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Specialty and age may contribute to suicidal thoughts among physicians
A physician’s specialty can make a difference when it comes to having suicidal thoughts. Doctors who specialize in family medicine, obstetrics-gynecology, and psychiatry reported double the rates of suicidal thoughts than doctors in oncology, rheumatology, and pulmonary medicine, according to Doctors’ Burden: Medscape Physician Suicide Report 2023.
“The specialties with the highest reporting of physician suicidal thoughts are also those with the greatest physician shortages, based on the number of job openings posted by recruiting sites,” said Peter Yellowlees, MD, professor of psychiatry and chief wellness officer at UC Davis Health.
Doctors in those specialties are overworked, which can lead to burnout, he said.
There’s also a generational divide among physicians who reported suicidal thoughts. Millennials (age 27-41) and Gen-X physicians (age 42-56) were more likely to report these thoughts than were Baby Boomers (age 57-75) and the Silent Generation (age 76-95).
“Younger physicians are more burned out – they may have less control over their lives and less meaning than some older doctors who can do what they want,” said Dr. Yellowlees.
One millennial respondent commented that being on call and being required to chart detailed notes in the EHR has contributed to her burnout. “I’m more impatient and make less time and effort to see my friends and family.”
One Silent Generation respondent commented, “I am semi-retired, I take no call, I work no weekends, I provide anesthesia care in my area of special expertise, I work clinically about 46 days a year. Life is good, particularly compared to my younger colleagues who are working 60-plus hours a week with evening work, weekend work, and call. I feel really sorry for them.”
When young people enter medical school, they’re quite healthy, with low rates of depression and burnout, said Dr. Yellowlees. Yet, studies have shown that rates of burnout and suicidal thoughts increased within 2 years. “That reflects what happens when a group of idealistic young people hit a horrible system,” he said.
Who’s responsible?
Millennials were three times as likely as baby boomers to say that a medical school or health care organization should be responsible when a student or physician commits suicide.
“Young physicians may expect more of their employers than my generation did, which we see in residency programs that have unionized,” said Dr. Yellowlees, a Baby Boomer.
“As more young doctors are employed by health care organizations, they also may expect more resources to be available to them, such as wellness programs,” he added.
Younger doctors also focus more on work-life balance than older doctors, including time off and having hobbies, he said. “They are much more rational in terms of their overall beliefs and expectations than the older generation.”
Whom doctors confide in
Nearly 60% of physician-respondents with suicidal thoughts said they confided in a professional or someone they knew. Men were just as likely as women to reach out to a therapist (38%), whereas men were slightly more likely to confide in a family member and women were slightly more likely to confide in a colleague.
“It’s interesting that women are more active in seeking support at work – they often have developed a network of colleagues to support each other’s careers and whom they can confide in,” said Dr. Yellowlees.
He emphasized that 40% of physicians said they didn’t confide in anyone when they had suicidal thoughts. Of those, just over half said they could cope without professional help.
One respondent commented, “It’s just a thought; nothing I would actually do.” Another commented, “Mental health professionals can’t fix the underlying reason for the problem.”
Many doctors were concerned about risking disclosure to their medical boards (42%); that it would show up on their insurance records (33%); and that their colleagues would find out (25%), according to the report.
One respondent commented, “I don’t trust doctors to keep it to themselves.”
Another barrier doctors mentioned was a lack of time to seek help. One commented, “Time. I have none, when am I supposed to find an hour for counseling?”
A version of this article originally appeared on Medscape.com.
A physician’s specialty can make a difference when it comes to having suicidal thoughts. Doctors who specialize in family medicine, obstetrics-gynecology, and psychiatry reported double the rates of suicidal thoughts than doctors in oncology, rheumatology, and pulmonary medicine, according to Doctors’ Burden: Medscape Physician Suicide Report 2023.
“The specialties with the highest reporting of physician suicidal thoughts are also those with the greatest physician shortages, based on the number of job openings posted by recruiting sites,” said Peter Yellowlees, MD, professor of psychiatry and chief wellness officer at UC Davis Health.
Doctors in those specialties are overworked, which can lead to burnout, he said.
There’s also a generational divide among physicians who reported suicidal thoughts. Millennials (age 27-41) and Gen-X physicians (age 42-56) were more likely to report these thoughts than were Baby Boomers (age 57-75) and the Silent Generation (age 76-95).
“Younger physicians are more burned out – they may have less control over their lives and less meaning than some older doctors who can do what they want,” said Dr. Yellowlees.
One millennial respondent commented that being on call and being required to chart detailed notes in the EHR has contributed to her burnout. “I’m more impatient and make less time and effort to see my friends and family.”
One Silent Generation respondent commented, “I am semi-retired, I take no call, I work no weekends, I provide anesthesia care in my area of special expertise, I work clinically about 46 days a year. Life is good, particularly compared to my younger colleagues who are working 60-plus hours a week with evening work, weekend work, and call. I feel really sorry for them.”
When young people enter medical school, they’re quite healthy, with low rates of depression and burnout, said Dr. Yellowlees. Yet, studies have shown that rates of burnout and suicidal thoughts increased within 2 years. “That reflects what happens when a group of idealistic young people hit a horrible system,” he said.
Who’s responsible?
Millennials were three times as likely as baby boomers to say that a medical school or health care organization should be responsible when a student or physician commits suicide.
“Young physicians may expect more of their employers than my generation did, which we see in residency programs that have unionized,” said Dr. Yellowlees, a Baby Boomer.
“As more young doctors are employed by health care organizations, they also may expect more resources to be available to them, such as wellness programs,” he added.
Younger doctors also focus more on work-life balance than older doctors, including time off and having hobbies, he said. “They are much more rational in terms of their overall beliefs and expectations than the older generation.”
Whom doctors confide in
Nearly 60% of physician-respondents with suicidal thoughts said they confided in a professional or someone they knew. Men were just as likely as women to reach out to a therapist (38%), whereas men were slightly more likely to confide in a family member and women were slightly more likely to confide in a colleague.
“It’s interesting that women are more active in seeking support at work – they often have developed a network of colleagues to support each other’s careers and whom they can confide in,” said Dr. Yellowlees.
He emphasized that 40% of physicians said they didn’t confide in anyone when they had suicidal thoughts. Of those, just over half said they could cope without professional help.
One respondent commented, “It’s just a thought; nothing I would actually do.” Another commented, “Mental health professionals can’t fix the underlying reason for the problem.”
Many doctors were concerned about risking disclosure to their medical boards (42%); that it would show up on their insurance records (33%); and that their colleagues would find out (25%), according to the report.
One respondent commented, “I don’t trust doctors to keep it to themselves.”
Another barrier doctors mentioned was a lack of time to seek help. One commented, “Time. I have none, when am I supposed to find an hour for counseling?”
A version of this article originally appeared on Medscape.com.
A physician’s specialty can make a difference when it comes to having suicidal thoughts. Doctors who specialize in family medicine, obstetrics-gynecology, and psychiatry reported double the rates of suicidal thoughts than doctors in oncology, rheumatology, and pulmonary medicine, according to Doctors’ Burden: Medscape Physician Suicide Report 2023.
“The specialties with the highest reporting of physician suicidal thoughts are also those with the greatest physician shortages, based on the number of job openings posted by recruiting sites,” said Peter Yellowlees, MD, professor of psychiatry and chief wellness officer at UC Davis Health.
Doctors in those specialties are overworked, which can lead to burnout, he said.
There’s also a generational divide among physicians who reported suicidal thoughts. Millennials (age 27-41) and Gen-X physicians (age 42-56) were more likely to report these thoughts than were Baby Boomers (age 57-75) and the Silent Generation (age 76-95).
“Younger physicians are more burned out – they may have less control over their lives and less meaning than some older doctors who can do what they want,” said Dr. Yellowlees.
One millennial respondent commented that being on call and being required to chart detailed notes in the EHR has contributed to her burnout. “I’m more impatient and make less time and effort to see my friends and family.”
One Silent Generation respondent commented, “I am semi-retired, I take no call, I work no weekends, I provide anesthesia care in my area of special expertise, I work clinically about 46 days a year. Life is good, particularly compared to my younger colleagues who are working 60-plus hours a week with evening work, weekend work, and call. I feel really sorry for them.”
When young people enter medical school, they’re quite healthy, with low rates of depression and burnout, said Dr. Yellowlees. Yet, studies have shown that rates of burnout and suicidal thoughts increased within 2 years. “That reflects what happens when a group of idealistic young people hit a horrible system,” he said.
Who’s responsible?
Millennials were three times as likely as baby boomers to say that a medical school or health care organization should be responsible when a student or physician commits suicide.
“Young physicians may expect more of their employers than my generation did, which we see in residency programs that have unionized,” said Dr. Yellowlees, a Baby Boomer.
“As more young doctors are employed by health care organizations, they also may expect more resources to be available to them, such as wellness programs,” he added.
Younger doctors also focus more on work-life balance than older doctors, including time off and having hobbies, he said. “They are much more rational in terms of their overall beliefs and expectations than the older generation.”
Whom doctors confide in
Nearly 60% of physician-respondents with suicidal thoughts said they confided in a professional or someone they knew. Men were just as likely as women to reach out to a therapist (38%), whereas men were slightly more likely to confide in a family member and women were slightly more likely to confide in a colleague.
“It’s interesting that women are more active in seeking support at work – they often have developed a network of colleagues to support each other’s careers and whom they can confide in,” said Dr. Yellowlees.
He emphasized that 40% of physicians said they didn’t confide in anyone when they had suicidal thoughts. Of those, just over half said they could cope without professional help.
One respondent commented, “It’s just a thought; nothing I would actually do.” Another commented, “Mental health professionals can’t fix the underlying reason for the problem.”
Many doctors were concerned about risking disclosure to their medical boards (42%); that it would show up on their insurance records (33%); and that their colleagues would find out (25%), according to the report.
One respondent commented, “I don’t trust doctors to keep it to themselves.”
Another barrier doctors mentioned was a lack of time to seek help. One commented, “Time. I have none, when am I supposed to find an hour for counseling?”
A version of this article originally appeared on Medscape.com.
CLL treatment: More infections among real-world patients
For example, “the rate of severe infection for ibrutinib in clinical trials ranged from 12.8% to 45% with median follow-up ranging from 27 to 65 months. In our study, the rate of severe infection was 45.3% within a shorter median follow-up period of 23.3 months,” said study lead author Amanda Tey, MPharm, a hematology pharmacist with Monash Health in Clayton, Australia, in an interview.
The results suggest that “real-world severe infection risk is higher than previously appreciated,” said Ms. Tey, whose findings were published in the European Journal of Hematology. “Poor performance status and a high comorbidity burden further increase this risk.”
According to the study, there are limited data about real-world infection rates for patients with CLL or B-cell lymphoma who take the three drugs.
Both the underlying blood cancer and the drugs themselves may disrupt the immune system in these patients, Ms. Tey noted. “Ibrutinib inhibits interleukin-2-inducible T-cell kinase, which has a role in T-cell maturation. Idelalisib reduces regulatory T-cell activity and natural killer cell and neutrophil inflammatory responses. Venetoclax is associated with a high rate of neutropenia.”
For the new retrospective, single-center study, researchers tracked adult patients who’d received the drugs from 2014 to 2021 in a hospital network serving 1.5 million people in the Australian state of Victoria. The primary outcome was severe infection of grade 3 or higher. Patients were excluded for such factors as having been primarily treated at other facilities, receiving less than 30 days of treatment, or having been treated for other indications such as primary central nervous system lymphoma.
Of the 67 patients in the study, the numbers taking the drugs were 53 (ibrutinib), 8 (idelalisib), and 6 (venetoclax). Eleven patients took more than one drug. Median age was 73 years, and 73% of patients were male.
Patients spent a median 23.3, 4.8, and 3.5 months taking ibrutinib, idelalisib, and venetoclax, respectively, before treatment stopped or data were collected. Patients were commonly prescribed antimicrobials to prevent pneumocystis jirovecii pneumonia and herpes simplex virus (HSV)/varicella zoster virus (VZV) infection.
Researchers found that 48% of the patients had at least one serious infection: 45% of those on ibrutinib, 63% of those on idelalisib, and 50% of those on venetoclax. Seven patients died of infections.
In comparison, the researchers reported, a systematic review of idelalisib in blood cancer clinical trials reported an overall infection rate of 28%, while clinical trials reported an infection rate of 17.5%-22% in patients taking venetoclax for CLL.
Poor performance status and higher levels of comorbidity were linked to higher risk of infection, and infections occurred at a median of 5.4 months.
Lead author Ms. Tey highlighted the fact that most of the patients in the new study had relapsed/refractory disease. The infection risk in the real-world first-line setting is unknown, she said. “Furthermore, due to the size of our study and high uptake of antimicrobial prophylaxis, the optimal prophylaxis strategy for these patients remains unclear.”
In an interview, infectious disease physician Gemma Reynolds, MChD, MPH, of Austin Health and Peter MacCallum Cancer Center in Melbourne, said the study findings reflect “a lot of what we know from other observational studies and clinical practice. There is a risk of infection, and serious infection, associated with these agents. Sometimes the pathogen is classically opportunistic, but often it is bacterial, and respiratory sites are common. Infections often occur early into a course of therapy.”
Dr. Reynolds, who didn’t take part in the study, urged colleagues to cast a wide net if a patient appears to have an infection but doesn’t respond to conventional therapies such as antibiotics. “Unusual infections are possible,” she said, and aggressive early workups may be advisable via blood cultures, viral swabs, sputum culture, early imaging, bronchoscopy, and preemptive monitoring in patients with a prior infection history with a disease such as CMV.
Alessandra Ferrajoli, MD, a hematologist/oncologist at MD Anderson Cancer Center who also didn’t take part in the study, agreed in an interview that the findings reflect those found in other reports. “It should be highlighted that the population studied is at particular high risk for infections given the high proportion of patients with recurrent disease (85%), many patients with concurrent hypogammaglobulinemia (64%), and the patient median age of 73 years and a high comorbidities burden,” she said. “In my view, this explains the higher rate of infections reported in this study, when compared to other case series.”
Dr. Ferrajoli added that there’s no standard antimicrobial prophylaxis for patients with B-cell malignancies receiving targeted therapies. “Anti-HSV/VZV prophylaxis is commonly implemented. Additional antiviral, antimicrobial, and antifungal prophylaxis should be used based on patients’ absolute neutrophil and T-cell count and individual risk factors, including prior history of infections such as CMV, prior splenectomy, and history of invasive fungal infections.”
The study was funded by Monash Health, the National Health and Medical Research Council (Australia), and the Society of Hospital Pharmacists of Australia. Ms. Tey reported no disclosures. Some of the study authors reported multiple disclosures. Dr. Reynolds discloses a PhD scholarship from the National Health and Medical Research Council. Dr. Ferrajoli reported no disclosures.
For example, “the rate of severe infection for ibrutinib in clinical trials ranged from 12.8% to 45% with median follow-up ranging from 27 to 65 months. In our study, the rate of severe infection was 45.3% within a shorter median follow-up period of 23.3 months,” said study lead author Amanda Tey, MPharm, a hematology pharmacist with Monash Health in Clayton, Australia, in an interview.
The results suggest that “real-world severe infection risk is higher than previously appreciated,” said Ms. Tey, whose findings were published in the European Journal of Hematology. “Poor performance status and a high comorbidity burden further increase this risk.”
According to the study, there are limited data about real-world infection rates for patients with CLL or B-cell lymphoma who take the three drugs.
Both the underlying blood cancer and the drugs themselves may disrupt the immune system in these patients, Ms. Tey noted. “Ibrutinib inhibits interleukin-2-inducible T-cell kinase, which has a role in T-cell maturation. Idelalisib reduces regulatory T-cell activity and natural killer cell and neutrophil inflammatory responses. Venetoclax is associated with a high rate of neutropenia.”
For the new retrospective, single-center study, researchers tracked adult patients who’d received the drugs from 2014 to 2021 in a hospital network serving 1.5 million people in the Australian state of Victoria. The primary outcome was severe infection of grade 3 or higher. Patients were excluded for such factors as having been primarily treated at other facilities, receiving less than 30 days of treatment, or having been treated for other indications such as primary central nervous system lymphoma.
Of the 67 patients in the study, the numbers taking the drugs were 53 (ibrutinib), 8 (idelalisib), and 6 (venetoclax). Eleven patients took more than one drug. Median age was 73 years, and 73% of patients were male.
Patients spent a median 23.3, 4.8, and 3.5 months taking ibrutinib, idelalisib, and venetoclax, respectively, before treatment stopped or data were collected. Patients were commonly prescribed antimicrobials to prevent pneumocystis jirovecii pneumonia and herpes simplex virus (HSV)/varicella zoster virus (VZV) infection.
Researchers found that 48% of the patients had at least one serious infection: 45% of those on ibrutinib, 63% of those on idelalisib, and 50% of those on venetoclax. Seven patients died of infections.
In comparison, the researchers reported, a systematic review of idelalisib in blood cancer clinical trials reported an overall infection rate of 28%, while clinical trials reported an infection rate of 17.5%-22% in patients taking venetoclax for CLL.
Poor performance status and higher levels of comorbidity were linked to higher risk of infection, and infections occurred at a median of 5.4 months.
Lead author Ms. Tey highlighted the fact that most of the patients in the new study had relapsed/refractory disease. The infection risk in the real-world first-line setting is unknown, she said. “Furthermore, due to the size of our study and high uptake of antimicrobial prophylaxis, the optimal prophylaxis strategy for these patients remains unclear.”
In an interview, infectious disease physician Gemma Reynolds, MChD, MPH, of Austin Health and Peter MacCallum Cancer Center in Melbourne, said the study findings reflect “a lot of what we know from other observational studies and clinical practice. There is a risk of infection, and serious infection, associated with these agents. Sometimes the pathogen is classically opportunistic, but often it is bacterial, and respiratory sites are common. Infections often occur early into a course of therapy.”
Dr. Reynolds, who didn’t take part in the study, urged colleagues to cast a wide net if a patient appears to have an infection but doesn’t respond to conventional therapies such as antibiotics. “Unusual infections are possible,” she said, and aggressive early workups may be advisable via blood cultures, viral swabs, sputum culture, early imaging, bronchoscopy, and preemptive monitoring in patients with a prior infection history with a disease such as CMV.
Alessandra Ferrajoli, MD, a hematologist/oncologist at MD Anderson Cancer Center who also didn’t take part in the study, agreed in an interview that the findings reflect those found in other reports. “It should be highlighted that the population studied is at particular high risk for infections given the high proportion of patients with recurrent disease (85%), many patients with concurrent hypogammaglobulinemia (64%), and the patient median age of 73 years and a high comorbidities burden,” she said. “In my view, this explains the higher rate of infections reported in this study, when compared to other case series.”
Dr. Ferrajoli added that there’s no standard antimicrobial prophylaxis for patients with B-cell malignancies receiving targeted therapies. “Anti-HSV/VZV prophylaxis is commonly implemented. Additional antiviral, antimicrobial, and antifungal prophylaxis should be used based on patients’ absolute neutrophil and T-cell count and individual risk factors, including prior history of infections such as CMV, prior splenectomy, and history of invasive fungal infections.”
The study was funded by Monash Health, the National Health and Medical Research Council (Australia), and the Society of Hospital Pharmacists of Australia. Ms. Tey reported no disclosures. Some of the study authors reported multiple disclosures. Dr. Reynolds discloses a PhD scholarship from the National Health and Medical Research Council. Dr. Ferrajoli reported no disclosures.
For example, “the rate of severe infection for ibrutinib in clinical trials ranged from 12.8% to 45% with median follow-up ranging from 27 to 65 months. In our study, the rate of severe infection was 45.3% within a shorter median follow-up period of 23.3 months,” said study lead author Amanda Tey, MPharm, a hematology pharmacist with Monash Health in Clayton, Australia, in an interview.
The results suggest that “real-world severe infection risk is higher than previously appreciated,” said Ms. Tey, whose findings were published in the European Journal of Hematology. “Poor performance status and a high comorbidity burden further increase this risk.”
According to the study, there are limited data about real-world infection rates for patients with CLL or B-cell lymphoma who take the three drugs.
Both the underlying blood cancer and the drugs themselves may disrupt the immune system in these patients, Ms. Tey noted. “Ibrutinib inhibits interleukin-2-inducible T-cell kinase, which has a role in T-cell maturation. Idelalisib reduces regulatory T-cell activity and natural killer cell and neutrophil inflammatory responses. Venetoclax is associated with a high rate of neutropenia.”
For the new retrospective, single-center study, researchers tracked adult patients who’d received the drugs from 2014 to 2021 in a hospital network serving 1.5 million people in the Australian state of Victoria. The primary outcome was severe infection of grade 3 or higher. Patients were excluded for such factors as having been primarily treated at other facilities, receiving less than 30 days of treatment, or having been treated for other indications such as primary central nervous system lymphoma.
Of the 67 patients in the study, the numbers taking the drugs were 53 (ibrutinib), 8 (idelalisib), and 6 (venetoclax). Eleven patients took more than one drug. Median age was 73 years, and 73% of patients were male.
Patients spent a median 23.3, 4.8, and 3.5 months taking ibrutinib, idelalisib, and venetoclax, respectively, before treatment stopped or data were collected. Patients were commonly prescribed antimicrobials to prevent pneumocystis jirovecii pneumonia and herpes simplex virus (HSV)/varicella zoster virus (VZV) infection.
Researchers found that 48% of the patients had at least one serious infection: 45% of those on ibrutinib, 63% of those on idelalisib, and 50% of those on venetoclax. Seven patients died of infections.
In comparison, the researchers reported, a systematic review of idelalisib in blood cancer clinical trials reported an overall infection rate of 28%, while clinical trials reported an infection rate of 17.5%-22% in patients taking venetoclax for CLL.
Poor performance status and higher levels of comorbidity were linked to higher risk of infection, and infections occurred at a median of 5.4 months.
Lead author Ms. Tey highlighted the fact that most of the patients in the new study had relapsed/refractory disease. The infection risk in the real-world first-line setting is unknown, she said. “Furthermore, due to the size of our study and high uptake of antimicrobial prophylaxis, the optimal prophylaxis strategy for these patients remains unclear.”
In an interview, infectious disease physician Gemma Reynolds, MChD, MPH, of Austin Health and Peter MacCallum Cancer Center in Melbourne, said the study findings reflect “a lot of what we know from other observational studies and clinical practice. There is a risk of infection, and serious infection, associated with these agents. Sometimes the pathogen is classically opportunistic, but often it is bacterial, and respiratory sites are common. Infections often occur early into a course of therapy.”
Dr. Reynolds, who didn’t take part in the study, urged colleagues to cast a wide net if a patient appears to have an infection but doesn’t respond to conventional therapies such as antibiotics. “Unusual infections are possible,” she said, and aggressive early workups may be advisable via blood cultures, viral swabs, sputum culture, early imaging, bronchoscopy, and preemptive monitoring in patients with a prior infection history with a disease such as CMV.
Alessandra Ferrajoli, MD, a hematologist/oncologist at MD Anderson Cancer Center who also didn’t take part in the study, agreed in an interview that the findings reflect those found in other reports. “It should be highlighted that the population studied is at particular high risk for infections given the high proportion of patients with recurrent disease (85%), many patients with concurrent hypogammaglobulinemia (64%), and the patient median age of 73 years and a high comorbidities burden,” she said. “In my view, this explains the higher rate of infections reported in this study, when compared to other case series.”
Dr. Ferrajoli added that there’s no standard antimicrobial prophylaxis for patients with B-cell malignancies receiving targeted therapies. “Anti-HSV/VZV prophylaxis is commonly implemented. Additional antiviral, antimicrobial, and antifungal prophylaxis should be used based on patients’ absolute neutrophil and T-cell count and individual risk factors, including prior history of infections such as CMV, prior splenectomy, and history of invasive fungal infections.”
The study was funded by Monash Health, the National Health and Medical Research Council (Australia), and the Society of Hospital Pharmacists of Australia. Ms. Tey reported no disclosures. Some of the study authors reported multiple disclosures. Dr. Reynolds discloses a PhD scholarship from the National Health and Medical Research Council. Dr. Ferrajoli reported no disclosures.
FROM THE EUROPEAN JOURNAL OF HEMATOLOGY
Atorvastatin cut anthracycline cardiac dysfunction in lymphoma
NEW ORLEANS – Atorvastatin treatment of patients with lymphoma undergoing treatment with an anthracycline significantly cut the incidence of incident cardiac dysfunction by about two-thirds during 12 months of treatment, in a multicenter, randomized trial with 300 enrolled patients.
“These data support the use of atorvastatin among patients with lymphoma being treated with anthracyclines where prevention of cardiac systolic dysfunction is important,” concluded Tomas G. Neilan, MD, at the joint scientific sessions of the American College of Cardiology and the World Heart Federation.
He highlighted that an important difference between the new study, STOP-CA, and a major prior study with a neutral effect published in 2022, was that STOP-CA “was powered for a major change” in cardiac function as the study’s primary outcome, a decline from baseline in left ventricular ejection fraction (LVEF) of at least 10% that also reduced ejection fraction to less than 55%.
“We can consider these medications [atorvastatin] for patients at higher risk for cardiac toxicity from anthracyclines, such as patients who receive a higher dose of an anthracycline, older patients, people with obesity, and women, commented Anita Deswal, MD, professor and chair of the department of cardiology at the University of Texas MD Anderson Cancer Center, Houston, who was not involved with the study.
A basis for an ‘important discussion’ with patients
“For patients receiving higher doses of anthracyclines, the STOP-CA trial says that whether to start a statin for cardiac protection is now an important discussion” for these patients to have with their treating clinicians. ”That was not the case before today,” commented Ronald M. Witteles, MD, a cardiologist and professor who specializes in cardio-oncology at Stanford (Calif.) University.
“For a patient being treated for lymphoma or for another cancer and treated with equal or higher anthracycline doses, such as patients with a sarcoma, this trial’s results at the very least warrant a discussion between physicians and patients to make the decision,” Dr. Witteles, who was not involved in the study, said in an interview. But he also cautioned that “whether an individual patient should take a statin in this scenario is still not a no-brainer. While the trial was positive, it was for an imaging rather than for a clinical endpoint.”
Experts noted that a similar study with the clinical endpoint of heart failure would require both many more randomized patients as well as much longer follow-up. STOP-CA was not powered for this endpoint. During its 12-month duration, a total of 11 patients developed heart failure, with no between group difference.
STOP-CA enrolled adults with lymphoma (Hodgkin or non-Hodgkin) and scheduled to undergo anthracycline treatment at eight U.S. centers and one in Canada, and excluded patients already on statin treatment or those for whom a statin was already indicated. Of the 300 enrolled patients, 286 had 12-month follow-up. Randomization assigned patients to receive either 40 mg daily of atorvastatin or placebo.
Their cumulative, median anthracycline dose was 300 mg/m2, which is typical for treating lymphoma, but higher than the typical dose use for patients with breast cancer. At baseline, average LVEF was 63%, and after 12 months this had declined to 59%. Forty-six of the 286 patients assessed after 12 months fulfilled the primary outcome of at least a 10–percentage point reduction from baseline in their LVEF and a decline in LVEF to less than 55%. Researchers used cardiac MR to assess LVEF at baseline, and in most patients at follow-up, but a minority of patients had their follow-up assessments by echocardiography because of logistical issues. Greater than 90% of patients were adherent to their assigned regimen.
Tripled incidence of cardiac dysfunction in placebo patients
The incidence of this outcome was 9% among the patients who received atorvastatin, and 22% among those on placebo, a significant difference. The calculated odds of the primary outcome was 2.9-fold more likely among the patients treated with placebo, compared with those who received atorvastatin, also a significant difference.
The study’s secondary outcome was patients who had at least a 5% drop from baseline in their LVEF and with a LVEF of less than 55% after 12 months. This outcome occurred in 13% of patients treated with atorvastatin and in 29% of those who received placebo, a significant difference.
The atorvastatin and placebo arms showed no significant differences in adverse events during the study, with roughly similar incidence rates for muscle pain, elevated liver enzymes, and renal failure. None of the enrolled patients developed myositis.
Atorvastatin treatment also produced an expected average 37% decline from baseline in levels of LDL cholesterol.
“This was a well-designed and important trial,” said Dr. Witteles. “Anthracyclines remain a mainstay of cancer therapies for a number of malignancies, such as lymphoma and sarcoma, and the cardiac side effects of development of cardiac dysfunction are unequivocally real.”
The importance of a clinically meaningful effect
The results especially contrast with the findings from the PREVENT study, published in 2022, which compared a daily, 40-mg atorvastatin treatment with placebo in 279 randomized patients with breast cancer and treated for 24 months. However, patients in PREVENT had a cumulative, median anthracycline dose of 240 mg/m2, and the study’s primary outcome was the average change from baseline in LVEF after 24 months of treatment, which was a reduction of 0.08 percentage points in the placebo arm, a nonsignificant difference.
In STOP-CA, the average change in LVEF from baseline was a 1–percentage point reduction in the placebo arm, compared with the atorvastatin-treated patients, a difference that was statistically significant, but “not clinically significant,” said Dr. Neilan, director of the cardio-oncology program at Massachusetts General Hospital, Boston. He cited the good fortune of the STOP-CA investigators when they received a recommendation from reviewers early on to design their study to track a clinically meaningful change in LVEF rather than just looking at the average overall change.
Dr. Deswal also noted that it is unlikely that future studies will examine the efficacy of a statin for preventing LVEF in patients across the range of cancers that are eligible for anthracycline treatment. As a result, she predicted that “we may have to extrapolate” the results from STOP-CA to patients with other cancer types.
STOP-CA received no commercial funding. Dr. Neilan has been a consultant for and received fees from Abbvie, Amgen, Bristol-Myers Squibb, CRC Oncology, Genentech, Roche, and Sanofi, and has received grant funding from AstraZeneca and Bristol Myers Squib. Dr. Deswal and Dr. Witteles had no relevant disclosures.
NEW ORLEANS – Atorvastatin treatment of patients with lymphoma undergoing treatment with an anthracycline significantly cut the incidence of incident cardiac dysfunction by about two-thirds during 12 months of treatment, in a multicenter, randomized trial with 300 enrolled patients.
“These data support the use of atorvastatin among patients with lymphoma being treated with anthracyclines where prevention of cardiac systolic dysfunction is important,” concluded Tomas G. Neilan, MD, at the joint scientific sessions of the American College of Cardiology and the World Heart Federation.
He highlighted that an important difference between the new study, STOP-CA, and a major prior study with a neutral effect published in 2022, was that STOP-CA “was powered for a major change” in cardiac function as the study’s primary outcome, a decline from baseline in left ventricular ejection fraction (LVEF) of at least 10% that also reduced ejection fraction to less than 55%.
“We can consider these medications [atorvastatin] for patients at higher risk for cardiac toxicity from anthracyclines, such as patients who receive a higher dose of an anthracycline, older patients, people with obesity, and women, commented Anita Deswal, MD, professor and chair of the department of cardiology at the University of Texas MD Anderson Cancer Center, Houston, who was not involved with the study.
A basis for an ‘important discussion’ with patients
“For patients receiving higher doses of anthracyclines, the STOP-CA trial says that whether to start a statin for cardiac protection is now an important discussion” for these patients to have with their treating clinicians. ”That was not the case before today,” commented Ronald M. Witteles, MD, a cardiologist and professor who specializes in cardio-oncology at Stanford (Calif.) University.
“For a patient being treated for lymphoma or for another cancer and treated with equal or higher anthracycline doses, such as patients with a sarcoma, this trial’s results at the very least warrant a discussion between physicians and patients to make the decision,” Dr. Witteles, who was not involved in the study, said in an interview. But he also cautioned that “whether an individual patient should take a statin in this scenario is still not a no-brainer. While the trial was positive, it was for an imaging rather than for a clinical endpoint.”
Experts noted that a similar study with the clinical endpoint of heart failure would require both many more randomized patients as well as much longer follow-up. STOP-CA was not powered for this endpoint. During its 12-month duration, a total of 11 patients developed heart failure, with no between group difference.
STOP-CA enrolled adults with lymphoma (Hodgkin or non-Hodgkin) and scheduled to undergo anthracycline treatment at eight U.S. centers and one in Canada, and excluded patients already on statin treatment or those for whom a statin was already indicated. Of the 300 enrolled patients, 286 had 12-month follow-up. Randomization assigned patients to receive either 40 mg daily of atorvastatin or placebo.
Their cumulative, median anthracycline dose was 300 mg/m2, which is typical for treating lymphoma, but higher than the typical dose use for patients with breast cancer. At baseline, average LVEF was 63%, and after 12 months this had declined to 59%. Forty-six of the 286 patients assessed after 12 months fulfilled the primary outcome of at least a 10–percentage point reduction from baseline in their LVEF and a decline in LVEF to less than 55%. Researchers used cardiac MR to assess LVEF at baseline, and in most patients at follow-up, but a minority of patients had their follow-up assessments by echocardiography because of logistical issues. Greater than 90% of patients were adherent to their assigned regimen.
Tripled incidence of cardiac dysfunction in placebo patients
The incidence of this outcome was 9% among the patients who received atorvastatin, and 22% among those on placebo, a significant difference. The calculated odds of the primary outcome was 2.9-fold more likely among the patients treated with placebo, compared with those who received atorvastatin, also a significant difference.
The study’s secondary outcome was patients who had at least a 5% drop from baseline in their LVEF and with a LVEF of less than 55% after 12 months. This outcome occurred in 13% of patients treated with atorvastatin and in 29% of those who received placebo, a significant difference.
The atorvastatin and placebo arms showed no significant differences in adverse events during the study, with roughly similar incidence rates for muscle pain, elevated liver enzymes, and renal failure. None of the enrolled patients developed myositis.
Atorvastatin treatment also produced an expected average 37% decline from baseline in levels of LDL cholesterol.
“This was a well-designed and important trial,” said Dr. Witteles. “Anthracyclines remain a mainstay of cancer therapies for a number of malignancies, such as lymphoma and sarcoma, and the cardiac side effects of development of cardiac dysfunction are unequivocally real.”
The importance of a clinically meaningful effect
The results especially contrast with the findings from the PREVENT study, published in 2022, which compared a daily, 40-mg atorvastatin treatment with placebo in 279 randomized patients with breast cancer and treated for 24 months. However, patients in PREVENT had a cumulative, median anthracycline dose of 240 mg/m2, and the study’s primary outcome was the average change from baseline in LVEF after 24 months of treatment, which was a reduction of 0.08 percentage points in the placebo arm, a nonsignificant difference.
In STOP-CA, the average change in LVEF from baseline was a 1–percentage point reduction in the placebo arm, compared with the atorvastatin-treated patients, a difference that was statistically significant, but “not clinically significant,” said Dr. Neilan, director of the cardio-oncology program at Massachusetts General Hospital, Boston. He cited the good fortune of the STOP-CA investigators when they received a recommendation from reviewers early on to design their study to track a clinically meaningful change in LVEF rather than just looking at the average overall change.
Dr. Deswal also noted that it is unlikely that future studies will examine the efficacy of a statin for preventing LVEF in patients across the range of cancers that are eligible for anthracycline treatment. As a result, she predicted that “we may have to extrapolate” the results from STOP-CA to patients with other cancer types.
STOP-CA received no commercial funding. Dr. Neilan has been a consultant for and received fees from Abbvie, Amgen, Bristol-Myers Squibb, CRC Oncology, Genentech, Roche, and Sanofi, and has received grant funding from AstraZeneca and Bristol Myers Squib. Dr. Deswal and Dr. Witteles had no relevant disclosures.
NEW ORLEANS – Atorvastatin treatment of patients with lymphoma undergoing treatment with an anthracycline significantly cut the incidence of incident cardiac dysfunction by about two-thirds during 12 months of treatment, in a multicenter, randomized trial with 300 enrolled patients.
“These data support the use of atorvastatin among patients with lymphoma being treated with anthracyclines where prevention of cardiac systolic dysfunction is important,” concluded Tomas G. Neilan, MD, at the joint scientific sessions of the American College of Cardiology and the World Heart Federation.
He highlighted that an important difference between the new study, STOP-CA, and a major prior study with a neutral effect published in 2022, was that STOP-CA “was powered for a major change” in cardiac function as the study’s primary outcome, a decline from baseline in left ventricular ejection fraction (LVEF) of at least 10% that also reduced ejection fraction to less than 55%.
“We can consider these medications [atorvastatin] for patients at higher risk for cardiac toxicity from anthracyclines, such as patients who receive a higher dose of an anthracycline, older patients, people with obesity, and women, commented Anita Deswal, MD, professor and chair of the department of cardiology at the University of Texas MD Anderson Cancer Center, Houston, who was not involved with the study.
A basis for an ‘important discussion’ with patients
“For patients receiving higher doses of anthracyclines, the STOP-CA trial says that whether to start a statin for cardiac protection is now an important discussion” for these patients to have with their treating clinicians. ”That was not the case before today,” commented Ronald M. Witteles, MD, a cardiologist and professor who specializes in cardio-oncology at Stanford (Calif.) University.
“For a patient being treated for lymphoma or for another cancer and treated with equal or higher anthracycline doses, such as patients with a sarcoma, this trial’s results at the very least warrant a discussion between physicians and patients to make the decision,” Dr. Witteles, who was not involved in the study, said in an interview. But he also cautioned that “whether an individual patient should take a statin in this scenario is still not a no-brainer. While the trial was positive, it was for an imaging rather than for a clinical endpoint.”
Experts noted that a similar study with the clinical endpoint of heart failure would require both many more randomized patients as well as much longer follow-up. STOP-CA was not powered for this endpoint. During its 12-month duration, a total of 11 patients developed heart failure, with no between group difference.
STOP-CA enrolled adults with lymphoma (Hodgkin or non-Hodgkin) and scheduled to undergo anthracycline treatment at eight U.S. centers and one in Canada, and excluded patients already on statin treatment or those for whom a statin was already indicated. Of the 300 enrolled patients, 286 had 12-month follow-up. Randomization assigned patients to receive either 40 mg daily of atorvastatin or placebo.
Their cumulative, median anthracycline dose was 300 mg/m2, which is typical for treating lymphoma, but higher than the typical dose use for patients with breast cancer. At baseline, average LVEF was 63%, and after 12 months this had declined to 59%. Forty-six of the 286 patients assessed after 12 months fulfilled the primary outcome of at least a 10–percentage point reduction from baseline in their LVEF and a decline in LVEF to less than 55%. Researchers used cardiac MR to assess LVEF at baseline, and in most patients at follow-up, but a minority of patients had their follow-up assessments by echocardiography because of logistical issues. Greater than 90% of patients were adherent to their assigned regimen.
Tripled incidence of cardiac dysfunction in placebo patients
The incidence of this outcome was 9% among the patients who received atorvastatin, and 22% among those on placebo, a significant difference. The calculated odds of the primary outcome was 2.9-fold more likely among the patients treated with placebo, compared with those who received atorvastatin, also a significant difference.
The study’s secondary outcome was patients who had at least a 5% drop from baseline in their LVEF and with a LVEF of less than 55% after 12 months. This outcome occurred in 13% of patients treated with atorvastatin and in 29% of those who received placebo, a significant difference.
The atorvastatin and placebo arms showed no significant differences in adverse events during the study, with roughly similar incidence rates for muscle pain, elevated liver enzymes, and renal failure. None of the enrolled patients developed myositis.
Atorvastatin treatment also produced an expected average 37% decline from baseline in levels of LDL cholesterol.
“This was a well-designed and important trial,” said Dr. Witteles. “Anthracyclines remain a mainstay of cancer therapies for a number of malignancies, such as lymphoma and sarcoma, and the cardiac side effects of development of cardiac dysfunction are unequivocally real.”
The importance of a clinically meaningful effect
The results especially contrast with the findings from the PREVENT study, published in 2022, which compared a daily, 40-mg atorvastatin treatment with placebo in 279 randomized patients with breast cancer and treated for 24 months. However, patients in PREVENT had a cumulative, median anthracycline dose of 240 mg/m2, and the study’s primary outcome was the average change from baseline in LVEF after 24 months of treatment, which was a reduction of 0.08 percentage points in the placebo arm, a nonsignificant difference.
In STOP-CA, the average change in LVEF from baseline was a 1–percentage point reduction in the placebo arm, compared with the atorvastatin-treated patients, a difference that was statistically significant, but “not clinically significant,” said Dr. Neilan, director of the cardio-oncology program at Massachusetts General Hospital, Boston. He cited the good fortune of the STOP-CA investigators when they received a recommendation from reviewers early on to design their study to track a clinically meaningful change in LVEF rather than just looking at the average overall change.
Dr. Deswal also noted that it is unlikely that future studies will examine the efficacy of a statin for preventing LVEF in patients across the range of cancers that are eligible for anthracycline treatment. As a result, she predicted that “we may have to extrapolate” the results from STOP-CA to patients with other cancer types.
STOP-CA received no commercial funding. Dr. Neilan has been a consultant for and received fees from Abbvie, Amgen, Bristol-Myers Squibb, CRC Oncology, Genentech, Roche, and Sanofi, and has received grant funding from AstraZeneca and Bristol Myers Squib. Dr. Deswal and Dr. Witteles had no relevant disclosures.
AT ACC 2023
NP-PA turf fights: Where the relationship can improve
– The U.S. Bureau of Labor Statistics forecasts a 40% increase in the NP workforce by 2031, coupled with a 28% rise in PAs.
In recent reports on the quality of the relationships involving these health care professions, survey respondents mostly gave positive accounts of collaboration, using words such as like “comradery,” “teamwork,” “congenial,” and “cohesion.” But all was not perfect. Where and how could these important health care provider relationships improve?
PAs: “Competition and collaboration’ with RNs
In a Medscape survey of more than 770 PAs about their working relationships with other health care professionals; 83% of them supported the idea of PAs and NPs practicing more independently from physicians, but sometimes it’s not easy to stay in their individual lanes.
One PA respondent complained that NPs get “more opportunities and preference,” another pointed to PA-NP “turf issues,” and a third griped about NPs’ “strong unions,” which have stoked more fighting about practice abilities and available settings.
Robert Blumm, MA, PA-C, a retired surgical and emergency medicine PA who regards himself as an advocate for both PAs and NPs, describes their interaction as a “mixture of competition and collaboration.”
On one hand, the two groups typically “cooperate and do an excellent job, incurring patient errors similar to or less than physician colleagues or senior residents.” On the other hand, Mr. Blumm conceded, there is some jealousy among PAs over NPs’ advantage in staffing and hiring decisions, “since they don’t need [direct physician] supervision ... and there are limits on how many PAs can be supervised by one physician.”
Most PA-NP interactions are collaborative, although many people emphasize the relatively few conflicts, said Jennifer Orozco, DMSc, PA-C, president and chair of the American Academy of PAs.
“We see that a lot in this country,” she said. “People try to drive a wedge, but it’s often a misnomer that there’s a lot of arguing and infighting.”
NPs: Different backgrounds, same goal
The Medscape survey also included information from 750 NPs on working relationships; 93% of them favored nurses and PAs working more independently from doctors.
April Kapu, DNP, ARPN, has worked closely with PAs for more than 20 years. “In my experience ... they complement one another as health team members, although the education and training are somewhat different,” said Ms. Kapu, , president of the American Association of Nurse Practitioners.
Some respondents noted the different educational trajectories for NPs and PAs. “Doctors and PAs are taught using the same model, but NPs are taught under the nursing model,” wrote a family medicine PA.
In emergency departments where Mr. Blumm has worked, ICU NPs have an edge over PAs in terms of preparation, organization, and the tabulation of formulas. On the other hand, some of Mr. Blumm’s fellow PAs were also emergency medicine technicians or respiratory therapists, who had “2 years of classroom training, on par with that of medical students.”
Must these differences in training and education foment conflict between NPs and PAs? “We all bring something different to the table,” said Ms. Kapu, who also is associate dean for clinical and community partnerships at Vanderbilt University, Nashville, Tenn. “It is important to respect each person’s entry point, education, and training.”
Differing personalities and environments
Numerous PA respondents said that individual personalities and work environments are more likely to trigger issues with NPs than are differences in training.
“It depends on the team and situation and who the people are, not the letters behind their names,” an emergency medicine PA wrote. A surgical PA noted that “group dynamics and work culture differ from place to place,” while a third PA agreed that “it’s personality dependent, not title dependent.”
No single formula will resolve areas of NP-PA conflict, Ms. Orozco said. “What works in Chicago might not work in rural Colorado or Texas or California, but we do have to come together. The overall focus should be on greater flexibility for PAs and NPs. Patients will fare better.”
Joint research, publishing could help
About a decade ago, Mr. Blumm joined with another PA and an NP to form the American College of Clinicians, the first joint PA-NP national professional organization. Although it disbanded after 6 years, owing to low membership, he hopes a similar collaboration will take off in the future.
“I also recommend that PAs and NPs publish articles together, with research as an excellent place to start,” he added. “PAs and NPs should stand together and be a source of healing for all our patients. Regardless of our titles, our responsibility is to bring healing together.”
A version of this article first appeared on Medscape.com.
– The U.S. Bureau of Labor Statistics forecasts a 40% increase in the NP workforce by 2031, coupled with a 28% rise in PAs.
In recent reports on the quality of the relationships involving these health care professions, survey respondents mostly gave positive accounts of collaboration, using words such as like “comradery,” “teamwork,” “congenial,” and “cohesion.” But all was not perfect. Where and how could these important health care provider relationships improve?
PAs: “Competition and collaboration’ with RNs
In a Medscape survey of more than 770 PAs about their working relationships with other health care professionals; 83% of them supported the idea of PAs and NPs practicing more independently from physicians, but sometimes it’s not easy to stay in their individual lanes.
One PA respondent complained that NPs get “more opportunities and preference,” another pointed to PA-NP “turf issues,” and a third griped about NPs’ “strong unions,” which have stoked more fighting about practice abilities and available settings.
Robert Blumm, MA, PA-C, a retired surgical and emergency medicine PA who regards himself as an advocate for both PAs and NPs, describes their interaction as a “mixture of competition and collaboration.”
On one hand, the two groups typically “cooperate and do an excellent job, incurring patient errors similar to or less than physician colleagues or senior residents.” On the other hand, Mr. Blumm conceded, there is some jealousy among PAs over NPs’ advantage in staffing and hiring decisions, “since they don’t need [direct physician] supervision ... and there are limits on how many PAs can be supervised by one physician.”
Most PA-NP interactions are collaborative, although many people emphasize the relatively few conflicts, said Jennifer Orozco, DMSc, PA-C, president and chair of the American Academy of PAs.
“We see that a lot in this country,” she said. “People try to drive a wedge, but it’s often a misnomer that there’s a lot of arguing and infighting.”
NPs: Different backgrounds, same goal
The Medscape survey also included information from 750 NPs on working relationships; 93% of them favored nurses and PAs working more independently from doctors.
April Kapu, DNP, ARPN, has worked closely with PAs for more than 20 years. “In my experience ... they complement one another as health team members, although the education and training are somewhat different,” said Ms. Kapu, , president of the American Association of Nurse Practitioners.
Some respondents noted the different educational trajectories for NPs and PAs. “Doctors and PAs are taught using the same model, but NPs are taught under the nursing model,” wrote a family medicine PA.
In emergency departments where Mr. Blumm has worked, ICU NPs have an edge over PAs in terms of preparation, organization, and the tabulation of formulas. On the other hand, some of Mr. Blumm’s fellow PAs were also emergency medicine technicians or respiratory therapists, who had “2 years of classroom training, on par with that of medical students.”
Must these differences in training and education foment conflict between NPs and PAs? “We all bring something different to the table,” said Ms. Kapu, who also is associate dean for clinical and community partnerships at Vanderbilt University, Nashville, Tenn. “It is important to respect each person’s entry point, education, and training.”
Differing personalities and environments
Numerous PA respondents said that individual personalities and work environments are more likely to trigger issues with NPs than are differences in training.
“It depends on the team and situation and who the people are, not the letters behind their names,” an emergency medicine PA wrote. A surgical PA noted that “group dynamics and work culture differ from place to place,” while a third PA agreed that “it’s personality dependent, not title dependent.”
No single formula will resolve areas of NP-PA conflict, Ms. Orozco said. “What works in Chicago might not work in rural Colorado or Texas or California, but we do have to come together. The overall focus should be on greater flexibility for PAs and NPs. Patients will fare better.”
Joint research, publishing could help
About a decade ago, Mr. Blumm joined with another PA and an NP to form the American College of Clinicians, the first joint PA-NP national professional organization. Although it disbanded after 6 years, owing to low membership, he hopes a similar collaboration will take off in the future.
“I also recommend that PAs and NPs publish articles together, with research as an excellent place to start,” he added. “PAs and NPs should stand together and be a source of healing for all our patients. Regardless of our titles, our responsibility is to bring healing together.”
A version of this article first appeared on Medscape.com.
– The U.S. Bureau of Labor Statistics forecasts a 40% increase in the NP workforce by 2031, coupled with a 28% rise in PAs.
In recent reports on the quality of the relationships involving these health care professions, survey respondents mostly gave positive accounts of collaboration, using words such as like “comradery,” “teamwork,” “congenial,” and “cohesion.” But all was not perfect. Where and how could these important health care provider relationships improve?
PAs: “Competition and collaboration’ with RNs
In a Medscape survey of more than 770 PAs about their working relationships with other health care professionals; 83% of them supported the idea of PAs and NPs practicing more independently from physicians, but sometimes it’s not easy to stay in their individual lanes.
One PA respondent complained that NPs get “more opportunities and preference,” another pointed to PA-NP “turf issues,” and a third griped about NPs’ “strong unions,” which have stoked more fighting about practice abilities and available settings.
Robert Blumm, MA, PA-C, a retired surgical and emergency medicine PA who regards himself as an advocate for both PAs and NPs, describes their interaction as a “mixture of competition and collaboration.”
On one hand, the two groups typically “cooperate and do an excellent job, incurring patient errors similar to or less than physician colleagues or senior residents.” On the other hand, Mr. Blumm conceded, there is some jealousy among PAs over NPs’ advantage in staffing and hiring decisions, “since they don’t need [direct physician] supervision ... and there are limits on how many PAs can be supervised by one physician.”
Most PA-NP interactions are collaborative, although many people emphasize the relatively few conflicts, said Jennifer Orozco, DMSc, PA-C, president and chair of the American Academy of PAs.
“We see that a lot in this country,” she said. “People try to drive a wedge, but it’s often a misnomer that there’s a lot of arguing and infighting.”
NPs: Different backgrounds, same goal
The Medscape survey also included information from 750 NPs on working relationships; 93% of them favored nurses and PAs working more independently from doctors.
April Kapu, DNP, ARPN, has worked closely with PAs for more than 20 years. “In my experience ... they complement one another as health team members, although the education and training are somewhat different,” said Ms. Kapu, , president of the American Association of Nurse Practitioners.
Some respondents noted the different educational trajectories for NPs and PAs. “Doctors and PAs are taught using the same model, but NPs are taught under the nursing model,” wrote a family medicine PA.
In emergency departments where Mr. Blumm has worked, ICU NPs have an edge over PAs in terms of preparation, organization, and the tabulation of formulas. On the other hand, some of Mr. Blumm’s fellow PAs were also emergency medicine technicians or respiratory therapists, who had “2 years of classroom training, on par with that of medical students.”
Must these differences in training and education foment conflict between NPs and PAs? “We all bring something different to the table,” said Ms. Kapu, who also is associate dean for clinical and community partnerships at Vanderbilt University, Nashville, Tenn. “It is important to respect each person’s entry point, education, and training.”
Differing personalities and environments
Numerous PA respondents said that individual personalities and work environments are more likely to trigger issues with NPs than are differences in training.
“It depends on the team and situation and who the people are, not the letters behind their names,” an emergency medicine PA wrote. A surgical PA noted that “group dynamics and work culture differ from place to place,” while a third PA agreed that “it’s personality dependent, not title dependent.”
No single formula will resolve areas of NP-PA conflict, Ms. Orozco said. “What works in Chicago might not work in rural Colorado or Texas or California, but we do have to come together. The overall focus should be on greater flexibility for PAs and NPs. Patients will fare better.”
Joint research, publishing could help
About a decade ago, Mr. Blumm joined with another PA and an NP to form the American College of Clinicians, the first joint PA-NP national professional organization. Although it disbanded after 6 years, owing to low membership, he hopes a similar collaboration will take off in the future.
“I also recommend that PAs and NPs publish articles together, with research as an excellent place to start,” he added. “PAs and NPs should stand together and be a source of healing for all our patients. Regardless of our titles, our responsibility is to bring healing together.”
A version of this article first appeared on Medscape.com.
Docs struggle to keep up with the flood of new medical knowledge. Here’s advice
making it much tougher for physicians to identify innovative findings and newer guidelines for helping patients. Yet not keeping up with the latest information can put doctors at risk.
“Most doctors are feeling lost about keeping up to date,” said John P.A. Ioannidis, MD, professor of medicine at Stanford (Calif.) University School of Medicine. “The vast majority of new studies are either wrong or not useful, but physicians cannot sort out which are those studies.”
The sheer number of new studies may even force some doctors to retreat from areas where they have not kept up, said Stephen A. Martin, MD, professor of family medicine and community health at the University of Massachusetts, Worcester. “When doctors don’t feel they can stay current, they may refer more cases to specialists or narrow their focus,” he said.
Some specialties have a greater challenge than others
Dr. Martin said the deluge of studies heavily impacts generalists because they have a wider field of information to keep up with. However, certain specialties like oncology are particularly flooded with new findings.
Specialties with the greatest number of published studies are reportedly oncology, cardiology, and neurology. A 2021 study found that the number of articles with the word “stroke” in them increased five times from 2000 to 2020. And investigative treatments targeting cancer nearly quadrupled just between 2010 and 2020.
What’s more, physicians spend a great deal of time sifting through studies that are ultimately useless. In a survey of internists by Univadis, which is part of WebMD/Medscape, 82% said that fewer than half of the studies they read actually had an impact on how they practice medicine.
“You often have to dig into an article and learn more about a finding before you now whether it’s useful,” Dr. Martin said. “And in the end, relatively few new findings are truly novel ones that are useful for patient care.”
So what can a physician do? First, find out what you don’t know
Looking for new findings needs to be carried out systematically, according to William B. Cutrer, MD, MEd, a pediatric intensivist who is associate dean for undergraduate medical education at Vanderbilt University School of Medicine, Nashville, Tenn.
“Before you start, you have to know what you don’t know, and that’s often not so easy,” he said. “You may get a spark about what you don’t know in an encounter with a patient or colleague or through patient outcomes data,” he said.
Dr. Martin, on the other hand, advocates a broad approach that involves finding out at least a little about everything in one’s field. “If you have a good base, you’re not starting from zero when you encounter a new clinical situation,” he said.
“The idea is that you don’t need to memorize most things, but you do need to know how to access them,” Dr. Martin said. “I memorize the things I do all the time, such as dosing or indicated testing, but I look up things that I don’t see that often and ones that have some complexity.”
Updating the old ways
For generations, doctors have stayed current by going to meetings, conversing with colleagues, and reading journals, but many physicians have updated these methods through various resources on the internet.
For example, meetings went virtual during the pandemic, and now that face-to-face meetings are back, many of them retain a virtual option, said Kevin Campbell, MD, a cardiologist at Health First Medical Group, Melbourne, Fla. “I typically go to one or two conferences a year, but I also learn a lot digitally,” he said.
As to journal reading, “assessing an article is an essential skill,” Dr. Cutrer said. “It’s important to quickly decide whether a journal article is worth reading or not. One answer to this problem is to consult summaries of important articles. But summaries are sometimes unhelpful, and it is hard to know which articles are significant. Therefore, doctors have been reaching out to others who can research the articles for them.”
For many years, some physicians have pooled their resources in journal clubs. “You get a chance to cross-cultivate your skills with others,” Dr. Ioannidis said. “But you need someone who is well informed and dedicated to run the journal club, using evidence-based principles.”
Dr. Cutrer said physicians like to cast their net wide because they are understandably wary of changing their practice based on one study. “Unless there is one large study that is really well designed, doctors will need two or more findings to be convinced,” he said. This requires having the ability to match studies across many journals.
Using research summaries
In the past two decades, physicians have gained access to countless summaries of journal articles prepared by armies of clinical experts working for review services such as the New England Journal of Medicine’s “Journal Watch,” Annals of Internal Medicine’s “In the Clinic,” and BMJ’s “State of the Arts.”
In addition to summarizing findings from a wide variety of journals in plain language, reviewers may compare them to similar studies and assess the validity of the finding by assigning a level of evidence.
Some commercial ventures provide similar services. Betsy Jones, executive vice president of clinical decisions at EBSCO, said the DynaMed service is now available through an app on the physician’s smartphone or through the electronic health record.
Physicians like this approach. Many specialists have noted that reading full-length articles was not an efficient use of their time, while even more said that reviews are efficient.
Exchanging information online
Physicians are increasingly keeping current by using the internet, especially on social media, Dr. Cutrer said. “Young doctors in particular are more likely to keep up digitally,” he said.
Internet-based information has become so widespread that disparities in health care from region to region have somewhat abated, according to Stuart J. Fischer, MD, an orthopedic surgeon at Summit Orthopaedics and Sports Medicine, New Jersey. “One positive outcome of this plethora of information today is that geographic disparities in clinical practice are not as great as they used to be,” he said.
Rather than chatting up colleagues in the hallway, many physicians have come to rely on internet-based discussion boards.
Blogs, podcasts, and Twitter
Blogs and podcasts, often focused on a specialty, can be a great way for physicians to keep up, said UMass Chan professor Dr. Martin. “Podcasts in particular have enhanced the ability to stay current,” he said. “You want to find someone you trust.”
Internal medicine podcasts include Annals on Call, where doctors discuss articles in the Annals of Internal Medicine, and the Curbsiders, where two internists interview a guest expert.
Orthopedic surgeons can visit podcasts like Nailed it, Orthobullets, the Ortho Show, and Inside Orthopedics. Neurologists can consult Brainwaves, Neurology Podcast, Practical Neurology Podcast, and Clinical Neurology with KD. And pediatricians can drop in on Talking Pediatrics, The Cribsiders, and PedsCases.
Meanwhile, Twitter has become a particularly effective way to broadcast new findings, speeding up the transition from the bench to the bedside, said Dr. Campbell, the Florida cardiologist.
“I visit cardio-specific resources on Twitter,” he said. “They can be real-time video chats or posted messages. They spur discussion like a journal club. Colleagues present cases and drop in and out of the discussion.”
Others are not as enthusiastic. Although Stanford’s Dr. Ioannidis is in the heart of the Silicon Valley, he is leery of some of the new digital methods. “I don’t use Twitter,” he says. “You just add more people to the process, which could only make things more confusing. I want to be able to think a lot about it.”
Cutting-edge knowledge at the point of care
Consulting the literature often takes place at the point of care, when a particular patient requires treatment. This can be done by using clinical decision support (CDS) and by using clinical practice guidelines (CPGs), which are typically developed by panels of doctors at specialty societies.
“It used to be that the doctor was expected to know everything,” said Ms. Jones at DynaMed. “Today there is no way to keep up with it all. Doctors often need a quick memory jog.”
Ms. Jones said the CDS result always requires the doctor’s interpretation. “It is up to the doctor to decide whether a new finding is the best choice for his or her patient,” she said.
Dr. Martin recommends going easy on point-of-care resources. “They can be used for showing a patient a differential diagnosis list or checking the cost of a procedure, but they are harder to use for novel developments that require time and context to evaluate their impact,” he said.
CPGs, meanwhile, have a high profile in the research world. In a 2018 study, Dr. Ioannidis found that 8 of the 15 most-cited articles were CPGs, disease definitions, or disease statistics.
Dr. Fischer said CPGs are typically based on thorough reviews of the literature, but they do involve experts’ interpretation of the science. “It can be difficult to obtain specific answers to some medical questions, especially for problems with complex treatments or variations,” he said.
As a result, Dr. Fischer said doctors have to use their judgment in applying CPGs to a specific patient. “For example, the orthopedic surgeon would normally recommend a total hip replacement for patients with a bad hip, but it might not be appropriate for an overweight patient.”
Stay skeptical
There are many novel ways for physicians to keep current, including summaries of articles, discussion boards, blogs, podcasts, Twitter, clinical decision support, and clinical practice guidelines.
Even with all these new services, though, doctors need to retain a healthy amount of skepticism about new research findings, Dr. Ioannidis said. “Ask yourself questions such as: Does it deal with a real problem? Am I getting the real information? Is it relevant to real patients? Is it offering good value for money?”
A version of this article first appeared on Medscape.com.
making it much tougher for physicians to identify innovative findings and newer guidelines for helping patients. Yet not keeping up with the latest information can put doctors at risk.
“Most doctors are feeling lost about keeping up to date,” said John P.A. Ioannidis, MD, professor of medicine at Stanford (Calif.) University School of Medicine. “The vast majority of new studies are either wrong or not useful, but physicians cannot sort out which are those studies.”
The sheer number of new studies may even force some doctors to retreat from areas where they have not kept up, said Stephen A. Martin, MD, professor of family medicine and community health at the University of Massachusetts, Worcester. “When doctors don’t feel they can stay current, they may refer more cases to specialists or narrow their focus,” he said.
Some specialties have a greater challenge than others
Dr. Martin said the deluge of studies heavily impacts generalists because they have a wider field of information to keep up with. However, certain specialties like oncology are particularly flooded with new findings.
Specialties with the greatest number of published studies are reportedly oncology, cardiology, and neurology. A 2021 study found that the number of articles with the word “stroke” in them increased five times from 2000 to 2020. And investigative treatments targeting cancer nearly quadrupled just between 2010 and 2020.
What’s more, physicians spend a great deal of time sifting through studies that are ultimately useless. In a survey of internists by Univadis, which is part of WebMD/Medscape, 82% said that fewer than half of the studies they read actually had an impact on how they practice medicine.
“You often have to dig into an article and learn more about a finding before you now whether it’s useful,” Dr. Martin said. “And in the end, relatively few new findings are truly novel ones that are useful for patient care.”
So what can a physician do? First, find out what you don’t know
Looking for new findings needs to be carried out systematically, according to William B. Cutrer, MD, MEd, a pediatric intensivist who is associate dean for undergraduate medical education at Vanderbilt University School of Medicine, Nashville, Tenn.
“Before you start, you have to know what you don’t know, and that’s often not so easy,” he said. “You may get a spark about what you don’t know in an encounter with a patient or colleague or through patient outcomes data,” he said.
Dr. Martin, on the other hand, advocates a broad approach that involves finding out at least a little about everything in one’s field. “If you have a good base, you’re not starting from zero when you encounter a new clinical situation,” he said.
“The idea is that you don’t need to memorize most things, but you do need to know how to access them,” Dr. Martin said. “I memorize the things I do all the time, such as dosing or indicated testing, but I look up things that I don’t see that often and ones that have some complexity.”
Updating the old ways
For generations, doctors have stayed current by going to meetings, conversing with colleagues, and reading journals, but many physicians have updated these methods through various resources on the internet.
For example, meetings went virtual during the pandemic, and now that face-to-face meetings are back, many of them retain a virtual option, said Kevin Campbell, MD, a cardiologist at Health First Medical Group, Melbourne, Fla. “I typically go to one or two conferences a year, but I also learn a lot digitally,” he said.
As to journal reading, “assessing an article is an essential skill,” Dr. Cutrer said. “It’s important to quickly decide whether a journal article is worth reading or not. One answer to this problem is to consult summaries of important articles. But summaries are sometimes unhelpful, and it is hard to know which articles are significant. Therefore, doctors have been reaching out to others who can research the articles for them.”
For many years, some physicians have pooled their resources in journal clubs. “You get a chance to cross-cultivate your skills with others,” Dr. Ioannidis said. “But you need someone who is well informed and dedicated to run the journal club, using evidence-based principles.”
Dr. Cutrer said physicians like to cast their net wide because they are understandably wary of changing their practice based on one study. “Unless there is one large study that is really well designed, doctors will need two or more findings to be convinced,” he said. This requires having the ability to match studies across many journals.
Using research summaries
In the past two decades, physicians have gained access to countless summaries of journal articles prepared by armies of clinical experts working for review services such as the New England Journal of Medicine’s “Journal Watch,” Annals of Internal Medicine’s “In the Clinic,” and BMJ’s “State of the Arts.”
In addition to summarizing findings from a wide variety of journals in plain language, reviewers may compare them to similar studies and assess the validity of the finding by assigning a level of evidence.
Some commercial ventures provide similar services. Betsy Jones, executive vice president of clinical decisions at EBSCO, said the DynaMed service is now available through an app on the physician’s smartphone or through the electronic health record.
Physicians like this approach. Many specialists have noted that reading full-length articles was not an efficient use of their time, while even more said that reviews are efficient.
Exchanging information online
Physicians are increasingly keeping current by using the internet, especially on social media, Dr. Cutrer said. “Young doctors in particular are more likely to keep up digitally,” he said.
Internet-based information has become so widespread that disparities in health care from region to region have somewhat abated, according to Stuart J. Fischer, MD, an orthopedic surgeon at Summit Orthopaedics and Sports Medicine, New Jersey. “One positive outcome of this plethora of information today is that geographic disparities in clinical practice are not as great as they used to be,” he said.
Rather than chatting up colleagues in the hallway, many physicians have come to rely on internet-based discussion boards.
Blogs, podcasts, and Twitter
Blogs and podcasts, often focused on a specialty, can be a great way for physicians to keep up, said UMass Chan professor Dr. Martin. “Podcasts in particular have enhanced the ability to stay current,” he said. “You want to find someone you trust.”
Internal medicine podcasts include Annals on Call, where doctors discuss articles in the Annals of Internal Medicine, and the Curbsiders, where two internists interview a guest expert.
Orthopedic surgeons can visit podcasts like Nailed it, Orthobullets, the Ortho Show, and Inside Orthopedics. Neurologists can consult Brainwaves, Neurology Podcast, Practical Neurology Podcast, and Clinical Neurology with KD. And pediatricians can drop in on Talking Pediatrics, The Cribsiders, and PedsCases.
Meanwhile, Twitter has become a particularly effective way to broadcast new findings, speeding up the transition from the bench to the bedside, said Dr. Campbell, the Florida cardiologist.
“I visit cardio-specific resources on Twitter,” he said. “They can be real-time video chats or posted messages. They spur discussion like a journal club. Colleagues present cases and drop in and out of the discussion.”
Others are not as enthusiastic. Although Stanford’s Dr. Ioannidis is in the heart of the Silicon Valley, he is leery of some of the new digital methods. “I don’t use Twitter,” he says. “You just add more people to the process, which could only make things more confusing. I want to be able to think a lot about it.”
Cutting-edge knowledge at the point of care
Consulting the literature often takes place at the point of care, when a particular patient requires treatment. This can be done by using clinical decision support (CDS) and by using clinical practice guidelines (CPGs), which are typically developed by panels of doctors at specialty societies.
“It used to be that the doctor was expected to know everything,” said Ms. Jones at DynaMed. “Today there is no way to keep up with it all. Doctors often need a quick memory jog.”
Ms. Jones said the CDS result always requires the doctor’s interpretation. “It is up to the doctor to decide whether a new finding is the best choice for his or her patient,” she said.
Dr. Martin recommends going easy on point-of-care resources. “They can be used for showing a patient a differential diagnosis list or checking the cost of a procedure, but they are harder to use for novel developments that require time and context to evaluate their impact,” he said.
CPGs, meanwhile, have a high profile in the research world. In a 2018 study, Dr. Ioannidis found that 8 of the 15 most-cited articles were CPGs, disease definitions, or disease statistics.
Dr. Fischer said CPGs are typically based on thorough reviews of the literature, but they do involve experts’ interpretation of the science. “It can be difficult to obtain specific answers to some medical questions, especially for problems with complex treatments or variations,” he said.
As a result, Dr. Fischer said doctors have to use their judgment in applying CPGs to a specific patient. “For example, the orthopedic surgeon would normally recommend a total hip replacement for patients with a bad hip, but it might not be appropriate for an overweight patient.”
Stay skeptical
There are many novel ways for physicians to keep current, including summaries of articles, discussion boards, blogs, podcasts, Twitter, clinical decision support, and clinical practice guidelines.
Even with all these new services, though, doctors need to retain a healthy amount of skepticism about new research findings, Dr. Ioannidis said. “Ask yourself questions such as: Does it deal with a real problem? Am I getting the real information? Is it relevant to real patients? Is it offering good value for money?”
A version of this article first appeared on Medscape.com.
making it much tougher for physicians to identify innovative findings and newer guidelines for helping patients. Yet not keeping up with the latest information can put doctors at risk.
“Most doctors are feeling lost about keeping up to date,” said John P.A. Ioannidis, MD, professor of medicine at Stanford (Calif.) University School of Medicine. “The vast majority of new studies are either wrong or not useful, but physicians cannot sort out which are those studies.”
The sheer number of new studies may even force some doctors to retreat from areas where they have not kept up, said Stephen A. Martin, MD, professor of family medicine and community health at the University of Massachusetts, Worcester. “When doctors don’t feel they can stay current, they may refer more cases to specialists or narrow their focus,” he said.
Some specialties have a greater challenge than others
Dr. Martin said the deluge of studies heavily impacts generalists because they have a wider field of information to keep up with. However, certain specialties like oncology are particularly flooded with new findings.
Specialties with the greatest number of published studies are reportedly oncology, cardiology, and neurology. A 2021 study found that the number of articles with the word “stroke” in them increased five times from 2000 to 2020. And investigative treatments targeting cancer nearly quadrupled just between 2010 and 2020.
What’s more, physicians spend a great deal of time sifting through studies that are ultimately useless. In a survey of internists by Univadis, which is part of WebMD/Medscape, 82% said that fewer than half of the studies they read actually had an impact on how they practice medicine.
“You often have to dig into an article and learn more about a finding before you now whether it’s useful,” Dr. Martin said. “And in the end, relatively few new findings are truly novel ones that are useful for patient care.”
So what can a physician do? First, find out what you don’t know
Looking for new findings needs to be carried out systematically, according to William B. Cutrer, MD, MEd, a pediatric intensivist who is associate dean for undergraduate medical education at Vanderbilt University School of Medicine, Nashville, Tenn.
“Before you start, you have to know what you don’t know, and that’s often not so easy,” he said. “You may get a spark about what you don’t know in an encounter with a patient or colleague or through patient outcomes data,” he said.
Dr. Martin, on the other hand, advocates a broad approach that involves finding out at least a little about everything in one’s field. “If you have a good base, you’re not starting from zero when you encounter a new clinical situation,” he said.
“The idea is that you don’t need to memorize most things, but you do need to know how to access them,” Dr. Martin said. “I memorize the things I do all the time, such as dosing or indicated testing, but I look up things that I don’t see that often and ones that have some complexity.”
Updating the old ways
For generations, doctors have stayed current by going to meetings, conversing with colleagues, and reading journals, but many physicians have updated these methods through various resources on the internet.
For example, meetings went virtual during the pandemic, and now that face-to-face meetings are back, many of them retain a virtual option, said Kevin Campbell, MD, a cardiologist at Health First Medical Group, Melbourne, Fla. “I typically go to one or two conferences a year, but I also learn a lot digitally,” he said.
As to journal reading, “assessing an article is an essential skill,” Dr. Cutrer said. “It’s important to quickly decide whether a journal article is worth reading or not. One answer to this problem is to consult summaries of important articles. But summaries are sometimes unhelpful, and it is hard to know which articles are significant. Therefore, doctors have been reaching out to others who can research the articles for them.”
For many years, some physicians have pooled their resources in journal clubs. “You get a chance to cross-cultivate your skills with others,” Dr. Ioannidis said. “But you need someone who is well informed and dedicated to run the journal club, using evidence-based principles.”
Dr. Cutrer said physicians like to cast their net wide because they are understandably wary of changing their practice based on one study. “Unless there is one large study that is really well designed, doctors will need two or more findings to be convinced,” he said. This requires having the ability to match studies across many journals.
Using research summaries
In the past two decades, physicians have gained access to countless summaries of journal articles prepared by armies of clinical experts working for review services such as the New England Journal of Medicine’s “Journal Watch,” Annals of Internal Medicine’s “In the Clinic,” and BMJ’s “State of the Arts.”
In addition to summarizing findings from a wide variety of journals in plain language, reviewers may compare them to similar studies and assess the validity of the finding by assigning a level of evidence.
Some commercial ventures provide similar services. Betsy Jones, executive vice president of clinical decisions at EBSCO, said the DynaMed service is now available through an app on the physician’s smartphone or through the electronic health record.
Physicians like this approach. Many specialists have noted that reading full-length articles was not an efficient use of their time, while even more said that reviews are efficient.
Exchanging information online
Physicians are increasingly keeping current by using the internet, especially on social media, Dr. Cutrer said. “Young doctors in particular are more likely to keep up digitally,” he said.
Internet-based information has become so widespread that disparities in health care from region to region have somewhat abated, according to Stuart J. Fischer, MD, an orthopedic surgeon at Summit Orthopaedics and Sports Medicine, New Jersey. “One positive outcome of this plethora of information today is that geographic disparities in clinical practice are not as great as they used to be,” he said.
Rather than chatting up colleagues in the hallway, many physicians have come to rely on internet-based discussion boards.
Blogs, podcasts, and Twitter
Blogs and podcasts, often focused on a specialty, can be a great way for physicians to keep up, said UMass Chan professor Dr. Martin. “Podcasts in particular have enhanced the ability to stay current,” he said. “You want to find someone you trust.”
Internal medicine podcasts include Annals on Call, where doctors discuss articles in the Annals of Internal Medicine, and the Curbsiders, where two internists interview a guest expert.
Orthopedic surgeons can visit podcasts like Nailed it, Orthobullets, the Ortho Show, and Inside Orthopedics. Neurologists can consult Brainwaves, Neurology Podcast, Practical Neurology Podcast, and Clinical Neurology with KD. And pediatricians can drop in on Talking Pediatrics, The Cribsiders, and PedsCases.
Meanwhile, Twitter has become a particularly effective way to broadcast new findings, speeding up the transition from the bench to the bedside, said Dr. Campbell, the Florida cardiologist.
“I visit cardio-specific resources on Twitter,” he said. “They can be real-time video chats or posted messages. They spur discussion like a journal club. Colleagues present cases and drop in and out of the discussion.”
Others are not as enthusiastic. Although Stanford’s Dr. Ioannidis is in the heart of the Silicon Valley, he is leery of some of the new digital methods. “I don’t use Twitter,” he says. “You just add more people to the process, which could only make things more confusing. I want to be able to think a lot about it.”
Cutting-edge knowledge at the point of care
Consulting the literature often takes place at the point of care, when a particular patient requires treatment. This can be done by using clinical decision support (CDS) and by using clinical practice guidelines (CPGs), which are typically developed by panels of doctors at specialty societies.
“It used to be that the doctor was expected to know everything,” said Ms. Jones at DynaMed. “Today there is no way to keep up with it all. Doctors often need a quick memory jog.”
Ms. Jones said the CDS result always requires the doctor’s interpretation. “It is up to the doctor to decide whether a new finding is the best choice for his or her patient,” she said.
Dr. Martin recommends going easy on point-of-care resources. “They can be used for showing a patient a differential diagnosis list or checking the cost of a procedure, but they are harder to use for novel developments that require time and context to evaluate their impact,” he said.
CPGs, meanwhile, have a high profile in the research world. In a 2018 study, Dr. Ioannidis found that 8 of the 15 most-cited articles were CPGs, disease definitions, or disease statistics.
Dr. Fischer said CPGs are typically based on thorough reviews of the literature, but they do involve experts’ interpretation of the science. “It can be difficult to obtain specific answers to some medical questions, especially for problems with complex treatments or variations,” he said.
As a result, Dr. Fischer said doctors have to use their judgment in applying CPGs to a specific patient. “For example, the orthopedic surgeon would normally recommend a total hip replacement for patients with a bad hip, but it might not be appropriate for an overweight patient.”
Stay skeptical
There are many novel ways for physicians to keep current, including summaries of articles, discussion boards, blogs, podcasts, Twitter, clinical decision support, and clinical practice guidelines.
Even with all these new services, though, doctors need to retain a healthy amount of skepticism about new research findings, Dr. Ioannidis said. “Ask yourself questions such as: Does it deal with a real problem? Am I getting the real information? Is it relevant to real patients? Is it offering good value for money?”
A version of this article first appeared on Medscape.com.
Transplant surgeon to 30,000 marathoners: Give me that liver
Surgeon goes the extra half mile for his patient
Sorry medical profession, but it’s Adam Bodzin’s world now. When a donor liver got stuck in the middle of the Philadelphia Half Marathon’s 30,000 participants, Dr. Bodzin, the transplant team’s lead surgeon, took matters into his own hands. And by hands, of course, we mean feet.
Still wearing his hospital scrubs, Dr. Bodzin ran more than half a mile to where the van carrying the liver was stranded, according to the Philadelphia Inquirer. Fortunately, he was able to hitch a ride in a police car for the return trip and didn’t have to run back through the crowd carrying his somewhat unusual package. By package, of course, we mean human liver.
It’s been 3 months since the surgery/marathon and it’s still not clear why the driver had such trouble getting through – he had been trying for more than an hour and half by the time Dr. Bodzin reached him – but the surgery half of the big event was deemed a success and the patient has recovered.
Rick Hasz, president and chief executive officer of the Gift of Life Donor Program, which coordinates organ donation for transplants in the Philadelphia region, told the newspaper that “Dr. Bodzin’s quick action demonstrated his commitment to honoring the selfless generosity of all donors and their families and gives hope to everyone waiting for a second chance at life.”
Should Dr. Bodzin consider a step up from the transplant team to another group that’s fighting for the common good? The recipient of the liver in question seems to think so. “I guess he has a cape on under that white jacket,” 66-year-old Charles Rowe told Fox29. You already know where we’re going with this, right?
Avengers … Assemble.
Your spleen’s due for its 5,000-mile oil change
The human body is an incredible biological machine, capable of performing a countless array of tasks automatically and essentially without flaw, but there’s always room for improvement. After all, there are animals that can regrow entire missing limbs or live for up to 500 years. It would be nice if we could get some of that going.
Rather than any of that cool stuff, a recent survey of 2,000 average Americans revealed that our ambitions for improving the human body are a bit more mundane. The big thing that would make our lives better and easier, according to three-fourths of Americans, would be a built-in “check engine” light in our bodies. Come on guys, starfish can literally be cut in half and not only survive, but become two starfish. Mantis shrimp can punch with a force thousands of times their own weight. If we could punch like they could, we could literally break steel with our fists. Wouldn’t we rather have that?
Apparently not. Fine, we’ll stick with the check engine light.
Maybe it isn’t a huge surprise that we’d like the extra help in figuring out what our body needs. According to the survey, more than 60% of Americans struggle to identify when their body is trying to tell them something important, and only one-third actively checked in with their health every day. Considering about 40% said they feel tired for much of the day and nearly half reported not having a meal with fruits or vegetables in the past 3 days, perhaps a gentle reminder wouldn’t be the worst thing in the world.
So, if we did have a built-in check engine light, what would we use it for? A majority said they’d like to be reminded to drink a glass of water, with 45% saying they wanted to know when to take a nap. Feeling thirsty or tired isn’t quite enough, it seems.
Of course, the technology certainly exists to make the human check engine light a reality. An implanted microchip could absolutely tell us to drink a glass of water, but that would put our health in the hands of tech companies, and you just know Meta and Elon Muskrat wouldn’t pass up the chance for monetization. “Oh, sorry, we could have notified the hospital that you were about to have a heart attack, but you didn’t pay your life subscription this month.”
Sext offenders show more than their, well, you know
As we have become more and more attached to our phones, especially post pandemic, it’s no surprise that sexting – sending sexually explicit images and messages with those phones – has become a fairly common way for people to sexually communicate. And with dating apps just another venture in the dating landscape, regardless of age, sexting is an easy avenue to incite a mood without being physically present.
A recent study, though, has linked sexting with anxiety, sleep issues, depression, and compulsive sexual behaviors. Yikes.
Although the researchers noted that sexting was primarily reciprocal (sending and receiving), “over 50% of adults report sending a sext, while women are up to four times more likely than men to report having received nonconsensual sexts,” said Brenda K. Wiederhold, PhD, editor-in-chief of Cyberpsychology, Behavior, and Social Networking, which published the study, in which Dr. Wiederhold was not involved.
Among the 2,160 U.S. college students who were involved, participants who had only sent sexts reported more anxiety, depression, and sleep problems than other groups (no sexting, received only, reciprocal). There was also a possible connection between sexting, marijuana use, and compulsive sexual behavior, the investigators said in a written statement.
Considering the study population, these data are perhaps not that surprising. For young adults, to receive or send an elusive nude is as common as it once was to give someone flowers. Not that the two things elicit the same reactions. “Many individuals reveal they enjoy consensual sexting and feel it empowers them and builds self-confidence,” Dr. Wiederhold added.
Receiving a nonconsensual sext, though, is definitely going to result in feeling violated and super awkward. Senders beware: Don’t be surprised if you’re ghosted after that.
Surgeon goes the extra half mile for his patient
Sorry medical profession, but it’s Adam Bodzin’s world now. When a donor liver got stuck in the middle of the Philadelphia Half Marathon’s 30,000 participants, Dr. Bodzin, the transplant team’s lead surgeon, took matters into his own hands. And by hands, of course, we mean feet.
Still wearing his hospital scrubs, Dr. Bodzin ran more than half a mile to where the van carrying the liver was stranded, according to the Philadelphia Inquirer. Fortunately, he was able to hitch a ride in a police car for the return trip and didn’t have to run back through the crowd carrying his somewhat unusual package. By package, of course, we mean human liver.
It’s been 3 months since the surgery/marathon and it’s still not clear why the driver had such trouble getting through – he had been trying for more than an hour and half by the time Dr. Bodzin reached him – but the surgery half of the big event was deemed a success and the patient has recovered.
Rick Hasz, president and chief executive officer of the Gift of Life Donor Program, which coordinates organ donation for transplants in the Philadelphia region, told the newspaper that “Dr. Bodzin’s quick action demonstrated his commitment to honoring the selfless generosity of all donors and their families and gives hope to everyone waiting for a second chance at life.”
Should Dr. Bodzin consider a step up from the transplant team to another group that’s fighting for the common good? The recipient of the liver in question seems to think so. “I guess he has a cape on under that white jacket,” 66-year-old Charles Rowe told Fox29. You already know where we’re going with this, right?
Avengers … Assemble.
Your spleen’s due for its 5,000-mile oil change
The human body is an incredible biological machine, capable of performing a countless array of tasks automatically and essentially without flaw, but there’s always room for improvement. After all, there are animals that can regrow entire missing limbs or live for up to 500 years. It would be nice if we could get some of that going.
Rather than any of that cool stuff, a recent survey of 2,000 average Americans revealed that our ambitions for improving the human body are a bit more mundane. The big thing that would make our lives better and easier, according to three-fourths of Americans, would be a built-in “check engine” light in our bodies. Come on guys, starfish can literally be cut in half and not only survive, but become two starfish. Mantis shrimp can punch with a force thousands of times their own weight. If we could punch like they could, we could literally break steel with our fists. Wouldn’t we rather have that?
Apparently not. Fine, we’ll stick with the check engine light.
Maybe it isn’t a huge surprise that we’d like the extra help in figuring out what our body needs. According to the survey, more than 60% of Americans struggle to identify when their body is trying to tell them something important, and only one-third actively checked in with their health every day. Considering about 40% said they feel tired for much of the day and nearly half reported not having a meal with fruits or vegetables in the past 3 days, perhaps a gentle reminder wouldn’t be the worst thing in the world.
So, if we did have a built-in check engine light, what would we use it for? A majority said they’d like to be reminded to drink a glass of water, with 45% saying they wanted to know when to take a nap. Feeling thirsty or tired isn’t quite enough, it seems.
Of course, the technology certainly exists to make the human check engine light a reality. An implanted microchip could absolutely tell us to drink a glass of water, but that would put our health in the hands of tech companies, and you just know Meta and Elon Muskrat wouldn’t pass up the chance for monetization. “Oh, sorry, we could have notified the hospital that you were about to have a heart attack, but you didn’t pay your life subscription this month.”
Sext offenders show more than their, well, you know
As we have become more and more attached to our phones, especially post pandemic, it’s no surprise that sexting – sending sexually explicit images and messages with those phones – has become a fairly common way for people to sexually communicate. And with dating apps just another venture in the dating landscape, regardless of age, sexting is an easy avenue to incite a mood without being physically present.
A recent study, though, has linked sexting with anxiety, sleep issues, depression, and compulsive sexual behaviors. Yikes.
Although the researchers noted that sexting was primarily reciprocal (sending and receiving), “over 50% of adults report sending a sext, while women are up to four times more likely than men to report having received nonconsensual sexts,” said Brenda K. Wiederhold, PhD, editor-in-chief of Cyberpsychology, Behavior, and Social Networking, which published the study, in which Dr. Wiederhold was not involved.
Among the 2,160 U.S. college students who were involved, participants who had only sent sexts reported more anxiety, depression, and sleep problems than other groups (no sexting, received only, reciprocal). There was also a possible connection between sexting, marijuana use, and compulsive sexual behavior, the investigators said in a written statement.
Considering the study population, these data are perhaps not that surprising. For young adults, to receive or send an elusive nude is as common as it once was to give someone flowers. Not that the two things elicit the same reactions. “Many individuals reveal they enjoy consensual sexting and feel it empowers them and builds self-confidence,” Dr. Wiederhold added.
Receiving a nonconsensual sext, though, is definitely going to result in feeling violated and super awkward. Senders beware: Don’t be surprised if you’re ghosted after that.
Surgeon goes the extra half mile for his patient
Sorry medical profession, but it’s Adam Bodzin’s world now. When a donor liver got stuck in the middle of the Philadelphia Half Marathon’s 30,000 participants, Dr. Bodzin, the transplant team’s lead surgeon, took matters into his own hands. And by hands, of course, we mean feet.
Still wearing his hospital scrubs, Dr. Bodzin ran more than half a mile to where the van carrying the liver was stranded, according to the Philadelphia Inquirer. Fortunately, he was able to hitch a ride in a police car for the return trip and didn’t have to run back through the crowd carrying his somewhat unusual package. By package, of course, we mean human liver.
It’s been 3 months since the surgery/marathon and it’s still not clear why the driver had such trouble getting through – he had been trying for more than an hour and half by the time Dr. Bodzin reached him – but the surgery half of the big event was deemed a success and the patient has recovered.
Rick Hasz, president and chief executive officer of the Gift of Life Donor Program, which coordinates organ donation for transplants in the Philadelphia region, told the newspaper that “Dr. Bodzin’s quick action demonstrated his commitment to honoring the selfless generosity of all donors and their families and gives hope to everyone waiting for a second chance at life.”
Should Dr. Bodzin consider a step up from the transplant team to another group that’s fighting for the common good? The recipient of the liver in question seems to think so. “I guess he has a cape on under that white jacket,” 66-year-old Charles Rowe told Fox29. You already know where we’re going with this, right?
Avengers … Assemble.
Your spleen’s due for its 5,000-mile oil change
The human body is an incredible biological machine, capable of performing a countless array of tasks automatically and essentially without flaw, but there’s always room for improvement. After all, there are animals that can regrow entire missing limbs or live for up to 500 years. It would be nice if we could get some of that going.
Rather than any of that cool stuff, a recent survey of 2,000 average Americans revealed that our ambitions for improving the human body are a bit more mundane. The big thing that would make our lives better and easier, according to three-fourths of Americans, would be a built-in “check engine” light in our bodies. Come on guys, starfish can literally be cut in half and not only survive, but become two starfish. Mantis shrimp can punch with a force thousands of times their own weight. If we could punch like they could, we could literally break steel with our fists. Wouldn’t we rather have that?
Apparently not. Fine, we’ll stick with the check engine light.
Maybe it isn’t a huge surprise that we’d like the extra help in figuring out what our body needs. According to the survey, more than 60% of Americans struggle to identify when their body is trying to tell them something important, and only one-third actively checked in with their health every day. Considering about 40% said they feel tired for much of the day and nearly half reported not having a meal with fruits or vegetables in the past 3 days, perhaps a gentle reminder wouldn’t be the worst thing in the world.
So, if we did have a built-in check engine light, what would we use it for? A majority said they’d like to be reminded to drink a glass of water, with 45% saying they wanted to know when to take a nap. Feeling thirsty or tired isn’t quite enough, it seems.
Of course, the technology certainly exists to make the human check engine light a reality. An implanted microchip could absolutely tell us to drink a glass of water, but that would put our health in the hands of tech companies, and you just know Meta and Elon Muskrat wouldn’t pass up the chance for monetization. “Oh, sorry, we could have notified the hospital that you were about to have a heart attack, but you didn’t pay your life subscription this month.”
Sext offenders show more than their, well, you know
As we have become more and more attached to our phones, especially post pandemic, it’s no surprise that sexting – sending sexually explicit images and messages with those phones – has become a fairly common way for people to sexually communicate. And with dating apps just another venture in the dating landscape, regardless of age, sexting is an easy avenue to incite a mood without being physically present.
A recent study, though, has linked sexting with anxiety, sleep issues, depression, and compulsive sexual behaviors. Yikes.
Although the researchers noted that sexting was primarily reciprocal (sending and receiving), “over 50% of adults report sending a sext, while women are up to four times more likely than men to report having received nonconsensual sexts,” said Brenda K. Wiederhold, PhD, editor-in-chief of Cyberpsychology, Behavior, and Social Networking, which published the study, in which Dr. Wiederhold was not involved.
Among the 2,160 U.S. college students who were involved, participants who had only sent sexts reported more anxiety, depression, and sleep problems than other groups (no sexting, received only, reciprocal). There was also a possible connection between sexting, marijuana use, and compulsive sexual behavior, the investigators said in a written statement.
Considering the study population, these data are perhaps not that surprising. For young adults, to receive or send an elusive nude is as common as it once was to give someone flowers. Not that the two things elicit the same reactions. “Many individuals reveal they enjoy consensual sexting and feel it empowers them and builds self-confidence,” Dr. Wiederhold added.
Receiving a nonconsensual sext, though, is definitely going to result in feeling violated and super awkward. Senders beware: Don’t be surprised if you’re ghosted after that.
Physician pleads guilty to 52 counts in opioid scheme
Jeffrey B. Sutton, DO, a neuromuscular medicine specialist, pled guilty on January 30 in federal court to 31 counts of illegally prescribing opioids and other controlled substances, 1 count of illegally distributing controlled substances, and 20 counts of health care fraud.
Prosecutors said Dr. Sutton admitted that he ignored warnings from prescription drug management organizations, insurers, and state authorities that he was prescribing excessively high dosages of opioids.
Dr. Sutton also admitted to ignoring patient requests to lower dosages and that he also ignored signs that patients were selling prescribed medications or otherwise engaging in illicit activity, including violations of a “pain management agreement” that he required them to sign.
The fraud counts pertained to Dr. Sutton billing Medicare, Medicaid, and other insurers for medically unnecessary visits that he required of patients so that he could prescribe inappropriate or unnecessary opioids.
In the charging document shared with this news organization, prosecutors said Dr. Sutton had sex with at least three patients, including during office visits and outside of the office. Occasionally, the physician would give opioids or other controlled substances – often benzodiazepines – to these patients, without a prescription or valid medical need.
Dr. Sutton escalated the dosage for one of those patients, even as the subjective pain score did not improve and when the patient’s urine tests showed the presence of THC and buprenorphine, but not any of the prescribed medications.
Another patient came to Dr. Sutton in 2007 with a warning that she had a history of “narcotic-seeking” behavior and diagnoses of depression, anxiety, paranoid schizophrenia, and obsessive-compulsive disorder.
The patient was hospitalized in 2018 for complications from benzodiazepine use (prescribed by Dr. Sutton). She weighed 80 pounds at the time. Dr. Sutton continued to prescribe benzodiazepines and extreme doses of opioids – in excess of 2,000 morphine equivalent dose – “despite recognizing and documenting repeated instances of noncompliance with treatment for psychiatric conditions, and despite the known contraindications of long-term opioid use for patients with these mental illnesses,” according to the charging document.
Dr. Sutton continued to prescribe opioids despite two hospitalizations for overdoses, more than 20 failed urine drug screens that showed presence of illicit drugs such as cocaine, and documented excessive use of alprazolam (Xanax) and methadone.
The physician surrendered his Drug Enforcement Administration Certificate of Registration of Controlled Substances Privileges in February 2022 “as an indication of your good faith in desiring to remedy any incorrect or unlawful practices on your part,” according to a letter to Dr. Sutton from the State Medical Board of Ohio. In that September 2022 letter, the Board notified Dr. Sutton of its intention to possibly suspend or revoke his license.
Dr. Sutton did not request a hearing, and the Board permanently revoked his medical license on January 16.
The court will sentence Dr. Sutton on May 23, according to a report by WFMJ.
A version of this article originally appeared on Medscape.com.
Jeffrey B. Sutton, DO, a neuromuscular medicine specialist, pled guilty on January 30 in federal court to 31 counts of illegally prescribing opioids and other controlled substances, 1 count of illegally distributing controlled substances, and 20 counts of health care fraud.
Prosecutors said Dr. Sutton admitted that he ignored warnings from prescription drug management organizations, insurers, and state authorities that he was prescribing excessively high dosages of opioids.
Dr. Sutton also admitted to ignoring patient requests to lower dosages and that he also ignored signs that patients were selling prescribed medications or otherwise engaging in illicit activity, including violations of a “pain management agreement” that he required them to sign.
The fraud counts pertained to Dr. Sutton billing Medicare, Medicaid, and other insurers for medically unnecessary visits that he required of patients so that he could prescribe inappropriate or unnecessary opioids.
In the charging document shared with this news organization, prosecutors said Dr. Sutton had sex with at least three patients, including during office visits and outside of the office. Occasionally, the physician would give opioids or other controlled substances – often benzodiazepines – to these patients, without a prescription or valid medical need.
Dr. Sutton escalated the dosage for one of those patients, even as the subjective pain score did not improve and when the patient’s urine tests showed the presence of THC and buprenorphine, but not any of the prescribed medications.
Another patient came to Dr. Sutton in 2007 with a warning that she had a history of “narcotic-seeking” behavior and diagnoses of depression, anxiety, paranoid schizophrenia, and obsessive-compulsive disorder.
The patient was hospitalized in 2018 for complications from benzodiazepine use (prescribed by Dr. Sutton). She weighed 80 pounds at the time. Dr. Sutton continued to prescribe benzodiazepines and extreme doses of opioids – in excess of 2,000 morphine equivalent dose – “despite recognizing and documenting repeated instances of noncompliance with treatment for psychiatric conditions, and despite the known contraindications of long-term opioid use for patients with these mental illnesses,” according to the charging document.
Dr. Sutton continued to prescribe opioids despite two hospitalizations for overdoses, more than 20 failed urine drug screens that showed presence of illicit drugs such as cocaine, and documented excessive use of alprazolam (Xanax) and methadone.
The physician surrendered his Drug Enforcement Administration Certificate of Registration of Controlled Substances Privileges in February 2022 “as an indication of your good faith in desiring to remedy any incorrect or unlawful practices on your part,” according to a letter to Dr. Sutton from the State Medical Board of Ohio. In that September 2022 letter, the Board notified Dr. Sutton of its intention to possibly suspend or revoke his license.
Dr. Sutton did not request a hearing, and the Board permanently revoked his medical license on January 16.
The court will sentence Dr. Sutton on May 23, according to a report by WFMJ.
A version of this article originally appeared on Medscape.com.
Jeffrey B. Sutton, DO, a neuromuscular medicine specialist, pled guilty on January 30 in federal court to 31 counts of illegally prescribing opioids and other controlled substances, 1 count of illegally distributing controlled substances, and 20 counts of health care fraud.
Prosecutors said Dr. Sutton admitted that he ignored warnings from prescription drug management organizations, insurers, and state authorities that he was prescribing excessively high dosages of opioids.
Dr. Sutton also admitted to ignoring patient requests to lower dosages and that he also ignored signs that patients were selling prescribed medications or otherwise engaging in illicit activity, including violations of a “pain management agreement” that he required them to sign.
The fraud counts pertained to Dr. Sutton billing Medicare, Medicaid, and other insurers for medically unnecessary visits that he required of patients so that he could prescribe inappropriate or unnecessary opioids.
In the charging document shared with this news organization, prosecutors said Dr. Sutton had sex with at least three patients, including during office visits and outside of the office. Occasionally, the physician would give opioids or other controlled substances – often benzodiazepines – to these patients, without a prescription or valid medical need.
Dr. Sutton escalated the dosage for one of those patients, even as the subjective pain score did not improve and when the patient’s urine tests showed the presence of THC and buprenorphine, but not any of the prescribed medications.
Another patient came to Dr. Sutton in 2007 with a warning that she had a history of “narcotic-seeking” behavior and diagnoses of depression, anxiety, paranoid schizophrenia, and obsessive-compulsive disorder.
The patient was hospitalized in 2018 for complications from benzodiazepine use (prescribed by Dr. Sutton). She weighed 80 pounds at the time. Dr. Sutton continued to prescribe benzodiazepines and extreme doses of opioids – in excess of 2,000 morphine equivalent dose – “despite recognizing and documenting repeated instances of noncompliance with treatment for psychiatric conditions, and despite the known contraindications of long-term opioid use for patients with these mental illnesses,” according to the charging document.
Dr. Sutton continued to prescribe opioids despite two hospitalizations for overdoses, more than 20 failed urine drug screens that showed presence of illicit drugs such as cocaine, and documented excessive use of alprazolam (Xanax) and methadone.
The physician surrendered his Drug Enforcement Administration Certificate of Registration of Controlled Substances Privileges in February 2022 “as an indication of your good faith in desiring to remedy any incorrect or unlawful practices on your part,” according to a letter to Dr. Sutton from the State Medical Board of Ohio. In that September 2022 letter, the Board notified Dr. Sutton of its intention to possibly suspend or revoke his license.
Dr. Sutton did not request a hearing, and the Board permanently revoked his medical license on January 16.
The court will sentence Dr. Sutton on May 23, according to a report by WFMJ.
A version of this article originally appeared on Medscape.com.
Hemophilia A gene therapy under FDA review
Hemophilia A (a deficiency of clotting Factor X) is the most common form of the disease, accounting for about 85% of patients.
The other type is hemophilia B (deficiency of clotting Factor VIII), and a gene therapy for this form of the disease has recently been launched – etranacogene dezaparvovec (Hemgenix), at the enormous price tag of $3.5 million.
Both products are comprised of a one-off intravenous IV infusion that delivers a functional gene via an adeno-associated virus that instructs the body to make the missing clotting factor. The hope is that this one-off infusion will act as a ‘cure’ and that the individual will be freed from life-long prophylaxis and/or treatment.
The new clinical data on valoctocogene roxaparvovec, published online in the New England Journal of Medicine, show that the beneficial effects from the gene are largely durable at 2 years, but they are anticipated to fade with time.
Two years after the one-time infusion, there remained “a significant reduction in the annualized bleeding rates” among 132 men who, at baseline, had severe hemophilia A requiring ongoing factor VIII prophylaxis, said the investigators, led by hematologist Johnny Mahlangu, MBBCh, MMed, of the University of the Witwatersrand, Johannesburg, South Africa.
However, the team predicted that median factor VIII activity would decrease below 10% of normal by year 3 or 5 depending on measurement technique, which would still translate to mild disease with an annualized bleeding rate of less than 1 episode per year.
“Although valoctocogene roxaparvovec may not eliminate bleeding, it potentially provides more consistent protection than factor VIII prophylaxis with less treatment burden,” the team said.
New questions
Data from the study “will directly inform therapeutic decision-making” in Europe, where valoctocogene roxaparvovec is already conditionally approved, and the United States, where it is awaiting approval by the FDA, says Lindsey George, MD, a hematologist and gene therapy specialist at Children’s Hospital of Philadelphia, in an accompanying editorial.
The study speaks to an ongoing concern about the durability of gene therapy for hemophilia but also raises new questions, she said.
For instance, while some patients had normal Factor VIII production and activity at 2 years, activity had dropped substantially in others, including in six men who resumed prophylaxis. “The cause of the decrease in factor VIII expression is an unanswered question,” and despite an anticipated U.S. price tag of around $2.5 million per treatment, “it is not possible [at the moment] to predict where an individual patient may fall within this range,” she writes.
Also, some subjects had elevations in liver aminotransferase levels that lasted for several months, including 2 years after infusion in 29% of subjects. Elevations in liver aminotransferase levels were treated with immune suppression for a median of 33 weeks.
“This is a unique finding with an undefined cause and long-term safety implications,” Dr. George said.
Getting to the bottom of such issues will be necessary for hemophilia gene therapy to fulfill its promise as “a one-time, lifelong, disease-ameliorating” fix for the condition, she asserted.
Study details
The new report followed up on the initial trial in 134 men who were treated with a single infusion of 6 × 1013 vector genomes per kilogram of body weight.
Among the 132 subjects available for 2-year evaluation, median factor VIII activity was in the range of mild hemophilia (6%-49% of normal) with an 84.5% reduction in bleeding events from baseline.
More than 80% of participants had no bleeding events requiring treatment, and there was a 98% reduction from baseline in mean use of exogenous factor VIII.
Overall, at year 2, 4.5% of subjects had factor VIII activity consistent with severe hemophilia A; 9.1% had activity consistent with moderate disease; 59.8% had activity consistent with mild disease; and 26.5% had activity in the normal range above 40 IU/dL. The investigators estimated that the typical half-life of the transgene-derived factor VIII production system is 123 weeks.
Among the six men who resumed prophylaxis, most had fewer bleeding events than when they were on prophylaxis before the infusion, investigators noted.
All the subjects developed antibodies to the virus delivery vector, precluding retreatment.
The work was funded by valoctocogene roxaparvovec maker BioMarin Pharmaceuticals. Several investigators are employees. Others reported ties to BioMarin and other companies; Dr. Mahlangu, for instance, reported research grants from BioMarin, Roche, Novo Nordisk, Pfizer, and others. Dr. George reported a research grant from Asklepios Biopharmaceutical and having a patent licensed to the company. The full list of author disclosures can be found with the original article.
A version of this article first appeared on Medscape.com.
Hemophilia A (a deficiency of clotting Factor X) is the most common form of the disease, accounting for about 85% of patients.
The other type is hemophilia B (deficiency of clotting Factor VIII), and a gene therapy for this form of the disease has recently been launched – etranacogene dezaparvovec (Hemgenix), at the enormous price tag of $3.5 million.
Both products are comprised of a one-off intravenous IV infusion that delivers a functional gene via an adeno-associated virus that instructs the body to make the missing clotting factor. The hope is that this one-off infusion will act as a ‘cure’ and that the individual will be freed from life-long prophylaxis and/or treatment.
The new clinical data on valoctocogene roxaparvovec, published online in the New England Journal of Medicine, show that the beneficial effects from the gene are largely durable at 2 years, but they are anticipated to fade with time.
Two years after the one-time infusion, there remained “a significant reduction in the annualized bleeding rates” among 132 men who, at baseline, had severe hemophilia A requiring ongoing factor VIII prophylaxis, said the investigators, led by hematologist Johnny Mahlangu, MBBCh, MMed, of the University of the Witwatersrand, Johannesburg, South Africa.
However, the team predicted that median factor VIII activity would decrease below 10% of normal by year 3 or 5 depending on measurement technique, which would still translate to mild disease with an annualized bleeding rate of less than 1 episode per year.
“Although valoctocogene roxaparvovec may not eliminate bleeding, it potentially provides more consistent protection than factor VIII prophylaxis with less treatment burden,” the team said.
New questions
Data from the study “will directly inform therapeutic decision-making” in Europe, where valoctocogene roxaparvovec is already conditionally approved, and the United States, where it is awaiting approval by the FDA, says Lindsey George, MD, a hematologist and gene therapy specialist at Children’s Hospital of Philadelphia, in an accompanying editorial.
The study speaks to an ongoing concern about the durability of gene therapy for hemophilia but also raises new questions, she said.
For instance, while some patients had normal Factor VIII production and activity at 2 years, activity had dropped substantially in others, including in six men who resumed prophylaxis. “The cause of the decrease in factor VIII expression is an unanswered question,” and despite an anticipated U.S. price tag of around $2.5 million per treatment, “it is not possible [at the moment] to predict where an individual patient may fall within this range,” she writes.
Also, some subjects had elevations in liver aminotransferase levels that lasted for several months, including 2 years after infusion in 29% of subjects. Elevations in liver aminotransferase levels were treated with immune suppression for a median of 33 weeks.
“This is a unique finding with an undefined cause and long-term safety implications,” Dr. George said.
Getting to the bottom of such issues will be necessary for hemophilia gene therapy to fulfill its promise as “a one-time, lifelong, disease-ameliorating” fix for the condition, she asserted.
Study details
The new report followed up on the initial trial in 134 men who were treated with a single infusion of 6 × 1013 vector genomes per kilogram of body weight.
Among the 132 subjects available for 2-year evaluation, median factor VIII activity was in the range of mild hemophilia (6%-49% of normal) with an 84.5% reduction in bleeding events from baseline.
More than 80% of participants had no bleeding events requiring treatment, and there was a 98% reduction from baseline in mean use of exogenous factor VIII.
Overall, at year 2, 4.5% of subjects had factor VIII activity consistent with severe hemophilia A; 9.1% had activity consistent with moderate disease; 59.8% had activity consistent with mild disease; and 26.5% had activity in the normal range above 40 IU/dL. The investigators estimated that the typical half-life of the transgene-derived factor VIII production system is 123 weeks.
Among the six men who resumed prophylaxis, most had fewer bleeding events than when they were on prophylaxis before the infusion, investigators noted.
All the subjects developed antibodies to the virus delivery vector, precluding retreatment.
The work was funded by valoctocogene roxaparvovec maker BioMarin Pharmaceuticals. Several investigators are employees. Others reported ties to BioMarin and other companies; Dr. Mahlangu, for instance, reported research grants from BioMarin, Roche, Novo Nordisk, Pfizer, and others. Dr. George reported a research grant from Asklepios Biopharmaceutical and having a patent licensed to the company. The full list of author disclosures can be found with the original article.
A version of this article first appeared on Medscape.com.
Hemophilia A (a deficiency of clotting Factor X) is the most common form of the disease, accounting for about 85% of patients.
The other type is hemophilia B (deficiency of clotting Factor VIII), and a gene therapy for this form of the disease has recently been launched – etranacogene dezaparvovec (Hemgenix), at the enormous price tag of $3.5 million.
Both products are comprised of a one-off intravenous IV infusion that delivers a functional gene via an adeno-associated virus that instructs the body to make the missing clotting factor. The hope is that this one-off infusion will act as a ‘cure’ and that the individual will be freed from life-long prophylaxis and/or treatment.
The new clinical data on valoctocogene roxaparvovec, published online in the New England Journal of Medicine, show that the beneficial effects from the gene are largely durable at 2 years, but they are anticipated to fade with time.
Two years after the one-time infusion, there remained “a significant reduction in the annualized bleeding rates” among 132 men who, at baseline, had severe hemophilia A requiring ongoing factor VIII prophylaxis, said the investigators, led by hematologist Johnny Mahlangu, MBBCh, MMed, of the University of the Witwatersrand, Johannesburg, South Africa.
However, the team predicted that median factor VIII activity would decrease below 10% of normal by year 3 or 5 depending on measurement technique, which would still translate to mild disease with an annualized bleeding rate of less than 1 episode per year.
“Although valoctocogene roxaparvovec may not eliminate bleeding, it potentially provides more consistent protection than factor VIII prophylaxis with less treatment burden,” the team said.
New questions
Data from the study “will directly inform therapeutic decision-making” in Europe, where valoctocogene roxaparvovec is already conditionally approved, and the United States, where it is awaiting approval by the FDA, says Lindsey George, MD, a hematologist and gene therapy specialist at Children’s Hospital of Philadelphia, in an accompanying editorial.
The study speaks to an ongoing concern about the durability of gene therapy for hemophilia but also raises new questions, she said.
For instance, while some patients had normal Factor VIII production and activity at 2 years, activity had dropped substantially in others, including in six men who resumed prophylaxis. “The cause of the decrease in factor VIII expression is an unanswered question,” and despite an anticipated U.S. price tag of around $2.5 million per treatment, “it is not possible [at the moment] to predict where an individual patient may fall within this range,” she writes.
Also, some subjects had elevations in liver aminotransferase levels that lasted for several months, including 2 years after infusion in 29% of subjects. Elevations in liver aminotransferase levels were treated with immune suppression for a median of 33 weeks.
“This is a unique finding with an undefined cause and long-term safety implications,” Dr. George said.
Getting to the bottom of such issues will be necessary for hemophilia gene therapy to fulfill its promise as “a one-time, lifelong, disease-ameliorating” fix for the condition, she asserted.
Study details
The new report followed up on the initial trial in 134 men who were treated with a single infusion of 6 × 1013 vector genomes per kilogram of body weight.
Among the 132 subjects available for 2-year evaluation, median factor VIII activity was in the range of mild hemophilia (6%-49% of normal) with an 84.5% reduction in bleeding events from baseline.
More than 80% of participants had no bleeding events requiring treatment, and there was a 98% reduction from baseline in mean use of exogenous factor VIII.
Overall, at year 2, 4.5% of subjects had factor VIII activity consistent with severe hemophilia A; 9.1% had activity consistent with moderate disease; 59.8% had activity consistent with mild disease; and 26.5% had activity in the normal range above 40 IU/dL. The investigators estimated that the typical half-life of the transgene-derived factor VIII production system is 123 weeks.
Among the six men who resumed prophylaxis, most had fewer bleeding events than when they were on prophylaxis before the infusion, investigators noted.
All the subjects developed antibodies to the virus delivery vector, precluding retreatment.
The work was funded by valoctocogene roxaparvovec maker BioMarin Pharmaceuticals. Several investigators are employees. Others reported ties to BioMarin and other companies; Dr. Mahlangu, for instance, reported research grants from BioMarin, Roche, Novo Nordisk, Pfizer, and others. Dr. George reported a research grant from Asklepios Biopharmaceutical and having a patent licensed to the company. The full list of author disclosures can be found with the original article.
A version of this article first appeared on Medscape.com.
FROM THE NEW ENGLAND JOURNAL OF MEDICINE
Efanesoctocog alfa treatment: ‘Victory’ over hemophilia A
One injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population, and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week.
Efanesoctocog alfa is currently under priority review by the U.S. Food and Drug Administration, and the target action date for the approval decision was set for Tuesday, February 28.
“Currently, [patients] often need to make trade-offs between bleed protection and dosing frequency,” study researcher Angela Weyand, MD, of Michigan Medicine, said in a press release. The current phase 3 XTEND-1 results assessing efanesoctocog alfa demonstrate that “we have the opportunity to provide near normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A.”
According to the researchers, efanesoctocog alfa is also the first investigational treatment for hemophilia A to surpass the von Willebrand factor half-life ceiling, which imposes a half-life limitation on current factor VIII therapies.
The study, which was funded by drugmakers Sanofi and Sobi, was published online in the New England Journal of Medicine.
Hemophilia A is a rare genetic disorder caused by a lack of blood clotting factor VIII. Normalizing factor VIII levels can help protect patients from spontaneous and traumatic bleeding but maintaining factor VIII levels in the normal (50-150 IU/dL) or close-to-normal range (> 40 to < 50 IU/dL) with currently available factor VIII therapies requires frequent administration, the study authors explained.
In the phase 3, open-label trial, the authors evaluated the efficacy, safety, and pharmacokinetics of efanesoctocog alfa for routine prophylaxis, treatment of bleeding episodes, and perioperative management in previously treated patients with severe hemophilia A.
Patients were 12 years of age or older with endogenous factor VIII activity of less than 1 IU/dL or a genotype known to produce severe hemophilia A. Patients were required to have had at least 150 previous exposure days to recombinant or plasma-derived factor VIII concentrates or cryoprecipitate.
Overall, 133 patients received once-weekly prophylaxis doses of 50 IU per kg of intravenous efanesoctocog alfa for 52 weeks (group A), and 26 patients received on-demand efanesoctocog alfa treatment for 26 weeks, followed by once-weekly prophylaxis with the medication for 26 weeks at the same 50 IU per kg dosage (group B). The primary endpoint was the mean annualized bleeding rate in group A.
Among those in group A, the annualized bleeding rate was 0 (interquartile range, 0-1.04) and the estimated mean annualized bleeding rate was 0.71. Overall, 65% of these patients (86 of 133) had no bleeding episodes and 93% had 0-2 bleeding episodes.
As for spontaneous bleeding, no episodes were reported in 80% of patients in group A (107 of 133) and 85% of patients (22 of 26) during the prophylaxis period in group B.
A total of 362 bleeding events occurred during the study, with most (268 of 362, or 74%) occurring in group B during the on-demand treatment period.
Among those in group A, switching from the prestudy standard of care to efanesoctocog alfa prophylaxis reduced the mean annualized bleeding rate from 2.96 to 0.69, a decrease of 77%. In group B, the mean annualized bleeding rate also decreased when patients switched from on-demand efanesoctocog alfa to prophylaxis (21.42 vs. 0.69).
Scores of physical health, joint health, and pain intensity were significantly improved within the 52 weeks. In patients with target joints at baseline, 100% of the target joints were resolved after at least 12 months of continuous prophylaxis.
In other words, not only did the treatment stop bleeding, but efanesoctocog alfa also improved the overall quality of life for patients, said lead author Annette von Drygalski MD, PharmD, of the University of California, San Diego.
“[Efanesoctocog] alfa’s half-life and clotting factor activity levels truly translated into a number of other patient outcomes,” Dr. von Drygalski told this news organization. “All these reductions in parameters, pain improvement, improvement in joint health, reduction in pain, and of course reduction of infusions really resulted in improved quality of life for most patients, and so that’s remarkable.”
In addition, no patients developed inhibitors to factor VIII and there were no reports of serious allergic reactions, anaphylaxis, or vascular thrombotic events.
Of the 159 patients who received at least one dose of efanesoctocog alfa, 123 (77%) had at least one adverse event that developed or worsened during the treatment period. The most common adverse events were headache, arthralgia, fall, and back pain.
In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.
“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” writes Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University, New Orleans.
The study was supported by Sanofi and Sobi. Both Dr. von Drygalski and Dr. Leissinger disclosed serving as consultants for Sanofi, among other disclosures. Other authors provided a range of disclosures, including serving as consultants for Sanofi and Sobi.
A version of this article first appeared on Medscape.com.
One injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population, and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week.
Efanesoctocog alfa is currently under priority review by the U.S. Food and Drug Administration, and the target action date for the approval decision was set for Tuesday, February 28.
“Currently, [patients] often need to make trade-offs between bleed protection and dosing frequency,” study researcher Angela Weyand, MD, of Michigan Medicine, said in a press release. The current phase 3 XTEND-1 results assessing efanesoctocog alfa demonstrate that “we have the opportunity to provide near normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A.”
According to the researchers, efanesoctocog alfa is also the first investigational treatment for hemophilia A to surpass the von Willebrand factor half-life ceiling, which imposes a half-life limitation on current factor VIII therapies.
The study, which was funded by drugmakers Sanofi and Sobi, was published online in the New England Journal of Medicine.
Hemophilia A is a rare genetic disorder caused by a lack of blood clotting factor VIII. Normalizing factor VIII levels can help protect patients from spontaneous and traumatic bleeding but maintaining factor VIII levels in the normal (50-150 IU/dL) or close-to-normal range (> 40 to < 50 IU/dL) with currently available factor VIII therapies requires frequent administration, the study authors explained.
In the phase 3, open-label trial, the authors evaluated the efficacy, safety, and pharmacokinetics of efanesoctocog alfa for routine prophylaxis, treatment of bleeding episodes, and perioperative management in previously treated patients with severe hemophilia A.
Patients were 12 years of age or older with endogenous factor VIII activity of less than 1 IU/dL or a genotype known to produce severe hemophilia A. Patients were required to have had at least 150 previous exposure days to recombinant or plasma-derived factor VIII concentrates or cryoprecipitate.
Overall, 133 patients received once-weekly prophylaxis doses of 50 IU per kg of intravenous efanesoctocog alfa for 52 weeks (group A), and 26 patients received on-demand efanesoctocog alfa treatment for 26 weeks, followed by once-weekly prophylaxis with the medication for 26 weeks at the same 50 IU per kg dosage (group B). The primary endpoint was the mean annualized bleeding rate in group A.
Among those in group A, the annualized bleeding rate was 0 (interquartile range, 0-1.04) and the estimated mean annualized bleeding rate was 0.71. Overall, 65% of these patients (86 of 133) had no bleeding episodes and 93% had 0-2 bleeding episodes.
As for spontaneous bleeding, no episodes were reported in 80% of patients in group A (107 of 133) and 85% of patients (22 of 26) during the prophylaxis period in group B.
A total of 362 bleeding events occurred during the study, with most (268 of 362, or 74%) occurring in group B during the on-demand treatment period.
Among those in group A, switching from the prestudy standard of care to efanesoctocog alfa prophylaxis reduced the mean annualized bleeding rate from 2.96 to 0.69, a decrease of 77%. In group B, the mean annualized bleeding rate also decreased when patients switched from on-demand efanesoctocog alfa to prophylaxis (21.42 vs. 0.69).
Scores of physical health, joint health, and pain intensity were significantly improved within the 52 weeks. In patients with target joints at baseline, 100% of the target joints were resolved after at least 12 months of continuous prophylaxis.
In other words, not only did the treatment stop bleeding, but efanesoctocog alfa also improved the overall quality of life for patients, said lead author Annette von Drygalski MD, PharmD, of the University of California, San Diego.
“[Efanesoctocog] alfa’s half-life and clotting factor activity levels truly translated into a number of other patient outcomes,” Dr. von Drygalski told this news organization. “All these reductions in parameters, pain improvement, improvement in joint health, reduction in pain, and of course reduction of infusions really resulted in improved quality of life for most patients, and so that’s remarkable.”
In addition, no patients developed inhibitors to factor VIII and there were no reports of serious allergic reactions, anaphylaxis, or vascular thrombotic events.
Of the 159 patients who received at least one dose of efanesoctocog alfa, 123 (77%) had at least one adverse event that developed or worsened during the treatment period. The most common adverse events were headache, arthralgia, fall, and back pain.
In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.
“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” writes Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University, New Orleans.
The study was supported by Sanofi and Sobi. Both Dr. von Drygalski and Dr. Leissinger disclosed serving as consultants for Sanofi, among other disclosures. Other authors provided a range of disclosures, including serving as consultants for Sanofi and Sobi.
A version of this article first appeared on Medscape.com.
One injection of efanesoctocog alfa, a factor VIII therapy, resolved almost all bleeding episodes (97%) in the overall patient population, and weekly prophylaxis provided mean factor VIII activity in the normal or near-normal range (> 40 IU/dL) for most of the week.
Efanesoctocog alfa is currently under priority review by the U.S. Food and Drug Administration, and the target action date for the approval decision was set for Tuesday, February 28.
“Currently, [patients] often need to make trade-offs between bleed protection and dosing frequency,” study researcher Angela Weyand, MD, of Michigan Medicine, said in a press release. The current phase 3 XTEND-1 results assessing efanesoctocog alfa demonstrate that “we have the opportunity to provide near normal factor activity levels for an extended period of time (the majority of a week) with a single dose, which is a first for hemophilia A.”
According to the researchers, efanesoctocog alfa is also the first investigational treatment for hemophilia A to surpass the von Willebrand factor half-life ceiling, which imposes a half-life limitation on current factor VIII therapies.
The study, which was funded by drugmakers Sanofi and Sobi, was published online in the New England Journal of Medicine.
Hemophilia A is a rare genetic disorder caused by a lack of blood clotting factor VIII. Normalizing factor VIII levels can help protect patients from spontaneous and traumatic bleeding but maintaining factor VIII levels in the normal (50-150 IU/dL) or close-to-normal range (> 40 to < 50 IU/dL) with currently available factor VIII therapies requires frequent administration, the study authors explained.
In the phase 3, open-label trial, the authors evaluated the efficacy, safety, and pharmacokinetics of efanesoctocog alfa for routine prophylaxis, treatment of bleeding episodes, and perioperative management in previously treated patients with severe hemophilia A.
Patients were 12 years of age or older with endogenous factor VIII activity of less than 1 IU/dL or a genotype known to produce severe hemophilia A. Patients were required to have had at least 150 previous exposure days to recombinant or plasma-derived factor VIII concentrates or cryoprecipitate.
Overall, 133 patients received once-weekly prophylaxis doses of 50 IU per kg of intravenous efanesoctocog alfa for 52 weeks (group A), and 26 patients received on-demand efanesoctocog alfa treatment for 26 weeks, followed by once-weekly prophylaxis with the medication for 26 weeks at the same 50 IU per kg dosage (group B). The primary endpoint was the mean annualized bleeding rate in group A.
Among those in group A, the annualized bleeding rate was 0 (interquartile range, 0-1.04) and the estimated mean annualized bleeding rate was 0.71. Overall, 65% of these patients (86 of 133) had no bleeding episodes and 93% had 0-2 bleeding episodes.
As for spontaneous bleeding, no episodes were reported in 80% of patients in group A (107 of 133) and 85% of patients (22 of 26) during the prophylaxis period in group B.
A total of 362 bleeding events occurred during the study, with most (268 of 362, or 74%) occurring in group B during the on-demand treatment period.
Among those in group A, switching from the prestudy standard of care to efanesoctocog alfa prophylaxis reduced the mean annualized bleeding rate from 2.96 to 0.69, a decrease of 77%. In group B, the mean annualized bleeding rate also decreased when patients switched from on-demand efanesoctocog alfa to prophylaxis (21.42 vs. 0.69).
Scores of physical health, joint health, and pain intensity were significantly improved within the 52 weeks. In patients with target joints at baseline, 100% of the target joints were resolved after at least 12 months of continuous prophylaxis.
In other words, not only did the treatment stop bleeding, but efanesoctocog alfa also improved the overall quality of life for patients, said lead author Annette von Drygalski MD, PharmD, of the University of California, San Diego.
“[Efanesoctocog] alfa’s half-life and clotting factor activity levels truly translated into a number of other patient outcomes,” Dr. von Drygalski told this news organization. “All these reductions in parameters, pain improvement, improvement in joint health, reduction in pain, and of course reduction of infusions really resulted in improved quality of life for most patients, and so that’s remarkable.”
In addition, no patients developed inhibitors to factor VIII and there were no reports of serious allergic reactions, anaphylaxis, or vascular thrombotic events.
Of the 159 patients who received at least one dose of efanesoctocog alfa, 123 (77%) had at least one adverse event that developed or worsened during the treatment period. The most common adverse events were headache, arthralgia, fall, and back pain.
In an accompanying editorial, Cindy Leissinger, MD, called efanesoctocog alfa a “victory” for patients with hemophilia A.
“In a crowded field of transformative therapies for hemophilia, efanesoctocog alfa stands out as a winner – a major therapeutic advance that achieves highly protective factor VIII levels with a once-weekly infusion,” writes Dr. Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University, New Orleans.
The study was supported by Sanofi and Sobi. Both Dr. von Drygalski and Dr. Leissinger disclosed serving as consultants for Sanofi, among other disclosures. Other authors provided a range of disclosures, including serving as consultants for Sanofi and Sobi.
A version of this article first appeared on Medscape.com.
FROM THE NEW ENGLAND JOURNAL OF MEDICINE
Europe approves first gene therapy for hemophilia B
The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.
The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.
People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.
The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.
“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.
A version of this article first appeared on Medscape.com.
The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.
The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.
People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.
The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.
“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.
A version of this article first appeared on Medscape.com.
The approval means that the product will now be available in all the countries of the European Union as well as the European Economic Area.
The gene therapy was approved in the United States in November 2022. It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
The treatment comprises a one-time infusion of a functional gene that acts as a blueprint for coagulation factor IX, a protein important for blood clotting, stated the manufacturer, CSL.
People living with hemophilia B currently require lifelong treatment of intravenous infusions of factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and well-being, the company explained in its press release.
The approval was based on findings from the pivotal HOPE-B trial, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; first results from this trial were reported at the 2020 annual meeting of the American Society of Hematology.
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity (with a mean factor IX activity of 36.9%), which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post treatment compared with the lead-in period, the company noted.
“Data from the HOPE-B study demonstrate the potential of Hemgenix to remove the need for routine prophylaxis by providing durable factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B,” said one of the trialists, Wolfgang Miesbach, MD, PHD, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European approval “marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints, and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,” Dr. Miesbach said in the company press release.
A version of this article first appeared on Medscape.com.