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The American College of Obstetricians and Gynecologists (ACOG) has updated its breast cancer screening guidelines, recommending that individuals at an average risk for breast cancer initiate mammography screening at age 40. This change reflects evolving evidence that starting earlier screening yields greater net benefits in reducing breast cancer mortality, particularly for certain racial groups with higher risk factors.

Breast cancer is the second leading cause of cancer deaths in American women overall and the leading cause of cancer deaths among Black and Hispanic women. Although mammography has long been recognized as a life-saving tool by detecting cancer early, there has been debate on when screening should begin due to concerns about overdiagnosis, false positives, and potential harms such as unnecessary biopsies.

Recent evidence has prompted ACOG to revise its recommendation for individuals assigned female at birth, including cisgender women, transgender men, and nonbinary individuals. This updated guidance includes individuals with dense breast tissue or a family history of breast cancer but excludes those with higher risk factors, such as a personal history of breast cancer or previous high-risk lesion on a breast biopsy, genetic mutations linked to higher cancer risk, or a history of high-dose radiation therapy to their chest at a young age.

Under the new guidelines, routine screening mammography should start at age 40 and can be performed annually or every 2 years, based on an informed, shared decision-making process that considers the benefits and potential harms of frequent screening.

Previously, ACOG recommended initiating screening between ages 40 and 50, depending on individual risk factors and preferences, with screening required by age 50 at the latest. However, several factors, including an increasing incidence of breast cancer in younger women, have influenced the decision to lower the recommended starting age.
 

Increasing Incidence Among Younger Women

Between 2015 and 2019, the incidence of invasive breast cancer in women aged 40-49 years increased by approximately 2% per year.

“There has been a concerning trend of increasing breast cancer diagnoses among women in their 40s, and new data shows that earlier screening could make a significant difference in decreasing breast cancer deaths,” said Eve Zaritsky, MD, FACOG, coauthor of the clinical practice update. “While screening can sometimes cause anxiety for people and even unnecessary follow-up, the benefits of diagnosing breast cancer earlier outweigh those risks enough to warrant starting to get mammograms at age 40.”

Studies commissioned by the US Preventive Services Task Force (USPSTF) show that starting mammography at age 40 provides a greater overall benefit than beginning at age 50. Early screening reduces the number of breast cancer deaths and increases life years gained when weighed against the harms of false positives, overdiagnosis, and benign biopsies.
 

Addressing Health Inequities

The benefits of earlier screening are expected to be particularly significant for Black women, who have disproportionately high mortality rates from breast cancer. Even though Black women have a lower overall incidence of breast cancer than White women, they have a 40% higher 5-year age-adjusted mortality rate from the disease and a 45% increased incidence of invasive breast cancer before age 50. Black women are also more likely to be diagnosed with aggressive subtypes, such as triple-negative breast cancer, which is harder to detect and treat and occurs at younger ages.

Racial disparities in breast cancer outcomes are deeply rooted in inequities in social determinants of health, such as access to care, housing, and environmental conditions. Black women are also less likely to receive timely or comprehensive treatment than White women, which contributes to worse survival rates even after adjusting for socioeconomic factors and insurance status.

“Our updated recommendation addresses important inequities in breast cancer diagnosis, treatment, and death, and we hope that the earlier initiation of mammography screening across the board will have a great net benefit in outcomes for Black women especially, who have been shown to have the poorest outcomes when it comes to breast cancer, in part because of long-standing inequities in social determinants of health,” added coauthor Cherie C. Hill, MD, FACOG.

ACOG’s updated recommendation aligns with that of other leading organizations, including the USPSTF, the National Comprehensive Cancer Network, the American College of Radiology, and the Society of Breast Imaging. This growing consensus among experts is expected to reduce confusion among clinicians and patients regarding when to begin screening, thus improving screening rates in individuals in the 40- to 49-year age group.

Zaritsky and Hill reported no conflicts of interest.
 

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

The American College of Obstetricians and Gynecologists (ACOG) has updated its breast cancer screening guidelines, recommending that individuals at an average risk for breast cancer initiate mammography screening at age 40. This change reflects evolving evidence that starting earlier screening yields greater net benefits in reducing breast cancer mortality, particularly for certain racial groups with higher risk factors.

Breast cancer is the second leading cause of cancer deaths in American women overall and the leading cause of cancer deaths among Black and Hispanic women. Although mammography has long been recognized as a life-saving tool by detecting cancer early, there has been debate on when screening should begin due to concerns about overdiagnosis, false positives, and potential harms such as unnecessary biopsies.

Recent evidence has prompted ACOG to revise its recommendation for individuals assigned female at birth, including cisgender women, transgender men, and nonbinary individuals. This updated guidance includes individuals with dense breast tissue or a family history of breast cancer but excludes those with higher risk factors, such as a personal history of breast cancer or previous high-risk lesion on a breast biopsy, genetic mutations linked to higher cancer risk, or a history of high-dose radiation therapy to their chest at a young age.

Under the new guidelines, routine screening mammography should start at age 40 and can be performed annually or every 2 years, based on an informed, shared decision-making process that considers the benefits and potential harms of frequent screening.

Previously, ACOG recommended initiating screening between ages 40 and 50, depending on individual risk factors and preferences, with screening required by age 50 at the latest. However, several factors, including an increasing incidence of breast cancer in younger women, have influenced the decision to lower the recommended starting age.
 

Increasing Incidence Among Younger Women

Between 2015 and 2019, the incidence of invasive breast cancer in women aged 40-49 years increased by approximately 2% per year.

“There has been a concerning trend of increasing breast cancer diagnoses among women in their 40s, and new data shows that earlier screening could make a significant difference in decreasing breast cancer deaths,” said Eve Zaritsky, MD, FACOG, coauthor of the clinical practice update. “While screening can sometimes cause anxiety for people and even unnecessary follow-up, the benefits of diagnosing breast cancer earlier outweigh those risks enough to warrant starting to get mammograms at age 40.”

Studies commissioned by the US Preventive Services Task Force (USPSTF) show that starting mammography at age 40 provides a greater overall benefit than beginning at age 50. Early screening reduces the number of breast cancer deaths and increases life years gained when weighed against the harms of false positives, overdiagnosis, and benign biopsies.
 

Addressing Health Inequities

The benefits of earlier screening are expected to be particularly significant for Black women, who have disproportionately high mortality rates from breast cancer. Even though Black women have a lower overall incidence of breast cancer than White women, they have a 40% higher 5-year age-adjusted mortality rate from the disease and a 45% increased incidence of invasive breast cancer before age 50. Black women are also more likely to be diagnosed with aggressive subtypes, such as triple-negative breast cancer, which is harder to detect and treat and occurs at younger ages.

Racial disparities in breast cancer outcomes are deeply rooted in inequities in social determinants of health, such as access to care, housing, and environmental conditions. Black women are also less likely to receive timely or comprehensive treatment than White women, which contributes to worse survival rates even after adjusting for socioeconomic factors and insurance status.

“Our updated recommendation addresses important inequities in breast cancer diagnosis, treatment, and death, and we hope that the earlier initiation of mammography screening across the board will have a great net benefit in outcomes for Black women especially, who have been shown to have the poorest outcomes when it comes to breast cancer, in part because of long-standing inequities in social determinants of health,” added coauthor Cherie C. Hill, MD, FACOG.

ACOG’s updated recommendation aligns with that of other leading organizations, including the USPSTF, the National Comprehensive Cancer Network, the American College of Radiology, and the Society of Breast Imaging. This growing consensus among experts is expected to reduce confusion among clinicians and patients regarding when to begin screening, thus improving screening rates in individuals in the 40- to 49-year age group.

Zaritsky and Hill reported no conflicts of interest.
 

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

The American College of Obstetricians and Gynecologists (ACOG) has updated its breast cancer screening guidelines, recommending that individuals at an average risk for breast cancer initiate mammography screening at age 40. This change reflects evolving evidence that starting earlier screening yields greater net benefits in reducing breast cancer mortality, particularly for certain racial groups with higher risk factors.

Breast cancer is the second leading cause of cancer deaths in American women overall and the leading cause of cancer deaths among Black and Hispanic women. Although mammography has long been recognized as a life-saving tool by detecting cancer early, there has been debate on when screening should begin due to concerns about overdiagnosis, false positives, and potential harms such as unnecessary biopsies.

Recent evidence has prompted ACOG to revise its recommendation for individuals assigned female at birth, including cisgender women, transgender men, and nonbinary individuals. This updated guidance includes individuals with dense breast tissue or a family history of breast cancer but excludes those with higher risk factors, such as a personal history of breast cancer or previous high-risk lesion on a breast biopsy, genetic mutations linked to higher cancer risk, or a history of high-dose radiation therapy to their chest at a young age.

Under the new guidelines, routine screening mammography should start at age 40 and can be performed annually or every 2 years, based on an informed, shared decision-making process that considers the benefits and potential harms of frequent screening.

Previously, ACOG recommended initiating screening between ages 40 and 50, depending on individual risk factors and preferences, with screening required by age 50 at the latest. However, several factors, including an increasing incidence of breast cancer in younger women, have influenced the decision to lower the recommended starting age.
 

Increasing Incidence Among Younger Women

Between 2015 and 2019, the incidence of invasive breast cancer in women aged 40-49 years increased by approximately 2% per year.

“There has been a concerning trend of increasing breast cancer diagnoses among women in their 40s, and new data shows that earlier screening could make a significant difference in decreasing breast cancer deaths,” said Eve Zaritsky, MD, FACOG, coauthor of the clinical practice update. “While screening can sometimes cause anxiety for people and even unnecessary follow-up, the benefits of diagnosing breast cancer earlier outweigh those risks enough to warrant starting to get mammograms at age 40.”

Studies commissioned by the US Preventive Services Task Force (USPSTF) show that starting mammography at age 40 provides a greater overall benefit than beginning at age 50. Early screening reduces the number of breast cancer deaths and increases life years gained when weighed against the harms of false positives, overdiagnosis, and benign biopsies.
 

Addressing Health Inequities

The benefits of earlier screening are expected to be particularly significant for Black women, who have disproportionately high mortality rates from breast cancer. Even though Black women have a lower overall incidence of breast cancer than White women, they have a 40% higher 5-year age-adjusted mortality rate from the disease and a 45% increased incidence of invasive breast cancer before age 50. Black women are also more likely to be diagnosed with aggressive subtypes, such as triple-negative breast cancer, which is harder to detect and treat and occurs at younger ages.

Racial disparities in breast cancer outcomes are deeply rooted in inequities in social determinants of health, such as access to care, housing, and environmental conditions. Black women are also less likely to receive timely or comprehensive treatment than White women, which contributes to worse survival rates even after adjusting for socioeconomic factors and insurance status.

“Our updated recommendation addresses important inequities in breast cancer diagnosis, treatment, and death, and we hope that the earlier initiation of mammography screening across the board will have a great net benefit in outcomes for Black women especially, who have been shown to have the poorest outcomes when it comes to breast cancer, in part because of long-standing inequities in social determinants of health,” added coauthor Cherie C. Hill, MD, FACOG.

ACOG’s updated recommendation aligns with that of other leading organizations, including the USPSTF, the National Comprehensive Cancer Network, the American College of Radiology, and the Society of Breast Imaging. This growing consensus among experts is expected to reduce confusion among clinicians and patients regarding when to begin screening, thus improving screening rates in individuals in the 40- to 49-year age group.

Zaritsky and Hill reported no conflicts of interest.
 

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

Cancer is becoming more common in younger generations. Data show that people under 50 are experiencing higher rates of cancer than any generation before them. As a genetic counselor, I hoped these upward trends in early-onset malignancies would slow with a better understanding of risk factors and prevention strategies. Unfortunately, the opposite is happening. Recent findings from the American Cancer Society reveal that the incidence of at least 17 of 34 cancer types is rising among GenX and Millennials. 

These statistics are alarming. I appreciate how easy it is for patients to get lost in the headlines about cancer, which may shape how they approach their healthcare. Each year, millions of Americans miss critical cancer screenings, with many citing fear of a positive test result as a leading reason. Others believe, despite the statistics, that cancer is not something they need to worry about until they are older. And then, of course, getting screened is not as easy as it should be. 

In my work, I meet with people from both younger and older generations who have either faced cancer themselves or witnessed a loved one experience the disease. One of the most common sentiments I hear from these patients is the desire to catch cancer earlier. My answer is always this: The first and most important step everyone can take is understanding their risk. 

For some, knowing they are at increased risk for cancer means starting screenings earlier — sometimes as early as age 25 — or getting screened with a more sensitive test. 

This proactive approach is the right one. Early detection can dramatically increase survival rates, sometimes by up to eightfold, depending on the type of cancer. It also significantly reduces the burden of total and cancer-specific healthcare costs. While screening may carry some potential risks, clinicians can minimize these risks by adhering to evidence-based guidelines, such as those from the American Cancer Society, and ensuring there is appropriate discussion of treatment options when a diagnosis is made.
 

Normalizing Cancer Risk Assessment and Screening 

A detailed cancer risk assessment and education about signs and symptoms should be part of every preventive care visit, regardless of someone’s age. Further, that cancer risk assessment should lead to clear recommendations and support for taking the next steps. 

This is where care advocacy and patient navigation come in. Care advocacy can improve outcomes at every stage of the cancer journey, from increasing screening rates to improving quality of life for survivors. I’ve seen first-hand how care advocates help patients overcome hurdles like long wait times for appointments they need, making both screening and diagnostic care easier to access. 

Now, with the finalization of a new rule from the Centers for Medicare & Medicaid Services, providers can bill for oncology navigation services that occur under their supervision. This formal recognition of care navigation affirms the value of these services not just clinically but financially as well. It will be through methods like care navigation, targeted outreach, and engaging educational resources — built into and covered by health plans — that patients will feel more in control over their health and have tools to help minimize the effects of cancer on the rest of their lives. 

These services benefit healthcare providers as well. Care navigation supports clinical care teams, from primary care providers to oncologists, by ensuring patients are seen before their cancer progresses to a more advanced stage. And even if patients follow screening recommendations for the rest of their lives and never get a positive result, they’ve still gained something invaluable: peace of mind, knowing they’ve taken an active role in their health. 
 

Fighting Fear With Routine

Treating cancer as a normal part of young people’s healthcare means helping them envision the disease as a condition that can be treated, much like a diagnosis of diabetes or high cholesterol. This mindset shift means quickly following up on a concerning symptom or screening result and reducing the time to start treatment if needed. And with treatment options and success rates for some cancers being better than ever, survivorship support must be built into every treatment plan from the start. Before treatment begins, healthcare providers should make time to talk about sometimes-overlooked key topics, such as reproductive options for people whose fertility may be affected by their cancer treatment, about plans for returning to work during or after treatment, and finding the right mental health support. 

Where we can’t prevent cancer, both primary care providers and oncologists can work together to help patients receive the right diagnosis and treatment as quickly as possible. Knowing insurance coverage has a direct effect on how early cancer is caught, for example, younger people need support in understanding and accessing benefits and resources that may be available through their existing healthcare channels, like some employer-sponsored health plans. Even if getting treated for cancer is inevitable for some, taking immediate action to get screened when it’s appropriate is the best thing we can do to lessen the impact of these rising cancer incidences across the country. At the end of the day, being afraid of cancer doesn’t decrease the chances of getting sick or dying from it. Proactive screening and early detection do. 
 

Brockman, Genetic Counselor, Color Health, Buffalo, New York, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

Cancer is becoming more common in younger generations. Data show that people under 50 are experiencing higher rates of cancer than any generation before them. As a genetic counselor, I hoped these upward trends in early-onset malignancies would slow with a better understanding of risk factors and prevention strategies. Unfortunately, the opposite is happening. Recent findings from the American Cancer Society reveal that the incidence of at least 17 of 34 cancer types is rising among GenX and Millennials. 

These statistics are alarming. I appreciate how easy it is for patients to get lost in the headlines about cancer, which may shape how they approach their healthcare. Each year, millions of Americans miss critical cancer screenings, with many citing fear of a positive test result as a leading reason. Others believe, despite the statistics, that cancer is not something they need to worry about until they are older. And then, of course, getting screened is not as easy as it should be. 

In my work, I meet with people from both younger and older generations who have either faced cancer themselves or witnessed a loved one experience the disease. One of the most common sentiments I hear from these patients is the desire to catch cancer earlier. My answer is always this: The first and most important step everyone can take is understanding their risk. 

For some, knowing they are at increased risk for cancer means starting screenings earlier — sometimes as early as age 25 — or getting screened with a more sensitive test. 

This proactive approach is the right one. Early detection can dramatically increase survival rates, sometimes by up to eightfold, depending on the type of cancer. It also significantly reduces the burden of total and cancer-specific healthcare costs. While screening may carry some potential risks, clinicians can minimize these risks by adhering to evidence-based guidelines, such as those from the American Cancer Society, and ensuring there is appropriate discussion of treatment options when a diagnosis is made.
 

Normalizing Cancer Risk Assessment and Screening 

A detailed cancer risk assessment and education about signs and symptoms should be part of every preventive care visit, regardless of someone’s age. Further, that cancer risk assessment should lead to clear recommendations and support for taking the next steps. 

This is where care advocacy and patient navigation come in. Care advocacy can improve outcomes at every stage of the cancer journey, from increasing screening rates to improving quality of life for survivors. I’ve seen first-hand how care advocates help patients overcome hurdles like long wait times for appointments they need, making both screening and diagnostic care easier to access. 

Now, with the finalization of a new rule from the Centers for Medicare & Medicaid Services, providers can bill for oncology navigation services that occur under their supervision. This formal recognition of care navigation affirms the value of these services not just clinically but financially as well. It will be through methods like care navigation, targeted outreach, and engaging educational resources — built into and covered by health plans — that patients will feel more in control over their health and have tools to help minimize the effects of cancer on the rest of their lives. 

These services benefit healthcare providers as well. Care navigation supports clinical care teams, from primary care providers to oncologists, by ensuring patients are seen before their cancer progresses to a more advanced stage. And even if patients follow screening recommendations for the rest of their lives and never get a positive result, they’ve still gained something invaluable: peace of mind, knowing they’ve taken an active role in their health. 
 

Fighting Fear With Routine

Treating cancer as a normal part of young people’s healthcare means helping them envision the disease as a condition that can be treated, much like a diagnosis of diabetes or high cholesterol. This mindset shift means quickly following up on a concerning symptom or screening result and reducing the time to start treatment if needed. And with treatment options and success rates for some cancers being better than ever, survivorship support must be built into every treatment plan from the start. Before treatment begins, healthcare providers should make time to talk about sometimes-overlooked key topics, such as reproductive options for people whose fertility may be affected by their cancer treatment, about plans for returning to work during or after treatment, and finding the right mental health support. 

Where we can’t prevent cancer, both primary care providers and oncologists can work together to help patients receive the right diagnosis and treatment as quickly as possible. Knowing insurance coverage has a direct effect on how early cancer is caught, for example, younger people need support in understanding and accessing benefits and resources that may be available through their existing healthcare channels, like some employer-sponsored health plans. Even if getting treated for cancer is inevitable for some, taking immediate action to get screened when it’s appropriate is the best thing we can do to lessen the impact of these rising cancer incidences across the country. At the end of the day, being afraid of cancer doesn’t decrease the chances of getting sick or dying from it. Proactive screening and early detection do. 
 

Brockman, Genetic Counselor, Color Health, Buffalo, New York, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

Cancer is becoming more common in younger generations. Data show that people under 50 are experiencing higher rates of cancer than any generation before them. As a genetic counselor, I hoped these upward trends in early-onset malignancies would slow with a better understanding of risk factors and prevention strategies. Unfortunately, the opposite is happening. Recent findings from the American Cancer Society reveal that the incidence of at least 17 of 34 cancer types is rising among GenX and Millennials. 

These statistics are alarming. I appreciate how easy it is for patients to get lost in the headlines about cancer, which may shape how they approach their healthcare. Each year, millions of Americans miss critical cancer screenings, with many citing fear of a positive test result as a leading reason. Others believe, despite the statistics, that cancer is not something they need to worry about until they are older. And then, of course, getting screened is not as easy as it should be. 

In my work, I meet with people from both younger and older generations who have either faced cancer themselves or witnessed a loved one experience the disease. One of the most common sentiments I hear from these patients is the desire to catch cancer earlier. My answer is always this: The first and most important step everyone can take is understanding their risk. 

For some, knowing they are at increased risk for cancer means starting screenings earlier — sometimes as early as age 25 — or getting screened with a more sensitive test. 

This proactive approach is the right one. Early detection can dramatically increase survival rates, sometimes by up to eightfold, depending on the type of cancer. It also significantly reduces the burden of total and cancer-specific healthcare costs. While screening may carry some potential risks, clinicians can minimize these risks by adhering to evidence-based guidelines, such as those from the American Cancer Society, and ensuring there is appropriate discussion of treatment options when a diagnosis is made.
 

Normalizing Cancer Risk Assessment and Screening 

A detailed cancer risk assessment and education about signs and symptoms should be part of every preventive care visit, regardless of someone’s age. Further, that cancer risk assessment should lead to clear recommendations and support for taking the next steps. 

This is where care advocacy and patient navigation come in. Care advocacy can improve outcomes at every stage of the cancer journey, from increasing screening rates to improving quality of life for survivors. I’ve seen first-hand how care advocates help patients overcome hurdles like long wait times for appointments they need, making both screening and diagnostic care easier to access. 

Now, with the finalization of a new rule from the Centers for Medicare & Medicaid Services, providers can bill for oncology navigation services that occur under their supervision. This formal recognition of care navigation affirms the value of these services not just clinically but financially as well. It will be through methods like care navigation, targeted outreach, and engaging educational resources — built into and covered by health plans — that patients will feel more in control over their health and have tools to help minimize the effects of cancer on the rest of their lives. 

These services benefit healthcare providers as well. Care navigation supports clinical care teams, from primary care providers to oncologists, by ensuring patients are seen before their cancer progresses to a more advanced stage. And even if patients follow screening recommendations for the rest of their lives and never get a positive result, they’ve still gained something invaluable: peace of mind, knowing they’ve taken an active role in their health. 
 

Fighting Fear With Routine

Treating cancer as a normal part of young people’s healthcare means helping them envision the disease as a condition that can be treated, much like a diagnosis of diabetes or high cholesterol. This mindset shift means quickly following up on a concerning symptom or screening result and reducing the time to start treatment if needed. And with treatment options and success rates for some cancers being better than ever, survivorship support must be built into every treatment plan from the start. Before treatment begins, healthcare providers should make time to talk about sometimes-overlooked key topics, such as reproductive options for people whose fertility may be affected by their cancer treatment, about plans for returning to work during or after treatment, and finding the right mental health support. 

Where we can’t prevent cancer, both primary care providers and oncologists can work together to help patients receive the right diagnosis and treatment as quickly as possible. Knowing insurance coverage has a direct effect on how early cancer is caught, for example, younger people need support in understanding and accessing benefits and resources that may be available through their existing healthcare channels, like some employer-sponsored health plans. Even if getting treated for cancer is inevitable for some, taking immediate action to get screened when it’s appropriate is the best thing we can do to lessen the impact of these rising cancer incidences across the country. At the end of the day, being afraid of cancer doesn’t decrease the chances of getting sick or dying from it. Proactive screening and early detection do. 
 

Brockman, Genetic Counselor, Color Health, Buffalo, New York, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

The US Department of Veterans Affairs (VA) will conduct a scientific assessment to find out in whether kidney cancer should be considered a presumptive service-connected condition for veterans exposed to per- and polyfluoroalkyl substances (PFAs). This assessment is the first step in the VA presumptive condition investigative process, which could allow exposed veterans who were exposed to PFAs during their service to access more VA services.

A class of more than 12,000 chemicals, PFAs have been used in the military since the early 1970s in many items, including military-grade firefighting foam. Studies have already suggested links between the so-called forever chemicals and cancer, particularly kidney cancer.

The US Department of Defense (DoD) is assessing contamination at hundreds of sites, while the National Defense Authorization Act in Fiscal Year 2020 mandated that DoD stop using those foams starting in October and remove all stocks from active and former installations and equipment. That may not happen until next year, though, because the DoD has requested a waiver through October 2025 and may extend it through 2026.

When a condition is considered presumptive, eligible veterans do not need to prove their service caused their disease to receive benefits. As part of the Biden Administration’s efforts to expand benefits and services for toxin-exposed veterans and their families, the VA expedited health care and benefits eligibility under the PACT Act by several years—including extending presumptions for head cancer, neck cancer, gastrointestinal cancer, reproductive cancer, lymphoma, pancreatic cancer, kidney cancer, melanoma, and hypertension for Vietnam era veterans. The VA has also extended presumptions for > 300 new conditions, most recently for male breast cancer, urethral cancer, and cancer of the paraurethral glands.

Whether a condition is an established presumptive condition or not, the VA will consider claims on a case-by-case basis and can grant disability compensation benefits if sufficient evidence of service connection is found. “[M]ake no mistake: Veterans should not wait for the outcome of this review to apply for the benefits and care they deserve,” VA Secretary Denis McDonough said in a release.  “If you’re a veteran and believe your military service has negatively impacted your health, we encourage you to apply for VA care and benefits today.”

The public has 30 days to comment on the proposed scientific assessment between PFAs exposure and kidney cancer via the Federal Register. The VA is set to host a listening session on Nov. 19, 2024, to allow individuals to share research and input. Interested individuals may register to participate. The public may also comment via either forum on other conditions that would benefit from review for potential service-connection.

The US Department of Veterans Affairs (VA) will conduct a scientific assessment to find out in whether kidney cancer should be considered a presumptive service-connected condition for veterans exposed to per- and polyfluoroalkyl substances (PFAs). This assessment is the first step in the VA presumptive condition investigative process, which could allow exposed veterans who were exposed to PFAs during their service to access more VA services.

A class of more than 12,000 chemicals, PFAs have been used in the military since the early 1970s in many items, including military-grade firefighting foam. Studies have already suggested links between the so-called forever chemicals and cancer, particularly kidney cancer.

The US Department of Defense (DoD) is assessing contamination at hundreds of sites, while the National Defense Authorization Act in Fiscal Year 2020 mandated that DoD stop using those foams starting in October and remove all stocks from active and former installations and equipment. That may not happen until next year, though, because the DoD has requested a waiver through October 2025 and may extend it through 2026.

When a condition is considered presumptive, eligible veterans do not need to prove their service caused their disease to receive benefits. As part of the Biden Administration’s efforts to expand benefits and services for toxin-exposed veterans and their families, the VA expedited health care and benefits eligibility under the PACT Act by several years—including extending presumptions for head cancer, neck cancer, gastrointestinal cancer, reproductive cancer, lymphoma, pancreatic cancer, kidney cancer, melanoma, and hypertension for Vietnam era veterans. The VA has also extended presumptions for > 300 new conditions, most recently for male breast cancer, urethral cancer, and cancer of the paraurethral glands.

Whether a condition is an established presumptive condition or not, the VA will consider claims on a case-by-case basis and can grant disability compensation benefits if sufficient evidence of service connection is found. “[M]ake no mistake: Veterans should not wait for the outcome of this review to apply for the benefits and care they deserve,” VA Secretary Denis McDonough said in a release.  “If you’re a veteran and believe your military service has negatively impacted your health, we encourage you to apply for VA care and benefits today.”

The public has 30 days to comment on the proposed scientific assessment between PFAs exposure and kidney cancer via the Federal Register. The VA is set to host a listening session on Nov. 19, 2024, to allow individuals to share research and input. Interested individuals may register to participate. The public may also comment via either forum on other conditions that would benefit from review for potential service-connection.

The US Department of Veterans Affairs (VA) will conduct a scientific assessment to find out in whether kidney cancer should be considered a presumptive service-connected condition for veterans exposed to per- and polyfluoroalkyl substances (PFAs). This assessment is the first step in the VA presumptive condition investigative process, which could allow exposed veterans who were exposed to PFAs during their service to access more VA services.

A class of more than 12,000 chemicals, PFAs have been used in the military since the early 1970s in many items, including military-grade firefighting foam. Studies have already suggested links between the so-called forever chemicals and cancer, particularly kidney cancer.

The US Department of Defense (DoD) is assessing contamination at hundreds of sites, while the National Defense Authorization Act in Fiscal Year 2020 mandated that DoD stop using those foams starting in October and remove all stocks from active and former installations and equipment. That may not happen until next year, though, because the DoD has requested a waiver through October 2025 and may extend it through 2026.

When a condition is considered presumptive, eligible veterans do not need to prove their service caused their disease to receive benefits. As part of the Biden Administration’s efforts to expand benefits and services for toxin-exposed veterans and their families, the VA expedited health care and benefits eligibility under the PACT Act by several years—including extending presumptions for head cancer, neck cancer, gastrointestinal cancer, reproductive cancer, lymphoma, pancreatic cancer, kidney cancer, melanoma, and hypertension for Vietnam era veterans. The VA has also extended presumptions for > 300 new conditions, most recently for male breast cancer, urethral cancer, and cancer of the paraurethral glands.

Whether a condition is an established presumptive condition or not, the VA will consider claims on a case-by-case basis and can grant disability compensation benefits if sufficient evidence of service connection is found. “[M]ake no mistake: Veterans should not wait for the outcome of this review to apply for the benefits and care they deserve,” VA Secretary Denis McDonough said in a release.  “If you’re a veteran and believe your military service has negatively impacted your health, we encourage you to apply for VA care and benefits today.”

The public has 30 days to comment on the proposed scientific assessment between PFAs exposure and kidney cancer via the Federal Register. The VA is set to host a listening session on Nov. 19, 2024, to allow individuals to share research and input. Interested individuals may register to participate. The public may also comment via either forum on other conditions that would benefit from review for potential service-connection.

The US Department of Veterans Affairs (VA) has announced that tele-emergency care (tele-EC) is now available nationwide. According to the VA, the expansion has already helped > 61,000 callers with a 59.4% case resolution rate, meaning veterans’ needs were resolved without them having to travel to urgent care or an emergency department.

Tele-EC does not replace the need for in-person emergency evaluation, but offers quick, virtual triage assessments for veterans in rural areas or those with mobility and transportation challenges when in-person immediate care can be difficult to access. The program is a part of VA Health Connect, which connects the caller to a clinical triage nurse, who connects the veteran to tele-emergency care when clinically appropriate. Tele-EC practitioners evaluate the veteran over the phone or on video and recommend treatment or follow-up, including in-person care if needed. In life-threatening emergencies, the clinical triage nurse will call 911 and stay on the phone with the veteran until help arrives. The VA however, says the best step for a veteran experiencing a life-threatening emergency is to immediately contact 911 as opposed to seeking support via tele-EC.

The program can save time not only through on-the-spot evaluation, but by avoiding drive and wait times. “Sometimes, you’re not sure whether what you’re experiencing is a minor emergency or not — and tele-emergency care can help you resolve those questions,” VA Under Secretary for Health Shereef Elnahal, MD, says. “Veterans can get immediate, virtual triage with a VA medical provider who has direct access to their medical records. This avoids having to potentially drive to the nearest emergency department and wait to be evaluated, if appropriate.”

Veterans enrolled in VA health care can now access tele-EC nationwide by calling VA Health Connect and through the VA Health Chat app. Veterans can find their local VA Health Connect number by searching for their facility.

The US Department of Veterans Affairs (VA) has announced that tele-emergency care (tele-EC) is now available nationwide. According to the VA, the expansion has already helped > 61,000 callers with a 59.4% case resolution rate, meaning veterans’ needs were resolved without them having to travel to urgent care or an emergency department.

Tele-EC does not replace the need for in-person emergency evaluation, but offers quick, virtual triage assessments for veterans in rural areas or those with mobility and transportation challenges when in-person immediate care can be difficult to access. The program is a part of VA Health Connect, which connects the caller to a clinical triage nurse, who connects the veteran to tele-emergency care when clinically appropriate. Tele-EC practitioners evaluate the veteran over the phone or on video and recommend treatment or follow-up, including in-person care if needed. In life-threatening emergencies, the clinical triage nurse will call 911 and stay on the phone with the veteran until help arrives. The VA however, says the best step for a veteran experiencing a life-threatening emergency is to immediately contact 911 as opposed to seeking support via tele-EC.

The program can save time not only through on-the-spot evaluation, but by avoiding drive and wait times. “Sometimes, you’re not sure whether what you’re experiencing is a minor emergency or not — and tele-emergency care can help you resolve those questions,” VA Under Secretary for Health Shereef Elnahal, MD, says. “Veterans can get immediate, virtual triage with a VA medical provider who has direct access to their medical records. This avoids having to potentially drive to the nearest emergency department and wait to be evaluated, if appropriate.”

Veterans enrolled in VA health care can now access tele-EC nationwide by calling VA Health Connect and through the VA Health Chat app. Veterans can find their local VA Health Connect number by searching for their facility.

The US Department of Veterans Affairs (VA) has announced that tele-emergency care (tele-EC) is now available nationwide. According to the VA, the expansion has already helped > 61,000 callers with a 59.4% case resolution rate, meaning veterans’ needs were resolved without them having to travel to urgent care or an emergency department.

Tele-EC does not replace the need for in-person emergency evaluation, but offers quick, virtual triage assessments for veterans in rural areas or those with mobility and transportation challenges when in-person immediate care can be difficult to access. The program is a part of VA Health Connect, which connects the caller to a clinical triage nurse, who connects the veteran to tele-emergency care when clinically appropriate. Tele-EC practitioners evaluate the veteran over the phone or on video and recommend treatment or follow-up, including in-person care if needed. In life-threatening emergencies, the clinical triage nurse will call 911 and stay on the phone with the veteran until help arrives. The VA however, says the best step for a veteran experiencing a life-threatening emergency is to immediately contact 911 as opposed to seeking support via tele-EC.

The program can save time not only through on-the-spot evaluation, but by avoiding drive and wait times. “Sometimes, you’re not sure whether what you’re experiencing is a minor emergency or not — and tele-emergency care can help you resolve those questions,” VA Under Secretary for Health Shereef Elnahal, MD, says. “Veterans can get immediate, virtual triage with a VA medical provider who has direct access to their medical records. This avoids having to potentially drive to the nearest emergency department and wait to be evaluated, if appropriate.”

Veterans enrolled in VA health care can now access tele-EC nationwide by calling VA Health Connect and through the VA Health Chat app. Veterans can find their local VA Health Connect number by searching for their facility.

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.

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.

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.

The global withdrawal of voxelotor (Oxbryta, Pfizer) has left clinicians who treat sickle cell disease (SCD) with the urgent task of reaching patients taking the medicine, while trying to understand why it was taken off the market.

The National Alliance of Sickle Cell Centers issued a statement urging patients not to stop voxelotor abruptly. Instead, they should work out plans with their physicians and medical teams for weaning plans.

“Don’t lose faith. This a step backward, but we will stay on the path to better outcomes for everyone,” said the alliance in a statement to patients and clinicians.

On September 25, Pfizer said it would withdraw all lots of voxelotor in all markets where it is approved. The New York–based drugmaker also said it was discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The cause was data that suggested “an imbalance in vaso-occlusive crises and fatal events which require further assessment.”

Pfizer told this news organization in an email exchange that it is focused on analyzing the data and will share updates in the future about presenting or publishing on this issue.

The withdrawal came amid increased scrutiny of the drug by the European Medicines Agency (EMA). The EMA in July began a review of voxelotor after data from a clinical trial showed that a higher number of deaths occurred with the drug than with placebo and another trial showed the total number of deaths was higher than anticipated.

On September 26, the EMA’s human medicines committee recommended suspending the marketing authorization of voxelotor, citing new safety data that emerged during the review. The drug had received marketing authorization for the European Union in 2022, the agency said.

The US Food and Drug Administration (FDA), which first cleared voxelotor for sale in 2019, also said it has been conducting a safety review of the drug. The agency continues to examine post-marketing clinical trial data for voxelotor, the real-world registry studies, and data from the FDA Adverse Event Reporting System. At the conclusion of this review, the FDA will communicate any additional findings, if necessary, the agency said.

The FDA said it appeared that more deaths and a higher rate of vaso-occlusive crisis occurred in patients taking voxelotor vs placebo in post-marketing clinical trials.

“Pfizer also observed a higher rate of vaso-occlusive crisis in patients with sickle cell disease receiving Oxbryta in two real-world registry studies,” the FDA said. “Based on the totality of clinical data, Pfizer has determined the benefit of Oxbryta does not outweigh the risk.”
 

Gene Therapy, Tried-and-True Hydroxyurea (HU)

As a field, SCD has drawn more interest in recent years, with significant gains made lately in cutting-edge projects.

The FDA in December approved two gene-editing treatments for patients aged 12 years or older. These are considered “milestone treatments” for a debilitating and potentially life-threatening blood disorder that affects about 100,000 people in the United States. Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics) is the first to use the gene-editing tool CRISPR. And lovotibeglogene autotemcel (Lyfgenia, bluebird bio) uses a different gene-editing tool called a lentiviral vector.

These advances have been covered widely by the news media but are not expected to be widely available, with the cost of these extensive treatments estimated around $2-$3 million per patient.

“Gene therapy is amazing in that it can offer a cure, but it’s very expensive and not all patients are suitable for it. Some have so much existing organ damage that it’s not an option for them,” said John Wood, MD, PhD, director of cardiovascular MRI at Children’s Hospital Los Angeles, Los Angeles, who does research on SCD.

“So it really is a great treatment for a very few people,” he said in an interview.

The mainstay of treatment for SCD remains a drug that Lydia Pecker, MD, a pediatric hematologist at Johns Hopkins University in Baltimore, describes as the “first, oldest, and best”: HU.

The FDA approved this in 1998 for use in SCD. It reduces the frequency of painful crises and acute chest syndrome and other complications of SCD that otherwise could be serious or even lethal, Pecker said.

“Older doctors can tell you that what they experienced with sickle cell disease in the hospitals has been completely transformed because of the high uptake of the drug,” she said, adding that it made a “profound” change. “We just don’t have data for any other agent that’s quite like that.”

Voxelotor had been a good second drug to add for some patients, in addition to HU and blood transfusions, Dr. Pecker noted. It was a first-line drug for those for whom transfusion and HU were not options, which constitutes a relatively small number of patients, she said.

“So we have, in the last 5 years, felt more hopeful because we had something else to offer,” she said.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology at Children’s Hospital of Philadelphia in Pennsylvania, said in an interview that her organization also had patients who appeared to benefit from voxelotor, some of whom had been participants in clinical trials.

Dr. Thompson, who has been a top researcher involved in the study of gene therapy, urged the need for companies to keep seeking to expand the options for people with SCD, even after the setback with voxelotor.

“I hope that there’s an appreciation for the need for continued investment in this very serious condition, for which there are insufficient options for treatments,” Dr. Thompson said. “So ongoing investment is really needed if we expect to make progress.”

Dr. Pecker disclosed ties with Novartis, Afimmune, the American Society of Hematology, and the National Institutes of Health. Thompson reported relationships with bluebird bio, Beam, Editas, Novartis, and Novo Nordisk.
 

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

The global withdrawal of voxelotor (Oxbryta, Pfizer) has left clinicians who treat sickle cell disease (SCD) with the urgent task of reaching patients taking the medicine, while trying to understand why it was taken off the market.

The National Alliance of Sickle Cell Centers issued a statement urging patients not to stop voxelotor abruptly. Instead, they should work out plans with their physicians and medical teams for weaning plans.

“Don’t lose faith. This a step backward, but we will stay on the path to better outcomes for everyone,” said the alliance in a statement to patients and clinicians.

On September 25, Pfizer said it would withdraw all lots of voxelotor in all markets where it is approved. The New York–based drugmaker also said it was discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The cause was data that suggested “an imbalance in vaso-occlusive crises and fatal events which require further assessment.”

Pfizer told this news organization in an email exchange that it is focused on analyzing the data and will share updates in the future about presenting or publishing on this issue.

The withdrawal came amid increased scrutiny of the drug by the European Medicines Agency (EMA). The EMA in July began a review of voxelotor after data from a clinical trial showed that a higher number of deaths occurred with the drug than with placebo and another trial showed the total number of deaths was higher than anticipated.

On September 26, the EMA’s human medicines committee recommended suspending the marketing authorization of voxelotor, citing new safety data that emerged during the review. The drug had received marketing authorization for the European Union in 2022, the agency said.

The US Food and Drug Administration (FDA), which first cleared voxelotor for sale in 2019, also said it has been conducting a safety review of the drug. The agency continues to examine post-marketing clinical trial data for voxelotor, the real-world registry studies, and data from the FDA Adverse Event Reporting System. At the conclusion of this review, the FDA will communicate any additional findings, if necessary, the agency said.

The FDA said it appeared that more deaths and a higher rate of vaso-occlusive crisis occurred in patients taking voxelotor vs placebo in post-marketing clinical trials.

“Pfizer also observed a higher rate of vaso-occlusive crisis in patients with sickle cell disease receiving Oxbryta in two real-world registry studies,” the FDA said. “Based on the totality of clinical data, Pfizer has determined the benefit of Oxbryta does not outweigh the risk.”
 

Gene Therapy, Tried-and-True Hydroxyurea (HU)

As a field, SCD has drawn more interest in recent years, with significant gains made lately in cutting-edge projects.

The FDA in December approved two gene-editing treatments for patients aged 12 years or older. These are considered “milestone treatments” for a debilitating and potentially life-threatening blood disorder that affects about 100,000 people in the United States. Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics) is the first to use the gene-editing tool CRISPR. And lovotibeglogene autotemcel (Lyfgenia, bluebird bio) uses a different gene-editing tool called a lentiviral vector.

These advances have been covered widely by the news media but are not expected to be widely available, with the cost of these extensive treatments estimated around $2-$3 million per patient.

“Gene therapy is amazing in that it can offer a cure, but it’s very expensive and not all patients are suitable for it. Some have so much existing organ damage that it’s not an option for them,” said John Wood, MD, PhD, director of cardiovascular MRI at Children’s Hospital Los Angeles, Los Angeles, who does research on SCD.

“So it really is a great treatment for a very few people,” he said in an interview.

The mainstay of treatment for SCD remains a drug that Lydia Pecker, MD, a pediatric hematologist at Johns Hopkins University in Baltimore, describes as the “first, oldest, and best”: HU.

The FDA approved this in 1998 for use in SCD. It reduces the frequency of painful crises and acute chest syndrome and other complications of SCD that otherwise could be serious or even lethal, Pecker said.

“Older doctors can tell you that what they experienced with sickle cell disease in the hospitals has been completely transformed because of the high uptake of the drug,” she said, adding that it made a “profound” change. “We just don’t have data for any other agent that’s quite like that.”

Voxelotor had been a good second drug to add for some patients, in addition to HU and blood transfusions, Dr. Pecker noted. It was a first-line drug for those for whom transfusion and HU were not options, which constitutes a relatively small number of patients, she said.

“So we have, in the last 5 years, felt more hopeful because we had something else to offer,” she said.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology at Children’s Hospital of Philadelphia in Pennsylvania, said in an interview that her organization also had patients who appeared to benefit from voxelotor, some of whom had been participants in clinical trials.

Dr. Thompson, who has been a top researcher involved in the study of gene therapy, urged the need for companies to keep seeking to expand the options for people with SCD, even after the setback with voxelotor.

“I hope that there’s an appreciation for the need for continued investment in this very serious condition, for which there are insufficient options for treatments,” Dr. Thompson said. “So ongoing investment is really needed if we expect to make progress.”

Dr. Pecker disclosed ties with Novartis, Afimmune, the American Society of Hematology, and the National Institutes of Health. Thompson reported relationships with bluebird bio, Beam, Editas, Novartis, and Novo Nordisk.
 

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

The global withdrawal of voxelotor (Oxbryta, Pfizer) has left clinicians who treat sickle cell disease (SCD) with the urgent task of reaching patients taking the medicine, while trying to understand why it was taken off the market.

The National Alliance of Sickle Cell Centers issued a statement urging patients not to stop voxelotor abruptly. Instead, they should work out plans with their physicians and medical teams for weaning plans.

“Don’t lose faith. This a step backward, but we will stay on the path to better outcomes for everyone,” said the alliance in a statement to patients and clinicians.

On September 25, Pfizer said it would withdraw all lots of voxelotor in all markets where it is approved. The New York–based drugmaker also said it was discontinuing all active voxelotor clinical trials and expanded access programs worldwide. The cause was data that suggested “an imbalance in vaso-occlusive crises and fatal events which require further assessment.”

Pfizer told this news organization in an email exchange that it is focused on analyzing the data and will share updates in the future about presenting or publishing on this issue.

The withdrawal came amid increased scrutiny of the drug by the European Medicines Agency (EMA). The EMA in July began a review of voxelotor after data from a clinical trial showed that a higher number of deaths occurred with the drug than with placebo and another trial showed the total number of deaths was higher than anticipated.

On September 26, the EMA’s human medicines committee recommended suspending the marketing authorization of voxelotor, citing new safety data that emerged during the review. The drug had received marketing authorization for the European Union in 2022, the agency said.

The US Food and Drug Administration (FDA), which first cleared voxelotor for sale in 2019, also said it has been conducting a safety review of the drug. The agency continues to examine post-marketing clinical trial data for voxelotor, the real-world registry studies, and data from the FDA Adverse Event Reporting System. At the conclusion of this review, the FDA will communicate any additional findings, if necessary, the agency said.

The FDA said it appeared that more deaths and a higher rate of vaso-occlusive crisis occurred in patients taking voxelotor vs placebo in post-marketing clinical trials.

“Pfizer also observed a higher rate of vaso-occlusive crisis in patients with sickle cell disease receiving Oxbryta in two real-world registry studies,” the FDA said. “Based on the totality of clinical data, Pfizer has determined the benefit of Oxbryta does not outweigh the risk.”
 

Gene Therapy, Tried-and-True Hydroxyurea (HU)

As a field, SCD has drawn more interest in recent years, with significant gains made lately in cutting-edge projects.

The FDA in December approved two gene-editing treatments for patients aged 12 years or older. These are considered “milestone treatments” for a debilitating and potentially life-threatening blood disorder that affects about 100,000 people in the United States. Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals and CRISPR Therapeutics) is the first to use the gene-editing tool CRISPR. And lovotibeglogene autotemcel (Lyfgenia, bluebird bio) uses a different gene-editing tool called a lentiviral vector.

These advances have been covered widely by the news media but are not expected to be widely available, with the cost of these extensive treatments estimated around $2-$3 million per patient.

“Gene therapy is amazing in that it can offer a cure, but it’s very expensive and not all patients are suitable for it. Some have so much existing organ damage that it’s not an option for them,” said John Wood, MD, PhD, director of cardiovascular MRI at Children’s Hospital Los Angeles, Los Angeles, who does research on SCD.

“So it really is a great treatment for a very few people,” he said in an interview.

The mainstay of treatment for SCD remains a drug that Lydia Pecker, MD, a pediatric hematologist at Johns Hopkins University in Baltimore, describes as the “first, oldest, and best”: HU.

The FDA approved this in 1998 for use in SCD. It reduces the frequency of painful crises and acute chest syndrome and other complications of SCD that otherwise could be serious or even lethal, Pecker said.

“Older doctors can tell you that what they experienced with sickle cell disease in the hospitals has been completely transformed because of the high uptake of the drug,” she said, adding that it made a “profound” change. “We just don’t have data for any other agent that’s quite like that.”

Voxelotor had been a good second drug to add for some patients, in addition to HU and blood transfusions, Dr. Pecker noted. It was a first-line drug for those for whom transfusion and HU were not options, which constitutes a relatively small number of patients, she said.

“So we have, in the last 5 years, felt more hopeful because we had something else to offer,” she said.

Alexis A. Thompson, MD, MPH, chief of the Division of Hematology at Children’s Hospital of Philadelphia in Pennsylvania, said in an interview that her organization also had patients who appeared to benefit from voxelotor, some of whom had been participants in clinical trials.

Dr. Thompson, who has been a top researcher involved in the study of gene therapy, urged the need for companies to keep seeking to expand the options for people with SCD, even after the setback with voxelotor.

“I hope that there’s an appreciation for the need for continued investment in this very serious condition, for which there are insufficient options for treatments,” Dr. Thompson said. “So ongoing investment is really needed if we expect to make progress.”

Dr. Pecker disclosed ties with Novartis, Afimmune, the American Society of Hematology, and the National Institutes of Health. Thompson reported relationships with bluebird bio, Beam, Editas, Novartis, and Novo Nordisk.
 

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

Research conducted at the US Department of Veterans Affairs (VA) could offer crucial insight into the hotly debated question of whether patients with head and neck cancer should have access to percutaneous endoscopic gastrostomy (PEG) before they develop malnutrition.

While no definitive conclusions can be drawn until a complete study is performed, early findings of a pilot trial are intriguing, said advanced practice oncology dietitian Katherine Petersen, MS, RDN, CSO, of the Phoenix VA Health Care System, who spoke in an interview with Federal Practitioner and at the annual meeting of the Association of VA Hematology/Oncology.

So far, the 12 patients with head and neck cancer who agreed to the placement of prophylactic feeding tubes prior to chemoradiation have had worse outcomes in some areas compared to the 9 patients who had tubes inserted when clinically indicated and the 12 who didn't need feeding tubes.

Petersen cautioned that the study is small and underpowered at this point. Still, she noted, "We're seeing a hint of exactly the opposite of what I expected. Those who get a tube prophylactically are doing worse than those who are getting it reactively or not at all, If that's the case, that's a really important outcome."

As Petersen explained, the placement of PEG feeding tubes is a hot topic in head and neck cancer care. Malnutrition affects about 80% of these patients and can contribute to mortality, raising the question of whether they should have access to feeding tubes placed prior to treatment in case enteral nutrition is needed.

In some patients with head and neck cancer, malnutrition may arise when tumors block food intake or prevent patients from swallowing. "But in my clinical experience, most often it's from the adverse effects of radiation and chemotherapy. Radiation creates burns inside their throat that make it hard to swallow. Or they have taste changes or really dry mouth," Petersen said.

"On top of these problems, chemotherapy can cause nausea and vomiting," she said. Placing feeding tube access may seem like a smart strategy to head off malnutrition as soon as it occurs. But, as Petersen noted, feeding tube use can lead to dependency as patients lose their ability to swallow. "There's a theory that if we give people feeding tubes, they'll go with the easier route of using a feeding tube and not keep swallowing. Then those swallowing muscles would weaken, and patients would end up permanently on a feeding tube."

In 2020, a retrospective VA study linked feeding tube dependence to lower overall survival in head and neck cancer patients. There are also risks to feeding tube placement, such as infection, pain, leakage, and inflammation.

But what if feeding tube valves are inserted prophylactically so they can be used for nutrition if needed? "We just haven't had any prospective studies to get to the heart of the matter and answer the question," she said. "It's hard to recruit. How do you convince somebody to randomly be assigned to have a hole poked in their stomach?"

For the new pilot study, researchers in Phoenix decided not to randomize patients. Instead, they asked them whether they'd accept the placement of feeding tube valves on a prophylactic basis.

Thirty-six veterans enrolled in 3 years, 33% of those were eligible. Twelve have died, 1 withdrew, and 2 were lost to follow-up.

Those in the prophylactic group had worse physical function and muscle strength over time, while those who received feeding tubes when needed had more adverse events.

Why might some outcomes be worse for patients who chose the prophylactic approach? "The answer is unclear," Petersen said. "Although one possibility is that those patients had higher-risk tumors and were more clued into their own risk."

"The goal now is to get funding for an expanded, multicenter study within the VA," Petersen said. The big question that she hopes to answer is: Does a prophylactic approach work? "Does it make a difference for patients in terms of how quickly they go back to living a full, meaningful life and be able to do all the things that they normally would do?"

A complete study would likely last 7 years, but helpful results may come earlier. "We are starting to see significant differences in terms of our main outcomes of physical function," Petersen said. "We only need 1 to 2 years of data for each patient to get to the heart of that."

The study is not funded, and Petersen reported no disclosures.

Research conducted at the US Department of Veterans Affairs (VA) could offer crucial insight into the hotly debated question of whether patients with head and neck cancer should have access to percutaneous endoscopic gastrostomy (PEG) before they develop malnutrition.

While no definitive conclusions can be drawn until a complete study is performed, early findings of a pilot trial are intriguing, said advanced practice oncology dietitian Katherine Petersen, MS, RDN, CSO, of the Phoenix VA Health Care System, who spoke in an interview with Federal Practitioner and at the annual meeting of the Association of VA Hematology/Oncology.

So far, the 12 patients with head and neck cancer who agreed to the placement of prophylactic feeding tubes prior to chemoradiation have had worse outcomes in some areas compared to the 9 patients who had tubes inserted when clinically indicated and the 12 who didn't need feeding tubes.

Petersen cautioned that the study is small and underpowered at this point. Still, she noted, "We're seeing a hint of exactly the opposite of what I expected. Those who get a tube prophylactically are doing worse than those who are getting it reactively or not at all, If that's the case, that's a really important outcome."

As Petersen explained, the placement of PEG feeding tubes is a hot topic in head and neck cancer care. Malnutrition affects about 80% of these patients and can contribute to mortality, raising the question of whether they should have access to feeding tubes placed prior to treatment in case enteral nutrition is needed.

In some patients with head and neck cancer, malnutrition may arise when tumors block food intake or prevent patients from swallowing. "But in my clinical experience, most often it's from the adverse effects of radiation and chemotherapy. Radiation creates burns inside their throat that make it hard to swallow. Or they have taste changes or really dry mouth," Petersen said.

"On top of these problems, chemotherapy can cause nausea and vomiting," she said. Placing feeding tube access may seem like a smart strategy to head off malnutrition as soon as it occurs. But, as Petersen noted, feeding tube use can lead to dependency as patients lose their ability to swallow. "There's a theory that if we give people feeding tubes, they'll go with the easier route of using a feeding tube and not keep swallowing. Then those swallowing muscles would weaken, and patients would end up permanently on a feeding tube."

In 2020, a retrospective VA study linked feeding tube dependence to lower overall survival in head and neck cancer patients. There are also risks to feeding tube placement, such as infection, pain, leakage, and inflammation.

But what if feeding tube valves are inserted prophylactically so they can be used for nutrition if needed? "We just haven't had any prospective studies to get to the heart of the matter and answer the question," she said. "It's hard to recruit. How do you convince somebody to randomly be assigned to have a hole poked in their stomach?"

For the new pilot study, researchers in Phoenix decided not to randomize patients. Instead, they asked them whether they'd accept the placement of feeding tube valves on a prophylactic basis.

Thirty-six veterans enrolled in 3 years, 33% of those were eligible. Twelve have died, 1 withdrew, and 2 were lost to follow-up.

Those in the prophylactic group had worse physical function and muscle strength over time, while those who received feeding tubes when needed had more adverse events.

Why might some outcomes be worse for patients who chose the prophylactic approach? "The answer is unclear," Petersen said. "Although one possibility is that those patients had higher-risk tumors and were more clued into their own risk."

"The goal now is to get funding for an expanded, multicenter study within the VA," Petersen said. The big question that she hopes to answer is: Does a prophylactic approach work? "Does it make a difference for patients in terms of how quickly they go back to living a full, meaningful life and be able to do all the things that they normally would do?"

A complete study would likely last 7 years, but helpful results may come earlier. "We are starting to see significant differences in terms of our main outcomes of physical function," Petersen said. "We only need 1 to 2 years of data for each patient to get to the heart of that."

The study is not funded, and Petersen reported no disclosures.

Research conducted at the US Department of Veterans Affairs (VA) could offer crucial insight into the hotly debated question of whether patients with head and neck cancer should have access to percutaneous endoscopic gastrostomy (PEG) before they develop malnutrition.

While no definitive conclusions can be drawn until a complete study is performed, early findings of a pilot trial are intriguing, said advanced practice oncology dietitian Katherine Petersen, MS, RDN, CSO, of the Phoenix VA Health Care System, who spoke in an interview with Federal Practitioner and at the annual meeting of the Association of VA Hematology/Oncology.

So far, the 12 patients with head and neck cancer who agreed to the placement of prophylactic feeding tubes prior to chemoradiation have had worse outcomes in some areas compared to the 9 patients who had tubes inserted when clinically indicated and the 12 who didn't need feeding tubes.

Petersen cautioned that the study is small and underpowered at this point. Still, she noted, "We're seeing a hint of exactly the opposite of what I expected. Those who get a tube prophylactically are doing worse than those who are getting it reactively or not at all, If that's the case, that's a really important outcome."

As Petersen explained, the placement of PEG feeding tubes is a hot topic in head and neck cancer care. Malnutrition affects about 80% of these patients and can contribute to mortality, raising the question of whether they should have access to feeding tubes placed prior to treatment in case enteral nutrition is needed.

In some patients with head and neck cancer, malnutrition may arise when tumors block food intake or prevent patients from swallowing. "But in my clinical experience, most often it's from the adverse effects of radiation and chemotherapy. Radiation creates burns inside their throat that make it hard to swallow. Or they have taste changes or really dry mouth," Petersen said.

"On top of these problems, chemotherapy can cause nausea and vomiting," she said. Placing feeding tube access may seem like a smart strategy to head off malnutrition as soon as it occurs. But, as Petersen noted, feeding tube use can lead to dependency as patients lose their ability to swallow. "There's a theory that if we give people feeding tubes, they'll go with the easier route of using a feeding tube and not keep swallowing. Then those swallowing muscles would weaken, and patients would end up permanently on a feeding tube."

In 2020, a retrospective VA study linked feeding tube dependence to lower overall survival in head and neck cancer patients. There are also risks to feeding tube placement, such as infection, pain, leakage, and inflammation.

But what if feeding tube valves are inserted prophylactically so they can be used for nutrition if needed? "We just haven't had any prospective studies to get to the heart of the matter and answer the question," she said. "It's hard to recruit. How do you convince somebody to randomly be assigned to have a hole poked in their stomach?"

For the new pilot study, researchers in Phoenix decided not to randomize patients. Instead, they asked them whether they'd accept the placement of feeding tube valves on a prophylactic basis.

Thirty-six veterans enrolled in 3 years, 33% of those were eligible. Twelve have died, 1 withdrew, and 2 were lost to follow-up.

Those in the prophylactic group had worse physical function and muscle strength over time, while those who received feeding tubes when needed had more adverse events.

Why might some outcomes be worse for patients who chose the prophylactic approach? "The answer is unclear," Petersen said. "Although one possibility is that those patients had higher-risk tumors and were more clued into their own risk."

"The goal now is to get funding for an expanded, multicenter study within the VA," Petersen said. The big question that she hopes to answer is: Does a prophylactic approach work? "Does it make a difference for patients in terms of how quickly they go back to living a full, meaningful life and be able to do all the things that they normally would do?"

A complete study would likely last 7 years, but helpful results may come earlier. "We are starting to see significant differences in terms of our main outcomes of physical function," Petersen said. "We only need 1 to 2 years of data for each patient to get to the heart of that."

The study is not funded, and Petersen reported no disclosures.

The Veterans Health Administration (VHA) provides care for about 10,000 transgender women, and clinicians must understand their distinctive needs for prostate cancer screening, a urologist told cancer specialists during a presentation at the 2024 annual meeting of the Association of VA Hematology/Oncology in Atlanta.

Even if they’ve undergone gender reassignment surgery, “all transgender women still have a prostate, so therefore they remain at risk of prostate cancer and could still be considered for prostate cancer screening,” said Farnoosh Nik-Ahd, MD, a resident physician at the University of California San Francisco. However, “clinicians and patients may not be aware of prostate cancer risk, so that they may not think [of screening] transgender women.”

Nik-Ahd also noted another complication: The results of prostate screening tests may be misleading in this population.

Transgender women were born biologically male but now identify as female. These individuals may have undergone gender reassignment surgery to remove male genitalia, but the procedures do not remove the prostate. They also might be taking estrogen therapy. “Prostate cancer is a hormonally driven cancer, and the exact impact of gender-affirming hormones on prostate cancer risk and development is unknown,” Nik-Ahd said.

In a 2023 study in JAMA, Nik-Ahd and colleagues identified 155 cases of prostate cancer in transgender women within the VHA (about 14 cases per year) from 2000 to 2022. Of these patients, 116 had never used estrogen, while 17 had used it previously and 22 used it at diagnosis.

The median age of patients was 61 years, 88% identified as White, and the median prostate-specific antigen (PSA) was 6.8 ng/mL. “Given estimates of 10,000 transgender women in the US Department of Veterans Affairs, 33 cases per year would be expected. Instead, only about 14 per year were observed,” the researchers wrote. “Lower rates may stem from less PSA screening owing to barriers including lack of prostate cancer risk awareness or stigma, the suppressive effects of estrogen on prostate cancer development, or prostate cancers being missed in transgender women because of misinterpretation of ‘normal’ PSA levels among those receiving gender-affirming hormone therapies.”

In the presentation, Nik-Ahd said, “PSA density, which is a marker of prostate cancer aggressiveness, was highest in transgender women who were actively on estrogen.”

She noted, “the existing thyrotropin reference ranges, which is what we use to interpret PSA values, are all based on data from cisgender men.” The ranges would be expected to be far lower in transgender women who are taking estrogen, potentially throwing off screening tests, she said, and “ultimately missing clinically significant prostate cancer.”

In the larger picture, there are no specific guidelines about PSA screening in transgender women, she said. 

A recent study published in JAMA by Nik-Ahd and colleagues examined PSA levels in 210 transgender women (mean age 60 years) treated within the VHA from 2000 to 2023. All were aged 40 to 80 years, had received estrogen for at least 6 months (mean duration 4.7 years), and didn’t have prostate cancer diagnoses.

“Median (IQR) PSA was 0.02 (0-0.2) ng/mL and the 95th percentile value was 0.6 ng/mL,” the report found. “PSAs were undetectable in 36% of patients (23% and 49% of PSAs in patients without and with orchiectomy, respectively).”

The researchers write that “the historic cut point of 4 ng/mL, often used as a threshold for further evaluation, is likely far too high a threshold for this population.”

Nik-Ahd noted, “clinicians should interpret PSA values in transgender women on estrogen with extreme caution. In this population, normal might actually not be normal, and a value that is considered normal might be very abnormal for somebody who is on estrogen. If you're unsure of whether a PSA value is appropriate for a transgender woman on estrogen, refer that patient to a urologist so they can undergo further evaluation.”

Farnoosh Nik-Ahd discloses consulting for Janssen.

The Veterans Health Administration (VHA) provides care for about 10,000 transgender women, and clinicians must understand their distinctive needs for prostate cancer screening, a urologist told cancer specialists during a presentation at the 2024 annual meeting of the Association of VA Hematology/Oncology in Atlanta.

Even if they’ve undergone gender reassignment surgery, “all transgender women still have a prostate, so therefore they remain at risk of prostate cancer and could still be considered for prostate cancer screening,” said Farnoosh Nik-Ahd, MD, a resident physician at the University of California San Francisco. However, “clinicians and patients may not be aware of prostate cancer risk, so that they may not think [of screening] transgender women.”

Nik-Ahd also noted another complication: The results of prostate screening tests may be misleading in this population.

Transgender women were born biologically male but now identify as female. These individuals may have undergone gender reassignment surgery to remove male genitalia, but the procedures do not remove the prostate. They also might be taking estrogen therapy. “Prostate cancer is a hormonally driven cancer, and the exact impact of gender-affirming hormones on prostate cancer risk and development is unknown,” Nik-Ahd said.

In a 2023 study in JAMA, Nik-Ahd and colleagues identified 155 cases of prostate cancer in transgender women within the VHA (about 14 cases per year) from 2000 to 2022. Of these patients, 116 had never used estrogen, while 17 had used it previously and 22 used it at diagnosis.

The median age of patients was 61 years, 88% identified as White, and the median prostate-specific antigen (PSA) was 6.8 ng/mL. “Given estimates of 10,000 transgender women in the US Department of Veterans Affairs, 33 cases per year would be expected. Instead, only about 14 per year were observed,” the researchers wrote. “Lower rates may stem from less PSA screening owing to barriers including lack of prostate cancer risk awareness or stigma, the suppressive effects of estrogen on prostate cancer development, or prostate cancers being missed in transgender women because of misinterpretation of ‘normal’ PSA levels among those receiving gender-affirming hormone therapies.”

In the presentation, Nik-Ahd said, “PSA density, which is a marker of prostate cancer aggressiveness, was highest in transgender women who were actively on estrogen.”

She noted, “the existing thyrotropin reference ranges, which is what we use to interpret PSA values, are all based on data from cisgender men.” The ranges would be expected to be far lower in transgender women who are taking estrogen, potentially throwing off screening tests, she said, and “ultimately missing clinically significant prostate cancer.”

In the larger picture, there are no specific guidelines about PSA screening in transgender women, she said. 

A recent study published in JAMA by Nik-Ahd and colleagues examined PSA levels in 210 transgender women (mean age 60 years) treated within the VHA from 2000 to 2023. All were aged 40 to 80 years, had received estrogen for at least 6 months (mean duration 4.7 years), and didn’t have prostate cancer diagnoses.

“Median (IQR) PSA was 0.02 (0-0.2) ng/mL and the 95th percentile value was 0.6 ng/mL,” the report found. “PSAs were undetectable in 36% of patients (23% and 49% of PSAs in patients without and with orchiectomy, respectively).”

The researchers write that “the historic cut point of 4 ng/mL, often used as a threshold for further evaluation, is likely far too high a threshold for this population.”

Nik-Ahd noted, “clinicians should interpret PSA values in transgender women on estrogen with extreme caution. In this population, normal might actually not be normal, and a value that is considered normal might be very abnormal for somebody who is on estrogen. If you're unsure of whether a PSA value is appropriate for a transgender woman on estrogen, refer that patient to a urologist so they can undergo further evaluation.”

Farnoosh Nik-Ahd discloses consulting for Janssen.

The Veterans Health Administration (VHA) provides care for about 10,000 transgender women, and clinicians must understand their distinctive needs for prostate cancer screening, a urologist told cancer specialists during a presentation at the 2024 annual meeting of the Association of VA Hematology/Oncology in Atlanta.

Even if they’ve undergone gender reassignment surgery, “all transgender women still have a prostate, so therefore they remain at risk of prostate cancer and could still be considered for prostate cancer screening,” said Farnoosh Nik-Ahd, MD, a resident physician at the University of California San Francisco. However, “clinicians and patients may not be aware of prostate cancer risk, so that they may not think [of screening] transgender women.”

Nik-Ahd also noted another complication: The results of prostate screening tests may be misleading in this population.

Transgender women were born biologically male but now identify as female. These individuals may have undergone gender reassignment surgery to remove male genitalia, but the procedures do not remove the prostate. They also might be taking estrogen therapy. “Prostate cancer is a hormonally driven cancer, and the exact impact of gender-affirming hormones on prostate cancer risk and development is unknown,” Nik-Ahd said.

In a 2023 study in JAMA, Nik-Ahd and colleagues identified 155 cases of prostate cancer in transgender women within the VHA (about 14 cases per year) from 2000 to 2022. Of these patients, 116 had never used estrogen, while 17 had used it previously and 22 used it at diagnosis.

The median age of patients was 61 years, 88% identified as White, and the median prostate-specific antigen (PSA) was 6.8 ng/mL. “Given estimates of 10,000 transgender women in the US Department of Veterans Affairs, 33 cases per year would be expected. Instead, only about 14 per year were observed,” the researchers wrote. “Lower rates may stem from less PSA screening owing to barriers including lack of prostate cancer risk awareness or stigma, the suppressive effects of estrogen on prostate cancer development, or prostate cancers being missed in transgender women because of misinterpretation of ‘normal’ PSA levels among those receiving gender-affirming hormone therapies.”

In the presentation, Nik-Ahd said, “PSA density, which is a marker of prostate cancer aggressiveness, was highest in transgender women who were actively on estrogen.”

She noted, “the existing thyrotropin reference ranges, which is what we use to interpret PSA values, are all based on data from cisgender men.” The ranges would be expected to be far lower in transgender women who are taking estrogen, potentially throwing off screening tests, she said, and “ultimately missing clinically significant prostate cancer.”

In the larger picture, there are no specific guidelines about PSA screening in transgender women, she said. 

A recent study published in JAMA by Nik-Ahd and colleagues examined PSA levels in 210 transgender women (mean age 60 years) treated within the VHA from 2000 to 2023. All were aged 40 to 80 years, had received estrogen for at least 6 months (mean duration 4.7 years), and didn’t have prostate cancer diagnoses.

“Median (IQR) PSA was 0.02 (0-0.2) ng/mL and the 95th percentile value was 0.6 ng/mL,” the report found. “PSAs were undetectable in 36% of patients (23% and 49% of PSAs in patients without and with orchiectomy, respectively).”

The researchers write that “the historic cut point of 4 ng/mL, often used as a threshold for further evaluation, is likely far too high a threshold for this population.”

Nik-Ahd noted, “clinicians should interpret PSA values in transgender women on estrogen with extreme caution. In this population, normal might actually not be normal, and a value that is considered normal might be very abnormal for somebody who is on estrogen. If you're unsure of whether a PSA value is appropriate for a transgender woman on estrogen, refer that patient to a urologist so they can undergo further evaluation.”

Farnoosh Nik-Ahd discloses consulting for Janssen.