dementias (ADRD), which has increasingly led to a focus on primary prevention.

Although previous research pointed to a link between socioeconomically disadvantaged neighborhoods and a greater risk for cognitive deficits, mild cognitive impairment, dementia, and poor brain health, the timeline for the emergence of that risk is unknown.

To fill in the gaps, investigators studied data on all 1.4 million New Zealand residents, dividing neighborhoods into quintiles based on level of disadvantage (assessed by the New Zealand Index of Deprivation) to see whether dementia diagnoses followed neighborhood socioeconomic gradients.

After adjusting for covariates, they found that overall, those living in disadvantaged areas were slightly more likely to develop dementia across the 20-year study period (adjusted hazard ratio [HR], 1.09; 95% CI, 1.08-1.10).

The more disadvantaged the neighborhood, the higher the dementia risk, with a 43% higher risk for ADRD among those in the highest quintile than among those in the lowest quintile (HR, 1.43; 95% CI, 1.36-1.49).

The effect was larger in men than in women and in younger vs older individuals, with the youngest age group showing 21% greater risk in women and 26% greater risk in men vs the oldest age group.

Dementia Prevention Starts Early

Researchers then turned to the Dunedin Study, a cohort of 938 New Zealanders (50% female) followed from birth to age 45 to track their psychological, social, and physiological health with brain scans, memory tests, and cognitive self-assessments.

The analysis suggested that by age 45, those living in more disadvantaged neighborhoods across adulthood had accumulated a significantly greater number of midlife risk factors for later ADRD.

They also had worse structural brain integrity, with each standard deviation increase in neighborhood disadvantage resulting in a thinner cortex, greater white matter hyperintensities volume, and older brain age.

Those living in poorer areas had lower cognitive test scores, reported more issues with everyday cognitive function, and showed a greater reduction in IQ from childhood to midlife. Analysis of brain scans also revealed mean brain ages 2.98 years older than those living in the least disadvantaged areas (P = .001).

Limitations included the study’s observational design, which could not establish causation, and the fact that the researchers did not have access to individual-level socioeconomic information for the entire population. Additionally, brain-integrity measures in the Dunedin Study were largely cross-sectional.

“If you want to truly prevent dementia, you’ve got to start early because 20 years before anyone will get a diagnosis, we’re seeing dementia’s emergence,” Dr. Reuben said. “And it could be even earlier.”

Funding for the study was provided by the National Institutes for Health; UK Medical Research Council; Health Research Council of New Zealand; Brain Research New Zealand; New Zealand Ministry of Business, Innovation, & Employment; and the Duke University and the University of North Carolina Alzheimer’s Disease Research Center. The authors declared no relevant financial relationships.

A version of this article appeared on Medscape.com.

Three genetically targeted drugs for Duchenne muscular dystrophy (DMD) — eteplirsen, golodirsen, and casimersen — cost the US health care system more than $3 billion between 2016 and 2022, despite a lack of confirmatory efficacy data, a new analysis showed. 

“We were certainly surprised to see how much was spent on these drugs during the period when we were still waiting for evidence to confirm whether or not they were effective,” study investigator Benjamin Rome, MD, MPH, with the Program on Regulation, Therapeutics, and Law, Harvard Medical School and Brigham and Women’s Hospital, Boston, told this news organization.

“With these drugs often costing over $1 million a year, these results show how spending can add up even for drugs that treat a rare disease,” Dr. Rome added. 

The study was published online March 11, 2024, in JAMA. 
 

No Confirmatory Research

Investigators estimated public and private spending on eteplirsen, golodirsen, and casimersen for DMD during 2016 and 2022 — years in which these drugs were marketed without the required confirmatory studies completed.

Annual net sales, which include rebates and statutory discounts to Medicaid or 340B entities, for the three drugs totaled $3.1 billion during the study period. Estimated Medicaid and Medicare spending accounted for $1.2 billion of that total. Of this total, Medicaid programs spent $1.1 billion (34% of US net sales) and Medicare spent $104 million (3% of US net sales).

Overall sales for the drugs increased from $7 million in 2016 to $879 million in 2022, while Medicaid and Medicare spending rose from $25 million in 2017 to $327 million in 2022.

Most of the spending on these therapies was for eteplirsen ($2.6 billion [82%]), “the efficacy of which has yet to be determined in a confirmatory trial more than 7 years after the drug’s accelerated approval,” the authors noted.

Of the total amount spent on the three drugs, US payers spent an estimated $301 million (10%) on casimersen and $263 million (8%) on golodirsen.

The findings point to the importance of follow up on drugs that are approved with preliminary evidence, Rome said. 

“Congress and the US Food and Drug Administration have already made some important changes to the accelerated approval pathway, so hopefully we won’t see cases of multi-year delays in the future,” he said.

“Payers, including public payers like Medicare and Medicaid, need tools to financially encourage companies to complete the follow-up trials, such as paying less for drugs with accelerated approval or engaging in outcomes-based contracts to ensure they don’t pay billions of dollars for drugs that ultimately turn out not to be effective,” Dr. Rome added.

Reached for comment, Adam C. Powell, PhD, president, Payer+Provider Syndicate, noted that when a condition impacts a small population, as is the case with muscular dystrophy, there are fewer people over which to spread the cost of treatment development.

Dr. Powell pointed to a recent report that showed the average cost of developing a new drug exceeds $2 billion. The finding in the current study, that three DMD treatments had combined net sales of $3.1 billion over a 7-year period, “suggests that their developers may not have yet recouped their development costs,” Dr. Powell told this news organization. 

“Unless the cost of drug development can be lessened through innovations in artificial intelligence or other means, high spending per patient for drugs addressing uncommon conditions is to be expected,” noted Dr. Powell, who was not part of the study. 

“That said, it is concerning when substantial funds are being spent by public payers on treatments that do not work,” he added. “As the authors suggest, one option is to tie reimbursement to efficacy. While patients living with deadly conditions cannot indefinitely wait for treatments to be validated, clawing back payments in the event of inefficacy is always an option.” 

The study was funded by Arnold Ventures. Dr. Rome reported receiving grants from the Elevance Health Public Policy Institute, the National Academy for State Health Policy, and several state prescription drug affordability boards outside the submitted work. Powell had no relevant disclosures.
 

A version of this article appeared on Medscape.com .

Three genetically targeted drugs for Duchenne muscular dystrophy (DMD) — eteplirsen, golodirsen, and casimersen — cost the US health care system more than $3 billion between 2016 and 2022, despite a lack of confirmatory efficacy data, a new analysis showed. 

“We were certainly surprised to see how much was spent on these drugs during the period when we were still waiting for evidence to confirm whether or not they were effective,” study investigator Benjamin Rome, MD, MPH, with the Program on Regulation, Therapeutics, and Law, Harvard Medical School and Brigham and Women’s Hospital, Boston, told this news organization.

“With these drugs often costing over $1 million a year, these results show how spending can add up even for drugs that treat a rare disease,” Dr. Rome added. 

The study was published online March 11, 2024, in JAMA. 
 

No Confirmatory Research

Investigators estimated public and private spending on eteplirsen, golodirsen, and casimersen for DMD during 2016 and 2022 — years in which these drugs were marketed without the required confirmatory studies completed.

Annual net sales, which include rebates and statutory discounts to Medicaid or 340B entities, for the three drugs totaled $3.1 billion during the study period. Estimated Medicaid and Medicare spending accounted for $1.2 billion of that total. Of this total, Medicaid programs spent $1.1 billion (34% of US net sales) and Medicare spent $104 million (3% of US net sales).

Overall sales for the drugs increased from $7 million in 2016 to $879 million in 2022, while Medicaid and Medicare spending rose from $25 million in 2017 to $327 million in 2022.

Most of the spending on these therapies was for eteplirsen ($2.6 billion [82%]), “the efficacy of which has yet to be determined in a confirmatory trial more than 7 years after the drug’s accelerated approval,” the authors noted.

Of the total amount spent on the three drugs, US payers spent an estimated $301 million (10%) on casimersen and $263 million (8%) on golodirsen.

The findings point to the importance of follow up on drugs that are approved with preliminary evidence, Rome said. 

“Congress and the US Food and Drug Administration have already made some important changes to the accelerated approval pathway, so hopefully we won’t see cases of multi-year delays in the future,” he said.

“Payers, including public payers like Medicare and Medicaid, need tools to financially encourage companies to complete the follow-up trials, such as paying less for drugs with accelerated approval or engaging in outcomes-based contracts to ensure they don’t pay billions of dollars for drugs that ultimately turn out not to be effective,” Dr. Rome added.

Reached for comment, Adam C. Powell, PhD, president, Payer+Provider Syndicate, noted that when a condition impacts a small population, as is the case with muscular dystrophy, there are fewer people over which to spread the cost of treatment development.

Dr. Powell pointed to a recent report that showed the average cost of developing a new drug exceeds $2 billion. The finding in the current study, that three DMD treatments had combined net sales of $3.1 billion over a 7-year period, “suggests that their developers may not have yet recouped their development costs,” Dr. Powell told this news organization. 

“Unless the cost of drug development can be lessened through innovations in artificial intelligence or other means, high spending per patient for drugs addressing uncommon conditions is to be expected,” noted Dr. Powell, who was not part of the study. 

“That said, it is concerning when substantial funds are being spent by public payers on treatments that do not work,” he added. “As the authors suggest, one option is to tie reimbursement to efficacy. While patients living with deadly conditions cannot indefinitely wait for treatments to be validated, clawing back payments in the event of inefficacy is always an option.” 

The study was funded by Arnold Ventures. Dr. Rome reported receiving grants from the Elevance Health Public Policy Institute, the National Academy for State Health Policy, and several state prescription drug affordability boards outside the submitted work. Powell had no relevant disclosures.
 

A version of this article appeared on Medscape.com .

Three genetically targeted drugs for Duchenne muscular dystrophy (DMD) — eteplirsen, golodirsen, and casimersen — cost the US health care system more than $3 billion between 2016 and 2022, despite a lack of confirmatory efficacy data, a new analysis showed. 

“We were certainly surprised to see how much was spent on these drugs during the period when we were still waiting for evidence to confirm whether or not they were effective,” study investigator Benjamin Rome, MD, MPH, with the Program on Regulation, Therapeutics, and Law, Harvard Medical School and Brigham and Women’s Hospital, Boston, told this news organization.

“With these drugs often costing over $1 million a year, these results show how spending can add up even for drugs that treat a rare disease,” Dr. Rome added. 

The study was published online March 11, 2024, in JAMA. 
 

No Confirmatory Research

Investigators estimated public and private spending on eteplirsen, golodirsen, and casimersen for DMD during 2016 and 2022 — years in which these drugs were marketed without the required confirmatory studies completed.

Annual net sales, which include rebates and statutory discounts to Medicaid or 340B entities, for the three drugs totaled $3.1 billion during the study period. Estimated Medicaid and Medicare spending accounted for $1.2 billion of that total. Of this total, Medicaid programs spent $1.1 billion (34% of US net sales) and Medicare spent $104 million (3% of US net sales).

Overall sales for the drugs increased from $7 million in 2016 to $879 million in 2022, while Medicaid and Medicare spending rose from $25 million in 2017 to $327 million in 2022.

Most of the spending on these therapies was for eteplirsen ($2.6 billion [82%]), “the efficacy of which has yet to be determined in a confirmatory trial more than 7 years after the drug’s accelerated approval,” the authors noted.

Of the total amount spent on the three drugs, US payers spent an estimated $301 million (10%) on casimersen and $263 million (8%) on golodirsen.

The findings point to the importance of follow up on drugs that are approved with preliminary evidence, Rome said. 

“Congress and the US Food and Drug Administration have already made some important changes to the accelerated approval pathway, so hopefully we won’t see cases of multi-year delays in the future,” he said.

“Payers, including public payers like Medicare and Medicaid, need tools to financially encourage companies to complete the follow-up trials, such as paying less for drugs with accelerated approval or engaging in outcomes-based contracts to ensure they don’t pay billions of dollars for drugs that ultimately turn out not to be effective,” Dr. Rome added.

Reached for comment, Adam C. Powell, PhD, president, Payer+Provider Syndicate, noted that when a condition impacts a small population, as is the case with muscular dystrophy, there are fewer people over which to spread the cost of treatment development.

Dr. Powell pointed to a recent report that showed the average cost of developing a new drug exceeds $2 billion. The finding in the current study, that three DMD treatments had combined net sales of $3.1 billion over a 7-year period, “suggests that their developers may not have yet recouped their development costs,” Dr. Powell told this news organization. 

“Unless the cost of drug development can be lessened through innovations in artificial intelligence or other means, high spending per patient for drugs addressing uncommon conditions is to be expected,” noted Dr. Powell, who was not part of the study. 

“That said, it is concerning when substantial funds are being spent by public payers on treatments that do not work,” he added. “As the authors suggest, one option is to tie reimbursement to efficacy. While patients living with deadly conditions cannot indefinitely wait for treatments to be validated, clawing back payments in the event of inefficacy is always an option.” 

The study was funded by Arnold Ventures. Dr. Rome reported receiving grants from the Elevance Health Public Policy Institute, the National Academy for State Health Policy, and several state prescription drug affordability boards outside the submitted work. Powell had no relevant disclosures.
 

A version of this article appeared on Medscape.com .