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Who’s an Anesthesiologist? Turf War Sparks Trademark Dispute
The turf war between two types of anesthesia providers is escalating:
At issue: Who can be called an anesthesiologist?
In its complaint, filed in June 2024 with the US Trademark Trial and Appeal Board, the 54,000-member physician society seeks to deny the nurse group the registration of its trademark. If ASA wins, it could sue AANA in federal court.
AANA denied the physicians’ allegations in its recent response to the complaint.
The dispute between the two associations comes at a time when physicians are facing challenges from providers such as nurse practitioners and physician assistants who seek new titles and more autonomy in medical decision-making.
A Controversial Name Change
In 2021, the 61,000-member AANA changed its name from the American Association of Nurse Anesthetists, saying the change “clarifies” the role of its members.
The ASA declared it was “gravely concerned” by the name change, which “confuses patients and creates discord in the care setting, ultimately risking patient safety.
“ ’Anesthesiologist’ has always been used to differentiate physicians trained in the science and study of anesthesiology from nonphysicians, including nurse anesthetists,” the physicians’ group said in a news release.
Chicago Intellectual Property Attorney Laura M. Schaefer, who represents AANA, told this news organization that certified registered nurse anesthetists (CRNAs) — “also known as nurse anesthesiologists or nurse anesthetists — have a 150-year track record of administering safe, effective anesthesia to patients in need of care. Not only are CRNAs highly trained and capable, they also use the exact same techniques to provide anesthesia as other anesthesiology professionals.”
Ms. Schaefer declined to comment further, and ASA declined to comment at all, citing pending litigation.
The scope of practice of nurse anesthetists has long been disputed. In mid-September, California health officials clarified what nurse anesthetists can do on the job after complaints about lack of oversight, The Modesto Bee reported.
According to nursing education site NurseJournal.org, CRNAs and anesthesiologists “perform many of the same duties,” although CRNAs are in more demand. Also, the site says some states require CRNAs to be supervised by anesthesiologists.
“It is possible that scope of practice debates are increasing in prominence due to the increase in demand for healthcare services, coupled with workforce shortages in certain areas,” Alice Chen, PhD, MBA, vice dean for research at the USC Sol Price School of Public Policy in Los Angeles, told this news organization. “For example, during COVID, the federal government temporarily expanded scope of practice to help address healthcare needs.”
She added her group’s research has shown that despite the large stakes perceived by both sides of the debate, changes in practice behavior were actually quite small in states that allowed CRNAs to practice without supervision.
“In fact, we found only modest reduction in anesthesiologist billing for supervision, and we did not find an increase in the supply of anesthesia care,” she noted.
Trademark law specialists told this news organization that they couldn’t predict which way the board will rule. However, they noted potential weaknesses of the ASA’s case.
Rebecca Tushnet, JD, a professor at Harvard Law School, Cambridge, Massachusetts, explained that a trademark “can’t misrepresent those goods or services in a way that deceives consumers.” However, if insurers, doctors, and hospitals are considered the “consumers” — and not patients — “then confusion is probably less likely because they will have relevant expertise to distinguish among groups.”
Christine Farley, JD, LLM, JSD, professor at American University Washington College of Law, said attacking the AANA’s trademark as deceptive may be one of the ASA’s strongest arguments. The suggestion, she said, is that “nurse anesthesiologist” is an oxymoron, like “jumbo shrimp.”
On the other hand, she said it’s not clear that people will miss the word “nurse” in AANA’s name and say, “ ’Well, obviously these people are doctors.’ So that that’s an uphill battle.”
What happens now? The Trademark Trial and Appeal Board will decide whether AANA’s trademark application should be granted or denied, said Kayla Jimenez, JD, a San Diego trademark attorney and adjunct law professor at the University of San Diego. The entire process can take 2-3 years, she said.
The board “cannot award attorneys’ fees or force a party to stop using a trademark,” she said. “You would have to go file a lawsuit in federal court if that is your endgame.” Also, she said, the board’s ultimate decision can be appealed in federal court.
Eric Goldman, JD, MBA, associate dean for research and professor at Santa Clara University School of Law, Santa Clara, California, doesn’t expect the trademark case will spell the end of this dispute.
“ASA is signaling that it will challenge AANA’s use of the term in multiple battlegrounds,” he said. “I see this as a move by ASA to contest AANA in every potentially relevant venue, even if neither side can score a knockout blow in the Trademark Trial and Appeal Board.”
Dr. Chen, Ms. Farley, Ms. Jimenez, and Mr. Goldman had no disclosures.
A version of this article appeared on Medscape.com.
The turf war between two types of anesthesia providers is escalating:
At issue: Who can be called an anesthesiologist?
In its complaint, filed in June 2024 with the US Trademark Trial and Appeal Board, the 54,000-member physician society seeks to deny the nurse group the registration of its trademark. If ASA wins, it could sue AANA in federal court.
AANA denied the physicians’ allegations in its recent response to the complaint.
The dispute between the two associations comes at a time when physicians are facing challenges from providers such as nurse practitioners and physician assistants who seek new titles and more autonomy in medical decision-making.
A Controversial Name Change
In 2021, the 61,000-member AANA changed its name from the American Association of Nurse Anesthetists, saying the change “clarifies” the role of its members.
The ASA declared it was “gravely concerned” by the name change, which “confuses patients and creates discord in the care setting, ultimately risking patient safety.
“ ’Anesthesiologist’ has always been used to differentiate physicians trained in the science and study of anesthesiology from nonphysicians, including nurse anesthetists,” the physicians’ group said in a news release.
Chicago Intellectual Property Attorney Laura M. Schaefer, who represents AANA, told this news organization that certified registered nurse anesthetists (CRNAs) — “also known as nurse anesthesiologists or nurse anesthetists — have a 150-year track record of administering safe, effective anesthesia to patients in need of care. Not only are CRNAs highly trained and capable, they also use the exact same techniques to provide anesthesia as other anesthesiology professionals.”
Ms. Schaefer declined to comment further, and ASA declined to comment at all, citing pending litigation.
The scope of practice of nurse anesthetists has long been disputed. In mid-September, California health officials clarified what nurse anesthetists can do on the job after complaints about lack of oversight, The Modesto Bee reported.
According to nursing education site NurseJournal.org, CRNAs and anesthesiologists “perform many of the same duties,” although CRNAs are in more demand. Also, the site says some states require CRNAs to be supervised by anesthesiologists.
“It is possible that scope of practice debates are increasing in prominence due to the increase in demand for healthcare services, coupled with workforce shortages in certain areas,” Alice Chen, PhD, MBA, vice dean for research at the USC Sol Price School of Public Policy in Los Angeles, told this news organization. “For example, during COVID, the federal government temporarily expanded scope of practice to help address healthcare needs.”
She added her group’s research has shown that despite the large stakes perceived by both sides of the debate, changes in practice behavior were actually quite small in states that allowed CRNAs to practice without supervision.
“In fact, we found only modest reduction in anesthesiologist billing for supervision, and we did not find an increase in the supply of anesthesia care,” she noted.
Trademark law specialists told this news organization that they couldn’t predict which way the board will rule. However, they noted potential weaknesses of the ASA’s case.
Rebecca Tushnet, JD, a professor at Harvard Law School, Cambridge, Massachusetts, explained that a trademark “can’t misrepresent those goods or services in a way that deceives consumers.” However, if insurers, doctors, and hospitals are considered the “consumers” — and not patients — “then confusion is probably less likely because they will have relevant expertise to distinguish among groups.”
Christine Farley, JD, LLM, JSD, professor at American University Washington College of Law, said attacking the AANA’s trademark as deceptive may be one of the ASA’s strongest arguments. The suggestion, she said, is that “nurse anesthesiologist” is an oxymoron, like “jumbo shrimp.”
On the other hand, she said it’s not clear that people will miss the word “nurse” in AANA’s name and say, “ ’Well, obviously these people are doctors.’ So that that’s an uphill battle.”
What happens now? The Trademark Trial and Appeal Board will decide whether AANA’s trademark application should be granted or denied, said Kayla Jimenez, JD, a San Diego trademark attorney and adjunct law professor at the University of San Diego. The entire process can take 2-3 years, she said.
The board “cannot award attorneys’ fees or force a party to stop using a trademark,” she said. “You would have to go file a lawsuit in federal court if that is your endgame.” Also, she said, the board’s ultimate decision can be appealed in federal court.
Eric Goldman, JD, MBA, associate dean for research and professor at Santa Clara University School of Law, Santa Clara, California, doesn’t expect the trademark case will spell the end of this dispute.
“ASA is signaling that it will challenge AANA’s use of the term in multiple battlegrounds,” he said. “I see this as a move by ASA to contest AANA in every potentially relevant venue, even if neither side can score a knockout blow in the Trademark Trial and Appeal Board.”
Dr. Chen, Ms. Farley, Ms. Jimenez, and Mr. Goldman had no disclosures.
A version of this article appeared on Medscape.com.
The turf war between two types of anesthesia providers is escalating:
At issue: Who can be called an anesthesiologist?
In its complaint, filed in June 2024 with the US Trademark Trial and Appeal Board, the 54,000-member physician society seeks to deny the nurse group the registration of its trademark. If ASA wins, it could sue AANA in federal court.
AANA denied the physicians’ allegations in its recent response to the complaint.
The dispute between the two associations comes at a time when physicians are facing challenges from providers such as nurse practitioners and physician assistants who seek new titles and more autonomy in medical decision-making.
A Controversial Name Change
In 2021, the 61,000-member AANA changed its name from the American Association of Nurse Anesthetists, saying the change “clarifies” the role of its members.
The ASA declared it was “gravely concerned” by the name change, which “confuses patients and creates discord in the care setting, ultimately risking patient safety.
“ ’Anesthesiologist’ has always been used to differentiate physicians trained in the science and study of anesthesiology from nonphysicians, including nurse anesthetists,” the physicians’ group said in a news release.
Chicago Intellectual Property Attorney Laura M. Schaefer, who represents AANA, told this news organization that certified registered nurse anesthetists (CRNAs) — “also known as nurse anesthesiologists or nurse anesthetists — have a 150-year track record of administering safe, effective anesthesia to patients in need of care. Not only are CRNAs highly trained and capable, they also use the exact same techniques to provide anesthesia as other anesthesiology professionals.”
Ms. Schaefer declined to comment further, and ASA declined to comment at all, citing pending litigation.
The scope of practice of nurse anesthetists has long been disputed. In mid-September, California health officials clarified what nurse anesthetists can do on the job after complaints about lack of oversight, The Modesto Bee reported.
According to nursing education site NurseJournal.org, CRNAs and anesthesiologists “perform many of the same duties,” although CRNAs are in more demand. Also, the site says some states require CRNAs to be supervised by anesthesiologists.
“It is possible that scope of practice debates are increasing in prominence due to the increase in demand for healthcare services, coupled with workforce shortages in certain areas,” Alice Chen, PhD, MBA, vice dean for research at the USC Sol Price School of Public Policy in Los Angeles, told this news organization. “For example, during COVID, the federal government temporarily expanded scope of practice to help address healthcare needs.”
She added her group’s research has shown that despite the large stakes perceived by both sides of the debate, changes in practice behavior were actually quite small in states that allowed CRNAs to practice without supervision.
“In fact, we found only modest reduction in anesthesiologist billing for supervision, and we did not find an increase in the supply of anesthesia care,” she noted.
Trademark law specialists told this news organization that they couldn’t predict which way the board will rule. However, they noted potential weaknesses of the ASA’s case.
Rebecca Tushnet, JD, a professor at Harvard Law School, Cambridge, Massachusetts, explained that a trademark “can’t misrepresent those goods or services in a way that deceives consumers.” However, if insurers, doctors, and hospitals are considered the “consumers” — and not patients — “then confusion is probably less likely because they will have relevant expertise to distinguish among groups.”
Christine Farley, JD, LLM, JSD, professor at American University Washington College of Law, said attacking the AANA’s trademark as deceptive may be one of the ASA’s strongest arguments. The suggestion, she said, is that “nurse anesthesiologist” is an oxymoron, like “jumbo shrimp.”
On the other hand, she said it’s not clear that people will miss the word “nurse” in AANA’s name and say, “ ’Well, obviously these people are doctors.’ So that that’s an uphill battle.”
What happens now? The Trademark Trial and Appeal Board will decide whether AANA’s trademark application should be granted or denied, said Kayla Jimenez, JD, a San Diego trademark attorney and adjunct law professor at the University of San Diego. The entire process can take 2-3 years, she said.
The board “cannot award attorneys’ fees or force a party to stop using a trademark,” she said. “You would have to go file a lawsuit in federal court if that is your endgame.” Also, she said, the board’s ultimate decision can be appealed in federal court.
Eric Goldman, JD, MBA, associate dean for research and professor at Santa Clara University School of Law, Santa Clara, California, doesn’t expect the trademark case will spell the end of this dispute.
“ASA is signaling that it will challenge AANA’s use of the term in multiple battlegrounds,” he said. “I see this as a move by ASA to contest AANA in every potentially relevant venue, even if neither side can score a knockout blow in the Trademark Trial and Appeal Board.”
Dr. Chen, Ms. Farley, Ms. Jimenez, and Mr. Goldman had no disclosures.
A version of this article appeared on Medscape.com.
Trial Looks at Early Use of Mycophenolate to Reduce Flares, Nephritis
Early use of mycophenolate mofetil (MMF), a drug used to dampen the immune system in organ transplant recipients, may reduce the risk for severe flares in patients with newly diagnosed systemic lupus erythematosus (SLE), according to results from a randomized, open-label, observer-blinded clinical trial.
In interviews, two SLE specialists who were not involved with the study said the research is preliminary but promising. However, another specialist criticized the paper’s reliance on unusual doses of prednisone and MMF, saying it “puts people on a treatment regimen that nobody ever uses.”
The Lupus Foundation of America estimates that about 16,000 people in the United States are diagnosed with lupus each year. “Our current treatment paradigm is to go pretty slowly and start treatment for new-onset, mild SLE with glucocorticoids, if necessary, and hydroxychloroquine,” said Karen H. Costenbader, MD, MPH, of Harvard Medical School and Harvard School of Public Health, Boston, Massachusetts.
Stronger immunosuppressive agents may be added as patients progress, she said.
Off-label use of MMF, which is approved by the Food and Drug Administration only for patients with certain organ transplants, may be appropriate in some cases, she said. “There is a big push to start immunosuppressives earlier, but we currently would reserve mycophenolate for those with severe manifestations — lupus nephritis; vasculitis; or lung, brain, or heart inflammation.”
In the trial, adult patients who received oral prednisone (starting at 0.5 mg/kg per day) and hydroxychloroquine sulfate (5 mg/kg per day) plus MMF (500 mg twice daily) for 96 weeks were less likely to develop severe flares than those who took the regimen without MMF (relative risk [RR], 0.39; 95% CI, 0.17-0.87; P = .01). Severe flares occurred in 10.8% of the MMF group (7 of 65 patients) and in 27.7% of the control group (18 of 65), Yijun You, MD, of Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China, and colleagues reported in JAMA Network Open.
Patients in the MMF group also had 89% lower risk for lupus nephritis than those in the control group (RR, 0.11; 95% CI, 0.01-0.85; P = .008), with kidney involvement occurring in 1.5% (1 of 65) vs 13.8% (9 of 65).
During 2018-2021, researchers recruited 130 patients in China aged 18-65 years with newly diagnosed SLE, a high titer of anti–double-stranded DNA (dsDNA) antibodies, and no major organ involvement (mean age, 34.5 years; 86.2% women). Patients’ initial 0.5–mg/kg per day prednisone dose was maintained for 4 weeks, then tapered by 5.0 mg every 2 weeks, and when the dose had been reduced to 20.0 mg/day, it was tapered by 5 mg every month and then gradually to 0.1-0.2 mg/kg per day. If patients had severe flares, they stopped taking MMF. (The study authors did not respond to requests for comment on the study.)
‘A Treatment Regimen That Nobody Ever Uses’
While Dr. Costenbader called the study “very interesting” and said “every person diagnosing or taking care of patients with lupus should be familiar” with it, she noted that the prednisone doses were high. “I am wondering why they used quite so much glucocorticoid for everyone. This may have masked some of the MMF effect and biased toward the null. They also used a low dose of MMF and did not ramp it up as we would normally to a full dose. That being said, it is remarkable that it was well-tolerated and resulted in better outcomes over the period of the trial.”
Daniel J. Wallace, MD, of Cedars-Sinai Medical Center, Los Angeles, California, and the University of California, Los Angeles, also highlighted the high doses of prednisone and low doses of MMF. “It’s a useless paper that puts people on a treatment regimen that nobody ever uses,” he said.
The rates of mild to moderate flares were similar between the control and intervention groups (38.5% vs 36.9%, respectively; RR, 0.96; P = .90). This finding is surprising, said Judith A. James, MD, PhD, executive vice president, chief medical officer, and head of the rheumatology clinic and Arthritis and Clinical Immunology Research Program at the Oklahoma Medical Research Foundation in Oklahoma City and also the Associate Vice Provost of Clinical & Translational Science, professor of medicine, and George Lynn Cross Research Professor at the University of Oklahoma Health Sciences Center in Oklahoma City. “It may be that mild flares have a different mechanism or are caused by noninflammatory endotypes that don’t respond to MMF.”
Dr. Costenbader noted that a risk-benefit analysis will need to be done to take the risks of MMF into account. “However, every time that a person flares or is not in lupus low-disease activity state, potentially permanent organ damage is done and the patient suffers,” she said. “Preventing lupus nephritis de novo was also seen — nine cases potentially prevented — and that is also really interesting. It would be amazing if we could completely avoid that life-threatening complication.”
MMF can cause miscarriage and boost the risk for birth defects, and the manufacturer says it can lower the effectiveness of birth control pills. It can also boost the risk for some cancers such as lymphoma and increase the risk for infections.
Surprisingly, the number of adverse events in the control and intervention groups were similar (35.4% vs 46.2%, respectively; RR, 1.30; 95% CI, 0.86-1.99; P = .20). They included infection (30.8% vs 33.8%, respectively; P = .70) and gastrointestinal tract events (16.9% for both; P > .99).
“There were overall pretty similar rates of side effects, but maybe this was because MMF dose was pretty low in the treated group, or the glucocorticoid dose was not so low in both groups,” Dr. Costenbader said. She also noted that “the risk of malignancy with MMF is longer term than this study. It may not show up for 5-10 or even more years, but we know it exists. Infections are also increased with MMF — some of which can be avoided with vaccines for COVID, pneumonia, influenza, shingles, etc. MMF also causes gastrointestinal intolerance, and people often are not able to take it because of nausea, vomiting, diarrhea, and elevated liver function tests.”
Dr. James said the infection rates “may be due to the higher doses of steroids patients in both groups are on for several months at the beginning of the study.”
A total of 12 patients in the MMF group discontinued the intervention for various reasons, and 6 were lost to follow-up. In the control group, 20 discontinued the intervention and two were lost to follow-up. However, all 130 patients in the trial were included in the primary and secondary outcome analyses.
Should clinicians consider prescribing MMF to patients with new-onset SLE? “We usually wait until later when there are indications of more severe disease, but here they started it from the time of diagnosis if the patient was anti-dsDNA positive. Given insurance restrictions in this country, we would be unlikely to be able to do that for many patients,” Dr. Costenbader said. “They likely also overtreated a lot of patients who didn’t need it. Due to our lack of more specific biomarkers and precision medicine for lupus, we do currently undertreat a lot of patients, as this study highlights, as well as overtreat others.”
How Much Might Cost Factor Into Treatment Decisions?
The study did not examine cost. Prednisone and hydroxychloroquine sulfate are inexpensive, but Dr. James said MMF can cost about $450 a month at the study dosage. However, “the average hospitalization without an ICU [intensive care unit] visit for an SLE patient is about $15,000-$20,000. If you can avoid one hospitalization, you can pay for nearly 4 years of MMF. More importantly, from a financial perspective, if you can convert a severe lupus patient to a mild/moderate lupus patient, then the annual costs of lupus decrease nearly by half, from about $52,000 per year to $25,000 per year.”
The study authors noted various limitations such as the small number of subjects, the need for a longer trial “to determine the advantages and disadvantages of early application of MMF,” and the fact that all subjects were Asian. The authors also called for confirmation via a double-blind, placebo-controlled study.
The study was funded by grants to the authors by the National Natural Science Foundation of China, Shanghai Rising-Star Program, Natural Science Foundation of Shanghai, Five-Year National Key R&D Program, and Ruijin–Zhongmei Huadong Lupus Funding. The authors had no disclosures. Dr. Costenbader disclosed consulting/research collaboration relationships with AstraZeneca, Amgen, Biogen, Bristol-Myers Squibb, GSK, Merck, Gilead, and Cabaletta. Dr. James and Dr. Wallace had no disclosures.
A version of this article first appeared on Medscape.com.
Early use of mycophenolate mofetil (MMF), a drug used to dampen the immune system in organ transplant recipients, may reduce the risk for severe flares in patients with newly diagnosed systemic lupus erythematosus (SLE), according to results from a randomized, open-label, observer-blinded clinical trial.
In interviews, two SLE specialists who were not involved with the study said the research is preliminary but promising. However, another specialist criticized the paper’s reliance on unusual doses of prednisone and MMF, saying it “puts people on a treatment regimen that nobody ever uses.”
The Lupus Foundation of America estimates that about 16,000 people in the United States are diagnosed with lupus each year. “Our current treatment paradigm is to go pretty slowly and start treatment for new-onset, mild SLE with glucocorticoids, if necessary, and hydroxychloroquine,” said Karen H. Costenbader, MD, MPH, of Harvard Medical School and Harvard School of Public Health, Boston, Massachusetts.
Stronger immunosuppressive agents may be added as patients progress, she said.
Off-label use of MMF, which is approved by the Food and Drug Administration only for patients with certain organ transplants, may be appropriate in some cases, she said. “There is a big push to start immunosuppressives earlier, but we currently would reserve mycophenolate for those with severe manifestations — lupus nephritis; vasculitis; or lung, brain, or heart inflammation.”
In the trial, adult patients who received oral prednisone (starting at 0.5 mg/kg per day) and hydroxychloroquine sulfate (5 mg/kg per day) plus MMF (500 mg twice daily) for 96 weeks were less likely to develop severe flares than those who took the regimen without MMF (relative risk [RR], 0.39; 95% CI, 0.17-0.87; P = .01). Severe flares occurred in 10.8% of the MMF group (7 of 65 patients) and in 27.7% of the control group (18 of 65), Yijun You, MD, of Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China, and colleagues reported in JAMA Network Open.
Patients in the MMF group also had 89% lower risk for lupus nephritis than those in the control group (RR, 0.11; 95% CI, 0.01-0.85; P = .008), with kidney involvement occurring in 1.5% (1 of 65) vs 13.8% (9 of 65).
During 2018-2021, researchers recruited 130 patients in China aged 18-65 years with newly diagnosed SLE, a high titer of anti–double-stranded DNA (dsDNA) antibodies, and no major organ involvement (mean age, 34.5 years; 86.2% women). Patients’ initial 0.5–mg/kg per day prednisone dose was maintained for 4 weeks, then tapered by 5.0 mg every 2 weeks, and when the dose had been reduced to 20.0 mg/day, it was tapered by 5 mg every month and then gradually to 0.1-0.2 mg/kg per day. If patients had severe flares, they stopped taking MMF. (The study authors did not respond to requests for comment on the study.)
‘A Treatment Regimen That Nobody Ever Uses’
While Dr. Costenbader called the study “very interesting” and said “every person diagnosing or taking care of patients with lupus should be familiar” with it, she noted that the prednisone doses were high. “I am wondering why they used quite so much glucocorticoid for everyone. This may have masked some of the MMF effect and biased toward the null. They also used a low dose of MMF and did not ramp it up as we would normally to a full dose. That being said, it is remarkable that it was well-tolerated and resulted in better outcomes over the period of the trial.”
Daniel J. Wallace, MD, of Cedars-Sinai Medical Center, Los Angeles, California, and the University of California, Los Angeles, also highlighted the high doses of prednisone and low doses of MMF. “It’s a useless paper that puts people on a treatment regimen that nobody ever uses,” he said.
The rates of mild to moderate flares were similar between the control and intervention groups (38.5% vs 36.9%, respectively; RR, 0.96; P = .90). This finding is surprising, said Judith A. James, MD, PhD, executive vice president, chief medical officer, and head of the rheumatology clinic and Arthritis and Clinical Immunology Research Program at the Oklahoma Medical Research Foundation in Oklahoma City and also the Associate Vice Provost of Clinical & Translational Science, professor of medicine, and George Lynn Cross Research Professor at the University of Oklahoma Health Sciences Center in Oklahoma City. “It may be that mild flares have a different mechanism or are caused by noninflammatory endotypes that don’t respond to MMF.”
Dr. Costenbader noted that a risk-benefit analysis will need to be done to take the risks of MMF into account. “However, every time that a person flares or is not in lupus low-disease activity state, potentially permanent organ damage is done and the patient suffers,” she said. “Preventing lupus nephritis de novo was also seen — nine cases potentially prevented — and that is also really interesting. It would be amazing if we could completely avoid that life-threatening complication.”
MMF can cause miscarriage and boost the risk for birth defects, and the manufacturer says it can lower the effectiveness of birth control pills. It can also boost the risk for some cancers such as lymphoma and increase the risk for infections.
Surprisingly, the number of adverse events in the control and intervention groups were similar (35.4% vs 46.2%, respectively; RR, 1.30; 95% CI, 0.86-1.99; P = .20). They included infection (30.8% vs 33.8%, respectively; P = .70) and gastrointestinal tract events (16.9% for both; P > .99).
“There were overall pretty similar rates of side effects, but maybe this was because MMF dose was pretty low in the treated group, or the glucocorticoid dose was not so low in both groups,” Dr. Costenbader said. She also noted that “the risk of malignancy with MMF is longer term than this study. It may not show up for 5-10 or even more years, but we know it exists. Infections are also increased with MMF — some of which can be avoided with vaccines for COVID, pneumonia, influenza, shingles, etc. MMF also causes gastrointestinal intolerance, and people often are not able to take it because of nausea, vomiting, diarrhea, and elevated liver function tests.”
Dr. James said the infection rates “may be due to the higher doses of steroids patients in both groups are on for several months at the beginning of the study.”
A total of 12 patients in the MMF group discontinued the intervention for various reasons, and 6 were lost to follow-up. In the control group, 20 discontinued the intervention and two were lost to follow-up. However, all 130 patients in the trial were included in the primary and secondary outcome analyses.
Should clinicians consider prescribing MMF to patients with new-onset SLE? “We usually wait until later when there are indications of more severe disease, but here they started it from the time of diagnosis if the patient was anti-dsDNA positive. Given insurance restrictions in this country, we would be unlikely to be able to do that for many patients,” Dr. Costenbader said. “They likely also overtreated a lot of patients who didn’t need it. Due to our lack of more specific biomarkers and precision medicine for lupus, we do currently undertreat a lot of patients, as this study highlights, as well as overtreat others.”
How Much Might Cost Factor Into Treatment Decisions?
The study did not examine cost. Prednisone and hydroxychloroquine sulfate are inexpensive, but Dr. James said MMF can cost about $450 a month at the study dosage. However, “the average hospitalization without an ICU [intensive care unit] visit for an SLE patient is about $15,000-$20,000. If you can avoid one hospitalization, you can pay for nearly 4 years of MMF. More importantly, from a financial perspective, if you can convert a severe lupus patient to a mild/moderate lupus patient, then the annual costs of lupus decrease nearly by half, from about $52,000 per year to $25,000 per year.”
The study authors noted various limitations such as the small number of subjects, the need for a longer trial “to determine the advantages and disadvantages of early application of MMF,” and the fact that all subjects were Asian. The authors also called for confirmation via a double-blind, placebo-controlled study.
The study was funded by grants to the authors by the National Natural Science Foundation of China, Shanghai Rising-Star Program, Natural Science Foundation of Shanghai, Five-Year National Key R&D Program, and Ruijin–Zhongmei Huadong Lupus Funding. The authors had no disclosures. Dr. Costenbader disclosed consulting/research collaboration relationships with AstraZeneca, Amgen, Biogen, Bristol-Myers Squibb, GSK, Merck, Gilead, and Cabaletta. Dr. James and Dr. Wallace had no disclosures.
A version of this article first appeared on Medscape.com.
Early use of mycophenolate mofetil (MMF), a drug used to dampen the immune system in organ transplant recipients, may reduce the risk for severe flares in patients with newly diagnosed systemic lupus erythematosus (SLE), according to results from a randomized, open-label, observer-blinded clinical trial.
In interviews, two SLE specialists who were not involved with the study said the research is preliminary but promising. However, another specialist criticized the paper’s reliance on unusual doses of prednisone and MMF, saying it “puts people on a treatment regimen that nobody ever uses.”
The Lupus Foundation of America estimates that about 16,000 people in the United States are diagnosed with lupus each year. “Our current treatment paradigm is to go pretty slowly and start treatment for new-onset, mild SLE with glucocorticoids, if necessary, and hydroxychloroquine,” said Karen H. Costenbader, MD, MPH, of Harvard Medical School and Harvard School of Public Health, Boston, Massachusetts.
Stronger immunosuppressive agents may be added as patients progress, she said.
Off-label use of MMF, which is approved by the Food and Drug Administration only for patients with certain organ transplants, may be appropriate in some cases, she said. “There is a big push to start immunosuppressives earlier, but we currently would reserve mycophenolate for those with severe manifestations — lupus nephritis; vasculitis; or lung, brain, or heart inflammation.”
In the trial, adult patients who received oral prednisone (starting at 0.5 mg/kg per day) and hydroxychloroquine sulfate (5 mg/kg per day) plus MMF (500 mg twice daily) for 96 weeks were less likely to develop severe flares than those who took the regimen without MMF (relative risk [RR], 0.39; 95% CI, 0.17-0.87; P = .01). Severe flares occurred in 10.8% of the MMF group (7 of 65 patients) and in 27.7% of the control group (18 of 65), Yijun You, MD, of Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China, and colleagues reported in JAMA Network Open.
Patients in the MMF group also had 89% lower risk for lupus nephritis than those in the control group (RR, 0.11; 95% CI, 0.01-0.85; P = .008), with kidney involvement occurring in 1.5% (1 of 65) vs 13.8% (9 of 65).
During 2018-2021, researchers recruited 130 patients in China aged 18-65 years with newly diagnosed SLE, a high titer of anti–double-stranded DNA (dsDNA) antibodies, and no major organ involvement (mean age, 34.5 years; 86.2% women). Patients’ initial 0.5–mg/kg per day prednisone dose was maintained for 4 weeks, then tapered by 5.0 mg every 2 weeks, and when the dose had been reduced to 20.0 mg/day, it was tapered by 5 mg every month and then gradually to 0.1-0.2 mg/kg per day. If patients had severe flares, they stopped taking MMF. (The study authors did not respond to requests for comment on the study.)
‘A Treatment Regimen That Nobody Ever Uses’
While Dr. Costenbader called the study “very interesting” and said “every person diagnosing or taking care of patients with lupus should be familiar” with it, she noted that the prednisone doses were high. “I am wondering why they used quite so much glucocorticoid for everyone. This may have masked some of the MMF effect and biased toward the null. They also used a low dose of MMF and did not ramp it up as we would normally to a full dose. That being said, it is remarkable that it was well-tolerated and resulted in better outcomes over the period of the trial.”
Daniel J. Wallace, MD, of Cedars-Sinai Medical Center, Los Angeles, California, and the University of California, Los Angeles, also highlighted the high doses of prednisone and low doses of MMF. “It’s a useless paper that puts people on a treatment regimen that nobody ever uses,” he said.
The rates of mild to moderate flares were similar between the control and intervention groups (38.5% vs 36.9%, respectively; RR, 0.96; P = .90). This finding is surprising, said Judith A. James, MD, PhD, executive vice president, chief medical officer, and head of the rheumatology clinic and Arthritis and Clinical Immunology Research Program at the Oklahoma Medical Research Foundation in Oklahoma City and also the Associate Vice Provost of Clinical & Translational Science, professor of medicine, and George Lynn Cross Research Professor at the University of Oklahoma Health Sciences Center in Oklahoma City. “It may be that mild flares have a different mechanism or are caused by noninflammatory endotypes that don’t respond to MMF.”
Dr. Costenbader noted that a risk-benefit analysis will need to be done to take the risks of MMF into account. “However, every time that a person flares or is not in lupus low-disease activity state, potentially permanent organ damage is done and the patient suffers,” she said. “Preventing lupus nephritis de novo was also seen — nine cases potentially prevented — and that is also really interesting. It would be amazing if we could completely avoid that life-threatening complication.”
MMF can cause miscarriage and boost the risk for birth defects, and the manufacturer says it can lower the effectiveness of birth control pills. It can also boost the risk for some cancers such as lymphoma and increase the risk for infections.
Surprisingly, the number of adverse events in the control and intervention groups were similar (35.4% vs 46.2%, respectively; RR, 1.30; 95% CI, 0.86-1.99; P = .20). They included infection (30.8% vs 33.8%, respectively; P = .70) and gastrointestinal tract events (16.9% for both; P > .99).
“There were overall pretty similar rates of side effects, but maybe this was because MMF dose was pretty low in the treated group, or the glucocorticoid dose was not so low in both groups,” Dr. Costenbader said. She also noted that “the risk of malignancy with MMF is longer term than this study. It may not show up for 5-10 or even more years, but we know it exists. Infections are also increased with MMF — some of which can be avoided with vaccines for COVID, pneumonia, influenza, shingles, etc. MMF also causes gastrointestinal intolerance, and people often are not able to take it because of nausea, vomiting, diarrhea, and elevated liver function tests.”
Dr. James said the infection rates “may be due to the higher doses of steroids patients in both groups are on for several months at the beginning of the study.”
A total of 12 patients in the MMF group discontinued the intervention for various reasons, and 6 were lost to follow-up. In the control group, 20 discontinued the intervention and two were lost to follow-up. However, all 130 patients in the trial were included in the primary and secondary outcome analyses.
Should clinicians consider prescribing MMF to patients with new-onset SLE? “We usually wait until later when there are indications of more severe disease, but here they started it from the time of diagnosis if the patient was anti-dsDNA positive. Given insurance restrictions in this country, we would be unlikely to be able to do that for many patients,” Dr. Costenbader said. “They likely also overtreated a lot of patients who didn’t need it. Due to our lack of more specific biomarkers and precision medicine for lupus, we do currently undertreat a lot of patients, as this study highlights, as well as overtreat others.”
How Much Might Cost Factor Into Treatment Decisions?
The study did not examine cost. Prednisone and hydroxychloroquine sulfate are inexpensive, but Dr. James said MMF can cost about $450 a month at the study dosage. However, “the average hospitalization without an ICU [intensive care unit] visit for an SLE patient is about $15,000-$20,000. If you can avoid one hospitalization, you can pay for nearly 4 years of MMF. More importantly, from a financial perspective, if you can convert a severe lupus patient to a mild/moderate lupus patient, then the annual costs of lupus decrease nearly by half, from about $52,000 per year to $25,000 per year.”
The study authors noted various limitations such as the small number of subjects, the need for a longer trial “to determine the advantages and disadvantages of early application of MMF,” and the fact that all subjects were Asian. The authors also called for confirmation via a double-blind, placebo-controlled study.
The study was funded by grants to the authors by the National Natural Science Foundation of China, Shanghai Rising-Star Program, Natural Science Foundation of Shanghai, Five-Year National Key R&D Program, and Ruijin–Zhongmei Huadong Lupus Funding. The authors had no disclosures. Dr. Costenbader disclosed consulting/research collaboration relationships with AstraZeneca, Amgen, Biogen, Bristol-Myers Squibb, GSK, Merck, Gilead, and Cabaletta. Dr. James and Dr. Wallace had no disclosures.
A version of this article first appeared on Medscape.com.
FROM JAMA NETWORK OPEN
Transgender Women and Prostate Cancer: It’s Complicated
The Veterans Health Administration (VHA) provides care for about 10,000 transgender women, and clinicians must understand their distinctive needs for prostate cancer screening, a urologist told cancer specialists during a presentation at the 2024 annual meeting of the Association of VA Hematology/Oncology in Atlanta.
Even if they’ve undergone gender reassignment surgery, “all transgender women still have a prostate, so therefore they remain at risk of prostate cancer and could still be considered for prostate cancer screening,” said Farnoosh Nik-Ahd, MD, a resident physician at the University of California San Francisco. However, “clinicians and patients may not be aware of prostate cancer risk, so that they may not think [of screening] transgender women.”
Nik-Ahd also noted another complication: The results of prostate screening tests may be misleading in this population.
Transgender women were born biologically male but now identify as female. These individuals may have undergone gender reassignment surgery to remove male genitalia, but the procedures do not remove the prostate. They also might be taking estrogen therapy. “Prostate cancer is a hormonally driven cancer, and the exact impact of gender-affirming hormones on prostate cancer risk and development is unknown,” Nik-Ahd said.
In a 2023 study in JAMA, Nik-Ahd and colleagues identified 155 cases of prostate cancer in transgender women within the VHA (about 14 cases per year) from 2000 to 2022. Of these patients, 116 had never used estrogen, while 17 had used it previously and 22 used it at diagnosis.
The median age of patients was 61 years, 88% identified as White, and the median prostate-specific antigen (PSA) was 6.8 ng/mL. “Given estimates of 10,000 transgender women in the US Department of Veterans Affairs, 33 cases per year would be expected. Instead, only about 14 per year were observed,” the researchers wrote. “Lower rates may stem from less PSA screening owing to barriers including lack of prostate cancer risk awareness or stigma, the suppressive effects of estrogen on prostate cancer development, or prostate cancers being missed in transgender women because of misinterpretation of ‘normal’ PSA levels among those receiving gender-affirming hormone therapies.”
In the presentation, Nik-Ahd said, “PSA density, which is a marker of prostate cancer aggressiveness, was highest in transgender women who were actively on estrogen.”
She noted, “the existing thyrotropin reference ranges, which is what we use to interpret PSA values, are all based on data from cisgender men.” The ranges would be expected to be far lower in transgender women who are taking estrogen, potentially throwing off screening tests, she said, and “ultimately missing clinically significant prostate cancer.”
In the larger picture, there are no specific guidelines about PSA screening in transgender women, she said.
A recent study published in JAMA by Nik-Ahd and colleagues examined PSA levels in 210 transgender women (mean age 60 years) treated within the VHA from 2000 to 2023. All were aged 40 to 80 years, had received estrogen for at least 6 months (mean duration 4.7 years), and didn’t have prostate cancer diagnoses.
“Median (IQR) PSA was 0.02 (0-0.2) ng/mL and the 95th percentile value was 0.6 ng/mL,” the report found. “PSAs were undetectable in 36% of patients (23% and 49% of PSAs in patients without and with orchiectomy, respectively).”
The researchers write that “the historic cut point of 4 ng/mL, often used as a threshold for further evaluation, is likely far too high a threshold for this population.”
Nik-Ahd noted, “clinicians should interpret PSA values in transgender women on estrogen with extreme caution. In this population, normal might actually not be normal, and a value that is considered normal might be very abnormal for somebody who is on estrogen. If you're unsure of whether a PSA value is appropriate for a transgender woman on estrogen, refer that patient to a urologist so they can undergo further evaluation.”
Farnoosh Nik-Ahd discloses consulting for Janssen.
The Veterans Health Administration (VHA) provides care for about 10,000 transgender women, and clinicians must understand their distinctive needs for prostate cancer screening, a urologist told cancer specialists during a presentation at the 2024 annual meeting of the Association of VA Hematology/Oncology in Atlanta.
Even if they’ve undergone gender reassignment surgery, “all transgender women still have a prostate, so therefore they remain at risk of prostate cancer and could still be considered for prostate cancer screening,” said Farnoosh Nik-Ahd, MD, a resident physician at the University of California San Francisco. However, “clinicians and patients may not be aware of prostate cancer risk, so that they may not think [of screening] transgender women.”
Nik-Ahd also noted another complication: The results of prostate screening tests may be misleading in this population.
Transgender women were born biologically male but now identify as female. These individuals may have undergone gender reassignment surgery to remove male genitalia, but the procedures do not remove the prostate. They also might be taking estrogen therapy. “Prostate cancer is a hormonally driven cancer, and the exact impact of gender-affirming hormones on prostate cancer risk and development is unknown,” Nik-Ahd said.
In a 2023 study in JAMA, Nik-Ahd and colleagues identified 155 cases of prostate cancer in transgender women within the VHA (about 14 cases per year) from 2000 to 2022. Of these patients, 116 had never used estrogen, while 17 had used it previously and 22 used it at diagnosis.
The median age of patients was 61 years, 88% identified as White, and the median prostate-specific antigen (PSA) was 6.8 ng/mL. “Given estimates of 10,000 transgender women in the US Department of Veterans Affairs, 33 cases per year would be expected. Instead, only about 14 per year were observed,” the researchers wrote. “Lower rates may stem from less PSA screening owing to barriers including lack of prostate cancer risk awareness or stigma, the suppressive effects of estrogen on prostate cancer development, or prostate cancers being missed in transgender women because of misinterpretation of ‘normal’ PSA levels among those receiving gender-affirming hormone therapies.”
In the presentation, Nik-Ahd said, “PSA density, which is a marker of prostate cancer aggressiveness, was highest in transgender women who were actively on estrogen.”
She noted, “the existing thyrotropin reference ranges, which is what we use to interpret PSA values, are all based on data from cisgender men.” The ranges would be expected to be far lower in transgender women who are taking estrogen, potentially throwing off screening tests, she said, and “ultimately missing clinically significant prostate cancer.”
In the larger picture, there are no specific guidelines about PSA screening in transgender women, she said.
A recent study published in JAMA by Nik-Ahd and colleagues examined PSA levels in 210 transgender women (mean age 60 years) treated within the VHA from 2000 to 2023. All were aged 40 to 80 years, had received estrogen for at least 6 months (mean duration 4.7 years), and didn’t have prostate cancer diagnoses.
“Median (IQR) PSA was 0.02 (0-0.2) ng/mL and the 95th percentile value was 0.6 ng/mL,” the report found. “PSAs were undetectable in 36% of patients (23% and 49% of PSAs in patients without and with orchiectomy, respectively).”
The researchers write that “the historic cut point of 4 ng/mL, often used as a threshold for further evaluation, is likely far too high a threshold for this population.”
Nik-Ahd noted, “clinicians should interpret PSA values in transgender women on estrogen with extreme caution. In this population, normal might actually not be normal, and a value that is considered normal might be very abnormal for somebody who is on estrogen. If you're unsure of whether a PSA value is appropriate for a transgender woman on estrogen, refer that patient to a urologist so they can undergo further evaluation.”
Farnoosh Nik-Ahd discloses consulting for Janssen.
The Veterans Health Administration (VHA) provides care for about 10,000 transgender women, and clinicians must understand their distinctive needs for prostate cancer screening, a urologist told cancer specialists during a presentation at the 2024 annual meeting of the Association of VA Hematology/Oncology in Atlanta.
Even if they’ve undergone gender reassignment surgery, “all transgender women still have a prostate, so therefore they remain at risk of prostate cancer and could still be considered for prostate cancer screening,” said Farnoosh Nik-Ahd, MD, a resident physician at the University of California San Francisco. However, “clinicians and patients may not be aware of prostate cancer risk, so that they may not think [of screening] transgender women.”
Nik-Ahd also noted another complication: The results of prostate screening tests may be misleading in this population.
Transgender women were born biologically male but now identify as female. These individuals may have undergone gender reassignment surgery to remove male genitalia, but the procedures do not remove the prostate. They also might be taking estrogen therapy. “Prostate cancer is a hormonally driven cancer, and the exact impact of gender-affirming hormones on prostate cancer risk and development is unknown,” Nik-Ahd said.
In a 2023 study in JAMA, Nik-Ahd and colleagues identified 155 cases of prostate cancer in transgender women within the VHA (about 14 cases per year) from 2000 to 2022. Of these patients, 116 had never used estrogen, while 17 had used it previously and 22 used it at diagnosis.
The median age of patients was 61 years, 88% identified as White, and the median prostate-specific antigen (PSA) was 6.8 ng/mL. “Given estimates of 10,000 transgender women in the US Department of Veterans Affairs, 33 cases per year would be expected. Instead, only about 14 per year were observed,” the researchers wrote. “Lower rates may stem from less PSA screening owing to barriers including lack of prostate cancer risk awareness or stigma, the suppressive effects of estrogen on prostate cancer development, or prostate cancers being missed in transgender women because of misinterpretation of ‘normal’ PSA levels among those receiving gender-affirming hormone therapies.”
In the presentation, Nik-Ahd said, “PSA density, which is a marker of prostate cancer aggressiveness, was highest in transgender women who were actively on estrogen.”
She noted, “the existing thyrotropin reference ranges, which is what we use to interpret PSA values, are all based on data from cisgender men.” The ranges would be expected to be far lower in transgender women who are taking estrogen, potentially throwing off screening tests, she said, and “ultimately missing clinically significant prostate cancer.”
In the larger picture, there are no specific guidelines about PSA screening in transgender women, she said.
A recent study published in JAMA by Nik-Ahd and colleagues examined PSA levels in 210 transgender women (mean age 60 years) treated within the VHA from 2000 to 2023. All were aged 40 to 80 years, had received estrogen for at least 6 months (mean duration 4.7 years), and didn’t have prostate cancer diagnoses.
“Median (IQR) PSA was 0.02 (0-0.2) ng/mL and the 95th percentile value was 0.6 ng/mL,” the report found. “PSAs were undetectable in 36% of patients (23% and 49% of PSAs in patients without and with orchiectomy, respectively).”
The researchers write that “the historic cut point of 4 ng/mL, often used as a threshold for further evaluation, is likely far too high a threshold for this population.”
Nik-Ahd noted, “clinicians should interpret PSA values in transgender women on estrogen with extreme caution. In this population, normal might actually not be normal, and a value that is considered normal might be very abnormal for somebody who is on estrogen. If you're unsure of whether a PSA value is appropriate for a transgender woman on estrogen, refer that patient to a urologist so they can undergo further evaluation.”
Farnoosh Nik-Ahd discloses consulting for Janssen.
No Matched Sibling Donor? Sickle Cell Experts Debate Next-Best Option
“If there is an indication for intervention, for a curative therapy, in the absence of a matched sibling donor, gene therapy is the first choice,” Jaap-Jan Boelens, MD, PhD, of Memorial Sloan Kettering Cancer Center, New York City, argued in a presentation at the annual meeting of the Society of Hematologic Oncology (SOHO) in Houston.
“In the registries, alternative transplant outcomes are pretty poor, although there is some encouraging data coming up. The time is not there yet when this is the [best] choice.”
But Adetola Kassim, MBBS, of Vanderbilt University Medical Center in Nashville, Tennessee, said patients with sickle cell disease (SCD) who don’t qualify for a matched sibling donor transplant can still have good transplant options. And the results can be impressive.
“Once you’re engrafted, and you don’t lose your graft, the effect in transplant is lifelong,” he said. When it comes to long-lasting effects, he added, “we’re not sure yet about gene therapy.”
As Dr. Kassim noted, SCD continues to take a huge toll.
“Median survival for patients with sickle cell anemia remains stuck in the fifth decade of life with no change in 25 years,” he said. Heart, lung, and kidney complications account for 50% of identifiable causes of death, followed by about 26% attributed to cardiovascular disease, he said. “The question here is about which therapy can impact the most debilitating complication in children, which is stroke, and improve survival in adults with progressive organ dysfunction.”
Dr. Boelens said there are “huge barriers” to stem cell transplant in SCD because only 15% of patients eligible for the treatment have a matched related donor, and only 10% have a matched related or unrelated donor.
“There’s also a lack of financial and psychosocial support in many of the families. There is also parental refusal because of the mortality risk, and there’s also physician refusal because hematologists aren’t always in the same hospitals as the transplant programs.”
Dr. Boelens highlighted a 2019 study of data from 2008-2017 that found outcomes in unmatched donor transplantations are “not great,” with higher risk for mortality and graft failure.
As an alternative, he said, two gene therapies, both gene “additions,” are now approved by the US Food and Drug Administration (FDA). They are exagamglogene autotemcel (exa-cel, Casgevy) and betibeglogene autotemcel (LentiGlobin, Zynteglo). There’s also a gene “correction” option in the works, but it’s not yet ready for prime time, he said.
In the two approved gene therapy treatments, stem cells are removed from the patient, modified/manufactured in an outside facility, and then engrafted.
The advantages of gene therapy include no need to find a donor or worry about graft resistance, and there’s no need for immunosuppression, he said. However, the process takes a long time, there’s limited long-term data, and there’s a risk for loss of fertility and other chemotherapy-related adverse effects.
For his part, Dr. Kassim noted how several groups are excluded from the strong outcomes in matched sibling donor stem-cell transplants: Children with strokes and no eligible donors, others without eligible donors, and adults with severe disease and organ dysfunction who are typically excluded.
“We need transplants with less toxicity and alternative donors,” he said. Another challenge: “How do we decrease graft failure without increasing transplant-related mortality?”
Researchers are exploring several strategies to adjust drug therapy during conditioning, Dr. Kassim said, and he led a promising phase II study that explored one approach. The results of that study were recently published in the journal Blood. Graft failures were very low in both adults and children, he said, and 2-year survival among 70 patients was 94.8%. The five deaths were related to infection.
The evidence about the various strategies shows that “virtually all SCD patients, except those with severe heart, lung, or kidney disease” can benefit from a curative transplant, Dr. Kassim said.
Dr. Boelens had no disclosures. Disclosures for Dr. Kassim were not provided, but he recently reported no disclosures in a report about transplants in SCD.
A version of this article appeared on Medscape.com.
“If there is an indication for intervention, for a curative therapy, in the absence of a matched sibling donor, gene therapy is the first choice,” Jaap-Jan Boelens, MD, PhD, of Memorial Sloan Kettering Cancer Center, New York City, argued in a presentation at the annual meeting of the Society of Hematologic Oncology (SOHO) in Houston.
“In the registries, alternative transplant outcomes are pretty poor, although there is some encouraging data coming up. The time is not there yet when this is the [best] choice.”
But Adetola Kassim, MBBS, of Vanderbilt University Medical Center in Nashville, Tennessee, said patients with sickle cell disease (SCD) who don’t qualify for a matched sibling donor transplant can still have good transplant options. And the results can be impressive.
“Once you’re engrafted, and you don’t lose your graft, the effect in transplant is lifelong,” he said. When it comes to long-lasting effects, he added, “we’re not sure yet about gene therapy.”
As Dr. Kassim noted, SCD continues to take a huge toll.
“Median survival for patients with sickle cell anemia remains stuck in the fifth decade of life with no change in 25 years,” he said. Heart, lung, and kidney complications account for 50% of identifiable causes of death, followed by about 26% attributed to cardiovascular disease, he said. “The question here is about which therapy can impact the most debilitating complication in children, which is stroke, and improve survival in adults with progressive organ dysfunction.”
Dr. Boelens said there are “huge barriers” to stem cell transplant in SCD because only 15% of patients eligible for the treatment have a matched related donor, and only 10% have a matched related or unrelated donor.
“There’s also a lack of financial and psychosocial support in many of the families. There is also parental refusal because of the mortality risk, and there’s also physician refusal because hematologists aren’t always in the same hospitals as the transplant programs.”
Dr. Boelens highlighted a 2019 study of data from 2008-2017 that found outcomes in unmatched donor transplantations are “not great,” with higher risk for mortality and graft failure.
As an alternative, he said, two gene therapies, both gene “additions,” are now approved by the US Food and Drug Administration (FDA). They are exagamglogene autotemcel (exa-cel, Casgevy) and betibeglogene autotemcel (LentiGlobin, Zynteglo). There’s also a gene “correction” option in the works, but it’s not yet ready for prime time, he said.
In the two approved gene therapy treatments, stem cells are removed from the patient, modified/manufactured in an outside facility, and then engrafted.
The advantages of gene therapy include no need to find a donor or worry about graft resistance, and there’s no need for immunosuppression, he said. However, the process takes a long time, there’s limited long-term data, and there’s a risk for loss of fertility and other chemotherapy-related adverse effects.
For his part, Dr. Kassim noted how several groups are excluded from the strong outcomes in matched sibling donor stem-cell transplants: Children with strokes and no eligible donors, others without eligible donors, and adults with severe disease and organ dysfunction who are typically excluded.
“We need transplants with less toxicity and alternative donors,” he said. Another challenge: “How do we decrease graft failure without increasing transplant-related mortality?”
Researchers are exploring several strategies to adjust drug therapy during conditioning, Dr. Kassim said, and he led a promising phase II study that explored one approach. The results of that study were recently published in the journal Blood. Graft failures were very low in both adults and children, he said, and 2-year survival among 70 patients was 94.8%. The five deaths were related to infection.
The evidence about the various strategies shows that “virtually all SCD patients, except those with severe heart, lung, or kidney disease” can benefit from a curative transplant, Dr. Kassim said.
Dr. Boelens had no disclosures. Disclosures for Dr. Kassim were not provided, but he recently reported no disclosures in a report about transplants in SCD.
A version of this article appeared on Medscape.com.
“If there is an indication for intervention, for a curative therapy, in the absence of a matched sibling donor, gene therapy is the first choice,” Jaap-Jan Boelens, MD, PhD, of Memorial Sloan Kettering Cancer Center, New York City, argued in a presentation at the annual meeting of the Society of Hematologic Oncology (SOHO) in Houston.
“In the registries, alternative transplant outcomes are pretty poor, although there is some encouraging data coming up. The time is not there yet when this is the [best] choice.”
But Adetola Kassim, MBBS, of Vanderbilt University Medical Center in Nashville, Tennessee, said patients with sickle cell disease (SCD) who don’t qualify for a matched sibling donor transplant can still have good transplant options. And the results can be impressive.
“Once you’re engrafted, and you don’t lose your graft, the effect in transplant is lifelong,” he said. When it comes to long-lasting effects, he added, “we’re not sure yet about gene therapy.”
As Dr. Kassim noted, SCD continues to take a huge toll.
“Median survival for patients with sickle cell anemia remains stuck in the fifth decade of life with no change in 25 years,” he said. Heart, lung, and kidney complications account for 50% of identifiable causes of death, followed by about 26% attributed to cardiovascular disease, he said. “The question here is about which therapy can impact the most debilitating complication in children, which is stroke, and improve survival in adults with progressive organ dysfunction.”
Dr. Boelens said there are “huge barriers” to stem cell transplant in SCD because only 15% of patients eligible for the treatment have a matched related donor, and only 10% have a matched related or unrelated donor.
“There’s also a lack of financial and psychosocial support in many of the families. There is also parental refusal because of the mortality risk, and there’s also physician refusal because hematologists aren’t always in the same hospitals as the transplant programs.”
Dr. Boelens highlighted a 2019 study of data from 2008-2017 that found outcomes in unmatched donor transplantations are “not great,” with higher risk for mortality and graft failure.
As an alternative, he said, two gene therapies, both gene “additions,” are now approved by the US Food and Drug Administration (FDA). They are exagamglogene autotemcel (exa-cel, Casgevy) and betibeglogene autotemcel (LentiGlobin, Zynteglo). There’s also a gene “correction” option in the works, but it’s not yet ready for prime time, he said.
In the two approved gene therapy treatments, stem cells are removed from the patient, modified/manufactured in an outside facility, and then engrafted.
The advantages of gene therapy include no need to find a donor or worry about graft resistance, and there’s no need for immunosuppression, he said. However, the process takes a long time, there’s limited long-term data, and there’s a risk for loss of fertility and other chemotherapy-related adverse effects.
For his part, Dr. Kassim noted how several groups are excluded from the strong outcomes in matched sibling donor stem-cell transplants: Children with strokes and no eligible donors, others without eligible donors, and adults with severe disease and organ dysfunction who are typically excluded.
“We need transplants with less toxicity and alternative donors,” he said. Another challenge: “How do we decrease graft failure without increasing transplant-related mortality?”
Researchers are exploring several strategies to adjust drug therapy during conditioning, Dr. Kassim said, and he led a promising phase II study that explored one approach. The results of that study were recently published in the journal Blood. Graft failures were very low in both adults and children, he said, and 2-year survival among 70 patients was 94.8%. The five deaths were related to infection.
The evidence about the various strategies shows that “virtually all SCD patients, except those with severe heart, lung, or kidney disease” can benefit from a curative transplant, Dr. Kassim said.
Dr. Boelens had no disclosures. Disclosures for Dr. Kassim were not provided, but he recently reported no disclosures in a report about transplants in SCD.
A version of this article appeared on Medscape.com.
FROM SOHO 2024
Treatment Options in MCL: What Are the Best Practices?
In the frontline setting, findings suggest that regimens should differ significantly on the basis of whether patients are older or younger, whereas more data are needed to understand whether treatment can overcome poor prognoses in patients with TP53 mutations, lymphoma specialist Nina Wagner-Johnston, MD, of Johns Hopkins University School of Medicine, Baltimore, said in a presentation at the annual meeting of the Society of Hematologic Oncology (SOHO) 2024 in Houston, Texas.
On the relapsed/refractory front, patients need better options after treatment with Bruton tyrosine kinase inhibitors or chimeric antigen receptor (CAR) T-cell therapy, Krish Patel, MD, a lymphoma specialist with Swedish Cancer Institute in Seattle, said in an adjoining presentation. Fortunately, he said, some treatments are showing early promise.
Here’s a closer look at the presentations by Dr. Wagner-Johnston and Dr. Patel.
Frontline MCL: Age Helps Determine Best Approach
“For older and less fit patients, the standard approach has typically been bendamustine (Bendeka, Treanda) and rituximab (Rituxan), and the median progression-free survival is about 4 years, with overall survival not reached at a median 5 years of follow-up,” Dr. Wagner-Johnston said.
Low doses of the chemotherapy drug cytarabine have been added to the bendamustine-rituximab regimen, with encouraging results, she said. “Certainly there’s more toxicity associated even with lower doses, but those data look fairly promising.”
For younger and fit patients, “the standard of care approach has been to administer intensive chemoimmunotherapy that contains high-dose cytarabine, and then that’s typically followed with an autologous stem cell transplant,” she said. A 2016 study reported median progression-free survival of 8.5 years and median overall survival of 12.7 years.
Now, second-generation Bruton tyrosine kinase inhibitors “look very promising” in the frontline setting, Dr. Wagner-Johnston said.
The road has been rocky, however. The SHINE trial of more than 500 patients aged over 65 found that adding ibrutinib to bendamustine-rituximab improved progression-free survival. “However, progression-free survival did not [connect] to an overall survival benefit, and that’s likely due to the toxicity seen with ibrutinib,” she said.
“It’s not surprising to many of you that ibrutinib has been removed from the FDA label for mantle cell lymphoma,” she said. However, “second-generation [Bruton tyrosine kinase inhibitors] are known to be associated with less toxicity and potentially increased potency.”
What about Bruton tyrosine kinase inhibitors in younger and fitter patients? The TRIANGLE trial demonstrated their benefit, Dr. Wagner-Johnston said, linking ibrutinib to improvement in progression-free survival.
However, “it’s really too early to evaluate the statistical significance for overall survival.” And while the study looks at therapy without stem cell transplant, she believes it’s too early to know whether that’s a good option.
Dr. Wagner-Johnston tackled another topic: Can Bruton tyrosine kinase inhibitors overcome the poor prognosis seen with MCL with TP53 mutation? For now, the limitations of research makes it “hard to know,” she said, although early results of the BOVen trial are promising.
Relapsed/Refractory MCL: Better Options Are Still Needed
In his presentation, Dr. Patel spoke about therapy in patients with MCL and relapsed/refractory disease. “We know that outcomes for patients who progress on covalent [Bruton tyrosine kinase inhibitors] is really dismal,” he said. “This has been shown by multiple groups now across the globe.”
Noncovalent Bruton tyrosine kinase inhibitors are now an option, he noted. “We do understand that they work for some patients, and it can be quite useful, but even noncovalent [Bruton tyrosine kinase inhibitors] themselves are susceptible to resistance mutations. We’ve seen that in the [chronic lymphocytic leukemia] world.”
Dr. Patel asked the audience, “Why not just give everybody CAR T-cells, post-[Bruton tyrosine kinase inhibitors]? You get a CAR T-cell! You get a CAR T-cell! Everybody gets one.”
However, he noted, “Unfortunately, mantle cell lymphoma patients experience the worst high-grade toxicity when receiving CD19[-targeted] CAR T-cells.”
Are there better options? At the moment, “really, really early data” suggest benefits from molecular glues and degraders, novel inhibitors, antibody-drug conjugates, novel CAR T-cells, and bispecific antibodies, Dr. Patel said.
“All of these tools are in clinical trials, and hopefully some of them will help,” he said.
Disclosures were not provided. Dr. Wagner-Johnston recently disclosed advisory committee/board of directors’ relationships with ADC Therapeutics, Regeneron, Calibr, and Verastem. Dr. Patel recently disclosed ties with a long list of pharmaceutical companies, including AbbVie, AstraZeneca, BeiGene, Bristol Myers Squibb, Genentech, Janssen, Merck, and others.
A version of this article first appeared on Medscape.com.
In the frontline setting, findings suggest that regimens should differ significantly on the basis of whether patients are older or younger, whereas more data are needed to understand whether treatment can overcome poor prognoses in patients with TP53 mutations, lymphoma specialist Nina Wagner-Johnston, MD, of Johns Hopkins University School of Medicine, Baltimore, said in a presentation at the annual meeting of the Society of Hematologic Oncology (SOHO) 2024 in Houston, Texas.
On the relapsed/refractory front, patients need better options after treatment with Bruton tyrosine kinase inhibitors or chimeric antigen receptor (CAR) T-cell therapy, Krish Patel, MD, a lymphoma specialist with Swedish Cancer Institute in Seattle, said in an adjoining presentation. Fortunately, he said, some treatments are showing early promise.
Here’s a closer look at the presentations by Dr. Wagner-Johnston and Dr. Patel.
Frontline MCL: Age Helps Determine Best Approach
“For older and less fit patients, the standard approach has typically been bendamustine (Bendeka, Treanda) and rituximab (Rituxan), and the median progression-free survival is about 4 years, with overall survival not reached at a median 5 years of follow-up,” Dr. Wagner-Johnston said.
Low doses of the chemotherapy drug cytarabine have been added to the bendamustine-rituximab regimen, with encouraging results, she said. “Certainly there’s more toxicity associated even with lower doses, but those data look fairly promising.”
For younger and fit patients, “the standard of care approach has been to administer intensive chemoimmunotherapy that contains high-dose cytarabine, and then that’s typically followed with an autologous stem cell transplant,” she said. A 2016 study reported median progression-free survival of 8.5 years and median overall survival of 12.7 years.
Now, second-generation Bruton tyrosine kinase inhibitors “look very promising” in the frontline setting, Dr. Wagner-Johnston said.
The road has been rocky, however. The SHINE trial of more than 500 patients aged over 65 found that adding ibrutinib to bendamustine-rituximab improved progression-free survival. “However, progression-free survival did not [connect] to an overall survival benefit, and that’s likely due to the toxicity seen with ibrutinib,” she said.
“It’s not surprising to many of you that ibrutinib has been removed from the FDA label for mantle cell lymphoma,” she said. However, “second-generation [Bruton tyrosine kinase inhibitors] are known to be associated with less toxicity and potentially increased potency.”
What about Bruton tyrosine kinase inhibitors in younger and fitter patients? The TRIANGLE trial demonstrated their benefit, Dr. Wagner-Johnston said, linking ibrutinib to improvement in progression-free survival.
However, “it’s really too early to evaluate the statistical significance for overall survival.” And while the study looks at therapy without stem cell transplant, she believes it’s too early to know whether that’s a good option.
Dr. Wagner-Johnston tackled another topic: Can Bruton tyrosine kinase inhibitors overcome the poor prognosis seen with MCL with TP53 mutation? For now, the limitations of research makes it “hard to know,” she said, although early results of the BOVen trial are promising.
Relapsed/Refractory MCL: Better Options Are Still Needed
In his presentation, Dr. Patel spoke about therapy in patients with MCL and relapsed/refractory disease. “We know that outcomes for patients who progress on covalent [Bruton tyrosine kinase inhibitors] is really dismal,” he said. “This has been shown by multiple groups now across the globe.”
Noncovalent Bruton tyrosine kinase inhibitors are now an option, he noted. “We do understand that they work for some patients, and it can be quite useful, but even noncovalent [Bruton tyrosine kinase inhibitors] themselves are susceptible to resistance mutations. We’ve seen that in the [chronic lymphocytic leukemia] world.”
Dr. Patel asked the audience, “Why not just give everybody CAR T-cells, post-[Bruton tyrosine kinase inhibitors]? You get a CAR T-cell! You get a CAR T-cell! Everybody gets one.”
However, he noted, “Unfortunately, mantle cell lymphoma patients experience the worst high-grade toxicity when receiving CD19[-targeted] CAR T-cells.”
Are there better options? At the moment, “really, really early data” suggest benefits from molecular glues and degraders, novel inhibitors, antibody-drug conjugates, novel CAR T-cells, and bispecific antibodies, Dr. Patel said.
“All of these tools are in clinical trials, and hopefully some of them will help,” he said.
Disclosures were not provided. Dr. Wagner-Johnston recently disclosed advisory committee/board of directors’ relationships with ADC Therapeutics, Regeneron, Calibr, and Verastem. Dr. Patel recently disclosed ties with a long list of pharmaceutical companies, including AbbVie, AstraZeneca, BeiGene, Bristol Myers Squibb, Genentech, Janssen, Merck, and others.
A version of this article first appeared on Medscape.com.
In the frontline setting, findings suggest that regimens should differ significantly on the basis of whether patients are older or younger, whereas more data are needed to understand whether treatment can overcome poor prognoses in patients with TP53 mutations, lymphoma specialist Nina Wagner-Johnston, MD, of Johns Hopkins University School of Medicine, Baltimore, said in a presentation at the annual meeting of the Society of Hematologic Oncology (SOHO) 2024 in Houston, Texas.
On the relapsed/refractory front, patients need better options after treatment with Bruton tyrosine kinase inhibitors or chimeric antigen receptor (CAR) T-cell therapy, Krish Patel, MD, a lymphoma specialist with Swedish Cancer Institute in Seattle, said in an adjoining presentation. Fortunately, he said, some treatments are showing early promise.
Here’s a closer look at the presentations by Dr. Wagner-Johnston and Dr. Patel.
Frontline MCL: Age Helps Determine Best Approach
“For older and less fit patients, the standard approach has typically been bendamustine (Bendeka, Treanda) and rituximab (Rituxan), and the median progression-free survival is about 4 years, with overall survival not reached at a median 5 years of follow-up,” Dr. Wagner-Johnston said.
Low doses of the chemotherapy drug cytarabine have been added to the bendamustine-rituximab regimen, with encouraging results, she said. “Certainly there’s more toxicity associated even with lower doses, but those data look fairly promising.”
For younger and fit patients, “the standard of care approach has been to administer intensive chemoimmunotherapy that contains high-dose cytarabine, and then that’s typically followed with an autologous stem cell transplant,” she said. A 2016 study reported median progression-free survival of 8.5 years and median overall survival of 12.7 years.
Now, second-generation Bruton tyrosine kinase inhibitors “look very promising” in the frontline setting, Dr. Wagner-Johnston said.
The road has been rocky, however. The SHINE trial of more than 500 patients aged over 65 found that adding ibrutinib to bendamustine-rituximab improved progression-free survival. “However, progression-free survival did not [connect] to an overall survival benefit, and that’s likely due to the toxicity seen with ibrutinib,” she said.
“It’s not surprising to many of you that ibrutinib has been removed from the FDA label for mantle cell lymphoma,” she said. However, “second-generation [Bruton tyrosine kinase inhibitors] are known to be associated with less toxicity and potentially increased potency.”
What about Bruton tyrosine kinase inhibitors in younger and fitter patients? The TRIANGLE trial demonstrated their benefit, Dr. Wagner-Johnston said, linking ibrutinib to improvement in progression-free survival.
However, “it’s really too early to evaluate the statistical significance for overall survival.” And while the study looks at therapy without stem cell transplant, she believes it’s too early to know whether that’s a good option.
Dr. Wagner-Johnston tackled another topic: Can Bruton tyrosine kinase inhibitors overcome the poor prognosis seen with MCL with TP53 mutation? For now, the limitations of research makes it “hard to know,” she said, although early results of the BOVen trial are promising.
Relapsed/Refractory MCL: Better Options Are Still Needed
In his presentation, Dr. Patel spoke about therapy in patients with MCL and relapsed/refractory disease. “We know that outcomes for patients who progress on covalent [Bruton tyrosine kinase inhibitors] is really dismal,” he said. “This has been shown by multiple groups now across the globe.”
Noncovalent Bruton tyrosine kinase inhibitors are now an option, he noted. “We do understand that they work for some patients, and it can be quite useful, but even noncovalent [Bruton tyrosine kinase inhibitors] themselves are susceptible to resistance mutations. We’ve seen that in the [chronic lymphocytic leukemia] world.”
Dr. Patel asked the audience, “Why not just give everybody CAR T-cells, post-[Bruton tyrosine kinase inhibitors]? You get a CAR T-cell! You get a CAR T-cell! Everybody gets one.”
However, he noted, “Unfortunately, mantle cell lymphoma patients experience the worst high-grade toxicity when receiving CD19[-targeted] CAR T-cells.”
Are there better options? At the moment, “really, really early data” suggest benefits from molecular glues and degraders, novel inhibitors, antibody-drug conjugates, novel CAR T-cells, and bispecific antibodies, Dr. Patel said.
“All of these tools are in clinical trials, and hopefully some of them will help,” he said.
Disclosures were not provided. Dr. Wagner-Johnston recently disclosed advisory committee/board of directors’ relationships with ADC Therapeutics, Regeneron, Calibr, and Verastem. Dr. Patel recently disclosed ties with a long list of pharmaceutical companies, including AbbVie, AstraZeneca, BeiGene, Bristol Myers Squibb, Genentech, Janssen, Merck, and others.
A version of this article first appeared on Medscape.com.
FROM SOHO 2024
Debate: Should Patients With CLL Take Breaks From Targeted Therapies?
At the annual meeting of the Society of Hematologic Oncology, two hematologist-oncologists — Inhye Ahn, MD, of Dana-Farber Cancer Institute in Boston, Massachusetts, and Kerry A. Rogers, MD, of Ohio State University in Columbus — faced off in a debate. Ahn said the drugs can indeed be discontinued, while Rogers argued against stopping the medications.
“When I talk to my own patient about standard of care options in CLL, I use the analogy of a marathon and a sprint,” Dr. Ahn said. A marathon refers to continuous treatment with Bruton’s kinase inhibitors given daily for years, while the sprint refers to the combination of venetoclax with an anti-CD20 monoclonal antibody given over 12 cycles for the frontline regimen and 2 years for refractory CLL.
“I tell them these are both considered very efficacious regimens and well tolerated, one is by IV [the venetoclax regimen] and the other isn’t [Bruton’s kinase inhibitors], and they have different toxicity profile. I ask them what would you do? The most common question that I get from my patient is, ‘why would anyone do a marathon?’ ”
It’s not solely the length of treatment that’s important, Dr. Ahn said, as toxicities from the long-term use of Bruton’s kinase inhibitors build up over time and can lead to hypertension, arrhythmia, and sudden cardiac death.
In addition, she said, infections can occur, as well as hampered vaccine response, an important risk in the era of the COVID-19 pandemic. The cost of the drugs is high and adds up over time, and continuous use can boost resistance.
Is there a way to turn the marathon of Bruton’s kinase inhibitor use into a sprint without hurting patients? The answer is yes, through temporary discontinuation, Dr. Ahn said, although she cautioned that early cessation could lead to disease flare. “We dipped into our own database of 84 CLL patients treated with ibrutinib, and our conclusion was that temporary dose interruption or dose reduction did not impact progression-free survival”
Moving forward, she said, “more research is needed to define the optimal regimen that would lead to treatment cessation, the optimal patient population, who would benefit most from the cessation strategy, treatment duration, and how we define success.” For her part, Dr. Rogers argued that the continuous use of Bruton’s kinase inhibitors is “really the most effective treatment we have in CLL.”
It’s clear that “responses deepen with continued treatment,” Dr. Rogers said, noting that remission times grow over years of treatment. She highlighted a 2022 study of patients with CLL who took ibrutinib that found complete remission or complete remission with incomplete hematologic recovery was 7% at 12 months and 34% at 7 years. When patients quit taking the drugs, “you don’t get to maximize your patient’s response to this treatment.”
Dr. Rogers also noted that the RESONATE-2 trial found that ibrutinib is linked to the longest median progression-free survival of any CLL treatment at 8.9 years. “That really struck me a very effective initial therapy.”
Indeed, “when you’re offering someone initial therapy with a Bruton’s kinase inhibitor as a continuous treatment strategy, you can tell people that they can expect a normal lifespan with this approach. That’s extremely important when you’re talking to patients about whether they might want to alter their leukemia treatment.”
Finally, she noted that discontinuation of ibrutinib was linked to shorter survival in early research. “This was the first suggestion that discontinuation is not good.”
Dr. Rogers said that discontinuing the drugs is sometimes necessary because of adverse events, but patients can “certainly switch to a more tolerable Bruton’s kinase inhibitor. With the options available today, that should be a strategy that’s considered.”
Audience members at the debate were invited to respond to a live online survey about whether Bruton’s kinase inhibitors can be discontinued. Among 49 respondents, most (52.3%) said no, 42.8% said yes, and the rest were undecided/other.
Disclosures for the speakers were not provided. Dr. Ahn disclosed consulting for BeiGene and AstraZeneca. Dr. Rogers disclosed receiving research funding from Genentech, AbbVie, Janssen, and Novartis; consulting for AstraZeneca, BeiGene, Janssen, Pharmacyclics, AbbVie, Genentech, and LOXO@Lilly; and receiving travel funding from AstraZeneca.
A version of this article appeared on Medscape.com.
At the annual meeting of the Society of Hematologic Oncology, two hematologist-oncologists — Inhye Ahn, MD, of Dana-Farber Cancer Institute in Boston, Massachusetts, and Kerry A. Rogers, MD, of Ohio State University in Columbus — faced off in a debate. Ahn said the drugs can indeed be discontinued, while Rogers argued against stopping the medications.
“When I talk to my own patient about standard of care options in CLL, I use the analogy of a marathon and a sprint,” Dr. Ahn said. A marathon refers to continuous treatment with Bruton’s kinase inhibitors given daily for years, while the sprint refers to the combination of venetoclax with an anti-CD20 monoclonal antibody given over 12 cycles for the frontline regimen and 2 years for refractory CLL.
“I tell them these are both considered very efficacious regimens and well tolerated, one is by IV [the venetoclax regimen] and the other isn’t [Bruton’s kinase inhibitors], and they have different toxicity profile. I ask them what would you do? The most common question that I get from my patient is, ‘why would anyone do a marathon?’ ”
It’s not solely the length of treatment that’s important, Dr. Ahn said, as toxicities from the long-term use of Bruton’s kinase inhibitors build up over time and can lead to hypertension, arrhythmia, and sudden cardiac death.
In addition, she said, infections can occur, as well as hampered vaccine response, an important risk in the era of the COVID-19 pandemic. The cost of the drugs is high and adds up over time, and continuous use can boost resistance.
Is there a way to turn the marathon of Bruton’s kinase inhibitor use into a sprint without hurting patients? The answer is yes, through temporary discontinuation, Dr. Ahn said, although she cautioned that early cessation could lead to disease flare. “We dipped into our own database of 84 CLL patients treated with ibrutinib, and our conclusion was that temporary dose interruption or dose reduction did not impact progression-free survival”
Moving forward, she said, “more research is needed to define the optimal regimen that would lead to treatment cessation, the optimal patient population, who would benefit most from the cessation strategy, treatment duration, and how we define success.” For her part, Dr. Rogers argued that the continuous use of Bruton’s kinase inhibitors is “really the most effective treatment we have in CLL.”
It’s clear that “responses deepen with continued treatment,” Dr. Rogers said, noting that remission times grow over years of treatment. She highlighted a 2022 study of patients with CLL who took ibrutinib that found complete remission or complete remission with incomplete hematologic recovery was 7% at 12 months and 34% at 7 years. When patients quit taking the drugs, “you don’t get to maximize your patient’s response to this treatment.”
Dr. Rogers also noted that the RESONATE-2 trial found that ibrutinib is linked to the longest median progression-free survival of any CLL treatment at 8.9 years. “That really struck me a very effective initial therapy.”
Indeed, “when you’re offering someone initial therapy with a Bruton’s kinase inhibitor as a continuous treatment strategy, you can tell people that they can expect a normal lifespan with this approach. That’s extremely important when you’re talking to patients about whether they might want to alter their leukemia treatment.”
Finally, she noted that discontinuation of ibrutinib was linked to shorter survival in early research. “This was the first suggestion that discontinuation is not good.”
Dr. Rogers said that discontinuing the drugs is sometimes necessary because of adverse events, but patients can “certainly switch to a more tolerable Bruton’s kinase inhibitor. With the options available today, that should be a strategy that’s considered.”
Audience members at the debate were invited to respond to a live online survey about whether Bruton’s kinase inhibitors can be discontinued. Among 49 respondents, most (52.3%) said no, 42.8% said yes, and the rest were undecided/other.
Disclosures for the speakers were not provided. Dr. Ahn disclosed consulting for BeiGene and AstraZeneca. Dr. Rogers disclosed receiving research funding from Genentech, AbbVie, Janssen, and Novartis; consulting for AstraZeneca, BeiGene, Janssen, Pharmacyclics, AbbVie, Genentech, and LOXO@Lilly; and receiving travel funding from AstraZeneca.
A version of this article appeared on Medscape.com.
At the annual meeting of the Society of Hematologic Oncology, two hematologist-oncologists — Inhye Ahn, MD, of Dana-Farber Cancer Institute in Boston, Massachusetts, and Kerry A. Rogers, MD, of Ohio State University in Columbus — faced off in a debate. Ahn said the drugs can indeed be discontinued, while Rogers argued against stopping the medications.
“When I talk to my own patient about standard of care options in CLL, I use the analogy of a marathon and a sprint,” Dr. Ahn said. A marathon refers to continuous treatment with Bruton’s kinase inhibitors given daily for years, while the sprint refers to the combination of venetoclax with an anti-CD20 monoclonal antibody given over 12 cycles for the frontline regimen and 2 years for refractory CLL.
“I tell them these are both considered very efficacious regimens and well tolerated, one is by IV [the venetoclax regimen] and the other isn’t [Bruton’s kinase inhibitors], and they have different toxicity profile. I ask them what would you do? The most common question that I get from my patient is, ‘why would anyone do a marathon?’ ”
It’s not solely the length of treatment that’s important, Dr. Ahn said, as toxicities from the long-term use of Bruton’s kinase inhibitors build up over time and can lead to hypertension, arrhythmia, and sudden cardiac death.
In addition, she said, infections can occur, as well as hampered vaccine response, an important risk in the era of the COVID-19 pandemic. The cost of the drugs is high and adds up over time, and continuous use can boost resistance.
Is there a way to turn the marathon of Bruton’s kinase inhibitor use into a sprint without hurting patients? The answer is yes, through temporary discontinuation, Dr. Ahn said, although she cautioned that early cessation could lead to disease flare. “We dipped into our own database of 84 CLL patients treated with ibrutinib, and our conclusion was that temporary dose interruption or dose reduction did not impact progression-free survival”
Moving forward, she said, “more research is needed to define the optimal regimen that would lead to treatment cessation, the optimal patient population, who would benefit most from the cessation strategy, treatment duration, and how we define success.” For her part, Dr. Rogers argued that the continuous use of Bruton’s kinase inhibitors is “really the most effective treatment we have in CLL.”
It’s clear that “responses deepen with continued treatment,” Dr. Rogers said, noting that remission times grow over years of treatment. She highlighted a 2022 study of patients with CLL who took ibrutinib that found complete remission or complete remission with incomplete hematologic recovery was 7% at 12 months and 34% at 7 years. When patients quit taking the drugs, “you don’t get to maximize your patient’s response to this treatment.”
Dr. Rogers also noted that the RESONATE-2 trial found that ibrutinib is linked to the longest median progression-free survival of any CLL treatment at 8.9 years. “That really struck me a very effective initial therapy.”
Indeed, “when you’re offering someone initial therapy with a Bruton’s kinase inhibitor as a continuous treatment strategy, you can tell people that they can expect a normal lifespan with this approach. That’s extremely important when you’re talking to patients about whether they might want to alter their leukemia treatment.”
Finally, she noted that discontinuation of ibrutinib was linked to shorter survival in early research. “This was the first suggestion that discontinuation is not good.”
Dr. Rogers said that discontinuing the drugs is sometimes necessary because of adverse events, but patients can “certainly switch to a more tolerable Bruton’s kinase inhibitor. With the options available today, that should be a strategy that’s considered.”
Audience members at the debate were invited to respond to a live online survey about whether Bruton’s kinase inhibitors can be discontinued. Among 49 respondents, most (52.3%) said no, 42.8% said yes, and the rest were undecided/other.
Disclosures for the speakers were not provided. Dr. Ahn disclosed consulting for BeiGene and AstraZeneca. Dr. Rogers disclosed receiving research funding from Genentech, AbbVie, Janssen, and Novartis; consulting for AstraZeneca, BeiGene, Janssen, Pharmacyclics, AbbVie, Genentech, and LOXO@Lilly; and receiving travel funding from AstraZeneca.
A version of this article appeared on Medscape.com.
FROM SOHO 2024
Could Aspirin Avert Bad Outcomes in Leukemia?
A new analysis hints that there may be a benefit from aspirin for hospitalized patients with leukemia. In a preliminary study, researchers found that aspirin users had much lower odds of intracranial bleeding, deep vein thrombosis, in-hospital mortality, and septic stroke.
Aspirin users also spent less time in the hospital and had less costly care.
No one is suggesting that clinicians give aspirin to hospitalized patients with leukemia when the drug is not otherwise indicated. However, the findings, released at the Society of Hematologic Oncology (SOHO 2024) meeting in Houston, do indicate that more research is warranted, study lead author Jayalekshmi Jayakumar, MD, of the Brooklyn Hospital Center in New York City, said in a presentation.
“We hope our study can act as background for further prospective and experimental studies to explore this association,” she said. “If we can establish causation, then aspirin has a potential to be a thromboprophylactic agent to enhance outcomes and reduce resource utilization among leukemia hospitalizations.”
Dr. Jayakumar noted that previous research has suggested aspirin may help prevent deep vein thrombosis in patients with breast and pancreatic cancer. And in blood cancer, animal research has suggested that aspirin may “promote apoptosis in leukemia cells and decrease the spread of leukemia cells through platelet inhibition,” she said.
However, “we do not have any prospective or retrospective studies to establish causation or to see if this actually has some value within the clinical practice,” she noted.
Dr. Jayakumar stated that new study aims to detect whether aspirin may be beneficial in leukemia. She and her colleagues retrospectively tracked 1,663,149 US hospitalizations of patients with leukemia from 2016 to 2020 via the National Inpatient Sample. Of those patients, 11.2% used aspirin, although the data didn’t say whether they started it during hospitalization, and dosages were not reported. Aspirin users were older (mean age, 74.53 years vs 64.83 years in nonusers).
After adjustment for confounders, aspirin users had lower odds of several conditions than nonusers:
- Epistaxis (odds ratio [OR], 0.63; 95% CI, 0.55-0.72; P < .001)
- Hemoptysis (OR, 0.71; 95% CI, 0.61-0.82; P < .001)
- Intracranial bleed (OR, 0.74; 95% CI, 0.64-0.85; P < .001)
- Deep vein thrombosis (OR, 0.72; 95% CI, 0.66-0.78; P < .001)
- In-hospital mortality (OR, 0.54; 95% CI, 0.50-0.58; P < .001)
- Sepsis (OR, 0.71; 95% CI, 0.68-0.75; P < .001)
- Septic shock (OR, 0.55; 95% CI, 0.50-0.60; P < .001)
There was no association reported for gastrointestinal bleeding, a possible side effect of aspirin use, or tumor lysis syndrome. Aspirin users also had a shorter typical stay (−2.8 days) and lower typical hospital charges ($40,719).
“We also found that aspirin users had a slightly reduced risk of minor bleeding and infection compared to non–aspirin users,” Dr. Jayakumar said.
In an interview, Dr. Jayakumar noted that the study is retrospective and declined to speculate on why aspirin may have benefits or why it may have the seemingly contradictory effect of reducing both blood clots and bleeding.
Aspirin is one of the least expensive drugs in existence.
In an interview, Richard M. Stone, MD, oncologist at Dana-Farber Cancer Institute in Boston, who’s familiar with the study findings but didn’t take part in the research, said the findings are “totally counterintuitive.”
“It doesn’t mean they should be rejected, but they should be highly scrutinized,” he said.
Dr. Stone added that bleeding is a major risk in leukemia due to low platelet counts, although platelet transplants can be helpful, and patients rarely die of bleeding. Thrombosis is also a problem in leukemia, he said, and it’s being increasingly recognized as a risk in acute myeloid leukemia.
No funding was reported. Dr. Jayakumar and Dr. Stone had no disclosures.
A version of this article appeared on Medscape.com.
A new analysis hints that there may be a benefit from aspirin for hospitalized patients with leukemia. In a preliminary study, researchers found that aspirin users had much lower odds of intracranial bleeding, deep vein thrombosis, in-hospital mortality, and septic stroke.
Aspirin users also spent less time in the hospital and had less costly care.
No one is suggesting that clinicians give aspirin to hospitalized patients with leukemia when the drug is not otherwise indicated. However, the findings, released at the Society of Hematologic Oncology (SOHO 2024) meeting in Houston, do indicate that more research is warranted, study lead author Jayalekshmi Jayakumar, MD, of the Brooklyn Hospital Center in New York City, said in a presentation.
“We hope our study can act as background for further prospective and experimental studies to explore this association,” she said. “If we can establish causation, then aspirin has a potential to be a thromboprophylactic agent to enhance outcomes and reduce resource utilization among leukemia hospitalizations.”
Dr. Jayakumar noted that previous research has suggested aspirin may help prevent deep vein thrombosis in patients with breast and pancreatic cancer. And in blood cancer, animal research has suggested that aspirin may “promote apoptosis in leukemia cells and decrease the spread of leukemia cells through platelet inhibition,” she said.
However, “we do not have any prospective or retrospective studies to establish causation or to see if this actually has some value within the clinical practice,” she noted.
Dr. Jayakumar stated that new study aims to detect whether aspirin may be beneficial in leukemia. She and her colleagues retrospectively tracked 1,663,149 US hospitalizations of patients with leukemia from 2016 to 2020 via the National Inpatient Sample. Of those patients, 11.2% used aspirin, although the data didn’t say whether they started it during hospitalization, and dosages were not reported. Aspirin users were older (mean age, 74.53 years vs 64.83 years in nonusers).
After adjustment for confounders, aspirin users had lower odds of several conditions than nonusers:
- Epistaxis (odds ratio [OR], 0.63; 95% CI, 0.55-0.72; P < .001)
- Hemoptysis (OR, 0.71; 95% CI, 0.61-0.82; P < .001)
- Intracranial bleed (OR, 0.74; 95% CI, 0.64-0.85; P < .001)
- Deep vein thrombosis (OR, 0.72; 95% CI, 0.66-0.78; P < .001)
- In-hospital mortality (OR, 0.54; 95% CI, 0.50-0.58; P < .001)
- Sepsis (OR, 0.71; 95% CI, 0.68-0.75; P < .001)
- Septic shock (OR, 0.55; 95% CI, 0.50-0.60; P < .001)
There was no association reported for gastrointestinal bleeding, a possible side effect of aspirin use, or tumor lysis syndrome. Aspirin users also had a shorter typical stay (−2.8 days) and lower typical hospital charges ($40,719).
“We also found that aspirin users had a slightly reduced risk of minor bleeding and infection compared to non–aspirin users,” Dr. Jayakumar said.
In an interview, Dr. Jayakumar noted that the study is retrospective and declined to speculate on why aspirin may have benefits or why it may have the seemingly contradictory effect of reducing both blood clots and bleeding.
Aspirin is one of the least expensive drugs in existence.
In an interview, Richard M. Stone, MD, oncologist at Dana-Farber Cancer Institute in Boston, who’s familiar with the study findings but didn’t take part in the research, said the findings are “totally counterintuitive.”
“It doesn’t mean they should be rejected, but they should be highly scrutinized,” he said.
Dr. Stone added that bleeding is a major risk in leukemia due to low platelet counts, although platelet transplants can be helpful, and patients rarely die of bleeding. Thrombosis is also a problem in leukemia, he said, and it’s being increasingly recognized as a risk in acute myeloid leukemia.
No funding was reported. Dr. Jayakumar and Dr. Stone had no disclosures.
A version of this article appeared on Medscape.com.
A new analysis hints that there may be a benefit from aspirin for hospitalized patients with leukemia. In a preliminary study, researchers found that aspirin users had much lower odds of intracranial bleeding, deep vein thrombosis, in-hospital mortality, and septic stroke.
Aspirin users also spent less time in the hospital and had less costly care.
No one is suggesting that clinicians give aspirin to hospitalized patients with leukemia when the drug is not otherwise indicated. However, the findings, released at the Society of Hematologic Oncology (SOHO 2024) meeting in Houston, do indicate that more research is warranted, study lead author Jayalekshmi Jayakumar, MD, of the Brooklyn Hospital Center in New York City, said in a presentation.
“We hope our study can act as background for further prospective and experimental studies to explore this association,” she said. “If we can establish causation, then aspirin has a potential to be a thromboprophylactic agent to enhance outcomes and reduce resource utilization among leukemia hospitalizations.”
Dr. Jayakumar noted that previous research has suggested aspirin may help prevent deep vein thrombosis in patients with breast and pancreatic cancer. And in blood cancer, animal research has suggested that aspirin may “promote apoptosis in leukemia cells and decrease the spread of leukemia cells through platelet inhibition,” she said.
However, “we do not have any prospective or retrospective studies to establish causation or to see if this actually has some value within the clinical practice,” she noted.
Dr. Jayakumar stated that new study aims to detect whether aspirin may be beneficial in leukemia. She and her colleagues retrospectively tracked 1,663,149 US hospitalizations of patients with leukemia from 2016 to 2020 via the National Inpatient Sample. Of those patients, 11.2% used aspirin, although the data didn’t say whether they started it during hospitalization, and dosages were not reported. Aspirin users were older (mean age, 74.53 years vs 64.83 years in nonusers).
After adjustment for confounders, aspirin users had lower odds of several conditions than nonusers:
- Epistaxis (odds ratio [OR], 0.63; 95% CI, 0.55-0.72; P < .001)
- Hemoptysis (OR, 0.71; 95% CI, 0.61-0.82; P < .001)
- Intracranial bleed (OR, 0.74; 95% CI, 0.64-0.85; P < .001)
- Deep vein thrombosis (OR, 0.72; 95% CI, 0.66-0.78; P < .001)
- In-hospital mortality (OR, 0.54; 95% CI, 0.50-0.58; P < .001)
- Sepsis (OR, 0.71; 95% CI, 0.68-0.75; P < .001)
- Septic shock (OR, 0.55; 95% CI, 0.50-0.60; P < .001)
There was no association reported for gastrointestinal bleeding, a possible side effect of aspirin use, or tumor lysis syndrome. Aspirin users also had a shorter typical stay (−2.8 days) and lower typical hospital charges ($40,719).
“We also found that aspirin users had a slightly reduced risk of minor bleeding and infection compared to non–aspirin users,” Dr. Jayakumar said.
In an interview, Dr. Jayakumar noted that the study is retrospective and declined to speculate on why aspirin may have benefits or why it may have the seemingly contradictory effect of reducing both blood clots and bleeding.
Aspirin is one of the least expensive drugs in existence.
In an interview, Richard M. Stone, MD, oncologist at Dana-Farber Cancer Institute in Boston, who’s familiar with the study findings but didn’t take part in the research, said the findings are “totally counterintuitive.”
“It doesn’t mean they should be rejected, but they should be highly scrutinized,” he said.
Dr. Stone added that bleeding is a major risk in leukemia due to low platelet counts, although platelet transplants can be helpful, and patients rarely die of bleeding. Thrombosis is also a problem in leukemia, he said, and it’s being increasingly recognized as a risk in acute myeloid leukemia.
No funding was reported. Dr. Jayakumar and Dr. Stone had no disclosures.
A version of this article appeared on Medscape.com.
FROM SOHO 2024
Wait, a Health Worker Surplus? Workforce Report Projects Big Surprises
A surprising new report by the Mercer consulting firm projects that the American healthcare workforce will face a small shortfall in 2028 — a shortage of less than 1% of all employees.
Mercer’s projections are rosier than federal workforce projections, which paint a grimmer picture of impending shortages.
“The labor market is a little more stabilized right now, and most healthcare systems are seeing less turnover,” Dan Lezotte, PhD, a partner with Mercer, said in an interview. But he noted “critical shortages” are still expected in some areas.
Mercer last projected workforce numbers in a 2020-2021 report released during the height of the COVID-19 pandemic. Now, “the labor market is drastically different,” Dr. Lezotte said. Health workforce shortages and surpluses have long varied significantly by region across the country.
The report forecasts a small surplus of physicians in 2028 but not in states such as California, New York, and Texas. The upper Midwest states will largely see doctor surpluses while Southern states face shortages. Some states with general physician surpluses may still experience shortages of specialists.
A surplus of nearly 30,000 registered nurses is expected, but New York, New Jersey, and Connecticut are projected to have a combined shortage of 16,000 nurses.
Overall, the report projects a shortage of more than 100,000 healthcare workers nationally by 2028. That’s less than 1% of the entire healthcare workforce of 18.6 million expected by then.
The report also predicts a shortage of nurse practitioners, especially in California and New York, and a shortage of 73,000 nursing assistants, especially in California, New York, and Texas.
“Healthcare systems are having the most difficulty hiring and hanging on to those workers who are supposed to take up the load off physicians and nurses,” Dr. Lezotte said. “They’re competing not only with other healthcare systems but with other industries like Amazon warehouses or McDonald’s in California paying $20 an hour. Healthcare was a little slow to keep up with that. In a lot of healthcare systems, that’s their biggest headache right now.”
On the other hand, the report projects a national surplus of 48,000 home health/personal care aides.
That surprised Bianca K. Frogner, PhD, director of the Center for Health Workforce Studies at the University of Washington, Seattle.
“We are seeing increasing movement of investments toward moving patients out of skilled nursing facilities and keeping them in the home and community, which requires many more home health aides,” Dr. Frogner said. “Given such high turnover in this occupation, it’s hard to know if the surplus is really a surplus or if they will quickly be employed.”
Dr. Frogner receives grants and contracts from not-for-profit entities to investigate issues related to the health workforce.
Dr. Lezotte said the report’s findings are based on data from sources such as public and private databases and job postings. According to the report, “projections were made up to 2028 based on historical data up to 2023,” and “supply projections were derived using a linear autoregressive model based on historical supply within each occupation and geography.”
It’s not clear why some states like New York are expected to have huge shortages, but migration might be a factor, along with a lack of nearby nursing schools, Dr. Lezotte said.
As for shortages, Dr. Lezotte said healthcare systems will have to understand their local workforce situation and adapt. “They’ll need to be more proactive about their employee value proposition” via competitive pay and benefits Flexibility regarding scheduling is also important.
“They’re going to have to figure out how to up their game,” he said.
What about states with surpluses? They might be target-rich environments for states facing shortages, he said.
Positive Outlook Not Shared by Other Researchers
Other workforce projections conflict with Mercer’s, according to Jean Moore, DrPH, and Gaetano Forte, MS, director and assistant director of the Center for Health Workforce Studies, School of Public Health, University at Albany, New York.
The National Center for Health Workforce Analysis projects a 10% shortage of registered nurses and a 13% shortage of physicians in 2031. The agency didn’t make projections for home health aides because that workforce is in flux.
Why are Mercer’s projections so different? Dr. Lezotte said other projections assume that equity efforts will bring healthcare to everyone who needs it. The report assumes this won’t happen, he said. As a result, it expects there will be fewer patients who need to be served by workers.
Other projections expect a shortage of 300,000 registered nurses by 2035, Mr. Forte said. But the number of nurse practitioners in New York is growing quickly, Dr. Moore said.
Dr. Moore said it’s difficult to interpret Mercer’s findings because the company doesn’t provide enough information about its methodology.
“At some level, it’s not particularly useful regarding what the next steps are,” she said. “Projections should make you think about what you should do to change and improve, to create more of what you need.”
The Center for Health Workforce Studies at the University of Albany has provided consulting services to multiple companies that provide healthcare workforce projections. It has no relationship with Mercer.
A version of this article first appeared on Medscape.com.
A surprising new report by the Mercer consulting firm projects that the American healthcare workforce will face a small shortfall in 2028 — a shortage of less than 1% of all employees.
Mercer’s projections are rosier than federal workforce projections, which paint a grimmer picture of impending shortages.
“The labor market is a little more stabilized right now, and most healthcare systems are seeing less turnover,” Dan Lezotte, PhD, a partner with Mercer, said in an interview. But he noted “critical shortages” are still expected in some areas.
Mercer last projected workforce numbers in a 2020-2021 report released during the height of the COVID-19 pandemic. Now, “the labor market is drastically different,” Dr. Lezotte said. Health workforce shortages and surpluses have long varied significantly by region across the country.
The report forecasts a small surplus of physicians in 2028 but not in states such as California, New York, and Texas. The upper Midwest states will largely see doctor surpluses while Southern states face shortages. Some states with general physician surpluses may still experience shortages of specialists.
A surplus of nearly 30,000 registered nurses is expected, but New York, New Jersey, and Connecticut are projected to have a combined shortage of 16,000 nurses.
Overall, the report projects a shortage of more than 100,000 healthcare workers nationally by 2028. That’s less than 1% of the entire healthcare workforce of 18.6 million expected by then.
The report also predicts a shortage of nurse practitioners, especially in California and New York, and a shortage of 73,000 nursing assistants, especially in California, New York, and Texas.
“Healthcare systems are having the most difficulty hiring and hanging on to those workers who are supposed to take up the load off physicians and nurses,” Dr. Lezotte said. “They’re competing not only with other healthcare systems but with other industries like Amazon warehouses or McDonald’s in California paying $20 an hour. Healthcare was a little slow to keep up with that. In a lot of healthcare systems, that’s their biggest headache right now.”
On the other hand, the report projects a national surplus of 48,000 home health/personal care aides.
That surprised Bianca K. Frogner, PhD, director of the Center for Health Workforce Studies at the University of Washington, Seattle.
“We are seeing increasing movement of investments toward moving patients out of skilled nursing facilities and keeping them in the home and community, which requires many more home health aides,” Dr. Frogner said. “Given such high turnover in this occupation, it’s hard to know if the surplus is really a surplus or if they will quickly be employed.”
Dr. Frogner receives grants and contracts from not-for-profit entities to investigate issues related to the health workforce.
Dr. Lezotte said the report’s findings are based on data from sources such as public and private databases and job postings. According to the report, “projections were made up to 2028 based on historical data up to 2023,” and “supply projections were derived using a linear autoregressive model based on historical supply within each occupation and geography.”
It’s not clear why some states like New York are expected to have huge shortages, but migration might be a factor, along with a lack of nearby nursing schools, Dr. Lezotte said.
As for shortages, Dr. Lezotte said healthcare systems will have to understand their local workforce situation and adapt. “They’ll need to be more proactive about their employee value proposition” via competitive pay and benefits Flexibility regarding scheduling is also important.
“They’re going to have to figure out how to up their game,” he said.
What about states with surpluses? They might be target-rich environments for states facing shortages, he said.
Positive Outlook Not Shared by Other Researchers
Other workforce projections conflict with Mercer’s, according to Jean Moore, DrPH, and Gaetano Forte, MS, director and assistant director of the Center for Health Workforce Studies, School of Public Health, University at Albany, New York.
The National Center for Health Workforce Analysis projects a 10% shortage of registered nurses and a 13% shortage of physicians in 2031. The agency didn’t make projections for home health aides because that workforce is in flux.
Why are Mercer’s projections so different? Dr. Lezotte said other projections assume that equity efforts will bring healthcare to everyone who needs it. The report assumes this won’t happen, he said. As a result, it expects there will be fewer patients who need to be served by workers.
Other projections expect a shortage of 300,000 registered nurses by 2035, Mr. Forte said. But the number of nurse practitioners in New York is growing quickly, Dr. Moore said.
Dr. Moore said it’s difficult to interpret Mercer’s findings because the company doesn’t provide enough information about its methodology.
“At some level, it’s not particularly useful regarding what the next steps are,” she said. “Projections should make you think about what you should do to change and improve, to create more of what you need.”
The Center for Health Workforce Studies at the University of Albany has provided consulting services to multiple companies that provide healthcare workforce projections. It has no relationship with Mercer.
A version of this article first appeared on Medscape.com.
A surprising new report by the Mercer consulting firm projects that the American healthcare workforce will face a small shortfall in 2028 — a shortage of less than 1% of all employees.
Mercer’s projections are rosier than federal workforce projections, which paint a grimmer picture of impending shortages.
“The labor market is a little more stabilized right now, and most healthcare systems are seeing less turnover,” Dan Lezotte, PhD, a partner with Mercer, said in an interview. But he noted “critical shortages” are still expected in some areas.
Mercer last projected workforce numbers in a 2020-2021 report released during the height of the COVID-19 pandemic. Now, “the labor market is drastically different,” Dr. Lezotte said. Health workforce shortages and surpluses have long varied significantly by region across the country.
The report forecasts a small surplus of physicians in 2028 but not in states such as California, New York, and Texas. The upper Midwest states will largely see doctor surpluses while Southern states face shortages. Some states with general physician surpluses may still experience shortages of specialists.
A surplus of nearly 30,000 registered nurses is expected, but New York, New Jersey, and Connecticut are projected to have a combined shortage of 16,000 nurses.
Overall, the report projects a shortage of more than 100,000 healthcare workers nationally by 2028. That’s less than 1% of the entire healthcare workforce of 18.6 million expected by then.
The report also predicts a shortage of nurse practitioners, especially in California and New York, and a shortage of 73,000 nursing assistants, especially in California, New York, and Texas.
“Healthcare systems are having the most difficulty hiring and hanging on to those workers who are supposed to take up the load off physicians and nurses,” Dr. Lezotte said. “They’re competing not only with other healthcare systems but with other industries like Amazon warehouses or McDonald’s in California paying $20 an hour. Healthcare was a little slow to keep up with that. In a lot of healthcare systems, that’s their biggest headache right now.”
On the other hand, the report projects a national surplus of 48,000 home health/personal care aides.
That surprised Bianca K. Frogner, PhD, director of the Center for Health Workforce Studies at the University of Washington, Seattle.
“We are seeing increasing movement of investments toward moving patients out of skilled nursing facilities and keeping them in the home and community, which requires many more home health aides,” Dr. Frogner said. “Given such high turnover in this occupation, it’s hard to know if the surplus is really a surplus or if they will quickly be employed.”
Dr. Frogner receives grants and contracts from not-for-profit entities to investigate issues related to the health workforce.
Dr. Lezotte said the report’s findings are based on data from sources such as public and private databases and job postings. According to the report, “projections were made up to 2028 based on historical data up to 2023,” and “supply projections were derived using a linear autoregressive model based on historical supply within each occupation and geography.”
It’s not clear why some states like New York are expected to have huge shortages, but migration might be a factor, along with a lack of nearby nursing schools, Dr. Lezotte said.
As for shortages, Dr. Lezotte said healthcare systems will have to understand their local workforce situation and adapt. “They’ll need to be more proactive about their employee value proposition” via competitive pay and benefits Flexibility regarding scheduling is also important.
“They’re going to have to figure out how to up their game,” he said.
What about states with surpluses? They might be target-rich environments for states facing shortages, he said.
Positive Outlook Not Shared by Other Researchers
Other workforce projections conflict with Mercer’s, according to Jean Moore, DrPH, and Gaetano Forte, MS, director and assistant director of the Center for Health Workforce Studies, School of Public Health, University at Albany, New York.
The National Center for Health Workforce Analysis projects a 10% shortage of registered nurses and a 13% shortage of physicians in 2031. The agency didn’t make projections for home health aides because that workforce is in flux.
Why are Mercer’s projections so different? Dr. Lezotte said other projections assume that equity efforts will bring healthcare to everyone who needs it. The report assumes this won’t happen, he said. As a result, it expects there will be fewer patients who need to be served by workers.
Other projections expect a shortage of 300,000 registered nurses by 2035, Mr. Forte said. But the number of nurse practitioners in New York is growing quickly, Dr. Moore said.
Dr. Moore said it’s difficult to interpret Mercer’s findings because the company doesn’t provide enough information about its methodology.
“At some level, it’s not particularly useful regarding what the next steps are,” she said. “Projections should make you think about what you should do to change and improve, to create more of what you need.”
The Center for Health Workforce Studies at the University of Albany has provided consulting services to multiple companies that provide healthcare workforce projections. It has no relationship with Mercer.
A version of this article first appeared on Medscape.com.
Primary Care Physicians Track an Average of 57 Quality Measures for Value-Based Care Pay
A new analysis suggests one reason doctors are wary of value-based care arrangements: Overkill.
Researchers found that primary care physicians in one large integrated health system were required to track an average of 57 different quality measures across multiple insurers that linked outcomes to payments under value-based contracts.
Medicare contracts were the most likely to pile quality measures on physicians with an average of 13.42 measures vs 10.07 for commercial insurer contracts and 5.37 for Medicaid contracts, reported Claire Boone, PhD, of the University of Chicago in Illinois and Providence Research Network, Portland, Oregon, and colleagues in JAMA Health Forum. The analysis, which may be the first of its kind, tracked 890 primary care physicians from 2020 to 2022.
The average of 57 quality measures per physician was unexpectedly high, Dr. Boone said in an interview.
“The magnitude of that number surprised us,” Dr. Boone said. “Primary care physicians and their practices have a lot on their plate. Now we know that one of those things is a very large number of different quality metrics to pay attention to, measure, report on, and implement.
Value-based care programs use quality measures to evaluate how well clinicians are doing their jobs and adjust reimbursement accordingly. A payer, for example, may raise reimbursements if a clinician has higher numbers of patients who meet quality measure standards for depression screening or blood pressure.
Dr. Boone said her research group is studying the impact of quality measures and was surprised that data showed individual primary care physicians had to deal with a high number of value-based contracts.
The researchers tracked value-based contracts for 890 physicians (58.3% women, 41.7% men) in an unidentified West Coast Health system. (Several study authors work for the Providence Health System, which serves several Western States and Texas.) The average number of patients per physician was 1309.
The physicians were part of an average of 11.18 value-based contracts (commercial insurers, 49.50%; Medicaid, 21.49%; and Medicare, 29.01%). This number grew from 9.39 in 2020 to 12.26 in 2022. Quality measure data weren’t available for 29% of contracts.
Quality measures were considered unique if they referenced different conditions.
For example, colorectal cancer screening is unique from depression screening. The researchers also considered measures for the same condition unique if the target value differed — for example, blood pressure control defined as < 140/90 vs blood pressure control defined as < 130/80, Dr. Boone said.
Dr. Boone said she expected payers to coordinate quality measures.
“The fact that they largely are not is really the main finding of this paper. Without coordination, the use of value-based contracts and quality measures at scale leads to many unique measures being used. This may reflect the fact that there are so many important tasks to do in primary care, and there’s no consensus on which ones should be included in quality-based contracts.”
Ronald N. Adler, MD, an associate professor in the Department of Family Medicine and Community Health at UMass Chan Medical School, Worcester, Massachusetts, who’s familiar with the findings but didn’t take part in the research, said the study offers something new — the quantification of quality measures.
He said in an interview that physicians deal with quality measures in different ways. Some clinicians “don’t really care,” and have an attitude of “this is not why I got into medicine.” But others “are very competitive around this, and it leads to a lot of a lot of stress. Trying to address 50-plus measures is impossible and demoralizing.”
The metrics may measure things like mammogram screening that are out of the physician’s control, Dr. Adler said. “I can recommend a mammogram, and my patient can choose not to do it. Or maybe my patient is homeless; she doesn’t have transportation, and it’s not a priority for her, even though she wants to do it.”
Patients may not take medication as prescribed, or they may be unable to afford it, he said. “Can they afford to eat healthy foods? Or is ramen all they can afford, and their sugars are through the roof? There are a lot of factors at play here that are independent of the quality of care provided by the doctor.”
As for his own approach, Dr. Adler said he worries about some measures more than others. “I’m very proactive about screening my patients for colon cancer and maybe a little less so about mammography.”
For colon cancer screening, “there are a lot of benefits and not that many harms as opposed to mammography, which has harms such as false positives and overdiagnosis of breast cancer.”
Dr. Adler is a member of the Quality Measure Alignment Taskforce in Massachusetts, which is trying to establish consensus on appropriate quality measures. But payer participation is voluntary. “Our health systems are too siloed ... so there is no readily available mechanism for enforcing such recommendations.”
Wayne Altman, MD, chair of Family Medicine at Tufts University School of Medicine, Boston, Massachusetts, is also familiar with the study findings but didn’t take part in the research. He said in an interview that clinicians shouldn’t have to deal with more than 5-10 quality measures in total.
He pointed out that many measures don’t make sense in certain populations. Titrating blood pressure to < 140/90 isn’t ideal for elderly patients because aggressive control can send their blood pressure dangerously low. “They’re going to fall down, break a hip, and likely die within a year. You have to have the right population and be aware of unintended consequences.”
Still, Dr. Adler noted, there’s an important role for quality measures in healthcare.
“We need data to inform our quality improvement activities, but they need to be the right measures. People can’t respond reasonably to improve on 50-plus measures,” he said. “They need to be consolidated and prioritized. It would be really helpful if we could have a much lower number of measures that are meaningful, safe, and connect to things that matter.”
No funding has been reported. Dr. Boone disclosed a grant from the National Institute on Aging. Dr. Adler and Dr. Altman had no disclosures.
A version of this article first appeared on Medscape.com.
A new analysis suggests one reason doctors are wary of value-based care arrangements: Overkill.
Researchers found that primary care physicians in one large integrated health system were required to track an average of 57 different quality measures across multiple insurers that linked outcomes to payments under value-based contracts.
Medicare contracts were the most likely to pile quality measures on physicians with an average of 13.42 measures vs 10.07 for commercial insurer contracts and 5.37 for Medicaid contracts, reported Claire Boone, PhD, of the University of Chicago in Illinois and Providence Research Network, Portland, Oregon, and colleagues in JAMA Health Forum. The analysis, which may be the first of its kind, tracked 890 primary care physicians from 2020 to 2022.
The average of 57 quality measures per physician was unexpectedly high, Dr. Boone said in an interview.
“The magnitude of that number surprised us,” Dr. Boone said. “Primary care physicians and their practices have a lot on their plate. Now we know that one of those things is a very large number of different quality metrics to pay attention to, measure, report on, and implement.
Value-based care programs use quality measures to evaluate how well clinicians are doing their jobs and adjust reimbursement accordingly. A payer, for example, may raise reimbursements if a clinician has higher numbers of patients who meet quality measure standards for depression screening or blood pressure.
Dr. Boone said her research group is studying the impact of quality measures and was surprised that data showed individual primary care physicians had to deal with a high number of value-based contracts.
The researchers tracked value-based contracts for 890 physicians (58.3% women, 41.7% men) in an unidentified West Coast Health system. (Several study authors work for the Providence Health System, which serves several Western States and Texas.) The average number of patients per physician was 1309.
The physicians were part of an average of 11.18 value-based contracts (commercial insurers, 49.50%; Medicaid, 21.49%; and Medicare, 29.01%). This number grew from 9.39 in 2020 to 12.26 in 2022. Quality measure data weren’t available for 29% of contracts.
Quality measures were considered unique if they referenced different conditions.
For example, colorectal cancer screening is unique from depression screening. The researchers also considered measures for the same condition unique if the target value differed — for example, blood pressure control defined as < 140/90 vs blood pressure control defined as < 130/80, Dr. Boone said.
Dr. Boone said she expected payers to coordinate quality measures.
“The fact that they largely are not is really the main finding of this paper. Without coordination, the use of value-based contracts and quality measures at scale leads to many unique measures being used. This may reflect the fact that there are so many important tasks to do in primary care, and there’s no consensus on which ones should be included in quality-based contracts.”
Ronald N. Adler, MD, an associate professor in the Department of Family Medicine and Community Health at UMass Chan Medical School, Worcester, Massachusetts, who’s familiar with the findings but didn’t take part in the research, said the study offers something new — the quantification of quality measures.
He said in an interview that physicians deal with quality measures in different ways. Some clinicians “don’t really care,” and have an attitude of “this is not why I got into medicine.” But others “are very competitive around this, and it leads to a lot of a lot of stress. Trying to address 50-plus measures is impossible and demoralizing.”
The metrics may measure things like mammogram screening that are out of the physician’s control, Dr. Adler said. “I can recommend a mammogram, and my patient can choose not to do it. Or maybe my patient is homeless; she doesn’t have transportation, and it’s not a priority for her, even though she wants to do it.”
Patients may not take medication as prescribed, or they may be unable to afford it, he said. “Can they afford to eat healthy foods? Or is ramen all they can afford, and their sugars are through the roof? There are a lot of factors at play here that are independent of the quality of care provided by the doctor.”
As for his own approach, Dr. Adler said he worries about some measures more than others. “I’m very proactive about screening my patients for colon cancer and maybe a little less so about mammography.”
For colon cancer screening, “there are a lot of benefits and not that many harms as opposed to mammography, which has harms such as false positives and overdiagnosis of breast cancer.”
Dr. Adler is a member of the Quality Measure Alignment Taskforce in Massachusetts, which is trying to establish consensus on appropriate quality measures. But payer participation is voluntary. “Our health systems are too siloed ... so there is no readily available mechanism for enforcing such recommendations.”
Wayne Altman, MD, chair of Family Medicine at Tufts University School of Medicine, Boston, Massachusetts, is also familiar with the study findings but didn’t take part in the research. He said in an interview that clinicians shouldn’t have to deal with more than 5-10 quality measures in total.
He pointed out that many measures don’t make sense in certain populations. Titrating blood pressure to < 140/90 isn’t ideal for elderly patients because aggressive control can send their blood pressure dangerously low. “They’re going to fall down, break a hip, and likely die within a year. You have to have the right population and be aware of unintended consequences.”
Still, Dr. Adler noted, there’s an important role for quality measures in healthcare.
“We need data to inform our quality improvement activities, but they need to be the right measures. People can’t respond reasonably to improve on 50-plus measures,” he said. “They need to be consolidated and prioritized. It would be really helpful if we could have a much lower number of measures that are meaningful, safe, and connect to things that matter.”
No funding has been reported. Dr. Boone disclosed a grant from the National Institute on Aging. Dr. Adler and Dr. Altman had no disclosures.
A version of this article first appeared on Medscape.com.
A new analysis suggests one reason doctors are wary of value-based care arrangements: Overkill.
Researchers found that primary care physicians in one large integrated health system were required to track an average of 57 different quality measures across multiple insurers that linked outcomes to payments under value-based contracts.
Medicare contracts were the most likely to pile quality measures on physicians with an average of 13.42 measures vs 10.07 for commercial insurer contracts and 5.37 for Medicaid contracts, reported Claire Boone, PhD, of the University of Chicago in Illinois and Providence Research Network, Portland, Oregon, and colleagues in JAMA Health Forum. The analysis, which may be the first of its kind, tracked 890 primary care physicians from 2020 to 2022.
The average of 57 quality measures per physician was unexpectedly high, Dr. Boone said in an interview.
“The magnitude of that number surprised us,” Dr. Boone said. “Primary care physicians and their practices have a lot on their plate. Now we know that one of those things is a very large number of different quality metrics to pay attention to, measure, report on, and implement.
Value-based care programs use quality measures to evaluate how well clinicians are doing their jobs and adjust reimbursement accordingly. A payer, for example, may raise reimbursements if a clinician has higher numbers of patients who meet quality measure standards for depression screening or blood pressure.
Dr. Boone said her research group is studying the impact of quality measures and was surprised that data showed individual primary care physicians had to deal with a high number of value-based contracts.
The researchers tracked value-based contracts for 890 physicians (58.3% women, 41.7% men) in an unidentified West Coast Health system. (Several study authors work for the Providence Health System, which serves several Western States and Texas.) The average number of patients per physician was 1309.
The physicians were part of an average of 11.18 value-based contracts (commercial insurers, 49.50%; Medicaid, 21.49%; and Medicare, 29.01%). This number grew from 9.39 in 2020 to 12.26 in 2022. Quality measure data weren’t available for 29% of contracts.
Quality measures were considered unique if they referenced different conditions.
For example, colorectal cancer screening is unique from depression screening. The researchers also considered measures for the same condition unique if the target value differed — for example, blood pressure control defined as < 140/90 vs blood pressure control defined as < 130/80, Dr. Boone said.
Dr. Boone said she expected payers to coordinate quality measures.
“The fact that they largely are not is really the main finding of this paper. Without coordination, the use of value-based contracts and quality measures at scale leads to many unique measures being used. This may reflect the fact that there are so many important tasks to do in primary care, and there’s no consensus on which ones should be included in quality-based contracts.”
Ronald N. Adler, MD, an associate professor in the Department of Family Medicine and Community Health at UMass Chan Medical School, Worcester, Massachusetts, who’s familiar with the findings but didn’t take part in the research, said the study offers something new — the quantification of quality measures.
He said in an interview that physicians deal with quality measures in different ways. Some clinicians “don’t really care,” and have an attitude of “this is not why I got into medicine.” But others “are very competitive around this, and it leads to a lot of a lot of stress. Trying to address 50-plus measures is impossible and demoralizing.”
The metrics may measure things like mammogram screening that are out of the physician’s control, Dr. Adler said. “I can recommend a mammogram, and my patient can choose not to do it. Or maybe my patient is homeless; she doesn’t have transportation, and it’s not a priority for her, even though she wants to do it.”
Patients may not take medication as prescribed, or they may be unable to afford it, he said. “Can they afford to eat healthy foods? Or is ramen all they can afford, and their sugars are through the roof? There are a lot of factors at play here that are independent of the quality of care provided by the doctor.”
As for his own approach, Dr. Adler said he worries about some measures more than others. “I’m very proactive about screening my patients for colon cancer and maybe a little less so about mammography.”
For colon cancer screening, “there are a lot of benefits and not that many harms as opposed to mammography, which has harms such as false positives and overdiagnosis of breast cancer.”
Dr. Adler is a member of the Quality Measure Alignment Taskforce in Massachusetts, which is trying to establish consensus on appropriate quality measures. But payer participation is voluntary. “Our health systems are too siloed ... so there is no readily available mechanism for enforcing such recommendations.”
Wayne Altman, MD, chair of Family Medicine at Tufts University School of Medicine, Boston, Massachusetts, is also familiar with the study findings but didn’t take part in the research. He said in an interview that clinicians shouldn’t have to deal with more than 5-10 quality measures in total.
He pointed out that many measures don’t make sense in certain populations. Titrating blood pressure to < 140/90 isn’t ideal for elderly patients because aggressive control can send their blood pressure dangerously low. “They’re going to fall down, break a hip, and likely die within a year. You have to have the right population and be aware of unintended consequences.”
Still, Dr. Adler noted, there’s an important role for quality measures in healthcare.
“We need data to inform our quality improvement activities, but they need to be the right measures. People can’t respond reasonably to improve on 50-plus measures,” he said. “They need to be consolidated and prioritized. It would be really helpful if we could have a much lower number of measures that are meaningful, safe, and connect to things that matter.”
No funding has been reported. Dr. Boone disclosed a grant from the National Institute on Aging. Dr. Adler and Dr. Altman had no disclosures.
A version of this article first appeared on Medscape.com.
FROM JAMA HEALTH FORUM
No Surprises Act: Private Equity Scores Big in Arbitrations
Four organizations owned by private equity firms — including two provider groups — dominated the No Surprises Act’s disputed bill arbitration process in its first year, filing about 70% of 657,040 cases against insurers in 2023, a new report finds.
The findings, recently published in Health Affairs, suggest that private equity–owned organizations are forcefully challenging insurers about payments for certain kinds of out-of-network care.
Their fighting stance has paid off: The percentage of resolved arbitration cases won by providers jumped from 72% in the first quarter of 2023 to 85% in the last quarter, and they were awarded a median of more than 300% the contracted in-network rates for the services in question.
With many more out-of-network bills disputed by providers than expected, “the system is not working exactly the way it was anticipated when this law was written,” lead author Jack Hoadley, PhD, a research professor emeritus at Georgetown University’s McCourt School of Public Policy, Washington, DC, told this news organization.
And, he said, the public and the federal government may end up paying a price.
Congress passed the No Surprises Act in 2020 and then-President Donald Trump signed it. The landmark bill, which went into effect in 2022, was designed to protect patients from unexpected and often exorbitant “surprise” bills after they received some kinds of out-of-network care.
Now, many types of providers are forbidden from billing patients beyond normal in-network costs. In these cases, health plans and out-of-network providers — who don’t have mutual agreements — must wrangle over payment amounts, which are intended to not exceed inflation-adjusted 2019 median levels.
A binding arbitration process kicks in when a provider and a health plan fail to agree about how much the plan will pay for a service. Then, a third-party arbitrator is called in to make a ruling that’s binding. The process is controversial, and a flurry of lawsuits from providers have challenged it.
The new report, which updates an earlier analysis, examines data about disputed cases from all of 2023.
Of the 657,040 new cases filed in 2023, about 70% came from four private equity-funded organizations: Team Health, SCP Health, Radiology Partners, and Envision, which each provide physician services.
About half of the 2023 cases were from just four states: Texas, Florida, Tennessee, and Georgia. The report says the four organizations are especially active in those states. In contrast, Connecticut, Maryland, Massachusetts, and Washington state each had just 1500 or fewer cases filed last year.
Health plans challenged a third of cases as ineligible, and 22% of all resolved cases were deemed ineligible.
Providers won 80% of resolved challenges in 2023, although it’s not clear how much money they reaped. Still, it’s clear that “in the vast majority of the cases, insurers have to pay larger amounts to the provider,” Dr. Hoadley said.
Radiologists made a median of at least 500% of the in-network rate in their cases. Surgeons and neurologists made even more money — a median of at least 800% of the in-network rate. Overall, providers made 322%-350% of in-network rates, depending on the quarter.
Dr. Hoadley cautioned that only a small percentage of medical payments are disputed. In those cases, “the amount that the insurer offers is accepted, and that’s the end of the story.”
Why are the providers often reaping much more than typical payments for in-network services? It’s “really hard to know,” Dr. Hoadley said. But one factor, he said, may be the fact that providers are able to offer evidence challenging that amounts that insurers say they paid previously: “Hey, when we were in network, we were paid this much.”
It’s not clear whether the dispute-and-arbitration system will cost insurers — and patients — more in the long run. The Congressional Budget Office actually thought the No Surprises Act might lower the growth of premiums slightly and save the federal government money, Dr. Hoadley said, but that could potentially not happen. The flood of litigation also contributes to uncertainty, he said.
Alan Sager, PhD, professor of Health Law, Policy, and Management at Boston University School of Public Health, told this news organization that premiums are bound to rise as insurers react to higher costs. He also expects that providers will question the value of being in-network. “If you’re out-of-network and can obtain much higher payments, why would any doctor or hospital remain in-network, especially since they don’t lose out on patient volume?”
Why are provider groups owned by private equity firms so aggressive at challenging health plans? Loren Adler, a fellow and associate director of the Brookings Institution’s Center on Health Policy, told this news organization that these companies play large roles in fields affected by the No Surprises Act. These include emergency medicine, radiology, and anesthesiology, said Mr. Adler, who’s also studied the No Surprises Act’s dispute/arbitration system.
Mr. Adler added that larger companies “are better suited to deal with technical complexities of this process and spend the sort of upfront money to go through it.”
In the big picture, Mr. Adler said, the new study “raises question of whether Congress at some point wants to try to basically bring prices from the arbitration process back in line with average in-network prices.”
The study was funded by the Commonwealth Fund and Arnold Ventures. Dr. Hoadley, Dr. Sager, and Mr. Adler had no disclosures.
A version of this article first appeared on Medscape.com.
Four organizations owned by private equity firms — including two provider groups — dominated the No Surprises Act’s disputed bill arbitration process in its first year, filing about 70% of 657,040 cases against insurers in 2023, a new report finds.
The findings, recently published in Health Affairs, suggest that private equity–owned organizations are forcefully challenging insurers about payments for certain kinds of out-of-network care.
Their fighting stance has paid off: The percentage of resolved arbitration cases won by providers jumped from 72% in the first quarter of 2023 to 85% in the last quarter, and they were awarded a median of more than 300% the contracted in-network rates for the services in question.
With many more out-of-network bills disputed by providers than expected, “the system is not working exactly the way it was anticipated when this law was written,” lead author Jack Hoadley, PhD, a research professor emeritus at Georgetown University’s McCourt School of Public Policy, Washington, DC, told this news organization.
And, he said, the public and the federal government may end up paying a price.
Congress passed the No Surprises Act in 2020 and then-President Donald Trump signed it. The landmark bill, which went into effect in 2022, was designed to protect patients from unexpected and often exorbitant “surprise” bills after they received some kinds of out-of-network care.
Now, many types of providers are forbidden from billing patients beyond normal in-network costs. In these cases, health plans and out-of-network providers — who don’t have mutual agreements — must wrangle over payment amounts, which are intended to not exceed inflation-adjusted 2019 median levels.
A binding arbitration process kicks in when a provider and a health plan fail to agree about how much the plan will pay for a service. Then, a third-party arbitrator is called in to make a ruling that’s binding. The process is controversial, and a flurry of lawsuits from providers have challenged it.
The new report, which updates an earlier analysis, examines data about disputed cases from all of 2023.
Of the 657,040 new cases filed in 2023, about 70% came from four private equity-funded organizations: Team Health, SCP Health, Radiology Partners, and Envision, which each provide physician services.
About half of the 2023 cases were from just four states: Texas, Florida, Tennessee, and Georgia. The report says the four organizations are especially active in those states. In contrast, Connecticut, Maryland, Massachusetts, and Washington state each had just 1500 or fewer cases filed last year.
Health plans challenged a third of cases as ineligible, and 22% of all resolved cases were deemed ineligible.
Providers won 80% of resolved challenges in 2023, although it’s not clear how much money they reaped. Still, it’s clear that “in the vast majority of the cases, insurers have to pay larger amounts to the provider,” Dr. Hoadley said.
Radiologists made a median of at least 500% of the in-network rate in their cases. Surgeons and neurologists made even more money — a median of at least 800% of the in-network rate. Overall, providers made 322%-350% of in-network rates, depending on the quarter.
Dr. Hoadley cautioned that only a small percentage of medical payments are disputed. In those cases, “the amount that the insurer offers is accepted, and that’s the end of the story.”
Why are the providers often reaping much more than typical payments for in-network services? It’s “really hard to know,” Dr. Hoadley said. But one factor, he said, may be the fact that providers are able to offer evidence challenging that amounts that insurers say they paid previously: “Hey, when we were in network, we were paid this much.”
It’s not clear whether the dispute-and-arbitration system will cost insurers — and patients — more in the long run. The Congressional Budget Office actually thought the No Surprises Act might lower the growth of premiums slightly and save the federal government money, Dr. Hoadley said, but that could potentially not happen. The flood of litigation also contributes to uncertainty, he said.
Alan Sager, PhD, professor of Health Law, Policy, and Management at Boston University School of Public Health, told this news organization that premiums are bound to rise as insurers react to higher costs. He also expects that providers will question the value of being in-network. “If you’re out-of-network and can obtain much higher payments, why would any doctor or hospital remain in-network, especially since they don’t lose out on patient volume?”
Why are provider groups owned by private equity firms so aggressive at challenging health plans? Loren Adler, a fellow and associate director of the Brookings Institution’s Center on Health Policy, told this news organization that these companies play large roles in fields affected by the No Surprises Act. These include emergency medicine, radiology, and anesthesiology, said Mr. Adler, who’s also studied the No Surprises Act’s dispute/arbitration system.
Mr. Adler added that larger companies “are better suited to deal with technical complexities of this process and spend the sort of upfront money to go through it.”
In the big picture, Mr. Adler said, the new study “raises question of whether Congress at some point wants to try to basically bring prices from the arbitration process back in line with average in-network prices.”
The study was funded by the Commonwealth Fund and Arnold Ventures. Dr. Hoadley, Dr. Sager, and Mr. Adler had no disclosures.
A version of this article first appeared on Medscape.com.
Four organizations owned by private equity firms — including two provider groups — dominated the No Surprises Act’s disputed bill arbitration process in its first year, filing about 70% of 657,040 cases against insurers in 2023, a new report finds.
The findings, recently published in Health Affairs, suggest that private equity–owned organizations are forcefully challenging insurers about payments for certain kinds of out-of-network care.
Their fighting stance has paid off: The percentage of resolved arbitration cases won by providers jumped from 72% in the first quarter of 2023 to 85% in the last quarter, and they were awarded a median of more than 300% the contracted in-network rates for the services in question.
With many more out-of-network bills disputed by providers than expected, “the system is not working exactly the way it was anticipated when this law was written,” lead author Jack Hoadley, PhD, a research professor emeritus at Georgetown University’s McCourt School of Public Policy, Washington, DC, told this news organization.
And, he said, the public and the federal government may end up paying a price.
Congress passed the No Surprises Act in 2020 and then-President Donald Trump signed it. The landmark bill, which went into effect in 2022, was designed to protect patients from unexpected and often exorbitant “surprise” bills after they received some kinds of out-of-network care.
Now, many types of providers are forbidden from billing patients beyond normal in-network costs. In these cases, health plans and out-of-network providers — who don’t have mutual agreements — must wrangle over payment amounts, which are intended to not exceed inflation-adjusted 2019 median levels.
A binding arbitration process kicks in when a provider and a health plan fail to agree about how much the plan will pay for a service. Then, a third-party arbitrator is called in to make a ruling that’s binding. The process is controversial, and a flurry of lawsuits from providers have challenged it.
The new report, which updates an earlier analysis, examines data about disputed cases from all of 2023.
Of the 657,040 new cases filed in 2023, about 70% came from four private equity-funded organizations: Team Health, SCP Health, Radiology Partners, and Envision, which each provide physician services.
About half of the 2023 cases were from just four states: Texas, Florida, Tennessee, and Georgia. The report says the four organizations are especially active in those states. In contrast, Connecticut, Maryland, Massachusetts, and Washington state each had just 1500 or fewer cases filed last year.
Health plans challenged a third of cases as ineligible, and 22% of all resolved cases were deemed ineligible.
Providers won 80% of resolved challenges in 2023, although it’s not clear how much money they reaped. Still, it’s clear that “in the vast majority of the cases, insurers have to pay larger amounts to the provider,” Dr. Hoadley said.
Radiologists made a median of at least 500% of the in-network rate in their cases. Surgeons and neurologists made even more money — a median of at least 800% of the in-network rate. Overall, providers made 322%-350% of in-network rates, depending on the quarter.
Dr. Hoadley cautioned that only a small percentage of medical payments are disputed. In those cases, “the amount that the insurer offers is accepted, and that’s the end of the story.”
Why are the providers often reaping much more than typical payments for in-network services? It’s “really hard to know,” Dr. Hoadley said. But one factor, he said, may be the fact that providers are able to offer evidence challenging that amounts that insurers say they paid previously: “Hey, when we were in network, we were paid this much.”
It’s not clear whether the dispute-and-arbitration system will cost insurers — and patients — more in the long run. The Congressional Budget Office actually thought the No Surprises Act might lower the growth of premiums slightly and save the federal government money, Dr. Hoadley said, but that could potentially not happen. The flood of litigation also contributes to uncertainty, he said.
Alan Sager, PhD, professor of Health Law, Policy, and Management at Boston University School of Public Health, told this news organization that premiums are bound to rise as insurers react to higher costs. He also expects that providers will question the value of being in-network. “If you’re out-of-network and can obtain much higher payments, why would any doctor or hospital remain in-network, especially since they don’t lose out on patient volume?”
Why are provider groups owned by private equity firms so aggressive at challenging health plans? Loren Adler, a fellow and associate director of the Brookings Institution’s Center on Health Policy, told this news organization that these companies play large roles in fields affected by the No Surprises Act. These include emergency medicine, radiology, and anesthesiology, said Mr. Adler, who’s also studied the No Surprises Act’s dispute/arbitration system.
Mr. Adler added that larger companies “are better suited to deal with technical complexities of this process and spend the sort of upfront money to go through it.”
In the big picture, Mr. Adler said, the new study “raises question of whether Congress at some point wants to try to basically bring prices from the arbitration process back in line with average in-network prices.”
The study was funded by the Commonwealth Fund and Arnold Ventures. Dr. Hoadley, Dr. Sager, and Mr. Adler had no disclosures.
A version of this article first appeared on Medscape.com.