AVAHO

Detection of circulating tumor DNA was significantly associated with worse rates of disease-free and overall survival in patients with stage I-III breast cancer, a new meta-analysis and systematic review found.

“Circulating tumor DNA (ctDNA) has been extensively studied as a prognostic biomarker in early breast cancer. However, there is a significant heterogeneity in the study results, which is probably related to the fact that each individual study included different patient populations, collected blood at different time points, and used different methods (assays) for ctDNA analysis,” said Guilherme Nader Marta, MD, of the Institut Jules Bordet, Anderlecht, Belgium, in an interview.

“The aim of our study was to summarize the available evidence that has been presented so far on this topic by performing a systematic review and meta-analysis including studies that reported the association between ctDNA detection and long-term outcomes,” said Dr. Nader Marta, who coauthored the new research, which was presented as a poster (Poster 26P) at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.
 

Methods and results

The authors identified 57 studies including data from 5,729 individuals with early breast cancer. The 44.5% for whom stages were reported consisted of 18.3% with stage I disease, 60.0% with stage II, and 21.5% with stage III. Patients’ ctDNA collection was divided into three groups: baseline, after neoadjuvant therapy (End-of-NAT), and during follow-up care; ctDNA assays were classified as tumor-informed or non–tumor-informed.

The detection of ctDNA at any time point during diagnosis and treatment was associated with worse disease-free survival (DFS) and overall survival (OS), compared with no ctDNA. The association was stronger in tumor-informed assays, the researchers said.

For disease-free survival, the overall multivariate hazard ratios were 2.5, 5.5, and 7.2 for ctDNA detection at baseline, End-of-NAT, and follow-up, respectively.

For overall survival, the overall multivariate hazard ratios were 3.0, 12.9, and 5.6, for ctDNA detection at baseline, End-of-NAT, and follow-up, respectively.

The pooled hazard ratios were numerically higher for both DFS and OS when ctDNA was detected at either End-of-NAT or follow-up.

In addition, detection of ctDNA was associated with a high degree of specificity (from 0.7 to 1.0) for breast cancer relapse; sensitivity ranged from 0.31 to 1.0, the researchers noted. The mean lead time from ctDNA detection to breast cancer recurrence in these cases was approximately 10 months.
 

Results show ctDNA detection is associated with worse survival

“Our study results demonstrate that ctDNA detection is associated with worse disease-free survival and overall survival in patients with early breast cancer, particularly when measured after treatment with tumor-informed assays,” Dr. Nader Marta said in an interview.

“As next steps, we need to build on this evidence to bring the potential benefits of this powerful prognostic tool to our patients,” said Dr. Nader Marta. “Ongoing studies exploring different management strategies based on serial ctDNA assessments will help us understand the exact role of this technology in our clinical practice.”

The study received no outside funding. Dr. Nader Marta disclosed relationships with companies including Roche and Bayer.
 

Detection of circulating tumor DNA was significantly associated with worse rates of disease-free and overall survival in patients with stage I-III breast cancer, a new meta-analysis and systematic review found.

“Circulating tumor DNA (ctDNA) has been extensively studied as a prognostic biomarker in early breast cancer. However, there is a significant heterogeneity in the study results, which is probably related to the fact that each individual study included different patient populations, collected blood at different time points, and used different methods (assays) for ctDNA analysis,” said Guilherme Nader Marta, MD, of the Institut Jules Bordet, Anderlecht, Belgium, in an interview.

“The aim of our study was to summarize the available evidence that has been presented so far on this topic by performing a systematic review and meta-analysis including studies that reported the association between ctDNA detection and long-term outcomes,” said Dr. Nader Marta, who coauthored the new research, which was presented as a poster (Poster 26P) at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.
 

Methods and results

The authors identified 57 studies including data from 5,729 individuals with early breast cancer. The 44.5% for whom stages were reported consisted of 18.3% with stage I disease, 60.0% with stage II, and 21.5% with stage III. Patients’ ctDNA collection was divided into three groups: baseline, after neoadjuvant therapy (End-of-NAT), and during follow-up care; ctDNA assays were classified as tumor-informed or non–tumor-informed.

The detection of ctDNA at any time point during diagnosis and treatment was associated with worse disease-free survival (DFS) and overall survival (OS), compared with no ctDNA. The association was stronger in tumor-informed assays, the researchers said.

For disease-free survival, the overall multivariate hazard ratios were 2.5, 5.5, and 7.2 for ctDNA detection at baseline, End-of-NAT, and follow-up, respectively.

For overall survival, the overall multivariate hazard ratios were 3.0, 12.9, and 5.6, for ctDNA detection at baseline, End-of-NAT, and follow-up, respectively.

The pooled hazard ratios were numerically higher for both DFS and OS when ctDNA was detected at either End-of-NAT or follow-up.

In addition, detection of ctDNA was associated with a high degree of specificity (from 0.7 to 1.0) for breast cancer relapse; sensitivity ranged from 0.31 to 1.0, the researchers noted. The mean lead time from ctDNA detection to breast cancer recurrence in these cases was approximately 10 months.
 

Results show ctDNA detection is associated with worse survival

“Our study results demonstrate that ctDNA detection is associated with worse disease-free survival and overall survival in patients with early breast cancer, particularly when measured after treatment with tumor-informed assays,” Dr. Nader Marta said in an interview.

“As next steps, we need to build on this evidence to bring the potential benefits of this powerful prognostic tool to our patients,” said Dr. Nader Marta. “Ongoing studies exploring different management strategies based on serial ctDNA assessments will help us understand the exact role of this technology in our clinical practice.”

The study received no outside funding. Dr. Nader Marta disclosed relationships with companies including Roche and Bayer.
 

Detection of circulating tumor DNA was significantly associated with worse rates of disease-free and overall survival in patients with stage I-III breast cancer, a new meta-analysis and systematic review found.

“Circulating tumor DNA (ctDNA) has been extensively studied as a prognostic biomarker in early breast cancer. However, there is a significant heterogeneity in the study results, which is probably related to the fact that each individual study included different patient populations, collected blood at different time points, and used different methods (assays) for ctDNA analysis,” said Guilherme Nader Marta, MD, of the Institut Jules Bordet, Anderlecht, Belgium, in an interview.

“The aim of our study was to summarize the available evidence that has been presented so far on this topic by performing a systematic review and meta-analysis including studies that reported the association between ctDNA detection and long-term outcomes,” said Dr. Nader Marta, who coauthored the new research, which was presented as a poster (Poster 26P) at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.
 

Methods and results

The authors identified 57 studies including data from 5,729 individuals with early breast cancer. The 44.5% for whom stages were reported consisted of 18.3% with stage I disease, 60.0% with stage II, and 21.5% with stage III. Patients’ ctDNA collection was divided into three groups: baseline, after neoadjuvant therapy (End-of-NAT), and during follow-up care; ctDNA assays were classified as tumor-informed or non–tumor-informed.

The detection of ctDNA at any time point during diagnosis and treatment was associated with worse disease-free survival (DFS) and overall survival (OS), compared with no ctDNA. The association was stronger in tumor-informed assays, the researchers said.

For disease-free survival, the overall multivariate hazard ratios were 2.5, 5.5, and 7.2 for ctDNA detection at baseline, End-of-NAT, and follow-up, respectively.

For overall survival, the overall multivariate hazard ratios were 3.0, 12.9, and 5.6, for ctDNA detection at baseline, End-of-NAT, and follow-up, respectively.

The pooled hazard ratios were numerically higher for both DFS and OS when ctDNA was detected at either End-of-NAT or follow-up.

In addition, detection of ctDNA was associated with a high degree of specificity (from 0.7 to 1.0) for breast cancer relapse; sensitivity ranged from 0.31 to 1.0, the researchers noted. The mean lead time from ctDNA detection to breast cancer recurrence in these cases was approximately 10 months.
 

Results show ctDNA detection is associated with worse survival

“Our study results demonstrate that ctDNA detection is associated with worse disease-free survival and overall survival in patients with early breast cancer, particularly when measured after treatment with tumor-informed assays,” Dr. Nader Marta said in an interview.

“As next steps, we need to build on this evidence to bring the potential benefits of this powerful prognostic tool to our patients,” said Dr. Nader Marta. “Ongoing studies exploring different management strategies based on serial ctDNA assessments will help us understand the exact role of this technology in our clinical practice.”

The study received no outside funding. Dr. Nader Marta disclosed relationships with companies including Roche and Bayer.
 

A new study finds that racial disparities in male breast cancer are persisting in the United States.

From 2000 to 2019, Black men were diagnosed at later ages than White males (median ages, 69 and 63 years, respectively) and were more likely to die from the disease (22.4% vs. 16.8%, respectively). Male breast cancer (MBC) was more likely to kill Black men in rural vs. urban areas (hazard ratio = 1.4; 95% confidence interval, 1.0-2.1; P less than .05). Among White males, in contrast, there was no difference on that front, according to the research, which was presented in a poster (Abstract No. 87P) at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.

It’s not clear why the disparities exist, said lead author Lekha Yadukumar, MBBS, an internal medicine resident at the Wright Center for Graduate Medical Education in Scranton, Penn., in an interview.

“Several potential factors may contribute to the higher rate of breast cancer diagnosis in older [Black] men, including the pathology of the disease, limited awareness about breast cancer, and potential barriers to accessibility,” she said. “The increased mortality among [Black men] may be linked to variations in tumor pathology and molecular biology. Social factors may also potentially impact survival rates, including [having] limited access to health care in rural areas and inadequate social support.”

Male breast cancer is rare, accounting for less than 1% of all breast cancer cases in the United States, according to the Breast Cancer Research Foundation. An estimated 2,700 men are diagnosed each year, and about 530 will die. Previous research has suggested Black men have worse outcomes than White men, but the data covered earlier years than the new study.
 

Methods and results

Dr. Yadukumar and colleagues retrospectively analyzed statistics from the Surveillance, Epidemiology, and End Results database for patients diagnosed with primary male breast cancer from 2000 to 2019 (n = 8,373; Black men, 1,111 [13.26%]; White men, 6,817 [81.41%]).

Median income didn’t affect mortality, whereas men in both racial groups were less likely to die if they were married vs. single/divorced (hazard ratio = 0.6; P less than .05).

Other studies have shown that “[Black American] men diagnosed with breast cancer experience longer time intervals before receiving treatment, encounter more severe disease manifestations, and exhibit lower rates of survivorship,” Dr. Yadukumar said. “Despite these findings, there remains a scarcity of genetic studies aimed at comprehending the underlying causes of these disparities. Moreover, there is a dearth of research investigating other factors that may influence survival outcomes among men with breast cancer.”
 

Findings reflect the disparities in female breast cancer

In an interview, Duke University, Durham, N.C., oncologist Arif Kamal, MD, MBA, MHS, the chief patient officer at the American Cancer Society, said the study is impressive since the number of patients is large for a rare cancer and the population is diverse. Plus, the findings reflect the disparities in female breast cancer, he noted.

“We know that Black women’s mortality is worse vs. White women in breast cancer, and we believe that most of that has nothing to do with cancer screening,” said Dr. Kamal, who was not involved in the new study. “When the clock starts from diagnosis onwards, you start to see less introduction to clinical trials and standard care medications and more time to treatment, surgery, and radiation,” he said.

“You see similar disparities as related to mortality in Black vs. White men,” he noted.

The new findings about higher death rates for Black men, especially in rural areas, suggest that “distance matters, and race matters,” he said. In rural areas, it can be hard to access pathologists, radiologists, and surgeons with more experience with breast cancer, he said.

But, he noted, the study finds that income doesn’t appear to be a factor.

In the big picture, he said, the results suggest that when it comes to barriers to better outcomes, “things that are systemic don’t make exceptions because you are a man vs. a woman.”

No study funding was reported. The study authors and Dr. Kamal have no relevant financial disclosures.
 

A new study finds that racial disparities in male breast cancer are persisting in the United States.

From 2000 to 2019, Black men were diagnosed at later ages than White males (median ages, 69 and 63 years, respectively) and were more likely to die from the disease (22.4% vs. 16.8%, respectively). Male breast cancer (MBC) was more likely to kill Black men in rural vs. urban areas (hazard ratio = 1.4; 95% confidence interval, 1.0-2.1; P less than .05). Among White males, in contrast, there was no difference on that front, according to the research, which was presented in a poster (Abstract No. 87P) at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.

It’s not clear why the disparities exist, said lead author Lekha Yadukumar, MBBS, an internal medicine resident at the Wright Center for Graduate Medical Education in Scranton, Penn., in an interview.

“Several potential factors may contribute to the higher rate of breast cancer diagnosis in older [Black] men, including the pathology of the disease, limited awareness about breast cancer, and potential barriers to accessibility,” she said. “The increased mortality among [Black men] may be linked to variations in tumor pathology and molecular biology. Social factors may also potentially impact survival rates, including [having] limited access to health care in rural areas and inadequate social support.”

Male breast cancer is rare, accounting for less than 1% of all breast cancer cases in the United States, according to the Breast Cancer Research Foundation. An estimated 2,700 men are diagnosed each year, and about 530 will die. Previous research has suggested Black men have worse outcomes than White men, but the data covered earlier years than the new study.
 

Methods and results

Dr. Yadukumar and colleagues retrospectively analyzed statistics from the Surveillance, Epidemiology, and End Results database for patients diagnosed with primary male breast cancer from 2000 to 2019 (n = 8,373; Black men, 1,111 [13.26%]; White men, 6,817 [81.41%]).

Median income didn’t affect mortality, whereas men in both racial groups were less likely to die if they were married vs. single/divorced (hazard ratio = 0.6; P less than .05).

Other studies have shown that “[Black American] men diagnosed with breast cancer experience longer time intervals before receiving treatment, encounter more severe disease manifestations, and exhibit lower rates of survivorship,” Dr. Yadukumar said. “Despite these findings, there remains a scarcity of genetic studies aimed at comprehending the underlying causes of these disparities. Moreover, there is a dearth of research investigating other factors that may influence survival outcomes among men with breast cancer.”
 

Findings reflect the disparities in female breast cancer

In an interview, Duke University, Durham, N.C., oncologist Arif Kamal, MD, MBA, MHS, the chief patient officer at the American Cancer Society, said the study is impressive since the number of patients is large for a rare cancer and the population is diverse. Plus, the findings reflect the disparities in female breast cancer, he noted.

“We know that Black women’s mortality is worse vs. White women in breast cancer, and we believe that most of that has nothing to do with cancer screening,” said Dr. Kamal, who was not involved in the new study. “When the clock starts from diagnosis onwards, you start to see less introduction to clinical trials and standard care medications and more time to treatment, surgery, and radiation,” he said.

“You see similar disparities as related to mortality in Black vs. White men,” he noted.

The new findings about higher death rates for Black men, especially in rural areas, suggest that “distance matters, and race matters,” he said. In rural areas, it can be hard to access pathologists, radiologists, and surgeons with more experience with breast cancer, he said.

But, he noted, the study finds that income doesn’t appear to be a factor.

In the big picture, he said, the results suggest that when it comes to barriers to better outcomes, “things that are systemic don’t make exceptions because you are a man vs. a woman.”

No study funding was reported. The study authors and Dr. Kamal have no relevant financial disclosures.
 

A new study finds that racial disparities in male breast cancer are persisting in the United States.

From 2000 to 2019, Black men were diagnosed at later ages than White males (median ages, 69 and 63 years, respectively) and were more likely to die from the disease (22.4% vs. 16.8%, respectively). Male breast cancer (MBC) was more likely to kill Black men in rural vs. urban areas (hazard ratio = 1.4; 95% confidence interval, 1.0-2.1; P less than .05). Among White males, in contrast, there was no difference on that front, according to the research, which was presented in a poster (Abstract No. 87P) at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.

It’s not clear why the disparities exist, said lead author Lekha Yadukumar, MBBS, an internal medicine resident at the Wright Center for Graduate Medical Education in Scranton, Penn., in an interview.

“Several potential factors may contribute to the higher rate of breast cancer diagnosis in older [Black] men, including the pathology of the disease, limited awareness about breast cancer, and potential barriers to accessibility,” she said. “The increased mortality among [Black men] may be linked to variations in tumor pathology and molecular biology. Social factors may also potentially impact survival rates, including [having] limited access to health care in rural areas and inadequate social support.”

Male breast cancer is rare, accounting for less than 1% of all breast cancer cases in the United States, according to the Breast Cancer Research Foundation. An estimated 2,700 men are diagnosed each year, and about 530 will die. Previous research has suggested Black men have worse outcomes than White men, but the data covered earlier years than the new study.
 

Methods and results

Dr. Yadukumar and colleagues retrospectively analyzed statistics from the Surveillance, Epidemiology, and End Results database for patients diagnosed with primary male breast cancer from 2000 to 2019 (n = 8,373; Black men, 1,111 [13.26%]; White men, 6,817 [81.41%]).

Median income didn’t affect mortality, whereas men in both racial groups were less likely to die if they were married vs. single/divorced (hazard ratio = 0.6; P less than .05).

Other studies have shown that “[Black American] men diagnosed with breast cancer experience longer time intervals before receiving treatment, encounter more severe disease manifestations, and exhibit lower rates of survivorship,” Dr. Yadukumar said. “Despite these findings, there remains a scarcity of genetic studies aimed at comprehending the underlying causes of these disparities. Moreover, there is a dearth of research investigating other factors that may influence survival outcomes among men with breast cancer.”
 

Findings reflect the disparities in female breast cancer

In an interview, Duke University, Durham, N.C., oncologist Arif Kamal, MD, MBA, MHS, the chief patient officer at the American Cancer Society, said the study is impressive since the number of patients is large for a rare cancer and the population is diverse. Plus, the findings reflect the disparities in female breast cancer, he noted.

“We know that Black women’s mortality is worse vs. White women in breast cancer, and we believe that most of that has nothing to do with cancer screening,” said Dr. Kamal, who was not involved in the new study. “When the clock starts from diagnosis onwards, you start to see less introduction to clinical trials and standard care medications and more time to treatment, surgery, and radiation,” he said.

“You see similar disparities as related to mortality in Black vs. White men,” he noted.

The new findings about higher death rates for Black men, especially in rural areas, suggest that “distance matters, and race matters,” he said. In rural areas, it can be hard to access pathologists, radiologists, and surgeons with more experience with breast cancer, he said.

But, he noted, the study finds that income doesn’t appear to be a factor.

In the big picture, he said, the results suggest that when it comes to barriers to better outcomes, “things that are systemic don’t make exceptions because you are a man vs. a woman.”

No study funding was reported. The study authors and Dr. Kamal have no relevant financial disclosures.
 

CHICAGO - The American Urological Association and the Society of Urologic Oncology (SUO) for the first time have taken a position on the type of biopsy men with prostate lesions should undergo, endorsing both transperineal and transrectal biopsy instead of choosing one over the other.

The new guidelines, issued at the annual meeting of the American Urological Association, contrast with 2021 recommendations from the European Association of Urologists (EAU), which regard the transperineal approach as superior to and safer than the transrectal approach.

The new guidelines state: “Clinicians may use either a transrectal or transperineal biopsy route when performing a biopsy. (Conditional Recommendation; Evidence Level: Grade C).” Grade C is the lowest grade of acceptance the guideline committee could issue, according to Daniel Lin, MD, vice-chair of the AUA guideline panel.

“The AUA looked at all the higher-level data comparing the two procedures. There was a lack of that data,” Dr. Lin, chief of urologic oncology at the University of Washington, Seattle, said in an interview. He said the literature consists mainly of systematic single-center reviews, rather than multicenter randomized trials.

But Hendrik Van Poppel, MD, policy chief for the EAU, said that in Europe, transrectal biopsies are now considered “medical malpractice.”

Philip Cornford, MD, associate professor of urology at the University of Liverpool, England, and chair of the prostate biopsy guidelines panel for the EAU, said the society in 2021 concluded that the transperineal approach is the preferred one.

The EAU stated that transperineal prostate biopsies should be performed “due to the lower risk of infectious complications.” The EAU described the evidence as strong: A meta-analysis of seven studies that included 1,330 patients showed that for patients undergoing transperineal biopsy, infectious complications were significantly reduced.

Dr. Cornford said in essence, the EAU made its decision out of concern about infections, whereas the AUA and SUO based their decision on the ability of the methods to detect cancer.

Advocates for transperineal procedures cite several studies that show that the rate of infection, including sepsis, with such biopsies is virtually zero.

However, Dr. Lin noted that the committee said existing data on infection did not support this position. He also cited a “a fairly compelling” single-center randomized study with 750 patients that showed no difference in infection rates. The study was presented at the AUA meeting.
 

Agents of death and destruction?

Badar Mian, MD, professor of surgery at Albany (N.Y.) Medical College, who led the study, told an AUA session that urology has been trapped in an “echo chamber” regarding the relative safety of biopsies.

Clinicians hear “loud proclamations, which get repeated and magnified, that there is a real zero risk of complications after transperineal biopsies as compared to the horrendous 5% to 10% or higher rate of transrectal biopsy complications and that you, with your transrectal biopsies, are the cause of death and destruction all around,” Dr. Mian said. “Well, if you step out of the echo chamber, what you’ll find is that the accurate complications amongst the two procedures are not that dramatically different, much less dramatic than what you’ve been told to believe.”

The campaign to end transrectal biopsies in Europe started in 2018 with the death of a Norwegian man who experienced an infection after the procedure. Truls Bjerklund Johansen, MD, who’d performed the biopsy on the patient and who worked with the man’s daughter to change national practice, persuaded the EAU to look at the issue.

Advocates also say transperineal biopsies are better at detecting anterior and apical cancers.

“I would agree the data on cancer detection is less convincing, but that is not the basis of the EAU recommendation,” Dr. Cornford said.

Arvin George, MD, leads the transperineal biopsy program at the University of Michigan, Ann Arbor, and directs the transperineal training program at the AUA’s annual meeting. He said his course was sold out early and included about 60 trainees.

Dr. George said the new guideline statement “is not an unequivocal endorsement for transperineal biopsy as the preferred approach for diagnostic sampling but rather an acknowledgment of this approach as an alternative option.”

He said that although the new position statement should increase awareness of the transperineal approach in the United States, “without a strong recommendation, the guideline statement is unlikely to spark a large switch to the transperineal biopsy but rather supports the continued slow and steady adoption.”

Matthew Allaway, DO, founder of Perineologic, developer of the PrecisionPoint Transperineal Access System, said industry figures show that about 10% of the 1.5 million prostate biopsies performed in the United States annually are performed transperineally, a doubling in 2 years.

Jeremy Grummet, MD, clinical professor of urology at Monash University, Melbourne, and leader of the TREXIT (Transperineal Exit) movement to abandon transrectal procedures, said the AUA guidelines are biased toward “physician convenience.”
 

Lack of training

The AUA said another reason it did not endorse the transperineal approach was that currently, American urologists lack training and experience with transperineal procedures.

Dr. Grummet blamed major medical centers for any gap in the familiarity of clinicians with transperineal biopsies, which have been available for more than a decade.

“It is incumbent on the leaders of urology departments globally to ensure that their colleagues are trained in transperineal biopsy and have access to the appropriate equipment,” he said in an interview. “Lack of training didn’t seem to prevent the rapid uptake of robotic prostatectomy – a far more complex procedure.”

The authors have disclosed no relevant financial relationships.
 

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

CHICAGO - The American Urological Association and the Society of Urologic Oncology (SUO) for the first time have taken a position on the type of biopsy men with prostate lesions should undergo, endorsing both transperineal and transrectal biopsy instead of choosing one over the other.

The new guidelines, issued at the annual meeting of the American Urological Association, contrast with 2021 recommendations from the European Association of Urologists (EAU), which regard the transperineal approach as superior to and safer than the transrectal approach.

The new guidelines state: “Clinicians may use either a transrectal or transperineal biopsy route when performing a biopsy. (Conditional Recommendation; Evidence Level: Grade C).” Grade C is the lowest grade of acceptance the guideline committee could issue, according to Daniel Lin, MD, vice-chair of the AUA guideline panel.

“The AUA looked at all the higher-level data comparing the two procedures. There was a lack of that data,” Dr. Lin, chief of urologic oncology at the University of Washington, Seattle, said in an interview. He said the literature consists mainly of systematic single-center reviews, rather than multicenter randomized trials.

But Hendrik Van Poppel, MD, policy chief for the EAU, said that in Europe, transrectal biopsies are now considered “medical malpractice.”

Philip Cornford, MD, associate professor of urology at the University of Liverpool, England, and chair of the prostate biopsy guidelines panel for the EAU, said the society in 2021 concluded that the transperineal approach is the preferred one.

The EAU stated that transperineal prostate biopsies should be performed “due to the lower risk of infectious complications.” The EAU described the evidence as strong: A meta-analysis of seven studies that included 1,330 patients showed that for patients undergoing transperineal biopsy, infectious complications were significantly reduced.

Dr. Cornford said in essence, the EAU made its decision out of concern about infections, whereas the AUA and SUO based their decision on the ability of the methods to detect cancer.

Advocates for transperineal procedures cite several studies that show that the rate of infection, including sepsis, with such biopsies is virtually zero.

However, Dr. Lin noted that the committee said existing data on infection did not support this position. He also cited a “a fairly compelling” single-center randomized study with 750 patients that showed no difference in infection rates. The study was presented at the AUA meeting.
 

Agents of death and destruction?

Badar Mian, MD, professor of surgery at Albany (N.Y.) Medical College, who led the study, told an AUA session that urology has been trapped in an “echo chamber” regarding the relative safety of biopsies.

Clinicians hear “loud proclamations, which get repeated and magnified, that there is a real zero risk of complications after transperineal biopsies as compared to the horrendous 5% to 10% or higher rate of transrectal biopsy complications and that you, with your transrectal biopsies, are the cause of death and destruction all around,” Dr. Mian said. “Well, if you step out of the echo chamber, what you’ll find is that the accurate complications amongst the two procedures are not that dramatically different, much less dramatic than what you’ve been told to believe.”

The campaign to end transrectal biopsies in Europe started in 2018 with the death of a Norwegian man who experienced an infection after the procedure. Truls Bjerklund Johansen, MD, who’d performed the biopsy on the patient and who worked with the man’s daughter to change national practice, persuaded the EAU to look at the issue.

Advocates also say transperineal biopsies are better at detecting anterior and apical cancers.

“I would agree the data on cancer detection is less convincing, but that is not the basis of the EAU recommendation,” Dr. Cornford said.

Arvin George, MD, leads the transperineal biopsy program at the University of Michigan, Ann Arbor, and directs the transperineal training program at the AUA’s annual meeting. He said his course was sold out early and included about 60 trainees.

Dr. George said the new guideline statement “is not an unequivocal endorsement for transperineal biopsy as the preferred approach for diagnostic sampling but rather an acknowledgment of this approach as an alternative option.”

He said that although the new position statement should increase awareness of the transperineal approach in the United States, “without a strong recommendation, the guideline statement is unlikely to spark a large switch to the transperineal biopsy but rather supports the continued slow and steady adoption.”

Matthew Allaway, DO, founder of Perineologic, developer of the PrecisionPoint Transperineal Access System, said industry figures show that about 10% of the 1.5 million prostate biopsies performed in the United States annually are performed transperineally, a doubling in 2 years.

Jeremy Grummet, MD, clinical professor of urology at Monash University, Melbourne, and leader of the TREXIT (Transperineal Exit) movement to abandon transrectal procedures, said the AUA guidelines are biased toward “physician convenience.”
 

Lack of training

The AUA said another reason it did not endorse the transperineal approach was that currently, American urologists lack training and experience with transperineal procedures.

Dr. Grummet blamed major medical centers for any gap in the familiarity of clinicians with transperineal biopsies, which have been available for more than a decade.

“It is incumbent on the leaders of urology departments globally to ensure that their colleagues are trained in transperineal biopsy and have access to the appropriate equipment,” he said in an interview. “Lack of training didn’t seem to prevent the rapid uptake of robotic prostatectomy – a far more complex procedure.”

The authors have disclosed no relevant financial relationships.
 

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

CHICAGO - The American Urological Association and the Society of Urologic Oncology (SUO) for the first time have taken a position on the type of biopsy men with prostate lesions should undergo, endorsing both transperineal and transrectal biopsy instead of choosing one over the other.

The new guidelines, issued at the annual meeting of the American Urological Association, contrast with 2021 recommendations from the European Association of Urologists (EAU), which regard the transperineal approach as superior to and safer than the transrectal approach.

The new guidelines state: “Clinicians may use either a transrectal or transperineal biopsy route when performing a biopsy. (Conditional Recommendation; Evidence Level: Grade C).” Grade C is the lowest grade of acceptance the guideline committee could issue, according to Daniel Lin, MD, vice-chair of the AUA guideline panel.

“The AUA looked at all the higher-level data comparing the two procedures. There was a lack of that data,” Dr. Lin, chief of urologic oncology at the University of Washington, Seattle, said in an interview. He said the literature consists mainly of systematic single-center reviews, rather than multicenter randomized trials.

But Hendrik Van Poppel, MD, policy chief for the EAU, said that in Europe, transrectal biopsies are now considered “medical malpractice.”

Philip Cornford, MD, associate professor of urology at the University of Liverpool, England, and chair of the prostate biopsy guidelines panel for the EAU, said the society in 2021 concluded that the transperineal approach is the preferred one.

The EAU stated that transperineal prostate biopsies should be performed “due to the lower risk of infectious complications.” The EAU described the evidence as strong: A meta-analysis of seven studies that included 1,330 patients showed that for patients undergoing transperineal biopsy, infectious complications were significantly reduced.

Dr. Cornford said in essence, the EAU made its decision out of concern about infections, whereas the AUA and SUO based their decision on the ability of the methods to detect cancer.

Advocates for transperineal procedures cite several studies that show that the rate of infection, including sepsis, with such biopsies is virtually zero.

However, Dr. Lin noted that the committee said existing data on infection did not support this position. He also cited a “a fairly compelling” single-center randomized study with 750 patients that showed no difference in infection rates. The study was presented at the AUA meeting.
 

Agents of death and destruction?

Badar Mian, MD, professor of surgery at Albany (N.Y.) Medical College, who led the study, told an AUA session that urology has been trapped in an “echo chamber” regarding the relative safety of biopsies.

Clinicians hear “loud proclamations, which get repeated and magnified, that there is a real zero risk of complications after transperineal biopsies as compared to the horrendous 5% to 10% or higher rate of transrectal biopsy complications and that you, with your transrectal biopsies, are the cause of death and destruction all around,” Dr. Mian said. “Well, if you step out of the echo chamber, what you’ll find is that the accurate complications amongst the two procedures are not that dramatically different, much less dramatic than what you’ve been told to believe.”

The campaign to end transrectal biopsies in Europe started in 2018 with the death of a Norwegian man who experienced an infection after the procedure. Truls Bjerklund Johansen, MD, who’d performed the biopsy on the patient and who worked with the man’s daughter to change national practice, persuaded the EAU to look at the issue.

Advocates also say transperineal biopsies are better at detecting anterior and apical cancers.

“I would agree the data on cancer detection is less convincing, but that is not the basis of the EAU recommendation,” Dr. Cornford said.

Arvin George, MD, leads the transperineal biopsy program at the University of Michigan, Ann Arbor, and directs the transperineal training program at the AUA’s annual meeting. He said his course was sold out early and included about 60 trainees.

Dr. George said the new guideline statement “is not an unequivocal endorsement for transperineal biopsy as the preferred approach for diagnostic sampling but rather an acknowledgment of this approach as an alternative option.”

He said that although the new position statement should increase awareness of the transperineal approach in the United States, “without a strong recommendation, the guideline statement is unlikely to spark a large switch to the transperineal biopsy but rather supports the continued slow and steady adoption.”

Matthew Allaway, DO, founder of Perineologic, developer of the PrecisionPoint Transperineal Access System, said industry figures show that about 10% of the 1.5 million prostate biopsies performed in the United States annually are performed transperineally, a doubling in 2 years.

Jeremy Grummet, MD, clinical professor of urology at Monash University, Melbourne, and leader of the TREXIT (Transperineal Exit) movement to abandon transrectal procedures, said the AUA guidelines are biased toward “physician convenience.”
 

Lack of training

The AUA said another reason it did not endorse the transperineal approach was that currently, American urologists lack training and experience with transperineal procedures.

Dr. Grummet blamed major medical centers for any gap in the familiarity of clinicians with transperineal biopsies, which have been available for more than a decade.

“It is incumbent on the leaders of urology departments globally to ensure that their colleagues are trained in transperineal biopsy and have access to the appropriate equipment,” he said in an interview. “Lack of training didn’t seem to prevent the rapid uptake of robotic prostatectomy – a far more complex procedure.”

The authors have disclosed no relevant financial relationships.
 

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

The U.S. Food and Drug Administration has approved avapritinib (AYVAKIT, Blueprint Medicines) for the treatment of adults with indolent systemic mastocytosis. The new approval, which expands use of the drug to patients with indolent systemic mastocytosis, represents the “first and only approved medicine” to treat this disease, according to the company press statement.

Avapritinib, a selective KIT mutation-targeted tyrosine kinase inhibitor, was approved in 2021 to treat advanced systemic mastocytosis, a rare and potentially fatal hematologic disorder. Nonadvanced forms include indolent or smoldering disease; advanced disease can progress to leukemia. The expanded approval now covers patients with indolent disease, which represents the majority of patients with systemic mastocytosis.

The drug is also approved for adults with unresectable or metastatic GIST that harbors a platelet-derived growth factor receptor alpha exon 18 mutation.

The approval is based on data from the phase 2 PIONEER trial. In the trial, 222 patients with moderate to severe indolent, systemic mastocytosis* were randomly assigned in a 2:1 ratio to receive either avapritinib 25 mg once daily plus best supportive care or placebo plus best supportive care.

The findings, published in February, revealed that patients who received avapritinib experienced significantly greater improvements in total symptom scores at 24 weeks (–15.6 vs. –9.2 for control patients). Significantly more patients in the avapritinib arm achieved greater than or equal to 50% reductions in serum tryptase (54% vs. 0%), bone marrow mast cell aggregates (53% vs. 23%), and KIT D816V variant allele fraction (68% vs. 6%).

Most adverse reactions were mild to moderate in severity and included eye edema, dizziness, peripheral edema, and flushing. Fewer than 1% of patients discontinued treatment because of serious adverse reactions.

“People with indolent systemic mastocytosis are significantly impacted by their disease symptoms, and many individuals self-isolate at home to protect against unpredictable external triggers,” Judith Kain Emmel, board chair of the Mast Cell Disease Society, said in the company press release. “Today’s approval is a historic moment for the [systemic mastocytosis] community and offers new hope for patients and their families.

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

Correction, 5/23/23: An earlier version of this article mischaracterized these patients' conditions. They had moderate to severe indolent, systemic mastocytosis.

The U.S. Food and Drug Administration has approved avapritinib (AYVAKIT, Blueprint Medicines) for the treatment of adults with indolent systemic mastocytosis. The new approval, which expands use of the drug to patients with indolent systemic mastocytosis, represents the “first and only approved medicine” to treat this disease, according to the company press statement.

Avapritinib, a selective KIT mutation-targeted tyrosine kinase inhibitor, was approved in 2021 to treat advanced systemic mastocytosis, a rare and potentially fatal hematologic disorder. Nonadvanced forms include indolent or smoldering disease; advanced disease can progress to leukemia. The expanded approval now covers patients with indolent disease, which represents the majority of patients with systemic mastocytosis.

The drug is also approved for adults with unresectable or metastatic GIST that harbors a platelet-derived growth factor receptor alpha exon 18 mutation.

The approval is based on data from the phase 2 PIONEER trial. In the trial, 222 patients with moderate to severe indolent, systemic mastocytosis* were randomly assigned in a 2:1 ratio to receive either avapritinib 25 mg once daily plus best supportive care or placebo plus best supportive care.

The findings, published in February, revealed that patients who received avapritinib experienced significantly greater improvements in total symptom scores at 24 weeks (–15.6 vs. –9.2 for control patients). Significantly more patients in the avapritinib arm achieved greater than or equal to 50% reductions in serum tryptase (54% vs. 0%), bone marrow mast cell aggregates (53% vs. 23%), and KIT D816V variant allele fraction (68% vs. 6%).

Most adverse reactions were mild to moderate in severity and included eye edema, dizziness, peripheral edema, and flushing. Fewer than 1% of patients discontinued treatment because of serious adverse reactions.

“People with indolent systemic mastocytosis are significantly impacted by their disease symptoms, and many individuals self-isolate at home to protect against unpredictable external triggers,” Judith Kain Emmel, board chair of the Mast Cell Disease Society, said in the company press release. “Today’s approval is a historic moment for the [systemic mastocytosis] community and offers new hope for patients and their families.

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

Correction, 5/23/23: An earlier version of this article mischaracterized these patients' conditions. They had moderate to severe indolent, systemic mastocytosis.

The U.S. Food and Drug Administration has approved avapritinib (AYVAKIT, Blueprint Medicines) for the treatment of adults with indolent systemic mastocytosis. The new approval, which expands use of the drug to patients with indolent systemic mastocytosis, represents the “first and only approved medicine” to treat this disease, according to the company press statement.

Avapritinib, a selective KIT mutation-targeted tyrosine kinase inhibitor, was approved in 2021 to treat advanced systemic mastocytosis, a rare and potentially fatal hematologic disorder. Nonadvanced forms include indolent or smoldering disease; advanced disease can progress to leukemia. The expanded approval now covers patients with indolent disease, which represents the majority of patients with systemic mastocytosis.

The drug is also approved for adults with unresectable or metastatic GIST that harbors a platelet-derived growth factor receptor alpha exon 18 mutation.

The approval is based on data from the phase 2 PIONEER trial. In the trial, 222 patients with moderate to severe indolent, systemic mastocytosis* were randomly assigned in a 2:1 ratio to receive either avapritinib 25 mg once daily plus best supportive care or placebo plus best supportive care.

The findings, published in February, revealed that patients who received avapritinib experienced significantly greater improvements in total symptom scores at 24 weeks (–15.6 vs. –9.2 for control patients). Significantly more patients in the avapritinib arm achieved greater than or equal to 50% reductions in serum tryptase (54% vs. 0%), bone marrow mast cell aggregates (53% vs. 23%), and KIT D816V variant allele fraction (68% vs. 6%).

Most adverse reactions were mild to moderate in severity and included eye edema, dizziness, peripheral edema, and flushing. Fewer than 1% of patients discontinued treatment because of serious adverse reactions.

“People with indolent systemic mastocytosis are significantly impacted by their disease symptoms, and many individuals self-isolate at home to protect against unpredictable external triggers,” Judith Kain Emmel, board chair of the Mast Cell Disease Society, said in the company press release. “Today’s approval is a historic moment for the [systemic mastocytosis] community and offers new hope for patients and their families.

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

Correction, 5/23/23: An earlier version of this article mischaracterized these patients' conditions. They had moderate to severe indolent, systemic mastocytosis.

Thrombophilia testing should be limited to specific circumstances, including when venous thromboembolism (VTE) is provoked by nonsurgical risk factors such as pregnancy or oral contraception use, according to new clinical practice guidelines released by the American Society of Hematology. Individuals with a family history of VTE and high-risk thrombophilia, and those with VTE at unusual body sites should also be tested, the guidelines panel agreed.

“These guidelines will potentially change practice – we know that providers and patients will make a shared treatment decision and we wanted to outline specific scenarios to guide that decision,” panel cochair and first author Saskia Middeldorp, MD, PhD, explained in a press release announcing the publication of the guidelines in Blood Advances.

Dr. Middeldorp is a professor of medicine and head of the department of internal medicine at Radboud University Medical Center, Nijmegen, the Netherlands.

The guidelines are the latest in an ASH series of VTE-related guidelines. ASH convened a multidisciplinary panel with clinical and methodological expertise to develop the guidelines, which were subject to public comment, and they “provide recommendations informed by case-based approaches and modeling to ensure the medical community can better diagnose and treat thrombophilia and people with the condition can make the best decisions for their care,” the press release explains.

Thrombophilia affects an estimated 10% of the population. Testing for the clotting disorder can be costly, and the use of testing to help guide treatment decisions is controversial.

“For decades there has been dispute about thrombophilia testing,” Dr. Middeldorp said. “We created a model about whether and when it would be useful to test for thrombophilia, and based on the model, we suggest it can be appropriate in [the specified] situations.

The panel agreed on 23 recommendations regarding thrombophilia testing and management. Most are based on “very low certainty” in the evidence because of modeling assumptions.

However, the panel agreed on a strong recommendation against testing the general population before starting combined oral contraceptives (COC), and a conditional recommendation for thrombophilia testing in:

• Patients with VTE associated with nonsurgical major transient or hormonal risk factors
• Patients with cerebral or splanchnic venous thrombosis in settings where anticoagulation would otherwise be discontinued
• Individuals with a family history of antithrombin, protein C, or protein S deficiency when considering thromboprophylaxis for minor provoking risk factors and for guidance related to the use of COC or hormone therapy
• Pregnant women with a family history of high-risk thrombophilia types
• Patients with cancer at low or intermediate risk of thrombosis and with a family history of VTE

“In all other instances, we suggest not testing for thrombophilia,” said Dr. Middeldorp.

The ASH guidelines largely mirror those of existing guidelines from a number of other organizations, but the recommendation in favor of testing for thrombophilia in patients with VTE provoked by a nonsurgical major transient risk factor or associated with COCs, hormone therapy, pregnancy or postpartum is new and “may cause considerable discussion, as many currently view these VTE episodes as provoked and are generally inclined to use short-term anticoagulation for such patients,” the guideline authors wrote.

“It is important to note, however, that most guidelines or guidance statements on thrombophilia testing did not distinguish between major and minor provoking risk factors, which current science suggests is appropriate,” they added.

Another novel recommendation is the suggestion to test for hereditary thrombophilia to guide the use of thromboprophylaxis during systemic treatment in ambulatory patients with cancer who are at low or intermediate risk for VTE and who have a family history of VTE.

“This new recommendation should be seen as a new application of an established risk stratification approach,” they said.

Additional research is urgently needed, particularly “large implementation studies comparing the impact, in terms of outcomes rates, among management strategies involving or not involving thrombophilia testing,” they noted.

The guideline was wholly funded by ASH. Dr. Middeldorp reported having no conflicts of interest.

Thrombophilia testing should be limited to specific circumstances, including when venous thromboembolism (VTE) is provoked by nonsurgical risk factors such as pregnancy or oral contraception use, according to new clinical practice guidelines released by the American Society of Hematology. Individuals with a family history of VTE and high-risk thrombophilia, and those with VTE at unusual body sites should also be tested, the guidelines panel agreed.

“These guidelines will potentially change practice – we know that providers and patients will make a shared treatment decision and we wanted to outline specific scenarios to guide that decision,” panel cochair and first author Saskia Middeldorp, MD, PhD, explained in a press release announcing the publication of the guidelines in Blood Advances.

Dr. Middeldorp is a professor of medicine and head of the department of internal medicine at Radboud University Medical Center, Nijmegen, the Netherlands.

The guidelines are the latest in an ASH series of VTE-related guidelines. ASH convened a multidisciplinary panel with clinical and methodological expertise to develop the guidelines, which were subject to public comment, and they “provide recommendations informed by case-based approaches and modeling to ensure the medical community can better diagnose and treat thrombophilia and people with the condition can make the best decisions for their care,” the press release explains.

Thrombophilia affects an estimated 10% of the population. Testing for the clotting disorder can be costly, and the use of testing to help guide treatment decisions is controversial.

“For decades there has been dispute about thrombophilia testing,” Dr. Middeldorp said. “We created a model about whether and when it would be useful to test for thrombophilia, and based on the model, we suggest it can be appropriate in [the specified] situations.

The panel agreed on 23 recommendations regarding thrombophilia testing and management. Most are based on “very low certainty” in the evidence because of modeling assumptions.

However, the panel agreed on a strong recommendation against testing the general population before starting combined oral contraceptives (COC), and a conditional recommendation for thrombophilia testing in:

• Patients with VTE associated with nonsurgical major transient or hormonal risk factors
• Patients with cerebral or splanchnic venous thrombosis in settings where anticoagulation would otherwise be discontinued
• Individuals with a family history of antithrombin, protein C, or protein S deficiency when considering thromboprophylaxis for minor provoking risk factors and for guidance related to the use of COC or hormone therapy
• Pregnant women with a family history of high-risk thrombophilia types
• Patients with cancer at low or intermediate risk of thrombosis and with a family history of VTE

“In all other instances, we suggest not testing for thrombophilia,” said Dr. Middeldorp.

The ASH guidelines largely mirror those of existing guidelines from a number of other organizations, but the recommendation in favor of testing for thrombophilia in patients with VTE provoked by a nonsurgical major transient risk factor or associated with COCs, hormone therapy, pregnancy or postpartum is new and “may cause considerable discussion, as many currently view these VTE episodes as provoked and are generally inclined to use short-term anticoagulation for such patients,” the guideline authors wrote.

“It is important to note, however, that most guidelines or guidance statements on thrombophilia testing did not distinguish between major and minor provoking risk factors, which current science suggests is appropriate,” they added.

Another novel recommendation is the suggestion to test for hereditary thrombophilia to guide the use of thromboprophylaxis during systemic treatment in ambulatory patients with cancer who are at low or intermediate risk for VTE and who have a family history of VTE.

“This new recommendation should be seen as a new application of an established risk stratification approach,” they said.

Additional research is urgently needed, particularly “large implementation studies comparing the impact, in terms of outcomes rates, among management strategies involving or not involving thrombophilia testing,” they noted.

The guideline was wholly funded by ASH. Dr. Middeldorp reported having no conflicts of interest.

Thrombophilia testing should be limited to specific circumstances, including when venous thromboembolism (VTE) is provoked by nonsurgical risk factors such as pregnancy or oral contraception use, according to new clinical practice guidelines released by the American Society of Hematology. Individuals with a family history of VTE and high-risk thrombophilia, and those with VTE at unusual body sites should also be tested, the guidelines panel agreed.

“These guidelines will potentially change practice – we know that providers and patients will make a shared treatment decision and we wanted to outline specific scenarios to guide that decision,” panel cochair and first author Saskia Middeldorp, MD, PhD, explained in a press release announcing the publication of the guidelines in Blood Advances.

Dr. Middeldorp is a professor of medicine and head of the department of internal medicine at Radboud University Medical Center, Nijmegen, the Netherlands.

The guidelines are the latest in an ASH series of VTE-related guidelines. ASH convened a multidisciplinary panel with clinical and methodological expertise to develop the guidelines, which were subject to public comment, and they “provide recommendations informed by case-based approaches and modeling to ensure the medical community can better diagnose and treat thrombophilia and people with the condition can make the best decisions for their care,” the press release explains.

Thrombophilia affects an estimated 10% of the population. Testing for the clotting disorder can be costly, and the use of testing to help guide treatment decisions is controversial.

“For decades there has been dispute about thrombophilia testing,” Dr. Middeldorp said. “We created a model about whether and when it would be useful to test for thrombophilia, and based on the model, we suggest it can be appropriate in [the specified] situations.

The panel agreed on 23 recommendations regarding thrombophilia testing and management. Most are based on “very low certainty” in the evidence because of modeling assumptions.

However, the panel agreed on a strong recommendation against testing the general population before starting combined oral contraceptives (COC), and a conditional recommendation for thrombophilia testing in:

• Patients with VTE associated with nonsurgical major transient or hormonal risk factors
• Patients with cerebral or splanchnic venous thrombosis in settings where anticoagulation would otherwise be discontinued
• Individuals with a family history of antithrombin, protein C, or protein S deficiency when considering thromboprophylaxis for minor provoking risk factors and for guidance related to the use of COC or hormone therapy
• Pregnant women with a family history of high-risk thrombophilia types
• Patients with cancer at low or intermediate risk of thrombosis and with a family history of VTE

“In all other instances, we suggest not testing for thrombophilia,” said Dr. Middeldorp.

The ASH guidelines largely mirror those of existing guidelines from a number of other organizations, but the recommendation in favor of testing for thrombophilia in patients with VTE provoked by a nonsurgical major transient risk factor or associated with COCs, hormone therapy, pregnancy or postpartum is new and “may cause considerable discussion, as many currently view these VTE episodes as provoked and are generally inclined to use short-term anticoagulation for such patients,” the guideline authors wrote.

“It is important to note, however, that most guidelines or guidance statements on thrombophilia testing did not distinguish between major and minor provoking risk factors, which current science suggests is appropriate,” they added.

Another novel recommendation is the suggestion to test for hereditary thrombophilia to guide the use of thromboprophylaxis during systemic treatment in ambulatory patients with cancer who are at low or intermediate risk for VTE and who have a family history of VTE.

“This new recommendation should be seen as a new application of an established risk stratification approach,” they said.

Additional research is urgently needed, particularly “large implementation studies comparing the impact, in terms of outcomes rates, among management strategies involving or not involving thrombophilia testing,” they noted.

The guideline was wholly funded by ASH. Dr. Middeldorp reported having no conflicts of interest.

In a sign of the power of combination therapy for high-risk chronic lymphocytic leukemia (CLL), a small new study showed that patients were more likely to reach undetectable measurable residual disease (U-MRD) when they added venetoclax to their drug regimen after a year or more of taking ibrutinib.

Of 45 patients, 57% reached U-MRD at 12 months, and 55% reached complete remission, according to the study, published in Leukemia.

By adding venetoclax, “you can get very deep remissions in high-risk patients with ibrutinib,” lead author Philip A. Thompson, MBBS, a hematologist-oncologist with the University of Melbourne and Peter MacCallum Cancer Center, also in Melbourne, said in an interview. “This is a significant advance for really high-risk patients.”

According to Dr. Thompson, Bruton’s tyrosine kinase inhibitors like ibrutinib have revolutionized the treatment of high-risk CLL by forcing the disease into remission for several years and allowing patients to avoid stem cell transplants. “But the drug doesn’t eradicate the disease,” he said, “so eventually these patients develop progression.”

The current hope, he said, is to use a combination therapy like ibrutinib and venetoclax to send CLL into remission with lower chance of drug resistance and then allow patients to stop taking the medications.

Previous research has supported the combination of ibrutinib and venetoclax in CLL in the frontline setting, and the European Commission approved it in 2022 for that use. But “ours is the first [study] that looked at patients who’ve been on ibrutinib for a long time and added venetoclax,” Dr. Thompson said. In some cases, he said, patients in the study had been on ibrutinib for several years.

For the new study, researchers at the University of Texas MD Anderson Cancer Center in Houston – where Dr. Thompson used to work – tracked 45 patients (average age, 68.5 years; 51-80 years) with CLL or small lymphocytic lymphoma who had MRD but no clinical disease progression. They all had at least 1 high-risk feature such as a mutated TP53. They’d been on ibrutinib for a median of 32 months (12-73 months), and two were in complete remission but with MRD.

An intention-to-treat analysis found that 71% reached U-MRD when they finished taking venetoclax. “We were actually pleasantly surprised by the high rate of undetectable MRD,” Dr. Thompson said.

At a median 41-month follow-up, 11% of patients had progressed, but none had died of CLL or Richter transformation, a deadly complication of CLL. “The main side effects were neutropenia and diarrhea, which we were manageable,” Dr. Thompson said.

It’s not clear why the drug combination is especially effective, he said, but it may be because the medications are synergistic. According to the National Cancer Institute, synergy in medicine refers to “the interaction of two or more drugs when their combined effect is greater than the sum of the effects seen when each drug is given alone.”

The findings suggest that “you can get deep remissions in high-risk patients with ibrutinib and venetoclax with very with good tolerability and very low risk of on-treatment progression,” Dr. Thompson said. “We don’t yet have enough progression events to talk about retreatment data, but we do feel that retreatment with Bruton’s tyrosine kinase inhibitors – plus or minus venetoclax – will be successful in the vast majority of patients.”

The combination can be given off label in the United States, Dr. Thompson added. As for expense, adding venetoclax will double the cost of ibrutinib. The two drugs are some of the most expensive medications in the world. But patients will save money if they can stop therapy when they reach remission.

In an interview, hematologist-oncologist Kerry A. Rogers, MD, of Ohio State University, Columbus, who is not involved with the study, praised the research: “While small, this study does say quite a bit about this as a strategy to help people discontinue ibrutinib prior to resistance developing.”

She noted that Bruton’s tyrosine kinase inhibitors “are generally given as a continuous monotherapy, and venetoclax is usually given for a fixed duration in combination with an anti-CD20 antibody.”

Going forward, she said, “the fact that the study was in high-risk patients who have the most to gain from such a combination suggests that similar or better rates of undetectable minimal residual disease might be seen in non–high-risk groups. Additional follow-up should be reported as well as use of this strategy in a larger group of patients before this should be considered a standard approach.”

AbbVie funding the study and provided study drugs. MD Anderson Cancer Center conducted the study and discloses funding from the National Cancer Institute. Dr. Thompson reported ties with AbbVie, Pharmacyclics, Lilly, Adaptive Biotechnologies, Janssen, BeiGene, and Genentech. The other study authors reported multiple disclosures. Dr. Rogers disclosed relationships with Genentech, AbbVie, Novartis, Janssen, Pharmacyclics, BeiGene, Lilly, and AstraZeneca.

In a sign of the power of combination therapy for high-risk chronic lymphocytic leukemia (CLL), a small new study showed that patients were more likely to reach undetectable measurable residual disease (U-MRD) when they added venetoclax to their drug regimen after a year or more of taking ibrutinib.

Of 45 patients, 57% reached U-MRD at 12 months, and 55% reached complete remission, according to the study, published in Leukemia.

By adding venetoclax, “you can get very deep remissions in high-risk patients with ibrutinib,” lead author Philip A. Thompson, MBBS, a hematologist-oncologist with the University of Melbourne and Peter MacCallum Cancer Center, also in Melbourne, said in an interview. “This is a significant advance for really high-risk patients.”

According to Dr. Thompson, Bruton’s tyrosine kinase inhibitors like ibrutinib have revolutionized the treatment of high-risk CLL by forcing the disease into remission for several years and allowing patients to avoid stem cell transplants. “But the drug doesn’t eradicate the disease,” he said, “so eventually these patients develop progression.”

The current hope, he said, is to use a combination therapy like ibrutinib and venetoclax to send CLL into remission with lower chance of drug resistance and then allow patients to stop taking the medications.

Previous research has supported the combination of ibrutinib and venetoclax in CLL in the frontline setting, and the European Commission approved it in 2022 for that use. But “ours is the first [study] that looked at patients who’ve been on ibrutinib for a long time and added venetoclax,” Dr. Thompson said. In some cases, he said, patients in the study had been on ibrutinib for several years.

For the new study, researchers at the University of Texas MD Anderson Cancer Center in Houston – where Dr. Thompson used to work – tracked 45 patients (average age, 68.5 years; 51-80 years) with CLL or small lymphocytic lymphoma who had MRD but no clinical disease progression. They all had at least 1 high-risk feature such as a mutated TP53. They’d been on ibrutinib for a median of 32 months (12-73 months), and two were in complete remission but with MRD.

An intention-to-treat analysis found that 71% reached U-MRD when they finished taking venetoclax. “We were actually pleasantly surprised by the high rate of undetectable MRD,” Dr. Thompson said.

At a median 41-month follow-up, 11% of patients had progressed, but none had died of CLL or Richter transformation, a deadly complication of CLL. “The main side effects were neutropenia and diarrhea, which we were manageable,” Dr. Thompson said.

It’s not clear why the drug combination is especially effective, he said, but it may be because the medications are synergistic. According to the National Cancer Institute, synergy in medicine refers to “the interaction of two or more drugs when their combined effect is greater than the sum of the effects seen when each drug is given alone.”

The findings suggest that “you can get deep remissions in high-risk patients with ibrutinib and venetoclax with very with good tolerability and very low risk of on-treatment progression,” Dr. Thompson said. “We don’t yet have enough progression events to talk about retreatment data, but we do feel that retreatment with Bruton’s tyrosine kinase inhibitors – plus or minus venetoclax – will be successful in the vast majority of patients.”

The combination can be given off label in the United States, Dr. Thompson added. As for expense, adding venetoclax will double the cost of ibrutinib. The two drugs are some of the most expensive medications in the world. But patients will save money if they can stop therapy when they reach remission.

In an interview, hematologist-oncologist Kerry A. Rogers, MD, of Ohio State University, Columbus, who is not involved with the study, praised the research: “While small, this study does say quite a bit about this as a strategy to help people discontinue ibrutinib prior to resistance developing.”

She noted that Bruton’s tyrosine kinase inhibitors “are generally given as a continuous monotherapy, and venetoclax is usually given for a fixed duration in combination with an anti-CD20 antibody.”

Going forward, she said, “the fact that the study was in high-risk patients who have the most to gain from such a combination suggests that similar or better rates of undetectable minimal residual disease might be seen in non–high-risk groups. Additional follow-up should be reported as well as use of this strategy in a larger group of patients before this should be considered a standard approach.”

AbbVie funding the study and provided study drugs. MD Anderson Cancer Center conducted the study and discloses funding from the National Cancer Institute. Dr. Thompson reported ties with AbbVie, Pharmacyclics, Lilly, Adaptive Biotechnologies, Janssen, BeiGene, and Genentech. The other study authors reported multiple disclosures. Dr. Rogers disclosed relationships with Genentech, AbbVie, Novartis, Janssen, Pharmacyclics, BeiGene, Lilly, and AstraZeneca.

In a sign of the power of combination therapy for high-risk chronic lymphocytic leukemia (CLL), a small new study showed that patients were more likely to reach undetectable measurable residual disease (U-MRD) when they added venetoclax to their drug regimen after a year or more of taking ibrutinib.

Of 45 patients, 57% reached U-MRD at 12 months, and 55% reached complete remission, according to the study, published in Leukemia.

By adding venetoclax, “you can get very deep remissions in high-risk patients with ibrutinib,” lead author Philip A. Thompson, MBBS, a hematologist-oncologist with the University of Melbourne and Peter MacCallum Cancer Center, also in Melbourne, said in an interview. “This is a significant advance for really high-risk patients.”

According to Dr. Thompson, Bruton’s tyrosine kinase inhibitors like ibrutinib have revolutionized the treatment of high-risk CLL by forcing the disease into remission for several years and allowing patients to avoid stem cell transplants. “But the drug doesn’t eradicate the disease,” he said, “so eventually these patients develop progression.”

The current hope, he said, is to use a combination therapy like ibrutinib and venetoclax to send CLL into remission with lower chance of drug resistance and then allow patients to stop taking the medications.

Previous research has supported the combination of ibrutinib and venetoclax in CLL in the frontline setting, and the European Commission approved it in 2022 for that use. But “ours is the first [study] that looked at patients who’ve been on ibrutinib for a long time and added venetoclax,” Dr. Thompson said. In some cases, he said, patients in the study had been on ibrutinib for several years.

For the new study, researchers at the University of Texas MD Anderson Cancer Center in Houston – where Dr. Thompson used to work – tracked 45 patients (average age, 68.5 years; 51-80 years) with CLL or small lymphocytic lymphoma who had MRD but no clinical disease progression. They all had at least 1 high-risk feature such as a mutated TP53. They’d been on ibrutinib for a median of 32 months (12-73 months), and two were in complete remission but with MRD.

An intention-to-treat analysis found that 71% reached U-MRD when they finished taking venetoclax. “We were actually pleasantly surprised by the high rate of undetectable MRD,” Dr. Thompson said.

At a median 41-month follow-up, 11% of patients had progressed, but none had died of CLL or Richter transformation, a deadly complication of CLL. “The main side effects were neutropenia and diarrhea, which we were manageable,” Dr. Thompson said.

It’s not clear why the drug combination is especially effective, he said, but it may be because the medications are synergistic. According to the National Cancer Institute, synergy in medicine refers to “the interaction of two or more drugs when their combined effect is greater than the sum of the effects seen when each drug is given alone.”

The findings suggest that “you can get deep remissions in high-risk patients with ibrutinib and venetoclax with very with good tolerability and very low risk of on-treatment progression,” Dr. Thompson said. “We don’t yet have enough progression events to talk about retreatment data, but we do feel that retreatment with Bruton’s tyrosine kinase inhibitors – plus or minus venetoclax – will be successful in the vast majority of patients.”

The combination can be given off label in the United States, Dr. Thompson added. As for expense, adding venetoclax will double the cost of ibrutinib. The two drugs are some of the most expensive medications in the world. But patients will save money if they can stop therapy when they reach remission.

In an interview, hematologist-oncologist Kerry A. Rogers, MD, of Ohio State University, Columbus, who is not involved with the study, praised the research: “While small, this study does say quite a bit about this as a strategy to help people discontinue ibrutinib prior to resistance developing.”

She noted that Bruton’s tyrosine kinase inhibitors “are generally given as a continuous monotherapy, and venetoclax is usually given for a fixed duration in combination with an anti-CD20 antibody.”

Going forward, she said, “the fact that the study was in high-risk patients who have the most to gain from such a combination suggests that similar or better rates of undetectable minimal residual disease might be seen in non–high-risk groups. Additional follow-up should be reported as well as use of this strategy in a larger group of patients before this should be considered a standard approach.”

AbbVie funding the study and provided study drugs. MD Anderson Cancer Center conducted the study and discloses funding from the National Cancer Institute. Dr. Thompson reported ties with AbbVie, Pharmacyclics, Lilly, Adaptive Biotechnologies, Janssen, BeiGene, and Genentech. The other study authors reported multiple disclosures. Dr. Rogers disclosed relationships with Genentech, AbbVie, Novartis, Janssen, Pharmacyclics, BeiGene, Lilly, and AstraZeneca.

The U.S. Food and Drug Administration approved epcoritamab-bysp (Epkinly, AbbVie and Genmab) today for adults with relapsed/refractory diffuse large B-cell lymphoma following at least two lines of systemic therapy. This includes patients with DLBCL arising from indolent lymphoma and high-grade B-cell lymphoma.

Epcoritamab is the first subcutaneous bispecific antibody approved for the indication. The biologic simultaneously binds CD3 on cytotoxic T cells to CD20 on lymphomic B cells, inducing T-cell mediated destruction.

“Together with our partner, AbbVie, we recognize the unmet need for safe, effective, and accessible treatments for patients with B-cell malignancies and we believe that epcoritamab has the potential to become a core therapy in this patient population,” Jan van de Winkel, PhD, CEO of Genmab, said in a press release announcing the FDA’s acceptance of its biologic licensing application in November 2022.

A potential competitor, Roche’s bispecific antibody mosunetuzumab (Lunsumio), was approved in December 2022. Mosunetuzumab has the same mechanism of action as epcoritamab but is indicated for relapsed or refractory follicular lymphoma after at least two lines of systemic therapy. A phase 3 trial is currently underway exploring epcoritamab for relapsed or refractory follicular lymphoma as part of combination therapy.

The current approval was based on the open-label phase 2 EPCORE NHL-1 trial conducted by AbbVie and Genmab. The trial’s efficacy population included 148 adults with relapsed or refractory CD20+ large B-cell lymphoma who had received at least two prior lines of therapy, including anti-CD20 therapies. Almost 40% had undergone CAR-T cell therapy.

Epcoritamab was administered initially once weekly, then every 2 weeks, and then every 4 weeks until disease progression or unacceptable toxicity. The trial had no comparator arm.

At a median follow-up of 10.7 months, the overall response rate was 61% and the complete response rate was 38%. At a median follow-up of 9.8 months among responders, the median duration of response was 15.6 months.

The prescribing information comes with a boxed warning for serious or life-threatening cytokine release syndrome and life-threatening or fatal immune effector cell–associated neurotoxicity syndrome. Warnings and precautions include infections and cytopenias.

Among the 157 patients who received epcoritamab in the trial at the recommended dose, grade 1-3 cytokine release syndrome occurred in 51%, immune effector cell–associated neurotoxicity syndrome occurred in 6% (with one fatal case), and 15% experienced serious infections.

The most common grade 3 or 4 events included neutropenia (14.6%), anemia (10.2%), and thrombocytopenia (5.7%).

The FDA recommends epcoritamab be administered subcutaneously in 28-day cycles until disease progression or unacceptable toxicity.

The FDA also noted that “this indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.”
 

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

The U.S. Food and Drug Administration approved epcoritamab-bysp (Epkinly, AbbVie and Genmab) today for adults with relapsed/refractory diffuse large B-cell lymphoma following at least two lines of systemic therapy. This includes patients with DLBCL arising from indolent lymphoma and high-grade B-cell lymphoma.

Epcoritamab is the first subcutaneous bispecific antibody approved for the indication. The biologic simultaneously binds CD3 on cytotoxic T cells to CD20 on lymphomic B cells, inducing T-cell mediated destruction.

“Together with our partner, AbbVie, we recognize the unmet need for safe, effective, and accessible treatments for patients with B-cell malignancies and we believe that epcoritamab has the potential to become a core therapy in this patient population,” Jan van de Winkel, PhD, CEO of Genmab, said in a press release announcing the FDA’s acceptance of its biologic licensing application in November 2022.

A potential competitor, Roche’s bispecific antibody mosunetuzumab (Lunsumio), was approved in December 2022. Mosunetuzumab has the same mechanism of action as epcoritamab but is indicated for relapsed or refractory follicular lymphoma after at least two lines of systemic therapy. A phase 3 trial is currently underway exploring epcoritamab for relapsed or refractory follicular lymphoma as part of combination therapy.

The current approval was based on the open-label phase 2 EPCORE NHL-1 trial conducted by AbbVie and Genmab. The trial’s efficacy population included 148 adults with relapsed or refractory CD20+ large B-cell lymphoma who had received at least two prior lines of therapy, including anti-CD20 therapies. Almost 40% had undergone CAR-T cell therapy.

Epcoritamab was administered initially once weekly, then every 2 weeks, and then every 4 weeks until disease progression or unacceptable toxicity. The trial had no comparator arm.

At a median follow-up of 10.7 months, the overall response rate was 61% and the complete response rate was 38%. At a median follow-up of 9.8 months among responders, the median duration of response was 15.6 months.

The prescribing information comes with a boxed warning for serious or life-threatening cytokine release syndrome and life-threatening or fatal immune effector cell–associated neurotoxicity syndrome. Warnings and precautions include infections and cytopenias.

Among the 157 patients who received epcoritamab in the trial at the recommended dose, grade 1-3 cytokine release syndrome occurred in 51%, immune effector cell–associated neurotoxicity syndrome occurred in 6% (with one fatal case), and 15% experienced serious infections.

The most common grade 3 or 4 events included neutropenia (14.6%), anemia (10.2%), and thrombocytopenia (5.7%).

The FDA recommends epcoritamab be administered subcutaneously in 28-day cycles until disease progression or unacceptable toxicity.

The FDA also noted that “this indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.”
 

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

The U.S. Food and Drug Administration approved epcoritamab-bysp (Epkinly, AbbVie and Genmab) today for adults with relapsed/refractory diffuse large B-cell lymphoma following at least two lines of systemic therapy. This includes patients with DLBCL arising from indolent lymphoma and high-grade B-cell lymphoma.

Epcoritamab is the first subcutaneous bispecific antibody approved for the indication. The biologic simultaneously binds CD3 on cytotoxic T cells to CD20 on lymphomic B cells, inducing T-cell mediated destruction.

“Together with our partner, AbbVie, we recognize the unmet need for safe, effective, and accessible treatments for patients with B-cell malignancies and we believe that epcoritamab has the potential to become a core therapy in this patient population,” Jan van de Winkel, PhD, CEO of Genmab, said in a press release announcing the FDA’s acceptance of its biologic licensing application in November 2022.

A potential competitor, Roche’s bispecific antibody mosunetuzumab (Lunsumio), was approved in December 2022. Mosunetuzumab has the same mechanism of action as epcoritamab but is indicated for relapsed or refractory follicular lymphoma after at least two lines of systemic therapy. A phase 3 trial is currently underway exploring epcoritamab for relapsed or refractory follicular lymphoma as part of combination therapy.

The current approval was based on the open-label phase 2 EPCORE NHL-1 trial conducted by AbbVie and Genmab. The trial’s efficacy population included 148 adults with relapsed or refractory CD20+ large B-cell lymphoma who had received at least two prior lines of therapy, including anti-CD20 therapies. Almost 40% had undergone CAR-T cell therapy.

Epcoritamab was administered initially once weekly, then every 2 weeks, and then every 4 weeks until disease progression or unacceptable toxicity. The trial had no comparator arm.

At a median follow-up of 10.7 months, the overall response rate was 61% and the complete response rate was 38%. At a median follow-up of 9.8 months among responders, the median duration of response was 15.6 months.

The prescribing information comes with a boxed warning for serious or life-threatening cytokine release syndrome and life-threatening or fatal immune effector cell–associated neurotoxicity syndrome. Warnings and precautions include infections and cytopenias.

Among the 157 patients who received epcoritamab in the trial at the recommended dose, grade 1-3 cytokine release syndrome occurred in 51%, immune effector cell–associated neurotoxicity syndrome occurred in 6% (with one fatal case), and 15% experienced serious infections.

The most common grade 3 or 4 events included neutropenia (14.6%), anemia (10.2%), and thrombocytopenia (5.7%).

The FDA recommends epcoritamab be administered subcutaneously in 28-day cycles until disease progression or unacceptable toxicity.

The FDA also noted that “this indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.”
 

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

A new strategy proposed by an international team of experts would limit the use of the prostate-specific antigen (PSA) test for screening tor prostate cancer to men who are younger than 70 years and who are at high risk or symptomatic.

This would reduce potential harms from overdiagnosis and overtreatment, the risk for which is high with the on-demand screening that is the current standard of care in most wealthy nations.

In a paper published online in The BMJ, the panel recommends instead a comprehensive nationwide program that would base PSA testing on individual patient risk and direct those with abnormal results to a managed system of imaging, targeted biopsy only if indicated, and subsequent active monitoring or treatment for those with more aggressive disease features.

Alternatively, government health programs could actively discourage widespread PSA testing and implement policies that would effectively limit PSA-based screening only to men with urologic symptoms warranting further exploration, said the authors, led by Andrew Vickers, PhD, a research epidemiologist at Memorial Sloan Kettering Cancer Center in New York.

“Although we believe that early detection of prostate cancer should involve shared decision making, the current approach of determining testing by shared decision making has resulted in the worst possible practical outcome of high levels of PSA testing and medical harm, with minimal benefit and inequity,” they wrote.

“To make better use of PSA testing, policy makers should choose between a comprehensive, risk adapted approach that is specifically designed to reduce overdiagnosis and overtreatment, or restricting PSA testing to people referred to urologists with symptoms. That choice will need to take into account wider patient and public perspective, as well as health economic concerns,” they continued.
 

Inappropriate testing

Since the Food and Drug Administration approved the first PSA screen in 1986 as a means for monitoring disease progression in patients being treated for prostate cancer, the test has remained controversial, embraced by some for its presumed ability to spot early prostate cancer but scorned by others for its equivocal results in patients with benign prostate pathology and for its potential to lead to overdiagnosis and overtreatment of low-grade disease in men who would otherwise be likely to die of other causes.

Currently, only Lithuania and Kazakhstan have government-supported population-based screening programs for prostate cancer. In contrast, the United States, United Kingdom, and other high-income countries have opted not to implement nationwide prostate cancer screening but allow so-called “informed choice testing,” in which men can receiving PSA screening after discussion with a primary care physician, urologist, or other specialist.

The U.S. Preventive Services Task Force recommends that for men aged 55-69 years, the decision to undergo PSA testing should be an individual one, based on an understanding of the risks and benefits. For men aged 70 or older, the task force flatly states, “Do not screen for prostate cancer.”

But as Dr. Vickers and colleagues noted, “high income countries that have made PSA testing available to men who request it after shared decision making with their physician now have a high prevalence of PSA testing with an inappropriate age distribution.”

For example, they pointed out that in the United Kingdom, men in their 80s are twice as likely as are men in their 50s to get a PSA test, even though men in the older age group are far less likely to have benefit and far more likely to experience harm from treatment. Similarly, in France, nearly one-third of men over 40 get an annual PSA test, with the highest incidence of PSA testing in men over age 70. There are also high rates of PSA testing in men over 70 in Italy, Germany, and Ireland.

“A key problem is that, in current routine care – and despite guidelines to the contrary – most men with an abnormal PSA result have prostate biopsy, even though only a minority will have aggressive prostate cancer,” Dr. Vickers and colleagues wrote. “Furthermore, most men with biopsy-detected cancers have either surgery or radiotherapy (with or without androgen deprivation therapy) even if they have low-risk tumors that are unlikely to cause cancer related morbidity or mortality.”

In addition, informed-choice PSA testing may lead to health inequities, the team noted, citing data from the United States, Canada, and Switzerland showing an inverse association between income and education and the likelihood of PSA testing. Also, in the United States and Canada, men from ethnic minority groups are less likely to have PSA testing.
 

Comprehensive risk-based program

Dr. Vickers and colleagues proposed that a “comprehensive, risk-based prostate cancer detection program based on best evidence on how to use PSA testing and manage subsequent diagnostic follow-up and treatment could reduce overdiagnosis and overtreatment.

“Such a program would restrict testing to men (and those not identifying as male but who have a prostate) aged 50-70, define testing intervals by PSA levels, stop testing early for those with lower PSA, offer biopsy only to those identified as at high risk of aggressive disease after a secondary test (such as magnetic resonance imaging [MRI] or blood markers), and limit treatment to those with high Gleason grade tumors,” they wrote.
 

‘Sound analysis’

Two experts who were not involved with the BMJ paper applaud the suggestions made in comments posted on the U.K. Science Media Centre.

Benjamin W. Lamb, MBBS, MA, PhD, a consultant urologist and surgeon at Barts Health NHS Trust in London, said the analysis conducted by the panel “is sound as there are known benefits from risk-adapted comprehensive screening trials in men aged 50-70, but discordance with current practice, meaning benefits and harms are not those seen in trials.”

However, he also said that the strategies proposed by the authors would be unlikely to prevent older, well-informed men from requesting and getting a PSA test.

“In my view, the emphasis should be on engaging younger and at-risk men rather than restricting access for older men,” he said, noting that the alternative proposal of restricting PSA testing “in my view, is not feasible.”

Nick James, MBBS, PhD, professor of prostate and bladder cancer research at the Institute of Cancer Research, London, and consultant oncologist at the Royal Marsden NHS Foundation Trust, said, “I agree with the authors and strongly support the implementation of a risk-based approach to PSA testing at a national level.

“There is an urgent need for a more equitable and targeted screening strategy, which could help address existing health disparities,” Dr. James said. “Currently, individuals from economically disadvantaged backgrounds are less likely to undergo PSA testing. Men in their 50s or younger, who may stand to benefit more from these tests, are also less likely to receive PSA tests compared to older men who benefit less. Linked to better diagnostic pathways with MRI, already standard in the UK, potential harms from overdiagnosis and overtreatment can be mitigated.”

The analysis was supported in part by the U.S. National Institutes of Health/National Cancer Institute with a Cancer Center Support Grant to Memorial Sloan Kettering Cancer Center, and NIH grants to coauthors. Dr. Vickers is a coinventor of the 4Kscore, a commercial test for predicting prostate biopsy outcome. He receives royalties from sales of the test and owns stock options in OPKO, which offers the test. Coauthor James W.F. Catto, PhD, disclosed ties to Astellas, AstraZeneca, BMS, Ferring, Gilead, Janssen, MSD, Nucleix, Photocure, QED Therapeutics, and Roche.

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

A new strategy proposed by an international team of experts would limit the use of the prostate-specific antigen (PSA) test for screening tor prostate cancer to men who are younger than 70 years and who are at high risk or symptomatic.

This would reduce potential harms from overdiagnosis and overtreatment, the risk for which is high with the on-demand screening that is the current standard of care in most wealthy nations.

In a paper published online in The BMJ, the panel recommends instead a comprehensive nationwide program that would base PSA testing on individual patient risk and direct those with abnormal results to a managed system of imaging, targeted biopsy only if indicated, and subsequent active monitoring or treatment for those with more aggressive disease features.

Alternatively, government health programs could actively discourage widespread PSA testing and implement policies that would effectively limit PSA-based screening only to men with urologic symptoms warranting further exploration, said the authors, led by Andrew Vickers, PhD, a research epidemiologist at Memorial Sloan Kettering Cancer Center in New York.

“Although we believe that early detection of prostate cancer should involve shared decision making, the current approach of determining testing by shared decision making has resulted in the worst possible practical outcome of high levels of PSA testing and medical harm, with minimal benefit and inequity,” they wrote.

“To make better use of PSA testing, policy makers should choose between a comprehensive, risk adapted approach that is specifically designed to reduce overdiagnosis and overtreatment, or restricting PSA testing to people referred to urologists with symptoms. That choice will need to take into account wider patient and public perspective, as well as health economic concerns,” they continued.
 

Inappropriate testing

Since the Food and Drug Administration approved the first PSA screen in 1986 as a means for monitoring disease progression in patients being treated for prostate cancer, the test has remained controversial, embraced by some for its presumed ability to spot early prostate cancer but scorned by others for its equivocal results in patients with benign prostate pathology and for its potential to lead to overdiagnosis and overtreatment of low-grade disease in men who would otherwise be likely to die of other causes.

Currently, only Lithuania and Kazakhstan have government-supported population-based screening programs for prostate cancer. In contrast, the United States, United Kingdom, and other high-income countries have opted not to implement nationwide prostate cancer screening but allow so-called “informed choice testing,” in which men can receiving PSA screening after discussion with a primary care physician, urologist, or other specialist.

The U.S. Preventive Services Task Force recommends that for men aged 55-69 years, the decision to undergo PSA testing should be an individual one, based on an understanding of the risks and benefits. For men aged 70 or older, the task force flatly states, “Do not screen for prostate cancer.”

But as Dr. Vickers and colleagues noted, “high income countries that have made PSA testing available to men who request it after shared decision making with their physician now have a high prevalence of PSA testing with an inappropriate age distribution.”

For example, they pointed out that in the United Kingdom, men in their 80s are twice as likely as are men in their 50s to get a PSA test, even though men in the older age group are far less likely to have benefit and far more likely to experience harm from treatment. Similarly, in France, nearly one-third of men over 40 get an annual PSA test, with the highest incidence of PSA testing in men over age 70. There are also high rates of PSA testing in men over 70 in Italy, Germany, and Ireland.

“A key problem is that, in current routine care – and despite guidelines to the contrary – most men with an abnormal PSA result have prostate biopsy, even though only a minority will have aggressive prostate cancer,” Dr. Vickers and colleagues wrote. “Furthermore, most men with biopsy-detected cancers have either surgery or radiotherapy (with or without androgen deprivation therapy) even if they have low-risk tumors that are unlikely to cause cancer related morbidity or mortality.”

In addition, informed-choice PSA testing may lead to health inequities, the team noted, citing data from the United States, Canada, and Switzerland showing an inverse association between income and education and the likelihood of PSA testing. Also, in the United States and Canada, men from ethnic minority groups are less likely to have PSA testing.
 

Comprehensive risk-based program

Dr. Vickers and colleagues proposed that a “comprehensive, risk-based prostate cancer detection program based on best evidence on how to use PSA testing and manage subsequent diagnostic follow-up and treatment could reduce overdiagnosis and overtreatment.

“Such a program would restrict testing to men (and those not identifying as male but who have a prostate) aged 50-70, define testing intervals by PSA levels, stop testing early for those with lower PSA, offer biopsy only to those identified as at high risk of aggressive disease after a secondary test (such as magnetic resonance imaging [MRI] or blood markers), and limit treatment to those with high Gleason grade tumors,” they wrote.
 

‘Sound analysis’

Two experts who were not involved with the BMJ paper applaud the suggestions made in comments posted on the U.K. Science Media Centre.

Benjamin W. Lamb, MBBS, MA, PhD, a consultant urologist and surgeon at Barts Health NHS Trust in London, said the analysis conducted by the panel “is sound as there are known benefits from risk-adapted comprehensive screening trials in men aged 50-70, but discordance with current practice, meaning benefits and harms are not those seen in trials.”

However, he also said that the strategies proposed by the authors would be unlikely to prevent older, well-informed men from requesting and getting a PSA test.

“In my view, the emphasis should be on engaging younger and at-risk men rather than restricting access for older men,” he said, noting that the alternative proposal of restricting PSA testing “in my view, is not feasible.”

Nick James, MBBS, PhD, professor of prostate and bladder cancer research at the Institute of Cancer Research, London, and consultant oncologist at the Royal Marsden NHS Foundation Trust, said, “I agree with the authors and strongly support the implementation of a risk-based approach to PSA testing at a national level.

“There is an urgent need for a more equitable and targeted screening strategy, which could help address existing health disparities,” Dr. James said. “Currently, individuals from economically disadvantaged backgrounds are less likely to undergo PSA testing. Men in their 50s or younger, who may stand to benefit more from these tests, are also less likely to receive PSA tests compared to older men who benefit less. Linked to better diagnostic pathways with MRI, already standard in the UK, potential harms from overdiagnosis and overtreatment can be mitigated.”

The analysis was supported in part by the U.S. National Institutes of Health/National Cancer Institute with a Cancer Center Support Grant to Memorial Sloan Kettering Cancer Center, and NIH grants to coauthors. Dr. Vickers is a coinventor of the 4Kscore, a commercial test for predicting prostate biopsy outcome. He receives royalties from sales of the test and owns stock options in OPKO, which offers the test. Coauthor James W.F. Catto, PhD, disclosed ties to Astellas, AstraZeneca, BMS, Ferring, Gilead, Janssen, MSD, Nucleix, Photocure, QED Therapeutics, and Roche.

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

A new strategy proposed by an international team of experts would limit the use of the prostate-specific antigen (PSA) test for screening tor prostate cancer to men who are younger than 70 years and who are at high risk or symptomatic.

This would reduce potential harms from overdiagnosis and overtreatment, the risk for which is high with the on-demand screening that is the current standard of care in most wealthy nations.

In a paper published online in The BMJ, the panel recommends instead a comprehensive nationwide program that would base PSA testing on individual patient risk and direct those with abnormal results to a managed system of imaging, targeted biopsy only if indicated, and subsequent active monitoring or treatment for those with more aggressive disease features.

Alternatively, government health programs could actively discourage widespread PSA testing and implement policies that would effectively limit PSA-based screening only to men with urologic symptoms warranting further exploration, said the authors, led by Andrew Vickers, PhD, a research epidemiologist at Memorial Sloan Kettering Cancer Center in New York.

“Although we believe that early detection of prostate cancer should involve shared decision making, the current approach of determining testing by shared decision making has resulted in the worst possible practical outcome of high levels of PSA testing and medical harm, with minimal benefit and inequity,” they wrote.

“To make better use of PSA testing, policy makers should choose between a comprehensive, risk adapted approach that is specifically designed to reduce overdiagnosis and overtreatment, or restricting PSA testing to people referred to urologists with symptoms. That choice will need to take into account wider patient and public perspective, as well as health economic concerns,” they continued.
 

Inappropriate testing

Since the Food and Drug Administration approved the first PSA screen in 1986 as a means for monitoring disease progression in patients being treated for prostate cancer, the test has remained controversial, embraced by some for its presumed ability to spot early prostate cancer but scorned by others for its equivocal results in patients with benign prostate pathology and for its potential to lead to overdiagnosis and overtreatment of low-grade disease in men who would otherwise be likely to die of other causes.

Currently, only Lithuania and Kazakhstan have government-supported population-based screening programs for prostate cancer. In contrast, the United States, United Kingdom, and other high-income countries have opted not to implement nationwide prostate cancer screening but allow so-called “informed choice testing,” in which men can receiving PSA screening after discussion with a primary care physician, urologist, or other specialist.

The U.S. Preventive Services Task Force recommends that for men aged 55-69 years, the decision to undergo PSA testing should be an individual one, based on an understanding of the risks and benefits. For men aged 70 or older, the task force flatly states, “Do not screen for prostate cancer.”

But as Dr. Vickers and colleagues noted, “high income countries that have made PSA testing available to men who request it after shared decision making with their physician now have a high prevalence of PSA testing with an inappropriate age distribution.”

For example, they pointed out that in the United Kingdom, men in their 80s are twice as likely as are men in their 50s to get a PSA test, even though men in the older age group are far less likely to have benefit and far more likely to experience harm from treatment. Similarly, in France, nearly one-third of men over 40 get an annual PSA test, with the highest incidence of PSA testing in men over age 70. There are also high rates of PSA testing in men over 70 in Italy, Germany, and Ireland.

“A key problem is that, in current routine care – and despite guidelines to the contrary – most men with an abnormal PSA result have prostate biopsy, even though only a minority will have aggressive prostate cancer,” Dr. Vickers and colleagues wrote. “Furthermore, most men with biopsy-detected cancers have either surgery or radiotherapy (with or without androgen deprivation therapy) even if they have low-risk tumors that are unlikely to cause cancer related morbidity or mortality.”

In addition, informed-choice PSA testing may lead to health inequities, the team noted, citing data from the United States, Canada, and Switzerland showing an inverse association between income and education and the likelihood of PSA testing. Also, in the United States and Canada, men from ethnic minority groups are less likely to have PSA testing.
 

Comprehensive risk-based program

Dr. Vickers and colleagues proposed that a “comprehensive, risk-based prostate cancer detection program based on best evidence on how to use PSA testing and manage subsequent diagnostic follow-up and treatment could reduce overdiagnosis and overtreatment.

“Such a program would restrict testing to men (and those not identifying as male but who have a prostate) aged 50-70, define testing intervals by PSA levels, stop testing early for those with lower PSA, offer biopsy only to those identified as at high risk of aggressive disease after a secondary test (such as magnetic resonance imaging [MRI] or blood markers), and limit treatment to those with high Gleason grade tumors,” they wrote.
 

‘Sound analysis’

Two experts who were not involved with the BMJ paper applaud the suggestions made in comments posted on the U.K. Science Media Centre.

Benjamin W. Lamb, MBBS, MA, PhD, a consultant urologist and surgeon at Barts Health NHS Trust in London, said the analysis conducted by the panel “is sound as there are known benefits from risk-adapted comprehensive screening trials in men aged 50-70, but discordance with current practice, meaning benefits and harms are not those seen in trials.”

However, he also said that the strategies proposed by the authors would be unlikely to prevent older, well-informed men from requesting and getting a PSA test.

“In my view, the emphasis should be on engaging younger and at-risk men rather than restricting access for older men,” he said, noting that the alternative proposal of restricting PSA testing “in my view, is not feasible.”

Nick James, MBBS, PhD, professor of prostate and bladder cancer research at the Institute of Cancer Research, London, and consultant oncologist at the Royal Marsden NHS Foundation Trust, said, “I agree with the authors and strongly support the implementation of a risk-based approach to PSA testing at a national level.

“There is an urgent need for a more equitable and targeted screening strategy, which could help address existing health disparities,” Dr. James said. “Currently, individuals from economically disadvantaged backgrounds are less likely to undergo PSA testing. Men in their 50s or younger, who may stand to benefit more from these tests, are also less likely to receive PSA tests compared to older men who benefit less. Linked to better diagnostic pathways with MRI, already standard in the UK, potential harms from overdiagnosis and overtreatment can be mitigated.”

The analysis was supported in part by the U.S. National Institutes of Health/National Cancer Institute with a Cancer Center Support Grant to Memorial Sloan Kettering Cancer Center, and NIH grants to coauthors. Dr. Vickers is a coinventor of the 4Kscore, a commercial test for predicting prostate biopsy outcome. He receives royalties from sales of the test and owns stock options in OPKO, which offers the test. Coauthor James W.F. Catto, PhD, disclosed ties to Astellas, AstraZeneca, BMS, Ferring, Gilead, Janssen, MSD, Nucleix, Photocure, QED Therapeutics, and Roche.

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

AURORA, COLO. – How often do you talk with patients about how to lower their out-of-pocket costs for medical care?

For most clinicians, the answer is: not often enough. But having those conversations can improve medication adherence and strengthen the patient-clinician relationship, according to panelists at the annual meeting of the Society of General Internal Medicine.

The inverse association between out-of-pocket expenditures and fidelity to prescriptions is clear. A 2020 study by the IQVIA Institute for Human Data Science, for example, found that rates of prescription abandonment are less than 5% when a given medication carries no out-of-pocket cost for patients. That figure rises to 45% when the cost is more than $125, and to 60% when it exceeds $500. One in five Americans said cost prevented them from adhering to medication regimens, according to a new study in JAMA Network Open.

The researchers surveyed more than 2,000 men and women, 40.4% of whom were aged 75 or older. They found that nearly 90% of respondents said they would not be uncomfortable being asked about drug costs before a visit with a physician. A similar share (89.5%) said they would welcome the use by their physician of a real-time tool to determine the cost of their medication.

But the survey results contained a note of warning for clinicians: A significant number of respondents said they would be “extremely” upset if the cost of their medication exceeded the estimate from the pricing tool. And many also said they would be “moderately” or “extremely” angry if their physician used a pricing tool but failed to share the results with them.

“Real-time benefit tools may support medication cost conversations and cost-conscious prescribing, and patients are enthusiastic about their use,” the authors write. “However, if disclosed prices are inaccurate, there is potential for harm through loss of confidence in the physician and nonadherence to prescribed medications.”

While having conversations about cost can be difficult for both clinicians and patients, studies have shown that patients who discuss cost concerns with their doctors feel as if they have stronger relationships as a result.

Clinicians often avoid conversations about out-of-pocket expenses because they don’t know specific price information, they lack solutions to address cost, or they are uncomfortable bringing up the issue.

One member of the audience at the SGIM meeting recalled a patient who worked in a warehouse for a large company. The man, who had type 2 diabetes, had medical insurance, but even with insurance, insulin was going to cost him $150 per month. He struggled to afford the necessary treatment.

“He looked at me and said, ‘What do they want me to do? Do they want me to actually not be able to work for them and not manage my diabetes?’ ”

The clinician said he offered empathy in the moment but felt he could do little else.

Panelists acknowledged that clinicians are crunched on time when seeing patients, but being willing to initiate conversations about cost with patients and to offer resources can help patients get necessary treatment.
 

Start the conversation

Panel member Caroline Sloan, MD, an assistant professor of medicine at Duke University, Durham, N.C., said making patients aware that you know cost can make a big difference.

The American College of Physicians advises clinicians to ask patients whether they are worried about the cost of care and to not assume which patients may have concerns.

The conversation could be started like this: “I’d like to discuss any concerns you might have about the cost of your health care.”

Normalize the concern by making it more general, and reassure your patient that your goal is to get them the best care. Say something like, “I’ve heard from many patients the cost of medications or tests is becoming hard to manage.”

Once a patient’s concerns are clear, you can direct them to resources for assistance in reducing their costs, Dr. Sloan said, such as ClearHealthCosts, FAIR Health, Healthcare Bluebook, New Choice Health, GoodRx, PharmacyChecker, HealthWell Foundation, Patient Advocate Foundation, Good Days, Good Health Will, Mercy Medical Angels, and the American Association of Family Physicians Neighborhood Navigator.

Dr. Sloan said she knows clinicians don’t have time to understand every insurance plan and other issues related to cost. “But at least know to ask about costs,” she said. “Practice, practice, practice. It feels awkward at first, but it gets easier every time.”

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

AURORA, COLO. – How often do you talk with patients about how to lower their out-of-pocket costs for medical care?

For most clinicians, the answer is: not often enough. But having those conversations can improve medication adherence and strengthen the patient-clinician relationship, according to panelists at the annual meeting of the Society of General Internal Medicine.

The inverse association between out-of-pocket expenditures and fidelity to prescriptions is clear. A 2020 study by the IQVIA Institute for Human Data Science, for example, found that rates of prescription abandonment are less than 5% when a given medication carries no out-of-pocket cost for patients. That figure rises to 45% when the cost is more than $125, and to 60% when it exceeds $500. One in five Americans said cost prevented them from adhering to medication regimens, according to a new study in JAMA Network Open.

The researchers surveyed more than 2,000 men and women, 40.4% of whom were aged 75 or older. They found that nearly 90% of respondents said they would not be uncomfortable being asked about drug costs before a visit with a physician. A similar share (89.5%) said they would welcome the use by their physician of a real-time tool to determine the cost of their medication.

But the survey results contained a note of warning for clinicians: A significant number of respondents said they would be “extremely” upset if the cost of their medication exceeded the estimate from the pricing tool. And many also said they would be “moderately” or “extremely” angry if their physician used a pricing tool but failed to share the results with them.

“Real-time benefit tools may support medication cost conversations and cost-conscious prescribing, and patients are enthusiastic about their use,” the authors write. “However, if disclosed prices are inaccurate, there is potential for harm through loss of confidence in the physician and nonadherence to prescribed medications.”

While having conversations about cost can be difficult for both clinicians and patients, studies have shown that patients who discuss cost concerns with their doctors feel as if they have stronger relationships as a result.

Clinicians often avoid conversations about out-of-pocket expenses because they don’t know specific price information, they lack solutions to address cost, or they are uncomfortable bringing up the issue.

One member of the audience at the SGIM meeting recalled a patient who worked in a warehouse for a large company. The man, who had type 2 diabetes, had medical insurance, but even with insurance, insulin was going to cost him $150 per month. He struggled to afford the necessary treatment.

“He looked at me and said, ‘What do they want me to do? Do they want me to actually not be able to work for them and not manage my diabetes?’ ”

The clinician said he offered empathy in the moment but felt he could do little else.

Panelists acknowledged that clinicians are crunched on time when seeing patients, but being willing to initiate conversations about cost with patients and to offer resources can help patients get necessary treatment.
 

Start the conversation

Panel member Caroline Sloan, MD, an assistant professor of medicine at Duke University, Durham, N.C., said making patients aware that you know cost can make a big difference.

The American College of Physicians advises clinicians to ask patients whether they are worried about the cost of care and to not assume which patients may have concerns.

The conversation could be started like this: “I’d like to discuss any concerns you might have about the cost of your health care.”

Normalize the concern by making it more general, and reassure your patient that your goal is to get them the best care. Say something like, “I’ve heard from many patients the cost of medications or tests is becoming hard to manage.”

Once a patient’s concerns are clear, you can direct them to resources for assistance in reducing their costs, Dr. Sloan said, such as ClearHealthCosts, FAIR Health, Healthcare Bluebook, New Choice Health, GoodRx, PharmacyChecker, HealthWell Foundation, Patient Advocate Foundation, Good Days, Good Health Will, Mercy Medical Angels, and the American Association of Family Physicians Neighborhood Navigator.

Dr. Sloan said she knows clinicians don’t have time to understand every insurance plan and other issues related to cost. “But at least know to ask about costs,” she said. “Practice, practice, practice. It feels awkward at first, but it gets easier every time.”

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

AURORA, COLO. – How often do you talk with patients about how to lower their out-of-pocket costs for medical care?

For most clinicians, the answer is: not often enough. But having those conversations can improve medication adherence and strengthen the patient-clinician relationship, according to panelists at the annual meeting of the Society of General Internal Medicine.

The inverse association between out-of-pocket expenditures and fidelity to prescriptions is clear. A 2020 study by the IQVIA Institute for Human Data Science, for example, found that rates of prescription abandonment are less than 5% when a given medication carries no out-of-pocket cost for patients. That figure rises to 45% when the cost is more than $125, and to 60% when it exceeds $500. One in five Americans said cost prevented them from adhering to medication regimens, according to a new study in JAMA Network Open.

The researchers surveyed more than 2,000 men and women, 40.4% of whom were aged 75 or older. They found that nearly 90% of respondents said they would not be uncomfortable being asked about drug costs before a visit with a physician. A similar share (89.5%) said they would welcome the use by their physician of a real-time tool to determine the cost of their medication.

But the survey results contained a note of warning for clinicians: A significant number of respondents said they would be “extremely” upset if the cost of their medication exceeded the estimate from the pricing tool. And many also said they would be “moderately” or “extremely” angry if their physician used a pricing tool but failed to share the results with them.

“Real-time benefit tools may support medication cost conversations and cost-conscious prescribing, and patients are enthusiastic about their use,” the authors write. “However, if disclosed prices are inaccurate, there is potential for harm through loss of confidence in the physician and nonadherence to prescribed medications.”

While having conversations about cost can be difficult for both clinicians and patients, studies have shown that patients who discuss cost concerns with their doctors feel as if they have stronger relationships as a result.

Clinicians often avoid conversations about out-of-pocket expenses because they don’t know specific price information, they lack solutions to address cost, or they are uncomfortable bringing up the issue.

One member of the audience at the SGIM meeting recalled a patient who worked in a warehouse for a large company. The man, who had type 2 diabetes, had medical insurance, but even with insurance, insulin was going to cost him $150 per month. He struggled to afford the necessary treatment.

“He looked at me and said, ‘What do they want me to do? Do they want me to actually not be able to work for them and not manage my diabetes?’ ”

The clinician said he offered empathy in the moment but felt he could do little else.

Panelists acknowledged that clinicians are crunched on time when seeing patients, but being willing to initiate conversations about cost with patients and to offer resources can help patients get necessary treatment.
 

Start the conversation

Panel member Caroline Sloan, MD, an assistant professor of medicine at Duke University, Durham, N.C., said making patients aware that you know cost can make a big difference.

The American College of Physicians advises clinicians to ask patients whether they are worried about the cost of care and to not assume which patients may have concerns.

The conversation could be started like this: “I’d like to discuss any concerns you might have about the cost of your health care.”

Normalize the concern by making it more general, and reassure your patient that your goal is to get them the best care. Say something like, “I’ve heard from many patients the cost of medications or tests is becoming hard to manage.”

Once a patient’s concerns are clear, you can direct them to resources for assistance in reducing their costs, Dr. Sloan said, such as ClearHealthCosts, FAIR Health, Healthcare Bluebook, New Choice Health, GoodRx, PharmacyChecker, HealthWell Foundation, Patient Advocate Foundation, Good Days, Good Health Will, Mercy Medical Angels, and the American Association of Family Physicians Neighborhood Navigator.

Dr. Sloan said she knows clinicians don’t have time to understand every insurance plan and other issues related to cost. “But at least know to ask about costs,” she said. “Practice, practice, practice. It feels awkward at first, but it gets easier every time.”

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

Drug shortages in the United States hit a 10-year high in the first quarter of 2023, according to data from the American Society of Health-System Pharmacists (ASHP). Among the top five drug classes affected by shortages are chemotherapy drugs used in the treatment of cancer, many of which do not have alternatives.

“The shortage of certain cancer drugs has become a serious and life-threatening issue for cancer patients across the country,” Karen E. Knudsen, MBA, PhD, chief executive officer of the American Cancer Society (ACS) and its advocacy affiliate, the American Cancer Society Cancer Action Network (ACS CAN), said in a statement. “I have heard from patients and practitioners who are directly experiencing the impact of these shortages.”

As of early May, there were 15 oncology drugs on the official Food and Drug Administration drug shortage list. The other top drug classes on shortage include drugs used for central nervous system (CNS) disorders,  antimicrobials, fluids and electrolytes, and hormones.

Factors blamed for the current shortages include expanded demand, supply shortages, limited manufacturing capacity, and low profit margins for generic therapies.

Dr. Knudsen emphasized that several of the oncology drugs now in short supply do not have an effective alternative. “As first-line treatments for a number of cancers, including triple-negative breast cancer, ovarian cancer, and leukemia often experienced by pediatric cancer patients, the shortage could lead to delays in treatment that could result in worse outcomes,” she said.

The ACS has listed the following oncology drugs and supportive agents as being in short supply: carboplatin injection used to treat triple negative breast cancer, ovarian, head, and neck cancers; fludarabine phosphate injection used for treating B-cell chronic lymphocytic leukemia; dacarbazine injection for treatment of skin cancer; amifostine injection; azacitidine injection; capecitabine tablets; cisplatin injection; cytarabine injection; dexamethasone sodium phosphate injection; hydrocortisone sodium succinate injection; leucovorin calcium lyophilized powder for injection; Lutetium Lu 177 vipivotide tetraxetan (Pluvicto) injection; methotrexate injection; pentostatin injection; and streptozocin (Zanosar) sterile powder.

Many of these drugs, such as cisplatin, are used in multiple regimens, so the issue is not limited to one specific cancer type.

In addition to these drugs, many products used in cancer care such as intravenous saline solutions are also in short supply, the ACS noted.
 

Two decades of shortages

Drug shortages in the United States have been a chronic problem for more than 2 decades, waxing and waning in intensity. In March, a hearing on drug shortages held by the Senate Committee on Homeland Security and Governmental Affairs noted that since 2001, the number of new drug shortages has ranged between 58 (in 2004) and 267 (in 2011). The trend toward new drug shortages declined from 2018 through 2021, but then rose to 160 in 2022.

The report further noted that the first quarter of 2023 marked the highest number of ongoing shortages by quarter since early 2018, with 301 active shortages as of March 31. For some drugs, the problem has become chronic, as more than 15 critical drug products have been in short supply for more than a decade, and 20 have been in shortage since at least 2015.

A 2022 survey of oncology pharmacists at 68 organizations nationwide showed that 63% of institutions reported one or more drug shortages every month, with a 34% increase in 2019, compared with 2018. Treatment delays, reduced doses, or alternative regimens were reported by 75% of respondents, the authors wrote.

Dr. Knudsen noted that the FDA is largely limited to working directly with the manufacturer on whatever issue is causing the shortage, as well as working with other manufacturers of the same product to urge them to ramp up production. 

“ACS CAN is urging Congress to look at longer-term solutions that change the fundamental underpinnings of the shortages,” she said. “In the meantime, we urge the industry to work with medical practitioners to help identify alternatives where possible to ensure that cancer patients’ treatments are not delayed.”
 

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

Drug shortages in the United States hit a 10-year high in the first quarter of 2023, according to data from the American Society of Health-System Pharmacists (ASHP). Among the top five drug classes affected by shortages are chemotherapy drugs used in the treatment of cancer, many of which do not have alternatives.

“The shortage of certain cancer drugs has become a serious and life-threatening issue for cancer patients across the country,” Karen E. Knudsen, MBA, PhD, chief executive officer of the American Cancer Society (ACS) and its advocacy affiliate, the American Cancer Society Cancer Action Network (ACS CAN), said in a statement. “I have heard from patients and practitioners who are directly experiencing the impact of these shortages.”

As of early May, there were 15 oncology drugs on the official Food and Drug Administration drug shortage list. The other top drug classes on shortage include drugs used for central nervous system (CNS) disorders,  antimicrobials, fluids and electrolytes, and hormones.

Factors blamed for the current shortages include expanded demand, supply shortages, limited manufacturing capacity, and low profit margins for generic therapies.

Dr. Knudsen emphasized that several of the oncology drugs now in short supply do not have an effective alternative. “As first-line treatments for a number of cancers, including triple-negative breast cancer, ovarian cancer, and leukemia often experienced by pediatric cancer patients, the shortage could lead to delays in treatment that could result in worse outcomes,” she said.

The ACS has listed the following oncology drugs and supportive agents as being in short supply: carboplatin injection used to treat triple negative breast cancer, ovarian, head, and neck cancers; fludarabine phosphate injection used for treating B-cell chronic lymphocytic leukemia; dacarbazine injection for treatment of skin cancer; amifostine injection; azacitidine injection; capecitabine tablets; cisplatin injection; cytarabine injection; dexamethasone sodium phosphate injection; hydrocortisone sodium succinate injection; leucovorin calcium lyophilized powder for injection; Lutetium Lu 177 vipivotide tetraxetan (Pluvicto) injection; methotrexate injection; pentostatin injection; and streptozocin (Zanosar) sterile powder.

Many of these drugs, such as cisplatin, are used in multiple regimens, so the issue is not limited to one specific cancer type.

In addition to these drugs, many products used in cancer care such as intravenous saline solutions are also in short supply, the ACS noted.
 

Two decades of shortages

Drug shortages in the United States have been a chronic problem for more than 2 decades, waxing and waning in intensity. In March, a hearing on drug shortages held by the Senate Committee on Homeland Security and Governmental Affairs noted that since 2001, the number of new drug shortages has ranged between 58 (in 2004) and 267 (in 2011). The trend toward new drug shortages declined from 2018 through 2021, but then rose to 160 in 2022.

The report further noted that the first quarter of 2023 marked the highest number of ongoing shortages by quarter since early 2018, with 301 active shortages as of March 31. For some drugs, the problem has become chronic, as more than 15 critical drug products have been in short supply for more than a decade, and 20 have been in shortage since at least 2015.

A 2022 survey of oncology pharmacists at 68 organizations nationwide showed that 63% of institutions reported one or more drug shortages every month, with a 34% increase in 2019, compared with 2018. Treatment delays, reduced doses, or alternative regimens were reported by 75% of respondents, the authors wrote.

Dr. Knudsen noted that the FDA is largely limited to working directly with the manufacturer on whatever issue is causing the shortage, as well as working with other manufacturers of the same product to urge them to ramp up production. 

“ACS CAN is urging Congress to look at longer-term solutions that change the fundamental underpinnings of the shortages,” she said. “In the meantime, we urge the industry to work with medical practitioners to help identify alternatives where possible to ensure that cancer patients’ treatments are not delayed.”
 

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

Drug shortages in the United States hit a 10-year high in the first quarter of 2023, according to data from the American Society of Health-System Pharmacists (ASHP). Among the top five drug classes affected by shortages are chemotherapy drugs used in the treatment of cancer, many of which do not have alternatives.

“The shortage of certain cancer drugs has become a serious and life-threatening issue for cancer patients across the country,” Karen E. Knudsen, MBA, PhD, chief executive officer of the American Cancer Society (ACS) and its advocacy affiliate, the American Cancer Society Cancer Action Network (ACS CAN), said in a statement. “I have heard from patients and practitioners who are directly experiencing the impact of these shortages.”

As of early May, there were 15 oncology drugs on the official Food and Drug Administration drug shortage list. The other top drug classes on shortage include drugs used for central nervous system (CNS) disorders,  antimicrobials, fluids and electrolytes, and hormones.

Factors blamed for the current shortages include expanded demand, supply shortages, limited manufacturing capacity, and low profit margins for generic therapies.

Dr. Knudsen emphasized that several of the oncology drugs now in short supply do not have an effective alternative. “As first-line treatments for a number of cancers, including triple-negative breast cancer, ovarian cancer, and leukemia often experienced by pediatric cancer patients, the shortage could lead to delays in treatment that could result in worse outcomes,” she said.

The ACS has listed the following oncology drugs and supportive agents as being in short supply: carboplatin injection used to treat triple negative breast cancer, ovarian, head, and neck cancers; fludarabine phosphate injection used for treating B-cell chronic lymphocytic leukemia; dacarbazine injection for treatment of skin cancer; amifostine injection; azacitidine injection; capecitabine tablets; cisplatin injection; cytarabine injection; dexamethasone sodium phosphate injection; hydrocortisone sodium succinate injection; leucovorin calcium lyophilized powder for injection; Lutetium Lu 177 vipivotide tetraxetan (Pluvicto) injection; methotrexate injection; pentostatin injection; and streptozocin (Zanosar) sterile powder.

Many of these drugs, such as cisplatin, are used in multiple regimens, so the issue is not limited to one specific cancer type.

In addition to these drugs, many products used in cancer care such as intravenous saline solutions are also in short supply, the ACS noted.
 

Two decades of shortages

Drug shortages in the United States have been a chronic problem for more than 2 decades, waxing and waning in intensity. In March, a hearing on drug shortages held by the Senate Committee on Homeland Security and Governmental Affairs noted that since 2001, the number of new drug shortages has ranged between 58 (in 2004) and 267 (in 2011). The trend toward new drug shortages declined from 2018 through 2021, but then rose to 160 in 2022.

The report further noted that the first quarter of 2023 marked the highest number of ongoing shortages by quarter since early 2018, with 301 active shortages as of March 31. For some drugs, the problem has become chronic, as more than 15 critical drug products have been in short supply for more than a decade, and 20 have been in shortage since at least 2015.

A 2022 survey of oncology pharmacists at 68 organizations nationwide showed that 63% of institutions reported one or more drug shortages every month, with a 34% increase in 2019, compared with 2018. Treatment delays, reduced doses, or alternative regimens were reported by 75% of respondents, the authors wrote.

Dr. Knudsen noted that the FDA is largely limited to working directly with the manufacturer on whatever issue is causing the shortage, as well as working with other manufacturers of the same product to urge them to ramp up production. 

“ACS CAN is urging Congress to look at longer-term solutions that change the fundamental underpinnings of the shortages,” she said. “In the meantime, we urge the industry to work with medical practitioners to help identify alternatives where possible to ensure that cancer patients’ treatments are not delayed.”
 

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