The randomized, placebo-controlled CLEAR Outcomes trial has shown a significant reduction in risk for a composite cardiovascular (CV) endpoint among its patients treated with the lipid-lowering agent bempedoic acid (Nexletol), the drug’s owner, Esperion, announced today.

The trial marks the first time an ATP-citrate lyase inhibitor has shown significant and “clinically meaningful” benefit for patients not adequately managed with standard lipid-modifying agents, Esperion president and CEO Sheldon Koenig said in a press release.

The brief statement provided only top-line results, without P values or other evidence of the magnitude of benefit in the active-therapy group. The company expects to present more complete results “at a key medical conference in the first quarter of 2023.”

CLEAR Outcomes had entered 14,014 patients with a history of or at high risk for CV disease events, elevated low-density lipoprotein cholesterol (LDL-C) levels, and demonstrated intolerance to at least two statins.

They were randomly assigned to bempedoic acid 180 mg once daily or placebo and followed for the primary endpoint of CV death, nonfatal myocardial infarction, nonfatal stroke, or coronary revascularization. The trial, conducted in 32 countries, launched in December 2016.  

Bempedoic acid is currently approved for adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease on maximally tolerated statins who require additional LDL-C lowering, the company states.

Concomitant use of bempedoic acid with simvastatin or pravastatin, the press release says, may lead to increased statin concentrations and risk for “simvastatin- or pravastatin-related myopathy.” Therefore, “use with greater than 20 mg of simvastatin or 40 mg of pravastatin should be avoided.”

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

The randomized, placebo-controlled CLEAR Outcomes trial has shown a significant reduction in risk for a composite cardiovascular (CV) endpoint among its patients treated with the lipid-lowering agent bempedoic acid (Nexletol), the drug’s owner, Esperion, announced today.

The trial marks the first time an ATP-citrate lyase inhibitor has shown significant and “clinically meaningful” benefit for patients not adequately managed with standard lipid-modifying agents, Esperion president and CEO Sheldon Koenig said in a press release.

The brief statement provided only top-line results, without P values or other evidence of the magnitude of benefit in the active-therapy group. The company expects to present more complete results “at a key medical conference in the first quarter of 2023.”

CLEAR Outcomes had entered 14,014 patients with a history of or at high risk for CV disease events, elevated low-density lipoprotein cholesterol (LDL-C) levels, and demonstrated intolerance to at least two statins.

They were randomly assigned to bempedoic acid 180 mg once daily or placebo and followed for the primary endpoint of CV death, nonfatal myocardial infarction, nonfatal stroke, or coronary revascularization. The trial, conducted in 32 countries, launched in December 2016.  

Bempedoic acid is currently approved for adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease on maximally tolerated statins who require additional LDL-C lowering, the company states.

Concomitant use of bempedoic acid with simvastatin or pravastatin, the press release says, may lead to increased statin concentrations and risk for “simvastatin- or pravastatin-related myopathy.” Therefore, “use with greater than 20 mg of simvastatin or 40 mg of pravastatin should be avoided.”

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

The randomized, placebo-controlled CLEAR Outcomes trial has shown a significant reduction in risk for a composite cardiovascular (CV) endpoint among its patients treated with the lipid-lowering agent bempedoic acid (Nexletol), the drug’s owner, Esperion, announced today.

The trial marks the first time an ATP-citrate lyase inhibitor has shown significant and “clinically meaningful” benefit for patients not adequately managed with standard lipid-modifying agents, Esperion president and CEO Sheldon Koenig said in a press release.

The brief statement provided only top-line results, without P values or other evidence of the magnitude of benefit in the active-therapy group. The company expects to present more complete results “at a key medical conference in the first quarter of 2023.”

CLEAR Outcomes had entered 14,014 patients with a history of or at high risk for CV disease events, elevated low-density lipoprotein cholesterol (LDL-C) levels, and demonstrated intolerance to at least two statins.

They were randomly assigned to bempedoic acid 180 mg once daily or placebo and followed for the primary endpoint of CV death, nonfatal myocardial infarction, nonfatal stroke, or coronary revascularization. The trial, conducted in 32 countries, launched in December 2016.  

Bempedoic acid is currently approved for adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease on maximally tolerated statins who require additional LDL-C lowering, the company states.

Concomitant use of bempedoic acid with simvastatin or pravastatin, the press release says, may lead to increased statin concentrations and risk for “simvastatin- or pravastatin-related myopathy.” Therefore, “use with greater than 20 mg of simvastatin or 40 mg of pravastatin should be avoided.”

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

When a secret shopper used telemedicine to request testosterone supplements, options were plentiful but good advice was scarce. As researchers showed, companies offered testosterone replacement therapy to the prospective buyer even though his stated T level was much higher than the cut-off for low testosterone levels.

Direct-to-consumer (DTC) delivery of testosterone or medications for erectile dysfunction has become routine. The many benefits of wide access include convenience and the ability to discuss sensitive topics in a safe environment. But as the new findings indicate, a lack of adherence to solid medical advice – such as guidance from the Endocrine Society and the American Urological Association – may be putting some people at risk for potentially harmful outcomes.

The Endocrine Society and the AUA state that only people with true testosterone deficiency should receive a T boost. Both groups discourage men who are planning to become parents in the near future from taking supplemental testosterone because of possible harms to their fertility.

“When guidelines are not being followed, there’s always the potential that we might not get the best outcomes for patients,” said Joshua A. Halpern, MD, a urologist at the Northwestern University Feinberg School of Medicine, Chicago, who led the study, which was published in JAMA Internal Medicine.

To conduct the research, the secret shopper approached seven different DTC testosterone websites with the same story for each: He was a 34-year-old man with low energy and libido who hoped to become a father soon. The customer noted that he had a testosterone level of 675 ng/dL, well above the 300 ng/dL the AUA considers low.

Despite these red flags – the normal T levels, the parenthood aspirations – representatives of almost every platform moved the shopper along toward receiving additional testosterone, with no attention to the possible harms to fertility. Only one platform declined to offer the testosterone because the shopper’s T levels were sufficient. In the other cases the secret shopper did not go forward with obtaining the medication, which would have required a prescription.

“Our goal with this study was to achieve a better understanding of what patients are experiencing and what’s out there,” Dr. Halpern said. While this study focused on cisgender patients, Dr. Halpern noted that it’s also important to understand the experiences of transgender patients who seek DTC hormonal therapy.

The research could help keep DTC companies more honest, according to Jesse N. Mills, MD, a urologist and men’s health specialist at the University of California, Los Angeles, who was not involved in the work. DTC platforms are financially incentivized to dispense medications regardless of need, unlike a traditional doctor who generally has no personal financial stake in a prescription.

“We need to keep the heat on DTC platforms,” Dr. Mills said, calling the article a “punchback” against the current DTC model for testosterone products. Dr. Mills said he is not opposed to telemedicine or DTC practices in general, adding that UCLA made a successful pivot to telemedicine during the pandemic.

“You can set up a lot of good care through video visits,” Dr. Mills said, as long as the system is ethical.

Dr. Halpern and Dr. Mills reported no relevant financial relationships.

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

When a secret shopper used telemedicine to request testosterone supplements, options were plentiful but good advice was scarce. As researchers showed, companies offered testosterone replacement therapy to the prospective buyer even though his stated T level was much higher than the cut-off for low testosterone levels.

Direct-to-consumer (DTC) delivery of testosterone or medications for erectile dysfunction has become routine. The many benefits of wide access include convenience and the ability to discuss sensitive topics in a safe environment. But as the new findings indicate, a lack of adherence to solid medical advice – such as guidance from the Endocrine Society and the American Urological Association – may be putting some people at risk for potentially harmful outcomes.

The Endocrine Society and the AUA state that only people with true testosterone deficiency should receive a T boost. Both groups discourage men who are planning to become parents in the near future from taking supplemental testosterone because of possible harms to their fertility.

“When guidelines are not being followed, there’s always the potential that we might not get the best outcomes for patients,” said Joshua A. Halpern, MD, a urologist at the Northwestern University Feinberg School of Medicine, Chicago, who led the study, which was published in JAMA Internal Medicine.

To conduct the research, the secret shopper approached seven different DTC testosterone websites with the same story for each: He was a 34-year-old man with low energy and libido who hoped to become a father soon. The customer noted that he had a testosterone level of 675 ng/dL, well above the 300 ng/dL the AUA considers low.

Despite these red flags – the normal T levels, the parenthood aspirations – representatives of almost every platform moved the shopper along toward receiving additional testosterone, with no attention to the possible harms to fertility. Only one platform declined to offer the testosterone because the shopper’s T levels were sufficient. In the other cases the secret shopper did not go forward with obtaining the medication, which would have required a prescription.

“Our goal with this study was to achieve a better understanding of what patients are experiencing and what’s out there,” Dr. Halpern said. While this study focused on cisgender patients, Dr. Halpern noted that it’s also important to understand the experiences of transgender patients who seek DTC hormonal therapy.

The research could help keep DTC companies more honest, according to Jesse N. Mills, MD, a urologist and men’s health specialist at the University of California, Los Angeles, who was not involved in the work. DTC platforms are financially incentivized to dispense medications regardless of need, unlike a traditional doctor who generally has no personal financial stake in a prescription.

“We need to keep the heat on DTC platforms,” Dr. Mills said, calling the article a “punchback” against the current DTC model for testosterone products. Dr. Mills said he is not opposed to telemedicine or DTC practices in general, adding that UCLA made a successful pivot to telemedicine during the pandemic.

“You can set up a lot of good care through video visits,” Dr. Mills said, as long as the system is ethical.

Dr. Halpern and Dr. Mills reported no relevant financial relationships.

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

When a secret shopper used telemedicine to request testosterone supplements, options were plentiful but good advice was scarce. As researchers showed, companies offered testosterone replacement therapy to the prospective buyer even though his stated T level was much higher than the cut-off for low testosterone levels.

Direct-to-consumer (DTC) delivery of testosterone or medications for erectile dysfunction has become routine. The many benefits of wide access include convenience and the ability to discuss sensitive topics in a safe environment. But as the new findings indicate, a lack of adherence to solid medical advice – such as guidance from the Endocrine Society and the American Urological Association – may be putting some people at risk for potentially harmful outcomes.

The Endocrine Society and the AUA state that only people with true testosterone deficiency should receive a T boost. Both groups discourage men who are planning to become parents in the near future from taking supplemental testosterone because of possible harms to their fertility.

“When guidelines are not being followed, there’s always the potential that we might not get the best outcomes for patients,” said Joshua A. Halpern, MD, a urologist at the Northwestern University Feinberg School of Medicine, Chicago, who led the study, which was published in JAMA Internal Medicine.

To conduct the research, the secret shopper approached seven different DTC testosterone websites with the same story for each: He was a 34-year-old man with low energy and libido who hoped to become a father soon. The customer noted that he had a testosterone level of 675 ng/dL, well above the 300 ng/dL the AUA considers low.

Despite these red flags – the normal T levels, the parenthood aspirations – representatives of almost every platform moved the shopper along toward receiving additional testosterone, with no attention to the possible harms to fertility. Only one platform declined to offer the testosterone because the shopper’s T levels were sufficient. In the other cases the secret shopper did not go forward with obtaining the medication, which would have required a prescription.

“Our goal with this study was to achieve a better understanding of what patients are experiencing and what’s out there,” Dr. Halpern said. While this study focused on cisgender patients, Dr. Halpern noted that it’s also important to understand the experiences of transgender patients who seek DTC hormonal therapy.

The research could help keep DTC companies more honest, according to Jesse N. Mills, MD, a urologist and men’s health specialist at the University of California, Los Angeles, who was not involved in the work. DTC platforms are financially incentivized to dispense medications regardless of need, unlike a traditional doctor who generally has no personal financial stake in a prescription.

“We need to keep the heat on DTC platforms,” Dr. Mills said, calling the article a “punchback” against the current DTC model for testosterone products. Dr. Mills said he is not opposed to telemedicine or DTC practices in general, adding that UCLA made a successful pivot to telemedicine during the pandemic.

“You can set up a lot of good care through video visits,” Dr. Mills said, as long as the system is ethical.

Dr. Halpern and Dr. Mills reported no relevant financial relationships.

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

NEW ORLEANS – Results of a new study could presage an end to the standard use of autologous hematopoietic stem cell transplantation (HSCT) for treating younger patients with mantle cell lymphoma (MCL), a researcher told colleagues.

First-line patients fared well on ibrutinib, a Bruton’s tyrosine kinase inhibitor, according to the findings.

Courtesy ASH

“Based on the results so far ... at least for the majority of patients, ibrutinib early will become the new standard,” said study lead author Martin Dreyling, MD, associate professor of medicine at Ludwig Maximilian University (LMU) Munich. Dr. Dreyling spoke in a news briefing and at a separate presentation at the annual meeting of the American Society of Hematology. “It might well be that specific subsets of patients may benefit from autologous transplant.”

MCL is a rare form of non-Hodgkin’s lymphoma that strikes cells in the mantle zone of lymph nodes. It is usually diagnosed in older men and often presents at an advanced stage. Multiple available treatments include rituximab/bendamustine, CAR-T cell therapy, stem cell transplants, and Bruton’s tyrosine kinase inhibitors. Ibrutinib is approved by the Food and Drug Administration only for refractory/relapsed cases, however.

Dr. Dreyling was a pioneer in confirming benefit from stem-cell transplants for MCL. “However,” he said, “no one likes autologous transplant because it also has side effects.”

For the new open-label study, Dr. Dreyling and colleagues in the European MCL Network in 2016 began recruiting patients with newly diagnosed, advanced stage II-IV MCL. The patients were younger than 65.

The subjects were randomly assigned to three trial arms: Standard treatment (high-dose cytarabine followed by autologous stem cell transplant and rituximab maintenance, n = 288), the standard treatment plus ibrutinib (n = 292), and ibrutinib without stem cell transplant (n = 290). The median age was 57, and 76% of patients were male.

The primary endpoint was failure-free survival at 31 months. Standard therapy was not superior to the ibrutinib without transplant group (72% vs. 86%, respectively, P = .9979). However, standard therapy with ibrutinib was superior to the standard therapy group (88% vs. 72%, respectively, P = .0008). The researchers haven’t finished their analysis of standard therapy with ibrutinib vs. ibrutinib without transplant.

Subjects in the standard therapy plus ibrutinib arm had more grade 3-5 adverse events than did the standard therapy and ibrutinib without transplant groups: Neutropenia, 44%, 17%, and 23%, respectively; leukopenia, 4%, 2%, and 2%; febrile neutropenia, 6%, 3%, and 3%; infections and infestations, 25%, 13%, and 19%; and cardiac disorders, 3%, 1%, 4%. P values were not provided.

In an interview, Ohio State University hematologist Narendranath Epperla, MD, MS, who was not involved in the study, said that this research reflects efforts to understand how novel agents such as ibrutinib and cellular therapies fit into MCL treatment. “We are trying to incorporate them in the frontline setting with either chemo backbone or with other targeted agents to improve outcomes and minimize toxicity. We are also trying to understand in whom auto-HCT can be precluded.”

The results of the new study appear promising, Dr. Epperla said, but he questioned the primary endpoint (failure-free survival instead of progress-free survival) and the short duration of the trial.

“I would like to see how the patients with high-risk features such as TP53 mutation, complex cytogenetics, and blastoid/pleomorphic variants did on the three arms,” Dr. Epperla said. “And I would like to see longer follow-up data before adapting this – [addition] of ibrutinib to the chemotherapy backbone without auto-HCT – into clinical practice.”

What’s next? Dr. Dreyling said that upcoming data will provide further insight into ibrutinib vs. stem-cell transplantation. And “within the next half year or so,” he said, “there will be a next generation of studies challenging chemotherapy overall in mantle cell lymphoma and substituting targeted treatment, hopefully achieving much better tolerability.”

Funding information was not provided. Dr. Dreyling disclosed ties with Lilly/Loxo, AstraZeneca, Novartis, Amgen, Roche, Janssen, Gilead/Kite, BMS/Celgene, Bayer, Abbvie, and Beigene. The other study authors reported various disclosures. Dr. Epperla disclosed a relationship with Pharmacyclics.

NEW ORLEANS – Results of a new study could presage an end to the standard use of autologous hematopoietic stem cell transplantation (HSCT) for treating younger patients with mantle cell lymphoma (MCL), a researcher told colleagues.

First-line patients fared well on ibrutinib, a Bruton’s tyrosine kinase inhibitor, according to the findings.

Courtesy ASH

“Based on the results so far ... at least for the majority of patients, ibrutinib early will become the new standard,” said study lead author Martin Dreyling, MD, associate professor of medicine at Ludwig Maximilian University (LMU) Munich. Dr. Dreyling spoke in a news briefing and at a separate presentation at the annual meeting of the American Society of Hematology. “It might well be that specific subsets of patients may benefit from autologous transplant.”

MCL is a rare form of non-Hodgkin’s lymphoma that strikes cells in the mantle zone of lymph nodes. It is usually diagnosed in older men and often presents at an advanced stage. Multiple available treatments include rituximab/bendamustine, CAR-T cell therapy, stem cell transplants, and Bruton’s tyrosine kinase inhibitors. Ibrutinib is approved by the Food and Drug Administration only for refractory/relapsed cases, however.

Dr. Dreyling was a pioneer in confirming benefit from stem-cell transplants for MCL. “However,” he said, “no one likes autologous transplant because it also has side effects.”

For the new open-label study, Dr. Dreyling and colleagues in the European MCL Network in 2016 began recruiting patients with newly diagnosed, advanced stage II-IV MCL. The patients were younger than 65.

The subjects were randomly assigned to three trial arms: Standard treatment (high-dose cytarabine followed by autologous stem cell transplant and rituximab maintenance, n = 288), the standard treatment plus ibrutinib (n = 292), and ibrutinib without stem cell transplant (n = 290). The median age was 57, and 76% of patients were male.

The primary endpoint was failure-free survival at 31 months. Standard therapy was not superior to the ibrutinib without transplant group (72% vs. 86%, respectively, P = .9979). However, standard therapy with ibrutinib was superior to the standard therapy group (88% vs. 72%, respectively, P = .0008). The researchers haven’t finished their analysis of standard therapy with ibrutinib vs. ibrutinib without transplant.

Subjects in the standard therapy plus ibrutinib arm had more grade 3-5 adverse events than did the standard therapy and ibrutinib without transplant groups: Neutropenia, 44%, 17%, and 23%, respectively; leukopenia, 4%, 2%, and 2%; febrile neutropenia, 6%, 3%, and 3%; infections and infestations, 25%, 13%, and 19%; and cardiac disorders, 3%, 1%, 4%. P values were not provided.

In an interview, Ohio State University hematologist Narendranath Epperla, MD, MS, who was not involved in the study, said that this research reflects efforts to understand how novel agents such as ibrutinib and cellular therapies fit into MCL treatment. “We are trying to incorporate them in the frontline setting with either chemo backbone or with other targeted agents to improve outcomes and minimize toxicity. We are also trying to understand in whom auto-HCT can be precluded.”

The results of the new study appear promising, Dr. Epperla said, but he questioned the primary endpoint (failure-free survival instead of progress-free survival) and the short duration of the trial.

“I would like to see how the patients with high-risk features such as TP53 mutation, complex cytogenetics, and blastoid/pleomorphic variants did on the three arms,” Dr. Epperla said. “And I would like to see longer follow-up data before adapting this – [addition] of ibrutinib to the chemotherapy backbone without auto-HCT – into clinical practice.”

What’s next? Dr. Dreyling said that upcoming data will provide further insight into ibrutinib vs. stem-cell transplantation. And “within the next half year or so,” he said, “there will be a next generation of studies challenging chemotherapy overall in mantle cell lymphoma and substituting targeted treatment, hopefully achieving much better tolerability.”

Funding information was not provided. Dr. Dreyling disclosed ties with Lilly/Loxo, AstraZeneca, Novartis, Amgen, Roche, Janssen, Gilead/Kite, BMS/Celgene, Bayer, Abbvie, and Beigene. The other study authors reported various disclosures. Dr. Epperla disclosed a relationship with Pharmacyclics.

NEW ORLEANS – Results of a new study could presage an end to the standard use of autologous hematopoietic stem cell transplantation (HSCT) for treating younger patients with mantle cell lymphoma (MCL), a researcher told colleagues.

First-line patients fared well on ibrutinib, a Bruton’s tyrosine kinase inhibitor, according to the findings.

Courtesy ASH

“Based on the results so far ... at least for the majority of patients, ibrutinib early will become the new standard,” said study lead author Martin Dreyling, MD, associate professor of medicine at Ludwig Maximilian University (LMU) Munich. Dr. Dreyling spoke in a news briefing and at a separate presentation at the annual meeting of the American Society of Hematology. “It might well be that specific subsets of patients may benefit from autologous transplant.”

MCL is a rare form of non-Hodgkin’s lymphoma that strikes cells in the mantle zone of lymph nodes. It is usually diagnosed in older men and often presents at an advanced stage. Multiple available treatments include rituximab/bendamustine, CAR-T cell therapy, stem cell transplants, and Bruton’s tyrosine kinase inhibitors. Ibrutinib is approved by the Food and Drug Administration only for refractory/relapsed cases, however.

Dr. Dreyling was a pioneer in confirming benefit from stem-cell transplants for MCL. “However,” he said, “no one likes autologous transplant because it also has side effects.”

For the new open-label study, Dr. Dreyling and colleagues in the European MCL Network in 2016 began recruiting patients with newly diagnosed, advanced stage II-IV MCL. The patients were younger than 65.

The subjects were randomly assigned to three trial arms: Standard treatment (high-dose cytarabine followed by autologous stem cell transplant and rituximab maintenance, n = 288), the standard treatment plus ibrutinib (n = 292), and ibrutinib without stem cell transplant (n = 290). The median age was 57, and 76% of patients were male.

The primary endpoint was failure-free survival at 31 months. Standard therapy was not superior to the ibrutinib without transplant group (72% vs. 86%, respectively, P = .9979). However, standard therapy with ibrutinib was superior to the standard therapy group (88% vs. 72%, respectively, P = .0008). The researchers haven’t finished their analysis of standard therapy with ibrutinib vs. ibrutinib without transplant.

Subjects in the standard therapy plus ibrutinib arm had more grade 3-5 adverse events than did the standard therapy and ibrutinib without transplant groups: Neutropenia, 44%, 17%, and 23%, respectively; leukopenia, 4%, 2%, and 2%; febrile neutropenia, 6%, 3%, and 3%; infections and infestations, 25%, 13%, and 19%; and cardiac disorders, 3%, 1%, 4%. P values were not provided.

In an interview, Ohio State University hematologist Narendranath Epperla, MD, MS, who was not involved in the study, said that this research reflects efforts to understand how novel agents such as ibrutinib and cellular therapies fit into MCL treatment. “We are trying to incorporate them in the frontline setting with either chemo backbone or with other targeted agents to improve outcomes and minimize toxicity. We are also trying to understand in whom auto-HCT can be precluded.”

The results of the new study appear promising, Dr. Epperla said, but he questioned the primary endpoint (failure-free survival instead of progress-free survival) and the short duration of the trial.

“I would like to see how the patients with high-risk features such as TP53 mutation, complex cytogenetics, and blastoid/pleomorphic variants did on the three arms,” Dr. Epperla said. “And I would like to see longer follow-up data before adapting this – [addition] of ibrutinib to the chemotherapy backbone without auto-HCT – into clinical practice.”

What’s next? Dr. Dreyling said that upcoming data will provide further insight into ibrutinib vs. stem-cell transplantation. And “within the next half year or so,” he said, “there will be a next generation of studies challenging chemotherapy overall in mantle cell lymphoma and substituting targeted treatment, hopefully achieving much better tolerability.”

Funding information was not provided. Dr. Dreyling disclosed ties with Lilly/Loxo, AstraZeneca, Novartis, Amgen, Roche, Janssen, Gilead/Kite, BMS/Celgene, Bayer, Abbvie, and Beigene. The other study authors reported various disclosures. Dr. Epperla disclosed a relationship with Pharmacyclics.

WASHINGTON – “We don’t get to use the ‘eliminate’ word all that often with a disease that’s taking thousands or tens of thousands – or worldwide, hundreds of thousands – of lives every year, but we have that opportunity with hepatitis C.”

So said Francis S. Collins, MD, PhD, special projects advisor to the Executive Office of the President of the United States, and former director of the National Institutes of Health, speaking at a special session outlining ambitious goals for a national plan to eliminate hepatitis C virus (HCV) infections by the year 2050.

The session was held at the annual meeting of the American Association for the Study of Liver Diseases.
 

A public health crisis

Dr. Collins labeled HCV a public health crisis, citing statistics from the Centers for Disease Control and Prevention that show that the rate of reported acute HCV infection cases increased 400% between 2010 and 2020, with the highest rates among young adults aged 20-39 years.

In addition, an estimated 2.4 million people in the United States are living with chronic HCV infections, but as many as 40% of these people are unaware of their infection, despite broad recommendations for the screening of all adults aged 18 years and older, he said.

“Our goal is to try to do something to change this,” Dr. Collins said. He noted that for the past 8 years we have had highly effective oral agents that don’t just treat the disease but cure it – 95%-97% of the time, with only 8-12 weeks of oral therapy and relatively few side effects.

“A wonderful story, one of the most exciting stories that’s come out of biomedical research in the last couple of decades,” he said.

Yet Dr. Collins also acknowledged that the task of developing a national plan is daunting, despite that pharmaceutical triumph.

National pharmacy claims data show that the number of persons treated for HCV with direct-acting antiviral agents (DAAs) in the United States declined from a high of 164,247 in 2015 to 83,740 in 2020.

Furthermore, CDC data from 2019 and 2020 show that, of persons with a diagnosis of HCV infection, only 23% of those on Medicaid, 28% of those on Medicare, and 35% of those with private insurance were treated for their infections.

“We have a huge gap here between the ability to know you have the disease and to get treatment, and we don’t see the numbers here for the uninsured, or people in prisons, but they’re probably much worse,” he said.
 

Obstacles abound, as do ways to overcome them

Current barriers to treatment include the aforementioned lack of awareness of infection, a “clunky” two-step diagnosis requiring an antibody test followed by an RNA or core antigen test necessitating three visits often separated by several weeks, and the high cost of treatment (around $90,000 per patient).

In addition, insurers commonly require proof that patients remain sober for extended periods, insist that treatment monitoring be performed by specialists only, and often approve treatment only for those patients who have documented evidence of liver damage.

“Does that make sense to you?” Dr. Collins asked. “You’ve got a cure for a liver disease, and you have to wait and show that the liver’s been damaged before you receive it? That just doesn’t fit,” he said.

Dr. Collins also pointed out that we’re dealing with hard-to-reach populations (underserved, uninsured, justice-involved), and people who are in tough times. “Anything that you put in the way as a barrier is going to make this worse in terms of its ability to be implemented,” he said.

To demonstrate how a coordinated HCV-elimination program could work, Dr. Collins pointed to a Medicaid cohort study in Louisiana conducted from July 2019 through December 2021, in which 8,867 patients started on therapy, 7,763 (88%) completed therapy, and 5,882 (66%) returned for testing. Of those tested, 5,285 (90%) had sustained virologic responses.

Another model of a hepatitis C elimination program was provided by the Veterans Health Administration. They received funding for an effort for all veterans, and in the space of 7 years were able to reach out even to some of their difficult-to-reach populations and achieve high diagnosis and treatment rates in a way that could be a model for what we would want to do across the nation, Dr. Collins noted.
 

Doing the math

Also at the session, Jagpreet Chhatwal, PhD, director of the Massachusetts General Hospital Institute for Technology Assessment and associate professor of radiology at Harvard Medical School, Boston, described outcomes projected by a mathematical simulation model of the HCV epidemic that he and his colleagues developed.

The HEP-SIM (Hepatitis C Disease Burden Simulation) model evaluates HCV prevalence trends, the number needed to screen and treat to eliminate HCV, HCV-associated clinical outcomes, the cost of an elimination program, and the cost savings that could be realized from preventing long-term complications.

The model seeks to determine whether the upfront costs of a national HCV elimination program could be offset by savings down the road. Specifically, it assumes that within the next 5 years 1.31 million individuals would be diagnosed with HCV and projects that within that time frame 1.52 million would need to be treated to meet HCV elimination goals.

The model shows that, compared with the status quo, a concerted campaign of screening and treatment would prevent more than 10,000 HCV-related deaths by 2030, and 91,000 deaths by 2050.

A coordinated screening program is also projected to prevent 17,000 cases of hepatocellular carcinoma by 2030 and 108,000 cases by 2050, as well as avert 29,000 cases of decompensated cirrhosis by 2030 and 93,000 such cases by 2050.

The cost savings associated with an HCV elimination plan would also be substantial, Dr. Chhatwal said.

According to the model, over the next decade the cumulative costs associated with HCV would decline by $14.2 billion, compared with the status quo. Nearly 80% of those savings ($11.2 billion) would be in Medicare and Medicaid.

The total projected savings from 2024 through 2050 – in disease management, testing, treatment, and pragmatic costs – are estimated at $59.3 billion, Dr. Chhatwal said.

“This is unprecedented,” he said. “We’re not just eliminating a disease as a public health threat but also saving money, which is not a common thing. That gives us a lot of impetus to implement such a program.”
 

Getting it done

Rachael L. Fleurence, PhD, MSc, a health economist currently serving as a senior advisor in the Executive Office of the President, summarized efforts to build a national HCV elimination program with input from federal health care agencies, state health leaders, patients, advocacy groups, drug manufacturers, and insurers.

She noted that a large component and focus of the program will be working on diagnostic test development but also accelerating bringing tests into the United States that are currently unavailable here. “These include point-of-care RNA diagnostic tests, as well as core antigen laboratory tests,” she said.

The program will be designed to offer broad access to curative anti-HCV drugs through a national subscription model that would make DAAs available to Medicaid recipients, justice-involved populations, the uninsured, and American Indians and Alaskan Natives who receive care through the Indian Health Service.

“On the Medicare and commercial insurance fronts, we’re still exploring different approaches, including potentially a co-pay assistance for Medicare beneficiaries, as well as working with commercial insurers to reduce barriers to access,” she said.

The program would also involve screening strategies extending to more settings, especially for high-risk populations, expanding the number of providers allowed to screen and treat HCV infections through telehealth, ensuring incentives for providers, and increasing the number of community health workers and case workers to improve linkage to care.

The next steps for the program would include funding to support the NIH’s RADx diagnostics program to accelerate access to testing, planning for the subscription model for DAA purchase, and launching pilot programs with the CDC, the Health Resources and Services Administration, the Substance Abuse and Mental Health Services Administration, and the Indian Health Service.
 

A call to action

Dr. Collins ended this portion of the program with an exhortation to AASLD members to do their part.

“We need your help,” Dr. Collins said. “This is a bold initiative, but it’s an opportunity. It’s even a responsibility. If we can actually succeed at this kind of outreach and save lives, and at the same time save money, how can we not do that?”

Dr. Collins, Dr. Chhatwal, and Dr. Fleurence each reported having no financial conflicts.

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

WASHINGTON – “We don’t get to use the ‘eliminate’ word all that often with a disease that’s taking thousands or tens of thousands – or worldwide, hundreds of thousands – of lives every year, but we have that opportunity with hepatitis C.”

So said Francis S. Collins, MD, PhD, special projects advisor to the Executive Office of the President of the United States, and former director of the National Institutes of Health, speaking at a special session outlining ambitious goals for a national plan to eliminate hepatitis C virus (HCV) infections by the year 2050.

The session was held at the annual meeting of the American Association for the Study of Liver Diseases.
 

A public health crisis

Dr. Collins labeled HCV a public health crisis, citing statistics from the Centers for Disease Control and Prevention that show that the rate of reported acute HCV infection cases increased 400% between 2010 and 2020, with the highest rates among young adults aged 20-39 years.

In addition, an estimated 2.4 million people in the United States are living with chronic HCV infections, but as many as 40% of these people are unaware of their infection, despite broad recommendations for the screening of all adults aged 18 years and older, he said.

“Our goal is to try to do something to change this,” Dr. Collins said. He noted that for the past 8 years we have had highly effective oral agents that don’t just treat the disease but cure it – 95%-97% of the time, with only 8-12 weeks of oral therapy and relatively few side effects.

“A wonderful story, one of the most exciting stories that’s come out of biomedical research in the last couple of decades,” he said.

Yet Dr. Collins also acknowledged that the task of developing a national plan is daunting, despite that pharmaceutical triumph.

National pharmacy claims data show that the number of persons treated for HCV with direct-acting antiviral agents (DAAs) in the United States declined from a high of 164,247 in 2015 to 83,740 in 2020.

Furthermore, CDC data from 2019 and 2020 show that, of persons with a diagnosis of HCV infection, only 23% of those on Medicaid, 28% of those on Medicare, and 35% of those with private insurance were treated for their infections.

“We have a huge gap here between the ability to know you have the disease and to get treatment, and we don’t see the numbers here for the uninsured, or people in prisons, but they’re probably much worse,” he said.
 

Obstacles abound, as do ways to overcome them

Current barriers to treatment include the aforementioned lack of awareness of infection, a “clunky” two-step diagnosis requiring an antibody test followed by an RNA or core antigen test necessitating three visits often separated by several weeks, and the high cost of treatment (around $90,000 per patient).

In addition, insurers commonly require proof that patients remain sober for extended periods, insist that treatment monitoring be performed by specialists only, and often approve treatment only for those patients who have documented evidence of liver damage.

“Does that make sense to you?” Dr. Collins asked. “You’ve got a cure for a liver disease, and you have to wait and show that the liver’s been damaged before you receive it? That just doesn’t fit,” he said.

Dr. Collins also pointed out that we’re dealing with hard-to-reach populations (underserved, uninsured, justice-involved), and people who are in tough times. “Anything that you put in the way as a barrier is going to make this worse in terms of its ability to be implemented,” he said.

To demonstrate how a coordinated HCV-elimination program could work, Dr. Collins pointed to a Medicaid cohort study in Louisiana conducted from July 2019 through December 2021, in which 8,867 patients started on therapy, 7,763 (88%) completed therapy, and 5,882 (66%) returned for testing. Of those tested, 5,285 (90%) had sustained virologic responses.

Another model of a hepatitis C elimination program was provided by the Veterans Health Administration. They received funding for an effort for all veterans, and in the space of 7 years were able to reach out even to some of their difficult-to-reach populations and achieve high diagnosis and treatment rates in a way that could be a model for what we would want to do across the nation, Dr. Collins noted.
 

Doing the math

Also at the session, Jagpreet Chhatwal, PhD, director of the Massachusetts General Hospital Institute for Technology Assessment and associate professor of radiology at Harvard Medical School, Boston, described outcomes projected by a mathematical simulation model of the HCV epidemic that he and his colleagues developed.

The HEP-SIM (Hepatitis C Disease Burden Simulation) model evaluates HCV prevalence trends, the number needed to screen and treat to eliminate HCV, HCV-associated clinical outcomes, the cost of an elimination program, and the cost savings that could be realized from preventing long-term complications.

The model seeks to determine whether the upfront costs of a national HCV elimination program could be offset by savings down the road. Specifically, it assumes that within the next 5 years 1.31 million individuals would be diagnosed with HCV and projects that within that time frame 1.52 million would need to be treated to meet HCV elimination goals.

The model shows that, compared with the status quo, a concerted campaign of screening and treatment would prevent more than 10,000 HCV-related deaths by 2030, and 91,000 deaths by 2050.

A coordinated screening program is also projected to prevent 17,000 cases of hepatocellular carcinoma by 2030 and 108,000 cases by 2050, as well as avert 29,000 cases of decompensated cirrhosis by 2030 and 93,000 such cases by 2050.

The cost savings associated with an HCV elimination plan would also be substantial, Dr. Chhatwal said.

According to the model, over the next decade the cumulative costs associated with HCV would decline by $14.2 billion, compared with the status quo. Nearly 80% of those savings ($11.2 billion) would be in Medicare and Medicaid.

The total projected savings from 2024 through 2050 – in disease management, testing, treatment, and pragmatic costs – are estimated at $59.3 billion, Dr. Chhatwal said.

“This is unprecedented,” he said. “We’re not just eliminating a disease as a public health threat but also saving money, which is not a common thing. That gives us a lot of impetus to implement such a program.”
 

Getting it done

Rachael L. Fleurence, PhD, MSc, a health economist currently serving as a senior advisor in the Executive Office of the President, summarized efforts to build a national HCV elimination program with input from federal health care agencies, state health leaders, patients, advocacy groups, drug manufacturers, and insurers.

She noted that a large component and focus of the program will be working on diagnostic test development but also accelerating bringing tests into the United States that are currently unavailable here. “These include point-of-care RNA diagnostic tests, as well as core antigen laboratory tests,” she said.

The program will be designed to offer broad access to curative anti-HCV drugs through a national subscription model that would make DAAs available to Medicaid recipients, justice-involved populations, the uninsured, and American Indians and Alaskan Natives who receive care through the Indian Health Service.

“On the Medicare and commercial insurance fronts, we’re still exploring different approaches, including potentially a co-pay assistance for Medicare beneficiaries, as well as working with commercial insurers to reduce barriers to access,” she said.

The program would also involve screening strategies extending to more settings, especially for high-risk populations, expanding the number of providers allowed to screen and treat HCV infections through telehealth, ensuring incentives for providers, and increasing the number of community health workers and case workers to improve linkage to care.

The next steps for the program would include funding to support the NIH’s RADx diagnostics program to accelerate access to testing, planning for the subscription model for DAA purchase, and launching pilot programs with the CDC, the Health Resources and Services Administration, the Substance Abuse and Mental Health Services Administration, and the Indian Health Service.
 

A call to action

Dr. Collins ended this portion of the program with an exhortation to AASLD members to do their part.

“We need your help,” Dr. Collins said. “This is a bold initiative, but it’s an opportunity. It’s even a responsibility. If we can actually succeed at this kind of outreach and save lives, and at the same time save money, how can we not do that?”

Dr. Collins, Dr. Chhatwal, and Dr. Fleurence each reported having no financial conflicts.

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

WASHINGTON – “We don’t get to use the ‘eliminate’ word all that often with a disease that’s taking thousands or tens of thousands – or worldwide, hundreds of thousands – of lives every year, but we have that opportunity with hepatitis C.”

So said Francis S. Collins, MD, PhD, special projects advisor to the Executive Office of the President of the United States, and former director of the National Institutes of Health, speaking at a special session outlining ambitious goals for a national plan to eliminate hepatitis C virus (HCV) infections by the year 2050.

The session was held at the annual meeting of the American Association for the Study of Liver Diseases.
 

A public health crisis

Dr. Collins labeled HCV a public health crisis, citing statistics from the Centers for Disease Control and Prevention that show that the rate of reported acute HCV infection cases increased 400% between 2010 and 2020, with the highest rates among young adults aged 20-39 years.

In addition, an estimated 2.4 million people in the United States are living with chronic HCV infections, but as many as 40% of these people are unaware of their infection, despite broad recommendations for the screening of all adults aged 18 years and older, he said.

“Our goal is to try to do something to change this,” Dr. Collins said. He noted that for the past 8 years we have had highly effective oral agents that don’t just treat the disease but cure it – 95%-97% of the time, with only 8-12 weeks of oral therapy and relatively few side effects.

“A wonderful story, one of the most exciting stories that’s come out of biomedical research in the last couple of decades,” he said.

Yet Dr. Collins also acknowledged that the task of developing a national plan is daunting, despite that pharmaceutical triumph.

National pharmacy claims data show that the number of persons treated for HCV with direct-acting antiviral agents (DAAs) in the United States declined from a high of 164,247 in 2015 to 83,740 in 2020.

Furthermore, CDC data from 2019 and 2020 show that, of persons with a diagnosis of HCV infection, only 23% of those on Medicaid, 28% of those on Medicare, and 35% of those with private insurance were treated for their infections.

“We have a huge gap here between the ability to know you have the disease and to get treatment, and we don’t see the numbers here for the uninsured, or people in prisons, but they’re probably much worse,” he said.
 

Obstacles abound, as do ways to overcome them

Current barriers to treatment include the aforementioned lack of awareness of infection, a “clunky” two-step diagnosis requiring an antibody test followed by an RNA or core antigen test necessitating three visits often separated by several weeks, and the high cost of treatment (around $90,000 per patient).

In addition, insurers commonly require proof that patients remain sober for extended periods, insist that treatment monitoring be performed by specialists only, and often approve treatment only for those patients who have documented evidence of liver damage.

“Does that make sense to you?” Dr. Collins asked. “You’ve got a cure for a liver disease, and you have to wait and show that the liver’s been damaged before you receive it? That just doesn’t fit,” he said.

Dr. Collins also pointed out that we’re dealing with hard-to-reach populations (underserved, uninsured, justice-involved), and people who are in tough times. “Anything that you put in the way as a barrier is going to make this worse in terms of its ability to be implemented,” he said.

To demonstrate how a coordinated HCV-elimination program could work, Dr. Collins pointed to a Medicaid cohort study in Louisiana conducted from July 2019 through December 2021, in which 8,867 patients started on therapy, 7,763 (88%) completed therapy, and 5,882 (66%) returned for testing. Of those tested, 5,285 (90%) had sustained virologic responses.

Another model of a hepatitis C elimination program was provided by the Veterans Health Administration. They received funding for an effort for all veterans, and in the space of 7 years were able to reach out even to some of their difficult-to-reach populations and achieve high diagnosis and treatment rates in a way that could be a model for what we would want to do across the nation, Dr. Collins noted.
 

Doing the math

Also at the session, Jagpreet Chhatwal, PhD, director of the Massachusetts General Hospital Institute for Technology Assessment and associate professor of radiology at Harvard Medical School, Boston, described outcomes projected by a mathematical simulation model of the HCV epidemic that he and his colleagues developed.

The HEP-SIM (Hepatitis C Disease Burden Simulation) model evaluates HCV prevalence trends, the number needed to screen and treat to eliminate HCV, HCV-associated clinical outcomes, the cost of an elimination program, and the cost savings that could be realized from preventing long-term complications.

The model seeks to determine whether the upfront costs of a national HCV elimination program could be offset by savings down the road. Specifically, it assumes that within the next 5 years 1.31 million individuals would be diagnosed with HCV and projects that within that time frame 1.52 million would need to be treated to meet HCV elimination goals.

The model shows that, compared with the status quo, a concerted campaign of screening and treatment would prevent more than 10,000 HCV-related deaths by 2030, and 91,000 deaths by 2050.

A coordinated screening program is also projected to prevent 17,000 cases of hepatocellular carcinoma by 2030 and 108,000 cases by 2050, as well as avert 29,000 cases of decompensated cirrhosis by 2030 and 93,000 such cases by 2050.

The cost savings associated with an HCV elimination plan would also be substantial, Dr. Chhatwal said.

According to the model, over the next decade the cumulative costs associated with HCV would decline by $14.2 billion, compared with the status quo. Nearly 80% of those savings ($11.2 billion) would be in Medicare and Medicaid.

The total projected savings from 2024 through 2050 – in disease management, testing, treatment, and pragmatic costs – are estimated at $59.3 billion, Dr. Chhatwal said.

“This is unprecedented,” he said. “We’re not just eliminating a disease as a public health threat but also saving money, which is not a common thing. That gives us a lot of impetus to implement such a program.”
 

Getting it done

Rachael L. Fleurence, PhD, MSc, a health economist currently serving as a senior advisor in the Executive Office of the President, summarized efforts to build a national HCV elimination program with input from federal health care agencies, state health leaders, patients, advocacy groups, drug manufacturers, and insurers.

She noted that a large component and focus of the program will be working on diagnostic test development but also accelerating bringing tests into the United States that are currently unavailable here. “These include point-of-care RNA diagnostic tests, as well as core antigen laboratory tests,” she said.

The program will be designed to offer broad access to curative anti-HCV drugs through a national subscription model that would make DAAs available to Medicaid recipients, justice-involved populations, the uninsured, and American Indians and Alaskan Natives who receive care through the Indian Health Service.

“On the Medicare and commercial insurance fronts, we’re still exploring different approaches, including potentially a co-pay assistance for Medicare beneficiaries, as well as working with commercial insurers to reduce barriers to access,” she said.

The program would also involve screening strategies extending to more settings, especially for high-risk populations, expanding the number of providers allowed to screen and treat HCV infections through telehealth, ensuring incentives for providers, and increasing the number of community health workers and case workers to improve linkage to care.

The next steps for the program would include funding to support the NIH’s RADx diagnostics program to accelerate access to testing, planning for the subscription model for DAA purchase, and launching pilot programs with the CDC, the Health Resources and Services Administration, the Substance Abuse and Mental Health Services Administration, and the Indian Health Service.
 

A call to action

Dr. Collins ended this portion of the program with an exhortation to AASLD members to do their part.

“We need your help,” Dr. Collins said. “This is a bold initiative, but it’s an opportunity. It’s even a responsibility. If we can actually succeed at this kind of outreach and save lives, and at the same time save money, how can we not do that?”

Dr. Collins, Dr. Chhatwal, and Dr. Fleurence each reported having no financial conflicts.

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

The dire shortage of pediatric hospital beds plaguing the nation in the fall of 2022 is a byproduct of financial decisions made by hospitals over the past decade, as they shuttered children’s wards, which often operate in the red, and expanded the number of beds available for more profitable endeavors like joint replacements and cancer care.

To cope with the flood of young patients sickened by a sweeping convergence of nasty bugs – especially respiratory syncytial virus, influenza, and coronavirus – medical centers nationwide have deployed triage tents, delayed elective surgeries, and transferred critically ill children out of state.

A major factor in the bed shortage is a years-long trend among hospitals of eliminating pediatric units, which tend to be less profitable than adult units, said Mark Wietecha, MS, MBA, CEO of the Children’s Hospital Association. Hospitals optimize revenue by striving to keep their beds 100% full – and filled with patients whose conditions command generous insurance reimbursements.

“It really has to do with dollars,” said Scott Krugman, MD, MS, vice chair of pediatrics at the Herman and Walter Samuelson Children’s Hospital at Sinai in Baltimore. “Hospitals rely on high-volume, high-reimbursement procedures from good payers to make money. There’s no incentive for hospitals to provide money-losing services.”

The number of pediatric inpatient units in hospitals fell 19% from 2008 to 2018, according to a study published in 2021 in the journal Pediatrics. Just this year, hospitals have closed pediatric units in Boston and Springfield, Mass.; Richmond, Va.; and Tulsa, Okla.

The current surge in dangerous respiratory illnesses among children is yet another example of how COVID-19 has upended the health care system. The lockdowns and isolation that marked the first years of the pandemic left kids largely unexposed – and still vulnerable – to viruses other than COVID for two winters, and doctors are now essentially treating multiple years’ worth of respiratory ailments.

The pandemic also accelerated changes in the health care industry that have left many communities with fewer hospital beds available for children who are acutely ill, along with fewer doctors and nurses to care for them.

When intensive care units were flooded with older COVID patients in 2020, some hospitals began using children’s beds to treat adults. Many of those pediatric beds haven’t been restored, said Daniel Rauch, MD, chair of the American Academy of Pediatrics’ committee on hospital care.

In addition, the relentless pace of the pandemic has spurred more than 230,000 health care providers – including doctors, nurses, and physician assistants – to quit. Before the pandemic, about 10% of nurses left their jobs every year; the rate has risen to about 20%, Dr. Wietecha said. He estimates that pediatric hospitals are unable to maintain as many as 10% of their beds because of staffing shortages.

“There is just not enough space for all the kids who need beds,” said Megan Ranney, MD, MPH, who works in several emergency departments in Providence, R.I., including Hasbro Children’s Hospital. The number of children seeking emergency care in recent weeks was 25% higher than the hospital’s previous record.

“We have doctors who are cleaning beds so we can get children into them faster,” said Dr. Ranney, a deputy dean at Brown University’s School of Public Health.

There’s not great money in treating kids. About 40% of U.S. children are covered by Medicaid, a joint federal-state program for low-income patients and people with disabilities. Base Medicaid rates are typically more than 20% below those paid by Medicare, the government insurance program for older adults, and are even lower when compared with private insurance. While specialty care for a range of common adult procedures, from knee and hip replacements to heart surgeries and cancer treatments, generates major profits for medical centers, hospitals complain they typically lose money on inpatient pediatric care.

When Tufts Children’s Hospital closed 41 pediatric beds this summer, hospital officials assured residents that young patients could receive care at nearby Boston Children’s Hospital. Now, Boston Children’s is delaying some elective surgeries to make room for kids who are acutely ill.

Dr. Rauch noted that children’s hospitals, which specialize in treating rare and serious conditions such as pediatric cancer, cystic fibrosis, and heart defects, simply aren’t designed to handle this season’s crush of kids acutely ill with respiratory bugs.

Even before the autumn’s viral trifecta, pediatric units were straining to absorb rising numbers of young people in acute mental distress. Stories abound of children in mental crises being marooned for weeks in emergency departments while awaiting transfer to a pediatric psychiatric unit. On a good day, Dr. Ranney said, 20% of pediatric emergency room beds at Hasbro Children’s Hospital are occupied by children experiencing mental health issues.

In hopes of adding pediatric capacity, the American Academy of Pediatrics joined the Children’s Hospital Association last month in calling on the White House to declare a national emergency due to child respiratory infections and provide additional resources to help cover the costs of care. The Biden administration has said that the flexibility hospital systems and providers have been given during the pandemic to sidestep certain staffing requirements also applies to RSV and flu.

Doernbecher Children’s Hospital at Oregon Health & Science University has shifted to “crisis standards of care,” enabling intensive care nurses to treat more patients than they’re usually assigned. Hospitals in Atlanta, Pittsburgh, and Aurora, Colorado, meanwhile, have resorted to treating young patients in overflow tents in parking lots.

Alex Kon, MD, a pediatric critical care physician at Community Medical Center in Missoula, Mont., said providers there have made plans to care for older kids in the adult intensive care unit, and to divert ambulances to other facilities when necessary. With only three pediatric ICUs in the state, that means young patients may be flown as far as Seattle or Spokane, Wash., or Idaho.

Hollis Lillard took her 1-year-old son, Calder, to an Army hospital in Northern Virginia last month after he experienced several days of fever, coughing, and labored breathing. They spent 7 anguished hours in the emergency room before the hospital found an open bed and transferred them by ambulance to Walter Reed National Military Medical Center in Maryland.

With proper therapy and instructions for home care, Calder’s virus was readily treatable: He recovered after he was given oxygen and treated with steroids, which fight inflammation, and albuterol, which counteracts bronchospasms. He was discharged the next day.

Although hospitalizations for RSV are falling, rates remain well above the norm for this time of year. And hospitals may not get much relief.

People can be infected with RSV more than once a year, and Dr. Krugman worries about a resurgence in the months to come. Because of the coronavirus, which competes with other viruses, “the usual seasonal pattern of viruses has gone out the window,” he said.

Like RSV, influenza arrived early this season. Both viruses usually peak around January. Three strains of flu are circulating and have caused an estimated 8.7 million illnesses, 78,000 hospitalizations, and 4,500 deaths, according to the Centers for Disease Control and Prevention.

Dr. Krugman doubts the health care industry will learn any quick lessons from the current crisis. “Unless there is a radical change in how we pay for pediatric hospital care,” Dr. Krugman said, “the bed shortage is only going to get worse.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

The dire shortage of pediatric hospital beds plaguing the nation in the fall of 2022 is a byproduct of financial decisions made by hospitals over the past decade, as they shuttered children’s wards, which often operate in the red, and expanded the number of beds available for more profitable endeavors like joint replacements and cancer care.

To cope with the flood of young patients sickened by a sweeping convergence of nasty bugs – especially respiratory syncytial virus, influenza, and coronavirus – medical centers nationwide have deployed triage tents, delayed elective surgeries, and transferred critically ill children out of state.

A major factor in the bed shortage is a years-long trend among hospitals of eliminating pediatric units, which tend to be less profitable than adult units, said Mark Wietecha, MS, MBA, CEO of the Children’s Hospital Association. Hospitals optimize revenue by striving to keep their beds 100% full – and filled with patients whose conditions command generous insurance reimbursements.

“It really has to do with dollars,” said Scott Krugman, MD, MS, vice chair of pediatrics at the Herman and Walter Samuelson Children’s Hospital at Sinai in Baltimore. “Hospitals rely on high-volume, high-reimbursement procedures from good payers to make money. There’s no incentive for hospitals to provide money-losing services.”

The number of pediatric inpatient units in hospitals fell 19% from 2008 to 2018, according to a study published in 2021 in the journal Pediatrics. Just this year, hospitals have closed pediatric units in Boston and Springfield, Mass.; Richmond, Va.; and Tulsa, Okla.

The current surge in dangerous respiratory illnesses among children is yet another example of how COVID-19 has upended the health care system. The lockdowns and isolation that marked the first years of the pandemic left kids largely unexposed – and still vulnerable – to viruses other than COVID for two winters, and doctors are now essentially treating multiple years’ worth of respiratory ailments.

The pandemic also accelerated changes in the health care industry that have left many communities with fewer hospital beds available for children who are acutely ill, along with fewer doctors and nurses to care for them.

When intensive care units were flooded with older COVID patients in 2020, some hospitals began using children’s beds to treat adults. Many of those pediatric beds haven’t been restored, said Daniel Rauch, MD, chair of the American Academy of Pediatrics’ committee on hospital care.

In addition, the relentless pace of the pandemic has spurred more than 230,000 health care providers – including doctors, nurses, and physician assistants – to quit. Before the pandemic, about 10% of nurses left their jobs every year; the rate has risen to about 20%, Dr. Wietecha said. He estimates that pediatric hospitals are unable to maintain as many as 10% of their beds because of staffing shortages.

“There is just not enough space for all the kids who need beds,” said Megan Ranney, MD, MPH, who works in several emergency departments in Providence, R.I., including Hasbro Children’s Hospital. The number of children seeking emergency care in recent weeks was 25% higher than the hospital’s previous record.

“We have doctors who are cleaning beds so we can get children into them faster,” said Dr. Ranney, a deputy dean at Brown University’s School of Public Health.

There’s not great money in treating kids. About 40% of U.S. children are covered by Medicaid, a joint federal-state program for low-income patients and people with disabilities. Base Medicaid rates are typically more than 20% below those paid by Medicare, the government insurance program for older adults, and are even lower when compared with private insurance. While specialty care for a range of common adult procedures, from knee and hip replacements to heart surgeries and cancer treatments, generates major profits for medical centers, hospitals complain they typically lose money on inpatient pediatric care.

When Tufts Children’s Hospital closed 41 pediatric beds this summer, hospital officials assured residents that young patients could receive care at nearby Boston Children’s Hospital. Now, Boston Children’s is delaying some elective surgeries to make room for kids who are acutely ill.

Dr. Rauch noted that children’s hospitals, which specialize in treating rare and serious conditions such as pediatric cancer, cystic fibrosis, and heart defects, simply aren’t designed to handle this season’s crush of kids acutely ill with respiratory bugs.

Even before the autumn’s viral trifecta, pediatric units were straining to absorb rising numbers of young people in acute mental distress. Stories abound of children in mental crises being marooned for weeks in emergency departments while awaiting transfer to a pediatric psychiatric unit. On a good day, Dr. Ranney said, 20% of pediatric emergency room beds at Hasbro Children’s Hospital are occupied by children experiencing mental health issues.

In hopes of adding pediatric capacity, the American Academy of Pediatrics joined the Children’s Hospital Association last month in calling on the White House to declare a national emergency due to child respiratory infections and provide additional resources to help cover the costs of care. The Biden administration has said that the flexibility hospital systems and providers have been given during the pandemic to sidestep certain staffing requirements also applies to RSV and flu.

Doernbecher Children’s Hospital at Oregon Health & Science University has shifted to “crisis standards of care,” enabling intensive care nurses to treat more patients than they’re usually assigned. Hospitals in Atlanta, Pittsburgh, and Aurora, Colorado, meanwhile, have resorted to treating young patients in overflow tents in parking lots.

Alex Kon, MD, a pediatric critical care physician at Community Medical Center in Missoula, Mont., said providers there have made plans to care for older kids in the adult intensive care unit, and to divert ambulances to other facilities when necessary. With only three pediatric ICUs in the state, that means young patients may be flown as far as Seattle or Spokane, Wash., or Idaho.

Hollis Lillard took her 1-year-old son, Calder, to an Army hospital in Northern Virginia last month after he experienced several days of fever, coughing, and labored breathing. They spent 7 anguished hours in the emergency room before the hospital found an open bed and transferred them by ambulance to Walter Reed National Military Medical Center in Maryland.

With proper therapy and instructions for home care, Calder’s virus was readily treatable: He recovered after he was given oxygen and treated with steroids, which fight inflammation, and albuterol, which counteracts bronchospasms. He was discharged the next day.

Although hospitalizations for RSV are falling, rates remain well above the norm for this time of year. And hospitals may not get much relief.

People can be infected with RSV more than once a year, and Dr. Krugman worries about a resurgence in the months to come. Because of the coronavirus, which competes with other viruses, “the usual seasonal pattern of viruses has gone out the window,” he said.

Like RSV, influenza arrived early this season. Both viruses usually peak around January. Three strains of flu are circulating and have caused an estimated 8.7 million illnesses, 78,000 hospitalizations, and 4,500 deaths, according to the Centers for Disease Control and Prevention.

Dr. Krugman doubts the health care industry will learn any quick lessons from the current crisis. “Unless there is a radical change in how we pay for pediatric hospital care,” Dr. Krugman said, “the bed shortage is only going to get worse.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

The dire shortage of pediatric hospital beds plaguing the nation in the fall of 2022 is a byproduct of financial decisions made by hospitals over the past decade, as they shuttered children’s wards, which often operate in the red, and expanded the number of beds available for more profitable endeavors like joint replacements and cancer care.

To cope with the flood of young patients sickened by a sweeping convergence of nasty bugs – especially respiratory syncytial virus, influenza, and coronavirus – medical centers nationwide have deployed triage tents, delayed elective surgeries, and transferred critically ill children out of state.

A major factor in the bed shortage is a years-long trend among hospitals of eliminating pediatric units, which tend to be less profitable than adult units, said Mark Wietecha, MS, MBA, CEO of the Children’s Hospital Association. Hospitals optimize revenue by striving to keep their beds 100% full – and filled with patients whose conditions command generous insurance reimbursements.

“It really has to do with dollars,” said Scott Krugman, MD, MS, vice chair of pediatrics at the Herman and Walter Samuelson Children’s Hospital at Sinai in Baltimore. “Hospitals rely on high-volume, high-reimbursement procedures from good payers to make money. There’s no incentive for hospitals to provide money-losing services.”

The number of pediatric inpatient units in hospitals fell 19% from 2008 to 2018, according to a study published in 2021 in the journal Pediatrics. Just this year, hospitals have closed pediatric units in Boston and Springfield, Mass.; Richmond, Va.; and Tulsa, Okla.

The current surge in dangerous respiratory illnesses among children is yet another example of how COVID-19 has upended the health care system. The lockdowns and isolation that marked the first years of the pandemic left kids largely unexposed – and still vulnerable – to viruses other than COVID for two winters, and doctors are now essentially treating multiple years’ worth of respiratory ailments.

The pandemic also accelerated changes in the health care industry that have left many communities with fewer hospital beds available for children who are acutely ill, along with fewer doctors and nurses to care for them.

When intensive care units were flooded with older COVID patients in 2020, some hospitals began using children’s beds to treat adults. Many of those pediatric beds haven’t been restored, said Daniel Rauch, MD, chair of the American Academy of Pediatrics’ committee on hospital care.

In addition, the relentless pace of the pandemic has spurred more than 230,000 health care providers – including doctors, nurses, and physician assistants – to quit. Before the pandemic, about 10% of nurses left their jobs every year; the rate has risen to about 20%, Dr. Wietecha said. He estimates that pediatric hospitals are unable to maintain as many as 10% of their beds because of staffing shortages.

“There is just not enough space for all the kids who need beds,” said Megan Ranney, MD, MPH, who works in several emergency departments in Providence, R.I., including Hasbro Children’s Hospital. The number of children seeking emergency care in recent weeks was 25% higher than the hospital’s previous record.

“We have doctors who are cleaning beds so we can get children into them faster,” said Dr. Ranney, a deputy dean at Brown University’s School of Public Health.

There’s not great money in treating kids. About 40% of U.S. children are covered by Medicaid, a joint federal-state program for low-income patients and people with disabilities. Base Medicaid rates are typically more than 20% below those paid by Medicare, the government insurance program for older adults, and are even lower when compared with private insurance. While specialty care for a range of common adult procedures, from knee and hip replacements to heart surgeries and cancer treatments, generates major profits for medical centers, hospitals complain they typically lose money on inpatient pediatric care.

When Tufts Children’s Hospital closed 41 pediatric beds this summer, hospital officials assured residents that young patients could receive care at nearby Boston Children’s Hospital. Now, Boston Children’s is delaying some elective surgeries to make room for kids who are acutely ill.

Dr. Rauch noted that children’s hospitals, which specialize in treating rare and serious conditions such as pediatric cancer, cystic fibrosis, and heart defects, simply aren’t designed to handle this season’s crush of kids acutely ill with respiratory bugs.

Even before the autumn’s viral trifecta, pediatric units were straining to absorb rising numbers of young people in acute mental distress. Stories abound of children in mental crises being marooned for weeks in emergency departments while awaiting transfer to a pediatric psychiatric unit. On a good day, Dr. Ranney said, 20% of pediatric emergency room beds at Hasbro Children’s Hospital are occupied by children experiencing mental health issues.

In hopes of adding pediatric capacity, the American Academy of Pediatrics joined the Children’s Hospital Association last month in calling on the White House to declare a national emergency due to child respiratory infections and provide additional resources to help cover the costs of care. The Biden administration has said that the flexibility hospital systems and providers have been given during the pandemic to sidestep certain staffing requirements also applies to RSV and flu.

Doernbecher Children’s Hospital at Oregon Health & Science University has shifted to “crisis standards of care,” enabling intensive care nurses to treat more patients than they’re usually assigned. Hospitals in Atlanta, Pittsburgh, and Aurora, Colorado, meanwhile, have resorted to treating young patients in overflow tents in parking lots.

Alex Kon, MD, a pediatric critical care physician at Community Medical Center in Missoula, Mont., said providers there have made plans to care for older kids in the adult intensive care unit, and to divert ambulances to other facilities when necessary. With only three pediatric ICUs in the state, that means young patients may be flown as far as Seattle or Spokane, Wash., or Idaho.

Hollis Lillard took her 1-year-old son, Calder, to an Army hospital in Northern Virginia last month after he experienced several days of fever, coughing, and labored breathing. They spent 7 anguished hours in the emergency room before the hospital found an open bed and transferred them by ambulance to Walter Reed National Military Medical Center in Maryland.

With proper therapy and instructions for home care, Calder’s virus was readily treatable: He recovered after he was given oxygen and treated with steroids, which fight inflammation, and albuterol, which counteracts bronchospasms. He was discharged the next day.

Although hospitalizations for RSV are falling, rates remain well above the norm for this time of year. And hospitals may not get much relief.

People can be infected with RSV more than once a year, and Dr. Krugman worries about a resurgence in the months to come. Because of the coronavirus, which competes with other viruses, “the usual seasonal pattern of viruses has gone out the window,” he said.

Like RSV, influenza arrived early this season. Both viruses usually peak around January. Three strains of flu are circulating and have caused an estimated 8.7 million illnesses, 78,000 hospitalizations, and 4,500 deaths, according to the Centers for Disease Control and Prevention.

Dr. Krugman doubts the health care industry will learn any quick lessons from the current crisis. “Unless there is a radical change in how we pay for pediatric hospital care,” Dr. Krugman said, “the bed shortage is only going to get worse.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Morality is often talked about in binary terms, black and white, right or wrong. That is how children 4-8 years old first conceive of it. Moral development progresses alongside motor, language, and social skills, but pediatricians typically do not screen for it or chart it. In adolescence, the ability for abstract thought develops and, once susceptibility to peer pressure lessens, nuances begin to shade the binary model. Honor codes become possible by the college years; scandals at colleges and military academies demonstrate that some 18- to 22-year-old young adults still lack that maturity. Lawrence Kohlberg, PhD, in the 1950s proposed a six-stage model of moral development and indicated that some people never achieve the upper stages.

Recently, neuroimaging research has demonstrated that the prefrontal cortex is still developing up to 25 years of age. Those data have ramifications for obtaining truly informed consent for medical procedures. Arbitrarily, driver’s licenses are issued at 16 years of age, the right to vote comes at age 18, and the purchase of alcohol allowed at age 21. Consent for medical care varies by state and by procedure. Treatment for general medical care, pregnancy, sexually transmitted infections, and mental health problems often have different age requirements. In some states, a 14-year-old can give consent for treatment of depression or pregnancy but cannot get a tattoo.

The rules for firearms are also complex and vary by state. Perhaps more important is the determination of medical, psychological, moral, and criminal conditions for which guns should be removed from someone’s access. Some states have created formalized red flag laws to accomplish this. Other states have informal procedures used by police and social workers rather than involving medical personnel. Recent gun-related tragedies at a St. Louis high school near me and at a Colorado Springs bar demonstrate deficiencies in the red flag approach, with multiple fatal consequences.

Some moral issues do not neatly fit the binary approach. There are many concerns that are better described by a paradigm that “all things are permissible but not everything is edifying.” Take as a model of this the consumption of alcohol. The risks of an occasional single drink are very small for most people. For some, one drink leads to binges of alcoholism. Others drink and drive. Over the centuries, various groups in various localities have counseled temperance. Some personally avoid alcohol to avoid leading others astray.

The United States from 1920 to 1933 carried out the national social experiment Prohibition that outlawed intoxicating beverages. Ultimately, the organized crime of black market alcohol production, distribution, and consumption was found to be worse than the disease. Alcohol use was again legalized. That is understandable. Still, I doubt Carrie Nation ever thought that the sponsorship of most major sporting events would rely heavily on beer and liquor companies. Legalization and promotion are two distinct acts.

A federal prohibition of marijuana since the 1950s similarly produced crime from drug dealing. It also induced many otherwise law-abiding citizens to be scofflaws. (I think the 55 mph national speed limit under President Nixon created greater numbers of scofflaws.) In my state recreational marijuana became legal as I write this column. But is that good? Wise? Edifying?

State lotteries were created partly to reduce the negative effects of the numbers games funding organized crime. That rationale is understandable, but why are states promoting the games with commercials and billion dollar payouts?

I find the “permissible but not edifying” paradigm helpful in many areas of medical ethics. The ethical concerns typically then fall into one of four categories: Slippery slope, bright lines, overuse, and conscientiously opting out. All are subspecies of the theme of going too far.

Oregon’s Death with Dignity Act was enacted 25 years ago. Nine other states now allow some form of medically assisted death. Most states prohibit it. As is now the case for abortion, medical care is typically legislated at the state level to reflect local values. Canada goes beyond assisted suicide and in the vast majority of cases employs active euthanasia with a physician pushing lethal drugs intravenously. That action is forbidden in the United States. Currently, euthanasia is involved in 10,000 deaths per year in Canada. A terminal illness is no longer needed; that requirement was ruled unconstitutional. By March 2023, intractable mental health problems will be eligible for the euthanasia cure in Canada. I have long considered myself a navigator of ethical slippery slopes because that is where the suffering people are. Canada has gone too far down this particular slope for my skill set.
 

Dr. Powell is a retired pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

Morality is often talked about in binary terms, black and white, right or wrong. That is how children 4-8 years old first conceive of it. Moral development progresses alongside motor, language, and social skills, but pediatricians typically do not screen for it or chart it. In adolescence, the ability for abstract thought develops and, once susceptibility to peer pressure lessens, nuances begin to shade the binary model. Honor codes become possible by the college years; scandals at colleges and military academies demonstrate that some 18- to 22-year-old young adults still lack that maturity. Lawrence Kohlberg, PhD, in the 1950s proposed a six-stage model of moral development and indicated that some people never achieve the upper stages.

Recently, neuroimaging research has demonstrated that the prefrontal cortex is still developing up to 25 years of age. Those data have ramifications for obtaining truly informed consent for medical procedures. Arbitrarily, driver’s licenses are issued at 16 years of age, the right to vote comes at age 18, and the purchase of alcohol allowed at age 21. Consent for medical care varies by state and by procedure. Treatment for general medical care, pregnancy, sexually transmitted infections, and mental health problems often have different age requirements. In some states, a 14-year-old can give consent for treatment of depression or pregnancy but cannot get a tattoo.

The rules for firearms are also complex and vary by state. Perhaps more important is the determination of medical, psychological, moral, and criminal conditions for which guns should be removed from someone’s access. Some states have created formalized red flag laws to accomplish this. Other states have informal procedures used by police and social workers rather than involving medical personnel. Recent gun-related tragedies at a St. Louis high school near me and at a Colorado Springs bar demonstrate deficiencies in the red flag approach, with multiple fatal consequences.

Some moral issues do not neatly fit the binary approach. There are many concerns that are better described by a paradigm that “all things are permissible but not everything is edifying.” Take as a model of this the consumption of alcohol. The risks of an occasional single drink are very small for most people. For some, one drink leads to binges of alcoholism. Others drink and drive. Over the centuries, various groups in various localities have counseled temperance. Some personally avoid alcohol to avoid leading others astray.

The United States from 1920 to 1933 carried out the national social experiment Prohibition that outlawed intoxicating beverages. Ultimately, the organized crime of black market alcohol production, distribution, and consumption was found to be worse than the disease. Alcohol use was again legalized. That is understandable. Still, I doubt Carrie Nation ever thought that the sponsorship of most major sporting events would rely heavily on beer and liquor companies. Legalization and promotion are two distinct acts.

A federal prohibition of marijuana since the 1950s similarly produced crime from drug dealing. It also induced many otherwise law-abiding citizens to be scofflaws. (I think the 55 mph national speed limit under President Nixon created greater numbers of scofflaws.) In my state recreational marijuana became legal as I write this column. But is that good? Wise? Edifying?

State lotteries were created partly to reduce the negative effects of the numbers games funding organized crime. That rationale is understandable, but why are states promoting the games with commercials and billion dollar payouts?

I find the “permissible but not edifying” paradigm helpful in many areas of medical ethics. The ethical concerns typically then fall into one of four categories: Slippery slope, bright lines, overuse, and conscientiously opting out. All are subspecies of the theme of going too far.

Oregon’s Death with Dignity Act was enacted 25 years ago. Nine other states now allow some form of medically assisted death. Most states prohibit it. As is now the case for abortion, medical care is typically legislated at the state level to reflect local values. Canada goes beyond assisted suicide and in the vast majority of cases employs active euthanasia with a physician pushing lethal drugs intravenously. That action is forbidden in the United States. Currently, euthanasia is involved in 10,000 deaths per year in Canada. A terminal illness is no longer needed; that requirement was ruled unconstitutional. By March 2023, intractable mental health problems will be eligible for the euthanasia cure in Canada. I have long considered myself a navigator of ethical slippery slopes because that is where the suffering people are. Canada has gone too far down this particular slope for my skill set.
 

Dr. Powell is a retired pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

Morality is often talked about in binary terms, black and white, right or wrong. That is how children 4-8 years old first conceive of it. Moral development progresses alongside motor, language, and social skills, but pediatricians typically do not screen for it or chart it. In adolescence, the ability for abstract thought develops and, once susceptibility to peer pressure lessens, nuances begin to shade the binary model. Honor codes become possible by the college years; scandals at colleges and military academies demonstrate that some 18- to 22-year-old young adults still lack that maturity. Lawrence Kohlberg, PhD, in the 1950s proposed a six-stage model of moral development and indicated that some people never achieve the upper stages.

Recently, neuroimaging research has demonstrated that the prefrontal cortex is still developing up to 25 years of age. Those data have ramifications for obtaining truly informed consent for medical procedures. Arbitrarily, driver’s licenses are issued at 16 years of age, the right to vote comes at age 18, and the purchase of alcohol allowed at age 21. Consent for medical care varies by state and by procedure. Treatment for general medical care, pregnancy, sexually transmitted infections, and mental health problems often have different age requirements. In some states, a 14-year-old can give consent for treatment of depression or pregnancy but cannot get a tattoo.

The rules for firearms are also complex and vary by state. Perhaps more important is the determination of medical, psychological, moral, and criminal conditions for which guns should be removed from someone’s access. Some states have created formalized red flag laws to accomplish this. Other states have informal procedures used by police and social workers rather than involving medical personnel. Recent gun-related tragedies at a St. Louis high school near me and at a Colorado Springs bar demonstrate deficiencies in the red flag approach, with multiple fatal consequences.

Some moral issues do not neatly fit the binary approach. There are many concerns that are better described by a paradigm that “all things are permissible but not everything is edifying.” Take as a model of this the consumption of alcohol. The risks of an occasional single drink are very small for most people. For some, one drink leads to binges of alcoholism. Others drink and drive. Over the centuries, various groups in various localities have counseled temperance. Some personally avoid alcohol to avoid leading others astray.

The United States from 1920 to 1933 carried out the national social experiment Prohibition that outlawed intoxicating beverages. Ultimately, the organized crime of black market alcohol production, distribution, and consumption was found to be worse than the disease. Alcohol use was again legalized. That is understandable. Still, I doubt Carrie Nation ever thought that the sponsorship of most major sporting events would rely heavily on beer and liquor companies. Legalization and promotion are two distinct acts.

A federal prohibition of marijuana since the 1950s similarly produced crime from drug dealing. It also induced many otherwise law-abiding citizens to be scofflaws. (I think the 55 mph national speed limit under President Nixon created greater numbers of scofflaws.) In my state recreational marijuana became legal as I write this column. But is that good? Wise? Edifying?

State lotteries were created partly to reduce the negative effects of the numbers games funding organized crime. That rationale is understandable, but why are states promoting the games with commercials and billion dollar payouts?

I find the “permissible but not edifying” paradigm helpful in many areas of medical ethics. The ethical concerns typically then fall into one of four categories: Slippery slope, bright lines, overuse, and conscientiously opting out. All are subspecies of the theme of going too far.

Oregon’s Death with Dignity Act was enacted 25 years ago. Nine other states now allow some form of medically assisted death. Most states prohibit it. As is now the case for abortion, medical care is typically legislated at the state level to reflect local values. Canada goes beyond assisted suicide and in the vast majority of cases employs active euthanasia with a physician pushing lethal drugs intravenously. That action is forbidden in the United States. Currently, euthanasia is involved in 10,000 deaths per year in Canada. A terminal illness is no longer needed; that requirement was ruled unconstitutional. By March 2023, intractable mental health problems will be eligible for the euthanasia cure in Canada. I have long considered myself a navigator of ethical slippery slopes because that is where the suffering people are. Canada has gone too far down this particular slope for my skill set.
 

Dr. Powell is a retired pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

The antidepressant vortioxetine (Trintellix) improved cognition, memory, and depressive symptoms in patients with comorbid major depressive disorder (MDD) and dementia.

The results of the 12-week open-label, single-group study are positive, study investigator Michael Cronquist Christensen, MPA, DrPH, a director with the Lundbeck pharmaceutical company, told this news organization before presenting the results in a poster at the 15th Clinical Trials on Alzheimer’s Disease conference.

“The study confirms earlier findings of improvement in both depressive symptoms and cognitive performance with vortioxetine in patients with depression and dementia and adds to this research that these clinical effects also extend to improvement in health-related quality of life and patients’ daily functioning,” Dr. Christensen said.

“It also demonstrates that patients with depression and comorbid dementia can be safely treated with 20 mg vortioxetine – starting dose of 5 mg for the first week and up-titration to 10 mg at day 8,” he added.

However, he reported that Lundbeck doesn’t plan to seek approval from the U.S. Food and Drug Administration for a new indication. Vortioxetine received FDA approval in 2013 to treat MDD, but 3 years later the agency rejected an expansion of its indication to include cognitive dysfunction.

“Vortioxetine is approved for MDD, but the product can be used in patients with MDD who have other diseases, including other mental illnesses,” Dr. Christensen said.

Potential neurotransmission modulator

Vortioxetine is a selective serotonin reuptake inhibitor and serotonin receptor modulator. According to Dr. Christensen, evidence suggests the drug’s receptor targets “have the potential to modulate neurotransmitter systems that are essential for regulation of cognitive function.”

The researchers recruited 83 individuals aged 55-85 with recurrent MDD that had started before the age of 55. All had MDD episodes within the previous 6 months and comorbid dementia for at least 6 months.

Of the participants, 65.9% were female. In addition, 42.7% had Alzheimer’s disease, 26.8% had mixed-type dementia, and the rest had other types of dementia.

The daily oral dose of vortioxetine started at 5 mg for up to week 1 and then was increased to 10 mg. It was then increased to 20 mg or decreased to 5 mg “based on investigator judgment and patient response.” The average daily dose was 12.3 mg.

In regard to the primary outcome, at week 12 (n = 70), scores on the Montgomery-Åsberg Depression Rating Scale (MADRS) fell by a mean of –12.4 (.78, P < .0001), which researchers deemed to be a significant reduction in severe symptoms.

“A significant and clinically meaningful effect was observed from week 1,” the researchers reported.

“As a basis for comparison, we typically see an improvement around 13-14 points during 8 weeks of antidepressant treatment in adults with MDD who do not have dementia,” Dr. Christensen added.

More than a third of patients (35.7%) saw a reduction in MADRS score by more than 50% at week 12, and 17.2% were considered to have reached MDD depression remission, defined as a MADRS score at or under 10.

For secondary outcomes, the total Digit Symbol Substitution test score grew by 0.65 (standardized effect size) by week 12, showing significant improvement (P < .0001). In addition, participants improved on some other cognitive measures, and Dr. Christensen noted that “significant improvement was also observed in the patients’ health-related quality of life and daily functioning.”

A third of patients had drug-related treatment-emergent adverse events.

Vortioxetine is one of the most expensive antidepressants: It has a list price of $444 a month, and no generic version is currently available.

Small trial, open-label design

In a comment, Claire Sexton, DPhil, senior director of scientific programs and outreach at the Alzheimer’s Association, said the study “reflects a valuable aspect of treatment research because of the close connection between depression and dementia. Depression is a known risk factor for dementia, including Alzheimer’s disease, and those who have dementia may experience depression.”

Alzheimer’s Association

She cautioned, however, that the trial was small and had an open-label design instead of the “gold standard” of a double-blinded trial with a control group.

The study was funded by Lundbeck, where Dr. Christensen is an employee. Another author is a Lundbeck employee, and a third author reported various disclosures. Dr. Sexton reported no disclosures.

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

The antidepressant vortioxetine (Trintellix) improved cognition, memory, and depressive symptoms in patients with comorbid major depressive disorder (MDD) and dementia.

The results of the 12-week open-label, single-group study are positive, study investigator Michael Cronquist Christensen, MPA, DrPH, a director with the Lundbeck pharmaceutical company, told this news organization before presenting the results in a poster at the 15th Clinical Trials on Alzheimer’s Disease conference.

“The study confirms earlier findings of improvement in both depressive symptoms and cognitive performance with vortioxetine in patients with depression and dementia and adds to this research that these clinical effects also extend to improvement in health-related quality of life and patients’ daily functioning,” Dr. Christensen said.

“It also demonstrates that patients with depression and comorbid dementia can be safely treated with 20 mg vortioxetine – starting dose of 5 mg for the first week and up-titration to 10 mg at day 8,” he added.

However, he reported that Lundbeck doesn’t plan to seek approval from the U.S. Food and Drug Administration for a new indication. Vortioxetine received FDA approval in 2013 to treat MDD, but 3 years later the agency rejected an expansion of its indication to include cognitive dysfunction.

“Vortioxetine is approved for MDD, but the product can be used in patients with MDD who have other diseases, including other mental illnesses,” Dr. Christensen said.

Potential neurotransmission modulator

Vortioxetine is a selective serotonin reuptake inhibitor and serotonin receptor modulator. According to Dr. Christensen, evidence suggests the drug’s receptor targets “have the potential to modulate neurotransmitter systems that are essential for regulation of cognitive function.”

The researchers recruited 83 individuals aged 55-85 with recurrent MDD that had started before the age of 55. All had MDD episodes within the previous 6 months and comorbid dementia for at least 6 months.

Of the participants, 65.9% were female. In addition, 42.7% had Alzheimer’s disease, 26.8% had mixed-type dementia, and the rest had other types of dementia.

The daily oral dose of vortioxetine started at 5 mg for up to week 1 and then was increased to 10 mg. It was then increased to 20 mg or decreased to 5 mg “based on investigator judgment and patient response.” The average daily dose was 12.3 mg.

In regard to the primary outcome, at week 12 (n = 70), scores on the Montgomery-Åsberg Depression Rating Scale (MADRS) fell by a mean of –12.4 (.78, P < .0001), which researchers deemed to be a significant reduction in severe symptoms.

“A significant and clinically meaningful effect was observed from week 1,” the researchers reported.

“As a basis for comparison, we typically see an improvement around 13-14 points during 8 weeks of antidepressant treatment in adults with MDD who do not have dementia,” Dr. Christensen added.

More than a third of patients (35.7%) saw a reduction in MADRS score by more than 50% at week 12, and 17.2% were considered to have reached MDD depression remission, defined as a MADRS score at or under 10.

For secondary outcomes, the total Digit Symbol Substitution test score grew by 0.65 (standardized effect size) by week 12, showing significant improvement (P < .0001). In addition, participants improved on some other cognitive measures, and Dr. Christensen noted that “significant improvement was also observed in the patients’ health-related quality of life and daily functioning.”

A third of patients had drug-related treatment-emergent adverse events.

Vortioxetine is one of the most expensive antidepressants: It has a list price of $444 a month, and no generic version is currently available.

Small trial, open-label design

In a comment, Claire Sexton, DPhil, senior director of scientific programs and outreach at the Alzheimer’s Association, said the study “reflects a valuable aspect of treatment research because of the close connection between depression and dementia. Depression is a known risk factor for dementia, including Alzheimer’s disease, and those who have dementia may experience depression.”

Alzheimer’s Association

She cautioned, however, that the trial was small and had an open-label design instead of the “gold standard” of a double-blinded trial with a control group.

The study was funded by Lundbeck, where Dr. Christensen is an employee. Another author is a Lundbeck employee, and a third author reported various disclosures. Dr. Sexton reported no disclosures.

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

The antidepressant vortioxetine (Trintellix) improved cognition, memory, and depressive symptoms in patients with comorbid major depressive disorder (MDD) and dementia.

The results of the 12-week open-label, single-group study are positive, study investigator Michael Cronquist Christensen, MPA, DrPH, a director with the Lundbeck pharmaceutical company, told this news organization before presenting the results in a poster at the 15th Clinical Trials on Alzheimer’s Disease conference.

“The study confirms earlier findings of improvement in both depressive symptoms and cognitive performance with vortioxetine in patients with depression and dementia and adds to this research that these clinical effects also extend to improvement in health-related quality of life and patients’ daily functioning,” Dr. Christensen said.

“It also demonstrates that patients with depression and comorbid dementia can be safely treated with 20 mg vortioxetine – starting dose of 5 mg for the first week and up-titration to 10 mg at day 8,” he added.

However, he reported that Lundbeck doesn’t plan to seek approval from the U.S. Food and Drug Administration for a new indication. Vortioxetine received FDA approval in 2013 to treat MDD, but 3 years later the agency rejected an expansion of its indication to include cognitive dysfunction.

“Vortioxetine is approved for MDD, but the product can be used in patients with MDD who have other diseases, including other mental illnesses,” Dr. Christensen said.

Potential neurotransmission modulator

Vortioxetine is a selective serotonin reuptake inhibitor and serotonin receptor modulator. According to Dr. Christensen, evidence suggests the drug’s receptor targets “have the potential to modulate neurotransmitter systems that are essential for regulation of cognitive function.”

The researchers recruited 83 individuals aged 55-85 with recurrent MDD that had started before the age of 55. All had MDD episodes within the previous 6 months and comorbid dementia for at least 6 months.

Of the participants, 65.9% were female. In addition, 42.7% had Alzheimer’s disease, 26.8% had mixed-type dementia, and the rest had other types of dementia.

The daily oral dose of vortioxetine started at 5 mg for up to week 1 and then was increased to 10 mg. It was then increased to 20 mg or decreased to 5 mg “based on investigator judgment and patient response.” The average daily dose was 12.3 mg.

In regard to the primary outcome, at week 12 (n = 70), scores on the Montgomery-Åsberg Depression Rating Scale (MADRS) fell by a mean of –12.4 (.78, P < .0001), which researchers deemed to be a significant reduction in severe symptoms.

“A significant and clinically meaningful effect was observed from week 1,” the researchers reported.

“As a basis for comparison, we typically see an improvement around 13-14 points during 8 weeks of antidepressant treatment in adults with MDD who do not have dementia,” Dr. Christensen added.

More than a third of patients (35.7%) saw a reduction in MADRS score by more than 50% at week 12, and 17.2% were considered to have reached MDD depression remission, defined as a MADRS score at or under 10.

For secondary outcomes, the total Digit Symbol Substitution test score grew by 0.65 (standardized effect size) by week 12, showing significant improvement (P < .0001). In addition, participants improved on some other cognitive measures, and Dr. Christensen noted that “significant improvement was also observed in the patients’ health-related quality of life and daily functioning.”

A third of patients had drug-related treatment-emergent adverse events.

Vortioxetine is one of the most expensive antidepressants: It has a list price of $444 a month, and no generic version is currently available.

Small trial, open-label design

In a comment, Claire Sexton, DPhil, senior director of scientific programs and outreach at the Alzheimer’s Association, said the study “reflects a valuable aspect of treatment research because of the close connection between depression and dementia. Depression is a known risk factor for dementia, including Alzheimer’s disease, and those who have dementia may experience depression.”

Alzheimer’s Association

She cautioned, however, that the trial was small and had an open-label design instead of the “gold standard” of a double-blinded trial with a control group.

The study was funded by Lundbeck, where Dr. Christensen is an employee. Another author is a Lundbeck employee, and a third author reported various disclosures. Dr. Sexton reported no disclosures.

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

Several years ago I visited my primary care provider in her new office. She had just left the practice where we had been coworkers for over a decade. I was “roomed” by Louise (not her real name) whom I had never met before. I assumed she had come with the new waiting room furniture. She took my vital signs, asked a few boilerplate questions, and told me that my PCP would be in shortly.

After our initial ping-pong match of how-are-things-going I mentioned to my old friend/PCP that I thought Louise needed to work on her person-to-person skills. She thanked me and said there were so many challenges in the new practice setting she hadn’t had a chance to work on staff training.

Courtesy Dr. William G. Wilkoff

When I returned 6 months later Louise was a different person. She appeared and sounded interested in who I was and left me in the room to wait feeling glad I had spoken up. I wasn’t surprised at the change, knowing my former coworker’s past history. I was confident that in time she would coach her new staff and continue to reinforce her message by setting an example by being a caring and concerned physician.

The old Louise certainly wasn’t a rude or unpleasant person but good customer service just didn’t come naturally to her. She thought she was doing a good job, at least as far as she understood what her job was supposed to be. On the whole spectrum of professional misbehavior she would barely warrant a pixel of color. Unfortunately, we are seeing and suffering through a surge of rude behavior and incivility not just in the medical community but across all segments of our society.

It is particularly troubling in health care, which has an organizational nonsystem that was initially paternalistic and male dominated but continues to be hierarchical even as gender stereotypes are becoming less rigid. Rudeness within a team, whether it is a medical office or a factory assembly line, can create a toxic work environment that can affect the quality of the product. In this case the end product is the health and wellness of our patients. While incivility within a team can occasionally be hidden from the patients, eventually it will surface and take its toll on the customer service component of the practice.

One wonders why so many of us are behaving rudely. Is it examples we see in the media, is it our political leaders, or is the pandemic a contributor? Has democracy run its course? Are we victims of our departure from organized religion? Do we need to reorganize our medical training to be less hierarchical? I don’t think there is any single cause nor do I believe we need to restructure our medical training system to remedy the situation. We will always need to transfer information and skills from people who have them to people who need to learn them. If there is a low-hanging fruit in the customer service orchard it is one person at a time deciding to behave in a civil manner toward fellow citizens.

While a colorful crop of political signs arrived in the run-up to the November election, recently, here on the midcoast of Maine, simple white-on-black signs have appeared saying “BE NICE.”

The good news is that being nice can be contagious. Simply think of that golden rule. Treat your patients/customers/coworkers as you would like to be treated yourself.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Several years ago I visited my primary care provider in her new office. She had just left the practice where we had been coworkers for over a decade. I was “roomed” by Louise (not her real name) whom I had never met before. I assumed she had come with the new waiting room furniture. She took my vital signs, asked a few boilerplate questions, and told me that my PCP would be in shortly.

After our initial ping-pong match of how-are-things-going I mentioned to my old friend/PCP that I thought Louise needed to work on her person-to-person skills. She thanked me and said there were so many challenges in the new practice setting she hadn’t had a chance to work on staff training.

Courtesy Dr. William G. Wilkoff

When I returned 6 months later Louise was a different person. She appeared and sounded interested in who I was and left me in the room to wait feeling glad I had spoken up. I wasn’t surprised at the change, knowing my former coworker’s past history. I was confident that in time she would coach her new staff and continue to reinforce her message by setting an example by being a caring and concerned physician.

The old Louise certainly wasn’t a rude or unpleasant person but good customer service just didn’t come naturally to her. She thought she was doing a good job, at least as far as she understood what her job was supposed to be. On the whole spectrum of professional misbehavior she would barely warrant a pixel of color. Unfortunately, we are seeing and suffering through a surge of rude behavior and incivility not just in the medical community but across all segments of our society.

It is particularly troubling in health care, which has an organizational nonsystem that was initially paternalistic and male dominated but continues to be hierarchical even as gender stereotypes are becoming less rigid. Rudeness within a team, whether it is a medical office or a factory assembly line, can create a toxic work environment that can affect the quality of the product. In this case the end product is the health and wellness of our patients. While incivility within a team can occasionally be hidden from the patients, eventually it will surface and take its toll on the customer service component of the practice.

One wonders why so many of us are behaving rudely. Is it examples we see in the media, is it our political leaders, or is the pandemic a contributor? Has democracy run its course? Are we victims of our departure from organized religion? Do we need to reorganize our medical training to be less hierarchical? I don’t think there is any single cause nor do I believe we need to restructure our medical training system to remedy the situation. We will always need to transfer information and skills from people who have them to people who need to learn them. If there is a low-hanging fruit in the customer service orchard it is one person at a time deciding to behave in a civil manner toward fellow citizens.

While a colorful crop of political signs arrived in the run-up to the November election, recently, here on the midcoast of Maine, simple white-on-black signs have appeared saying “BE NICE.”

The good news is that being nice can be contagious. Simply think of that golden rule. Treat your patients/customers/coworkers as you would like to be treated yourself.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Several years ago I visited my primary care provider in her new office. She had just left the practice where we had been coworkers for over a decade. I was “roomed” by Louise (not her real name) whom I had never met before. I assumed she had come with the new waiting room furniture. She took my vital signs, asked a few boilerplate questions, and told me that my PCP would be in shortly.

After our initial ping-pong match of how-are-things-going I mentioned to my old friend/PCP that I thought Louise needed to work on her person-to-person skills. She thanked me and said there were so many challenges in the new practice setting she hadn’t had a chance to work on staff training.

Courtesy Dr. William G. Wilkoff

When I returned 6 months later Louise was a different person. She appeared and sounded interested in who I was and left me in the room to wait feeling glad I had spoken up. I wasn’t surprised at the change, knowing my former coworker’s past history. I was confident that in time she would coach her new staff and continue to reinforce her message by setting an example by being a caring and concerned physician.

The old Louise certainly wasn’t a rude or unpleasant person but good customer service just didn’t come naturally to her. She thought she was doing a good job, at least as far as she understood what her job was supposed to be. On the whole spectrum of professional misbehavior she would barely warrant a pixel of color. Unfortunately, we are seeing and suffering through a surge of rude behavior and incivility not just in the medical community but across all segments of our society.

It is particularly troubling in health care, which has an organizational nonsystem that was initially paternalistic and male dominated but continues to be hierarchical even as gender stereotypes are becoming less rigid. Rudeness within a team, whether it is a medical office or a factory assembly line, can create a toxic work environment that can affect the quality of the product. In this case the end product is the health and wellness of our patients. While incivility within a team can occasionally be hidden from the patients, eventually it will surface and take its toll on the customer service component of the practice.

One wonders why so many of us are behaving rudely. Is it examples we see in the media, is it our political leaders, or is the pandemic a contributor? Has democracy run its course? Are we victims of our departure from organized religion? Do we need to reorganize our medical training to be less hierarchical? I don’t think there is any single cause nor do I believe we need to restructure our medical training system to remedy the situation. We will always need to transfer information and skills from people who have them to people who need to learn them. If there is a low-hanging fruit in the customer service orchard it is one person at a time deciding to behave in a civil manner toward fellow citizens.

While a colorful crop of political signs arrived in the run-up to the November election, recently, here on the midcoast of Maine, simple white-on-black signs have appeared saying “BE NICE.”

The good news is that being nice can be contagious. Simply think of that golden rule. Treat your patients/customers/coworkers as you would like to be treated yourself.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Two years ago, Ashley Hingston had a miscarriage. The 35-year-old Floridian and her husband had been going through in vitro fertilization when they received the news no one wants to get, but which many in their position reflexively expect: “You are going to lose the pregnancy.” 

On the other end of the line was Ms. Hingston’s physician, who offered her advice and comfort to ease the pain of the bad news.

“I was a complete wreck and could barely even talk,” Ms. Hingston recalled. “But I think my doctor knew what I was asking, and she was doing the best to answer the questions I had: Why did this happen? What does this mean? And she sat and listened to me.”

An estimated 2% of babies born in the United States each year are the result of IVF, according to 2019 data from the Centers for Disease Control and Prevention. The process is often emotionally, physically, and economically taxing for patients. According to the CDC, the chance a pregnancy will be successful through IVF is 21.3%. Consequently, doctors often find themselves the bearers of bad news.

But interaction with a care team or a string of nurses and providers, rather than a physician, is the norm for IVF patients, according to Aimee Eyvazzadeh, MD, a specialist in infertility and reproductive endocrinology in San Ramon, Calif.

“Patients see a doctor for all of 10 minutes and then they are handed off to a care team who don’t know their whole story,” she said.

For Dr. Eyvazzadeh and other health care professionals, physicians must improve the ways they share bad news, and how they divide tasks with care teams.

Personalized care works best

Providing personalized care will improve how IVF patients respond to bad news, according to Dr. Eyvazzadeh and others.

“When people have gone through so much trauma, anything you say to them, sometimes they can’t process it very well, so they have to see the information in different ways,” she told this news organization. “After each phone call, I’ll actually type up a summary for them, with links and articles for them to read, so they are directed in a way that I think is healthy so they aren’t pulled into a rabbit hole.”

Dr. Eyvazzadeh said she encourages her patients to seek counseling during IVF treatment, and even pays for their initial psychiatric consultation. Not many doctors do this, she noted.

“Taking the time to allow the patient to process the bad news is vital,” said Linda Kim, PhD, a psychiatrist at Moon Mental Health, to whom Dr. Eyvazzadeh refers couples. Sometimes, several calls are necessary.

“Rather than thinking of the conversation as a linear process, consider it a sphere of processing,” Dr. Kim said. “The patient may need space to grieve, may ask many questions, may need to clarify what happened, or may need to vent and release frustration. This is space that the patient needs to process the bad news.” (See below for more tips on how to share bad news with your patients.)

Many care teams are skilled in delivering bad news to patients, according to Liz Grill, PsyD, a psychologist at Weill Cornell Medicine, New York. The challenge for them is ensuring new nurses and clinicians continue to have empathy training, she said.

“You want to make sure clinicians are building relationships, and empathy. Whether there is a protocol to build that level of empathy, or if they have their own innate ability to build empathy, it’s about communicating in the right way,” Dr. Grill told this news organization.

But Mark Trolice, MD, a reproductive endocrinologist and infertility specialist at the University of Central Florida, Orlando, agreed that nurses should not deliver bad news, even if they have the expertise and the compassion to do so.

“It’s the doctor’s responsibility to make that call. It’s a very difficult call and it puts an unnecessary burden on your care team to be making these calls all the time,” Dr. Trolice said. “I feel the patient wants to hear from the physician who oversaw their cycle and did the procedure and embryo transfer. It shows a tremendous amount of responsibility and commitment on the part of the physician.”

Dr. Trolice also recommended clinicians refer to the HEART (Hearing, Empathy, Apology, Response, and Thanks) guidelines to ensure proper conversations with patients about bad news.

“You give the patient time to process the information and ask questions, and then we schedule another time to talk about plans going forward,” he said.

“Patients can feel powerless and not in control of what is happening, or even over their own bodies,” Dr. Kim added. “To counteract this, it can be helpful to outline projected steps as much as possible.”

For Dr. Eyvazzadeh, caring for an IVF patient is a matter of knowing your strengths.

Providing links to web resources, recommending an organic diet, and sending them to support groups (see below) are helpful, she said. “For some people, their strength isn’t engaging with patients on the same level that I do. But I still feel like there are ways that we can still make the patients feel cared for without being extreme.”

Tips on how to share bad news with patients

A guide often cited by clinicians when delivering bad news is the Buckman Six Step Protocol:

• Get the physical context right.
• Find out how much the patient knows.
• Find out how much the patient wants to know.
• Share the information.
• Respond to the patient’s feelings.
• Plan and follow through.

Linda Kim, PhD, notes that patient preference in receiving bad news is often culturally mediated. She recommends asking patients how they would want to receive bad news, especially in during IVF process, where there can be many challenges over the course of treatment. Dr. Kim also recommends these steps:

• Get as much information in advance as possible and ask your patient directly how they want bad news. When you are meeting a patient and their families for the first time, and they are filling out their intake paperwork or health forms, you may consider adding a section on “What is your preferred method of communication?” And after that, you might add, “What is the best way to tell you challenging or difficult news? Would you prefer to be by yourself or with a loved one? Please elaborate any additional preferences.” Everyone is different, and it can be helpful to hear from the patient directly how they would like to receive bad news. It will not only meet them where they are during a difficult time, it will also demonstrate to the patient that you are respecting their preferences and involving those preferences in the process.
• Try to leave enough time for a difficult conversation with a patient. Even better is if a clinician can prepare a patient that there is some disappointing or difficult news to share.
• Finally, offer discussion on next steps. It never hurts to ask the patient directly when they are ready to discuss next steps. This may take a few hours, a few days, or even a few months or longer.

Social media as support

Monica Wunderman, a patient of Dr. Eyvazzadeh’s since 2020, began her own social media campaign on Instagram to find support and give support to women experiencing IVF.

“I started scrolling and liking posts, and a girl reached out to me to ask if I needed help” in the form of emotional support, she recalls.

Instagram became a haven for Ms. Wunderman to share information, experiences, and support with others. It also allowed her to create a network of support and meet other women, like Ashley Hingston, going through similar struggles.

Ms. Wunderman has been through four rounds of IVF so far. Three were completely unsuccessful; the last ended in miscarriage. Although she and her husband are trying again with a surrogate, the uncertainty remains. And she feels the health care system should be offering her – and the other would-be parents she has met online – more support.

“We place such importance as a society on growing families,” she says. “But then we do very little to support those who want them and struggle.”

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

Two years ago, Ashley Hingston had a miscarriage. The 35-year-old Floridian and her husband had been going through in vitro fertilization when they received the news no one wants to get, but which many in their position reflexively expect: “You are going to lose the pregnancy.” 

On the other end of the line was Ms. Hingston’s physician, who offered her advice and comfort to ease the pain of the bad news.

“I was a complete wreck and could barely even talk,” Ms. Hingston recalled. “But I think my doctor knew what I was asking, and she was doing the best to answer the questions I had: Why did this happen? What does this mean? And she sat and listened to me.”

An estimated 2% of babies born in the United States each year are the result of IVF, according to 2019 data from the Centers for Disease Control and Prevention. The process is often emotionally, physically, and economically taxing for patients. According to the CDC, the chance a pregnancy will be successful through IVF is 21.3%. Consequently, doctors often find themselves the bearers of bad news.

But interaction with a care team or a string of nurses and providers, rather than a physician, is the norm for IVF patients, according to Aimee Eyvazzadeh, MD, a specialist in infertility and reproductive endocrinology in San Ramon, Calif.

“Patients see a doctor for all of 10 minutes and then they are handed off to a care team who don’t know their whole story,” she said.

For Dr. Eyvazzadeh and other health care professionals, physicians must improve the ways they share bad news, and how they divide tasks with care teams.

Personalized care works best

Providing personalized care will improve how IVF patients respond to bad news, according to Dr. Eyvazzadeh and others.

“When people have gone through so much trauma, anything you say to them, sometimes they can’t process it very well, so they have to see the information in different ways,” she told this news organization. “After each phone call, I’ll actually type up a summary for them, with links and articles for them to read, so they are directed in a way that I think is healthy so they aren’t pulled into a rabbit hole.”

Dr. Eyvazzadeh said she encourages her patients to seek counseling during IVF treatment, and even pays for their initial psychiatric consultation. Not many doctors do this, she noted.

“Taking the time to allow the patient to process the bad news is vital,” said Linda Kim, PhD, a psychiatrist at Moon Mental Health, to whom Dr. Eyvazzadeh refers couples. Sometimes, several calls are necessary.

“Rather than thinking of the conversation as a linear process, consider it a sphere of processing,” Dr. Kim said. “The patient may need space to grieve, may ask many questions, may need to clarify what happened, or may need to vent and release frustration. This is space that the patient needs to process the bad news.” (See below for more tips on how to share bad news with your patients.)

Many care teams are skilled in delivering bad news to patients, according to Liz Grill, PsyD, a psychologist at Weill Cornell Medicine, New York. The challenge for them is ensuring new nurses and clinicians continue to have empathy training, she said.

“You want to make sure clinicians are building relationships, and empathy. Whether there is a protocol to build that level of empathy, or if they have their own innate ability to build empathy, it’s about communicating in the right way,” Dr. Grill told this news organization.

But Mark Trolice, MD, a reproductive endocrinologist and infertility specialist at the University of Central Florida, Orlando, agreed that nurses should not deliver bad news, even if they have the expertise and the compassion to do so.

“It’s the doctor’s responsibility to make that call. It’s a very difficult call and it puts an unnecessary burden on your care team to be making these calls all the time,” Dr. Trolice said. “I feel the patient wants to hear from the physician who oversaw their cycle and did the procedure and embryo transfer. It shows a tremendous amount of responsibility and commitment on the part of the physician.”

Dr. Trolice also recommended clinicians refer to the HEART (Hearing, Empathy, Apology, Response, and Thanks) guidelines to ensure proper conversations with patients about bad news.

“You give the patient time to process the information and ask questions, and then we schedule another time to talk about plans going forward,” he said.

“Patients can feel powerless and not in control of what is happening, or even over their own bodies,” Dr. Kim added. “To counteract this, it can be helpful to outline projected steps as much as possible.”

For Dr. Eyvazzadeh, caring for an IVF patient is a matter of knowing your strengths.

Providing links to web resources, recommending an organic diet, and sending them to support groups (see below) are helpful, she said. “For some people, their strength isn’t engaging with patients on the same level that I do. But I still feel like there are ways that we can still make the patients feel cared for without being extreme.”

Tips on how to share bad news with patients

A guide often cited by clinicians when delivering bad news is the Buckman Six Step Protocol:

• Get the physical context right.
• Find out how much the patient knows.
• Find out how much the patient wants to know.
• Share the information.
• Respond to the patient’s feelings.
• Plan and follow through.

Linda Kim, PhD, notes that patient preference in receiving bad news is often culturally mediated. She recommends asking patients how they would want to receive bad news, especially in during IVF process, where there can be many challenges over the course of treatment. Dr. Kim also recommends these steps:

• Get as much information in advance as possible and ask your patient directly how they want bad news. When you are meeting a patient and their families for the first time, and they are filling out their intake paperwork or health forms, you may consider adding a section on “What is your preferred method of communication?” And after that, you might add, “What is the best way to tell you challenging or difficult news? Would you prefer to be by yourself or with a loved one? Please elaborate any additional preferences.” Everyone is different, and it can be helpful to hear from the patient directly how they would like to receive bad news. It will not only meet them where they are during a difficult time, it will also demonstrate to the patient that you are respecting their preferences and involving those preferences in the process.
• Try to leave enough time for a difficult conversation with a patient. Even better is if a clinician can prepare a patient that there is some disappointing or difficult news to share.
• Finally, offer discussion on next steps. It never hurts to ask the patient directly when they are ready to discuss next steps. This may take a few hours, a few days, or even a few months or longer.

Social media as support

Monica Wunderman, a patient of Dr. Eyvazzadeh’s since 2020, began her own social media campaign on Instagram to find support and give support to women experiencing IVF.

“I started scrolling and liking posts, and a girl reached out to me to ask if I needed help” in the form of emotional support, she recalls.

Instagram became a haven for Ms. Wunderman to share information, experiences, and support with others. It also allowed her to create a network of support and meet other women, like Ashley Hingston, going through similar struggles.

Ms. Wunderman has been through four rounds of IVF so far. Three were completely unsuccessful; the last ended in miscarriage. Although she and her husband are trying again with a surrogate, the uncertainty remains. And she feels the health care system should be offering her – and the other would-be parents she has met online – more support.

“We place such importance as a society on growing families,” she says. “But then we do very little to support those who want them and struggle.”

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

Two years ago, Ashley Hingston had a miscarriage. The 35-year-old Floridian and her husband had been going through in vitro fertilization when they received the news no one wants to get, but which many in their position reflexively expect: “You are going to lose the pregnancy.” 

On the other end of the line was Ms. Hingston’s physician, who offered her advice and comfort to ease the pain of the bad news.

“I was a complete wreck and could barely even talk,” Ms. Hingston recalled. “But I think my doctor knew what I was asking, and she was doing the best to answer the questions I had: Why did this happen? What does this mean? And she sat and listened to me.”

An estimated 2% of babies born in the United States each year are the result of IVF, according to 2019 data from the Centers for Disease Control and Prevention. The process is often emotionally, physically, and economically taxing for patients. According to the CDC, the chance a pregnancy will be successful through IVF is 21.3%. Consequently, doctors often find themselves the bearers of bad news.

But interaction with a care team or a string of nurses and providers, rather than a physician, is the norm for IVF patients, according to Aimee Eyvazzadeh, MD, a specialist in infertility and reproductive endocrinology in San Ramon, Calif.

“Patients see a doctor for all of 10 minutes and then they are handed off to a care team who don’t know their whole story,” she said.

For Dr. Eyvazzadeh and other health care professionals, physicians must improve the ways they share bad news, and how they divide tasks with care teams.

Personalized care works best

Providing personalized care will improve how IVF patients respond to bad news, according to Dr. Eyvazzadeh and others.

“When people have gone through so much trauma, anything you say to them, sometimes they can’t process it very well, so they have to see the information in different ways,” she told this news organization. “After each phone call, I’ll actually type up a summary for them, with links and articles for them to read, so they are directed in a way that I think is healthy so they aren’t pulled into a rabbit hole.”

Dr. Eyvazzadeh said she encourages her patients to seek counseling during IVF treatment, and even pays for their initial psychiatric consultation. Not many doctors do this, she noted.

“Taking the time to allow the patient to process the bad news is vital,” said Linda Kim, PhD, a psychiatrist at Moon Mental Health, to whom Dr. Eyvazzadeh refers couples. Sometimes, several calls are necessary.

“Rather than thinking of the conversation as a linear process, consider it a sphere of processing,” Dr. Kim said. “The patient may need space to grieve, may ask many questions, may need to clarify what happened, or may need to vent and release frustration. This is space that the patient needs to process the bad news.” (See below for more tips on how to share bad news with your patients.)

Many care teams are skilled in delivering bad news to patients, according to Liz Grill, PsyD, a psychologist at Weill Cornell Medicine, New York. The challenge for them is ensuring new nurses and clinicians continue to have empathy training, she said.

“You want to make sure clinicians are building relationships, and empathy. Whether there is a protocol to build that level of empathy, or if they have their own innate ability to build empathy, it’s about communicating in the right way,” Dr. Grill told this news organization.

But Mark Trolice, MD, a reproductive endocrinologist and infertility specialist at the University of Central Florida, Orlando, agreed that nurses should not deliver bad news, even if they have the expertise and the compassion to do so.

“It’s the doctor’s responsibility to make that call. It’s a very difficult call and it puts an unnecessary burden on your care team to be making these calls all the time,” Dr. Trolice said. “I feel the patient wants to hear from the physician who oversaw their cycle and did the procedure and embryo transfer. It shows a tremendous amount of responsibility and commitment on the part of the physician.”

Dr. Trolice also recommended clinicians refer to the HEART (Hearing, Empathy, Apology, Response, and Thanks) guidelines to ensure proper conversations with patients about bad news.

“You give the patient time to process the information and ask questions, and then we schedule another time to talk about plans going forward,” he said.

“Patients can feel powerless and not in control of what is happening, or even over their own bodies,” Dr. Kim added. “To counteract this, it can be helpful to outline projected steps as much as possible.”

For Dr. Eyvazzadeh, caring for an IVF patient is a matter of knowing your strengths.

Providing links to web resources, recommending an organic diet, and sending them to support groups (see below) are helpful, she said. “For some people, their strength isn’t engaging with patients on the same level that I do. But I still feel like there are ways that we can still make the patients feel cared for without being extreme.”

Tips on how to share bad news with patients

A guide often cited by clinicians when delivering bad news is the Buckman Six Step Protocol:

• Get the physical context right.
• Find out how much the patient knows.
• Find out how much the patient wants to know.
• Share the information.
• Respond to the patient’s feelings.
• Plan and follow through.

Linda Kim, PhD, notes that patient preference in receiving bad news is often culturally mediated. She recommends asking patients how they would want to receive bad news, especially in during IVF process, where there can be many challenges over the course of treatment. Dr. Kim also recommends these steps:

• Get as much information in advance as possible and ask your patient directly how they want bad news. When you are meeting a patient and their families for the first time, and they are filling out their intake paperwork or health forms, you may consider adding a section on “What is your preferred method of communication?” And after that, you might add, “What is the best way to tell you challenging or difficult news? Would you prefer to be by yourself or with a loved one? Please elaborate any additional preferences.” Everyone is different, and it can be helpful to hear from the patient directly how they would like to receive bad news. It will not only meet them where they are during a difficult time, it will also demonstrate to the patient that you are respecting their preferences and involving those preferences in the process.
• Try to leave enough time for a difficult conversation with a patient. Even better is if a clinician can prepare a patient that there is some disappointing or difficult news to share.
• Finally, offer discussion on next steps. It never hurts to ask the patient directly when they are ready to discuss next steps. This may take a few hours, a few days, or even a few months or longer.

Social media as support

Monica Wunderman, a patient of Dr. Eyvazzadeh’s since 2020, began her own social media campaign on Instagram to find support and give support to women experiencing IVF.

“I started scrolling and liking posts, and a girl reached out to me to ask if I needed help” in the form of emotional support, she recalls.

Instagram became a haven for Ms. Wunderman to share information, experiences, and support with others. It also allowed her to create a network of support and meet other women, like Ashley Hingston, going through similar struggles.

Ms. Wunderman has been through four rounds of IVF so far. Three were completely unsuccessful; the last ended in miscarriage. Although she and her husband are trying again with a surrogate, the uncertainty remains. And she feels the health care system should be offering her – and the other would-be parents she has met online – more support.

“We place such importance as a society on growing families,” she says. “But then we do very little to support those who want them and struggle.”

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

Evidence summary

GAHT may improve depression and quality of life, but not anxiety

A well-done systematic review of transgender men and transgender women demonstrated that GAHT of more than a year’s duration was associated with modestly improved standardized scores for QOL, depression, and possibly anxiety.¹ It was also associated with improved scores for depression in transgender adolescents.

The authors identified 15 prospective cohort studies (n = 626 transgender adults [mean age, 25-34 years]; 198 transgender adolescent girls and boys [mean age, 15-16 years]), 2 retrospective cohort studies (n = 1756 adults; mean age, 25-32 years), and 4 cross-sectional studies (n = 336 adults; mean age, 30-37 years).

Researchers recruited participants using strict eligibility criteria (psychiatric evaluation and formal diagnosis of gender dysphoria), with no prior history of GAHT, largely from gender-affirming specialty clinics at university hospitals. Most studies were conducted after the year 2000, predominantly in Europe (8 studies in Italy; 2 each in Belgium, the Netherlands, the United States, and Spain).

GAHT comprised testosterone for transgender men (14 studies used injectable testosterone cypionate, enanthate, undecanoate, or transdermal gels), estrogens (usually with an anti-androgen such as cyproterone acetate or spironolactone) for transgender women (10 studies used transdermal, oral, or injectable estradiol valerate or conjugated estrogens), and gonadotropin-releasing hormone (GnRH) therapy for transgender adolescents (3 studies).

Researchers evaluated the outcomes of QOL, depression, and anxiety with standardized scores on validated screening tools and suicide (2 studies) by medical records. GAHT in adult transgender men and transgender women was associated with modest improvements in QOL (3 of 5 studies) and depression (8 of 12 studies), and some improvement in anxiety scores (2 of 8 studies; see TABLE¹). There was insufficient evidence to determine whether GAHT had any effect on suicide. In adolescent transgender girls and boys, GAHT was associated with modest improvements in depression but not QOL or anxiety scores.

The authors rated the strength of evidence from the included studies as low, based on study quality (small study sizes, uncontrolled confounding factors, and risk of bias in study designs).

Additional research supports GAHT’s association with improved outcomes

Three studies, published after the systematic review, evaluated outcomes before and after GAHT and found similar results. All studies recruited treatment-seeking participants from specialty clinics.

Continue to: An Australian propsective longitudinal..

An Australian prospective longitudinal controlled study (n = 77 transgender adults; 103 cisgender controls) evaluated GAHT outcomes after 6 months and found a significant reduction in gender dysphoria scores in both transgender males (adjusted mean difference [aMD] = –6.8; 95% CI, –8.7 to –4.9; P < .001) and transgender females (aMD = –4.2; 95% CI, –6.2 to –2.2; P < .001) vs controls. QOL scores (emotional well-being, social functioning) improved only for transgender males (well-being: aMD = +7.5; 95% CI, 1.3 to 13.6; P < .018; social functioning: aMD = +12.5; 95% CI, 2.8 to 22.2; P = .011).²

A US prospective cohort study (n = 104 adolescents; mean age, 16 years) examined the effect of GnRH and/or GAHT over a 12-month period and found significant decreases in standardized scores for depression (adjusted odds ratio [aOR] = 0.4; 95% CI, 0.17-0.95) and suicidality (aOR = 0.27; 95% CI, 0.11-0.65) but not for anxiety. Participants who did not receive hormonal interventions had increased scores for depression and suicidality at 3 and 6 months’ follow-up.³

Gender-affirming hormone therapy in adult transgender men and transgender women was associated with modest improvements in QOL and depression, and some improvement in anxiety scores.

A prospective cohort study from the UK (n = 178 transgender adults) examined outcomes before and after GAHT treatment over 18 months and found significant decreases in standardized scores for depression (transgender males: –2.1; 95% CI, –3.2 to –1.2; P < .001; transgender females: –1.9; 95% CI, –2.8 to –1.0; P < .001) but not for anxiety.⁴

A large US study shows GAHT may reduce depression scores

A recent large cross-sectional study from the United States (n = 11,914 transgender or nonbinary youth, ages 13-24 years) found that receiving GAHT was associated with significantly lower odds of recent depression (aOR = 0.73; P < .001) and suicidality (aOR = 0.74; P < .001) compared to those who wanted GAHT but did not receive it. The authors were unable to differentiate the effects of receiving GAHT from the effects of parental support for their child’s gender identity, which may be a confounding factor.⁵

Recommendations from others

The World Professional Association for Transgender Health Standards of Care state that “gender incongruence that causes clinically significant distress and impairment often requires medically necessary clinical interventions” and recommends “health care professionals initiate and continue gender-affirming hormone therapy … due to demonstrated improvement in psychosocial functioning and quality of life.”⁶ The Endocrine Society Position Statement on Transgender Health states that “medical intervention for transgender youth and adults (including … hormone therapy) is effective, relatively safe (when appropriately monitored), and has been established as the standard of care.”⁷ The American Academy of Family Physicians “supports gender-­affirming care as an evidence-informed intervention that can promote health equity for gender-diverse individuals.”⁸

Editor’s takeaway

Family physicians commonly address many factors that can impact the QOL for our patients with gender dysphoria: lack of fixed residence, underemployment, food insecurity, and trauma. GAHT, especially in male-to-female transgender patients, may further improve QOL without evidence of harm.

Evidence summary

GAHT may improve depression and quality of life, but not anxiety

A well-done systematic review of transgender men and transgender women demonstrated that GAHT of more than a year’s duration was associated with modestly improved standardized scores for QOL, depression, and possibly anxiety.¹ It was also associated with improved scores for depression in transgender adolescents.

The authors identified 15 prospective cohort studies (n = 626 transgender adults [mean age, 25-34 years]; 198 transgender adolescent girls and boys [mean age, 15-16 years]), 2 retrospective cohort studies (n = 1756 adults; mean age, 25-32 years), and 4 cross-sectional studies (n = 336 adults; mean age, 30-37 years).

Researchers recruited participants using strict eligibility criteria (psychiatric evaluation and formal diagnosis of gender dysphoria), with no prior history of GAHT, largely from gender-affirming specialty clinics at university hospitals. Most studies were conducted after the year 2000, predominantly in Europe (8 studies in Italy; 2 each in Belgium, the Netherlands, the United States, and Spain).

GAHT comprised testosterone for transgender men (14 studies used injectable testosterone cypionate, enanthate, undecanoate, or transdermal gels), estrogens (usually with an anti-androgen such as cyproterone acetate or spironolactone) for transgender women (10 studies used transdermal, oral, or injectable estradiol valerate or conjugated estrogens), and gonadotropin-releasing hormone (GnRH) therapy for transgender adolescents (3 studies).

Researchers evaluated the outcomes of QOL, depression, and anxiety with standardized scores on validated screening tools and suicide (2 studies) by medical records. GAHT in adult transgender men and transgender women was associated with modest improvements in QOL (3 of 5 studies) and depression (8 of 12 studies), and some improvement in anxiety scores (2 of 8 studies; see TABLE¹). There was insufficient evidence to determine whether GAHT had any effect on suicide. In adolescent transgender girls and boys, GAHT was associated with modest improvements in depression but not QOL or anxiety scores.

The authors rated the strength of evidence from the included studies as low, based on study quality (small study sizes, uncontrolled confounding factors, and risk of bias in study designs).

Additional research supports GAHT’s association with improved outcomes

Three studies, published after the systematic review, evaluated outcomes before and after GAHT and found similar results. All studies recruited treatment-seeking participants from specialty clinics.

Continue to: An Australian propsective longitudinal..

An Australian prospective longitudinal controlled study (n = 77 transgender adults; 103 cisgender controls) evaluated GAHT outcomes after 6 months and found a significant reduction in gender dysphoria scores in both transgender males (adjusted mean difference [aMD] = –6.8; 95% CI, –8.7 to –4.9; P < .001) and transgender females (aMD = –4.2; 95% CI, –6.2 to –2.2; P < .001) vs controls. QOL scores (emotional well-being, social functioning) improved only for transgender males (well-being: aMD = +7.5; 95% CI, 1.3 to 13.6; P < .018; social functioning: aMD = +12.5; 95% CI, 2.8 to 22.2; P = .011).²

A US prospective cohort study (n = 104 adolescents; mean age, 16 years) examined the effect of GnRH and/or GAHT over a 12-month period and found significant decreases in standardized scores for depression (adjusted odds ratio [aOR] = 0.4; 95% CI, 0.17-0.95) and suicidality (aOR = 0.27; 95% CI, 0.11-0.65) but not for anxiety. Participants who did not receive hormonal interventions had increased scores for depression and suicidality at 3 and 6 months’ follow-up.³

Gender-affirming hormone therapy in adult transgender men and transgender women was associated with modest improvements in QOL and depression, and some improvement in anxiety scores.

A prospective cohort study from the UK (n = 178 transgender adults) examined outcomes before and after GAHT treatment over 18 months and found significant decreases in standardized scores for depression (transgender males: –2.1; 95% CI, –3.2 to –1.2; P < .001; transgender females: –1.9; 95% CI, –2.8 to –1.0; P < .001) but not for anxiety.⁴

A large US study shows GAHT may reduce depression scores

A recent large cross-sectional study from the United States (n = 11,914 transgender or nonbinary youth, ages 13-24 years) found that receiving GAHT was associated with significantly lower odds of recent depression (aOR = 0.73; P < .001) and suicidality (aOR = 0.74; P < .001) compared to those who wanted GAHT but did not receive it. The authors were unable to differentiate the effects of receiving GAHT from the effects of parental support for their child’s gender identity, which may be a confounding factor.⁵

Recommendations from others

The World Professional Association for Transgender Health Standards of Care state that “gender incongruence that causes clinically significant distress and impairment often requires medically necessary clinical interventions” and recommends “health care professionals initiate and continue gender-affirming hormone therapy … due to demonstrated improvement in psychosocial functioning and quality of life.”⁶ The Endocrine Society Position Statement on Transgender Health states that “medical intervention for transgender youth and adults (including … hormone therapy) is effective, relatively safe (when appropriately monitored), and has been established as the standard of care.”⁷ The American Academy of Family Physicians “supports gender-­affirming care as an evidence-informed intervention that can promote health equity for gender-diverse individuals.”⁸

Editor’s takeaway

Family physicians commonly address many factors that can impact the QOL for our patients with gender dysphoria: lack of fixed residence, underemployment, food insecurity, and trauma. GAHT, especially in male-to-female transgender patients, may further improve QOL without evidence of harm.

Evidence summary

GAHT may improve depression and quality of life, but not anxiety

A well-done systematic review of transgender men and transgender women demonstrated that GAHT of more than a year’s duration was associated with modestly improved standardized scores for QOL, depression, and possibly anxiety.¹ It was also associated with improved scores for depression in transgender adolescents.

The authors identified 15 prospective cohort studies (n = 626 transgender adults [mean age, 25-34 years]; 198 transgender adolescent girls and boys [mean age, 15-16 years]), 2 retrospective cohort studies (n = 1756 adults; mean age, 25-32 years), and 4 cross-sectional studies (n = 336 adults; mean age, 30-37 years).

Researchers recruited participants using strict eligibility criteria (psychiatric evaluation and formal diagnosis of gender dysphoria), with no prior history of GAHT, largely from gender-affirming specialty clinics at university hospitals. Most studies were conducted after the year 2000, predominantly in Europe (8 studies in Italy; 2 each in Belgium, the Netherlands, the United States, and Spain).

GAHT comprised testosterone for transgender men (14 studies used injectable testosterone cypionate, enanthate, undecanoate, or transdermal gels), estrogens (usually with an anti-androgen such as cyproterone acetate or spironolactone) for transgender women (10 studies used transdermal, oral, or injectable estradiol valerate or conjugated estrogens), and gonadotropin-releasing hormone (GnRH) therapy for transgender adolescents (3 studies).

Researchers evaluated the outcomes of QOL, depression, and anxiety with standardized scores on validated screening tools and suicide (2 studies) by medical records. GAHT in adult transgender men and transgender women was associated with modest improvements in QOL (3 of 5 studies) and depression (8 of 12 studies), and some improvement in anxiety scores (2 of 8 studies; see TABLE¹). There was insufficient evidence to determine whether GAHT had any effect on suicide. In adolescent transgender girls and boys, GAHT was associated with modest improvements in depression but not QOL or anxiety scores.

The authors rated the strength of evidence from the included studies as low, based on study quality (small study sizes, uncontrolled confounding factors, and risk of bias in study designs).

Additional research supports GAHT’s association with improved outcomes

Three studies, published after the systematic review, evaluated outcomes before and after GAHT and found similar results. All studies recruited treatment-seeking participants from specialty clinics.

Continue to: An Australian propsective longitudinal..

An Australian prospective longitudinal controlled study (n = 77 transgender adults; 103 cisgender controls) evaluated GAHT outcomes after 6 months and found a significant reduction in gender dysphoria scores in both transgender males (adjusted mean difference [aMD] = –6.8; 95% CI, –8.7 to –4.9; P < .001) and transgender females (aMD = –4.2; 95% CI, –6.2 to –2.2; P < .001) vs controls. QOL scores (emotional well-being, social functioning) improved only for transgender males (well-being: aMD = +7.5; 95% CI, 1.3 to 13.6; P < .018; social functioning: aMD = +12.5; 95% CI, 2.8 to 22.2; P = .011).²

A US prospective cohort study (n = 104 adolescents; mean age, 16 years) examined the effect of GnRH and/or GAHT over a 12-month period and found significant decreases in standardized scores for depression (adjusted odds ratio [aOR] = 0.4; 95% CI, 0.17-0.95) and suicidality (aOR = 0.27; 95% CI, 0.11-0.65) but not for anxiety. Participants who did not receive hormonal interventions had increased scores for depression and suicidality at 3 and 6 months’ follow-up.³

Gender-affirming hormone therapy in adult transgender men and transgender women was associated with modest improvements in QOL and depression, and some improvement in anxiety scores.

A prospective cohort study from the UK (n = 178 transgender adults) examined outcomes before and after GAHT treatment over 18 months and found significant decreases in standardized scores for depression (transgender males: –2.1; 95% CI, –3.2 to –1.2; P < .001; transgender females: –1.9; 95% CI, –2.8 to –1.0; P < .001) but not for anxiety.⁴

A large US study shows GAHT may reduce depression scores

A recent large cross-sectional study from the United States (n = 11,914 transgender or nonbinary youth, ages 13-24 years) found that receiving GAHT was associated with significantly lower odds of recent depression (aOR = 0.73; P < .001) and suicidality (aOR = 0.74; P < .001) compared to those who wanted GAHT but did not receive it. The authors were unable to differentiate the effects of receiving GAHT from the effects of parental support for their child’s gender identity, which may be a confounding factor.⁵

Recommendations from others

The World Professional Association for Transgender Health Standards of Care state that “gender incongruence that causes clinically significant distress and impairment often requires medically necessary clinical interventions” and recommends “health care professionals initiate and continue gender-affirming hormone therapy … due to demonstrated improvement in psychosocial functioning and quality of life.”⁶ The Endocrine Society Position Statement on Transgender Health states that “medical intervention for transgender youth and adults (including … hormone therapy) is effective, relatively safe (when appropriately monitored), and has been established as the standard of care.”⁷ The American Academy of Family Physicians “supports gender-­affirming care as an evidence-informed intervention that can promote health equity for gender-diverse individuals.”⁸

Editor’s takeaway

Family physicians commonly address many factors that can impact the QOL for our patients with gender dysphoria: lack of fixed residence, underemployment, food insecurity, and trauma. GAHT, especially in male-to-female transgender patients, may further improve QOL without evidence of harm.