Conference Coverage

Wildfire-related air pollution in Europe kills more than non-wildfire air pollution. As climate change exacerbates the frequency and violence of wildfires, researchers are studying the health implications of mitigation methods such as prescribed fires.

Presenting at the annual congress of the European Respiratory Society (ERS), Cathryn Tonne, PhD, an environmental epidemiologist at the Instituto de Salud Global de Barcelona, Spain, said wildfire-related PM^2.5 is more toxic than general PM^2.5, leading to significantly higher mortality rates.

Prescribed, controlled fires have been employed worldwide to reduce the chance of uncontrolled, catastrophic fires. However, researchers wonder whether the techniques reduce the overall fire-related PM^2.5 or add up to it. “Prescribed fire increases ecosystem resilience and can reduce the risk of catastrophic wildfire,” said Jason Sacks, MPH, an epidemiologist in the Center for Public Health and Environmental Assessment in the Office of Research and Development at the Environmental Protection Agency (EPA), at the congress. “But it also leads to poorer air quality and health impacts, and we still don’t know what this means at a regional scale.”
 

Wildfire Pollution Kills More Than Other Air Pollution

Researchers at the Instituto de Salud Global de Barcelona used a large dataset of daily mortality data from 32 European countries collected through the EARLY-ADAPT project. They utilized the SILAM model to derive daily average concentrations of wildfire-related PM^2.5, non-fire PM^2.5, and total PM^2.5 levels. They also employed GEOSTAT population grids at a 1-km resolution to calculate the attributable number of deaths across different regions, specifically focusing on data from 2006, 2011, and 2018.

The data analysis indicated that the relative risk per unit of PM^2.5 is substantially larger for wildfire-related PM2.5, compared with non-fire PM2.5. “We essentially assume that wildfire smoke PM^2.5 has the same toxicity as total PM2.5, but it’s increasingly clear that’s likely not the case,” Dr. Tonne said, presenting the study.

When employing exposure-response functions (ERFs) specific to wildfire smoke, researchers found that the attributable deaths from all causes of wildfire PM^2.5 were approximately 10 times larger than those calculated using total PM^2.5 exposure estimates. Dr. Tonne explained that this stark difference highlights the critical need for tailored ERFs that accurately reflect the unique health risks posed by wildfire smoke.

“Respiratory mortality usually has the strongest relative risks, and we’re seeing that in this study as well,” Dr. Tonne said. “Wildfire smoke seems to operate through quite immediate mechanisms, likely through inflammation and oxidative stress.”

One significant challenge of the study was the lack of uniform spatial resolution across all countries involved in the analysis. This inconsistency may affect how accurately mortality estimates can be attributed to specific PM^2.5 sources. Additionally, the study had limited statistical power for generating age- and sex-specific mortality estimates, which could obscure important demographic differences in vulnerability to wildfire smoke exposure. The analysis was also constrained to data available only up to 2020, thereby excluding critical wildfire events from subsequent years, such as those in 2022 and 2023, which may have further elucidated the health impacts of wildfire smoke in Europe.
 

Fires Prescription

Prescribed fires or controlled burns are intentional fires set by land managers under carefully managed conditions.

Historically, many forested areas have been subjected to fire suppression practices, which allow combustible materials like dry leaves, twigs, and shrubs to accumulate over time. This buildup leads to a higher likelihood of severe, uncontrollable wildfires. Prescribed fires can reduce these fuel loads and improve the health and resilience of ecosystems.

They release fewer pollutants and emissions than the large-scale, unmanageable wildfires they help prevent because they happen at lower temperatures. But they still introduce pollutants in the air that can negatively affect nearby communities’ health.

People with preexisting respiratory conditions, such as asthma or chronic obstructive pulmonary disease (COPD), are particularly vulnerable to smoke, which can trigger health issues like breathing difficulties, coughing, and eye irritation. The cumulative impact of increased burns raises concerns about long-term air quality, especially in densely populated areas. “We need to understand if we’re actually tipping the scale to having less wildfire smoke or just increasing the total amount of smoke.”

Mitigation strategies include accurately picking the right timing and weather conditions to determine when and where to conduct controlled burns and effective and timely communication to inform local communities about upcoming burns, the potential for smoke exposure, and how to protect themselves.

There is a growing need to improve public messaging around prescribed fires, Mr. Sacks said, because often the message communicated is oversimplified, such as “there will be smoke, but don’t worry. But that’s not the message we want to convey, especially for people with asthma or COPD.”

Instead, he said public health agencies should provide clearer, science-based guidance on the risks for smoke exposure and practical steps people can take to reduce their risk.
 

What Can Doctors Do?

Chris Carlsten, MD, director of the Centre for Lung Health and professor and head of the Respiratory Medicine Division at the University of British Columbia, Vancouver, Canada, told this news organization that determining whether an exacerbation of a respiratory condition is caused by fire exposure or other factors, such as viral infections, is complex because both can trigger similar responses and may complement each other. “It’s very difficult for any individual to know whether, when they’re having an exacerbation of asthma or COPD, that’s due to the fire,” he said. Fire smoke also increases infection risks, further complicating diagnosis.

Dr. Carlsten suggested that physicians could recommend preventative use of inhalers for at-risk patients when wildfires occur rather than waiting for symptoms to worsen. “That is a really interesting idea that could be practical.” Still, he advises caution, stressing that patients should consult their providers because not all may react well to increased inhaler use.

He also highlighted a significant shift in the healthcare landscape, noting that traditionally, the focus has been on the cardiovascular impacts of pollution, particularly traffic-related pollution. However, as wildfire smoke becomes a growing issue, the focus is shifting back to respiratory problems, with profound implications for healthcare resources, budgets, and drug approvals based on the burden of respiratory disease. “Fire smoke is becoming more of a problem. This swing back to respiratory has huge implications for healthcare systems and respiratory disease burden.”

Mr. Sacks and Dr. Carlsten reported no relevant financial relationships. The study presented by Dr. Tonne received funding from the European Union’s Horizon Europe research and innovation programme under Grant Agreement No. 101057131.
 

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

Wildfire-related air pollution in Europe kills more than non-wildfire air pollution. As climate change exacerbates the frequency and violence of wildfires, researchers are studying the health implications of mitigation methods such as prescribed fires.

Presenting at the annual congress of the European Respiratory Society (ERS), Cathryn Tonne, PhD, an environmental epidemiologist at the Instituto de Salud Global de Barcelona, Spain, said wildfire-related PM^2.5 is more toxic than general PM^2.5, leading to significantly higher mortality rates.

Prescribed, controlled fires have been employed worldwide to reduce the chance of uncontrolled, catastrophic fires. However, researchers wonder whether the techniques reduce the overall fire-related PM^2.5 or add up to it. “Prescribed fire increases ecosystem resilience and can reduce the risk of catastrophic wildfire,” said Jason Sacks, MPH, an epidemiologist in the Center for Public Health and Environmental Assessment in the Office of Research and Development at the Environmental Protection Agency (EPA), at the congress. “But it also leads to poorer air quality and health impacts, and we still don’t know what this means at a regional scale.”
 

Wildfire Pollution Kills More Than Other Air Pollution

Researchers at the Instituto de Salud Global de Barcelona used a large dataset of daily mortality data from 32 European countries collected through the EARLY-ADAPT project. They utilized the SILAM model to derive daily average concentrations of wildfire-related PM^2.5, non-fire PM^2.5, and total PM^2.5 levels. They also employed GEOSTAT population grids at a 1-km resolution to calculate the attributable number of deaths across different regions, specifically focusing on data from 2006, 2011, and 2018.

The data analysis indicated that the relative risk per unit of PM^2.5 is substantially larger for wildfire-related PM2.5, compared with non-fire PM2.5. “We essentially assume that wildfire smoke PM^2.5 has the same toxicity as total PM2.5, but it’s increasingly clear that’s likely not the case,” Dr. Tonne said, presenting the study.

When employing exposure-response functions (ERFs) specific to wildfire smoke, researchers found that the attributable deaths from all causes of wildfire PM^2.5 were approximately 10 times larger than those calculated using total PM^2.5 exposure estimates. Dr. Tonne explained that this stark difference highlights the critical need for tailored ERFs that accurately reflect the unique health risks posed by wildfire smoke.

“Respiratory mortality usually has the strongest relative risks, and we’re seeing that in this study as well,” Dr. Tonne said. “Wildfire smoke seems to operate through quite immediate mechanisms, likely through inflammation and oxidative stress.”

One significant challenge of the study was the lack of uniform spatial resolution across all countries involved in the analysis. This inconsistency may affect how accurately mortality estimates can be attributed to specific PM^2.5 sources. Additionally, the study had limited statistical power for generating age- and sex-specific mortality estimates, which could obscure important demographic differences in vulnerability to wildfire smoke exposure. The analysis was also constrained to data available only up to 2020, thereby excluding critical wildfire events from subsequent years, such as those in 2022 and 2023, which may have further elucidated the health impacts of wildfire smoke in Europe.
 

Fires Prescription

Prescribed fires or controlled burns are intentional fires set by land managers under carefully managed conditions.

Historically, many forested areas have been subjected to fire suppression practices, which allow combustible materials like dry leaves, twigs, and shrubs to accumulate over time. This buildup leads to a higher likelihood of severe, uncontrollable wildfires. Prescribed fires can reduce these fuel loads and improve the health and resilience of ecosystems.

They release fewer pollutants and emissions than the large-scale, unmanageable wildfires they help prevent because they happen at lower temperatures. But they still introduce pollutants in the air that can negatively affect nearby communities’ health.

People with preexisting respiratory conditions, such as asthma or chronic obstructive pulmonary disease (COPD), are particularly vulnerable to smoke, which can trigger health issues like breathing difficulties, coughing, and eye irritation. The cumulative impact of increased burns raises concerns about long-term air quality, especially in densely populated areas. “We need to understand if we’re actually tipping the scale to having less wildfire smoke or just increasing the total amount of smoke.”

Mitigation strategies include accurately picking the right timing and weather conditions to determine when and where to conduct controlled burns and effective and timely communication to inform local communities about upcoming burns, the potential for smoke exposure, and how to protect themselves.

There is a growing need to improve public messaging around prescribed fires, Mr. Sacks said, because often the message communicated is oversimplified, such as “there will be smoke, but don’t worry. But that’s not the message we want to convey, especially for people with asthma or COPD.”

Instead, he said public health agencies should provide clearer, science-based guidance on the risks for smoke exposure and practical steps people can take to reduce their risk.
 

What Can Doctors Do?

Chris Carlsten, MD, director of the Centre for Lung Health and professor and head of the Respiratory Medicine Division at the University of British Columbia, Vancouver, Canada, told this news organization that determining whether an exacerbation of a respiratory condition is caused by fire exposure or other factors, such as viral infections, is complex because both can trigger similar responses and may complement each other. “It’s very difficult for any individual to know whether, when they’re having an exacerbation of asthma or COPD, that’s due to the fire,” he said. Fire smoke also increases infection risks, further complicating diagnosis.

Dr. Carlsten suggested that physicians could recommend preventative use of inhalers for at-risk patients when wildfires occur rather than waiting for symptoms to worsen. “That is a really interesting idea that could be practical.” Still, he advises caution, stressing that patients should consult their providers because not all may react well to increased inhaler use.

He also highlighted a significant shift in the healthcare landscape, noting that traditionally, the focus has been on the cardiovascular impacts of pollution, particularly traffic-related pollution. However, as wildfire smoke becomes a growing issue, the focus is shifting back to respiratory problems, with profound implications for healthcare resources, budgets, and drug approvals based on the burden of respiratory disease. “Fire smoke is becoming more of a problem. This swing back to respiratory has huge implications for healthcare systems and respiratory disease burden.”

Mr. Sacks and Dr. Carlsten reported no relevant financial relationships. The study presented by Dr. Tonne received funding from the European Union’s Horizon Europe research and innovation programme under Grant Agreement No. 101057131.
 

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

Wildfire-related air pollution in Europe kills more than non-wildfire air pollution. As climate change exacerbates the frequency and violence of wildfires, researchers are studying the health implications of mitigation methods such as prescribed fires.

Presenting at the annual congress of the European Respiratory Society (ERS), Cathryn Tonne, PhD, an environmental epidemiologist at the Instituto de Salud Global de Barcelona, Spain, said wildfire-related PM^2.5 is more toxic than general PM^2.5, leading to significantly higher mortality rates.

Prescribed, controlled fires have been employed worldwide to reduce the chance of uncontrolled, catastrophic fires. However, researchers wonder whether the techniques reduce the overall fire-related PM^2.5 or add up to it. “Prescribed fire increases ecosystem resilience and can reduce the risk of catastrophic wildfire,” said Jason Sacks, MPH, an epidemiologist in the Center for Public Health and Environmental Assessment in the Office of Research and Development at the Environmental Protection Agency (EPA), at the congress. “But it also leads to poorer air quality and health impacts, and we still don’t know what this means at a regional scale.”
 

Wildfire Pollution Kills More Than Other Air Pollution

Researchers at the Instituto de Salud Global de Barcelona used a large dataset of daily mortality data from 32 European countries collected through the EARLY-ADAPT project. They utilized the SILAM model to derive daily average concentrations of wildfire-related PM^2.5, non-fire PM^2.5, and total PM^2.5 levels. They also employed GEOSTAT population grids at a 1-km resolution to calculate the attributable number of deaths across different regions, specifically focusing on data from 2006, 2011, and 2018.

The data analysis indicated that the relative risk per unit of PM^2.5 is substantially larger for wildfire-related PM2.5, compared with non-fire PM2.5. “We essentially assume that wildfire smoke PM^2.5 has the same toxicity as total PM2.5, but it’s increasingly clear that’s likely not the case,” Dr. Tonne said, presenting the study.

When employing exposure-response functions (ERFs) specific to wildfire smoke, researchers found that the attributable deaths from all causes of wildfire PM^2.5 were approximately 10 times larger than those calculated using total PM^2.5 exposure estimates. Dr. Tonne explained that this stark difference highlights the critical need for tailored ERFs that accurately reflect the unique health risks posed by wildfire smoke.

“Respiratory mortality usually has the strongest relative risks, and we’re seeing that in this study as well,” Dr. Tonne said. “Wildfire smoke seems to operate through quite immediate mechanisms, likely through inflammation and oxidative stress.”

One significant challenge of the study was the lack of uniform spatial resolution across all countries involved in the analysis. This inconsistency may affect how accurately mortality estimates can be attributed to specific PM^2.5 sources. Additionally, the study had limited statistical power for generating age- and sex-specific mortality estimates, which could obscure important demographic differences in vulnerability to wildfire smoke exposure. The analysis was also constrained to data available only up to 2020, thereby excluding critical wildfire events from subsequent years, such as those in 2022 and 2023, which may have further elucidated the health impacts of wildfire smoke in Europe.
 

Fires Prescription

Prescribed fires or controlled burns are intentional fires set by land managers under carefully managed conditions.

Historically, many forested areas have been subjected to fire suppression practices, which allow combustible materials like dry leaves, twigs, and shrubs to accumulate over time. This buildup leads to a higher likelihood of severe, uncontrollable wildfires. Prescribed fires can reduce these fuel loads and improve the health and resilience of ecosystems.

They release fewer pollutants and emissions than the large-scale, unmanageable wildfires they help prevent because they happen at lower temperatures. But they still introduce pollutants in the air that can negatively affect nearby communities’ health.

People with preexisting respiratory conditions, such as asthma or chronic obstructive pulmonary disease (COPD), are particularly vulnerable to smoke, which can trigger health issues like breathing difficulties, coughing, and eye irritation. The cumulative impact of increased burns raises concerns about long-term air quality, especially in densely populated areas. “We need to understand if we’re actually tipping the scale to having less wildfire smoke or just increasing the total amount of smoke.”

Mitigation strategies include accurately picking the right timing and weather conditions to determine when and where to conduct controlled burns and effective and timely communication to inform local communities about upcoming burns, the potential for smoke exposure, and how to protect themselves.

There is a growing need to improve public messaging around prescribed fires, Mr. Sacks said, because often the message communicated is oversimplified, such as “there will be smoke, but don’t worry. But that’s not the message we want to convey, especially for people with asthma or COPD.”

Instead, he said public health agencies should provide clearer, science-based guidance on the risks for smoke exposure and practical steps people can take to reduce their risk.
 

What Can Doctors Do?

Chris Carlsten, MD, director of the Centre for Lung Health and professor and head of the Respiratory Medicine Division at the University of British Columbia, Vancouver, Canada, told this news organization that determining whether an exacerbation of a respiratory condition is caused by fire exposure or other factors, such as viral infections, is complex because both can trigger similar responses and may complement each other. “It’s very difficult for any individual to know whether, when they’re having an exacerbation of asthma or COPD, that’s due to the fire,” he said. Fire smoke also increases infection risks, further complicating diagnosis.

Dr. Carlsten suggested that physicians could recommend preventative use of inhalers for at-risk patients when wildfires occur rather than waiting for symptoms to worsen. “That is a really interesting idea that could be practical.” Still, he advises caution, stressing that patients should consult their providers because not all may react well to increased inhaler use.

He also highlighted a significant shift in the healthcare landscape, noting that traditionally, the focus has been on the cardiovascular impacts of pollution, particularly traffic-related pollution. However, as wildfire smoke becomes a growing issue, the focus is shifting back to respiratory problems, with profound implications for healthcare resources, budgets, and drug approvals based on the burden of respiratory disease. “Fire smoke is becoming more of a problem. This swing back to respiratory has huge implications for healthcare systems and respiratory disease burden.”

Mr. Sacks and Dr. Carlsten reported no relevant financial relationships. The study presented by Dr. Tonne received funding from the European Union’s Horizon Europe research and innovation programme under Grant Agreement No. 101057131.
 

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

ORLANDO, FLORIDA — Pediatricians and other healthcare providers have a valuable role to play in screening parents for firearm ownership and offering counseling on safe storage practices, according to researchers who presented their findings at the American Academy of Pediatrics (AAP) 2024 National Conference.

An estimated 4.6 million US homes with children have firearms that are loaded and unlocked, a risk factor for youth suicide, yet only about half of parents of suicidal children had been screened for gun ownership in the hospital even as most would be receptive to both firearm screening and counseling, found one study in Texas.

In another study in Colorado, nearly all firearm owners believed that securely storing guns reduces the risk for firearm injury or death, but owners were less likely than non-owners to believe suicide is preventable or that removing a gun from the home reduces the risk for injury or death.

“Previous studies have shown that when pediatricians discuss the importance of armed safe storage guidance with families, families are actually more likely to go home and store firearms safely — storing them locked, unloaded, and separate from the ammunition,” said study author Taylor Rosenbaum, MD, a former pediatric fellow at Baylor College of Medicine/Texas Children’s Hospital in Houston and now an assistant professor at Children’s Hospital University of Miami. “However, previous studies have also shown that pediatricians really are not discussing firearm safe storage with our patients and their families, and we see this both in the outpatient setting, but especially in the inpatient setting for youth suicides, which have risen since 2020 and now are the second leading cause of death for those who are 10-24 years old in the United States.”

University of Miami

Firearm Safety Is a Necessary Conversation

The leading cause of death among children and teens aged 1-19 years is actually firearms, which are also the most fatal method for suicide. While only 4% of all suicide attempts in youth are fatal, 90% of those attempted with a firearm are fatal, Dr. Rosenbaum said. In addition, she said, 80% of the guns used in attempted suicide by children and teens belonged to a family member, and an estimated 70% of firearm-related suicides in youth can be prevented with safe storage of guns.

“This really gives us, as pediatricians, something actionable to do during these hospitalizations” for suicidal ideation or attempts, Dr. Rosenbaum said. “We know that when pediatricians discuss the importance of firearm safe storage guidance with families, they’re more likely to store their firearm safely,” Dr. Rosenbaum said. “We also know that families are not being screened for firearm ownership, that caregivers of youth who are in the hospital for suicidal thoughts or actions want their healthcare team to be screening for firearms, to be giving them information on how to safely secure their firearms, and to be providing free firearm blocks.”

Nathan Boonstra, MD, a general pediatrician at Blank Children’s Hospital, Des Moines, Iowa, said these findings are encouraging in terms of the opportunity pediatricians have.

“There is so much politicization around even basic firearm safety that pediatricians might shy away from the topic, but this research is reassuring that parents are receptive to our advice on safe gun storage,” said Dr. Boonstra, who was not involved in any of this presented research. “It’s especially important for pediatricians to address home firearms when their patient has a history of suicidal ideation or an attempt.”
 

Reducing the Risk

The Colorado findings similarly reinforce the opportunity physicians have to help caregivers reduce suicide risk, according to Maya Haasz, MD, an associate professor of pediatrics and emergency medicine at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

“Only 60% of firearm owners believed that removing firearms from the home in times of mental health crisis can decrease the risk of suicide,” she said. “These findings are really concerning, but what we found on the flip side was that 93% of firearm owners actually believe that secure storage can overall decrease the risk for firearm injury and death. So overall, we are underestimating the risk for suicide in our community, and we’re also underestimating our ability to prevent it.”

University of Colorado

Survey Results

Dr. Rosenbaum’s team conducted the survey in Houston with caregivers whose children were 8-21 years old and hospitalized for suicidal ideation or attempts at a large children’s hospital and two nearby community hospitals between June 2023 and May 2024. The respondents were 46% White and 23% Black, and 47% of the population were Hispanic, all but three of whom were not gun owners.

Among 244 potential participants, only 150 were eligible and approached, and 100 of these completed the surveys, including 26% firearm owners and 68% non-owners. Most of the youth (74%) were aged 14-17 years, and about three in four respondents were their mothers. Only half of the respondents (51%) said the healthcare provider had asked them whether they owned a gun.

One of the key findings Dr. Rosenbaum highlighted was the receptiveness of firearm-owning caregivers to advice from healthcare providers about ownership. If the healthcare team advised parents not to have any guns in the home for the safety of their child with self-arm, 58% of the firearm owners would follow the advice and 27% would consider it, with none saying they would be offended by it.

Among the firearm owners, 81% said their guns were safely secured where they did not believe their child could access it, which meant one in five youth had unsecured access to firearms. Most of the gun owners (77%), like the non-owners (70%), were “not at all worried” about their child getting ahold of a gun in the home, though 11.5% of the firearm owners were “very worried” about it. Interestingly, more gun owners (19%) were very worried about their children accessing a gun outside their home, a concern shared by 37% of non-owners. Nearly twice as many gun owners (46%) as non-owners (25%) were not at all worried about their child getting a gun outside the home.

The vast majority of respondents — 88% of gun owners and 91% of non-owners — felt it was “very important for the healthcare team to ask parents of children with suicidal ideation/attempts about firearms in the home.” Similarly, high proportions believed it was important for the healthcare team to counsel those parents on safe gun storage. Although only 69% of firearm owners believed it was important to distribute firearm locks in the hospital, 81% would be interested in receiving a free one. Significantly more of the non-owners (80%; P = .02) believed free lock distribution was important, and 72% of non-owners would also be interested in one.

About half the respondents (55%) preferred to hear firearm counseling one-on-one from a provider, whereas 31% would like written information and 27% would be interested in a video. In terms of what information parents preferred to receive, a little over half of owners (54%) and non-owners (56%) were interested in how or when (50% and 40%, respectively) to discuss the topic with their child. Only about a third (35% owners and 37% non-owners) wanted information on how to discuss the topic with the parents of their child’s friends.

The survey’s biggest limitations after its small size were the selection bias of those willing to complete the survey and potential response bias from the self-reported data.

The study of Colorado caregivers, just published in Pediatrics, surveyed 512 Colorado caregivers in April-May 2023 to learn about their beliefs and perceptions regarding firearms, firearm storage and risk, and youth suicide (2024 Oct 1;154[4]:e2024066930. doi: 10.1542/peds.2024-066930). Just over half the respondents (52%) had grown up in a household with firearms, and 44% currently lived in a household with a gun. The sample was 43% men and 88% White, predominantly non-Hispanic (75%), with 11% living in rural areas and 19% who currently or previously served in the military. Most (79%) had a child age 12 or younger in the home.

Only about one in four caregivers (24%) correctly answered that suicide is the leading cause of firearm death in Colorado, with similar rates of correct responses among both firearm owners and non-firearm owners. Both groups were also similarly likely (64% overall) to be concerned about youth suicide in their community, though those from homes with firearms were less likely to be concerned about youth suicide in their own family (28%) than those from homes without firearms (39%; P = .013).

In addition, caregivers from homes with versus without firearms were considerably less likely to believe suicide can be prevented (48% vs 69%) and were less likely to believe that temporarily removing a firearm from the home reduces the risk for gun injury or death (60% vs 78%; P < .001 for both comparisons).

Firearm owners were also much less likely than non-owners to believe keeping a gun in the home makes it more dangerous (7% vs 29%) and over twice as likely to think keeping a firearm makes their home safer (52% vs 22%; P < .001). The vast majority of respondents (89%) believed secure storage of guns reduces the risk for injury or death, though the response was higher for firearm owners (93%) than for non-owners (86%; P < .001).

“Our finding that most firearm owners believe that secure firearm storage is protective against firearm injury is a promising messaging strategy,” the authors wrote. “It presents a preventive education opportunity for adults living with children who have mental health concerns, who may benefit most from secure in-home storage and/or temporary and voluntary storage of firearms away from home.”
 

Firearm Injuries

A separate study at the AAP conference underscored the devastating impact of firearm injuries even among those who survive, whether self-inflicted or not, and the potential for reducing healthcare treatment and costs from effective prevention efforts. A national analysis of pediatric inpatient data from 2017 to 2020 calculated how much greater the burden of healthcare treatment and costs is for firearm injuries of any kind compared with penetrating traumas and blunt traumas.

“As a surgical resident, I have seen these patients who make it into the trauma bed that we are then faced to care for,” said Colleen Nofi, DO, PhD, MBA, a general surgery resident at Cohen Children’s Medical Center at Northwell Health in New York. “Anecdotally, we understand that the devastation and injury caused by bullets far outweighs the injuries caused by other trauma mechanisms,” but the actual calculation of the burden hasn’t been studied.

Northwell Health

A version of this article appeared on Medscape.com.

ORLANDO, FLORIDA — Pediatricians and other healthcare providers have a valuable role to play in screening parents for firearm ownership and offering counseling on safe storage practices, according to researchers who presented their findings at the American Academy of Pediatrics (AAP) 2024 National Conference.

An estimated 4.6 million US homes with children have firearms that are loaded and unlocked, a risk factor for youth suicide, yet only about half of parents of suicidal children had been screened for gun ownership in the hospital even as most would be receptive to both firearm screening and counseling, found one study in Texas.

In another study in Colorado, nearly all firearm owners believed that securely storing guns reduces the risk for firearm injury or death, but owners were less likely than non-owners to believe suicide is preventable or that removing a gun from the home reduces the risk for injury or death.

“Previous studies have shown that when pediatricians discuss the importance of armed safe storage guidance with families, families are actually more likely to go home and store firearms safely — storing them locked, unloaded, and separate from the ammunition,” said study author Taylor Rosenbaum, MD, a former pediatric fellow at Baylor College of Medicine/Texas Children’s Hospital in Houston and now an assistant professor at Children’s Hospital University of Miami. “However, previous studies have also shown that pediatricians really are not discussing firearm safe storage with our patients and their families, and we see this both in the outpatient setting, but especially in the inpatient setting for youth suicides, which have risen since 2020 and now are the second leading cause of death for those who are 10-24 years old in the United States.”

University of Miami

Firearm Safety Is a Necessary Conversation

The leading cause of death among children and teens aged 1-19 years is actually firearms, which are also the most fatal method for suicide. While only 4% of all suicide attempts in youth are fatal, 90% of those attempted with a firearm are fatal, Dr. Rosenbaum said. In addition, she said, 80% of the guns used in attempted suicide by children and teens belonged to a family member, and an estimated 70% of firearm-related suicides in youth can be prevented with safe storage of guns.

“This really gives us, as pediatricians, something actionable to do during these hospitalizations” for suicidal ideation or attempts, Dr. Rosenbaum said. “We know that when pediatricians discuss the importance of firearm safe storage guidance with families, they’re more likely to store their firearm safely,” Dr. Rosenbaum said. “We also know that families are not being screened for firearm ownership, that caregivers of youth who are in the hospital for suicidal thoughts or actions want their healthcare team to be screening for firearms, to be giving them information on how to safely secure their firearms, and to be providing free firearm blocks.”

Nathan Boonstra, MD, a general pediatrician at Blank Children’s Hospital, Des Moines, Iowa, said these findings are encouraging in terms of the opportunity pediatricians have.

“There is so much politicization around even basic firearm safety that pediatricians might shy away from the topic, but this research is reassuring that parents are receptive to our advice on safe gun storage,” said Dr. Boonstra, who was not involved in any of this presented research. “It’s especially important for pediatricians to address home firearms when their patient has a history of suicidal ideation or an attempt.”
 

Reducing the Risk

The Colorado findings similarly reinforce the opportunity physicians have to help caregivers reduce suicide risk, according to Maya Haasz, MD, an associate professor of pediatrics and emergency medicine at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

“Only 60% of firearm owners believed that removing firearms from the home in times of mental health crisis can decrease the risk of suicide,” she said. “These findings are really concerning, but what we found on the flip side was that 93% of firearm owners actually believe that secure storage can overall decrease the risk for firearm injury and death. So overall, we are underestimating the risk for suicide in our community, and we’re also underestimating our ability to prevent it.”

University of Colorado

Survey Results

Dr. Rosenbaum’s team conducted the survey in Houston with caregivers whose children were 8-21 years old and hospitalized for suicidal ideation or attempts at a large children’s hospital and two nearby community hospitals between June 2023 and May 2024. The respondents were 46% White and 23% Black, and 47% of the population were Hispanic, all but three of whom were not gun owners.

Among 244 potential participants, only 150 were eligible and approached, and 100 of these completed the surveys, including 26% firearm owners and 68% non-owners. Most of the youth (74%) were aged 14-17 years, and about three in four respondents were their mothers. Only half of the respondents (51%) said the healthcare provider had asked them whether they owned a gun.

One of the key findings Dr. Rosenbaum highlighted was the receptiveness of firearm-owning caregivers to advice from healthcare providers about ownership. If the healthcare team advised parents not to have any guns in the home for the safety of their child with self-arm, 58% of the firearm owners would follow the advice and 27% would consider it, with none saying they would be offended by it.

Among the firearm owners, 81% said their guns were safely secured where they did not believe their child could access it, which meant one in five youth had unsecured access to firearms. Most of the gun owners (77%), like the non-owners (70%), were “not at all worried” about their child getting ahold of a gun in the home, though 11.5% of the firearm owners were “very worried” about it. Interestingly, more gun owners (19%) were very worried about their children accessing a gun outside their home, a concern shared by 37% of non-owners. Nearly twice as many gun owners (46%) as non-owners (25%) were not at all worried about their child getting a gun outside the home.

The vast majority of respondents — 88% of gun owners and 91% of non-owners — felt it was “very important for the healthcare team to ask parents of children with suicidal ideation/attempts about firearms in the home.” Similarly, high proportions believed it was important for the healthcare team to counsel those parents on safe gun storage. Although only 69% of firearm owners believed it was important to distribute firearm locks in the hospital, 81% would be interested in receiving a free one. Significantly more of the non-owners (80%; P = .02) believed free lock distribution was important, and 72% of non-owners would also be interested in one.

About half the respondents (55%) preferred to hear firearm counseling one-on-one from a provider, whereas 31% would like written information and 27% would be interested in a video. In terms of what information parents preferred to receive, a little over half of owners (54%) and non-owners (56%) were interested in how or when (50% and 40%, respectively) to discuss the topic with their child. Only about a third (35% owners and 37% non-owners) wanted information on how to discuss the topic with the parents of their child’s friends.

The survey’s biggest limitations after its small size were the selection bias of those willing to complete the survey and potential response bias from the self-reported data.

The study of Colorado caregivers, just published in Pediatrics, surveyed 512 Colorado caregivers in April-May 2023 to learn about their beliefs and perceptions regarding firearms, firearm storage and risk, and youth suicide (2024 Oct 1;154[4]:e2024066930. doi: 10.1542/peds.2024-066930). Just over half the respondents (52%) had grown up in a household with firearms, and 44% currently lived in a household with a gun. The sample was 43% men and 88% White, predominantly non-Hispanic (75%), with 11% living in rural areas and 19% who currently or previously served in the military. Most (79%) had a child age 12 or younger in the home.

Only about one in four caregivers (24%) correctly answered that suicide is the leading cause of firearm death in Colorado, with similar rates of correct responses among both firearm owners and non-firearm owners. Both groups were also similarly likely (64% overall) to be concerned about youth suicide in their community, though those from homes with firearms were less likely to be concerned about youth suicide in their own family (28%) than those from homes without firearms (39%; P = .013).

In addition, caregivers from homes with versus without firearms were considerably less likely to believe suicide can be prevented (48% vs 69%) and were less likely to believe that temporarily removing a firearm from the home reduces the risk for gun injury or death (60% vs 78%; P < .001 for both comparisons).

Firearm owners were also much less likely than non-owners to believe keeping a gun in the home makes it more dangerous (7% vs 29%) and over twice as likely to think keeping a firearm makes their home safer (52% vs 22%; P < .001). The vast majority of respondents (89%) believed secure storage of guns reduces the risk for injury or death, though the response was higher for firearm owners (93%) than for non-owners (86%; P < .001).

“Our finding that most firearm owners believe that secure firearm storage is protective against firearm injury is a promising messaging strategy,” the authors wrote. “It presents a preventive education opportunity for adults living with children who have mental health concerns, who may benefit most from secure in-home storage and/or temporary and voluntary storage of firearms away from home.”
 

Firearm Injuries

A separate study at the AAP conference underscored the devastating impact of firearm injuries even among those who survive, whether self-inflicted or not, and the potential for reducing healthcare treatment and costs from effective prevention efforts. A national analysis of pediatric inpatient data from 2017 to 2020 calculated how much greater the burden of healthcare treatment and costs is for firearm injuries of any kind compared with penetrating traumas and blunt traumas.

“As a surgical resident, I have seen these patients who make it into the trauma bed that we are then faced to care for,” said Colleen Nofi, DO, PhD, MBA, a general surgery resident at Cohen Children’s Medical Center at Northwell Health in New York. “Anecdotally, we understand that the devastation and injury caused by bullets far outweighs the injuries caused by other trauma mechanisms,” but the actual calculation of the burden hasn’t been studied.

Northwell Health

A version of this article appeared on Medscape.com.

ORLANDO, FLORIDA — Pediatricians and other healthcare providers have a valuable role to play in screening parents for firearm ownership and offering counseling on safe storage practices, according to researchers who presented their findings at the American Academy of Pediatrics (AAP) 2024 National Conference.

An estimated 4.6 million US homes with children have firearms that are loaded and unlocked, a risk factor for youth suicide, yet only about half of parents of suicidal children had been screened for gun ownership in the hospital even as most would be receptive to both firearm screening and counseling, found one study in Texas.

In another study in Colorado, nearly all firearm owners believed that securely storing guns reduces the risk for firearm injury or death, but owners were less likely than non-owners to believe suicide is preventable or that removing a gun from the home reduces the risk for injury or death.

“Previous studies have shown that when pediatricians discuss the importance of armed safe storage guidance with families, families are actually more likely to go home and store firearms safely — storing them locked, unloaded, and separate from the ammunition,” said study author Taylor Rosenbaum, MD, a former pediatric fellow at Baylor College of Medicine/Texas Children’s Hospital in Houston and now an assistant professor at Children’s Hospital University of Miami. “However, previous studies have also shown that pediatricians really are not discussing firearm safe storage with our patients and their families, and we see this both in the outpatient setting, but especially in the inpatient setting for youth suicides, which have risen since 2020 and now are the second leading cause of death for those who are 10-24 years old in the United States.”

University of Miami

Firearm Safety Is a Necessary Conversation

The leading cause of death among children and teens aged 1-19 years is actually firearms, which are also the most fatal method for suicide. While only 4% of all suicide attempts in youth are fatal, 90% of those attempted with a firearm are fatal, Dr. Rosenbaum said. In addition, she said, 80% of the guns used in attempted suicide by children and teens belonged to a family member, and an estimated 70% of firearm-related suicides in youth can be prevented with safe storage of guns.

“This really gives us, as pediatricians, something actionable to do during these hospitalizations” for suicidal ideation or attempts, Dr. Rosenbaum said. “We know that when pediatricians discuss the importance of firearm safe storage guidance with families, they’re more likely to store their firearm safely,” Dr. Rosenbaum said. “We also know that families are not being screened for firearm ownership, that caregivers of youth who are in the hospital for suicidal thoughts or actions want their healthcare team to be screening for firearms, to be giving them information on how to safely secure their firearms, and to be providing free firearm blocks.”

Nathan Boonstra, MD, a general pediatrician at Blank Children’s Hospital, Des Moines, Iowa, said these findings are encouraging in terms of the opportunity pediatricians have.

“There is so much politicization around even basic firearm safety that pediatricians might shy away from the topic, but this research is reassuring that parents are receptive to our advice on safe gun storage,” said Dr. Boonstra, who was not involved in any of this presented research. “It’s especially important for pediatricians to address home firearms when their patient has a history of suicidal ideation or an attempt.”
 

Reducing the Risk

The Colorado findings similarly reinforce the opportunity physicians have to help caregivers reduce suicide risk, according to Maya Haasz, MD, an associate professor of pediatrics and emergency medicine at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

“Only 60% of firearm owners believed that removing firearms from the home in times of mental health crisis can decrease the risk of suicide,” she said. “These findings are really concerning, but what we found on the flip side was that 93% of firearm owners actually believe that secure storage can overall decrease the risk for firearm injury and death. So overall, we are underestimating the risk for suicide in our community, and we’re also underestimating our ability to prevent it.”

University of Colorado

Survey Results

Dr. Rosenbaum’s team conducted the survey in Houston with caregivers whose children were 8-21 years old and hospitalized for suicidal ideation or attempts at a large children’s hospital and two nearby community hospitals between June 2023 and May 2024. The respondents were 46% White and 23% Black, and 47% of the population were Hispanic, all but three of whom were not gun owners.

Among 244 potential participants, only 150 were eligible and approached, and 100 of these completed the surveys, including 26% firearm owners and 68% non-owners. Most of the youth (74%) were aged 14-17 years, and about three in four respondents were their mothers. Only half of the respondents (51%) said the healthcare provider had asked them whether they owned a gun.

One of the key findings Dr. Rosenbaum highlighted was the receptiveness of firearm-owning caregivers to advice from healthcare providers about ownership. If the healthcare team advised parents not to have any guns in the home for the safety of their child with self-arm, 58% of the firearm owners would follow the advice and 27% would consider it, with none saying they would be offended by it.

Among the firearm owners, 81% said their guns were safely secured where they did not believe their child could access it, which meant one in five youth had unsecured access to firearms. Most of the gun owners (77%), like the non-owners (70%), were “not at all worried” about their child getting ahold of a gun in the home, though 11.5% of the firearm owners were “very worried” about it. Interestingly, more gun owners (19%) were very worried about their children accessing a gun outside their home, a concern shared by 37% of non-owners. Nearly twice as many gun owners (46%) as non-owners (25%) were not at all worried about their child getting a gun outside the home.

The vast majority of respondents — 88% of gun owners and 91% of non-owners — felt it was “very important for the healthcare team to ask parents of children with suicidal ideation/attempts about firearms in the home.” Similarly, high proportions believed it was important for the healthcare team to counsel those parents on safe gun storage. Although only 69% of firearm owners believed it was important to distribute firearm locks in the hospital, 81% would be interested in receiving a free one. Significantly more of the non-owners (80%; P = .02) believed free lock distribution was important, and 72% of non-owners would also be interested in one.

About half the respondents (55%) preferred to hear firearm counseling one-on-one from a provider, whereas 31% would like written information and 27% would be interested in a video. In terms of what information parents preferred to receive, a little over half of owners (54%) and non-owners (56%) were interested in how or when (50% and 40%, respectively) to discuss the topic with their child. Only about a third (35% owners and 37% non-owners) wanted information on how to discuss the topic with the parents of their child’s friends.

The survey’s biggest limitations after its small size were the selection bias of those willing to complete the survey and potential response bias from the self-reported data.

The study of Colorado caregivers, just published in Pediatrics, surveyed 512 Colorado caregivers in April-May 2023 to learn about their beliefs and perceptions regarding firearms, firearm storage and risk, and youth suicide (2024 Oct 1;154[4]:e2024066930. doi: 10.1542/peds.2024-066930). Just over half the respondents (52%) had grown up in a household with firearms, and 44% currently lived in a household with a gun. The sample was 43% men and 88% White, predominantly non-Hispanic (75%), with 11% living in rural areas and 19% who currently or previously served in the military. Most (79%) had a child age 12 or younger in the home.

Only about one in four caregivers (24%) correctly answered that suicide is the leading cause of firearm death in Colorado, with similar rates of correct responses among both firearm owners and non-firearm owners. Both groups were also similarly likely (64% overall) to be concerned about youth suicide in their community, though those from homes with firearms were less likely to be concerned about youth suicide in their own family (28%) than those from homes without firearms (39%; P = .013).

In addition, caregivers from homes with versus without firearms were considerably less likely to believe suicide can be prevented (48% vs 69%) and were less likely to believe that temporarily removing a firearm from the home reduces the risk for gun injury or death (60% vs 78%; P < .001 for both comparisons).

Firearm owners were also much less likely than non-owners to believe keeping a gun in the home makes it more dangerous (7% vs 29%) and over twice as likely to think keeping a firearm makes their home safer (52% vs 22%; P < .001). The vast majority of respondents (89%) believed secure storage of guns reduces the risk for injury or death, though the response was higher for firearm owners (93%) than for non-owners (86%; P < .001).

“Our finding that most firearm owners believe that secure firearm storage is protective against firearm injury is a promising messaging strategy,” the authors wrote. “It presents a preventive education opportunity for adults living with children who have mental health concerns, who may benefit most from secure in-home storage and/or temporary and voluntary storage of firearms away from home.”
 

Firearm Injuries

A separate study at the AAP conference underscored the devastating impact of firearm injuries even among those who survive, whether self-inflicted or not, and the potential for reducing healthcare treatment and costs from effective prevention efforts. A national analysis of pediatric inpatient data from 2017 to 2020 calculated how much greater the burden of healthcare treatment and costs is for firearm injuries of any kind compared with penetrating traumas and blunt traumas.

“As a surgical resident, I have seen these patients who make it into the trauma bed that we are then faced to care for,” said Colleen Nofi, DO, PhD, MBA, a general surgery resident at Cohen Children’s Medical Center at Northwell Health in New York. “Anecdotally, we understand that the devastation and injury caused by bullets far outweighs the injuries caused by other trauma mechanisms,” but the actual calculation of the burden hasn’t been studied.

Northwell Health

A version of this article appeared on Medscape.com.

An international study, Colive Voice, presented at the European Association for the Study of Diabetes (EASD) 2024 conference, shows that patients with type 2 diabetes (T2D) have different voice characteristics compared with healthy controls of the same age and gender. These results “open up possibilities for developing a first-line, noninvasive, and rapid screening tool for T2D, feasible with just a few seconds of voice recording on a smartphone or during consultations,” explained the study’s principal investigator Guy Fagherazzi, PhD, a diabetes epidemiologist at the Luxembourg Institute of Health, in an interview with this news organization.

How did the idea of detecting diabetes through voice come about?

During the COVID-19 pandemic, we began analyzing voice recordings from patients with chronic diseases. We wanted to find solutions to assess people’s health remotely, without physical contact. We quickly realized that this approach could be extended to other diseases. Because my main research focus has always been diabetes, I looked into how voice characteristics might correlate with diabetes. Previous studies had indicated that patients with diabetes have distinct voices compared with the general population, and this insight formed the starting point.

What mechanism could explain why patients with T2D have different voice characteristics?

It’s challenging to pinpoint a single factor that would explain why patients with T2D have different voices from those without diabetes. Several factors are involved.

Some biological mechanisms, especially those affecting the vascular system, influence symptoms in people with metabolic diseases such as diabetes. For example, people with T2D have more frequent cardiorespiratory fatigue. Obesity and overweight are also key factors, as these conditions can slightly alter vocal parameters compared with people of normal weight. Hypertension, common in patients with T2D, adds to the complexity.

Neurologic complications can affect the nerves and muscles involved in voice production, particularly the vocal cords.

Therefore, respiratory fatigue, neuropathies, and other conditions such as dehydration and gastric acid reflux, which are more common in patients with diabetes, can contribute to differences in voice.

These differences might not be noticeable to the human ear. That’s why we often don’t notice the link between voice and diabetes. However, technological advancements in signal processing and artificial intelligence allow us to extract a large amount of information from these subtle variations. By analyzing these small differences, we can detect diabetes with a reasonable degree of accuracy.
 

In your study, you mention that voice tone can indicate diabetic status. Could you elaborate?

Yes, voice tone can be affected, though it’s a complex, multidimensional phenomenon.

Patients who have had diabetes for 5-10 years, or longer, tend to have a rougher voice than those without diabetes of the same age and gender. In our study, we were able to extract many voice characteristics from the raw audio signal, which is why it’s difficult to isolate one specific factor that stands out.
 

Is there a difference in voice changes between patients with well-managed diabetes and those whose disease is uncontrolled?

The roughness of the voice tends to increase with the duration of diabetes. It’s more noticeable in people with poorly controlled diabetes. Our hypothesis, based on the results we presented at the EASD conference, is that fluctuations in blood sugar levels, both hypo- and hyperglycemia, may cause short-term changes in the voice. There are also many subtle, rapid changes that could potentially be detected, though we haven’t confirmed this yet. We’re currently conducting additional studies to explore this.

Why did you ask participants to read a passage from the  Universal Declaration of Human Rights?

We used a highly standardized approach. Participants completed several recordings, including holding the sound “Aaaaaa” for as long as possible in one breath. They also read a passage, which helps us better distinguish between patients with and those without diabetes. This method works slightly better than other sounds typically used for analyzing diseases. We chose this particular text in the participant’s native language because it’s neutral and doesn’t trigger emotional fluctuations. Because Colive Voice is an international, multilingual study, we use official translations in various languages.

Your research focuses on T2D. Do you plan to study type 1 diabetes (T1D) as well?

We believe that individuals with T1D also exhibit voice changes over time. However, our current focus is on T2D because our goal is to develop large-scale screening methods. T1D, typically diagnosed in childhood, requires different screening approaches. For now, our research mainly involves adults.

Were there any gender differences in the accuracy of your voice analysis?

Yes, voice studies generally show that women have different vocal signatures from men, partly owing to hormonal fluctuations that affect pitch and tone. Detecting differences between healthy individuals and those with diabetes can sometimes be more challenging in women, depending on the condition. In our study, we achieved about 70% accuracy for women compared with 75% for men.

The EASD results focused on a US-based population. When can we expect data from France?

We started with the US because we could quickly gather a large number of patients. Now, we’re expanding to global and language-specific analyses. French data are certainly a priority, and we’re working on it. We encourage people to participate — it takes only 20 minutes and contributes to innovative research on noninvasive diabetes detection. Participants can sign up at www.colivevoice.org

Dr. Fagherazzi heads the Deep Digital Phenotyping laboratory and the Department of Precision Health at the Luxembourg Institute of Health. His research focuses on integrating new technologies and digital data into diabetes research. He has declared no relevant financial relationships. 

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

An international study, Colive Voice, presented at the European Association for the Study of Diabetes (EASD) 2024 conference, shows that patients with type 2 diabetes (T2D) have different voice characteristics compared with healthy controls of the same age and gender. These results “open up possibilities for developing a first-line, noninvasive, and rapid screening tool for T2D, feasible with just a few seconds of voice recording on a smartphone or during consultations,” explained the study’s principal investigator Guy Fagherazzi, PhD, a diabetes epidemiologist at the Luxembourg Institute of Health, in an interview with this news organization.

How did the idea of detecting diabetes through voice come about?

During the COVID-19 pandemic, we began analyzing voice recordings from patients with chronic diseases. We wanted to find solutions to assess people’s health remotely, without physical contact. We quickly realized that this approach could be extended to other diseases. Because my main research focus has always been diabetes, I looked into how voice characteristics might correlate with diabetes. Previous studies had indicated that patients with diabetes have distinct voices compared with the general population, and this insight formed the starting point.

What mechanism could explain why patients with T2D have different voice characteristics?

It’s challenging to pinpoint a single factor that would explain why patients with T2D have different voices from those without diabetes. Several factors are involved.

Some biological mechanisms, especially those affecting the vascular system, influence symptoms in people with metabolic diseases such as diabetes. For example, people with T2D have more frequent cardiorespiratory fatigue. Obesity and overweight are also key factors, as these conditions can slightly alter vocal parameters compared with people of normal weight. Hypertension, common in patients with T2D, adds to the complexity.

Neurologic complications can affect the nerves and muscles involved in voice production, particularly the vocal cords.

Therefore, respiratory fatigue, neuropathies, and other conditions such as dehydration and gastric acid reflux, which are more common in patients with diabetes, can contribute to differences in voice.

These differences might not be noticeable to the human ear. That’s why we often don’t notice the link between voice and diabetes. However, technological advancements in signal processing and artificial intelligence allow us to extract a large amount of information from these subtle variations. By analyzing these small differences, we can detect diabetes with a reasonable degree of accuracy.
 

In your study, you mention that voice tone can indicate diabetic status. Could you elaborate?

Yes, voice tone can be affected, though it’s a complex, multidimensional phenomenon.

Patients who have had diabetes for 5-10 years, or longer, tend to have a rougher voice than those without diabetes of the same age and gender. In our study, we were able to extract many voice characteristics from the raw audio signal, which is why it’s difficult to isolate one specific factor that stands out.
 

Is there a difference in voice changes between patients with well-managed diabetes and those whose disease is uncontrolled?

The roughness of the voice tends to increase with the duration of diabetes. It’s more noticeable in people with poorly controlled diabetes. Our hypothesis, based on the results we presented at the EASD conference, is that fluctuations in blood sugar levels, both hypo- and hyperglycemia, may cause short-term changes in the voice. There are also many subtle, rapid changes that could potentially be detected, though we haven’t confirmed this yet. We’re currently conducting additional studies to explore this.

Why did you ask participants to read a passage from the  Universal Declaration of Human Rights?

We used a highly standardized approach. Participants completed several recordings, including holding the sound “Aaaaaa” for as long as possible in one breath. They also read a passage, which helps us better distinguish between patients with and those without diabetes. This method works slightly better than other sounds typically used for analyzing diseases. We chose this particular text in the participant’s native language because it’s neutral and doesn’t trigger emotional fluctuations. Because Colive Voice is an international, multilingual study, we use official translations in various languages.

Your research focuses on T2D. Do you plan to study type 1 diabetes (T1D) as well?

We believe that individuals with T1D also exhibit voice changes over time. However, our current focus is on T2D because our goal is to develop large-scale screening methods. T1D, typically diagnosed in childhood, requires different screening approaches. For now, our research mainly involves adults.

Were there any gender differences in the accuracy of your voice analysis?

Yes, voice studies generally show that women have different vocal signatures from men, partly owing to hormonal fluctuations that affect pitch and tone. Detecting differences between healthy individuals and those with diabetes can sometimes be more challenging in women, depending on the condition. In our study, we achieved about 70% accuracy for women compared with 75% for men.

The EASD results focused on a US-based population. When can we expect data from France?

We started with the US because we could quickly gather a large number of patients. Now, we’re expanding to global and language-specific analyses. French data are certainly a priority, and we’re working on it. We encourage people to participate — it takes only 20 minutes and contributes to innovative research on noninvasive diabetes detection. Participants can sign up at www.colivevoice.org

Dr. Fagherazzi heads the Deep Digital Phenotyping laboratory and the Department of Precision Health at the Luxembourg Institute of Health. His research focuses on integrating new technologies and digital data into diabetes research. He has declared no relevant financial relationships. 

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

An international study, Colive Voice, presented at the European Association for the Study of Diabetes (EASD) 2024 conference, shows that patients with type 2 diabetes (T2D) have different voice characteristics compared with healthy controls of the same age and gender. These results “open up possibilities for developing a first-line, noninvasive, and rapid screening tool for T2D, feasible with just a few seconds of voice recording on a smartphone or during consultations,” explained the study’s principal investigator Guy Fagherazzi, PhD, a diabetes epidemiologist at the Luxembourg Institute of Health, in an interview with this news organization.

How did the idea of detecting diabetes through voice come about?

During the COVID-19 pandemic, we began analyzing voice recordings from patients with chronic diseases. We wanted to find solutions to assess people’s health remotely, without physical contact. We quickly realized that this approach could be extended to other diseases. Because my main research focus has always been diabetes, I looked into how voice characteristics might correlate with diabetes. Previous studies had indicated that patients with diabetes have distinct voices compared with the general population, and this insight formed the starting point.

What mechanism could explain why patients with T2D have different voice characteristics?

It’s challenging to pinpoint a single factor that would explain why patients with T2D have different voices from those without diabetes. Several factors are involved.

Some biological mechanisms, especially those affecting the vascular system, influence symptoms in people with metabolic diseases such as diabetes. For example, people with T2D have more frequent cardiorespiratory fatigue. Obesity and overweight are also key factors, as these conditions can slightly alter vocal parameters compared with people of normal weight. Hypertension, common in patients with T2D, adds to the complexity.

Neurologic complications can affect the nerves and muscles involved in voice production, particularly the vocal cords.

Therefore, respiratory fatigue, neuropathies, and other conditions such as dehydration and gastric acid reflux, which are more common in patients with diabetes, can contribute to differences in voice.

These differences might not be noticeable to the human ear. That’s why we often don’t notice the link between voice and diabetes. However, technological advancements in signal processing and artificial intelligence allow us to extract a large amount of information from these subtle variations. By analyzing these small differences, we can detect diabetes with a reasonable degree of accuracy.
 

In your study, you mention that voice tone can indicate diabetic status. Could you elaborate?

Yes, voice tone can be affected, though it’s a complex, multidimensional phenomenon.

Patients who have had diabetes for 5-10 years, or longer, tend to have a rougher voice than those without diabetes of the same age and gender. In our study, we were able to extract many voice characteristics from the raw audio signal, which is why it’s difficult to isolate one specific factor that stands out.
 

Is there a difference in voice changes between patients with well-managed diabetes and those whose disease is uncontrolled?

The roughness of the voice tends to increase with the duration of diabetes. It’s more noticeable in people with poorly controlled diabetes. Our hypothesis, based on the results we presented at the EASD conference, is that fluctuations in blood sugar levels, both hypo- and hyperglycemia, may cause short-term changes in the voice. There are also many subtle, rapid changes that could potentially be detected, though we haven’t confirmed this yet. We’re currently conducting additional studies to explore this.

Why did you ask participants to read a passage from the  Universal Declaration of Human Rights?

We used a highly standardized approach. Participants completed several recordings, including holding the sound “Aaaaaa” for as long as possible in one breath. They also read a passage, which helps us better distinguish between patients with and those without diabetes. This method works slightly better than other sounds typically used for analyzing diseases. We chose this particular text in the participant’s native language because it’s neutral and doesn’t trigger emotional fluctuations. Because Colive Voice is an international, multilingual study, we use official translations in various languages.

Your research focuses on T2D. Do you plan to study type 1 diabetes (T1D) as well?

We believe that individuals with T1D also exhibit voice changes over time. However, our current focus is on T2D because our goal is to develop large-scale screening methods. T1D, typically diagnosed in childhood, requires different screening approaches. For now, our research mainly involves adults.

Were there any gender differences in the accuracy of your voice analysis?

Yes, voice studies generally show that women have different vocal signatures from men, partly owing to hormonal fluctuations that affect pitch and tone. Detecting differences between healthy individuals and those with diabetes can sometimes be more challenging in women, depending on the condition. In our study, we achieved about 70% accuracy for women compared with 75% for men.

The EASD results focused on a US-based population. When can we expect data from France?

We started with the US because we could quickly gather a large number of patients. Now, we’re expanding to global and language-specific analyses. French data are certainly a priority, and we’re working on it. We encourage people to participate — it takes only 20 minutes and contributes to innovative research on noninvasive diabetes detection. Participants can sign up at www.colivevoice.org

Dr. Fagherazzi heads the Deep Digital Phenotyping laboratory and the Department of Precision Health at the Luxembourg Institute of Health. His research focuses on integrating new technologies and digital data into diabetes research. He has declared no relevant financial relationships. 

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

The US Department of Veterans Affairs (VA) now has a permanent pharmacogenomics service that provides genetic tests to give clinicians insight into the best medication options for their patients.

The tests, which have no extra cost, are available to all veterans, said pharmacist Jill S. Bates, PharmD, MS, executive director of the VA National Pharmacogenomics Program, who spoke in an interview and a presentation at the annual meeting of the Association of VA Hematology/Oncology.

Genetic testing is “a tool that can help optimize care that we provide for veterans,” she said. “Pharmacogenomics is additional information to help the clinician make a decision. We know that most veterans—greater than 90%—carry a variant in a pharmacogenomics gene that is actionable.”

The genetic tests can provide insight into the optimal medication for multiple conditions such as mental illness, gastrointestinal disorders, cancer, pain, and heart disease. According to a 2019 analysis of over 6 years of data, more than half of the VA patient population used medications whose efficacy may have been affected by detectable genetic variants.

For instance, Bates said tests can let clinicians know whether patients are susceptible to statin-associated muscle adverse effects if they take simvastatin, the cholesterol medication. An estimated 25.6% of the VA population has this variant.

Elsewhere on the cardiac front, an estimated 58.3% of the VA population has a genetic variant that increases sensitivity to the blood thinner warfarin.

Testing could help psychiatrists determine whether certain medications should not be prescribed—or should be prescribed at lower doses—in patients who’ve had adverse reactions to antidepressants, Bates said.

In cancer, Bates said, genetic testing can identify patients who have a genetic variant that boosts toxicity from fluoropyrimidine chemotherapy treatments, which include capecitabine, floxuridine, and fluorouracil. Meanwhile, an estimated 0.9% will have no reaction or limited reaction to capecitabine and fluorouracil, and 4.8% will have hypersensitivity to carbamazepine and oxcarbazepine. 

Tests can also identify a genetic variant that can lead to poor metabolism of the chemotherapy drug irinotecan, which is used to treat colon cancer. “In those patients, you’d want to reduce the dose by 20%,” Bates said. In other cases, alternate drugs may be the best strategy to address genetic variations.

Prior to 2019, clinicians had to order pharmacogenomic tests outside of the VA system, according to Bates. That year, a donation from Sanford Health brought VA pharmacogenomics to 40 pilot sites. Since then, more than 88,000 tests have been performed.

The VA has now made its pharmacogenomic program permanent, Bates said. As of early September, testing was available at 139 VA sites and is coming soon to 4 more. It’s not available at another 23 sites that are scattered across the country.

A tool in the VA electronic health record now reminds clinicians about the availability of genetic testing and allows them to order tests. However, testing isn’t available for patients who have had liver transplants or certain bone marrow transplants.

The VA is working on developing decision-making tools to help clinicians determine when the tests are appropriate, Bates said. It typically takes 2 to 3 weeks to get results, she said, adding that external laboratories provide results. “We eventually would like to bring in all pharmacogenomics testing to be conducted within the VA enterprise.”

Bates reported that she had no disclosures.

The US Department of Veterans Affairs (VA) now has a permanent pharmacogenomics service that provides genetic tests to give clinicians insight into the best medication options for their patients.

The tests, which have no extra cost, are available to all veterans, said pharmacist Jill S. Bates, PharmD, MS, executive director of the VA National Pharmacogenomics Program, who spoke in an interview and a presentation at the annual meeting of the Association of VA Hematology/Oncology.

Genetic testing is “a tool that can help optimize care that we provide for veterans,” she said. “Pharmacogenomics is additional information to help the clinician make a decision. We know that most veterans—greater than 90%—carry a variant in a pharmacogenomics gene that is actionable.”

The genetic tests can provide insight into the optimal medication for multiple conditions such as mental illness, gastrointestinal disorders, cancer, pain, and heart disease. According to a 2019 analysis of over 6 years of data, more than half of the VA patient population used medications whose efficacy may have been affected by detectable genetic variants.

For instance, Bates said tests can let clinicians know whether patients are susceptible to statin-associated muscle adverse effects if they take simvastatin, the cholesterol medication. An estimated 25.6% of the VA population has this variant.

Elsewhere on the cardiac front, an estimated 58.3% of the VA population has a genetic variant that increases sensitivity to the blood thinner warfarin.

Testing could help psychiatrists determine whether certain medications should not be prescribed—or should be prescribed at lower doses—in patients who’ve had adverse reactions to antidepressants, Bates said.

In cancer, Bates said, genetic testing can identify patients who have a genetic variant that boosts toxicity from fluoropyrimidine chemotherapy treatments, which include capecitabine, floxuridine, and fluorouracil. Meanwhile, an estimated 0.9% will have no reaction or limited reaction to capecitabine and fluorouracil, and 4.8% will have hypersensitivity to carbamazepine and oxcarbazepine. 

Tests can also identify a genetic variant that can lead to poor metabolism of the chemotherapy drug irinotecan, which is used to treat colon cancer. “In those patients, you’d want to reduce the dose by 20%,” Bates said. In other cases, alternate drugs may be the best strategy to address genetic variations.

Prior to 2019, clinicians had to order pharmacogenomic tests outside of the VA system, according to Bates. That year, a donation from Sanford Health brought VA pharmacogenomics to 40 pilot sites. Since then, more than 88,000 tests have been performed.

The VA has now made its pharmacogenomic program permanent, Bates said. As of early September, testing was available at 139 VA sites and is coming soon to 4 more. It’s not available at another 23 sites that are scattered across the country.

A tool in the VA electronic health record now reminds clinicians about the availability of genetic testing and allows them to order tests. However, testing isn’t available for patients who have had liver transplants or certain bone marrow transplants.

The VA is working on developing decision-making tools to help clinicians determine when the tests are appropriate, Bates said. It typically takes 2 to 3 weeks to get results, she said, adding that external laboratories provide results. “We eventually would like to bring in all pharmacogenomics testing to be conducted within the VA enterprise.”

Bates reported that she had no disclosures.

The US Department of Veterans Affairs (VA) now has a permanent pharmacogenomics service that provides genetic tests to give clinicians insight into the best medication options for their patients.

The tests, which have no extra cost, are available to all veterans, said pharmacist Jill S. Bates, PharmD, MS, executive director of the VA National Pharmacogenomics Program, who spoke in an interview and a presentation at the annual meeting of the Association of VA Hematology/Oncology.

Genetic testing is “a tool that can help optimize care that we provide for veterans,” she said. “Pharmacogenomics is additional information to help the clinician make a decision. We know that most veterans—greater than 90%—carry a variant in a pharmacogenomics gene that is actionable.”

The genetic tests can provide insight into the optimal medication for multiple conditions such as mental illness, gastrointestinal disorders, cancer, pain, and heart disease. According to a 2019 analysis of over 6 years of data, more than half of the VA patient population used medications whose efficacy may have been affected by detectable genetic variants.

For instance, Bates said tests can let clinicians know whether patients are susceptible to statin-associated muscle adverse effects if they take simvastatin, the cholesterol medication. An estimated 25.6% of the VA population has this variant.

Elsewhere on the cardiac front, an estimated 58.3% of the VA population has a genetic variant that increases sensitivity to the blood thinner warfarin.

Testing could help psychiatrists determine whether certain medications should not be prescribed—or should be prescribed at lower doses—in patients who’ve had adverse reactions to antidepressants, Bates said.

In cancer, Bates said, genetic testing can identify patients who have a genetic variant that boosts toxicity from fluoropyrimidine chemotherapy treatments, which include capecitabine, floxuridine, and fluorouracil. Meanwhile, an estimated 0.9% will have no reaction or limited reaction to capecitabine and fluorouracil, and 4.8% will have hypersensitivity to carbamazepine and oxcarbazepine. 

Tests can also identify a genetic variant that can lead to poor metabolism of the chemotherapy drug irinotecan, which is used to treat colon cancer. “In those patients, you’d want to reduce the dose by 20%,” Bates said. In other cases, alternate drugs may be the best strategy to address genetic variations.

Prior to 2019, clinicians had to order pharmacogenomic tests outside of the VA system, according to Bates. That year, a donation from Sanford Health brought VA pharmacogenomics to 40 pilot sites. Since then, more than 88,000 tests have been performed.

The VA has now made its pharmacogenomic program permanent, Bates said. As of early September, testing was available at 139 VA sites and is coming soon to 4 more. It’s not available at another 23 sites that are scattered across the country.

A tool in the VA electronic health record now reminds clinicians about the availability of genetic testing and allows them to order tests. However, testing isn’t available for patients who have had liver transplants or certain bone marrow transplants.

The VA is working on developing decision-making tools to help clinicians determine when the tests are appropriate, Bates said. It typically takes 2 to 3 weeks to get results, she said, adding that external laboratories provide results. “We eventually would like to bring in all pharmacogenomics testing to be conducted within the VA enterprise.”

Bates reported that she had no disclosures.

COPENHAGEN — Serum glial fibrillary acidic protein (sGFAP) is quickly maturing as a biomarker to predict disability in patients with multiple sclerosis (MS), but it will add information to, not compete with, serum neurofilament light chain (sNFl) levels, according to multiple independent studies.

The basic consensus is that “elevated sNFl levels predict inflammatory-associated worsening, while sGFAP values correlate with progression independent of inflammation,” said Enric Monreal, MD, Immunology Department, Ramón y Cajal University Hospital, Madrid, Spain.

This key message was repeated by several researchers presenting data at the 2024 ECTRIMS 2004 meeting, including one delivered as a latebreaker. There was also general agreement that sGFAP will eventually be a routine prognostic tool even if more data are needed to validate how it will be used in routine MS management.
 

A New Biomarker for MS Disability Progression

Although apparently reliable for predicting MS disability, “sGFAP is about 5 years behind where we are with sNFl,” said Evan Madill, MD, a clinical research fellow at the Brigham Multiple Sclerosis Research Center, Harvard Medical School, Boston. He does think, however, that it is coming to clinical practice.

In the study he presented, 744 patients from the Brigham MS Research Center database were evaluated retrospectively for sGFAP levels and subsequent disability progression. Among this cohort, for which sGFAP levels were collected at baseline and over time, 46.5% had 6-month confirmed disability progression (CDP) over follow-up.

On univariate analysis, sGFAP levels correlated with and predicted CDP, need for a new ambulatory aid, and conversion to secondary progressive MS (SPMS). For patients less than 60 years of age, all of these correlations were highly significant (P ≤ .002). On multivariate analysis, the significance was preserved for CDP (P = .032) and for need of a new ambulatory aid (P = .007), but it was lost for SPMS conversion.

Notably, his data suggest that a one-time baseline measurement of sGFAP was more useful than change in sGFAP as a predictor.

It is unclear why sGFAP is less predictive in older individuals, but Dr. Madill speculated that non-MS phenomena might play a role at older ages. Treatment did not influence sGFAP levels in this study, but Dr. Madill said most of the data were collected before anti-CD20 monoclonal antibodies were widely available.

The observational study data presented by Dr. Monreal involved 725 patients drawn from 13 European hospitals. sGFAP and sNFl levels were evaluated from blood drawn within 12 months of MS onset. Over time these biomarkers had overlapping but different predictive strengths.

Consistent with previously published studies, which link elevations in sNFl to neuronal damage and elevations in sGFAP to astrogliosis, sGFAP was found to be more useful for predicting progression independent of relapse activity (PIRA), particularly in patients with low sNFl levels.

Increases in sNFl were associated with an increased risk of both PIRA and relapse-associated worsening (RAW), but sNFl was more closely associated with RAW in untreated patients. The risk of PIRA and RAW were similar across GFAP and sNFl levels in those patients treated with high-efficacy disease-modifying therapies (DMT).

Overall, when stratifying the cohort into three groups, those with both low sNFl and low GFAP, those with high sNFl with low GFAP, and those with high GFAP and low sNFl, the relative risks of disability associated with PIRA and RAW diverged, suggesting these biomarkers correlate with different processes of progression.
 

Comparing sGFAP and sNFl

This same principle was explored further in the latebreaking presentation by Ahmed Abdelhak, MD, a clinical instructor, Weill Institute for Neurosciences, University of California, San Francisco. The objective of his study was to compare sGFAP and sNFl for predicting PIRA in patients on treatment.

The study included 212 patients from the Swiss Multiple Sclerosis Cohort who were started on fingolimod or on B-cell depleting therapies like rituximab. After correcting for sex, age at onset, baseline Expanded Disability Status Scale (EDSS) scores, and other variables, Dr. Abdelhak also reported that the predictive values for PIRA were different for sGFAP relative to sNFl at least on the group level.

However, in this study, unlike the analysis of the Brigham MS Research Center data, changes in sGFAP over time when on treatment did have prognostic value, and there was a relationship between sGFAP levels and treatment. Although reductions in GFAP predicted less disability progression whether patients were treated with fingolimod B-cell depleting therapies, that patterns were different. Dr. Abdelhak, like the other investigators speaking at ECTRIMS, also said the data so far favor sGFAP over sNFl for predicting PIRA.

Each z-score unit change in sGFAP corresponded to a 47% lower risk of PIRA in follow-up over 6.8 years, Dr. Abdelhak reported, adding that the predictive value of sGFAP was “numerically stronger than the corresponding relation for sNFl.”

So far, clinical utility of sGFAP remains speculative. Most of the correlations he presented were on a group rather than the individual level. Moreover, Dr. Abdelhak cautioned that these correlations, based on observational data, do not necessarily reflect causation.

Nonetheless, remarking on the parallels of his data on sGFAP and sNFl with other studies presented at the ECTRIMS meeting, Dr. Abdelhak foresees a time when GFAP will be a prognostic tool, offering relative simplicity and lower cost than the current standard of imaging. He also sees a role in clinical research.

“Monitoring of sGFAP dynamics following DMT initiation could be used to prognosticate long-term PIRA risk and provide insights valuable for design and interpretation of trial outcomes,” he said.

Dr. Monreal reported financial relationships with Almirall, Biogen, Bristol-Myers Squibb, Janssen, Merck, Novartis, Roche, and Sanofi. Dr. Madill and Dr. Abdelhak reported no potential conflicts of interest.

COPENHAGEN — Serum glial fibrillary acidic protein (sGFAP) is quickly maturing as a biomarker to predict disability in patients with multiple sclerosis (MS), but it will add information to, not compete with, serum neurofilament light chain (sNFl) levels, according to multiple independent studies.

The basic consensus is that “elevated sNFl levels predict inflammatory-associated worsening, while sGFAP values correlate with progression independent of inflammation,” said Enric Monreal, MD, Immunology Department, Ramón y Cajal University Hospital, Madrid, Spain.

This key message was repeated by several researchers presenting data at the 2024 ECTRIMS 2004 meeting, including one delivered as a latebreaker. There was also general agreement that sGFAP will eventually be a routine prognostic tool even if more data are needed to validate how it will be used in routine MS management.
 

A New Biomarker for MS Disability Progression

Although apparently reliable for predicting MS disability, “sGFAP is about 5 years behind where we are with sNFl,” said Evan Madill, MD, a clinical research fellow at the Brigham Multiple Sclerosis Research Center, Harvard Medical School, Boston. He does think, however, that it is coming to clinical practice.

In the study he presented, 744 patients from the Brigham MS Research Center database were evaluated retrospectively for sGFAP levels and subsequent disability progression. Among this cohort, for which sGFAP levels were collected at baseline and over time, 46.5% had 6-month confirmed disability progression (CDP) over follow-up.

On univariate analysis, sGFAP levels correlated with and predicted CDP, need for a new ambulatory aid, and conversion to secondary progressive MS (SPMS). For patients less than 60 years of age, all of these correlations were highly significant (P ≤ .002). On multivariate analysis, the significance was preserved for CDP (P = .032) and for need of a new ambulatory aid (P = .007), but it was lost for SPMS conversion.

Notably, his data suggest that a one-time baseline measurement of sGFAP was more useful than change in sGFAP as a predictor.

It is unclear why sGFAP is less predictive in older individuals, but Dr. Madill speculated that non-MS phenomena might play a role at older ages. Treatment did not influence sGFAP levels in this study, but Dr. Madill said most of the data were collected before anti-CD20 monoclonal antibodies were widely available.

The observational study data presented by Dr. Monreal involved 725 patients drawn from 13 European hospitals. sGFAP and sNFl levels were evaluated from blood drawn within 12 months of MS onset. Over time these biomarkers had overlapping but different predictive strengths.

Consistent with previously published studies, which link elevations in sNFl to neuronal damage and elevations in sGFAP to astrogliosis, sGFAP was found to be more useful for predicting progression independent of relapse activity (PIRA), particularly in patients with low sNFl levels.

Increases in sNFl were associated with an increased risk of both PIRA and relapse-associated worsening (RAW), but sNFl was more closely associated with RAW in untreated patients. The risk of PIRA and RAW were similar across GFAP and sNFl levels in those patients treated with high-efficacy disease-modifying therapies (DMT).

Overall, when stratifying the cohort into three groups, those with both low sNFl and low GFAP, those with high sNFl with low GFAP, and those with high GFAP and low sNFl, the relative risks of disability associated with PIRA and RAW diverged, suggesting these biomarkers correlate with different processes of progression.
 

Comparing sGFAP and sNFl

This same principle was explored further in the latebreaking presentation by Ahmed Abdelhak, MD, a clinical instructor, Weill Institute for Neurosciences, University of California, San Francisco. The objective of his study was to compare sGFAP and sNFl for predicting PIRA in patients on treatment.

The study included 212 patients from the Swiss Multiple Sclerosis Cohort who were started on fingolimod or on B-cell depleting therapies like rituximab. After correcting for sex, age at onset, baseline Expanded Disability Status Scale (EDSS) scores, and other variables, Dr. Abdelhak also reported that the predictive values for PIRA were different for sGFAP relative to sNFl at least on the group level.

However, in this study, unlike the analysis of the Brigham MS Research Center data, changes in sGFAP over time when on treatment did have prognostic value, and there was a relationship between sGFAP levels and treatment. Although reductions in GFAP predicted less disability progression whether patients were treated with fingolimod B-cell depleting therapies, that patterns were different. Dr. Abdelhak, like the other investigators speaking at ECTRIMS, also said the data so far favor sGFAP over sNFl for predicting PIRA.

Each z-score unit change in sGFAP corresponded to a 47% lower risk of PIRA in follow-up over 6.8 years, Dr. Abdelhak reported, adding that the predictive value of sGFAP was “numerically stronger than the corresponding relation for sNFl.”

So far, clinical utility of sGFAP remains speculative. Most of the correlations he presented were on a group rather than the individual level. Moreover, Dr. Abdelhak cautioned that these correlations, based on observational data, do not necessarily reflect causation.

Nonetheless, remarking on the parallels of his data on sGFAP and sNFl with other studies presented at the ECTRIMS meeting, Dr. Abdelhak foresees a time when GFAP will be a prognostic tool, offering relative simplicity and lower cost than the current standard of imaging. He also sees a role in clinical research.

“Monitoring of sGFAP dynamics following DMT initiation could be used to prognosticate long-term PIRA risk and provide insights valuable for design and interpretation of trial outcomes,” he said.

Dr. Monreal reported financial relationships with Almirall, Biogen, Bristol-Myers Squibb, Janssen, Merck, Novartis, Roche, and Sanofi. Dr. Madill and Dr. Abdelhak reported no potential conflicts of interest.

COPENHAGEN — Serum glial fibrillary acidic protein (sGFAP) is quickly maturing as a biomarker to predict disability in patients with multiple sclerosis (MS), but it will add information to, not compete with, serum neurofilament light chain (sNFl) levels, according to multiple independent studies.

The basic consensus is that “elevated sNFl levels predict inflammatory-associated worsening, while sGFAP values correlate with progression independent of inflammation,” said Enric Monreal, MD, Immunology Department, Ramón y Cajal University Hospital, Madrid, Spain.

This key message was repeated by several researchers presenting data at the 2024 ECTRIMS 2004 meeting, including one delivered as a latebreaker. There was also general agreement that sGFAP will eventually be a routine prognostic tool even if more data are needed to validate how it will be used in routine MS management.
 

A New Biomarker for MS Disability Progression

Although apparently reliable for predicting MS disability, “sGFAP is about 5 years behind where we are with sNFl,” said Evan Madill, MD, a clinical research fellow at the Brigham Multiple Sclerosis Research Center, Harvard Medical School, Boston. He does think, however, that it is coming to clinical practice.

In the study he presented, 744 patients from the Brigham MS Research Center database were evaluated retrospectively for sGFAP levels and subsequent disability progression. Among this cohort, for which sGFAP levels were collected at baseline and over time, 46.5% had 6-month confirmed disability progression (CDP) over follow-up.

On univariate analysis, sGFAP levels correlated with and predicted CDP, need for a new ambulatory aid, and conversion to secondary progressive MS (SPMS). For patients less than 60 years of age, all of these correlations were highly significant (P ≤ .002). On multivariate analysis, the significance was preserved for CDP (P = .032) and for need of a new ambulatory aid (P = .007), but it was lost for SPMS conversion.

Notably, his data suggest that a one-time baseline measurement of sGFAP was more useful than change in sGFAP as a predictor.

It is unclear why sGFAP is less predictive in older individuals, but Dr. Madill speculated that non-MS phenomena might play a role at older ages. Treatment did not influence sGFAP levels in this study, but Dr. Madill said most of the data were collected before anti-CD20 monoclonal antibodies were widely available.

The observational study data presented by Dr. Monreal involved 725 patients drawn from 13 European hospitals. sGFAP and sNFl levels were evaluated from blood drawn within 12 months of MS onset. Over time these biomarkers had overlapping but different predictive strengths.

Consistent with previously published studies, which link elevations in sNFl to neuronal damage and elevations in sGFAP to astrogliosis, sGFAP was found to be more useful for predicting progression independent of relapse activity (PIRA), particularly in patients with low sNFl levels.

Increases in sNFl were associated with an increased risk of both PIRA and relapse-associated worsening (RAW), but sNFl was more closely associated with RAW in untreated patients. The risk of PIRA and RAW were similar across GFAP and sNFl levels in those patients treated with high-efficacy disease-modifying therapies (DMT).

Overall, when stratifying the cohort into three groups, those with both low sNFl and low GFAP, those with high sNFl with low GFAP, and those with high GFAP and low sNFl, the relative risks of disability associated with PIRA and RAW diverged, suggesting these biomarkers correlate with different processes of progression.
 

Comparing sGFAP and sNFl

This same principle was explored further in the latebreaking presentation by Ahmed Abdelhak, MD, a clinical instructor, Weill Institute for Neurosciences, University of California, San Francisco. The objective of his study was to compare sGFAP and sNFl for predicting PIRA in patients on treatment.

The study included 212 patients from the Swiss Multiple Sclerosis Cohort who were started on fingolimod or on B-cell depleting therapies like rituximab. After correcting for sex, age at onset, baseline Expanded Disability Status Scale (EDSS) scores, and other variables, Dr. Abdelhak also reported that the predictive values for PIRA were different for sGFAP relative to sNFl at least on the group level.

However, in this study, unlike the analysis of the Brigham MS Research Center data, changes in sGFAP over time when on treatment did have prognostic value, and there was a relationship between sGFAP levels and treatment. Although reductions in GFAP predicted less disability progression whether patients were treated with fingolimod B-cell depleting therapies, that patterns were different. Dr. Abdelhak, like the other investigators speaking at ECTRIMS, also said the data so far favor sGFAP over sNFl for predicting PIRA.

Each z-score unit change in sGFAP corresponded to a 47% lower risk of PIRA in follow-up over 6.8 years, Dr. Abdelhak reported, adding that the predictive value of sGFAP was “numerically stronger than the corresponding relation for sNFl.”

So far, clinical utility of sGFAP remains speculative. Most of the correlations he presented were on a group rather than the individual level. Moreover, Dr. Abdelhak cautioned that these correlations, based on observational data, do not necessarily reflect causation.

Nonetheless, remarking on the parallels of his data on sGFAP and sNFl with other studies presented at the ECTRIMS meeting, Dr. Abdelhak foresees a time when GFAP will be a prognostic tool, offering relative simplicity and lower cost than the current standard of imaging. He also sees a role in clinical research.

“Monitoring of sGFAP dynamics following DMT initiation could be used to prognosticate long-term PIRA risk and provide insights valuable for design and interpretation of trial outcomes,” he said.

Dr. Monreal reported financial relationships with Almirall, Biogen, Bristol-Myers Squibb, Janssen, Merck, Novartis, Roche, and Sanofi. Dr. Madill and Dr. Abdelhak reported no potential conflicts of interest.

For patients with intermediate- or low-risk localized prostate cancer, intensity-modulated radiation therapy (IMRT) and proton beam therapy are both safe and effective options, according to results of the phase 3 randomized controlled PARTIQoL trial.

With both techniques, disease control rates were over 90%, with virtually no difference in bowel function or other quality-of-life ratings after 2 years, reported Jason Efstathiou, MD, PhD, with Massachusetts General Hospital, Boston, at the annual meeting of the American Society for Radiation Oncology (ASTRO).

“This is a tremendous study [that] really shows us we have two great options, with equal results across the board for both control rates and toxicity rates,” said Sameer Keole, MD, incoming ASTRO president, during a press briefing.

“These control rates are phenomenal, and the complication rates were very low,” continued Dr. Keole, with the Mayo Clinic in Phoenix, Arizona. “I think men can go and seek definitive treatment when it’s appropriate with a radiation oncologist and know that whether it’s proton therapy or IMRT; it’s an excellent treatment option.”

Overall, about 70% of new cases of prostate cancer each year are localized disease, which represents about 200,000 patients in the United States each year, Dr. Efstathiou explained. These patients have several treatment options, including different choices for external beam radiation therapy.

“Because many of these patients are going to survive their cancer and live many years after treatment, quality of life becomes paramount because they’re at risk for long-term posttreatment morbidity,” Dr. Efstathiou said. “Quality of life will inform their decision-making.”

Dr. Efstathiou noted that proton beam therapy comes with certain dosimetric advantages with the potential to reduce morbidity and improve cancer outcomes, but it is generally more resource intensive and costly than IMRT.

The PARTIQoL multicenter, phase 3, randomized trial compared patient-reported quality of life after external beam radiation using either IMRT or proton beam therapy to determine whether one performs better on the local control and toxicity fronts.

After stratifying by institution, age (< 65 years vs ≥ 65 years), rectal spacer use (no vs yes), and moderate hypofractionation (no vs yes), participants were randomized to either proton beam therapy or IMRT.

Patients were followed longitudinally for 60 months after completing radiotherapy. The primary endpoint was bowel function at 24 months using the Expanded Prostate Cancer Index Composite (EPIC) instrument. Secondary outcomes included urinary and erectile function, sexual function, toxicity and efficacy, or disease control endpoints.

Of the 450 patients randomized, 221 of 226 (97.8%) randomized to proton beam therapy and 216 of 224 (96.4%) randomized to IMRT started on their respective treatments, and 167 and 162, respectively, completed the EPIC at 24 months. This represents about a 27% rate of missing data, which “was much better than anticipated,” Dr. Efstathiou noted.

For the primary endpoint, there was no difference between proton beam therapy and IMRT in mean change of the EPIC bowel score at 24 months, with both treatment groups showing only a small, clinically nonrelevant decline from baseline. There was only about a 2% decrease on a 100-point scale in bowel quality of life after 2 years, Dr. Efstathiou reported.

Similarly, the team noted no difference in bowel function at earlier or later time points. “We see some small fluctuations, but at no time point did these reach statistical significance,” he noted.

There were also no differences observed in the other domains at any point, including urinary incontinence, urinary irritation, or sexual function.

Turning to disease control, Dr. Efstathiou and colleagues found no difference between the two groups in progression-free survival. The progression-free survival rate was 99% at 24 months and 93.7% at 60 months with IMRT, compared with 98.1% at 24 months and 93.4% at 60 months with proton beam therapy.

When looking at key subgroups or factors, the team reported no sustained difference in any quality-of-life domain or in cancer control.

Patient monitoring over a longer follow-up period is ongoing. Dr. Efstathiou noted that the PARTIQoL trial was limited to localized low- and intermediate-risk prostate cancer patients receiving either conventionally or moderately hypofractionated therapy. The trial also did not address the full range of disease scope, including higher risk disease, nodal therapy, concurrent use of hormonal therapy or other systemic therapy, local recurrent situations, or retreatment situations.

Dr. Efstathiou noted that because both proton therapy and IMRT continue to evolve, there is ongoing work to optimize the delivery of both.

Overall, the PARTIQoL trial results demonstrate “equivalent outcomes, with superb cancer control rates and extremely low toxicity from both treatments,” Jessica Karen Wong, MD, MEng, who wasn’t involved in the study, told this news organization.

“Both are excellent treatments for low- and intermediate-risk prostate cancer patients,” said Dr. Wong, Department of Radiation Oncology, Fox Chase Cancer Center, Philadelphia, Pennsylvania. “This study corroborates prior single and multi-institutional experiences with the statistical power and rigorous methods of a clinical trial. Dr Efstathiou and authors should be commended for this comprehensive and well-run trial.”

Discussant for the study, Curtiland Deville, MD, of Johns Hopkins University School of Medicine, Baltimore, Maryland, agreed that patients in the trial did “exceedingly well,” regardless of whether patients received IMRT or proton therapy.

Dr. Deville said the “fundamental question regarding the use of proton therapy for prostate cancer remains — is there a clinical benefit to protons that justifies their increased costs in this setting? In a cost-neutral setting, it may still be considered very reasonable to deliver proton therapy for prostate cancer.”

In his view, this study is “practice informing” but not yet “practice changing as we await the imminent findings of the COMPARE trial,” which uses a pragmatic design powered to assess the co-primary patient-reported outcome endpoints of EPIC bowel summary, urinary function, and sexual function scores at 2 years, and which enrolled over 2500 patients.

The study has no commercial funding. Dr. Efstathiou disclosed various relationships with IBA Proton Therapy, Blue Earth Diagnostics, Boston Scientific, AstraZeneca, Genentech, Lantheus/Progenics, Astellas/Pfizer, Elekta, Uptodate, Merck, Roivant Pharma, Myovant Sciences, EMD Serono, Bayer Healthcare, Janssen, Pfizer, Progenics Pharmaceuticals, Gilead, Angiodynamics, and Clarity Pharmaceuticals. Dr. Keole and Dr. Wong had no relevant disclosures. Dr. Deville is deputy editor of the ASTRO Red Journal.

A version of this article appeared on Medscape.com.

For patients with intermediate- or low-risk localized prostate cancer, intensity-modulated radiation therapy (IMRT) and proton beam therapy are both safe and effective options, according to results of the phase 3 randomized controlled PARTIQoL trial.

With both techniques, disease control rates were over 90%, with virtually no difference in bowel function or other quality-of-life ratings after 2 years, reported Jason Efstathiou, MD, PhD, with Massachusetts General Hospital, Boston, at the annual meeting of the American Society for Radiation Oncology (ASTRO).

“This is a tremendous study [that] really shows us we have two great options, with equal results across the board for both control rates and toxicity rates,” said Sameer Keole, MD, incoming ASTRO president, during a press briefing.

“These control rates are phenomenal, and the complication rates were very low,” continued Dr. Keole, with the Mayo Clinic in Phoenix, Arizona. “I think men can go and seek definitive treatment when it’s appropriate with a radiation oncologist and know that whether it’s proton therapy or IMRT; it’s an excellent treatment option.”

Overall, about 70% of new cases of prostate cancer each year are localized disease, which represents about 200,000 patients in the United States each year, Dr. Efstathiou explained. These patients have several treatment options, including different choices for external beam radiation therapy.

“Because many of these patients are going to survive their cancer and live many years after treatment, quality of life becomes paramount because they’re at risk for long-term posttreatment morbidity,” Dr. Efstathiou said. “Quality of life will inform their decision-making.”

Dr. Efstathiou noted that proton beam therapy comes with certain dosimetric advantages with the potential to reduce morbidity and improve cancer outcomes, but it is generally more resource intensive and costly than IMRT.

The PARTIQoL multicenter, phase 3, randomized trial compared patient-reported quality of life after external beam radiation using either IMRT or proton beam therapy to determine whether one performs better on the local control and toxicity fronts.

After stratifying by institution, age (< 65 years vs ≥ 65 years), rectal spacer use (no vs yes), and moderate hypofractionation (no vs yes), participants were randomized to either proton beam therapy or IMRT.

Patients were followed longitudinally for 60 months after completing radiotherapy. The primary endpoint was bowel function at 24 months using the Expanded Prostate Cancer Index Composite (EPIC) instrument. Secondary outcomes included urinary and erectile function, sexual function, toxicity and efficacy, or disease control endpoints.

Of the 450 patients randomized, 221 of 226 (97.8%) randomized to proton beam therapy and 216 of 224 (96.4%) randomized to IMRT started on their respective treatments, and 167 and 162, respectively, completed the EPIC at 24 months. This represents about a 27% rate of missing data, which “was much better than anticipated,” Dr. Efstathiou noted.

For the primary endpoint, there was no difference between proton beam therapy and IMRT in mean change of the EPIC bowel score at 24 months, with both treatment groups showing only a small, clinically nonrelevant decline from baseline. There was only about a 2% decrease on a 100-point scale in bowel quality of life after 2 years, Dr. Efstathiou reported.

Similarly, the team noted no difference in bowel function at earlier or later time points. “We see some small fluctuations, but at no time point did these reach statistical significance,” he noted.

There were also no differences observed in the other domains at any point, including urinary incontinence, urinary irritation, or sexual function.

Turning to disease control, Dr. Efstathiou and colleagues found no difference between the two groups in progression-free survival. The progression-free survival rate was 99% at 24 months and 93.7% at 60 months with IMRT, compared with 98.1% at 24 months and 93.4% at 60 months with proton beam therapy.

When looking at key subgroups or factors, the team reported no sustained difference in any quality-of-life domain or in cancer control.

Patient monitoring over a longer follow-up period is ongoing. Dr. Efstathiou noted that the PARTIQoL trial was limited to localized low- and intermediate-risk prostate cancer patients receiving either conventionally or moderately hypofractionated therapy. The trial also did not address the full range of disease scope, including higher risk disease, nodal therapy, concurrent use of hormonal therapy or other systemic therapy, local recurrent situations, or retreatment situations.

Dr. Efstathiou noted that because both proton therapy and IMRT continue to evolve, there is ongoing work to optimize the delivery of both.

Overall, the PARTIQoL trial results demonstrate “equivalent outcomes, with superb cancer control rates and extremely low toxicity from both treatments,” Jessica Karen Wong, MD, MEng, who wasn’t involved in the study, told this news organization.

“Both are excellent treatments for low- and intermediate-risk prostate cancer patients,” said Dr. Wong, Department of Radiation Oncology, Fox Chase Cancer Center, Philadelphia, Pennsylvania. “This study corroborates prior single and multi-institutional experiences with the statistical power and rigorous methods of a clinical trial. Dr Efstathiou and authors should be commended for this comprehensive and well-run trial.”

Discussant for the study, Curtiland Deville, MD, of Johns Hopkins University School of Medicine, Baltimore, Maryland, agreed that patients in the trial did “exceedingly well,” regardless of whether patients received IMRT or proton therapy.

Dr. Deville said the “fundamental question regarding the use of proton therapy for prostate cancer remains — is there a clinical benefit to protons that justifies their increased costs in this setting? In a cost-neutral setting, it may still be considered very reasonable to deliver proton therapy for prostate cancer.”

In his view, this study is “practice informing” but not yet “practice changing as we await the imminent findings of the COMPARE trial,” which uses a pragmatic design powered to assess the co-primary patient-reported outcome endpoints of EPIC bowel summary, urinary function, and sexual function scores at 2 years, and which enrolled over 2500 patients.

The study has no commercial funding. Dr. Efstathiou disclosed various relationships with IBA Proton Therapy, Blue Earth Diagnostics, Boston Scientific, AstraZeneca, Genentech, Lantheus/Progenics, Astellas/Pfizer, Elekta, Uptodate, Merck, Roivant Pharma, Myovant Sciences, EMD Serono, Bayer Healthcare, Janssen, Pfizer, Progenics Pharmaceuticals, Gilead, Angiodynamics, and Clarity Pharmaceuticals. Dr. Keole and Dr. Wong had no relevant disclosures. Dr. Deville is deputy editor of the ASTRO Red Journal.

A version of this article appeared on Medscape.com.

For patients with intermediate- or low-risk localized prostate cancer, intensity-modulated radiation therapy (IMRT) and proton beam therapy are both safe and effective options, according to results of the phase 3 randomized controlled PARTIQoL trial.

With both techniques, disease control rates were over 90%, with virtually no difference in bowel function or other quality-of-life ratings after 2 years, reported Jason Efstathiou, MD, PhD, with Massachusetts General Hospital, Boston, at the annual meeting of the American Society for Radiation Oncology (ASTRO).

“This is a tremendous study [that] really shows us we have two great options, with equal results across the board for both control rates and toxicity rates,” said Sameer Keole, MD, incoming ASTRO president, during a press briefing.

“These control rates are phenomenal, and the complication rates were very low,” continued Dr. Keole, with the Mayo Clinic in Phoenix, Arizona. “I think men can go and seek definitive treatment when it’s appropriate with a radiation oncologist and know that whether it’s proton therapy or IMRT; it’s an excellent treatment option.”

Overall, about 70% of new cases of prostate cancer each year are localized disease, which represents about 200,000 patients in the United States each year, Dr. Efstathiou explained. These patients have several treatment options, including different choices for external beam radiation therapy.

“Because many of these patients are going to survive their cancer and live many years after treatment, quality of life becomes paramount because they’re at risk for long-term posttreatment morbidity,” Dr. Efstathiou said. “Quality of life will inform their decision-making.”

Dr. Efstathiou noted that proton beam therapy comes with certain dosimetric advantages with the potential to reduce morbidity and improve cancer outcomes, but it is generally more resource intensive and costly than IMRT.

The PARTIQoL multicenter, phase 3, randomized trial compared patient-reported quality of life after external beam radiation using either IMRT or proton beam therapy to determine whether one performs better on the local control and toxicity fronts.

After stratifying by institution, age (< 65 years vs ≥ 65 years), rectal spacer use (no vs yes), and moderate hypofractionation (no vs yes), participants were randomized to either proton beam therapy or IMRT.

Patients were followed longitudinally for 60 months after completing radiotherapy. The primary endpoint was bowel function at 24 months using the Expanded Prostate Cancer Index Composite (EPIC) instrument. Secondary outcomes included urinary and erectile function, sexual function, toxicity and efficacy, or disease control endpoints.

Of the 450 patients randomized, 221 of 226 (97.8%) randomized to proton beam therapy and 216 of 224 (96.4%) randomized to IMRT started on their respective treatments, and 167 and 162, respectively, completed the EPIC at 24 months. This represents about a 27% rate of missing data, which “was much better than anticipated,” Dr. Efstathiou noted.

For the primary endpoint, there was no difference between proton beam therapy and IMRT in mean change of the EPIC bowel score at 24 months, with both treatment groups showing only a small, clinically nonrelevant decline from baseline. There was only about a 2% decrease on a 100-point scale in bowel quality of life after 2 years, Dr. Efstathiou reported.

Similarly, the team noted no difference in bowel function at earlier or later time points. “We see some small fluctuations, but at no time point did these reach statistical significance,” he noted.

There were also no differences observed in the other domains at any point, including urinary incontinence, urinary irritation, or sexual function.

Turning to disease control, Dr. Efstathiou and colleagues found no difference between the two groups in progression-free survival. The progression-free survival rate was 99% at 24 months and 93.7% at 60 months with IMRT, compared with 98.1% at 24 months and 93.4% at 60 months with proton beam therapy.

When looking at key subgroups or factors, the team reported no sustained difference in any quality-of-life domain or in cancer control.

Patient monitoring over a longer follow-up period is ongoing. Dr. Efstathiou noted that the PARTIQoL trial was limited to localized low- and intermediate-risk prostate cancer patients receiving either conventionally or moderately hypofractionated therapy. The trial also did not address the full range of disease scope, including higher risk disease, nodal therapy, concurrent use of hormonal therapy or other systemic therapy, local recurrent situations, or retreatment situations.

Dr. Efstathiou noted that because both proton therapy and IMRT continue to evolve, there is ongoing work to optimize the delivery of both.

Overall, the PARTIQoL trial results demonstrate “equivalent outcomes, with superb cancer control rates and extremely low toxicity from both treatments,” Jessica Karen Wong, MD, MEng, who wasn’t involved in the study, told this news organization.

“Both are excellent treatments for low- and intermediate-risk prostate cancer patients,” said Dr. Wong, Department of Radiation Oncology, Fox Chase Cancer Center, Philadelphia, Pennsylvania. “This study corroborates prior single and multi-institutional experiences with the statistical power and rigorous methods of a clinical trial. Dr Efstathiou and authors should be commended for this comprehensive and well-run trial.”

Discussant for the study, Curtiland Deville, MD, of Johns Hopkins University School of Medicine, Baltimore, Maryland, agreed that patients in the trial did “exceedingly well,” regardless of whether patients received IMRT or proton therapy.

Dr. Deville said the “fundamental question regarding the use of proton therapy for prostate cancer remains — is there a clinical benefit to protons that justifies their increased costs in this setting? In a cost-neutral setting, it may still be considered very reasonable to deliver proton therapy for prostate cancer.”

In his view, this study is “practice informing” but not yet “practice changing as we await the imminent findings of the COMPARE trial,” which uses a pragmatic design powered to assess the co-primary patient-reported outcome endpoints of EPIC bowel summary, urinary function, and sexual function scores at 2 years, and which enrolled over 2500 patients.

The study has no commercial funding. Dr. Efstathiou disclosed various relationships with IBA Proton Therapy, Blue Earth Diagnostics, Boston Scientific, AstraZeneca, Genentech, Lantheus/Progenics, Astellas/Pfizer, Elekta, Uptodate, Merck, Roivant Pharma, Myovant Sciences, EMD Serono, Bayer Healthcare, Janssen, Pfizer, Progenics Pharmaceuticals, Gilead, Angiodynamics, and Clarity Pharmaceuticals. Dr. Keole and Dr. Wong had no relevant disclosures. Dr. Deville is deputy editor of the ASTRO Red Journal.

A version of this article appeared on Medscape.com.

Neoadjuvant concurrent chemoradiotherapy significantly improves survival outcomes over adjuvant sequential chemoradiotherapy (ASCRT) in patients with locally advanced esophageal squamous cell carcinoma, confirmed the first randomized trial to directly compare the two approaches.

Yaoyao Zhu, MD, Department of Radiation Oncology, Shanghai Pulmonary Hospital, Shanghai, China, presented the new research at the annual World Conference on Lung Cancer on September 10.

Based on the findings, neoadjuvant concurrent chemoradiotherapy (NCCRT) followed by surgical resection “should be regarded as the standard of care for patients with locally advanced esophageal squamous cell carcinoma (ESCC) in the Chinese population,” Dr. Zhu said.
 

Different Approaches in ESCC

Dr. Zhu began her presentation by underscoring that in Western countries, NCCRT followed by surgery has been the standard treatment for locally advanced, resectable esophageal cancer since the publication of the CROSS trial in 2012, which compared neoadjuvant therapy plus surgery with surgery alone.

This demonstrated that preoperative chemoradiotherapy improved survival by 34% in patients with potentially curable esophageal or esophagogastric junction cancer, while adverse event rates were deemed “acceptable.”

In contrast, in most centers in China, clinicians opt for performing surgery followed by ASCRT.

Dr. Zhu pointed out that as previous randomized controlled trials have used surgery alone as the comparator arm, it has not been shown definitively that NCCRT plus surgery is superior to surgery followed by ASCRT.

The researchers, therefore, conducted the NEOTERIC trial, which enrolled patients with clinically resectable, locally advanced ESCC, defined as clinical stage T1-2N1M0 or T3-4N0-1M0.

They were randomized to one of two arms. The NCCRT arm involved 6 weeks of carboplatin plus paclitaxel chemotherapy alongside radiotherapy delivered as 50.4 Gy over 28 fractions. After an interval of 4-6 weeks, the patients underwent surgery, followed by an optional two cycles of carboplatin plus paclitaxel 4-6 weeks later.

In the ASCRT arm, patients underwent surgery straightaway, waited for 4-6 weeks, then had two cycles of carboplatin plus paclitaxel 3 weeks apart, followed by the same radiotherapy regimen as in the first arm. About 2-4 weeks later, patients could then undergo another two cycles of carboplatin plus paclitaxel.
 

More Than Doubling of Survival Outcomes

One hundred patients were assigned to NCCRT and 104 to ASCRT. There were no significant differences between the groups in terms of their baseline characteristics.

The vast majority of patients were men, just over two thirds were smokers, and the median age was around 60 years. The median tumor length was approximately 5 cm, and around half of tumors were located in the middle third of the esophagus.

Median disease-free survival was markedly longer with NCCRT, at 51.0 months vs 14.0 months in the ASCRT arm (P = .01). Similarly, median overall survival was far longer with neoadjuvant therapy, at 79.0 months, vs 38.0 months when waiting until after surgery to provide chemoradiotherapy (P = .025).

There were no significant differences in postsurgical complications between the two arms, and no significant differences in rates of grade 3-4 hematologic and nonhematologic toxicities. There were also no chemoradiotherapy-related deaths.

The most common toxicities across the two study arms were esophagitis, neutropenia, thrombocytopenia, and leukopenia.

Overall, the rates of recurrence were significantly lower with NCCRT than with ASCRT (58.0% vs 66.3%; P = .020). This included significant reductions in both locoregional (P = .012) and distant recurrence (P = .009).

Jaffer A. Ajani, MD, University of Texas MD Anderson Cancer Center, Houston, underlined that the experimental arm of the trial, with neoadjuvant chemoradiotherapy, “has been the standard of care in the United States for a long time, particularly for squamous carcinoma.”

However, he said in an interview that it is not a standard of care in China and clinicians continue with adjuvant therapy. This is despite a recent study conducted in Hong Kong that concluded that patients should not be given any treatment after surgery “because they do worse” than those given neoadjuvant therapy, he continued.

While Dr. Ajani noted that the current analysis is underpowered to provide a definitive conclusion, it remains “an important study for Chinese patients.

“Hopefully, it will be well advertised in China, and all the providers switch [to NCCRT]. This could push them to abandon what in the West was considered harmful.”

Dr. Ajani explained the reason neoadjuvant therapy performs better than adjuvant chemoradiotherapy is it “may be mopping up some of the micro metastatic disease, which is difficult to do after surgery,” especially as many patients cannot tolerate postoperative treatment.

“It may be that the majority of patients don’t even get [adjuvant therapy], and those who get it don’t seem to benefit.”

Vishwanath Sathyanarayanan, MD, PhD, Senior Consultant, Professor and Academic Advisor, Department of Medical Oncology, Apollo Cancer Centers, Bangalore, India, agreed that the study reinforces that “NCCRT continues to remain the standard of care in locally advanced resectable esophageal squamous cell carcinoma.”

Consequently, there are “no implications for clinical practice” for providers in the West from these study results, “particularly as NCCRT significantly improves outcomes vs ASCRT with a similar toxicity profile,” he said in an interview.

No funding was declared. Dr. Zhu declared no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

Neoadjuvant concurrent chemoradiotherapy significantly improves survival outcomes over adjuvant sequential chemoradiotherapy (ASCRT) in patients with locally advanced esophageal squamous cell carcinoma, confirmed the first randomized trial to directly compare the two approaches.

Yaoyao Zhu, MD, Department of Radiation Oncology, Shanghai Pulmonary Hospital, Shanghai, China, presented the new research at the annual World Conference on Lung Cancer on September 10.

Based on the findings, neoadjuvant concurrent chemoradiotherapy (NCCRT) followed by surgical resection “should be regarded as the standard of care for patients with locally advanced esophageal squamous cell carcinoma (ESCC) in the Chinese population,” Dr. Zhu said.
 

Different Approaches in ESCC

Dr. Zhu began her presentation by underscoring that in Western countries, NCCRT followed by surgery has been the standard treatment for locally advanced, resectable esophageal cancer since the publication of the CROSS trial in 2012, which compared neoadjuvant therapy plus surgery with surgery alone.

This demonstrated that preoperative chemoradiotherapy improved survival by 34% in patients with potentially curable esophageal or esophagogastric junction cancer, while adverse event rates were deemed “acceptable.”

In contrast, in most centers in China, clinicians opt for performing surgery followed by ASCRT.

Dr. Zhu pointed out that as previous randomized controlled trials have used surgery alone as the comparator arm, it has not been shown definitively that NCCRT plus surgery is superior to surgery followed by ASCRT.

The researchers, therefore, conducted the NEOTERIC trial, which enrolled patients with clinically resectable, locally advanced ESCC, defined as clinical stage T1-2N1M0 or T3-4N0-1M0.

They were randomized to one of two arms. The NCCRT arm involved 6 weeks of carboplatin plus paclitaxel chemotherapy alongside radiotherapy delivered as 50.4 Gy over 28 fractions. After an interval of 4-6 weeks, the patients underwent surgery, followed by an optional two cycles of carboplatin plus paclitaxel 4-6 weeks later.

In the ASCRT arm, patients underwent surgery straightaway, waited for 4-6 weeks, then had two cycles of carboplatin plus paclitaxel 3 weeks apart, followed by the same radiotherapy regimen as in the first arm. About 2-4 weeks later, patients could then undergo another two cycles of carboplatin plus paclitaxel.
 

More Than Doubling of Survival Outcomes

One hundred patients were assigned to NCCRT and 104 to ASCRT. There were no significant differences between the groups in terms of their baseline characteristics.

The vast majority of patients were men, just over two thirds were smokers, and the median age was around 60 years. The median tumor length was approximately 5 cm, and around half of tumors were located in the middle third of the esophagus.

Median disease-free survival was markedly longer with NCCRT, at 51.0 months vs 14.0 months in the ASCRT arm (P = .01). Similarly, median overall survival was far longer with neoadjuvant therapy, at 79.0 months, vs 38.0 months when waiting until after surgery to provide chemoradiotherapy (P = .025).

There were no significant differences in postsurgical complications between the two arms, and no significant differences in rates of grade 3-4 hematologic and nonhematologic toxicities. There were also no chemoradiotherapy-related deaths.

The most common toxicities across the two study arms were esophagitis, neutropenia, thrombocytopenia, and leukopenia.

Overall, the rates of recurrence were significantly lower with NCCRT than with ASCRT (58.0% vs 66.3%; P = .020). This included significant reductions in both locoregional (P = .012) and distant recurrence (P = .009).

Jaffer A. Ajani, MD, University of Texas MD Anderson Cancer Center, Houston, underlined that the experimental arm of the trial, with neoadjuvant chemoradiotherapy, “has been the standard of care in the United States for a long time, particularly for squamous carcinoma.”

However, he said in an interview that it is not a standard of care in China and clinicians continue with adjuvant therapy. This is despite a recent study conducted in Hong Kong that concluded that patients should not be given any treatment after surgery “because they do worse” than those given neoadjuvant therapy, he continued.

While Dr. Ajani noted that the current analysis is underpowered to provide a definitive conclusion, it remains “an important study for Chinese patients.

“Hopefully, it will be well advertised in China, and all the providers switch [to NCCRT]. This could push them to abandon what in the West was considered harmful.”

Dr. Ajani explained the reason neoadjuvant therapy performs better than adjuvant chemoradiotherapy is it “may be mopping up some of the micro metastatic disease, which is difficult to do after surgery,” especially as many patients cannot tolerate postoperative treatment.

“It may be that the majority of patients don’t even get [adjuvant therapy], and those who get it don’t seem to benefit.”

Vishwanath Sathyanarayanan, MD, PhD, Senior Consultant, Professor and Academic Advisor, Department of Medical Oncology, Apollo Cancer Centers, Bangalore, India, agreed that the study reinforces that “NCCRT continues to remain the standard of care in locally advanced resectable esophageal squamous cell carcinoma.”

Consequently, there are “no implications for clinical practice” for providers in the West from these study results, “particularly as NCCRT significantly improves outcomes vs ASCRT with a similar toxicity profile,” he said in an interview.

No funding was declared. Dr. Zhu declared no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

Neoadjuvant concurrent chemoradiotherapy significantly improves survival outcomes over adjuvant sequential chemoradiotherapy (ASCRT) in patients with locally advanced esophageal squamous cell carcinoma, confirmed the first randomized trial to directly compare the two approaches.

Yaoyao Zhu, MD, Department of Radiation Oncology, Shanghai Pulmonary Hospital, Shanghai, China, presented the new research at the annual World Conference on Lung Cancer on September 10.

Based on the findings, neoadjuvant concurrent chemoradiotherapy (NCCRT) followed by surgical resection “should be regarded as the standard of care for patients with locally advanced esophageal squamous cell carcinoma (ESCC) in the Chinese population,” Dr. Zhu said.
 

Different Approaches in ESCC

Dr. Zhu began her presentation by underscoring that in Western countries, NCCRT followed by surgery has been the standard treatment for locally advanced, resectable esophageal cancer since the publication of the CROSS trial in 2012, which compared neoadjuvant therapy plus surgery with surgery alone.

This demonstrated that preoperative chemoradiotherapy improved survival by 34% in patients with potentially curable esophageal or esophagogastric junction cancer, while adverse event rates were deemed “acceptable.”

In contrast, in most centers in China, clinicians opt for performing surgery followed by ASCRT.

Dr. Zhu pointed out that as previous randomized controlled trials have used surgery alone as the comparator arm, it has not been shown definitively that NCCRT plus surgery is superior to surgery followed by ASCRT.

The researchers, therefore, conducted the NEOTERIC trial, which enrolled patients with clinically resectable, locally advanced ESCC, defined as clinical stage T1-2N1M0 or T3-4N0-1M0.

They were randomized to one of two arms. The NCCRT arm involved 6 weeks of carboplatin plus paclitaxel chemotherapy alongside radiotherapy delivered as 50.4 Gy over 28 fractions. After an interval of 4-6 weeks, the patients underwent surgery, followed by an optional two cycles of carboplatin plus paclitaxel 4-6 weeks later.

In the ASCRT arm, patients underwent surgery straightaway, waited for 4-6 weeks, then had two cycles of carboplatin plus paclitaxel 3 weeks apart, followed by the same radiotherapy regimen as in the first arm. About 2-4 weeks later, patients could then undergo another two cycles of carboplatin plus paclitaxel.
 

More Than Doubling of Survival Outcomes

One hundred patients were assigned to NCCRT and 104 to ASCRT. There were no significant differences between the groups in terms of their baseline characteristics.

The vast majority of patients were men, just over two thirds were smokers, and the median age was around 60 years. The median tumor length was approximately 5 cm, and around half of tumors were located in the middle third of the esophagus.

Median disease-free survival was markedly longer with NCCRT, at 51.0 months vs 14.0 months in the ASCRT arm (P = .01). Similarly, median overall survival was far longer with neoadjuvant therapy, at 79.0 months, vs 38.0 months when waiting until after surgery to provide chemoradiotherapy (P = .025).

There were no significant differences in postsurgical complications between the two arms, and no significant differences in rates of grade 3-4 hematologic and nonhematologic toxicities. There were also no chemoradiotherapy-related deaths.

The most common toxicities across the two study arms were esophagitis, neutropenia, thrombocytopenia, and leukopenia.

Overall, the rates of recurrence were significantly lower with NCCRT than with ASCRT (58.0% vs 66.3%; P = .020). This included significant reductions in both locoregional (P = .012) and distant recurrence (P = .009).

Jaffer A. Ajani, MD, University of Texas MD Anderson Cancer Center, Houston, underlined that the experimental arm of the trial, with neoadjuvant chemoradiotherapy, “has been the standard of care in the United States for a long time, particularly for squamous carcinoma.”

However, he said in an interview that it is not a standard of care in China and clinicians continue with adjuvant therapy. This is despite a recent study conducted in Hong Kong that concluded that patients should not be given any treatment after surgery “because they do worse” than those given neoadjuvant therapy, he continued.

While Dr. Ajani noted that the current analysis is underpowered to provide a definitive conclusion, it remains “an important study for Chinese patients.

“Hopefully, it will be well advertised in China, and all the providers switch [to NCCRT]. This could push them to abandon what in the West was considered harmful.”

Dr. Ajani explained the reason neoadjuvant therapy performs better than adjuvant chemoradiotherapy is it “may be mopping up some of the micro metastatic disease, which is difficult to do after surgery,” especially as many patients cannot tolerate postoperative treatment.

“It may be that the majority of patients don’t even get [adjuvant therapy], and those who get it don’t seem to benefit.”

Vishwanath Sathyanarayanan, MD, PhD, Senior Consultant, Professor and Academic Advisor, Department of Medical Oncology, Apollo Cancer Centers, Bangalore, India, agreed that the study reinforces that “NCCRT continues to remain the standard of care in locally advanced resectable esophageal squamous cell carcinoma.”

Consequently, there are “no implications for clinical practice” for providers in the West from these study results, “particularly as NCCRT significantly improves outcomes vs ASCRT with a similar toxicity profile,” he said in an interview.

No funding was declared. Dr. Zhu declared no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

A single infusion of the chimeric antigen receptor (CAR) T-cell therapy ciltacabtagene autoleucel, or cilta-cel (CARVYKTI, Janssen Biotech, Inc.), reduces the risk for death by 45% vs standard-of-care (SoC) therapies in patients with lenalidomide-refractory multiple myeloma, according to the latest data from the phase 3 CARTITUDE-4 study.

“Cilta-cel is the first CAR T-cell therapy to demonstrate an overall survival benefit in multiple myeloma,” María-Victoria Mateos, MD, PhD, said during a presentation of the updated CARTITUDE-4 data at the annual meeting of the International Myeloma Society in late September.

A prespecified overall survival (OS) analysis at a median follow-up of 34 months showed that median OS was not reached in either the cilta-cel or SoC therapy arm (hazard ratio [HR], 0.55). The 30-month OS rates were 76% and 64% in the arms, respectively, said Dr. Mateos, a professor at the University Hospital of Salamanca, Spain.

The significant OS benefit was sustained across all prespecified subgroups, she noted.

The US Food and Drug Administration first approved cilta-cel in 2022 for use after at least four prior lines of therapy in patients with lenalidomide-resistant multiple myeloma based on findings from the CARTITUDE-1 trial. In April 2024, based on progression-free survival (PFS) findings at median follow-up of 16 months in CARTITUDE-4 (HR for progression/death vs SoC, 0.26), that approval was expanded to include patients with lenalidomide-refractory multiple myeloma after one or more prior lines of therapy.

“CARVYKTI demonstrated remarkable efficacy as a personalized, one-time infusion in the earlier treatment of relapsed/refractory multiple myeloma as shown through the CARTITUDE-4 study results,” study coauthor Binod Dhakal, MD, of the Medical College of Wisconsin, in Milwaukee, stated in a press release announcing that expansion. “With this approval, I’m excited for patients who may have the opportunity for a treatment-free period for their multiple myeloma as early as first relapse, with the hope of eliminating the burden of having to be on continuous treatment while living with this challenging disease.”

At the latest analysis, PFS was not reached in the cilta-cel arm and was 11.79 months with SoC, Dr. Mateos said.

The 30-month PFS rates were 59% and 26%, respectively (HR, 0.29), and the PFS benefit was observed across prespecified subgroups.

Patients in the cilta-cel arm also had better complete response rates (77% vs 24%), overall response rates (85% vs 67%), and minimal residual disease-negativity rates (62% vs 18%).

Median duration of response was not reached with cilta-cel and was 18.69 months with SoC, and median time to symptom worsening was not reached vs 34.33 months, respectively.

Safety at the latest update was consistent with prior analyses.

The CARTITUDE findings continue to support the overall benefit-risk profile of cilta-cel vs SoC in patients with lenalidomide-refractory multiple myeloma as early as after the first relapse, Dr. Mateos concluded.

Despite the “compelling efficacy” of cilta-cel, there remains a need for “a safer and equally (if not more) effective CAR-T product” in this setting, Manni Mohyuddin, MD, told this news organization.

“The trial does not change my practice,” said Dr. Mohyuddin, an assistant professor in the multiple myeloma program at Huntsman Cancer Institute, University of Utah, Salt Lake City.

“We must recognize that the control arm [in CARTITUDE-4] isn’t the best available standard of care,” he explained, noting that carfilzomib-containing triplets were not allowed. “Furthermore, overall survival is dependent on access to good therapies upon relapse, and patients in the control arm did not cross over to get cilta-cel at the time of relapse.

“We do not know if overall survival benefit would have been present if the control arm was better and if there was access to better post-protocol therapy.”

Toxicity is also a concern, he said.

“I think of it as high risk-high reward. There was a sevenfold increased incidence of secondary hematological malignancies in the cilta-cel arm compared to standard of care — this is a very concerning signal that dampens my enthusiasm to use this drug early for everyone,” he added.

For example, although Parkinsonism was rare, it generally did not resolve and lasted years, resolving in only 13% of affected patients, with a median time to resolution of 523 days.

“These are horrible odds, and for many patients there may be safer options,” he noted, adding that “cilta-cel is an option I would consider for some relapses (very early relapse while still on multi-agent therapy, high-risk disease), but otherwise I think personally it’s too toxic for most first relapses.”

Dr. Mateos reported relationships with AbbVie, Amgen, BMS, GSK, Janssen, Kite, Oncopeptides, Pfizer, Regeneron, Roche, Sanofi, Stemline Therapeutics, and Takeda. Dr. Mohyuddin had no disclosures.
 

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

A single infusion of the chimeric antigen receptor (CAR) T-cell therapy ciltacabtagene autoleucel, or cilta-cel (CARVYKTI, Janssen Biotech, Inc.), reduces the risk for death by 45% vs standard-of-care (SoC) therapies in patients with lenalidomide-refractory multiple myeloma, according to the latest data from the phase 3 CARTITUDE-4 study.

“Cilta-cel is the first CAR T-cell therapy to demonstrate an overall survival benefit in multiple myeloma,” María-Victoria Mateos, MD, PhD, said during a presentation of the updated CARTITUDE-4 data at the annual meeting of the International Myeloma Society in late September.

A prespecified overall survival (OS) analysis at a median follow-up of 34 months showed that median OS was not reached in either the cilta-cel or SoC therapy arm (hazard ratio [HR], 0.55). The 30-month OS rates were 76% and 64% in the arms, respectively, said Dr. Mateos, a professor at the University Hospital of Salamanca, Spain.

The significant OS benefit was sustained across all prespecified subgroups, she noted.

The US Food and Drug Administration first approved cilta-cel in 2022 for use after at least four prior lines of therapy in patients with lenalidomide-resistant multiple myeloma based on findings from the CARTITUDE-1 trial. In April 2024, based on progression-free survival (PFS) findings at median follow-up of 16 months in CARTITUDE-4 (HR for progression/death vs SoC, 0.26), that approval was expanded to include patients with lenalidomide-refractory multiple myeloma after one or more prior lines of therapy.

“CARVYKTI demonstrated remarkable efficacy as a personalized, one-time infusion in the earlier treatment of relapsed/refractory multiple myeloma as shown through the CARTITUDE-4 study results,” study coauthor Binod Dhakal, MD, of the Medical College of Wisconsin, in Milwaukee, stated in a press release announcing that expansion. “With this approval, I’m excited for patients who may have the opportunity for a treatment-free period for their multiple myeloma as early as first relapse, with the hope of eliminating the burden of having to be on continuous treatment while living with this challenging disease.”

At the latest analysis, PFS was not reached in the cilta-cel arm and was 11.79 months with SoC, Dr. Mateos said.

The 30-month PFS rates were 59% and 26%, respectively (HR, 0.29), and the PFS benefit was observed across prespecified subgroups.

Patients in the cilta-cel arm also had better complete response rates (77% vs 24%), overall response rates (85% vs 67%), and minimal residual disease-negativity rates (62% vs 18%).

Median duration of response was not reached with cilta-cel and was 18.69 months with SoC, and median time to symptom worsening was not reached vs 34.33 months, respectively.

Safety at the latest update was consistent with prior analyses.

The CARTITUDE findings continue to support the overall benefit-risk profile of cilta-cel vs SoC in patients with lenalidomide-refractory multiple myeloma as early as after the first relapse, Dr. Mateos concluded.

Despite the “compelling efficacy” of cilta-cel, there remains a need for “a safer and equally (if not more) effective CAR-T product” in this setting, Manni Mohyuddin, MD, told this news organization.

“The trial does not change my practice,” said Dr. Mohyuddin, an assistant professor in the multiple myeloma program at Huntsman Cancer Institute, University of Utah, Salt Lake City.

“We must recognize that the control arm [in CARTITUDE-4] isn’t the best available standard of care,” he explained, noting that carfilzomib-containing triplets were not allowed. “Furthermore, overall survival is dependent on access to good therapies upon relapse, and patients in the control arm did not cross over to get cilta-cel at the time of relapse.

“We do not know if overall survival benefit would have been present if the control arm was better and if there was access to better post-protocol therapy.”

Toxicity is also a concern, he said.

“I think of it as high risk-high reward. There was a sevenfold increased incidence of secondary hematological malignancies in the cilta-cel arm compared to standard of care — this is a very concerning signal that dampens my enthusiasm to use this drug early for everyone,” he added.

For example, although Parkinsonism was rare, it generally did not resolve and lasted years, resolving in only 13% of affected patients, with a median time to resolution of 523 days.

“These are horrible odds, and for many patients there may be safer options,” he noted, adding that “cilta-cel is an option I would consider for some relapses (very early relapse while still on multi-agent therapy, high-risk disease), but otherwise I think personally it’s too toxic for most first relapses.”

Dr. Mateos reported relationships with AbbVie, Amgen, BMS, GSK, Janssen, Kite, Oncopeptides, Pfizer, Regeneron, Roche, Sanofi, Stemline Therapeutics, and Takeda. Dr. Mohyuddin had no disclosures.
 

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

A single infusion of the chimeric antigen receptor (CAR) T-cell therapy ciltacabtagene autoleucel, or cilta-cel (CARVYKTI, Janssen Biotech, Inc.), reduces the risk for death by 45% vs standard-of-care (SoC) therapies in patients with lenalidomide-refractory multiple myeloma, according to the latest data from the phase 3 CARTITUDE-4 study.

“Cilta-cel is the first CAR T-cell therapy to demonstrate an overall survival benefit in multiple myeloma,” María-Victoria Mateos, MD, PhD, said during a presentation of the updated CARTITUDE-4 data at the annual meeting of the International Myeloma Society in late September.

A prespecified overall survival (OS) analysis at a median follow-up of 34 months showed that median OS was not reached in either the cilta-cel or SoC therapy arm (hazard ratio [HR], 0.55). The 30-month OS rates were 76% and 64% in the arms, respectively, said Dr. Mateos, a professor at the University Hospital of Salamanca, Spain.

The significant OS benefit was sustained across all prespecified subgroups, she noted.

The US Food and Drug Administration first approved cilta-cel in 2022 for use after at least four prior lines of therapy in patients with lenalidomide-resistant multiple myeloma based on findings from the CARTITUDE-1 trial. In April 2024, based on progression-free survival (PFS) findings at median follow-up of 16 months in CARTITUDE-4 (HR for progression/death vs SoC, 0.26), that approval was expanded to include patients with lenalidomide-refractory multiple myeloma after one or more prior lines of therapy.

“CARVYKTI demonstrated remarkable efficacy as a personalized, one-time infusion in the earlier treatment of relapsed/refractory multiple myeloma as shown through the CARTITUDE-4 study results,” study coauthor Binod Dhakal, MD, of the Medical College of Wisconsin, in Milwaukee, stated in a press release announcing that expansion. “With this approval, I’m excited for patients who may have the opportunity for a treatment-free period for their multiple myeloma as early as first relapse, with the hope of eliminating the burden of having to be on continuous treatment while living with this challenging disease.”

At the latest analysis, PFS was not reached in the cilta-cel arm and was 11.79 months with SoC, Dr. Mateos said.

The 30-month PFS rates were 59% and 26%, respectively (HR, 0.29), and the PFS benefit was observed across prespecified subgroups.

Patients in the cilta-cel arm also had better complete response rates (77% vs 24%), overall response rates (85% vs 67%), and minimal residual disease-negativity rates (62% vs 18%).

Median duration of response was not reached with cilta-cel and was 18.69 months with SoC, and median time to symptom worsening was not reached vs 34.33 months, respectively.

Safety at the latest update was consistent with prior analyses.

The CARTITUDE findings continue to support the overall benefit-risk profile of cilta-cel vs SoC in patients with lenalidomide-refractory multiple myeloma as early as after the first relapse, Dr. Mateos concluded.

Despite the “compelling efficacy” of cilta-cel, there remains a need for “a safer and equally (if not more) effective CAR-T product” in this setting, Manni Mohyuddin, MD, told this news organization.

“The trial does not change my practice,” said Dr. Mohyuddin, an assistant professor in the multiple myeloma program at Huntsman Cancer Institute, University of Utah, Salt Lake City.

“We must recognize that the control arm [in CARTITUDE-4] isn’t the best available standard of care,” he explained, noting that carfilzomib-containing triplets were not allowed. “Furthermore, overall survival is dependent on access to good therapies upon relapse, and patients in the control arm did not cross over to get cilta-cel at the time of relapse.

“We do not know if overall survival benefit would have been present if the control arm was better and if there was access to better post-protocol therapy.”

Toxicity is also a concern, he said.

“I think of it as high risk-high reward. There was a sevenfold increased incidence of secondary hematological malignancies in the cilta-cel arm compared to standard of care — this is a very concerning signal that dampens my enthusiasm to use this drug early for everyone,” he added.

For example, although Parkinsonism was rare, it generally did not resolve and lasted years, resolving in only 13% of affected patients, with a median time to resolution of 523 days.

“These are horrible odds, and for many patients there may be safer options,” he noted, adding that “cilta-cel is an option I would consider for some relapses (very early relapse while still on multi-agent therapy, high-risk disease), but otherwise I think personally it’s too toxic for most first relapses.”

Dr. Mateos reported relationships with AbbVie, Amgen, BMS, GSK, Janssen, Kite, Oncopeptides, Pfizer, Regeneron, Roche, Sanofi, Stemline Therapeutics, and Takeda. Dr. Mohyuddin had no disclosures.
 

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

In the treatment of patients with newly diagnosed multiple myeloma (MM) who are not eligible for stem cell transplant or are transplant-deferred, adding subcutaneous daratumumab to bortezomib/lenalidomide/dexamethasone (D-VRd) significantly improves minimal residual disease (MRD) outcomes among patients, the first results from the phase 3 CEPHEUS trial showed.

“CEPHEUS is the first phase 3 daratumumab trial with a primary endpoint of MRD negativity,” said first author Saad Z. Usmani, MD, of Memorial Sloan Kettering Cancer Center, New York City, in presenting late-breaking findings at the annual meeting of the International Myeloma Society in Rio de Janeiro, Brazil in late September.

“We found that adding daratumumab to VRd significantly improved depth and duration of response,” Dr. Usmani said. “[The quadruplet regimen] has the potential to improve clinical outcomes for transplant-ineligible or transplant-deferred patients with newly diagnosed MM who can tolerate bortezomib.”

For newly diagnosed patients with MM who are not eligible for a stem cell transplant, the triplet MAIA regimen of daratumumab, lenalidomide, and dexamethasone is a recommended standard of care, having shown a median overall survival of 7.5 years.

However, for those who are transplant eligible, the PERSEUS regimen of D-VRd followed by daratumumab/lenalidomide maintenance, has shown significant progress-free survival benefits compared with the standard of care.

For the ongoing, multicenter, open-label CEPHEUS study, Dr. Usmani and his colleagues investigated the efficacy of the quadruplet D-VRd regimen compared with VRd alone among newly diagnosed patients who are transplant-ineligible or deferred (not planned as initial therapy).

In the trial, 395 adult patients with transplant-ineligible or transplant-deferred newly diagnosed MM all were initially treated with eight 21-day cycles of VRd, followed by 28-day cycles of lenalidomide until disease progression.

The patients were then randomized to VRd either with (n = 197) or without (n = 198) subcutaneous daratumumab.

Those receiving daratumumab received the subcutaneous therapy weekly in cycles 1 and 2, every 3 weeks in cycles 3-8, and every 4 weeks in cycles 9 or more, until disease progression.

The patients had a median age of 70 years; 28.1% had International Staging System stage III disease, and 13.2% had high-risk cytogenetics.

For the primary endpoint, with a median follow-up of 58.7 months, those in the daratumumab group had a significantly higher rate of being MRD-negative (60.9%) than the VRd-only group (39.4%; odds ratio [OR], 2.37; P < .0001).

Likewise, progression-free survival (PFS) was significantly improved with the daratumumab regimen vs VRd (hazard ratio [HR], 0.57; P = .0005).

A median PFS was not reached for daratumumab plus VRd, compared with 52.6 months for the VRd group, while estimated 54-month PFS rates were 68.1% vs 49.5%, respectively.

A complete response or better was achieved among 81.2% in the daratumumab regimen vs 61.6% with VRd alone (P < .0001) and a sustained rate of MRD-negativity was achieved in 48.7% vs 26.3%, respectively (P < .0001).

There was a trend of overall survival in favor of daratumumab plus VRd (HR, 0.85), with an HR of 0.69 in a sensitivity analysis adjusting for deaths related to COVID-19.

Patients in the daratumumab group had a substantially longer median duration of treatment (56.3 months) than the VRd-only group (34.3 months), with the most common reason for treatment discontinuation being disease progression.

The benefit of daratumumab was generally consistent across the study’s prespecified subgroups, and the relative dose intensity of VRd was not affected by combination with daratumumab.

In terms of safety, treatment-emergent adverse events (TEAEs) were consistent with the known profile of daratumumab and VRd, with grade 5 TEAEs comparable between the two groups after adjusting for treatment exposure.

Quality of life, as measured by EORTC QLQ-C30 score, was improved in both arms over time, with no detriment related to treatment with daratumumab.

Of note, frail patients were not included in the trial. Asked in the Q and A why they were excluded, Dr. Usmani explained that “all of these options are wonderful for our patients, and we are entering a phase where quadruplet therapies will become a mainstay for majority of patients.

“But we have to be careful as we think about not overtreating patients or giving too many side effects of therapies, and that’s why it made sense for us to exclude the frail patients.”

Along those lines, he noted that a key concern in the CEPHEUS trial was tolerance of bortezomib.

“Peripheral sensory neuropathy tends to occur in about half of the patients receiving bortezomib, and about half of that number is grade 2 or higher,” he noted in an interview.

“In some patients, the symptoms do not completely resolve. [Therefore], in transplant-ineligible patients, quadruple regimens may be more relevant for the fit or intermediate-fit patients.”

He concluded that “the CEPHEUS trial compliments the MAIA regimen in supporting a daratumumab-based quadruplet or triplet standard-of-care option across transplant-ineligible patients and those deferring transplant.”

Commenting on the study, Philippe Moreau, MD, who is president of the IMS, noted that “the CEPHEUS study is important because [determining] the best treatment upfront for elderly patients is very important.”

“We need confirmation of the very good results achieved with the IMROZ trial, which showed an estimated 5-year PFS of 63.2%, said Dr. Moreau, professor of clinical hematology and head of the translational research program in hematology and oncology at the University Hospital of Nantes, France.

“If we can achieve the same results, we will have the confirmation that quadruplet is probably here to stay,” Dr. Moreau said.

Dr. Usmani disclosed relationships with Abbvie, Amgen, BioPharma, Bristol Myers Squibb, Celgene, GSK, Janssen, Merck, Pharmacyclics, Sanofi, Seattle Genetics, SkylineOx, and Takeda.
 

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

In the treatment of patients with newly diagnosed multiple myeloma (MM) who are not eligible for stem cell transplant or are transplant-deferred, adding subcutaneous daratumumab to bortezomib/lenalidomide/dexamethasone (D-VRd) significantly improves minimal residual disease (MRD) outcomes among patients, the first results from the phase 3 CEPHEUS trial showed.

“CEPHEUS is the first phase 3 daratumumab trial with a primary endpoint of MRD negativity,” said first author Saad Z. Usmani, MD, of Memorial Sloan Kettering Cancer Center, New York City, in presenting late-breaking findings at the annual meeting of the International Myeloma Society in Rio de Janeiro, Brazil in late September.

“We found that adding daratumumab to VRd significantly improved depth and duration of response,” Dr. Usmani said. “[The quadruplet regimen] has the potential to improve clinical outcomes for transplant-ineligible or transplant-deferred patients with newly diagnosed MM who can tolerate bortezomib.”

For newly diagnosed patients with MM who are not eligible for a stem cell transplant, the triplet MAIA regimen of daratumumab, lenalidomide, and dexamethasone is a recommended standard of care, having shown a median overall survival of 7.5 years.

However, for those who are transplant eligible, the PERSEUS regimen of D-VRd followed by daratumumab/lenalidomide maintenance, has shown significant progress-free survival benefits compared with the standard of care.

For the ongoing, multicenter, open-label CEPHEUS study, Dr. Usmani and his colleagues investigated the efficacy of the quadruplet D-VRd regimen compared with VRd alone among newly diagnosed patients who are transplant-ineligible or deferred (not planned as initial therapy).

In the trial, 395 adult patients with transplant-ineligible or transplant-deferred newly diagnosed MM all were initially treated with eight 21-day cycles of VRd, followed by 28-day cycles of lenalidomide until disease progression.

The patients were then randomized to VRd either with (n = 197) or without (n = 198) subcutaneous daratumumab.

Those receiving daratumumab received the subcutaneous therapy weekly in cycles 1 and 2, every 3 weeks in cycles 3-8, and every 4 weeks in cycles 9 or more, until disease progression.

The patients had a median age of 70 years; 28.1% had International Staging System stage III disease, and 13.2% had high-risk cytogenetics.

For the primary endpoint, with a median follow-up of 58.7 months, those in the daratumumab group had a significantly higher rate of being MRD-negative (60.9%) than the VRd-only group (39.4%; odds ratio [OR], 2.37; P < .0001).

Likewise, progression-free survival (PFS) was significantly improved with the daratumumab regimen vs VRd (hazard ratio [HR], 0.57; P = .0005).

A median PFS was not reached for daratumumab plus VRd, compared with 52.6 months for the VRd group, while estimated 54-month PFS rates were 68.1% vs 49.5%, respectively.

A complete response or better was achieved among 81.2% in the daratumumab regimen vs 61.6% with VRd alone (P < .0001) and a sustained rate of MRD-negativity was achieved in 48.7% vs 26.3%, respectively (P < .0001).

There was a trend of overall survival in favor of daratumumab plus VRd (HR, 0.85), with an HR of 0.69 in a sensitivity analysis adjusting for deaths related to COVID-19.

Patients in the daratumumab group had a substantially longer median duration of treatment (56.3 months) than the VRd-only group (34.3 months), with the most common reason for treatment discontinuation being disease progression.

The benefit of daratumumab was generally consistent across the study’s prespecified subgroups, and the relative dose intensity of VRd was not affected by combination with daratumumab.

In terms of safety, treatment-emergent adverse events (TEAEs) were consistent with the known profile of daratumumab and VRd, with grade 5 TEAEs comparable between the two groups after adjusting for treatment exposure.

Quality of life, as measured by EORTC QLQ-C30 score, was improved in both arms over time, with no detriment related to treatment with daratumumab.

Of note, frail patients were not included in the trial. Asked in the Q and A why they were excluded, Dr. Usmani explained that “all of these options are wonderful for our patients, and we are entering a phase where quadruplet therapies will become a mainstay for majority of patients.

“But we have to be careful as we think about not overtreating patients or giving too many side effects of therapies, and that’s why it made sense for us to exclude the frail patients.”

Along those lines, he noted that a key concern in the CEPHEUS trial was tolerance of bortezomib.

“Peripheral sensory neuropathy tends to occur in about half of the patients receiving bortezomib, and about half of that number is grade 2 or higher,” he noted in an interview.

“In some patients, the symptoms do not completely resolve. [Therefore], in transplant-ineligible patients, quadruple regimens may be more relevant for the fit or intermediate-fit patients.”

He concluded that “the CEPHEUS trial compliments the MAIA regimen in supporting a daratumumab-based quadruplet or triplet standard-of-care option across transplant-ineligible patients and those deferring transplant.”

Commenting on the study, Philippe Moreau, MD, who is president of the IMS, noted that “the CEPHEUS study is important because [determining] the best treatment upfront for elderly patients is very important.”

“We need confirmation of the very good results achieved with the IMROZ trial, which showed an estimated 5-year PFS of 63.2%, said Dr. Moreau, professor of clinical hematology and head of the translational research program in hematology and oncology at the University Hospital of Nantes, France.

“If we can achieve the same results, we will have the confirmation that quadruplet is probably here to stay,” Dr. Moreau said.

Dr. Usmani disclosed relationships with Abbvie, Amgen, BioPharma, Bristol Myers Squibb, Celgene, GSK, Janssen, Merck, Pharmacyclics, Sanofi, Seattle Genetics, SkylineOx, and Takeda.
 

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

In the treatment of patients with newly diagnosed multiple myeloma (MM) who are not eligible for stem cell transplant or are transplant-deferred, adding subcutaneous daratumumab to bortezomib/lenalidomide/dexamethasone (D-VRd) significantly improves minimal residual disease (MRD) outcomes among patients, the first results from the phase 3 CEPHEUS trial showed.

“CEPHEUS is the first phase 3 daratumumab trial with a primary endpoint of MRD negativity,” said first author Saad Z. Usmani, MD, of Memorial Sloan Kettering Cancer Center, New York City, in presenting late-breaking findings at the annual meeting of the International Myeloma Society in Rio de Janeiro, Brazil in late September.

“We found that adding daratumumab to VRd significantly improved depth and duration of response,” Dr. Usmani said. “[The quadruplet regimen] has the potential to improve clinical outcomes for transplant-ineligible or transplant-deferred patients with newly diagnosed MM who can tolerate bortezomib.”

For newly diagnosed patients with MM who are not eligible for a stem cell transplant, the triplet MAIA regimen of daratumumab, lenalidomide, and dexamethasone is a recommended standard of care, having shown a median overall survival of 7.5 years.

However, for those who are transplant eligible, the PERSEUS regimen of D-VRd followed by daratumumab/lenalidomide maintenance, has shown significant progress-free survival benefits compared with the standard of care.

For the ongoing, multicenter, open-label CEPHEUS study, Dr. Usmani and his colleagues investigated the efficacy of the quadruplet D-VRd regimen compared with VRd alone among newly diagnosed patients who are transplant-ineligible or deferred (not planned as initial therapy).

In the trial, 395 adult patients with transplant-ineligible or transplant-deferred newly diagnosed MM all were initially treated with eight 21-day cycles of VRd, followed by 28-day cycles of lenalidomide until disease progression.

The patients were then randomized to VRd either with (n = 197) or without (n = 198) subcutaneous daratumumab.

Those receiving daratumumab received the subcutaneous therapy weekly in cycles 1 and 2, every 3 weeks in cycles 3-8, and every 4 weeks in cycles 9 or more, until disease progression.

The patients had a median age of 70 years; 28.1% had International Staging System stage III disease, and 13.2% had high-risk cytogenetics.

For the primary endpoint, with a median follow-up of 58.7 months, those in the daratumumab group had a significantly higher rate of being MRD-negative (60.9%) than the VRd-only group (39.4%; odds ratio [OR], 2.37; P < .0001).

Likewise, progression-free survival (PFS) was significantly improved with the daratumumab regimen vs VRd (hazard ratio [HR], 0.57; P = .0005).

A median PFS was not reached for daratumumab plus VRd, compared with 52.6 months for the VRd group, while estimated 54-month PFS rates were 68.1% vs 49.5%, respectively.

A complete response or better was achieved among 81.2% in the daratumumab regimen vs 61.6% with VRd alone (P < .0001) and a sustained rate of MRD-negativity was achieved in 48.7% vs 26.3%, respectively (P < .0001).

There was a trend of overall survival in favor of daratumumab plus VRd (HR, 0.85), with an HR of 0.69 in a sensitivity analysis adjusting for deaths related to COVID-19.

Patients in the daratumumab group had a substantially longer median duration of treatment (56.3 months) than the VRd-only group (34.3 months), with the most common reason for treatment discontinuation being disease progression.

The benefit of daratumumab was generally consistent across the study’s prespecified subgroups, and the relative dose intensity of VRd was not affected by combination with daratumumab.

In terms of safety, treatment-emergent adverse events (TEAEs) were consistent with the known profile of daratumumab and VRd, with grade 5 TEAEs comparable between the two groups after adjusting for treatment exposure.

Quality of life, as measured by EORTC QLQ-C30 score, was improved in both arms over time, with no detriment related to treatment with daratumumab.

Of note, frail patients were not included in the trial. Asked in the Q and A why they were excluded, Dr. Usmani explained that “all of these options are wonderful for our patients, and we are entering a phase where quadruplet therapies will become a mainstay for majority of patients.

“But we have to be careful as we think about not overtreating patients or giving too many side effects of therapies, and that’s why it made sense for us to exclude the frail patients.”

Along those lines, he noted that a key concern in the CEPHEUS trial was tolerance of bortezomib.

“Peripheral sensory neuropathy tends to occur in about half of the patients receiving bortezomib, and about half of that number is grade 2 or higher,” he noted in an interview.

“In some patients, the symptoms do not completely resolve. [Therefore], in transplant-ineligible patients, quadruple regimens may be more relevant for the fit or intermediate-fit patients.”

He concluded that “the CEPHEUS trial compliments the MAIA regimen in supporting a daratumumab-based quadruplet or triplet standard-of-care option across transplant-ineligible patients and those deferring transplant.”

Commenting on the study, Philippe Moreau, MD, who is president of the IMS, noted that “the CEPHEUS study is important because [determining] the best treatment upfront for elderly patients is very important.”

“We need confirmation of the very good results achieved with the IMROZ trial, which showed an estimated 5-year PFS of 63.2%, said Dr. Moreau, professor of clinical hematology and head of the translational research program in hematology and oncology at the University Hospital of Nantes, France.

“If we can achieve the same results, we will have the confirmation that quadruplet is probably here to stay,” Dr. Moreau said.

Dr. Usmani disclosed relationships with Abbvie, Amgen, BioPharma, Bristol Myers Squibb, Celgene, GSK, Janssen, Merck, Pharmacyclics, Sanofi, Seattle Genetics, SkylineOx, and Takeda.
 

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

TOPLINE:

Home-based phototherapy for plaque and guttate psoriasis is as effective as office-based phototherapy, according to results of the randomized Light Treatment Effectiveness study.

METHODOLOGY:

• The pragmatic, investigator-initiated, open-label, noninferiority, randomized trial compared the effectiveness of 12 weeks of treatment with narrow-band ultraviolet B phototherapy administered at home (n = 393) vs at the doctor’s office (n = 390).
• Overall, 783 patients with plaque or guttate psoriasis (mean age, 48 years; 48% women) were enrolled at 42 academic and private clinical dermatology practices in the United States from March 1, 2019, to December 4, 2023, and were followed up through June 2024. At baseline, the mean Physician Global Assessment (PGA) and the mean Dermatology Life Quality Index (DLQI) scores were 2.7 and 12.2, respectively.
• The two co-primary endpoints were a PGA score ≤ 1 indicating clear or almost clear skin and a DLQI score ≤ 5.

TAKEAWAY:

• At 12 weeks, a PGA score ≤ 1 was achieved in 32.8% of patients using home-based phototherapy and in 25.6% of those who received office-based phototherapy (P < .001).
• At 12 weeks, a DLQI score ≤ 5 was achieved in 52.4% and 33.6% of home- and office-treated patients, respectively (P < .001).
• Similar benefits were seen across all Fitzpatrick skin types.
• A higher percentage of patients were adherent to home-based (51.4%) vs office-based (15.9%) phototherapy (P < .001).

IN PRACTICE:

“These data support the use of home phototherapy as a first-line treatment option for psoriasis,” and “efforts are needed to make home and office phototherapy more available to patients,” said the study’s lead author.

SOURCE:

Joel M. Gelfand, MD, director of the Psoriasis and Phototherapy Treatment Center at the University of Pennsylvania, Philadelphia, presented the findings at the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis meeting during the annual meeting of the European Academy of Dermatology and Venereology, with simultaneous publication in JAMA Dermatology.

LIMITATIONS:

This was an open-label trial and because of its pragmatic design, outcome data were missing. The cost of the home-based phototherapy equipment used in the study was $6040.88, which was mostly covered by Medicare, but direct costs to patients may have varied depending on their insurance plan.

DISCLOSURES:

The Patient-Centered Outcomes Research Institute funded the study. Daavlin provided and shipped machines for home-based phototherapy to patients at no cost. Dr. Gelfand disclosed serving as a consultant for AbbVie, Artax, Bristol-Myers Squibb, Boehringer Ingelheim, Celldex, and other companies. The full list of author disclosures can be found in the published study.

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

TOPLINE:

Home-based phototherapy for plaque and guttate psoriasis is as effective as office-based phototherapy, according to results of the randomized Light Treatment Effectiveness study.

METHODOLOGY:

• The pragmatic, investigator-initiated, open-label, noninferiority, randomized trial compared the effectiveness of 12 weeks of treatment with narrow-band ultraviolet B phototherapy administered at home (n = 393) vs at the doctor’s office (n = 390).
• Overall, 783 patients with plaque or guttate psoriasis (mean age, 48 years; 48% women) were enrolled at 42 academic and private clinical dermatology practices in the United States from March 1, 2019, to December 4, 2023, and were followed up through June 2024. At baseline, the mean Physician Global Assessment (PGA) and the mean Dermatology Life Quality Index (DLQI) scores were 2.7 and 12.2, respectively.
• The two co-primary endpoints were a PGA score ≤ 1 indicating clear or almost clear skin and a DLQI score ≤ 5.

TAKEAWAY:

• At 12 weeks, a PGA score ≤ 1 was achieved in 32.8% of patients using home-based phototherapy and in 25.6% of those who received office-based phototherapy (P < .001).
• At 12 weeks, a DLQI score ≤ 5 was achieved in 52.4% and 33.6% of home- and office-treated patients, respectively (P < .001).
• Similar benefits were seen across all Fitzpatrick skin types.
• A higher percentage of patients were adherent to home-based (51.4%) vs office-based (15.9%) phototherapy (P < .001).

IN PRACTICE:

“These data support the use of home phototherapy as a first-line treatment option for psoriasis,” and “efforts are needed to make home and office phototherapy more available to patients,” said the study’s lead author.

SOURCE:

Joel M. Gelfand, MD, director of the Psoriasis and Phototherapy Treatment Center at the University of Pennsylvania, Philadelphia, presented the findings at the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis meeting during the annual meeting of the European Academy of Dermatology and Venereology, with simultaneous publication in JAMA Dermatology.

LIMITATIONS:

This was an open-label trial and because of its pragmatic design, outcome data were missing. The cost of the home-based phototherapy equipment used in the study was $6040.88, which was mostly covered by Medicare, but direct costs to patients may have varied depending on their insurance plan.

DISCLOSURES:

The Patient-Centered Outcomes Research Institute funded the study. Daavlin provided and shipped machines for home-based phototherapy to patients at no cost. Dr. Gelfand disclosed serving as a consultant for AbbVie, Artax, Bristol-Myers Squibb, Boehringer Ingelheim, Celldex, and other companies. The full list of author disclosures can be found in the published study.

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

TOPLINE:

Home-based phototherapy for plaque and guttate psoriasis is as effective as office-based phototherapy, according to results of the randomized Light Treatment Effectiveness study.

METHODOLOGY:

• The pragmatic, investigator-initiated, open-label, noninferiority, randomized trial compared the effectiveness of 12 weeks of treatment with narrow-band ultraviolet B phototherapy administered at home (n = 393) vs at the doctor’s office (n = 390).
• Overall, 783 patients with plaque or guttate psoriasis (mean age, 48 years; 48% women) were enrolled at 42 academic and private clinical dermatology practices in the United States from March 1, 2019, to December 4, 2023, and were followed up through June 2024. At baseline, the mean Physician Global Assessment (PGA) and the mean Dermatology Life Quality Index (DLQI) scores were 2.7 and 12.2, respectively.
• The two co-primary endpoints were a PGA score ≤ 1 indicating clear or almost clear skin and a DLQI score ≤ 5.

TAKEAWAY:

• At 12 weeks, a PGA score ≤ 1 was achieved in 32.8% of patients using home-based phototherapy and in 25.6% of those who received office-based phototherapy (P < .001).
• At 12 weeks, a DLQI score ≤ 5 was achieved in 52.4% and 33.6% of home- and office-treated patients, respectively (P < .001).
• Similar benefits were seen across all Fitzpatrick skin types.
• A higher percentage of patients were adherent to home-based (51.4%) vs office-based (15.9%) phototherapy (P < .001).

IN PRACTICE:

“These data support the use of home phototherapy as a first-line treatment option for psoriasis,” and “efforts are needed to make home and office phototherapy more available to patients,” said the study’s lead author.

SOURCE:

Joel M. Gelfand, MD, director of the Psoriasis and Phototherapy Treatment Center at the University of Pennsylvania, Philadelphia, presented the findings at the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis meeting during the annual meeting of the European Academy of Dermatology and Venereology, with simultaneous publication in JAMA Dermatology.

LIMITATIONS:

This was an open-label trial and because of its pragmatic design, outcome data were missing. The cost of the home-based phototherapy equipment used in the study was $6040.88, which was mostly covered by Medicare, but direct costs to patients may have varied depending on their insurance plan.

DISCLOSURES:

The Patient-Centered Outcomes Research Institute funded the study. Daavlin provided and shipped machines for home-based phototherapy to patients at no cost. Dr. Gelfand disclosed serving as a consultant for AbbVie, Artax, Bristol-Myers Squibb, Boehringer Ingelheim, Celldex, and other companies. The full list of author disclosures can be found in the published study.

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