Feature

“Maintenance therapy is big business,” warned Benjamin Derman, MD, assistant professor of medicine at the University of Chicago. He was commenting on the financial implications of new data suggesting that physicians may need to rethink maintenance with lenalidomide (Revlimid and generics) for multiple myeloma (MM).

For more than 10 years, ongoing treatment with lenalidomide following autologous hematopoietic stem cell transplantation (ASCT) has been the unchallenged gold standard.

The new findings were from the MASTER study, published in The Lancet Haematology, along with an invited commentary by Dr. Derman. In MASTER, patients who showed no evidence of disease after transplantation and two phases of consolidation therapy had the opportunity to avoid lenalidomide maintenance.

In the lenalidomide-free group, just 9% of patients without high-risk chromosome abnormalities or just one HRCA progressed within 2 years. About 47% of patients with two or more HRCAs progressed within 2 years.

The MASTER authors concluded that modern regimens of induction plus ASCT/consolidation might be good enough for many patients. Avoiding maintenance therapy “lead to most patients with newly diagnosed multiple myeloma reaching an MRD [minimal residual disease]-free, treatment-free state with a low risk of disease progression.” They also cautioned that the approach was “not optimal” for high-risk patients.

“We have been indoctrinated into continuous therapy,” said lead author Luciano Costa, MD, professor of medicine at the University of Alabama at Birmingham. “This was a reasonable approach at the time when [induction and consolidation] therapy was not as effective.”

Lenalidomide for post-ASCT maintenance became a guideline standard following a pivotal study published in the New England Journal of Medicine in 2012. The study showed that lenalidomide maintenance after transplantation almost doubled the time to progression (P < .001) and improved survival (P = .03).

Shaji Kumar, MD, is chair of the National Comprehensive Cancer Network Multiple Myeloma Guidelines and professor of medicine at the Mayo Clinic in Rochester, Minn.

Dr. Kumar said that the MASTER results alone are not sufficient to change current guidelines because the study was a single-arm, uncontrolled, phase 2 trial. However, there are “multiple reasons why we would like to stop treatment at some point in time,” Dr. Kumar said.

“Quality of life, the financial cost, and the toxicity are three main reasons why we would like to discontinue the maintenance or give maintenance only for the amount of time that a patient needs it,” Dr. Kumar added. “So then the question comes up, how do we identify the people who need long term treatment versus the people who don’t?”

“Response” in MM is conventionally classified by criteria laid down by the International Myeloma Working Group. However, the MASTER trial made use of a different measure: MRD negativity, in which myeloma cells can no longer be detected in bone-marrow aspirate at a level of 1 in 100,000 (10^–5) or, in some studies, 1 in 1 million (10^–6).

MRD is a rare bird in oncology: A surrogate endpoint that provides answers faster than progression-free survival or overall survival but is a reliable guide to both. In 2020 a team headed by Nikhil Munshi, MD, professor of medicine at Harvard Medical School, Boston, published a large meta-analysis showing that a negative MRD in a patient with MM was significantly prognostic for both progression-free survival (hazard ratio, 0.33; P < .001) and overall survival (HR, 0.45; P < .001).

In an interview from 2022, Dr. Munshi explained that patients with MRD negativity are not necessarily “cured”: “Simply, physiologically, it means that if a patient has one [myeloma] cell in a million, that cell is going to take a much longer time to grow up to be myeloma.”

In MASTER, which was based at five U.S. academic medical centers, 81% of participants (96/118) achieved MRD negativity at the 10^–5 cutoff. Eighty-four people (71%) had two consecutive MRD-negative results and did not go on to lenalidomide maintenance. Instead, they were monitored with lab tests every 8 weeks for the first 24 weeks and every 16 weeks thereafter and assessed for any changes in MRD after 6 months and 18 months.

The median age in MASTER was 61 years, 43% were women, and 20% were non-Hispanic Black. About 20% of participants had two or more HRCAs, 37% had one HRCA, and 43% had no HRCAs. All participants had four 28-day cycles of induction with Dara-KRd (daratumumab, carfilzomib, lenalidomide, and dexamethasone). This was followed by ASCT and up to two phases of consolidation with Dara-KRd.

MASTER is not the only study to show that MRD-guided discontinuation of lenalidomide seems feasible in some patients. In November 2023, Spanish researchers published a study in Blood testing a combination of lenalidomide, dexamethasone, and ixazomib. The trial allowed MRD-negative patients to stop therapy after 2 years. Progression was 17.2% over the following 4 years in the group that dropped maintenance, which included high-risk patients. The authors concluded that their results “support the safety of maintenance therapy discontinuation in patients with negative MRD at 2 years.”

These two trials are conspicuous by their rarity.

Said Dr. Derman: “We haven’t done a great job until recently of designing trials that look into discontinuation.”

Both Dr. Derman and Dr. Costa raised the elephant in the room: industry funding.

“Maintenance therapy is big business,” said Dr. Derman. He added that he had experienced problems in the past obtaining industry funding for research that involved stopping therapy.

Dr. Costa, coauthor of the MASTER trial, agreed in part: “Most pharmaceutical companies do not embark on trials like this because they’re primarily doing registration trials.” MASTER garnered some industry funding, however, and Dr. Costa found that encouraging.

How much money is at stake? In other words, what are the financial savings if patients with zero to one HRCAs who are MRD negative start to take treatment holidays from lenalidomide maintenance?

In the United States in 2019 approximately 6,410 patients received ASCT. The MASTER publication stated that “around 85%” of newly diagnosed MM patients have zero to one HRCAs and that 73% of these patients were able to stop therapy in the trial. This suggests that, each year, approximately 4,000 new patients might be eligible to avoid lenalidomide after ASCT.

The price tag of lenalidomide is approximately $20,000 per month in the United States, according to Dr. Derman. A cohort of 4,000 patients avoiding lenalidomide each year represents lost revenue of $80 million per month or almost $1 billion per year. And this does not take into account patients already on lenalidomide from previous years – or sales outside the United States. The MM multiple research pipeline reflects a lack of enthusiasm for paring down maintenance.

There are currently 229 interventional clinical studies in MM taking place nationwide. Of these, just three trials are testing what happens when patients stop therapy in the post-ASCT setting and none of the three is sponsored by industry (NCT04108624, NCT05091372, and NCT04071457). (All data from clinicaltrials.gov; search covered phase 2, 3, or 4 studies still accruing data; descriptions hand-checked; search terms: maintenance/consolidation/post-ASCT.)

Dr. Derman said that it is “incumbent on investigators” to carry out the studies to identify who is eligible to stop therapy because industry is “probably always going to err on the side of treating more.”

Sergio Giralt, MD, head of the adult bone marrow transplant service at Memorial Sloan Kettering Cancer Center, New York, was an author of the key 2012 study that enshrined lenalidomide maintenance in the guidelines. Dr. Giralt expressed concerns about the single-arm design of MASTER and said he would like to see a randomized study where some patients continued treatment and others stopped.

Dr. Giralt cautioned: “If you’re MRD negative, the chances of having to deal with your disease in the next 5 years is one in five.” Physicians could certainly “have a conversation” with patients who are MRD negative about stopping therapy, but this would need to be weighed against the need for bone-marrow biopsies every 3-6 months to check progress. (In MASTER, MRD was checked at 6 and 18 months.)

Dr. Kumar believes that “we need to pursue the concept of decreasing the duration of treatment.” However, newer immunotherapies may be the answer: “Who knows? That may be the future, that we will do more of this hit-and-run approach rather than trying to keep them persistently on something.”

Dr. Derman said: “I personally think that the data is already there ... [MASTER] shows that perhaps this notion of indefinite maintenance therapy is one that really has to go by the wayside ... patients should have the option to consider with their physician [the chance to] potentially discontinue treatment.”

For 15 years, relentless lenalidomide maintenance has “quite rightly been the strongest pillar of therapy”, said Dr. Costa. “But for patients, this is not something that they easily embrace – it’s not ideal that you are going to have to take therapy for the rest of your life.”

Dr. Costa concluded: “I don’t think we had a single patient who was reluctant to stop therapy.”

Dr. Munshi reported relationships with Adaptive, Abbvie, Amgen, Bristol-Myers Squibb, Celgene, Janssen, Karyopharm, Legend, Millennium, Novartis, Pfizer, and he is the scientific founder of Oncopep and DCT. Dr. Derman disclosed ties with Janssen, Cota, and BMS. Dr. Costa reported ties with Amgen, Cota, Janssen, BMS, AbbVie, Ionis, Genentech, Sanofi, Karyopharm, AstraZeneca, Adaptive Biotechnologies, Takeda, and Pfizer. Dr. Kumar declared relationships with AbbVie, Amgen, BMS, GlaxoSmithKline, Karyopharm, Regeneron, Roche, Sanofi, Takeda, and BeiGene. Dr. Giralt reported ties with Amgen, CSL Behring, Caladrius, Celgene, Ceramedix, ExpertConnect, GlaxoSmithKline, Janssen, Karyopharm, Kite Pharmaceuticals, Magnolia Innovation, Novartis, Omeros, Pfizer, Physicians’ Education Resource, Sanofi, TRM Oncology, and Xcenda.

“Maintenance therapy is big business,” warned Benjamin Derman, MD, assistant professor of medicine at the University of Chicago. He was commenting on the financial implications of new data suggesting that physicians may need to rethink maintenance with lenalidomide (Revlimid and generics) for multiple myeloma (MM).

For more than 10 years, ongoing treatment with lenalidomide following autologous hematopoietic stem cell transplantation (ASCT) has been the unchallenged gold standard.

The new findings were from the MASTER study, published in The Lancet Haematology, along with an invited commentary by Dr. Derman. In MASTER, patients who showed no evidence of disease after transplantation and two phases of consolidation therapy had the opportunity to avoid lenalidomide maintenance.

In the lenalidomide-free group, just 9% of patients without high-risk chromosome abnormalities or just one HRCA progressed within 2 years. About 47% of patients with two or more HRCAs progressed within 2 years.

The MASTER authors concluded that modern regimens of induction plus ASCT/consolidation might be good enough for many patients. Avoiding maintenance therapy “lead to most patients with newly diagnosed multiple myeloma reaching an MRD [minimal residual disease]-free, treatment-free state with a low risk of disease progression.” They also cautioned that the approach was “not optimal” for high-risk patients.

“We have been indoctrinated into continuous therapy,” said lead author Luciano Costa, MD, professor of medicine at the University of Alabama at Birmingham. “This was a reasonable approach at the time when [induction and consolidation] therapy was not as effective.”

Lenalidomide for post-ASCT maintenance became a guideline standard following a pivotal study published in the New England Journal of Medicine in 2012. The study showed that lenalidomide maintenance after transplantation almost doubled the time to progression (P < .001) and improved survival (P = .03).

Shaji Kumar, MD, is chair of the National Comprehensive Cancer Network Multiple Myeloma Guidelines and professor of medicine at the Mayo Clinic in Rochester, Minn.

Dr. Kumar said that the MASTER results alone are not sufficient to change current guidelines because the study was a single-arm, uncontrolled, phase 2 trial. However, there are “multiple reasons why we would like to stop treatment at some point in time,” Dr. Kumar said.

“Quality of life, the financial cost, and the toxicity are three main reasons why we would like to discontinue the maintenance or give maintenance only for the amount of time that a patient needs it,” Dr. Kumar added. “So then the question comes up, how do we identify the people who need long term treatment versus the people who don’t?”

“Response” in MM is conventionally classified by criteria laid down by the International Myeloma Working Group. However, the MASTER trial made use of a different measure: MRD negativity, in which myeloma cells can no longer be detected in bone-marrow aspirate at a level of 1 in 100,000 (10^–5) or, in some studies, 1 in 1 million (10^–6).

MRD is a rare bird in oncology: A surrogate endpoint that provides answers faster than progression-free survival or overall survival but is a reliable guide to both. In 2020 a team headed by Nikhil Munshi, MD, professor of medicine at Harvard Medical School, Boston, published a large meta-analysis showing that a negative MRD in a patient with MM was significantly prognostic for both progression-free survival (hazard ratio, 0.33; P < .001) and overall survival (HR, 0.45; P < .001).

In an interview from 2022, Dr. Munshi explained that patients with MRD negativity are not necessarily “cured”: “Simply, physiologically, it means that if a patient has one [myeloma] cell in a million, that cell is going to take a much longer time to grow up to be myeloma.”

In MASTER, which was based at five U.S. academic medical centers, 81% of participants (96/118) achieved MRD negativity at the 10^–5 cutoff. Eighty-four people (71%) had two consecutive MRD-negative results and did not go on to lenalidomide maintenance. Instead, they were monitored with lab tests every 8 weeks for the first 24 weeks and every 16 weeks thereafter and assessed for any changes in MRD after 6 months and 18 months.

The median age in MASTER was 61 years, 43% were women, and 20% were non-Hispanic Black. About 20% of participants had two or more HRCAs, 37% had one HRCA, and 43% had no HRCAs. All participants had four 28-day cycles of induction with Dara-KRd (daratumumab, carfilzomib, lenalidomide, and dexamethasone). This was followed by ASCT and up to two phases of consolidation with Dara-KRd.

MASTER is not the only study to show that MRD-guided discontinuation of lenalidomide seems feasible in some patients. In November 2023, Spanish researchers published a study in Blood testing a combination of lenalidomide, dexamethasone, and ixazomib. The trial allowed MRD-negative patients to stop therapy after 2 years. Progression was 17.2% over the following 4 years in the group that dropped maintenance, which included high-risk patients. The authors concluded that their results “support the safety of maintenance therapy discontinuation in patients with negative MRD at 2 years.”

These two trials are conspicuous by their rarity.

Said Dr. Derman: “We haven’t done a great job until recently of designing trials that look into discontinuation.”

Both Dr. Derman and Dr. Costa raised the elephant in the room: industry funding.

“Maintenance therapy is big business,” said Dr. Derman. He added that he had experienced problems in the past obtaining industry funding for research that involved stopping therapy.

Dr. Costa, coauthor of the MASTER trial, agreed in part: “Most pharmaceutical companies do not embark on trials like this because they’re primarily doing registration trials.” MASTER garnered some industry funding, however, and Dr. Costa found that encouraging.

How much money is at stake? In other words, what are the financial savings if patients with zero to one HRCAs who are MRD negative start to take treatment holidays from lenalidomide maintenance?

In the United States in 2019 approximately 6,410 patients received ASCT. The MASTER publication stated that “around 85%” of newly diagnosed MM patients have zero to one HRCAs and that 73% of these patients were able to stop therapy in the trial. This suggests that, each year, approximately 4,000 new patients might be eligible to avoid lenalidomide after ASCT.

The price tag of lenalidomide is approximately $20,000 per month in the United States, according to Dr. Derman. A cohort of 4,000 patients avoiding lenalidomide each year represents lost revenue of $80 million per month or almost $1 billion per year. And this does not take into account patients already on lenalidomide from previous years – or sales outside the United States. The MM multiple research pipeline reflects a lack of enthusiasm for paring down maintenance.

There are currently 229 interventional clinical studies in MM taking place nationwide. Of these, just three trials are testing what happens when patients stop therapy in the post-ASCT setting and none of the three is sponsored by industry (NCT04108624, NCT05091372, and NCT04071457). (All data from clinicaltrials.gov; search covered phase 2, 3, or 4 studies still accruing data; descriptions hand-checked; search terms: maintenance/consolidation/post-ASCT.)

Dr. Derman said that it is “incumbent on investigators” to carry out the studies to identify who is eligible to stop therapy because industry is “probably always going to err on the side of treating more.”

Sergio Giralt, MD, head of the adult bone marrow transplant service at Memorial Sloan Kettering Cancer Center, New York, was an author of the key 2012 study that enshrined lenalidomide maintenance in the guidelines. Dr. Giralt expressed concerns about the single-arm design of MASTER and said he would like to see a randomized study where some patients continued treatment and others stopped.

Dr. Giralt cautioned: “If you’re MRD negative, the chances of having to deal with your disease in the next 5 years is one in five.” Physicians could certainly “have a conversation” with patients who are MRD negative about stopping therapy, but this would need to be weighed against the need for bone-marrow biopsies every 3-6 months to check progress. (In MASTER, MRD was checked at 6 and 18 months.)

Dr. Kumar believes that “we need to pursue the concept of decreasing the duration of treatment.” However, newer immunotherapies may be the answer: “Who knows? That may be the future, that we will do more of this hit-and-run approach rather than trying to keep them persistently on something.”

Dr. Derman said: “I personally think that the data is already there ... [MASTER] shows that perhaps this notion of indefinite maintenance therapy is one that really has to go by the wayside ... patients should have the option to consider with their physician [the chance to] potentially discontinue treatment.”

For 15 years, relentless lenalidomide maintenance has “quite rightly been the strongest pillar of therapy”, said Dr. Costa. “But for patients, this is not something that they easily embrace – it’s not ideal that you are going to have to take therapy for the rest of your life.”

Dr. Costa concluded: “I don’t think we had a single patient who was reluctant to stop therapy.”

Dr. Munshi reported relationships with Adaptive, Abbvie, Amgen, Bristol-Myers Squibb, Celgene, Janssen, Karyopharm, Legend, Millennium, Novartis, Pfizer, and he is the scientific founder of Oncopep and DCT. Dr. Derman disclosed ties with Janssen, Cota, and BMS. Dr. Costa reported ties with Amgen, Cota, Janssen, BMS, AbbVie, Ionis, Genentech, Sanofi, Karyopharm, AstraZeneca, Adaptive Biotechnologies, Takeda, and Pfizer. Dr. Kumar declared relationships with AbbVie, Amgen, BMS, GlaxoSmithKline, Karyopharm, Regeneron, Roche, Sanofi, Takeda, and BeiGene. Dr. Giralt reported ties with Amgen, CSL Behring, Caladrius, Celgene, Ceramedix, ExpertConnect, GlaxoSmithKline, Janssen, Karyopharm, Kite Pharmaceuticals, Magnolia Innovation, Novartis, Omeros, Pfizer, Physicians’ Education Resource, Sanofi, TRM Oncology, and Xcenda.

“Maintenance therapy is big business,” warned Benjamin Derman, MD, assistant professor of medicine at the University of Chicago. He was commenting on the financial implications of new data suggesting that physicians may need to rethink maintenance with lenalidomide (Revlimid and generics) for multiple myeloma (MM).

For more than 10 years, ongoing treatment with lenalidomide following autologous hematopoietic stem cell transplantation (ASCT) has been the unchallenged gold standard.

The new findings were from the MASTER study, published in The Lancet Haematology, along with an invited commentary by Dr. Derman. In MASTER, patients who showed no evidence of disease after transplantation and two phases of consolidation therapy had the opportunity to avoid lenalidomide maintenance.

In the lenalidomide-free group, just 9% of patients without high-risk chromosome abnormalities or just one HRCA progressed within 2 years. About 47% of patients with two or more HRCAs progressed within 2 years.

The MASTER authors concluded that modern regimens of induction plus ASCT/consolidation might be good enough for many patients. Avoiding maintenance therapy “lead to most patients with newly diagnosed multiple myeloma reaching an MRD [minimal residual disease]-free, treatment-free state with a low risk of disease progression.” They also cautioned that the approach was “not optimal” for high-risk patients.

“We have been indoctrinated into continuous therapy,” said lead author Luciano Costa, MD, professor of medicine at the University of Alabama at Birmingham. “This was a reasonable approach at the time when [induction and consolidation] therapy was not as effective.”

Lenalidomide for post-ASCT maintenance became a guideline standard following a pivotal study published in the New England Journal of Medicine in 2012. The study showed that lenalidomide maintenance after transplantation almost doubled the time to progression (P < .001) and improved survival (P = .03).

Shaji Kumar, MD, is chair of the National Comprehensive Cancer Network Multiple Myeloma Guidelines and professor of medicine at the Mayo Clinic in Rochester, Minn.

Dr. Kumar said that the MASTER results alone are not sufficient to change current guidelines because the study was a single-arm, uncontrolled, phase 2 trial. However, there are “multiple reasons why we would like to stop treatment at some point in time,” Dr. Kumar said.

“Quality of life, the financial cost, and the toxicity are three main reasons why we would like to discontinue the maintenance or give maintenance only for the amount of time that a patient needs it,” Dr. Kumar added. “So then the question comes up, how do we identify the people who need long term treatment versus the people who don’t?”

“Response” in MM is conventionally classified by criteria laid down by the International Myeloma Working Group. However, the MASTER trial made use of a different measure: MRD negativity, in which myeloma cells can no longer be detected in bone-marrow aspirate at a level of 1 in 100,000 (10^–5) or, in some studies, 1 in 1 million (10^–6).

MRD is a rare bird in oncology: A surrogate endpoint that provides answers faster than progression-free survival or overall survival but is a reliable guide to both. In 2020 a team headed by Nikhil Munshi, MD, professor of medicine at Harvard Medical School, Boston, published a large meta-analysis showing that a negative MRD in a patient with MM was significantly prognostic for both progression-free survival (hazard ratio, 0.33; P < .001) and overall survival (HR, 0.45; P < .001).

In an interview from 2022, Dr. Munshi explained that patients with MRD negativity are not necessarily “cured”: “Simply, physiologically, it means that if a patient has one [myeloma] cell in a million, that cell is going to take a much longer time to grow up to be myeloma.”

In MASTER, which was based at five U.S. academic medical centers, 81% of participants (96/118) achieved MRD negativity at the 10^–5 cutoff. Eighty-four people (71%) had two consecutive MRD-negative results and did not go on to lenalidomide maintenance. Instead, they were monitored with lab tests every 8 weeks for the first 24 weeks and every 16 weeks thereafter and assessed for any changes in MRD after 6 months and 18 months.

The median age in MASTER was 61 years, 43% were women, and 20% were non-Hispanic Black. About 20% of participants had two or more HRCAs, 37% had one HRCA, and 43% had no HRCAs. All participants had four 28-day cycles of induction with Dara-KRd (daratumumab, carfilzomib, lenalidomide, and dexamethasone). This was followed by ASCT and up to two phases of consolidation with Dara-KRd.

MASTER is not the only study to show that MRD-guided discontinuation of lenalidomide seems feasible in some patients. In November 2023, Spanish researchers published a study in Blood testing a combination of lenalidomide, dexamethasone, and ixazomib. The trial allowed MRD-negative patients to stop therapy after 2 years. Progression was 17.2% over the following 4 years in the group that dropped maintenance, which included high-risk patients. The authors concluded that their results “support the safety of maintenance therapy discontinuation in patients with negative MRD at 2 years.”

These two trials are conspicuous by their rarity.

Said Dr. Derman: “We haven’t done a great job until recently of designing trials that look into discontinuation.”

Both Dr. Derman and Dr. Costa raised the elephant in the room: industry funding.

“Maintenance therapy is big business,” said Dr. Derman. He added that he had experienced problems in the past obtaining industry funding for research that involved stopping therapy.

Dr. Costa, coauthor of the MASTER trial, agreed in part: “Most pharmaceutical companies do not embark on trials like this because they’re primarily doing registration trials.” MASTER garnered some industry funding, however, and Dr. Costa found that encouraging.

How much money is at stake? In other words, what are the financial savings if patients with zero to one HRCAs who are MRD negative start to take treatment holidays from lenalidomide maintenance?

In the United States in 2019 approximately 6,410 patients received ASCT. The MASTER publication stated that “around 85%” of newly diagnosed MM patients have zero to one HRCAs and that 73% of these patients were able to stop therapy in the trial. This suggests that, each year, approximately 4,000 new patients might be eligible to avoid lenalidomide after ASCT.

The price tag of lenalidomide is approximately $20,000 per month in the United States, according to Dr. Derman. A cohort of 4,000 patients avoiding lenalidomide each year represents lost revenue of $80 million per month or almost $1 billion per year. And this does not take into account patients already on lenalidomide from previous years – or sales outside the United States. The MM multiple research pipeline reflects a lack of enthusiasm for paring down maintenance.

There are currently 229 interventional clinical studies in MM taking place nationwide. Of these, just three trials are testing what happens when patients stop therapy in the post-ASCT setting and none of the three is sponsored by industry (NCT04108624, NCT05091372, and NCT04071457). (All data from clinicaltrials.gov; search covered phase 2, 3, or 4 studies still accruing data; descriptions hand-checked; search terms: maintenance/consolidation/post-ASCT.)

Dr. Derman said that it is “incumbent on investigators” to carry out the studies to identify who is eligible to stop therapy because industry is “probably always going to err on the side of treating more.”

Sergio Giralt, MD, head of the adult bone marrow transplant service at Memorial Sloan Kettering Cancer Center, New York, was an author of the key 2012 study that enshrined lenalidomide maintenance in the guidelines. Dr. Giralt expressed concerns about the single-arm design of MASTER and said he would like to see a randomized study where some patients continued treatment and others stopped.

Dr. Giralt cautioned: “If you’re MRD negative, the chances of having to deal with your disease in the next 5 years is one in five.” Physicians could certainly “have a conversation” with patients who are MRD negative about stopping therapy, but this would need to be weighed against the need for bone-marrow biopsies every 3-6 months to check progress. (In MASTER, MRD was checked at 6 and 18 months.)

Dr. Kumar believes that “we need to pursue the concept of decreasing the duration of treatment.” However, newer immunotherapies may be the answer: “Who knows? That may be the future, that we will do more of this hit-and-run approach rather than trying to keep them persistently on something.”

Dr. Derman said: “I personally think that the data is already there ... [MASTER] shows that perhaps this notion of indefinite maintenance therapy is one that really has to go by the wayside ... patients should have the option to consider with their physician [the chance to] potentially discontinue treatment.”

For 15 years, relentless lenalidomide maintenance has “quite rightly been the strongest pillar of therapy”, said Dr. Costa. “But for patients, this is not something that they easily embrace – it’s not ideal that you are going to have to take therapy for the rest of your life.”

Dr. Costa concluded: “I don’t think we had a single patient who was reluctant to stop therapy.”

Dr. Munshi reported relationships with Adaptive, Abbvie, Amgen, Bristol-Myers Squibb, Celgene, Janssen, Karyopharm, Legend, Millennium, Novartis, Pfizer, and he is the scientific founder of Oncopep and DCT. Dr. Derman disclosed ties with Janssen, Cota, and BMS. Dr. Costa reported ties with Amgen, Cota, Janssen, BMS, AbbVie, Ionis, Genentech, Sanofi, Karyopharm, AstraZeneca, Adaptive Biotechnologies, Takeda, and Pfizer. Dr. Kumar declared relationships with AbbVie, Amgen, BMS, GlaxoSmithKline, Karyopharm, Regeneron, Roche, Sanofi, Takeda, and BeiGene. Dr. Giralt reported ties with Amgen, CSL Behring, Caladrius, Celgene, Ceramedix, ExpertConnect, GlaxoSmithKline, Janssen, Karyopharm, Kite Pharmaceuticals, Magnolia Innovation, Novartis, Omeros, Pfizer, Physicians’ Education Resource, Sanofi, TRM Oncology, and Xcenda.

In 2022, two colleagues from Johns Hopkins University, Baltimore, Bhuchitra Singh, MD, MPH, MS, MBA, and James Segars Jr., MD, reviewed the available literature to evaluate the effectiveness of newer minimally invasive therapies in reducing bleeding and improving the quality of life and control of symptoms linked to uterine fibroids. 

Their goal, according to Dr. Segars, a professor of obstetrics and gynecology and director of the division of women’s health research at Johns Hopkins, was to help guide clinicians and patients in making decisions about the use of the newer therapies, including radiofrequency ablation and ultrasound-guided removal of lesions. 

But he and Dr. Singh, the director of clinical research at the Howard W. and Georgeanna Seegar Jones Laboratory of Reproductive Sciences and Women’s Health Research, were surprised by their findings. “The outcomes were relatively the same,” Dr. Segars said. “All of the modalities lead to significant reduction in bleeding and other fibroid-related symptoms.” 

The data on long-term complications and risk for recurrence are sparse for some of the newer approaches, and not enough high-quality long-term studies have been conducted for the Food and Drug Administration to approve them as fertility-sparing treatments.

But perhaps, the biggest challenge now is to ensure that women can take advantage of these newer therapies, with large gaps in both the diagnosis of fibroids and geographic access to minimally invasive treatments.
 

A widespread condition widely underdiagnosed 

Uterine fibroids occur in most women (the incidence rises with age) and can be found in up to 70% of women by the time they reach menopause. Risk factors include family history, increasing interval since last birth, hypertension, and obesity. Increasing parity and use of oral contraceptives are protective.

But as many as 50% of cases go undiagnosed, and one reason for this is the failure of clinicians to dig deeply enough into women’s menstrual histories to diagnose fibroids. 

“The most common cause of anemia is heavy menstrual bleeding,” said Shannon Laughlin-Tommaso, MD, MPH, a professor of obstetrics and gynecology at Mayo Clinic in Rochester, Minn. She frequently sees patients who have already undergone colonoscopy to work-up the source of their anemia before anyone suspects that fibroids are the culprit. 

“When women tell us about their periods, what they’ve been told is normal [bleeding] – or what they’ve always had and considered normal – is actually kind of on the heavier spectrum,” she said. 

Ideally, treatment for uterine fibroids would fix abnormally prolonged or heavy menstrual bleeding, relieve pain, and ameliorate symptoms associated with an enlarged uterus, such as pelvic pressure, urinary frequency, and constipation. And the fibroids would never recur.

By those measures, hysterectomy fits the bill: Success rates in relieving symptoms are high, and the risk for recurrence is zero. But the procedure carries significant drawbacks: short-term complications of surgery, including infection, bleeding, and injury to the bowels and bladder along with potential long-term risks for cardiovascular disease, cancer, ovarian failure and premature menopause, depression, and decline in cognitive function. Those factors loom even larger for women who still hope to have children. 

For that reason, the American College of Obstetricians and Gynecologists recommends myomectomy, or surgical removal of individual fibroids, for women who desire uterine preservation or future pregnancy. And the literature here is solid, according to Dr. Singh, who found that 95% of myomectomy patients achieved control of their bleeding symptoms, whether it was via laparoscopy, hysteroscopy, or laparotomy. Up to 40% of women may develop new fibroids, or leiomyomas, within 3 years, although only 12.2% required a second surgery up to after 5 years. 

But myomectomy is invasive, requiring general anesthesia, incisions in the uterus, and stitches to close the organ. 

Newer techniques have emerged that can effectively treat symptoms of fibroids without requiring surgery. Uterine artery embolization (UAE), which involves passing a catheter into the femoral artery, or laparoscopic uterine artery occlusion can be used to cut off the blood supply of the fibroid. Other techniques, including focused ultrasound surgery and radiofrequency ablation (RFA), use various forms of energy to heat and ablate fibroids. The latter two can be performed in outpatient settings and often without general anesthesia.

Approved for use in 1994, UAE has the most data available, with reduction in the volume of fibroids and uterine tissue lasting up to 5 years, and rates of reintervention of 19%-38% between 2 and 5 years after the procedure. Dr. Singh’s review found that 79%-98.5% of recipients of the procedure reported declines in bleeding that persisted for several years, which is comparable to myomectomy. Quality of life and pain scores also showed good improvement, with follow-up in the different studies ranging from 12 months to over 5 years, the analysis showed.

UAE does have its drawbacks. In rare cases, embolization can deprive the entire uterus and ovaries of blood, which can cause ovarian dysfunction and potentially result in premature menopause, although this outcome is most common in women who are older than 45 years. The procedure can often also be painful enough that overnight hospitalization is required.

Focused ultrasound surgeries, which include magnetic resonance–guided focused ultrasound surgery (MRgFUS) and high-intensity focused ultrasound (HIFU), were approved by the FDA in 2004. Focused ultrasound waves pass through the abdominal wall and produce significant heating, causing a burn that destroys the targeted tissue without damaging surrounding tissue. As with UAE, improvements in fibroid-associated bleeding and measures of quality of life were similar to those after myomectomy up to 3 years later.

But Dr. Singh noted that both focused ultrasound and RFA can damage the skin or internal organs. “[As] always with the thermal interventions, there is the probability of skin as well as internal organs that might get the thermal energy if it’s not focused correctly on to the fibroid itself,” he said. In addition, MRgFUS is not an option for women who are not good candidates to undergo an MRI, such as those with claustrophobia or pacemakers.

Also, with focus ultrasound and RFA, “we do worry about that fibroid getting blood flow back,” which can lead to recurrence of heavy menstrual bleeding, Dr. Laughlin-Tommaso noted. 

Although data on RFA are limited to 12 months of follow-up, most women reported meaningful reductions in bleeding symptoms. Longer follow-up has been reported for bleeding symptoms after MRgFUS, with similar results up to 3 years later. 

For Leslie Hansen-Lindner, MD, chief of obstetrics and gynecology at Atrium Health in Charlotte, N.C., choosing the right procedure starts with a patient-centered conversation weighing the pros and cons of the options and the woman’s goals. 

“Is their goal to reduce the size and impact of their fibroid, bleed less, and have a better quality of life on their period?” Dr. Hansen-Lindner said. “Or is their goal to have the entire fibroid removed?” 

If the former, an RFA is appealing to many women. If the latter, laparoscopic or mini-laparotomy myomectomy might be a better choice. Although fewer than 10% of patients require surgical reintervention at 3 years of follow-up for RFA, myomectomy has more consistent long-term evidence showing that fewer women require re-intervention and preserve their fertility, she added. 

Age also plays a role in the decision: The closer a woman is to menopause, the less likely she is to experience a recurrence, so a less-invasive procedure is preferable. But for younger women hoping to become pregnant, the lower risk for recurrence and good prognosis for future fertility might sway the choice toward myomectomy.

The first laparoscopic RFA procedures were approved for uterine fibroids in 2012. Dr. Hansen-Lindner is a proponent of transcervical fibroid ablation (TFA), a newer RFA procedure that the FDA approved in 2018. Performed through the cervix, TFA requires no incisions and can generally be done without general anesthesia. Eligible candidates would be any woman with symptomatic fibroids, such as heavy menstrual bleeding, pain, or bulk symptoms. The contraindications are few.

“It’s going to come down to size and location of fibroids, and whether or not they would be accessible by the TFA,” Dr. Hansen-Lindner said. “I have to make sure that there isn’t a fibroid blocking their cervix and that the fibroids are accessible with this device.” 

TFA also is not suitable for removing most submucosal lesions, which typically must be removed by hysteroscopic myomectomy. Dr. Hansen-Lindner said that she often uses TFA in conjunction with hysteroscopic myomectomy for this scenario. Although data on pregnancy after RFA (including TFA), MRgFUS, and HIFU are lacking, Gynesonics, the manufacturer of the Sonata System (the device that delivers radiofrequency energy to shrink the fibroid) has documented 79 pregnancies among the 2,200 women who have undergone TFA in the United States since 2018.
 

Disparities hampering care

Uterine fibroids are a particular problem for Black women, whose symptoms are more likely to be ignored by clinicians, according to Jodie Katon, PhD, a core investigator at the Veterans Affairs Greater Los Angeles Center for the Study of Healthcare Innovation, Implementation and Policy. Dr. Katon cited studies in which Black women interviewed about their experiences reported a consistent theme: Clinicians dismissed their symptoms, told them these were nothing to worry about, and advised them to lose weight. Those interactions not only delayed diagnosis among Black women but also led many of them to mistrust clinicians and avoid the health care system altogether.

The failure of clinicians to take their complaints seriously is just one of the disparities affecting Black women. In reviewing the literature, Dr. Laughlin-Tommaso, who also serves as the associate dean for Education Diversity, Equity, and Inclusion at the Mayo Clinic, found that African American women experience two to three times the risk for fibroids, compared with White women, as well as earlier onset and more severe disease, as measured by number and size of the lesions. 

According to Dr. Katon, the etiology of fibroids is still poorly understood. “What we do know is that Black women are disproportionately exposed to a variety of factors that we have shown through observational studies are associated with increased risk of development of uterine fibroids.” 

The list includes factors like stress; interpersonal racism; early age at menarche; various indicators of poor diets, such as vitamin D deficiency; the use of certain beauty products, specifically hair straighteners; as well as exposure to air pollution and other environmental toxins.

Laughlin-Tommaso also pointed to historical disparities in management, citing a doubled risk for hysterectomy for Black women in a study published in 2007 despite survey data suggesting that Black women report being more interested in uterine-preserving therapies rather than a hysterectomy.
 

Breaking down barriers of access to new treatments

Dr. Laughlin-Tommaso looked at more recent trends in the management of fibroids using data from the multicenter COMPARE-UF study, which enrolled women between 2015 and 2020 undergoing fibroid treatment into a longitudinal registry to track their outcomes. She found that Black women underwent hysterectomies at a lower rate than did White women and were instead more likely to undergo myomectomy or UAE. 

Some of the change may reflect lack of approved minimally invasive procedures before 2000. “But now that we have expanded options, I think most women are opting not to have a hysterectomy,” Dr. Laughlin-Tommaso said.

Dr. Katon has research funding from the VA to look more closely at racial disparities in the treatment of fibroids. In a study published in April 2023, she reported some surprising trends. 

During the period from 2010 to 2018, she found that Black veterans diagnosed with fibroids were less likely than White veterans were to receive treatment, regardless of their age or the severity of their symptoms. This finding held even among women with anemia, which should have been a clear indication for treatment.

But, as in the COMPARE-UF study, the subset of Black veterans who received an interventional treatment were less likely than their White peers were to undergo hysterectomy in favor of a fertility-sparing treatment as their initial procedure. Dr. Katon called it a “welcome but unexpected finding.” 

But another significant barrier remains: The two newest types of procedures, RFA and guided focused ultrasound, are not commonly performed outside of tertiary care facilities. However, studies have found that all these procedures are cost effective (studies for myomectomy, UAE, MRgFUS, and TFA). The implementation of a category 1 billing code for laparoscopic RFA in 2017 has led more insurance companies to cover the service, and a category 1 code will be available for TFA effective January 2024. 

Although RFA does require investment in specialized equipment, which limits facilities from offering the procedure, any gynecologist who routinely performs hysteroscopy can easily learn to do TFA. And the VA, which is committed to eliminating disparities in women’s health, established a 2-year advanced fellowship in minimally invasive gynecologic surgery in 2022 to help expand their capacity to offer these procedures. 

The VA has been rapidly expanding their gynecology services, and Katon said that she is confident that ultrasound-guided procedures and RFA will become more available within the system. “I would say we’re keeping pace. And in some ways, you know, as a national system we may be positioned to actually outpace the rest of the U.S.”

Dr. Segars reported prior research funding for clinical trials from BioSpecifics Technologies, Bayer, Allergan, AbbVie, and ObsEva and currently receives funding from Myovant Sciences. Dr. Hansen-Lindner reported personal fees from Gynesonics. Dr. Singh, Dr. Laughlin-Tommaso, and Dr. Katon reported no financial conflicts of interest. 

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

In 2022, two colleagues from Johns Hopkins University, Baltimore, Bhuchitra Singh, MD, MPH, MS, MBA, and James Segars Jr., MD, reviewed the available literature to evaluate the effectiveness of newer minimally invasive therapies in reducing bleeding and improving the quality of life and control of symptoms linked to uterine fibroids. 

Their goal, according to Dr. Segars, a professor of obstetrics and gynecology and director of the division of women’s health research at Johns Hopkins, was to help guide clinicians and patients in making decisions about the use of the newer therapies, including radiofrequency ablation and ultrasound-guided removal of lesions. 

But he and Dr. Singh, the director of clinical research at the Howard W. and Georgeanna Seegar Jones Laboratory of Reproductive Sciences and Women’s Health Research, were surprised by their findings. “The outcomes were relatively the same,” Dr. Segars said. “All of the modalities lead to significant reduction in bleeding and other fibroid-related symptoms.” 

The data on long-term complications and risk for recurrence are sparse for some of the newer approaches, and not enough high-quality long-term studies have been conducted for the Food and Drug Administration to approve them as fertility-sparing treatments.

But perhaps, the biggest challenge now is to ensure that women can take advantage of these newer therapies, with large gaps in both the diagnosis of fibroids and geographic access to minimally invasive treatments.
 

A widespread condition widely underdiagnosed 

Uterine fibroids occur in most women (the incidence rises with age) and can be found in up to 70% of women by the time they reach menopause. Risk factors include family history, increasing interval since last birth, hypertension, and obesity. Increasing parity and use of oral contraceptives are protective.

But as many as 50% of cases go undiagnosed, and one reason for this is the failure of clinicians to dig deeply enough into women’s menstrual histories to diagnose fibroids. 

“The most common cause of anemia is heavy menstrual bleeding,” said Shannon Laughlin-Tommaso, MD, MPH, a professor of obstetrics and gynecology at Mayo Clinic in Rochester, Minn. She frequently sees patients who have already undergone colonoscopy to work-up the source of their anemia before anyone suspects that fibroids are the culprit. 

“When women tell us about their periods, what they’ve been told is normal [bleeding] – or what they’ve always had and considered normal – is actually kind of on the heavier spectrum,” she said. 

Ideally, treatment for uterine fibroids would fix abnormally prolonged or heavy menstrual bleeding, relieve pain, and ameliorate symptoms associated with an enlarged uterus, such as pelvic pressure, urinary frequency, and constipation. And the fibroids would never recur.

By those measures, hysterectomy fits the bill: Success rates in relieving symptoms are high, and the risk for recurrence is zero. But the procedure carries significant drawbacks: short-term complications of surgery, including infection, bleeding, and injury to the bowels and bladder along with potential long-term risks for cardiovascular disease, cancer, ovarian failure and premature menopause, depression, and decline in cognitive function. Those factors loom even larger for women who still hope to have children. 

For that reason, the American College of Obstetricians and Gynecologists recommends myomectomy, or surgical removal of individual fibroids, for women who desire uterine preservation or future pregnancy. And the literature here is solid, according to Dr. Singh, who found that 95% of myomectomy patients achieved control of their bleeding symptoms, whether it was via laparoscopy, hysteroscopy, or laparotomy. Up to 40% of women may develop new fibroids, or leiomyomas, within 3 years, although only 12.2% required a second surgery up to after 5 years. 

But myomectomy is invasive, requiring general anesthesia, incisions in the uterus, and stitches to close the organ. 

Newer techniques have emerged that can effectively treat symptoms of fibroids without requiring surgery. Uterine artery embolization (UAE), which involves passing a catheter into the femoral artery, or laparoscopic uterine artery occlusion can be used to cut off the blood supply of the fibroid. Other techniques, including focused ultrasound surgery and radiofrequency ablation (RFA), use various forms of energy to heat and ablate fibroids. The latter two can be performed in outpatient settings and often without general anesthesia.

Approved for use in 1994, UAE has the most data available, with reduction in the volume of fibroids and uterine tissue lasting up to 5 years, and rates of reintervention of 19%-38% between 2 and 5 years after the procedure. Dr. Singh’s review found that 79%-98.5% of recipients of the procedure reported declines in bleeding that persisted for several years, which is comparable to myomectomy. Quality of life and pain scores also showed good improvement, with follow-up in the different studies ranging from 12 months to over 5 years, the analysis showed.

UAE does have its drawbacks. In rare cases, embolization can deprive the entire uterus and ovaries of blood, which can cause ovarian dysfunction and potentially result in premature menopause, although this outcome is most common in women who are older than 45 years. The procedure can often also be painful enough that overnight hospitalization is required.

Focused ultrasound surgeries, which include magnetic resonance–guided focused ultrasound surgery (MRgFUS) and high-intensity focused ultrasound (HIFU), were approved by the FDA in 2004. Focused ultrasound waves pass through the abdominal wall and produce significant heating, causing a burn that destroys the targeted tissue without damaging surrounding tissue. As with UAE, improvements in fibroid-associated bleeding and measures of quality of life were similar to those after myomectomy up to 3 years later.

But Dr. Singh noted that both focused ultrasound and RFA can damage the skin or internal organs. “[As] always with the thermal interventions, there is the probability of skin as well as internal organs that might get the thermal energy if it’s not focused correctly on to the fibroid itself,” he said. In addition, MRgFUS is not an option for women who are not good candidates to undergo an MRI, such as those with claustrophobia or pacemakers.

Also, with focus ultrasound and RFA, “we do worry about that fibroid getting blood flow back,” which can lead to recurrence of heavy menstrual bleeding, Dr. Laughlin-Tommaso noted. 

Although data on RFA are limited to 12 months of follow-up, most women reported meaningful reductions in bleeding symptoms. Longer follow-up has been reported for bleeding symptoms after MRgFUS, with similar results up to 3 years later. 

For Leslie Hansen-Lindner, MD, chief of obstetrics and gynecology at Atrium Health in Charlotte, N.C., choosing the right procedure starts with a patient-centered conversation weighing the pros and cons of the options and the woman’s goals. 

“Is their goal to reduce the size and impact of their fibroid, bleed less, and have a better quality of life on their period?” Dr. Hansen-Lindner said. “Or is their goal to have the entire fibroid removed?” 

If the former, an RFA is appealing to many women. If the latter, laparoscopic or mini-laparotomy myomectomy might be a better choice. Although fewer than 10% of patients require surgical reintervention at 3 years of follow-up for RFA, myomectomy has more consistent long-term evidence showing that fewer women require re-intervention and preserve their fertility, she added. 

Age also plays a role in the decision: The closer a woman is to menopause, the less likely she is to experience a recurrence, so a less-invasive procedure is preferable. But for younger women hoping to become pregnant, the lower risk for recurrence and good prognosis for future fertility might sway the choice toward myomectomy.

The first laparoscopic RFA procedures were approved for uterine fibroids in 2012. Dr. Hansen-Lindner is a proponent of transcervical fibroid ablation (TFA), a newer RFA procedure that the FDA approved in 2018. Performed through the cervix, TFA requires no incisions and can generally be done without general anesthesia. Eligible candidates would be any woman with symptomatic fibroids, such as heavy menstrual bleeding, pain, or bulk symptoms. The contraindications are few.

“It’s going to come down to size and location of fibroids, and whether or not they would be accessible by the TFA,” Dr. Hansen-Lindner said. “I have to make sure that there isn’t a fibroid blocking their cervix and that the fibroids are accessible with this device.” 

TFA also is not suitable for removing most submucosal lesions, which typically must be removed by hysteroscopic myomectomy. Dr. Hansen-Lindner said that she often uses TFA in conjunction with hysteroscopic myomectomy for this scenario. Although data on pregnancy after RFA (including TFA), MRgFUS, and HIFU are lacking, Gynesonics, the manufacturer of the Sonata System (the device that delivers radiofrequency energy to shrink the fibroid) has documented 79 pregnancies among the 2,200 women who have undergone TFA in the United States since 2018.
 

Disparities hampering care

Uterine fibroids are a particular problem for Black women, whose symptoms are more likely to be ignored by clinicians, according to Jodie Katon, PhD, a core investigator at the Veterans Affairs Greater Los Angeles Center for the Study of Healthcare Innovation, Implementation and Policy. Dr. Katon cited studies in which Black women interviewed about their experiences reported a consistent theme: Clinicians dismissed their symptoms, told them these were nothing to worry about, and advised them to lose weight. Those interactions not only delayed diagnosis among Black women but also led many of them to mistrust clinicians and avoid the health care system altogether.

The failure of clinicians to take their complaints seriously is just one of the disparities affecting Black women. In reviewing the literature, Dr. Laughlin-Tommaso, who also serves as the associate dean for Education Diversity, Equity, and Inclusion at the Mayo Clinic, found that African American women experience two to three times the risk for fibroids, compared with White women, as well as earlier onset and more severe disease, as measured by number and size of the lesions. 

According to Dr. Katon, the etiology of fibroids is still poorly understood. “What we do know is that Black women are disproportionately exposed to a variety of factors that we have shown through observational studies are associated with increased risk of development of uterine fibroids.” 

The list includes factors like stress; interpersonal racism; early age at menarche; various indicators of poor diets, such as vitamin D deficiency; the use of certain beauty products, specifically hair straighteners; as well as exposure to air pollution and other environmental toxins.

Laughlin-Tommaso also pointed to historical disparities in management, citing a doubled risk for hysterectomy for Black women in a study published in 2007 despite survey data suggesting that Black women report being more interested in uterine-preserving therapies rather than a hysterectomy.
 

Breaking down barriers of access to new treatments

Dr. Laughlin-Tommaso looked at more recent trends in the management of fibroids using data from the multicenter COMPARE-UF study, which enrolled women between 2015 and 2020 undergoing fibroid treatment into a longitudinal registry to track their outcomes. She found that Black women underwent hysterectomies at a lower rate than did White women and were instead more likely to undergo myomectomy or UAE. 

Some of the change may reflect lack of approved minimally invasive procedures before 2000. “But now that we have expanded options, I think most women are opting not to have a hysterectomy,” Dr. Laughlin-Tommaso said.

Dr. Katon has research funding from the VA to look more closely at racial disparities in the treatment of fibroids. In a study published in April 2023, she reported some surprising trends. 

During the period from 2010 to 2018, she found that Black veterans diagnosed with fibroids were less likely than White veterans were to receive treatment, regardless of their age or the severity of their symptoms. This finding held even among women with anemia, which should have been a clear indication for treatment.

But, as in the COMPARE-UF study, the subset of Black veterans who received an interventional treatment were less likely than their White peers were to undergo hysterectomy in favor of a fertility-sparing treatment as their initial procedure. Dr. Katon called it a “welcome but unexpected finding.” 

But another significant barrier remains: The two newest types of procedures, RFA and guided focused ultrasound, are not commonly performed outside of tertiary care facilities. However, studies have found that all these procedures are cost effective (studies for myomectomy, UAE, MRgFUS, and TFA). The implementation of a category 1 billing code for laparoscopic RFA in 2017 has led more insurance companies to cover the service, and a category 1 code will be available for TFA effective January 2024. 

Although RFA does require investment in specialized equipment, which limits facilities from offering the procedure, any gynecologist who routinely performs hysteroscopy can easily learn to do TFA. And the VA, which is committed to eliminating disparities in women’s health, established a 2-year advanced fellowship in minimally invasive gynecologic surgery in 2022 to help expand their capacity to offer these procedures. 

The VA has been rapidly expanding their gynecology services, and Katon said that she is confident that ultrasound-guided procedures and RFA will become more available within the system. “I would say we’re keeping pace. And in some ways, you know, as a national system we may be positioned to actually outpace the rest of the U.S.”

Dr. Segars reported prior research funding for clinical trials from BioSpecifics Technologies, Bayer, Allergan, AbbVie, and ObsEva and currently receives funding from Myovant Sciences. Dr. Hansen-Lindner reported personal fees from Gynesonics. Dr. Singh, Dr. Laughlin-Tommaso, and Dr. Katon reported no financial conflicts of interest. 

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

In 2022, two colleagues from Johns Hopkins University, Baltimore, Bhuchitra Singh, MD, MPH, MS, MBA, and James Segars Jr., MD, reviewed the available literature to evaluate the effectiveness of newer minimally invasive therapies in reducing bleeding and improving the quality of life and control of symptoms linked to uterine fibroids. 

Their goal, according to Dr. Segars, a professor of obstetrics and gynecology and director of the division of women’s health research at Johns Hopkins, was to help guide clinicians and patients in making decisions about the use of the newer therapies, including radiofrequency ablation and ultrasound-guided removal of lesions. 

But he and Dr. Singh, the director of clinical research at the Howard W. and Georgeanna Seegar Jones Laboratory of Reproductive Sciences and Women’s Health Research, were surprised by their findings. “The outcomes were relatively the same,” Dr. Segars said. “All of the modalities lead to significant reduction in bleeding and other fibroid-related symptoms.” 

The data on long-term complications and risk for recurrence are sparse for some of the newer approaches, and not enough high-quality long-term studies have been conducted for the Food and Drug Administration to approve them as fertility-sparing treatments.

But perhaps, the biggest challenge now is to ensure that women can take advantage of these newer therapies, with large gaps in both the diagnosis of fibroids and geographic access to minimally invasive treatments.
 

A widespread condition widely underdiagnosed 

Uterine fibroids occur in most women (the incidence rises with age) and can be found in up to 70% of women by the time they reach menopause. Risk factors include family history, increasing interval since last birth, hypertension, and obesity. Increasing parity and use of oral contraceptives are protective.

But as many as 50% of cases go undiagnosed, and one reason for this is the failure of clinicians to dig deeply enough into women’s menstrual histories to diagnose fibroids. 

“The most common cause of anemia is heavy menstrual bleeding,” said Shannon Laughlin-Tommaso, MD, MPH, a professor of obstetrics and gynecology at Mayo Clinic in Rochester, Minn. She frequently sees patients who have already undergone colonoscopy to work-up the source of their anemia before anyone suspects that fibroids are the culprit. 

“When women tell us about their periods, what they’ve been told is normal [bleeding] – or what they’ve always had and considered normal – is actually kind of on the heavier spectrum,” she said. 

Ideally, treatment for uterine fibroids would fix abnormally prolonged or heavy menstrual bleeding, relieve pain, and ameliorate symptoms associated with an enlarged uterus, such as pelvic pressure, urinary frequency, and constipation. And the fibroids would never recur.

By those measures, hysterectomy fits the bill: Success rates in relieving symptoms are high, and the risk for recurrence is zero. But the procedure carries significant drawbacks: short-term complications of surgery, including infection, bleeding, and injury to the bowels and bladder along with potential long-term risks for cardiovascular disease, cancer, ovarian failure and premature menopause, depression, and decline in cognitive function. Those factors loom even larger for women who still hope to have children. 

For that reason, the American College of Obstetricians and Gynecologists recommends myomectomy, or surgical removal of individual fibroids, for women who desire uterine preservation or future pregnancy. And the literature here is solid, according to Dr. Singh, who found that 95% of myomectomy patients achieved control of their bleeding symptoms, whether it was via laparoscopy, hysteroscopy, or laparotomy. Up to 40% of women may develop new fibroids, or leiomyomas, within 3 years, although only 12.2% required a second surgery up to after 5 years. 

But myomectomy is invasive, requiring general anesthesia, incisions in the uterus, and stitches to close the organ. 

Newer techniques have emerged that can effectively treat symptoms of fibroids without requiring surgery. Uterine artery embolization (UAE), which involves passing a catheter into the femoral artery, or laparoscopic uterine artery occlusion can be used to cut off the blood supply of the fibroid. Other techniques, including focused ultrasound surgery and radiofrequency ablation (RFA), use various forms of energy to heat and ablate fibroids. The latter two can be performed in outpatient settings and often without general anesthesia.

Approved for use in 1994, UAE has the most data available, with reduction in the volume of fibroids and uterine tissue lasting up to 5 years, and rates of reintervention of 19%-38% between 2 and 5 years after the procedure. Dr. Singh’s review found that 79%-98.5% of recipients of the procedure reported declines in bleeding that persisted for several years, which is comparable to myomectomy. Quality of life and pain scores also showed good improvement, with follow-up in the different studies ranging from 12 months to over 5 years, the analysis showed.

UAE does have its drawbacks. In rare cases, embolization can deprive the entire uterus and ovaries of blood, which can cause ovarian dysfunction and potentially result in premature menopause, although this outcome is most common in women who are older than 45 years. The procedure can often also be painful enough that overnight hospitalization is required.

Focused ultrasound surgeries, which include magnetic resonance–guided focused ultrasound surgery (MRgFUS) and high-intensity focused ultrasound (HIFU), were approved by the FDA in 2004. Focused ultrasound waves pass through the abdominal wall and produce significant heating, causing a burn that destroys the targeted tissue without damaging surrounding tissue. As with UAE, improvements in fibroid-associated bleeding and measures of quality of life were similar to those after myomectomy up to 3 years later.

But Dr. Singh noted that both focused ultrasound and RFA can damage the skin or internal organs. “[As] always with the thermal interventions, there is the probability of skin as well as internal organs that might get the thermal energy if it’s not focused correctly on to the fibroid itself,” he said. In addition, MRgFUS is not an option for women who are not good candidates to undergo an MRI, such as those with claustrophobia or pacemakers.

Also, with focus ultrasound and RFA, “we do worry about that fibroid getting blood flow back,” which can lead to recurrence of heavy menstrual bleeding, Dr. Laughlin-Tommaso noted. 

Although data on RFA are limited to 12 months of follow-up, most women reported meaningful reductions in bleeding symptoms. Longer follow-up has been reported for bleeding symptoms after MRgFUS, with similar results up to 3 years later. 

For Leslie Hansen-Lindner, MD, chief of obstetrics and gynecology at Atrium Health in Charlotte, N.C., choosing the right procedure starts with a patient-centered conversation weighing the pros and cons of the options and the woman’s goals. 

“Is their goal to reduce the size and impact of their fibroid, bleed less, and have a better quality of life on their period?” Dr. Hansen-Lindner said. “Or is their goal to have the entire fibroid removed?” 

If the former, an RFA is appealing to many women. If the latter, laparoscopic or mini-laparotomy myomectomy might be a better choice. Although fewer than 10% of patients require surgical reintervention at 3 years of follow-up for RFA, myomectomy has more consistent long-term evidence showing that fewer women require re-intervention and preserve their fertility, she added. 

Age also plays a role in the decision: The closer a woman is to menopause, the less likely she is to experience a recurrence, so a less-invasive procedure is preferable. But for younger women hoping to become pregnant, the lower risk for recurrence and good prognosis for future fertility might sway the choice toward myomectomy.

The first laparoscopic RFA procedures were approved for uterine fibroids in 2012. Dr. Hansen-Lindner is a proponent of transcervical fibroid ablation (TFA), a newer RFA procedure that the FDA approved in 2018. Performed through the cervix, TFA requires no incisions and can generally be done without general anesthesia. Eligible candidates would be any woman with symptomatic fibroids, such as heavy menstrual bleeding, pain, or bulk symptoms. The contraindications are few.

“It’s going to come down to size and location of fibroids, and whether or not they would be accessible by the TFA,” Dr. Hansen-Lindner said. “I have to make sure that there isn’t a fibroid blocking their cervix and that the fibroids are accessible with this device.” 

TFA also is not suitable for removing most submucosal lesions, which typically must be removed by hysteroscopic myomectomy. Dr. Hansen-Lindner said that she often uses TFA in conjunction with hysteroscopic myomectomy for this scenario. Although data on pregnancy after RFA (including TFA), MRgFUS, and HIFU are lacking, Gynesonics, the manufacturer of the Sonata System (the device that delivers radiofrequency energy to shrink the fibroid) has documented 79 pregnancies among the 2,200 women who have undergone TFA in the United States since 2018.
 

Disparities hampering care

Uterine fibroids are a particular problem for Black women, whose symptoms are more likely to be ignored by clinicians, according to Jodie Katon, PhD, a core investigator at the Veterans Affairs Greater Los Angeles Center for the Study of Healthcare Innovation, Implementation and Policy. Dr. Katon cited studies in which Black women interviewed about their experiences reported a consistent theme: Clinicians dismissed their symptoms, told them these were nothing to worry about, and advised them to lose weight. Those interactions not only delayed diagnosis among Black women but also led many of them to mistrust clinicians and avoid the health care system altogether.

The failure of clinicians to take their complaints seriously is just one of the disparities affecting Black women. In reviewing the literature, Dr. Laughlin-Tommaso, who also serves as the associate dean for Education Diversity, Equity, and Inclusion at the Mayo Clinic, found that African American women experience two to three times the risk for fibroids, compared with White women, as well as earlier onset and more severe disease, as measured by number and size of the lesions. 

According to Dr. Katon, the etiology of fibroids is still poorly understood. “What we do know is that Black women are disproportionately exposed to a variety of factors that we have shown through observational studies are associated with increased risk of development of uterine fibroids.” 

The list includes factors like stress; interpersonal racism; early age at menarche; various indicators of poor diets, such as vitamin D deficiency; the use of certain beauty products, specifically hair straighteners; as well as exposure to air pollution and other environmental toxins.

Laughlin-Tommaso also pointed to historical disparities in management, citing a doubled risk for hysterectomy for Black women in a study published in 2007 despite survey data suggesting that Black women report being more interested in uterine-preserving therapies rather than a hysterectomy.
 

Breaking down barriers of access to new treatments

Dr. Laughlin-Tommaso looked at more recent trends in the management of fibroids using data from the multicenter COMPARE-UF study, which enrolled women between 2015 and 2020 undergoing fibroid treatment into a longitudinal registry to track their outcomes. She found that Black women underwent hysterectomies at a lower rate than did White women and were instead more likely to undergo myomectomy or UAE. 

Some of the change may reflect lack of approved minimally invasive procedures before 2000. “But now that we have expanded options, I think most women are opting not to have a hysterectomy,” Dr. Laughlin-Tommaso said.

Dr. Katon has research funding from the VA to look more closely at racial disparities in the treatment of fibroids. In a study published in April 2023, she reported some surprising trends. 

During the period from 2010 to 2018, she found that Black veterans diagnosed with fibroids were less likely than White veterans were to receive treatment, regardless of their age or the severity of their symptoms. This finding held even among women with anemia, which should have been a clear indication for treatment.

But, as in the COMPARE-UF study, the subset of Black veterans who received an interventional treatment were less likely than their White peers were to undergo hysterectomy in favor of a fertility-sparing treatment as their initial procedure. Dr. Katon called it a “welcome but unexpected finding.” 

But another significant barrier remains: The two newest types of procedures, RFA and guided focused ultrasound, are not commonly performed outside of tertiary care facilities. However, studies have found that all these procedures are cost effective (studies for myomectomy, UAE, MRgFUS, and TFA). The implementation of a category 1 billing code for laparoscopic RFA in 2017 has led more insurance companies to cover the service, and a category 1 code will be available for TFA effective January 2024. 

Although RFA does require investment in specialized equipment, which limits facilities from offering the procedure, any gynecologist who routinely performs hysteroscopy can easily learn to do TFA. And the VA, which is committed to eliminating disparities in women’s health, established a 2-year advanced fellowship in minimally invasive gynecologic surgery in 2022 to help expand their capacity to offer these procedures. 

The VA has been rapidly expanding their gynecology services, and Katon said that she is confident that ultrasound-guided procedures and RFA will become more available within the system. “I would say we’re keeping pace. And in some ways, you know, as a national system we may be positioned to actually outpace the rest of the U.S.”

Dr. Segars reported prior research funding for clinical trials from BioSpecifics Technologies, Bayer, Allergan, AbbVie, and ObsEva and currently receives funding from Myovant Sciences. Dr. Hansen-Lindner reported personal fees from Gynesonics. Dr. Singh, Dr. Laughlin-Tommaso, and Dr. Katon reported no financial conflicts of interest. 

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

Bariatric surgery continues to play a major role in obesity management despite the emergence of potent new weight-loss medications, according to two experts who spoke at an Endocrine Society science writers briefing.

“Bariatric surgery is safe, effective, and unfortunately underutilized for treating obesity and its complications,” said Jaime Almandoz, MD, medical director of the Weight Wellness Program at the University of Texas Southwestern Medical Center, Dallas.

Added Dr. Almandoz, who is triple board-certified in internal medicine, endocrinology, and obesity medicine, “Sometimes this gets presented in a linear fashion. ‘We’ll try lifestyle first, and if that doesn’t work, we’ll try medications, and if that doesn’t work, we’ll try surgery.’ But sometimes we might need to go straight to surgery instead of going through medications first, because it may be the most effective and evidence-based treatment for the person in the office in front of you.”

Moreover, he pointed out that currently, Medicare and many private insurers don’t cover antiobesity medications but do cover bariatric surgery.

Indeed, Srividya Kidambi, MD, professor and chief of endocrinology and molecular medicine at the Medical College of Wisconsin/Froedtert Hospital, Milwaukee, said there are certain types of patients for whom she might consider bariatric surgery first. One would be a person with a body mass index (BMI) greater than 40 kg/m² or with a BMI greater than 35 kg/m² and severe comorbidities.

Another, she said, would be young, relatively healthy people with obesity who have no comorbid conditions. “We know that if we stop the medication, the weight comes back. So, if I see a 20- to 25-year-old, am I really to commit them to lifelong therapy, or is bariatric surgery a better option in these cases? These drugs have not been around that long ... so I tend to recommend bariatric surgery in some patients.”

During the recent briefing, Dr. Almandoz summarized the evidence base for the benefits of bariatric surgery beyond weight loss, which include remission of type 2 diabetes and fatty liver disease, reduction of the risks of cardiovascular disease and cancer, and increased life expectancy.

“Everyone seems to be talking about GLP-1s for facilitating weight loss and treating obesity. ... What I want to do is provide a counterpoint to accessible therapies that are covered by more insurance plans and that may, in fact, have a better evidence base for treating obesity and its related complications,” he said in his introduction.

Bariatric surgery has been used for decades, and many centers of excellence perform it, with greatly reduced complication rates seen today than in the past. “It’s comparable to having a gallbladder surgery in terms of perioperative risk,” he noted.

Medicare and private insurers generally cover bariatric surgery for people with BMI greater than 40 kg/m2 or 35-39 kg/m² and at least one weight-related comorbidity, including type 2 diabetes, obstructive sleep apnea, hypertension, atherosclerotic disease, hyperlipidemia, and fatty liver disease.

Data suggest that weight reduction of about 3% can lead to meaningful reductions in blood glucose and triglyceride levels, but weight loss of 15% or greater is associated with reductions in cardiovascular events and type 2 diabetes remission. Lifestyle modification typically produces about 5% weight loss, compared with 20%-35% with bariatric surgery with sleeve gastrectomy or gastric bypass.

Older weight loss medications produced weight loss of 5%-10%; only the newer medications, semaglutide 2.4 mg and tirzepatide, come close to that. Weight loss with semaglutide is about 15%, while tirzepatide can produce weight loss of up to 22%. But, there are still issues with affordability, access, and lack of coverage, Dr. Almandoz noted.

One recent randomized trial of more than 400 individuals showed that bariatric surgery was more effective than lifestyle and medical therapies for treating metabolic-associated steatohepatitis without worsening of fibrosis.

Another showed that the surgery was associated with fewer major adverse liver outcomes among people who already had MASH. That same study showed a 70% reduction in cardiovascular events with bariatric surgery.

For patients with type 2 diabetes, numerous trials have demonstrated long-term remission and reduced A1c at 5 years and 10 years post surgery, along with reductions in microvascular and macrovascular complications.

Other data suggest that a shorter history of type 2 diabetes is among the factors predicting remission with bariatric surgery. “Oftentimes, both patients and providers will wait until the diabetes is quite advanced before they even have the conversation about weight loss or even bariatric surgery. This suggests that if we intervene earlier in the course of disease, when it is less severe and less advanced, we have a higher rate of causing remission in the diabetes,” Dr. Almandoz said.

The American Diabetes Association’s Standards of Care incorporate bariatric surgery as either “recommended” or “may be considered” to treat type 2 diabetes, depending on BMI level, for those who don’t achieve durable weight loss with nonsurgical methods, he noted.

A retrospective cohort study showed significant reductions in cardiovascular outcomes with bariatric surgery among people with baseline cardiovascular disease. “This is not just about bariatric surgery to cause weight loss. This is about the multitude of effects that happen when we treat obesity as a disease with highly effective therapies such as surgery,” he said.

Even cancer risk and cancer-related mortality were significantly reduced with bariatric surgery, another study found.

And in the long-term Swedish Obese Subjects Study, among people with obesity, bariatric surgery was associated with a 3-year increase in life expectancy, compared with not undergoing surgery.

However, Dr. Almandoz also pointed out that some patients may benefit from both weight-loss medication and bariatric surgery. “Once someone has undergone pharmacotherapy, there may still be a role for bariatric procedures in helping to optimize body weight and control body weight long term. And likewise for those who have undergone bariatric surgery, there’s also a role for pharmacotherapy in terms of treating insufficient weight loss or weight recurrence after bariatric surgery. ... So I think there’s clearly a role for integration of therapies.”

Dr. Almandoz serves as consultant/advisory board member for Novo Nordisk, Boehringer Ingelheim, and Eli Lilly. Dr. Kidambi is director of TOPS Center for Metabolic Research and is medical editor of TOPS Magazine, for which her institution receives an honorarium.

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

Bariatric surgery continues to play a major role in obesity management despite the emergence of potent new weight-loss medications, according to two experts who spoke at an Endocrine Society science writers briefing.

“Bariatric surgery is safe, effective, and unfortunately underutilized for treating obesity and its complications,” said Jaime Almandoz, MD, medical director of the Weight Wellness Program at the University of Texas Southwestern Medical Center, Dallas.

Added Dr. Almandoz, who is triple board-certified in internal medicine, endocrinology, and obesity medicine, “Sometimes this gets presented in a linear fashion. ‘We’ll try lifestyle first, and if that doesn’t work, we’ll try medications, and if that doesn’t work, we’ll try surgery.’ But sometimes we might need to go straight to surgery instead of going through medications first, because it may be the most effective and evidence-based treatment for the person in the office in front of you.”

Moreover, he pointed out that currently, Medicare and many private insurers don’t cover antiobesity medications but do cover bariatric surgery.

Indeed, Srividya Kidambi, MD, professor and chief of endocrinology and molecular medicine at the Medical College of Wisconsin/Froedtert Hospital, Milwaukee, said there are certain types of patients for whom she might consider bariatric surgery first. One would be a person with a body mass index (BMI) greater than 40 kg/m² or with a BMI greater than 35 kg/m² and severe comorbidities.

Another, she said, would be young, relatively healthy people with obesity who have no comorbid conditions. “We know that if we stop the medication, the weight comes back. So, if I see a 20- to 25-year-old, am I really to commit them to lifelong therapy, or is bariatric surgery a better option in these cases? These drugs have not been around that long ... so I tend to recommend bariatric surgery in some patients.”

During the recent briefing, Dr. Almandoz summarized the evidence base for the benefits of bariatric surgery beyond weight loss, which include remission of type 2 diabetes and fatty liver disease, reduction of the risks of cardiovascular disease and cancer, and increased life expectancy.

“Everyone seems to be talking about GLP-1s for facilitating weight loss and treating obesity. ... What I want to do is provide a counterpoint to accessible therapies that are covered by more insurance plans and that may, in fact, have a better evidence base for treating obesity and its related complications,” he said in his introduction.

Bariatric surgery has been used for decades, and many centers of excellence perform it, with greatly reduced complication rates seen today than in the past. “It’s comparable to having a gallbladder surgery in terms of perioperative risk,” he noted.

Medicare and private insurers generally cover bariatric surgery for people with BMI greater than 40 kg/m2 or 35-39 kg/m² and at least one weight-related comorbidity, including type 2 diabetes, obstructive sleep apnea, hypertension, atherosclerotic disease, hyperlipidemia, and fatty liver disease.

Data suggest that weight reduction of about 3% can lead to meaningful reductions in blood glucose and triglyceride levels, but weight loss of 15% or greater is associated with reductions in cardiovascular events and type 2 diabetes remission. Lifestyle modification typically produces about 5% weight loss, compared with 20%-35% with bariatric surgery with sleeve gastrectomy or gastric bypass.

Older weight loss medications produced weight loss of 5%-10%; only the newer medications, semaglutide 2.4 mg and tirzepatide, come close to that. Weight loss with semaglutide is about 15%, while tirzepatide can produce weight loss of up to 22%. But, there are still issues with affordability, access, and lack of coverage, Dr. Almandoz noted.

One recent randomized trial of more than 400 individuals showed that bariatric surgery was more effective than lifestyle and medical therapies for treating metabolic-associated steatohepatitis without worsening of fibrosis.

Another showed that the surgery was associated with fewer major adverse liver outcomes among people who already had MASH. That same study showed a 70% reduction in cardiovascular events with bariatric surgery.

For patients with type 2 diabetes, numerous trials have demonstrated long-term remission and reduced A1c at 5 years and 10 years post surgery, along with reductions in microvascular and macrovascular complications.

Other data suggest that a shorter history of type 2 diabetes is among the factors predicting remission with bariatric surgery. “Oftentimes, both patients and providers will wait until the diabetes is quite advanced before they even have the conversation about weight loss or even bariatric surgery. This suggests that if we intervene earlier in the course of disease, when it is less severe and less advanced, we have a higher rate of causing remission in the diabetes,” Dr. Almandoz said.

The American Diabetes Association’s Standards of Care incorporate bariatric surgery as either “recommended” or “may be considered” to treat type 2 diabetes, depending on BMI level, for those who don’t achieve durable weight loss with nonsurgical methods, he noted.

A retrospective cohort study showed significant reductions in cardiovascular outcomes with bariatric surgery among people with baseline cardiovascular disease. “This is not just about bariatric surgery to cause weight loss. This is about the multitude of effects that happen when we treat obesity as a disease with highly effective therapies such as surgery,” he said.

Even cancer risk and cancer-related mortality were significantly reduced with bariatric surgery, another study found.

And in the long-term Swedish Obese Subjects Study, among people with obesity, bariatric surgery was associated with a 3-year increase in life expectancy, compared with not undergoing surgery.

However, Dr. Almandoz also pointed out that some patients may benefit from both weight-loss medication and bariatric surgery. “Once someone has undergone pharmacotherapy, there may still be a role for bariatric procedures in helping to optimize body weight and control body weight long term. And likewise for those who have undergone bariatric surgery, there’s also a role for pharmacotherapy in terms of treating insufficient weight loss or weight recurrence after bariatric surgery. ... So I think there’s clearly a role for integration of therapies.”

Dr. Almandoz serves as consultant/advisory board member for Novo Nordisk, Boehringer Ingelheim, and Eli Lilly. Dr. Kidambi is director of TOPS Center for Metabolic Research and is medical editor of TOPS Magazine, for which her institution receives an honorarium.

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

Bariatric surgery continues to play a major role in obesity management despite the emergence of potent new weight-loss medications, according to two experts who spoke at an Endocrine Society science writers briefing.

“Bariatric surgery is safe, effective, and unfortunately underutilized for treating obesity and its complications,” said Jaime Almandoz, MD, medical director of the Weight Wellness Program at the University of Texas Southwestern Medical Center, Dallas.

Added Dr. Almandoz, who is triple board-certified in internal medicine, endocrinology, and obesity medicine, “Sometimes this gets presented in a linear fashion. ‘We’ll try lifestyle first, and if that doesn’t work, we’ll try medications, and if that doesn’t work, we’ll try surgery.’ But sometimes we might need to go straight to surgery instead of going through medications first, because it may be the most effective and evidence-based treatment for the person in the office in front of you.”

Moreover, he pointed out that currently, Medicare and many private insurers don’t cover antiobesity medications but do cover bariatric surgery.

Indeed, Srividya Kidambi, MD, professor and chief of endocrinology and molecular medicine at the Medical College of Wisconsin/Froedtert Hospital, Milwaukee, said there are certain types of patients for whom she might consider bariatric surgery first. One would be a person with a body mass index (BMI) greater than 40 kg/m² or with a BMI greater than 35 kg/m² and severe comorbidities.

Another, she said, would be young, relatively healthy people with obesity who have no comorbid conditions. “We know that if we stop the medication, the weight comes back. So, if I see a 20- to 25-year-old, am I really to commit them to lifelong therapy, or is bariatric surgery a better option in these cases? These drugs have not been around that long ... so I tend to recommend bariatric surgery in some patients.”

During the recent briefing, Dr. Almandoz summarized the evidence base for the benefits of bariatric surgery beyond weight loss, which include remission of type 2 diabetes and fatty liver disease, reduction of the risks of cardiovascular disease and cancer, and increased life expectancy.

“Everyone seems to be talking about GLP-1s for facilitating weight loss and treating obesity. ... What I want to do is provide a counterpoint to accessible therapies that are covered by more insurance plans and that may, in fact, have a better evidence base for treating obesity and its related complications,” he said in his introduction.

Bariatric surgery has been used for decades, and many centers of excellence perform it, with greatly reduced complication rates seen today than in the past. “It’s comparable to having a gallbladder surgery in terms of perioperative risk,” he noted.

Medicare and private insurers generally cover bariatric surgery for people with BMI greater than 40 kg/m2 or 35-39 kg/m² and at least one weight-related comorbidity, including type 2 diabetes, obstructive sleep apnea, hypertension, atherosclerotic disease, hyperlipidemia, and fatty liver disease.

Data suggest that weight reduction of about 3% can lead to meaningful reductions in blood glucose and triglyceride levels, but weight loss of 15% or greater is associated with reductions in cardiovascular events and type 2 diabetes remission. Lifestyle modification typically produces about 5% weight loss, compared with 20%-35% with bariatric surgery with sleeve gastrectomy or gastric bypass.

Older weight loss medications produced weight loss of 5%-10%; only the newer medications, semaglutide 2.4 mg and tirzepatide, come close to that. Weight loss with semaglutide is about 15%, while tirzepatide can produce weight loss of up to 22%. But, there are still issues with affordability, access, and lack of coverage, Dr. Almandoz noted.

One recent randomized trial of more than 400 individuals showed that bariatric surgery was more effective than lifestyle and medical therapies for treating metabolic-associated steatohepatitis without worsening of fibrosis.

Another showed that the surgery was associated with fewer major adverse liver outcomes among people who already had MASH. That same study showed a 70% reduction in cardiovascular events with bariatric surgery.

For patients with type 2 diabetes, numerous trials have demonstrated long-term remission and reduced A1c at 5 years and 10 years post surgery, along with reductions in microvascular and macrovascular complications.

Other data suggest that a shorter history of type 2 diabetes is among the factors predicting remission with bariatric surgery. “Oftentimes, both patients and providers will wait until the diabetes is quite advanced before they even have the conversation about weight loss or even bariatric surgery. This suggests that if we intervene earlier in the course of disease, when it is less severe and less advanced, we have a higher rate of causing remission in the diabetes,” Dr. Almandoz said.

The American Diabetes Association’s Standards of Care incorporate bariatric surgery as either “recommended” or “may be considered” to treat type 2 diabetes, depending on BMI level, for those who don’t achieve durable weight loss with nonsurgical methods, he noted.

A retrospective cohort study showed significant reductions in cardiovascular outcomes with bariatric surgery among people with baseline cardiovascular disease. “This is not just about bariatric surgery to cause weight loss. This is about the multitude of effects that happen when we treat obesity as a disease with highly effective therapies such as surgery,” he said.

Even cancer risk and cancer-related mortality were significantly reduced with bariatric surgery, another study found.

And in the long-term Swedish Obese Subjects Study, among people with obesity, bariatric surgery was associated with a 3-year increase in life expectancy, compared with not undergoing surgery.

However, Dr. Almandoz also pointed out that some patients may benefit from both weight-loss medication and bariatric surgery. “Once someone has undergone pharmacotherapy, there may still be a role for bariatric procedures in helping to optimize body weight and control body weight long term. And likewise for those who have undergone bariatric surgery, there’s also a role for pharmacotherapy in terms of treating insufficient weight loss or weight recurrence after bariatric surgery. ... So I think there’s clearly a role for integration of therapies.”

Dr. Almandoz serves as consultant/advisory board member for Novo Nordisk, Boehringer Ingelheim, and Eli Lilly. Dr. Kidambi is director of TOPS Center for Metabolic Research and is medical editor of TOPS Magazine, for which her institution receives an honorarium.

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

In April 2020, as many Americans prepared to spend the Easter holiday in lockdown, pop star Mariah Carey released a video honoring the “sacrifices and courage” of frontline workers battling COVID-19 – her 1993 hit, “Hero.”

“The sorrow that you know will melt away,” Ms. Carey sang. “When you feel like hope is gone,” the song continued, strength and answers can be found within, and “a hero lies in you.”

For health care professionals, the reality of 2020 wasn’t quite so uplifting. PPE shortages and spillover ICUs had many feeling helpless, exhausted, and overwhelmed. Few if any medical professionals felt their sorrows “melt away.”

We can’t expect depth and nuance from pop songs, but we can find in them the imagery that runs through our culture. The “hero narrative” – the idea that doctors, nurses, and others in health care have superhuman endurance and selflessness – has long been an undercurrent in the medical field.

And yet, without a workforce willing to perform without adequate sleep, food, or time off, the health care system couldn’t function, says Brian Park, MD, MPH, a family medicine physician at Oregon Health & Science University, Portland. At many academic health centers, for example, residents are “the bedrock of the workforce,” he explains. If they didn’t work 80-100 hours per week, those systems wouldn’t exist.

So, how do we look at the health care system in a way that is both grateful and critical, Dr. Park wonders. “How do we honor extreme acts of heroism and also acknowledge that the system sometimes gets by on the acts of heroes to patch up some of the brokenness and fragmentation within it?”

Put simply: What makes “heroism” necessary in the first place?
 

Heroes are determined

Ala Stanford, MD, a pediatric surgeon in Philadelphia, has frequently been called a “health care hero.” Given the title by CNN in 2021, she has received numerous other awards and accolades, featured in Fortune Magazine’s “World’s 50 Greatest Leaders” in 2021 and USA Today’s “Women of the Year” in 2022.

In 2020, Dr. Stanford was sheltering in place and watching “way too much” cable news. “They would play solemn music and show photos of all the people who had died,” she recalls. “I thought, ‘All these people are Black or brown. What is going on?’”

The standard explanation was that people of color were more vulnerable because they were more likely to be essential workers or have chronic health conditions. But Dr. Stanford believed this was only part of the story. The reason she saw that local Black communities had higher positivity rates was because people couldn’t get a COVID test.

Dr. Stanford got call after call from Philadelphians who had been turned away from testing centers. When she questioned colleagues, “they gave me every reason under the sun,” Dr. Stanford says. “It was because someone took public transportation, and they were only testing people in cars, or because they weren’t over 65, or because they didn’t have other comorbid health conditions, or because they weren’t a health care worker, or because they hadn’t traveled to China ...” The list went on.

Dr. Stanford appealed to local, state, and federal health authorities. Finally, she took matters into her own hands. She found tests, packed a van with masks, gowns, and gloves, and drove across the city going door to door. Eventually, she organized testing in the parking lots of Black churches, sometimes seeing more than 400 people per day.

The services were funded entirely through her own bank account and donations until she was eventually awarded a CDC grant through the Coronavirus Aid, Relief, and Economic Security (CARES) Act of 2020 and began to receive contracts from the city.

Since then, Dr. Stanford’s mission has evolved. She and her team provided COVID vaccinations to thousands, and in 2021, opened the Dr. Ala Stanford Center for Health Equity. The center offers primary care for all ages in underserved communities.

Still, Dr. Stanford doesn’t think of herself as a hero, and she stresses that many other people contributed to her success. “I think the world was on fire, and we were all firefighters,” Dr. Stanford says. “Someone said to me, ‘Ala, you ran to the fire and everyone else was running away from it, and you didn’t have to.’ … I feel like I was able to galvanize people to realize the power that they actually had. Maybe independently, they couldn’t do a whole lot, but collectively, we were a force.”
 

Heroes are selfless

Nicole Jackson, RN, an emergency room manager and nurse at Advocate Trinity Hospital in Chicago, was recently honored as a Health Care Hero by the American Red Cross of Greater Chicago.

On June 23, 2022, Jackson’s emergency department was understaffed and struggling with an influx of patients when three gunshot victims arrived. Two needed to be transferred to a trauma center, and one – with multiple gunshot wounds – required a critical care nurse in the ambulance. But the ETA for that transport was 90 minutes, which meant the patient might not survive. Although Ms. Jackson was already working beyond her shift, she rode in the ambulance with the patient herself and probably saved his life.

While this incident stood out to a colleague who nominated her for the Red Cross award, Ms. Jackson finds herself working extra hours fairly often. “Since COVID, that’s pretty much been like any other hospital,” she says. “We’ve had staffing challenges that we work through every day. So, the nurses come, they show up, and they do the best that they can with what we have to keep our patients safe.”

A 2022 survey by McKinsey estimated that by 2025, there could be a gap of 200,000 to 450,000 nurses in the United States. A two-year impact assessment from the American Nurses Foundation found that among more than 12,500 nurses, 40% were considering leaving their positions before the pandemic. By 2022, that number had jumped to 52% with the top reasons being insufficient staffing and negative effects on health and well-being.

Can the “hero narrative” help that situation? Ms. Jackson says she doesn’t see herself as a hero, but the supportive environment and gestures of recognition by staff do make her feel appreciated. These include daily messages offering “kudos” and nominations for the DAISY Award, which she herself received in 2022.

“I have people who I have encouraged to become nurses,” Ms. Jackson says, “and when they saw [the award], they were really excited about becoming a nurse.”
 

Heroes are strong

Jasmine Marcelin, MD, an infectious disease physician with Nebraska Medicine in Omaha, understands the need for heroes as symbols and sources of inspiration. Dr. Marcelin is a fan of the superhero movie genre. There is value, she says, in feeling hope and excitement while watching Superman or Wonder Woman save the day. Who doesn’t want to believe (if only briefly) that the good guys will always win?

In reality, Dr. Marcelin says, “none of us are invincible.” And it’s dangerous to forget that “the people behind the symbols are also human.”

In 2021, Dr. Marcelin gave a TEDx talk entitled, “The Myth of the Health Care Hero.” In it she discussed the extreme physical and mental toll of the pandemic on health care workers and urged her audience to think less about extravagant praise and more about their personal responsibilities. “We don’t want or need to be called heroes,” Dr. Marcelin said. “Right now, our love language is action. We need your help, and we cannot save the world on our own.”

Dr. Marcelin also sees links between superhuman expectations and the high levels of burnout in the medical field.

“It’s a systemic issue,” she explains, “where it requires a revamping and revitalization of the entire psyche of health care to recognize that the people working within this profession are human. And the things that we think and feel and need are the same as anybody else.”
 

Heroes are self-sacrificing 


Well-being, burnout, and disengagement in health care has become a focus for Oregon Health & Science’s Dr. Park, who is also director of RELATE Lab, an organization that aims to make health care more human-centered and equitable through leadership training, research, and community organizing.

For him, hearing neighbors banging pots and pans during the early pandemic was complicated. “The first phase for me was, ‘Thank you. I feel seen. I feel appreciated,’ ” he says. “Yes, I’m wearing a mask. I’m going in. I’m changing in the garage when I come home, so my kid and my partner don’t get sick.”

But after a while, the cheers started to feel like pressure. “Have I done anything heroic today?” Dr. Park asked himself. “Have I been as heroic as my friend who is in the hospital in the ICU? I don’t deserve this, so don’t bang those pots and pans for me.”

When your identity becomes about being a hero, Dr. Park says, when that becomes the standard by which you measure yourself, the result is often a sense of shame.

“I think a lot of people feel ashamed that they feel burnout,” he says, “because they’re supposed to be heroes, putting on their capes and masks. They’re waking up and saying, ‘I’m exhausted, and I can’t play that part today. But I know that’s the social expectation of me.’ “
 

Heroes are noble

There may not be a clear solution, but for many health care professionals, symbolic gestures alone are inadequate and, in certain cases, insulting.

On Doctor’s Day 2023, Alok Patel, MD, a pediatric hospitalist, tweeted a photo of an appreciation “gift” for staff from an unnamed hospital. The small items had metaphorical meanings – a rubber band “as a reminder to stay flexible,” a quarter “as a reminder to ‘call’ for help,” etc.

“Welcome to how you give thanks to ‘health care heroes,’ ” Dr. Patel tweeted.

For Dr. Patel, the issue is not lavish gifts but a need for an attitude shift. He recalls colleagues who felt ashamed asking for mental health services or time off, “because they were bombarded by the hero narrative, by the manufactured pressure that they needed to put their jobs above their own health – because that’s what ‘heroes’ do. I’m willing to bet most physicians would rather receive a sincere email with a transparent plan to better support health care workers than any Doctor’s Day gift,” he says.

In Dr. Marcelin’s TEDx talk, she quotes Spider-Man’s classic adage, “With great power, comes great responsibility.” She argues that this motto doesn’t just apply to those who can fly or deflect bullets; that’s not what heroism is. In fact, most people have their own definition of the word.

For Dr. Stanford, a hero is “someone who is selfless, putting the needs of others before their own.” Dr. Park believes there are no individual heroes. “It’s the work of the collective that’s truly heroic.”

By those standards, clearly anyone can step up, offer help, act with courage and kindness, and be heroic. “We humans, as ordinary as we are, can be extraordinary by using our power to do what’s right,” Dr. Marcelin says, “because there’s no such thing as health care heroes, just good people doing the right thing.”

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

In April 2020, as many Americans prepared to spend the Easter holiday in lockdown, pop star Mariah Carey released a video honoring the “sacrifices and courage” of frontline workers battling COVID-19 – her 1993 hit, “Hero.”

“The sorrow that you know will melt away,” Ms. Carey sang. “When you feel like hope is gone,” the song continued, strength and answers can be found within, and “a hero lies in you.”

For health care professionals, the reality of 2020 wasn’t quite so uplifting. PPE shortages and spillover ICUs had many feeling helpless, exhausted, and overwhelmed. Few if any medical professionals felt their sorrows “melt away.”

We can’t expect depth and nuance from pop songs, but we can find in them the imagery that runs through our culture. The “hero narrative” – the idea that doctors, nurses, and others in health care have superhuman endurance and selflessness – has long been an undercurrent in the medical field.

And yet, without a workforce willing to perform without adequate sleep, food, or time off, the health care system couldn’t function, says Brian Park, MD, MPH, a family medicine physician at Oregon Health & Science University, Portland. At many academic health centers, for example, residents are “the bedrock of the workforce,” he explains. If they didn’t work 80-100 hours per week, those systems wouldn’t exist.

So, how do we look at the health care system in a way that is both grateful and critical, Dr. Park wonders. “How do we honor extreme acts of heroism and also acknowledge that the system sometimes gets by on the acts of heroes to patch up some of the brokenness and fragmentation within it?”

Put simply: What makes “heroism” necessary in the first place?
 

Heroes are determined

Ala Stanford, MD, a pediatric surgeon in Philadelphia, has frequently been called a “health care hero.” Given the title by CNN in 2021, she has received numerous other awards and accolades, featured in Fortune Magazine’s “World’s 50 Greatest Leaders” in 2021 and USA Today’s “Women of the Year” in 2022.

In 2020, Dr. Stanford was sheltering in place and watching “way too much” cable news. “They would play solemn music and show photos of all the people who had died,” she recalls. “I thought, ‘All these people are Black or brown. What is going on?’”

The standard explanation was that people of color were more vulnerable because they were more likely to be essential workers or have chronic health conditions. But Dr. Stanford believed this was only part of the story. The reason she saw that local Black communities had higher positivity rates was because people couldn’t get a COVID test.

Dr. Stanford got call after call from Philadelphians who had been turned away from testing centers. When she questioned colleagues, “they gave me every reason under the sun,” Dr. Stanford says. “It was because someone took public transportation, and they were only testing people in cars, or because they weren’t over 65, or because they didn’t have other comorbid health conditions, or because they weren’t a health care worker, or because they hadn’t traveled to China ...” The list went on.

Dr. Stanford appealed to local, state, and federal health authorities. Finally, she took matters into her own hands. She found tests, packed a van with masks, gowns, and gloves, and drove across the city going door to door. Eventually, she organized testing in the parking lots of Black churches, sometimes seeing more than 400 people per day.

The services were funded entirely through her own bank account and donations until she was eventually awarded a CDC grant through the Coronavirus Aid, Relief, and Economic Security (CARES) Act of 2020 and began to receive contracts from the city.

Since then, Dr. Stanford’s mission has evolved. She and her team provided COVID vaccinations to thousands, and in 2021, opened the Dr. Ala Stanford Center for Health Equity. The center offers primary care for all ages in underserved communities.

Still, Dr. Stanford doesn’t think of herself as a hero, and she stresses that many other people contributed to her success. “I think the world was on fire, and we were all firefighters,” Dr. Stanford says. “Someone said to me, ‘Ala, you ran to the fire and everyone else was running away from it, and you didn’t have to.’ … I feel like I was able to galvanize people to realize the power that they actually had. Maybe independently, they couldn’t do a whole lot, but collectively, we were a force.”
 

Heroes are selfless

Nicole Jackson, RN, an emergency room manager and nurse at Advocate Trinity Hospital in Chicago, was recently honored as a Health Care Hero by the American Red Cross of Greater Chicago.

On June 23, 2022, Jackson’s emergency department was understaffed and struggling with an influx of patients when three gunshot victims arrived. Two needed to be transferred to a trauma center, and one – with multiple gunshot wounds – required a critical care nurse in the ambulance. But the ETA for that transport was 90 minutes, which meant the patient might not survive. Although Ms. Jackson was already working beyond her shift, she rode in the ambulance with the patient herself and probably saved his life.

While this incident stood out to a colleague who nominated her for the Red Cross award, Ms. Jackson finds herself working extra hours fairly often. “Since COVID, that’s pretty much been like any other hospital,” she says. “We’ve had staffing challenges that we work through every day. So, the nurses come, they show up, and they do the best that they can with what we have to keep our patients safe.”

A 2022 survey by McKinsey estimated that by 2025, there could be a gap of 200,000 to 450,000 nurses in the United States. A two-year impact assessment from the American Nurses Foundation found that among more than 12,500 nurses, 40% were considering leaving their positions before the pandemic. By 2022, that number had jumped to 52% with the top reasons being insufficient staffing and negative effects on health and well-being.

Can the “hero narrative” help that situation? Ms. Jackson says she doesn’t see herself as a hero, but the supportive environment and gestures of recognition by staff do make her feel appreciated. These include daily messages offering “kudos” and nominations for the DAISY Award, which she herself received in 2022.

“I have people who I have encouraged to become nurses,” Ms. Jackson says, “and when they saw [the award], they were really excited about becoming a nurse.”
 

Heroes are strong

Jasmine Marcelin, MD, an infectious disease physician with Nebraska Medicine in Omaha, understands the need for heroes as symbols and sources of inspiration. Dr. Marcelin is a fan of the superhero movie genre. There is value, she says, in feeling hope and excitement while watching Superman or Wonder Woman save the day. Who doesn’t want to believe (if only briefly) that the good guys will always win?

In reality, Dr. Marcelin says, “none of us are invincible.” And it’s dangerous to forget that “the people behind the symbols are also human.”

In 2021, Dr. Marcelin gave a TEDx talk entitled, “The Myth of the Health Care Hero.” In it she discussed the extreme physical and mental toll of the pandemic on health care workers and urged her audience to think less about extravagant praise and more about their personal responsibilities. “We don’t want or need to be called heroes,” Dr. Marcelin said. “Right now, our love language is action. We need your help, and we cannot save the world on our own.”

Dr. Marcelin also sees links between superhuman expectations and the high levels of burnout in the medical field.

“It’s a systemic issue,” she explains, “where it requires a revamping and revitalization of the entire psyche of health care to recognize that the people working within this profession are human. And the things that we think and feel and need are the same as anybody else.”
 

Heroes are self-sacrificing 


Well-being, burnout, and disengagement in health care has become a focus for Oregon Health & Science’s Dr. Park, who is also director of RELATE Lab, an organization that aims to make health care more human-centered and equitable through leadership training, research, and community organizing.

For him, hearing neighbors banging pots and pans during the early pandemic was complicated. “The first phase for me was, ‘Thank you. I feel seen. I feel appreciated,’ ” he says. “Yes, I’m wearing a mask. I’m going in. I’m changing in the garage when I come home, so my kid and my partner don’t get sick.”

But after a while, the cheers started to feel like pressure. “Have I done anything heroic today?” Dr. Park asked himself. “Have I been as heroic as my friend who is in the hospital in the ICU? I don’t deserve this, so don’t bang those pots and pans for me.”

When your identity becomes about being a hero, Dr. Park says, when that becomes the standard by which you measure yourself, the result is often a sense of shame.

“I think a lot of people feel ashamed that they feel burnout,” he says, “because they’re supposed to be heroes, putting on their capes and masks. They’re waking up and saying, ‘I’m exhausted, and I can’t play that part today. But I know that’s the social expectation of me.’ “
 

Heroes are noble

There may not be a clear solution, but for many health care professionals, symbolic gestures alone are inadequate and, in certain cases, insulting.

On Doctor’s Day 2023, Alok Patel, MD, a pediatric hospitalist, tweeted a photo of an appreciation “gift” for staff from an unnamed hospital. The small items had metaphorical meanings – a rubber band “as a reminder to stay flexible,” a quarter “as a reminder to ‘call’ for help,” etc.

“Welcome to how you give thanks to ‘health care heroes,’ ” Dr. Patel tweeted.

For Dr. Patel, the issue is not lavish gifts but a need for an attitude shift. He recalls colleagues who felt ashamed asking for mental health services or time off, “because they were bombarded by the hero narrative, by the manufactured pressure that they needed to put their jobs above their own health – because that’s what ‘heroes’ do. I’m willing to bet most physicians would rather receive a sincere email with a transparent plan to better support health care workers than any Doctor’s Day gift,” he says.

In Dr. Marcelin’s TEDx talk, she quotes Spider-Man’s classic adage, “With great power, comes great responsibility.” She argues that this motto doesn’t just apply to those who can fly or deflect bullets; that’s not what heroism is. In fact, most people have their own definition of the word.

For Dr. Stanford, a hero is “someone who is selfless, putting the needs of others before their own.” Dr. Park believes there are no individual heroes. “It’s the work of the collective that’s truly heroic.”

By those standards, clearly anyone can step up, offer help, act with courage and kindness, and be heroic. “We humans, as ordinary as we are, can be extraordinary by using our power to do what’s right,” Dr. Marcelin says, “because there’s no such thing as health care heroes, just good people doing the right thing.”

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

In April 2020, as many Americans prepared to spend the Easter holiday in lockdown, pop star Mariah Carey released a video honoring the “sacrifices and courage” of frontline workers battling COVID-19 – her 1993 hit, “Hero.”

“The sorrow that you know will melt away,” Ms. Carey sang. “When you feel like hope is gone,” the song continued, strength and answers can be found within, and “a hero lies in you.”

For health care professionals, the reality of 2020 wasn’t quite so uplifting. PPE shortages and spillover ICUs had many feeling helpless, exhausted, and overwhelmed. Few if any medical professionals felt their sorrows “melt away.”

We can’t expect depth and nuance from pop songs, but we can find in them the imagery that runs through our culture. The “hero narrative” – the idea that doctors, nurses, and others in health care have superhuman endurance and selflessness – has long been an undercurrent in the medical field.

And yet, without a workforce willing to perform without adequate sleep, food, or time off, the health care system couldn’t function, says Brian Park, MD, MPH, a family medicine physician at Oregon Health & Science University, Portland. At many academic health centers, for example, residents are “the bedrock of the workforce,” he explains. If they didn’t work 80-100 hours per week, those systems wouldn’t exist.

So, how do we look at the health care system in a way that is both grateful and critical, Dr. Park wonders. “How do we honor extreme acts of heroism and also acknowledge that the system sometimes gets by on the acts of heroes to patch up some of the brokenness and fragmentation within it?”

Put simply: What makes “heroism” necessary in the first place?
 

Heroes are determined

Ala Stanford, MD, a pediatric surgeon in Philadelphia, has frequently been called a “health care hero.” Given the title by CNN in 2021, she has received numerous other awards and accolades, featured in Fortune Magazine’s “World’s 50 Greatest Leaders” in 2021 and USA Today’s “Women of the Year” in 2022.

In 2020, Dr. Stanford was sheltering in place and watching “way too much” cable news. “They would play solemn music and show photos of all the people who had died,” she recalls. “I thought, ‘All these people are Black or brown. What is going on?’”

The standard explanation was that people of color were more vulnerable because they were more likely to be essential workers or have chronic health conditions. But Dr. Stanford believed this was only part of the story. The reason she saw that local Black communities had higher positivity rates was because people couldn’t get a COVID test.

Dr. Stanford got call after call from Philadelphians who had been turned away from testing centers. When she questioned colleagues, “they gave me every reason under the sun,” Dr. Stanford says. “It was because someone took public transportation, and they were only testing people in cars, or because they weren’t over 65, or because they didn’t have other comorbid health conditions, or because they weren’t a health care worker, or because they hadn’t traveled to China ...” The list went on.

Dr. Stanford appealed to local, state, and federal health authorities. Finally, she took matters into her own hands. She found tests, packed a van with masks, gowns, and gloves, and drove across the city going door to door. Eventually, she organized testing in the parking lots of Black churches, sometimes seeing more than 400 people per day.

The services were funded entirely through her own bank account and donations until she was eventually awarded a CDC grant through the Coronavirus Aid, Relief, and Economic Security (CARES) Act of 2020 and began to receive contracts from the city.

Since then, Dr. Stanford’s mission has evolved. She and her team provided COVID vaccinations to thousands, and in 2021, opened the Dr. Ala Stanford Center for Health Equity. The center offers primary care for all ages in underserved communities.

Still, Dr. Stanford doesn’t think of herself as a hero, and she stresses that many other people contributed to her success. “I think the world was on fire, and we were all firefighters,” Dr. Stanford says. “Someone said to me, ‘Ala, you ran to the fire and everyone else was running away from it, and you didn’t have to.’ … I feel like I was able to galvanize people to realize the power that they actually had. Maybe independently, they couldn’t do a whole lot, but collectively, we were a force.”
 

Heroes are selfless

Nicole Jackson, RN, an emergency room manager and nurse at Advocate Trinity Hospital in Chicago, was recently honored as a Health Care Hero by the American Red Cross of Greater Chicago.

On June 23, 2022, Jackson’s emergency department was understaffed and struggling with an influx of patients when three gunshot victims arrived. Two needed to be transferred to a trauma center, and one – with multiple gunshot wounds – required a critical care nurse in the ambulance. But the ETA for that transport was 90 minutes, which meant the patient might not survive. Although Ms. Jackson was already working beyond her shift, she rode in the ambulance with the patient herself and probably saved his life.

While this incident stood out to a colleague who nominated her for the Red Cross award, Ms. Jackson finds herself working extra hours fairly often. “Since COVID, that’s pretty much been like any other hospital,” she says. “We’ve had staffing challenges that we work through every day. So, the nurses come, they show up, and they do the best that they can with what we have to keep our patients safe.”

A 2022 survey by McKinsey estimated that by 2025, there could be a gap of 200,000 to 450,000 nurses in the United States. A two-year impact assessment from the American Nurses Foundation found that among more than 12,500 nurses, 40% were considering leaving their positions before the pandemic. By 2022, that number had jumped to 52% with the top reasons being insufficient staffing and negative effects on health and well-being.

Can the “hero narrative” help that situation? Ms. Jackson says she doesn’t see herself as a hero, but the supportive environment and gestures of recognition by staff do make her feel appreciated. These include daily messages offering “kudos” and nominations for the DAISY Award, which she herself received in 2022.

“I have people who I have encouraged to become nurses,” Ms. Jackson says, “and when they saw [the award], they were really excited about becoming a nurse.”
 

Heroes are strong

Jasmine Marcelin, MD, an infectious disease physician with Nebraska Medicine in Omaha, understands the need for heroes as symbols and sources of inspiration. Dr. Marcelin is a fan of the superhero movie genre. There is value, she says, in feeling hope and excitement while watching Superman or Wonder Woman save the day. Who doesn’t want to believe (if only briefly) that the good guys will always win?

In reality, Dr. Marcelin says, “none of us are invincible.” And it’s dangerous to forget that “the people behind the symbols are also human.”

In 2021, Dr. Marcelin gave a TEDx talk entitled, “The Myth of the Health Care Hero.” In it she discussed the extreme physical and mental toll of the pandemic on health care workers and urged her audience to think less about extravagant praise and more about their personal responsibilities. “We don’t want or need to be called heroes,” Dr. Marcelin said. “Right now, our love language is action. We need your help, and we cannot save the world on our own.”

Dr. Marcelin also sees links between superhuman expectations and the high levels of burnout in the medical field.

“It’s a systemic issue,” she explains, “where it requires a revamping and revitalization of the entire psyche of health care to recognize that the people working within this profession are human. And the things that we think and feel and need are the same as anybody else.”
 

Heroes are self-sacrificing 


Well-being, burnout, and disengagement in health care has become a focus for Oregon Health & Science’s Dr. Park, who is also director of RELATE Lab, an organization that aims to make health care more human-centered and equitable through leadership training, research, and community organizing.

For him, hearing neighbors banging pots and pans during the early pandemic was complicated. “The first phase for me was, ‘Thank you. I feel seen. I feel appreciated,’ ” he says. “Yes, I’m wearing a mask. I’m going in. I’m changing in the garage when I come home, so my kid and my partner don’t get sick.”

But after a while, the cheers started to feel like pressure. “Have I done anything heroic today?” Dr. Park asked himself. “Have I been as heroic as my friend who is in the hospital in the ICU? I don’t deserve this, so don’t bang those pots and pans for me.”

When your identity becomes about being a hero, Dr. Park says, when that becomes the standard by which you measure yourself, the result is often a sense of shame.

“I think a lot of people feel ashamed that they feel burnout,” he says, “because they’re supposed to be heroes, putting on their capes and masks. They’re waking up and saying, ‘I’m exhausted, and I can’t play that part today. But I know that’s the social expectation of me.’ “
 

Heroes are noble

There may not be a clear solution, but for many health care professionals, symbolic gestures alone are inadequate and, in certain cases, insulting.

On Doctor’s Day 2023, Alok Patel, MD, a pediatric hospitalist, tweeted a photo of an appreciation “gift” for staff from an unnamed hospital. The small items had metaphorical meanings – a rubber band “as a reminder to stay flexible,” a quarter “as a reminder to ‘call’ for help,” etc.

“Welcome to how you give thanks to ‘health care heroes,’ ” Dr. Patel tweeted.

For Dr. Patel, the issue is not lavish gifts but a need for an attitude shift. He recalls colleagues who felt ashamed asking for mental health services or time off, “because they were bombarded by the hero narrative, by the manufactured pressure that they needed to put their jobs above their own health – because that’s what ‘heroes’ do. I’m willing to bet most physicians would rather receive a sincere email with a transparent plan to better support health care workers than any Doctor’s Day gift,” he says.

In Dr. Marcelin’s TEDx talk, she quotes Spider-Man’s classic adage, “With great power, comes great responsibility.” She argues that this motto doesn’t just apply to those who can fly or deflect bullets; that’s not what heroism is. In fact, most people have their own definition of the word.

For Dr. Stanford, a hero is “someone who is selfless, putting the needs of others before their own.” Dr. Park believes there are no individual heroes. “It’s the work of the collective that’s truly heroic.”

By those standards, clearly anyone can step up, offer help, act with courage and kindness, and be heroic. “We humans, as ordinary as we are, can be extraordinary by using our power to do what’s right,” Dr. Marcelin says, “because there’s no such thing as health care heroes, just good people doing the right thing.”

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

Earlier this year, I stumbled across a podcast in a content update email from the Journal of the American Medical Association. The moderator was interviewing the first author of a study comparing hydrocortisone and fludrocortisone (hydro/fludro) to hydrocortisone alone for treatment of septic shock. In the introduction, the author commented on the discordance in practice among his peers at his hospital. It seemed that there was no consensus on whether fludrocortisone was necessary.

I thought this issue had been settled with publication of the COIITSS trial in 2010. This study randomly assigned 509 patients with septic shock to hydro/fludro versus hydrocortisone alone. There was a nonsignificant reduction in mortality with hydro/fludro and everyone I knew stopped adding fludrocortisone for septic shock. It wasn’t included in guidelines (and still isn›t). I figured the only docs still using it were also prescribing ivermectin and vitamin C – another treatment touted to work in an apocryphal podcast.

It wasn’t just COIITSS that killed fludrocortisone for me. Back in 2002, I was a loyal adherent. That year, a randomized controlled trial (RCT) published by “the lord of corticosteroids for critical illness” doctor, Djillali Annane, found benefit to hydro/fludro in septic shock . Everyone in that study had a cosyntropin stim test and only certain subgroups had better outcomes. As a medical resident paying obeisance to all things evidence-based medicine, I rigidly adopted their protocol for all septic patients. I also kept their insulin between 80 and 110 mg/dL, prescribed drotrecogin alfa, and made sure they were floating in crystalloid. But those are topics for another time.

Subsequent trials and meta-analyses cast doubt on the need for the stim test, and a consensus around hydrocortisone at moderate doses for patients with septic shock emerged. Because one part of the Annane protocol was already deemed unnecessary (the cosyntropin stim test), it was easy to dismiss fludrocortisone after COIITTS was published. Yes, I read Annane’s 2018 APROCCHSS trial, and I’m aware that it found that hydro/fludro reduced 90-day mortality. Like others, I rationalized this finding by framing it as a function of baseline mortality. The two Annane RCTs that found that hydro/fludro reduced mortality in enrolled patients who were considerably more likely to die than those enrolled in RCTs of hydrocortisone alone were negative. It was the target population mortality rate and not the addition of fludrocortisone that made the difference, right?
 

Rethinking hydro/fludro

The author interviewed for the recent JAMA podcast forced me to rethink my blithe dismissal of fludrocortisone. He contended that the COIITTS trial was underpowered and the two Annane RCTs that used fludrocortisone supply the evidence that shows corticosteroids reduce septic shock mortality. As discussed earlier, he found clinical equipoise among his colleagues. Last, he invoked pleiotropic mineralocorticoid effects, such as activation of innate immunity and clearance of alveolar fluid, to support the need to reexamine hydro/fludro.

In his study, he used Big Data to compare hospital records from 2016 to 2020. He analyzed a total of 88,275 patients with septic shock. Most were prescribed hydrocortisone alone (85,995 [97.4%] vs. only 2.6% hydro/fludro). After a number of statistical adjustments and sensitivity analyses, the authors concluded that the addition of fludrocortisone to hydrocortisone for patients with septic shock provides a 3.7% absolute risk reduction in mortality (or discharge to hospice) when compared with hydrocortisone alone. That’s a number needed to treat of 28 to prevent one death (or discharge to hospice).
 

Key takeaways

The study isn’t perfect. In their methods section they use terms like “ensemble machine learner (super learner)” and “immortal time bias.” The first is a fancy way of saying they did a form of propensity scoring, which in turn is a fancy way of saying they tried to control for confounding. The second is a way to adjust for time delays between drug administration. Both are attempts to compensate for the observational design, as is their argument for biologic plausibility. Here they’re on particularly thin ice when trying to prove causal inference. Biologic plausibility is never hard to find; after all, what compound doesn’t have pleiotropic effects? Furthermore, the analysis lacks any data to support their biologic plausibility hypothesis that fludrocortisone’s effect on mortality is mediated via activation of innate immunity and/or clearance of alveolar fluid.

The editorial accompanying this Big Data study endorsed adding fludrocortisone. We have very little that reduces ICU mortality so the low number needed to treat is enticing, especially in light of the low risk from adverse events, so I’m going to start using it. Do I think I’ll save one life for every 28 patients with septic shock to whom I give hydro/fludro instead of hydrocortisone alone? I sure don’t. No way an oral mineralocorticoid at that dose has that type of impact on top of hydrocortisone alone. I still believe that the Annane studies are positive because of the mortality rate in the population enrolled and not because fludrocortisone was added. It all comes full circle, though – 20 years after I abandoned hydro/fludro, I’m going back to it.

Aaron B. Holley, MD, is a professor of medicine at Uniformed Services University in Bethesda, Md., and a pulmonary/critical care and sleep medicine physician at MedStar Washington Hospital Center in Washington, D.C.

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

Earlier this year, I stumbled across a podcast in a content update email from the Journal of the American Medical Association. The moderator was interviewing the first author of a study comparing hydrocortisone and fludrocortisone (hydro/fludro) to hydrocortisone alone for treatment of septic shock. In the introduction, the author commented on the discordance in practice among his peers at his hospital. It seemed that there was no consensus on whether fludrocortisone was necessary.

I thought this issue had been settled with publication of the COIITSS trial in 2010. This study randomly assigned 509 patients with septic shock to hydro/fludro versus hydrocortisone alone. There was a nonsignificant reduction in mortality with hydro/fludro and everyone I knew stopped adding fludrocortisone for septic shock. It wasn’t included in guidelines (and still isn›t). I figured the only docs still using it were also prescribing ivermectin and vitamin C – another treatment touted to work in an apocryphal podcast.

It wasn’t just COIITSS that killed fludrocortisone for me. Back in 2002, I was a loyal adherent. That year, a randomized controlled trial (RCT) published by “the lord of corticosteroids for critical illness” doctor, Djillali Annane, found benefit to hydro/fludro in septic shock . Everyone in that study had a cosyntropin stim test and only certain subgroups had better outcomes. As a medical resident paying obeisance to all things evidence-based medicine, I rigidly adopted their protocol for all septic patients. I also kept their insulin between 80 and 110 mg/dL, prescribed drotrecogin alfa, and made sure they were floating in crystalloid. But those are topics for another time.

Subsequent trials and meta-analyses cast doubt on the need for the stim test, and a consensus around hydrocortisone at moderate doses for patients with septic shock emerged. Because one part of the Annane protocol was already deemed unnecessary (the cosyntropin stim test), it was easy to dismiss fludrocortisone after COIITTS was published. Yes, I read Annane’s 2018 APROCCHSS trial, and I’m aware that it found that hydro/fludro reduced 90-day mortality. Like others, I rationalized this finding by framing it as a function of baseline mortality. The two Annane RCTs that found that hydro/fludro reduced mortality in enrolled patients who were considerably more likely to die than those enrolled in RCTs of hydrocortisone alone were negative. It was the target population mortality rate and not the addition of fludrocortisone that made the difference, right?
 

Rethinking hydro/fludro

The author interviewed for the recent JAMA podcast forced me to rethink my blithe dismissal of fludrocortisone. He contended that the COIITTS trial was underpowered and the two Annane RCTs that used fludrocortisone supply the evidence that shows corticosteroids reduce septic shock mortality. As discussed earlier, he found clinical equipoise among his colleagues. Last, he invoked pleiotropic mineralocorticoid effects, such as activation of innate immunity and clearance of alveolar fluid, to support the need to reexamine hydro/fludro.

In his study, he used Big Data to compare hospital records from 2016 to 2020. He analyzed a total of 88,275 patients with septic shock. Most were prescribed hydrocortisone alone (85,995 [97.4%] vs. only 2.6% hydro/fludro). After a number of statistical adjustments and sensitivity analyses, the authors concluded that the addition of fludrocortisone to hydrocortisone for patients with septic shock provides a 3.7% absolute risk reduction in mortality (or discharge to hospice) when compared with hydrocortisone alone. That’s a number needed to treat of 28 to prevent one death (or discharge to hospice).
 

Key takeaways

The study isn’t perfect. In their methods section they use terms like “ensemble machine learner (super learner)” and “immortal time bias.” The first is a fancy way of saying they did a form of propensity scoring, which in turn is a fancy way of saying they tried to control for confounding. The second is a way to adjust for time delays between drug administration. Both are attempts to compensate for the observational design, as is their argument for biologic plausibility. Here they’re on particularly thin ice when trying to prove causal inference. Biologic plausibility is never hard to find; after all, what compound doesn’t have pleiotropic effects? Furthermore, the analysis lacks any data to support their biologic plausibility hypothesis that fludrocortisone’s effect on mortality is mediated via activation of innate immunity and/or clearance of alveolar fluid.

The editorial accompanying this Big Data study endorsed adding fludrocortisone. We have very little that reduces ICU mortality so the low number needed to treat is enticing, especially in light of the low risk from adverse events, so I’m going to start using it. Do I think I’ll save one life for every 28 patients with septic shock to whom I give hydro/fludro instead of hydrocortisone alone? I sure don’t. No way an oral mineralocorticoid at that dose has that type of impact on top of hydrocortisone alone. I still believe that the Annane studies are positive because of the mortality rate in the population enrolled and not because fludrocortisone was added. It all comes full circle, though – 20 years after I abandoned hydro/fludro, I’m going back to it.

Aaron B. Holley, MD, is a professor of medicine at Uniformed Services University in Bethesda, Md., and a pulmonary/critical care and sleep medicine physician at MedStar Washington Hospital Center in Washington, D.C.

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

Earlier this year, I stumbled across a podcast in a content update email from the Journal of the American Medical Association. The moderator was interviewing the first author of a study comparing hydrocortisone and fludrocortisone (hydro/fludro) to hydrocortisone alone for treatment of septic shock. In the introduction, the author commented on the discordance in practice among his peers at his hospital. It seemed that there was no consensus on whether fludrocortisone was necessary.

I thought this issue had been settled with publication of the COIITSS trial in 2010. This study randomly assigned 509 patients with septic shock to hydro/fludro versus hydrocortisone alone. There was a nonsignificant reduction in mortality with hydro/fludro and everyone I knew stopped adding fludrocortisone for septic shock. It wasn’t included in guidelines (and still isn›t). I figured the only docs still using it were also prescribing ivermectin and vitamin C – another treatment touted to work in an apocryphal podcast.

It wasn’t just COIITSS that killed fludrocortisone for me. Back in 2002, I was a loyal adherent. That year, a randomized controlled trial (RCT) published by “the lord of corticosteroids for critical illness” doctor, Djillali Annane, found benefit to hydro/fludro in septic shock . Everyone in that study had a cosyntropin stim test and only certain subgroups had better outcomes. As a medical resident paying obeisance to all things evidence-based medicine, I rigidly adopted their protocol for all septic patients. I also kept their insulin between 80 and 110 mg/dL, prescribed drotrecogin alfa, and made sure they were floating in crystalloid. But those are topics for another time.

Subsequent trials and meta-analyses cast doubt on the need for the stim test, and a consensus around hydrocortisone at moderate doses for patients with septic shock emerged. Because one part of the Annane protocol was already deemed unnecessary (the cosyntropin stim test), it was easy to dismiss fludrocortisone after COIITTS was published. Yes, I read Annane’s 2018 APROCCHSS trial, and I’m aware that it found that hydro/fludro reduced 90-day mortality. Like others, I rationalized this finding by framing it as a function of baseline mortality. The two Annane RCTs that found that hydro/fludro reduced mortality in enrolled patients who were considerably more likely to die than those enrolled in RCTs of hydrocortisone alone were negative. It was the target population mortality rate and not the addition of fludrocortisone that made the difference, right?
 

Rethinking hydro/fludro

The author interviewed for the recent JAMA podcast forced me to rethink my blithe dismissal of fludrocortisone. He contended that the COIITTS trial was underpowered and the two Annane RCTs that used fludrocortisone supply the evidence that shows corticosteroids reduce septic shock mortality. As discussed earlier, he found clinical equipoise among his colleagues. Last, he invoked pleiotropic mineralocorticoid effects, such as activation of innate immunity and clearance of alveolar fluid, to support the need to reexamine hydro/fludro.

In his study, he used Big Data to compare hospital records from 2016 to 2020. He analyzed a total of 88,275 patients with septic shock. Most were prescribed hydrocortisone alone (85,995 [97.4%] vs. only 2.6% hydro/fludro). After a number of statistical adjustments and sensitivity analyses, the authors concluded that the addition of fludrocortisone to hydrocortisone for patients with septic shock provides a 3.7% absolute risk reduction in mortality (or discharge to hospice) when compared with hydrocortisone alone. That’s a number needed to treat of 28 to prevent one death (or discharge to hospice).
 

Key takeaways

The study isn’t perfect. In their methods section they use terms like “ensemble machine learner (super learner)” and “immortal time bias.” The first is a fancy way of saying they did a form of propensity scoring, which in turn is a fancy way of saying they tried to control for confounding. The second is a way to adjust for time delays between drug administration. Both are attempts to compensate for the observational design, as is their argument for biologic plausibility. Here they’re on particularly thin ice when trying to prove causal inference. Biologic plausibility is never hard to find; after all, what compound doesn’t have pleiotropic effects? Furthermore, the analysis lacks any data to support their biologic plausibility hypothesis that fludrocortisone’s effect on mortality is mediated via activation of innate immunity and/or clearance of alveolar fluid.

The editorial accompanying this Big Data study endorsed adding fludrocortisone. We have very little that reduces ICU mortality so the low number needed to treat is enticing, especially in light of the low risk from adverse events, so I’m going to start using it. Do I think I’ll save one life for every 28 patients with septic shock to whom I give hydro/fludro instead of hydrocortisone alone? I sure don’t. No way an oral mineralocorticoid at that dose has that type of impact on top of hydrocortisone alone. I still believe that the Annane studies are positive because of the mortality rate in the population enrolled and not because fludrocortisone was added. It all comes full circle, though – 20 years after I abandoned hydro/fludro, I’m going back to it.

Aaron B. Holley, MD, is a professor of medicine at Uniformed Services University in Bethesda, Md., and a pulmonary/critical care and sleep medicine physician at MedStar Washington Hospital Center in Washington, D.C.

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

Adam Boggon, MBChB, was working at the Royal Free Hospital in North London during the city’s second wave of COVID-19. “I was effectively living in the hospital,” he recalled. “It felt like I was going 10,000 miles per hour, trying to corral hundreds of medical students and doctors.”

During a national lockdown, there were few places Dr. Boggon could escape to, but the Hampstead Heath swimming ponds mostly remained open. He swam there regularly to exercise and recharge even in winter.

“Swimming in cold water takes you out of yourself,” Dr. Boggon said. “It was such a release for someone who grew up in a rural place and had access to green space, even though the water is murky.” It also hovers around 50 °F (10 °C).

Jumping into cold water, well, kind of stinks. So why do it? It’s not only for bragging rights. A growing number of studies suggest significant mental and physical health benefits to swimming in cold water, specifically to improve depression symptoms and even ease inflammatory conditions.

And a lot of that research is driven by medical pros who love to do it themselves.

For Dr. Boggon, swimming in frigid water is uncomfortable, but he feels that a sensation of calmness follows that makes the plunge more than worth it. Now a Fulbright Scholar at Harvard, where he studies public health and health management, Dr. Boggon is able to frequent the fabled Walden Pond just outside of Boston.

As Thoreau himself said, “You can never have enough of nature.”

Yes, even if it’s really, really cold.
 

Taking a deeper dive

Heather Massey, PhD, a senior lecturer in Sport, Health, and Exercise Science at University of Portsmouth, blames her father, a dinghy sailor, for her affinity for cold-water swimming.

And she’s done more than most, including an epic 16-hour crossing of the English Channel. The water temperature was in the upper-50s °F, and she swam without a wetsuit. “Time just seemed to collapse,” she has shared about the experience.

While working on her PhD and studying the effects of environmental physiology, in particular what happens to the body when it gets hot or cold, Dr. Massey’s hobby and studies seemed to coalesce.

Her research initially focused on the hazards around being in cold open water. But she also noticed a growing trend of people claiming health benefits from the practice. “People started to talk about experiencing improved symptoms of depression or improved mental health from their activities in the water,” she said.

She partnered with another outdoor swimming enthusiast, Hannah Denton, a counseling psychologist working for the National Health Service in the United Kingdom. Ms. Denton was publishing papers on the potential impact that outdoor swimming may have on people with depression and how it could improve mental health in general. She also regularly engages in cold-water swims to boost feelings of mindfulness and peace.

“Having the experience of being so close to nature, as well as the strong sensory experience of being in cold water, does really encourage you to be in the moment,” Ms. Denton wrote in an article for the Sussex Mindfulness Centre. “My experiences of sea swimming and mindfulness support each other. Both have made me feel more comfortable with my body, to have more of a present moment focus, to pay attention to my breathing, and to gain distance from difficult thoughts.”

Over the past few years, Dr. Massey and Ms. Denton have moved from fairly small-scale studies with no real controls to today, completing a randomized controlled trial and looking at the impact that outdoor swimming may have on people living with mild to moderate depression.

“At first, people sort of thought our idea was a bit wacky,” said Dr. Massey. “Now, the popularity of open-water swimming has really blossomed, and so has this area of research. We’re starting to build more rigor into the work.”

Like all the researchers and physicians interviewed for this article, Dr. Massey hesitates to claim that cold-water swimming is a “cure” that should be medicalized.

“It’s not about prescribing it or forcing people to do it,” said Dr. Massey. “This is not something that a doctor should write on a prescription and say you should go and have eight 1-hour sessions of swimming.”
 

(Not yet) a common cure

Enter into the conversation Mark Harper, MD, PhD, consultant anesthetist at Sussex University Hospitals in the United Kingdom and Kristiansand, Norway. Dr. Harper is the author of the 2022 book, Chill: The Cold Water Swim Cure – A Transformative Guide to Renew Your Body and Mind.

Dr. Harper grew up swimming in pools, and it wasn’t until his pool closed for 2 weeks that he ventured into the sea. He recalled walking up the beach afterward, thinking, God, this feels good, and from that moment on, he became hooked on outdoor swimming and curious about its therapeutic potential.

The “cure” in the book’s title, Dr. Harper explained, is being used in the historical sense of “treatment,” as in the first medical book about sea-bathing written over 250 years ago. Dr. Harper acknowledged that the connection to health is still speculative. “However, the circumstantial evidence, the feedback from participants and early study data for its benefits are now very strong,” he said.

In a small study published in 2022, Dr. Harper and colleagues took 59 people with anxiety and depression and put them through a sea-swimming course. Afterward, 80% showed a clinically significant improvement in their mental health.

More recently, Dr. Harper and his team of researchers released a survey to determine how many people were using cold-water swimming as a treatment for a mental or physical ailment. “We thought 30 or 40 people would respond, but we ended up with over 700,” he said. “The majority were using it for mental health but also included inflammation-related conditions.”

Over 2 decades, Dr. Harper has seen dramatic success stories. In his book, he recalled a good friend who, in his early 20s, suffered from Crohn’s disease so badly he couldn’t walk up the steps to his parents’ house. The friend turned to outdoor cold swimming as a low-impact workout and began noticing the symptoms of his disease were improving. Within months, he was able to go off his medications. In 2022, he completed 52 triathlons: one per week for the entire year.
 

How cold exposure may play with your brain

Vaibhav Diwadkar, PhD, professor of psychiatry and behavioral neurosciences at Wayne State University, in Detroit, is studying how human brain networks respond to cold exposure. Dr. Diwadkar and his colleague, Otto Muzik, PhD, began by putting volunteers in a rubber suit with thin tubing and infusing the tubing with temperature-controlled water. Meanwhile, they collected functional brain imaging data to analyze which parts of the brain were responding as body temperature changed.

The data showed that the cold exposure made certain areas of the brain very active, including some that have been associated with the regulation of mood.

Dr. Diwadkar posits that controlled exposure to cold serves as a low-level stressor that knocks different systems within the brain and body out of homeostasis. Once the stress is removed, the brain responds by releasing neurotransmitters that enhance mood, frequently leading to feelings of euphoria in participants.

“We don’t have direct evidence of such a mechanism, but it’s a reasonable speculation,” said Dr. Diwadkar.

However, he pointed out that science writers in the media often portray topics such as this one in black and white, which is “oversimplifying the scientific complexity of biology.”

Clearly, more research needs to be done on the potential therapeutic benefits of cold-water swimming. But for those suffering from anxiety, depression, or chronic illness, if taking a cold dip makes you feel better, the why and how might be beside the point.

Plus, as Dr. Harper pointed out, it’s an easy and accessible therapy.

“All you need is some water – enough to submerge your entire body in – that’s less than 68 °F (20 °C),” he said. “If you stay long enough to get over that initial shock, which is just 2 or 3 minutes, then you’ve got the effect. If you get out and want to go back in again, then you’ve done it right.”
 

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

Adam Boggon, MBChB, was working at the Royal Free Hospital in North London during the city’s second wave of COVID-19. “I was effectively living in the hospital,” he recalled. “It felt like I was going 10,000 miles per hour, trying to corral hundreds of medical students and doctors.”

During a national lockdown, there were few places Dr. Boggon could escape to, but the Hampstead Heath swimming ponds mostly remained open. He swam there regularly to exercise and recharge even in winter.

“Swimming in cold water takes you out of yourself,” Dr. Boggon said. “It was such a release for someone who grew up in a rural place and had access to green space, even though the water is murky.” It also hovers around 50 °F (10 °C).

Jumping into cold water, well, kind of stinks. So why do it? It’s not only for bragging rights. A growing number of studies suggest significant mental and physical health benefits to swimming in cold water, specifically to improve depression symptoms and even ease inflammatory conditions.

And a lot of that research is driven by medical pros who love to do it themselves.

For Dr. Boggon, swimming in frigid water is uncomfortable, but he feels that a sensation of calmness follows that makes the plunge more than worth it. Now a Fulbright Scholar at Harvard, where he studies public health and health management, Dr. Boggon is able to frequent the fabled Walden Pond just outside of Boston.

As Thoreau himself said, “You can never have enough of nature.”

Yes, even if it’s really, really cold.
 

Taking a deeper dive

Heather Massey, PhD, a senior lecturer in Sport, Health, and Exercise Science at University of Portsmouth, blames her father, a dinghy sailor, for her affinity for cold-water swimming.

And she’s done more than most, including an epic 16-hour crossing of the English Channel. The water temperature was in the upper-50s °F, and she swam without a wetsuit. “Time just seemed to collapse,” she has shared about the experience.

While working on her PhD and studying the effects of environmental physiology, in particular what happens to the body when it gets hot or cold, Dr. Massey’s hobby and studies seemed to coalesce.

Her research initially focused on the hazards around being in cold open water. But she also noticed a growing trend of people claiming health benefits from the practice. “People started to talk about experiencing improved symptoms of depression or improved mental health from their activities in the water,” she said.

She partnered with another outdoor swimming enthusiast, Hannah Denton, a counseling psychologist working for the National Health Service in the United Kingdom. Ms. Denton was publishing papers on the potential impact that outdoor swimming may have on people with depression and how it could improve mental health in general. She also regularly engages in cold-water swims to boost feelings of mindfulness and peace.

“Having the experience of being so close to nature, as well as the strong sensory experience of being in cold water, does really encourage you to be in the moment,” Ms. Denton wrote in an article for the Sussex Mindfulness Centre. “My experiences of sea swimming and mindfulness support each other. Both have made me feel more comfortable with my body, to have more of a present moment focus, to pay attention to my breathing, and to gain distance from difficult thoughts.”

Over the past few years, Dr. Massey and Ms. Denton have moved from fairly small-scale studies with no real controls to today, completing a randomized controlled trial and looking at the impact that outdoor swimming may have on people living with mild to moderate depression.

“At first, people sort of thought our idea was a bit wacky,” said Dr. Massey. “Now, the popularity of open-water swimming has really blossomed, and so has this area of research. We’re starting to build more rigor into the work.”

Like all the researchers and physicians interviewed for this article, Dr. Massey hesitates to claim that cold-water swimming is a “cure” that should be medicalized.

“It’s not about prescribing it or forcing people to do it,” said Dr. Massey. “This is not something that a doctor should write on a prescription and say you should go and have eight 1-hour sessions of swimming.”
 

(Not yet) a common cure

Enter into the conversation Mark Harper, MD, PhD, consultant anesthetist at Sussex University Hospitals in the United Kingdom and Kristiansand, Norway. Dr. Harper is the author of the 2022 book, Chill: The Cold Water Swim Cure – A Transformative Guide to Renew Your Body and Mind.

Dr. Harper grew up swimming in pools, and it wasn’t until his pool closed for 2 weeks that he ventured into the sea. He recalled walking up the beach afterward, thinking, God, this feels good, and from that moment on, he became hooked on outdoor swimming and curious about its therapeutic potential.

The “cure” in the book’s title, Dr. Harper explained, is being used in the historical sense of “treatment,” as in the first medical book about sea-bathing written over 250 years ago. Dr. Harper acknowledged that the connection to health is still speculative. “However, the circumstantial evidence, the feedback from participants and early study data for its benefits are now very strong,” he said.

In a small study published in 2022, Dr. Harper and colleagues took 59 people with anxiety and depression and put them through a sea-swimming course. Afterward, 80% showed a clinically significant improvement in their mental health.

More recently, Dr. Harper and his team of researchers released a survey to determine how many people were using cold-water swimming as a treatment for a mental or physical ailment. “We thought 30 or 40 people would respond, but we ended up with over 700,” he said. “The majority were using it for mental health but also included inflammation-related conditions.”

Over 2 decades, Dr. Harper has seen dramatic success stories. In his book, he recalled a good friend who, in his early 20s, suffered from Crohn’s disease so badly he couldn’t walk up the steps to his parents’ house. The friend turned to outdoor cold swimming as a low-impact workout and began noticing the symptoms of his disease were improving. Within months, he was able to go off his medications. In 2022, he completed 52 triathlons: one per week for the entire year.
 

How cold exposure may play with your brain

Vaibhav Diwadkar, PhD, professor of psychiatry and behavioral neurosciences at Wayne State University, in Detroit, is studying how human brain networks respond to cold exposure. Dr. Diwadkar and his colleague, Otto Muzik, PhD, began by putting volunteers in a rubber suit with thin tubing and infusing the tubing with temperature-controlled water. Meanwhile, they collected functional brain imaging data to analyze which parts of the brain were responding as body temperature changed.

The data showed that the cold exposure made certain areas of the brain very active, including some that have been associated with the regulation of mood.

Dr. Diwadkar posits that controlled exposure to cold serves as a low-level stressor that knocks different systems within the brain and body out of homeostasis. Once the stress is removed, the brain responds by releasing neurotransmitters that enhance mood, frequently leading to feelings of euphoria in participants.

“We don’t have direct evidence of such a mechanism, but it’s a reasonable speculation,” said Dr. Diwadkar.

However, he pointed out that science writers in the media often portray topics such as this one in black and white, which is “oversimplifying the scientific complexity of biology.”

Clearly, more research needs to be done on the potential therapeutic benefits of cold-water swimming. But for those suffering from anxiety, depression, or chronic illness, if taking a cold dip makes you feel better, the why and how might be beside the point.

Plus, as Dr. Harper pointed out, it’s an easy and accessible therapy.

“All you need is some water – enough to submerge your entire body in – that’s less than 68 °F (20 °C),” he said. “If you stay long enough to get over that initial shock, which is just 2 or 3 minutes, then you’ve got the effect. If you get out and want to go back in again, then you’ve done it right.”
 

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

Adam Boggon, MBChB, was working at the Royal Free Hospital in North London during the city’s second wave of COVID-19. “I was effectively living in the hospital,” he recalled. “It felt like I was going 10,000 miles per hour, trying to corral hundreds of medical students and doctors.”

During a national lockdown, there were few places Dr. Boggon could escape to, but the Hampstead Heath swimming ponds mostly remained open. He swam there regularly to exercise and recharge even in winter.

“Swimming in cold water takes you out of yourself,” Dr. Boggon said. “It was such a release for someone who grew up in a rural place and had access to green space, even though the water is murky.” It also hovers around 50 °F (10 °C).

Jumping into cold water, well, kind of stinks. So why do it? It’s not only for bragging rights. A growing number of studies suggest significant mental and physical health benefits to swimming in cold water, specifically to improve depression symptoms and even ease inflammatory conditions.

And a lot of that research is driven by medical pros who love to do it themselves.

For Dr. Boggon, swimming in frigid water is uncomfortable, but he feels that a sensation of calmness follows that makes the plunge more than worth it. Now a Fulbright Scholar at Harvard, where he studies public health and health management, Dr. Boggon is able to frequent the fabled Walden Pond just outside of Boston.

As Thoreau himself said, “You can never have enough of nature.”

Yes, even if it’s really, really cold.
 

Taking a deeper dive

Heather Massey, PhD, a senior lecturer in Sport, Health, and Exercise Science at University of Portsmouth, blames her father, a dinghy sailor, for her affinity for cold-water swimming.

And she’s done more than most, including an epic 16-hour crossing of the English Channel. The water temperature was in the upper-50s °F, and she swam without a wetsuit. “Time just seemed to collapse,” she has shared about the experience.

While working on her PhD and studying the effects of environmental physiology, in particular what happens to the body when it gets hot or cold, Dr. Massey’s hobby and studies seemed to coalesce.

Her research initially focused on the hazards around being in cold open water. But she also noticed a growing trend of people claiming health benefits from the practice. “People started to talk about experiencing improved symptoms of depression or improved mental health from their activities in the water,” she said.

She partnered with another outdoor swimming enthusiast, Hannah Denton, a counseling psychologist working for the National Health Service in the United Kingdom. Ms. Denton was publishing papers on the potential impact that outdoor swimming may have on people with depression and how it could improve mental health in general. She also regularly engages in cold-water swims to boost feelings of mindfulness and peace.

“Having the experience of being so close to nature, as well as the strong sensory experience of being in cold water, does really encourage you to be in the moment,” Ms. Denton wrote in an article for the Sussex Mindfulness Centre. “My experiences of sea swimming and mindfulness support each other. Both have made me feel more comfortable with my body, to have more of a present moment focus, to pay attention to my breathing, and to gain distance from difficult thoughts.”

Over the past few years, Dr. Massey and Ms. Denton have moved from fairly small-scale studies with no real controls to today, completing a randomized controlled trial and looking at the impact that outdoor swimming may have on people living with mild to moderate depression.

“At first, people sort of thought our idea was a bit wacky,” said Dr. Massey. “Now, the popularity of open-water swimming has really blossomed, and so has this area of research. We’re starting to build more rigor into the work.”

Like all the researchers and physicians interviewed for this article, Dr. Massey hesitates to claim that cold-water swimming is a “cure” that should be medicalized.

“It’s not about prescribing it or forcing people to do it,” said Dr. Massey. “This is not something that a doctor should write on a prescription and say you should go and have eight 1-hour sessions of swimming.”
 

(Not yet) a common cure

Enter into the conversation Mark Harper, MD, PhD, consultant anesthetist at Sussex University Hospitals in the United Kingdom and Kristiansand, Norway. Dr. Harper is the author of the 2022 book, Chill: The Cold Water Swim Cure – A Transformative Guide to Renew Your Body and Mind.

Dr. Harper grew up swimming in pools, and it wasn’t until his pool closed for 2 weeks that he ventured into the sea. He recalled walking up the beach afterward, thinking, God, this feels good, and from that moment on, he became hooked on outdoor swimming and curious about its therapeutic potential.

The “cure” in the book’s title, Dr. Harper explained, is being used in the historical sense of “treatment,” as in the first medical book about sea-bathing written over 250 years ago. Dr. Harper acknowledged that the connection to health is still speculative. “However, the circumstantial evidence, the feedback from participants and early study data for its benefits are now very strong,” he said.

In a small study published in 2022, Dr. Harper and colleagues took 59 people with anxiety and depression and put them through a sea-swimming course. Afterward, 80% showed a clinically significant improvement in their mental health.

More recently, Dr. Harper and his team of researchers released a survey to determine how many people were using cold-water swimming as a treatment for a mental or physical ailment. “We thought 30 or 40 people would respond, but we ended up with over 700,” he said. “The majority were using it for mental health but also included inflammation-related conditions.”

Over 2 decades, Dr. Harper has seen dramatic success stories. In his book, he recalled a good friend who, in his early 20s, suffered from Crohn’s disease so badly he couldn’t walk up the steps to his parents’ house. The friend turned to outdoor cold swimming as a low-impact workout and began noticing the symptoms of his disease were improving. Within months, he was able to go off his medications. In 2022, he completed 52 triathlons: one per week for the entire year.
 

How cold exposure may play with your brain

Vaibhav Diwadkar, PhD, professor of psychiatry and behavioral neurosciences at Wayne State University, in Detroit, is studying how human brain networks respond to cold exposure. Dr. Diwadkar and his colleague, Otto Muzik, PhD, began by putting volunteers in a rubber suit with thin tubing and infusing the tubing with temperature-controlled water. Meanwhile, they collected functional brain imaging data to analyze which parts of the brain were responding as body temperature changed.

The data showed that the cold exposure made certain areas of the brain very active, including some that have been associated with the regulation of mood.

Dr. Diwadkar posits that controlled exposure to cold serves as a low-level stressor that knocks different systems within the brain and body out of homeostasis. Once the stress is removed, the brain responds by releasing neurotransmitters that enhance mood, frequently leading to feelings of euphoria in participants.

“We don’t have direct evidence of such a mechanism, but it’s a reasonable speculation,” said Dr. Diwadkar.

However, he pointed out that science writers in the media often portray topics such as this one in black and white, which is “oversimplifying the scientific complexity of biology.”

Clearly, more research needs to be done on the potential therapeutic benefits of cold-water swimming. But for those suffering from anxiety, depression, or chronic illness, if taking a cold dip makes you feel better, the why and how might be beside the point.

Plus, as Dr. Harper pointed out, it’s an easy and accessible therapy.

“All you need is some water – enough to submerge your entire body in – that’s less than 68 °F (20 °C),” he said. “If you stay long enough to get over that initial shock, which is just 2 or 3 minutes, then you’ve got the effect. If you get out and want to go back in again, then you’ve done it right.”
 

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

Money talks.

The United States faces a serious shortage of primary care physicians for many reasons, but one, in particular, is inescapable: compensation.

Substantial disparities between what primary care physicians earn relative to specialists like orthopedists and cardiologists can weigh into medical students’ decisions about which field to choose. Plus, the system that Medicare and other health plans use to pay doctors generally places more value on doing procedures like replacing a knee or inserting a stent than on delivering the whole-person, long-term health care management that primary care physicians provide.

As a result of those pay disparities, and the punishing workload typically faced by primary care physicians, more new doctors are becoming specialists, often leaving patients with fewer choices for primary care.

“There is a public out there that is dissatisfied with the lack of access to a routine source of care,” said Christopher Koller, president of the Milbank Memorial Fund, a foundation that focuses on improving population health and health equity. “That’s not going to be addressed until we pay for it.”

Primary care is the foundation of our health care system, the only area in which providing more services – such as childhood vaccines and regular blood pressure screenings – is linked to better population health and more equitable outcomes, according to the National Academies of Sciences, Engineering, and Medicine, in a recently published report on how to rebuild primary care. Without it, the national academies wrote, “minor health problems can spiral into chronic disease,” with poor disease management, ED overuse, and unsustainable costs. Yet for decades, the United States has underinvested in primary care. It accounted for less than 5% of health care spending in 2020 – significantly less than the average spending by countries that are members of the Organization for Economic Cooperation and Development, according to the report.

A $26 billion piece of bipartisan legislation proposed last month by Sen. Bernie Sanders (I-Vt.), chair of the Senate Health, Education, Labor, and Pensions Committee, and Sen. Roger Marshall (R-Kan.) would bolster primary care by increasing training opportunities for doctors and nurses and expanding access to community health centers. Policy experts say the bill would provide important support, but it’s not enough. It doesn’t touch compensation.

“We need primary care to be paid differently and to be paid more, and that starts with Medicare,” Mr. Koller said.
 

How Medicare drives payment

Medicare, which covers 65 million people who are 65 and older or who have certain long-term disabilities, finances more than a fifth of all health care spending — giving it significant muscle in the health care market. Private health plans typically base their payment amounts on the Medicare system, so what Medicare pays is crucial.

Under the Medicare payment system, the amount the program pays for a medical service is determined by three geographically weighted components: a physician’s work, including time and intensity; the practice’s expense, such as overhead and equipment; and professional insurance. It tends to reward specialties that emphasize procedures, such as repairing a hernia or removing a tumor, more than primary care, where the focus is on talking with patients, answering questions, and educating them about managing their chronic conditions.

Medical students may not be familiar with the particulars of how the payment system works, but their clinical training exposes them to a punishing workload and burnout that is contributing to the shortage of primary care physicians, projected to reach up to 48,000 by 2034, according to estimates from the Association of American Medical Colleges.

The earnings differential between primary care and other specialists is also not lost on them. Average annual compensation for doctors who focus on primary care – family medicine, internists, and pediatricians – ranges from an average of about $250,000 to $275,000, according to Medscape’s annual physician compensation report. Many specialists make more than twice as much: Plastic surgeons top the compensation list at $619,000 annually, followed by orthopedists ($573,000) and cardiologists ($507,000).

“I think the major issues in terms of the primary care physician pipeline are the compensation and the work of primary care,” said Russ Phillips, MD, an internist and the director of the Harvard Medical School Center for Primary Care, Boston. “You have to really want to be a primary care physician when that student will make one-third of what students going into dermatology will make.”

According to statistics from the National Resident Matching Program, which tracks the number of residency slots available for graduating medical students and the number of slots filled, 89% of 5,088 family medicine residency slots were filled in 2023, compared with a 93% residency fill rate overall. Internists had a higher fill rate, 96%, but a significant proportion of internal medicine residents eventually practice in a specialty area rather than in primary care.

No one would claim that doctors are poorly paid, but with the average medical student graduating with just over $200,000 in medical school debt, making a good salary matters.
 

Not in it for the money

Still, it’s a misperception that student debt always drives the decision whether to go into primary care, said Len Marquez, senior director of government relations and legislative advocacy at the Association of American Medical Colleges.

For Anitza Quintero, 24, a second-year medical student at the Geisinger Commonwealth School of Medicine in rural Pennsylvania, primary care is a logical extension of her interest in helping children and immigrants. Ms. Quintero’s family came to the United States on a raft from Cuba before she was born. She plans to focus on internal medicine and pediatrics.

“I want to keep going to help my family and other families,” Ms. Quintero said. “There’s obviously something attractive about having a specialty and a high pay grade,” Still, she wants to work “where the whole body is involved,” she said, adding that long-term doctor-patient relationships are “also attractive.”

Ms. Quintero is part of the Abigail Geisinger Scholars Program, which aims to recruit primary care physicians and psychiatrists to the rural health system in part with a promise of medical school loan forgiveness. Health care shortages tend to be more acute in rural areas.

These students’ education costs are covered, and they receive a $2,000 monthly stipend. They can do their residency elsewhere, but upon completing it they return to Geisinger for a primary care job with the health care system. Every year of work there erases one year of the debt covered by their award. If they don’t take a job with the health care system, they must repay the amount they received.
 

Payment imbalances a source of tension

In recent years, the Centers for Medicare & Medicaid Services, which administers the Medicare program, has made changes to address some of the payment imbalances between primary care and specialist services. The agency has expanded the office visit services for which providers can bill to manage their patients, including adding nonprocedural billing codes for providing transitional care, chronic care management, and advance care planning.

In 2024’s final physician fee schedule, the agency plans to allow another new code to take effect, G2211. It would let physicians bill for complex patient evaluation and management services. Any physician could use the code, but it is expected that primary care physicians would use it more frequently than specialists. Congress has delayed implementation of the code since 2021.

The new code is a tiny piece of overall payment reform, “but it is critically important, and it is our top priority on the Hill right now,” said Shari Erickson, chief advocacy officer for the American College of Physicians.

It also triggered a tussle that highlights ongoing tension in Medicare physician payment rules.

The American College of Surgeons and 18 other specialty groups published a statement describing the new code as “unnecessary.” They oppose its implementation because it would primarily benefit primary care providers who, they say, already have the flexibility to bill more for more complex visits.

But the real issue is that, under federal law, changes to Medicare physician payments must preserve budget neutrality, a zero-sum arrangement in which payment increases for primary care providers mean payment decreases elsewhere.

“If they want to keep it, they need to pay for it,” said Christian Shalgian, director of the division of advocacy and health policy for the American College of Surgeons, noting that his organization will continue to oppose implementation otherwise.

Still, there’s general agreement that strengthening the primary care system through payment reform won’t be accomplished by tinkering with billing codes.

The current fee-for-service system doesn’t fully accommodate the time and effort primary care physicians put into “small-ticket” activities like emails and phone calls, reviews of lab results, and consultation reports. A better arrangement, they say, would be to pay primary care physicians a set monthly amount per patient to provide all their care, a system called capitation.

“We’re much better off paying on a per capita basis, get that monthly payment paid in advance plus some extra amount for other things,” said Paul Ginsburg, PhD, a senior fellow at the University of Southern California Schaeffer Center for Health Policy and Economics and former commissioner of the Medicare Payment Advisory Commission.

But if adding a single five-character code to Medicare’s payment rules has proved challenging, imagine the heavy lift involved in overhauling the program’s entire physician payment system. MedPAC and the national academies, both of which provide advice to Congress, have weighed in on the broad outlines of what such a transformation might look like. And there are targeted efforts in Congress: For instance, a bill that would add an annual inflation update to Medicare physician payments and a proposal to address budget neutrality. But it’s unclear whether lawmakers have strong interest in taking action.

“The fact that Medicare has been squeezing physician payment rates for 2 decades is making reforming their structure more difficult,” said Dr. Ginsburg. “The losers are more sensitive to reductions in the rates for the procedures they do.”

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.

Money talks.

The United States faces a serious shortage of primary care physicians for many reasons, but one, in particular, is inescapable: compensation.

Substantial disparities between what primary care physicians earn relative to specialists like orthopedists and cardiologists can weigh into medical students’ decisions about which field to choose. Plus, the system that Medicare and other health plans use to pay doctors generally places more value on doing procedures like replacing a knee or inserting a stent than on delivering the whole-person, long-term health care management that primary care physicians provide.

As a result of those pay disparities, and the punishing workload typically faced by primary care physicians, more new doctors are becoming specialists, often leaving patients with fewer choices for primary care.

“There is a public out there that is dissatisfied with the lack of access to a routine source of care,” said Christopher Koller, president of the Milbank Memorial Fund, a foundation that focuses on improving population health and health equity. “That’s not going to be addressed until we pay for it.”

Primary care is the foundation of our health care system, the only area in which providing more services – such as childhood vaccines and regular blood pressure screenings – is linked to better population health and more equitable outcomes, according to the National Academies of Sciences, Engineering, and Medicine, in a recently published report on how to rebuild primary care. Without it, the national academies wrote, “minor health problems can spiral into chronic disease,” with poor disease management, ED overuse, and unsustainable costs. Yet for decades, the United States has underinvested in primary care. It accounted for less than 5% of health care spending in 2020 – significantly less than the average spending by countries that are members of the Organization for Economic Cooperation and Development, according to the report.

A $26 billion piece of bipartisan legislation proposed last month by Sen. Bernie Sanders (I-Vt.), chair of the Senate Health, Education, Labor, and Pensions Committee, and Sen. Roger Marshall (R-Kan.) would bolster primary care by increasing training opportunities for doctors and nurses and expanding access to community health centers. Policy experts say the bill would provide important support, but it’s not enough. It doesn’t touch compensation.

“We need primary care to be paid differently and to be paid more, and that starts with Medicare,” Mr. Koller said.
 

How Medicare drives payment

Medicare, which covers 65 million people who are 65 and older or who have certain long-term disabilities, finances more than a fifth of all health care spending — giving it significant muscle in the health care market. Private health plans typically base their payment amounts on the Medicare system, so what Medicare pays is crucial.

Under the Medicare payment system, the amount the program pays for a medical service is determined by three geographically weighted components: a physician’s work, including time and intensity; the practice’s expense, such as overhead and equipment; and professional insurance. It tends to reward specialties that emphasize procedures, such as repairing a hernia or removing a tumor, more than primary care, where the focus is on talking with patients, answering questions, and educating them about managing their chronic conditions.

Medical students may not be familiar with the particulars of how the payment system works, but their clinical training exposes them to a punishing workload and burnout that is contributing to the shortage of primary care physicians, projected to reach up to 48,000 by 2034, according to estimates from the Association of American Medical Colleges.

The earnings differential between primary care and other specialists is also not lost on them. Average annual compensation for doctors who focus on primary care – family medicine, internists, and pediatricians – ranges from an average of about $250,000 to $275,000, according to Medscape’s annual physician compensation report. Many specialists make more than twice as much: Plastic surgeons top the compensation list at $619,000 annually, followed by orthopedists ($573,000) and cardiologists ($507,000).

“I think the major issues in terms of the primary care physician pipeline are the compensation and the work of primary care,” said Russ Phillips, MD, an internist and the director of the Harvard Medical School Center for Primary Care, Boston. “You have to really want to be a primary care physician when that student will make one-third of what students going into dermatology will make.”

According to statistics from the National Resident Matching Program, which tracks the number of residency slots available for graduating medical students and the number of slots filled, 89% of 5,088 family medicine residency slots were filled in 2023, compared with a 93% residency fill rate overall. Internists had a higher fill rate, 96%, but a significant proportion of internal medicine residents eventually practice in a specialty area rather than in primary care.

No one would claim that doctors are poorly paid, but with the average medical student graduating with just over $200,000 in medical school debt, making a good salary matters.
 

Not in it for the money

Still, it’s a misperception that student debt always drives the decision whether to go into primary care, said Len Marquez, senior director of government relations and legislative advocacy at the Association of American Medical Colleges.

For Anitza Quintero, 24, a second-year medical student at the Geisinger Commonwealth School of Medicine in rural Pennsylvania, primary care is a logical extension of her interest in helping children and immigrants. Ms. Quintero’s family came to the United States on a raft from Cuba before she was born. She plans to focus on internal medicine and pediatrics.

“I want to keep going to help my family and other families,” Ms. Quintero said. “There’s obviously something attractive about having a specialty and a high pay grade,” Still, she wants to work “where the whole body is involved,” she said, adding that long-term doctor-patient relationships are “also attractive.”

Ms. Quintero is part of the Abigail Geisinger Scholars Program, which aims to recruit primary care physicians and psychiatrists to the rural health system in part with a promise of medical school loan forgiveness. Health care shortages tend to be more acute in rural areas.

These students’ education costs are covered, and they receive a $2,000 monthly stipend. They can do their residency elsewhere, but upon completing it they return to Geisinger for a primary care job with the health care system. Every year of work there erases one year of the debt covered by their award. If they don’t take a job with the health care system, they must repay the amount they received.
 

Payment imbalances a source of tension

In recent years, the Centers for Medicare & Medicaid Services, which administers the Medicare program, has made changes to address some of the payment imbalances between primary care and specialist services. The agency has expanded the office visit services for which providers can bill to manage their patients, including adding nonprocedural billing codes for providing transitional care, chronic care management, and advance care planning.

In 2024’s final physician fee schedule, the agency plans to allow another new code to take effect, G2211. It would let physicians bill for complex patient evaluation and management services. Any physician could use the code, but it is expected that primary care physicians would use it more frequently than specialists. Congress has delayed implementation of the code since 2021.

The new code is a tiny piece of overall payment reform, “but it is critically important, and it is our top priority on the Hill right now,” said Shari Erickson, chief advocacy officer for the American College of Physicians.

It also triggered a tussle that highlights ongoing tension in Medicare physician payment rules.

The American College of Surgeons and 18 other specialty groups published a statement describing the new code as “unnecessary.” They oppose its implementation because it would primarily benefit primary care providers who, they say, already have the flexibility to bill more for more complex visits.

But the real issue is that, under federal law, changes to Medicare physician payments must preserve budget neutrality, a zero-sum arrangement in which payment increases for primary care providers mean payment decreases elsewhere.

“If they want to keep it, they need to pay for it,” said Christian Shalgian, director of the division of advocacy and health policy for the American College of Surgeons, noting that his organization will continue to oppose implementation otherwise.

Still, there’s general agreement that strengthening the primary care system through payment reform won’t be accomplished by tinkering with billing codes.

The current fee-for-service system doesn’t fully accommodate the time and effort primary care physicians put into “small-ticket” activities like emails and phone calls, reviews of lab results, and consultation reports. A better arrangement, they say, would be to pay primary care physicians a set monthly amount per patient to provide all their care, a system called capitation.

“We’re much better off paying on a per capita basis, get that monthly payment paid in advance plus some extra amount for other things,” said Paul Ginsburg, PhD, a senior fellow at the University of Southern California Schaeffer Center for Health Policy and Economics and former commissioner of the Medicare Payment Advisory Commission.

But if adding a single five-character code to Medicare’s payment rules has proved challenging, imagine the heavy lift involved in overhauling the program’s entire physician payment system. MedPAC and the national academies, both of which provide advice to Congress, have weighed in on the broad outlines of what such a transformation might look like. And there are targeted efforts in Congress: For instance, a bill that would add an annual inflation update to Medicare physician payments and a proposal to address budget neutrality. But it’s unclear whether lawmakers have strong interest in taking action.

“The fact that Medicare has been squeezing physician payment rates for 2 decades is making reforming their structure more difficult,” said Dr. Ginsburg. “The losers are more sensitive to reductions in the rates for the procedures they do.”

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.

Money talks.

The United States faces a serious shortage of primary care physicians for many reasons, but one, in particular, is inescapable: compensation.

Substantial disparities between what primary care physicians earn relative to specialists like orthopedists and cardiologists can weigh into medical students’ decisions about which field to choose. Plus, the system that Medicare and other health plans use to pay doctors generally places more value on doing procedures like replacing a knee or inserting a stent than on delivering the whole-person, long-term health care management that primary care physicians provide.

As a result of those pay disparities, and the punishing workload typically faced by primary care physicians, more new doctors are becoming specialists, often leaving patients with fewer choices for primary care.

“There is a public out there that is dissatisfied with the lack of access to a routine source of care,” said Christopher Koller, president of the Milbank Memorial Fund, a foundation that focuses on improving population health and health equity. “That’s not going to be addressed until we pay for it.”

Primary care is the foundation of our health care system, the only area in which providing more services – such as childhood vaccines and regular blood pressure screenings – is linked to better population health and more equitable outcomes, according to the National Academies of Sciences, Engineering, and Medicine, in a recently published report on how to rebuild primary care. Without it, the national academies wrote, “minor health problems can spiral into chronic disease,” with poor disease management, ED overuse, and unsustainable costs. Yet for decades, the United States has underinvested in primary care. It accounted for less than 5% of health care spending in 2020 – significantly less than the average spending by countries that are members of the Organization for Economic Cooperation and Development, according to the report.

A $26 billion piece of bipartisan legislation proposed last month by Sen. Bernie Sanders (I-Vt.), chair of the Senate Health, Education, Labor, and Pensions Committee, and Sen. Roger Marshall (R-Kan.) would bolster primary care by increasing training opportunities for doctors and nurses and expanding access to community health centers. Policy experts say the bill would provide important support, but it’s not enough. It doesn’t touch compensation.

“We need primary care to be paid differently and to be paid more, and that starts with Medicare,” Mr. Koller said.
 

How Medicare drives payment

Medicare, which covers 65 million people who are 65 and older or who have certain long-term disabilities, finances more than a fifth of all health care spending — giving it significant muscle in the health care market. Private health plans typically base their payment amounts on the Medicare system, so what Medicare pays is crucial.

Under the Medicare payment system, the amount the program pays for a medical service is determined by three geographically weighted components: a physician’s work, including time and intensity; the practice’s expense, such as overhead and equipment; and professional insurance. It tends to reward specialties that emphasize procedures, such as repairing a hernia or removing a tumor, more than primary care, where the focus is on talking with patients, answering questions, and educating them about managing their chronic conditions.

Medical students may not be familiar with the particulars of how the payment system works, but their clinical training exposes them to a punishing workload and burnout that is contributing to the shortage of primary care physicians, projected to reach up to 48,000 by 2034, according to estimates from the Association of American Medical Colleges.

The earnings differential between primary care and other specialists is also not lost on them. Average annual compensation for doctors who focus on primary care – family medicine, internists, and pediatricians – ranges from an average of about $250,000 to $275,000, according to Medscape’s annual physician compensation report. Many specialists make more than twice as much: Plastic surgeons top the compensation list at $619,000 annually, followed by orthopedists ($573,000) and cardiologists ($507,000).

“I think the major issues in terms of the primary care physician pipeline are the compensation and the work of primary care,” said Russ Phillips, MD, an internist and the director of the Harvard Medical School Center for Primary Care, Boston. “You have to really want to be a primary care physician when that student will make one-third of what students going into dermatology will make.”

According to statistics from the National Resident Matching Program, which tracks the number of residency slots available for graduating medical students and the number of slots filled, 89% of 5,088 family medicine residency slots were filled in 2023, compared with a 93% residency fill rate overall. Internists had a higher fill rate, 96%, but a significant proportion of internal medicine residents eventually practice in a specialty area rather than in primary care.

No one would claim that doctors are poorly paid, but with the average medical student graduating with just over $200,000 in medical school debt, making a good salary matters.
 

Not in it for the money

Still, it’s a misperception that student debt always drives the decision whether to go into primary care, said Len Marquez, senior director of government relations and legislative advocacy at the Association of American Medical Colleges.

For Anitza Quintero, 24, a second-year medical student at the Geisinger Commonwealth School of Medicine in rural Pennsylvania, primary care is a logical extension of her interest in helping children and immigrants. Ms. Quintero’s family came to the United States on a raft from Cuba before she was born. She plans to focus on internal medicine and pediatrics.

“I want to keep going to help my family and other families,” Ms. Quintero said. “There’s obviously something attractive about having a specialty and a high pay grade,” Still, she wants to work “where the whole body is involved,” she said, adding that long-term doctor-patient relationships are “also attractive.”

Ms. Quintero is part of the Abigail Geisinger Scholars Program, which aims to recruit primary care physicians and psychiatrists to the rural health system in part with a promise of medical school loan forgiveness. Health care shortages tend to be more acute in rural areas.

These students’ education costs are covered, and they receive a $2,000 monthly stipend. They can do their residency elsewhere, but upon completing it they return to Geisinger for a primary care job with the health care system. Every year of work there erases one year of the debt covered by their award. If they don’t take a job with the health care system, they must repay the amount they received.
 

Payment imbalances a source of tension

In recent years, the Centers for Medicare & Medicaid Services, which administers the Medicare program, has made changes to address some of the payment imbalances between primary care and specialist services. The agency has expanded the office visit services for which providers can bill to manage their patients, including adding nonprocedural billing codes for providing transitional care, chronic care management, and advance care planning.

In 2024’s final physician fee schedule, the agency plans to allow another new code to take effect, G2211. It would let physicians bill for complex patient evaluation and management services. Any physician could use the code, but it is expected that primary care physicians would use it more frequently than specialists. Congress has delayed implementation of the code since 2021.

The new code is a tiny piece of overall payment reform, “but it is critically important, and it is our top priority on the Hill right now,” said Shari Erickson, chief advocacy officer for the American College of Physicians.

It also triggered a tussle that highlights ongoing tension in Medicare physician payment rules.

The American College of Surgeons and 18 other specialty groups published a statement describing the new code as “unnecessary.” They oppose its implementation because it would primarily benefit primary care providers who, they say, already have the flexibility to bill more for more complex visits.

But the real issue is that, under federal law, changes to Medicare physician payments must preserve budget neutrality, a zero-sum arrangement in which payment increases for primary care providers mean payment decreases elsewhere.

“If they want to keep it, they need to pay for it,” said Christian Shalgian, director of the division of advocacy and health policy for the American College of Surgeons, noting that his organization will continue to oppose implementation otherwise.

Still, there’s general agreement that strengthening the primary care system through payment reform won’t be accomplished by tinkering with billing codes.

The current fee-for-service system doesn’t fully accommodate the time and effort primary care physicians put into “small-ticket” activities like emails and phone calls, reviews of lab results, and consultation reports. A better arrangement, they say, would be to pay primary care physicians a set monthly amount per patient to provide all their care, a system called capitation.

“We’re much better off paying on a per capita basis, get that monthly payment paid in advance plus some extra amount for other things,” said Paul Ginsburg, PhD, a senior fellow at the University of Southern California Schaeffer Center for Health Policy and Economics and former commissioner of the Medicare Payment Advisory Commission.

But if adding a single five-character code to Medicare’s payment rules has proved challenging, imagine the heavy lift involved in overhauling the program’s entire physician payment system. MedPAC and the national academies, both of which provide advice to Congress, have weighed in on the broad outlines of what such a transformation might look like. And there are targeted efforts in Congress: For instance, a bill that would add an annual inflation update to Medicare physician payments and a proposal to address budget neutrality. But it’s unclear whether lawmakers have strong interest in taking action.

“The fact that Medicare has been squeezing physician payment rates for 2 decades is making reforming their structure more difficult,” said Dr. Ginsburg. “The losers are more sensitive to reductions in the rates for the procedures they do.”

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about KFF.

An innovative surgical procedure combining breast implants and an artificial lung may help more patients with severe lung disease survive to receive transplants. The case was described in a press conference sponsored by Northwestern University, Evanston, Ill.

In May 2023, a surgical team at Northwestern removed both infected lungs from David “Davey” Bauer, aged 34 years, and temporarily used breast implants to hold his heart in place until new lungs were available.

In April 2023, Mr. Bauer, a longtime smoker and vaper, experienced shortness of breath. His girlfriend, Susan Gore, took him to an urgent care center, and he returned home, but “the next morning he couldn’t walk,” Ms. Gore said in the press conference. A trip to the ED yielded a diagnosis of influenza A, followed rapidly by a bacterial lung infection that proved resistant to antibiotics. Mr. Bauer had no prior medical history of serious illness, but he was soon in an intensive care unit. His condition continued to decline, and a double lung transplant was his only option.

Northwestern University

Back from the brink

Mr. Bauer made the transfer to Chicago despite being critically ill. He was in dire need of a lung transplant, and the only way to resolve his infection was to remove the lungs, said Ankit Bharat, MD, chief of thoracic surgery and director of Northwestern Medicine Canning Thoracic Institute, in the press conference.

Northwestern University

“Something needed to be done right away,” Dr. Bharat said. Mr. Bauer’s lungs were removed and the chest cavity was extensively debrided to remove the infection.

Then it was time for outside-the-box thinking. “With the lungs taken out, we needed something to support the heart,” he said. Breast implants came to mind, and double Ds were the largest available.

In addition, the surgeons created an artificial lung system of conduits to keep Mr. Bauer’s blood pumping. “We wanted to maintain the natural blood flow in the body that would be present if the lungs were there,” Dr. Bharat explained.

Plastic surgeons at Northwestern gave Mr. Bauer’s surgical team “a crash course” in managing the breast implants, Dr. Bharat said. The team anticipated that their novel surgical solution would need to last for weeks, but Mr. Bauer’s condition improved immediately once the infected lungs were removed. He was placed on a double-lung transplant list, and the team received an offer of new lungs within 24 hours.

The breast implants were removed, the new lungs were implanted, and Bauer spent several months in the ICU before his discharge to rehabilitation therapy at the end of September, according to a Northwestern press release.

This type of procedure could help patients with infections who need transplants but are too sick to undergo them, Dr. Bharat said in the press conference. In Mr. Bauer’s case, “a lot of stars aligned,” including Bauer’s rapid improvement and the quick availability of a perfect lung match, Dr. Bharat said. Many patients don’t survive to the point of transplant.

“We were surprised how quickly he recovered once we removed the infected lungs,” Dr. Bharat noted. The quick recovery may be in part because of Bauer’s youth and relative good health, but “this was uncharted territory.”

Mr. Bauer’s case is the first use of this particular surgical technique, although the team drew on lessons learned in other surgical settings, such as removal of both lungs to prevent cross-contamination in patients with cancer, he added.
 

Causes and effects

As for the factors that contributed to Mr. Bauer’s initial infection, “there is a lot we don’t know, but we can try to put things together,” said Dr. Bharat. Just as many factors lined up to promote Mr. Bauer’s recovery, many factors lined up to cause the problem, including long-standing smoking and vaping. Although some still view vaping as a safer alternative to smoking, patient data and experiences do not support this claim. “We know for a fact that both of them cause harm,” he added.

Mr. Bauer started smoking cigarettes at age 21 and typically smoked a pack of cigarettes each day before switching to vaping in 2014. In addition, Mr. Bauer had not been vaccinated against the flu, and his flu infection was followed by a bacterial infection.

Bacterial infections followed by hospitalizations are not new as an effect of vaping; a series of articles described the ongoing epidemic of e-cigarette or vaping product use–associated lung injury (EVALI). Patients with EVALI often present at urgent care centers, as Bauer did, with symptoms of flu or pneumonia, and they are often given medication and sent home.

Looking ahead: “We expect that Davey will fully recover and live a normal life,” although he will remain in Chicago for another year for monitoring, said Rade Tomic, MD, pulmonologist and medical director of the Northwestern Medicine Canning Thoracic Institute lung transplant program, in the press conference.

Mr. Bauer expressed his thanks to the surgical team, who also presented him with another gift: a T-shirt with his newly chosen nickname, “DD Davey.” “I feel so blessed, I got a second chance at life,” Mr. Bauer said in the press conference. “You should not inhale anything into your lungs except oxygen.”

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

An innovative surgical procedure combining breast implants and an artificial lung may help more patients with severe lung disease survive to receive transplants. The case was described in a press conference sponsored by Northwestern University, Evanston, Ill.

In May 2023, a surgical team at Northwestern removed both infected lungs from David “Davey” Bauer, aged 34 years, and temporarily used breast implants to hold his heart in place until new lungs were available.

In April 2023, Mr. Bauer, a longtime smoker and vaper, experienced shortness of breath. His girlfriend, Susan Gore, took him to an urgent care center, and he returned home, but “the next morning he couldn’t walk,” Ms. Gore said in the press conference. A trip to the ED yielded a diagnosis of influenza A, followed rapidly by a bacterial lung infection that proved resistant to antibiotics. Mr. Bauer had no prior medical history of serious illness, but he was soon in an intensive care unit. His condition continued to decline, and a double lung transplant was his only option.

Northwestern University

Back from the brink

Mr. Bauer made the transfer to Chicago despite being critically ill. He was in dire need of a lung transplant, and the only way to resolve his infection was to remove the lungs, said Ankit Bharat, MD, chief of thoracic surgery and director of Northwestern Medicine Canning Thoracic Institute, in the press conference.

Northwestern University

“Something needed to be done right away,” Dr. Bharat said. Mr. Bauer’s lungs were removed and the chest cavity was extensively debrided to remove the infection.

Then it was time for outside-the-box thinking. “With the lungs taken out, we needed something to support the heart,” he said. Breast implants came to mind, and double Ds were the largest available.

In addition, the surgeons created an artificial lung system of conduits to keep Mr. Bauer’s blood pumping. “We wanted to maintain the natural blood flow in the body that would be present if the lungs were there,” Dr. Bharat explained.

Plastic surgeons at Northwestern gave Mr. Bauer’s surgical team “a crash course” in managing the breast implants, Dr. Bharat said. The team anticipated that their novel surgical solution would need to last for weeks, but Mr. Bauer’s condition improved immediately once the infected lungs were removed. He was placed on a double-lung transplant list, and the team received an offer of new lungs within 24 hours.

The breast implants were removed, the new lungs were implanted, and Bauer spent several months in the ICU before his discharge to rehabilitation therapy at the end of September, according to a Northwestern press release.

This type of procedure could help patients with infections who need transplants but are too sick to undergo them, Dr. Bharat said in the press conference. In Mr. Bauer’s case, “a lot of stars aligned,” including Bauer’s rapid improvement and the quick availability of a perfect lung match, Dr. Bharat said. Many patients don’t survive to the point of transplant.

“We were surprised how quickly he recovered once we removed the infected lungs,” Dr. Bharat noted. The quick recovery may be in part because of Bauer’s youth and relative good health, but “this was uncharted territory.”

Mr. Bauer’s case is the first use of this particular surgical technique, although the team drew on lessons learned in other surgical settings, such as removal of both lungs to prevent cross-contamination in patients with cancer, he added.
 

Causes and effects

As for the factors that contributed to Mr. Bauer’s initial infection, “there is a lot we don’t know, but we can try to put things together,” said Dr. Bharat. Just as many factors lined up to promote Mr. Bauer’s recovery, many factors lined up to cause the problem, including long-standing smoking and vaping. Although some still view vaping as a safer alternative to smoking, patient data and experiences do not support this claim. “We know for a fact that both of them cause harm,” he added.

Mr. Bauer started smoking cigarettes at age 21 and typically smoked a pack of cigarettes each day before switching to vaping in 2014. In addition, Mr. Bauer had not been vaccinated against the flu, and his flu infection was followed by a bacterial infection.

Bacterial infections followed by hospitalizations are not new as an effect of vaping; a series of articles described the ongoing epidemic of e-cigarette or vaping product use–associated lung injury (EVALI). Patients with EVALI often present at urgent care centers, as Bauer did, with symptoms of flu or pneumonia, and they are often given medication and sent home.

Looking ahead: “We expect that Davey will fully recover and live a normal life,” although he will remain in Chicago for another year for monitoring, said Rade Tomic, MD, pulmonologist and medical director of the Northwestern Medicine Canning Thoracic Institute lung transplant program, in the press conference.

Mr. Bauer expressed his thanks to the surgical team, who also presented him with another gift: a T-shirt with his newly chosen nickname, “DD Davey.” “I feel so blessed, I got a second chance at life,” Mr. Bauer said in the press conference. “You should not inhale anything into your lungs except oxygen.”

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

An innovative surgical procedure combining breast implants and an artificial lung may help more patients with severe lung disease survive to receive transplants. The case was described in a press conference sponsored by Northwestern University, Evanston, Ill.

In May 2023, a surgical team at Northwestern removed both infected lungs from David “Davey” Bauer, aged 34 years, and temporarily used breast implants to hold his heart in place until new lungs were available.

In April 2023, Mr. Bauer, a longtime smoker and vaper, experienced shortness of breath. His girlfriend, Susan Gore, took him to an urgent care center, and he returned home, but “the next morning he couldn’t walk,” Ms. Gore said in the press conference. A trip to the ED yielded a diagnosis of influenza A, followed rapidly by a bacterial lung infection that proved resistant to antibiotics. Mr. Bauer had no prior medical history of serious illness, but he was soon in an intensive care unit. His condition continued to decline, and a double lung transplant was his only option.

Northwestern University

Back from the brink

Mr. Bauer made the transfer to Chicago despite being critically ill. He was in dire need of a lung transplant, and the only way to resolve his infection was to remove the lungs, said Ankit Bharat, MD, chief of thoracic surgery and director of Northwestern Medicine Canning Thoracic Institute, in the press conference.

Northwestern University

“Something needed to be done right away,” Dr. Bharat said. Mr. Bauer’s lungs were removed and the chest cavity was extensively debrided to remove the infection.

Then it was time for outside-the-box thinking. “With the lungs taken out, we needed something to support the heart,” he said. Breast implants came to mind, and double Ds were the largest available.

In addition, the surgeons created an artificial lung system of conduits to keep Mr. Bauer’s blood pumping. “We wanted to maintain the natural blood flow in the body that would be present if the lungs were there,” Dr. Bharat explained.

Plastic surgeons at Northwestern gave Mr. Bauer’s surgical team “a crash course” in managing the breast implants, Dr. Bharat said. The team anticipated that their novel surgical solution would need to last for weeks, but Mr. Bauer’s condition improved immediately once the infected lungs were removed. He was placed on a double-lung transplant list, and the team received an offer of new lungs within 24 hours.

The breast implants were removed, the new lungs were implanted, and Bauer spent several months in the ICU before his discharge to rehabilitation therapy at the end of September, according to a Northwestern press release.

This type of procedure could help patients with infections who need transplants but are too sick to undergo them, Dr. Bharat said in the press conference. In Mr. Bauer’s case, “a lot of stars aligned,” including Bauer’s rapid improvement and the quick availability of a perfect lung match, Dr. Bharat said. Many patients don’t survive to the point of transplant.

“We were surprised how quickly he recovered once we removed the infected lungs,” Dr. Bharat noted. The quick recovery may be in part because of Bauer’s youth and relative good health, but “this was uncharted territory.”

Mr. Bauer’s case is the first use of this particular surgical technique, although the team drew on lessons learned in other surgical settings, such as removal of both lungs to prevent cross-contamination in patients with cancer, he added.
 

Causes and effects

As for the factors that contributed to Mr. Bauer’s initial infection, “there is a lot we don’t know, but we can try to put things together,” said Dr. Bharat. Just as many factors lined up to promote Mr. Bauer’s recovery, many factors lined up to cause the problem, including long-standing smoking and vaping. Although some still view vaping as a safer alternative to smoking, patient data and experiences do not support this claim. “We know for a fact that both of them cause harm,” he added.

Mr. Bauer started smoking cigarettes at age 21 and typically smoked a pack of cigarettes each day before switching to vaping in 2014. In addition, Mr. Bauer had not been vaccinated against the flu, and his flu infection was followed by a bacterial infection.

Bacterial infections followed by hospitalizations are not new as an effect of vaping; a series of articles described the ongoing epidemic of e-cigarette or vaping product use–associated lung injury (EVALI). Patients with EVALI often present at urgent care centers, as Bauer did, with symptoms of flu or pneumonia, and they are often given medication and sent home.

Looking ahead: “We expect that Davey will fully recover and live a normal life,” although he will remain in Chicago for another year for monitoring, said Rade Tomic, MD, pulmonologist and medical director of the Northwestern Medicine Canning Thoracic Institute lung transplant program, in the press conference.

Mr. Bauer expressed his thanks to the surgical team, who also presented him with another gift: a T-shirt with his newly chosen nickname, “DD Davey.” “I feel so blessed, I got a second chance at life,” Mr. Bauer said in the press conference. “You should not inhale anything into your lungs except oxygen.”

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

A, B, C, D, E: It’s a short, menacing alphabet representing the five types of virus causing viral hepatitis, a sickness afflicting some 400 million people around the world today.

Hepatitis viruses are a set of very different pathogens that kill 1.4 million people annually and infect more than HIV and the malaria pathogen do combined. Most of the deaths are from cirrhosis of the liver or hepatic cancer due to chronic infections with hepatitis viruses B or C, picked up through contact with contaminated blood.

Hepatitis B was the first of the five to be discovered, in the 1960s, by biochemist Baruch S. Blumberg, MD. Hepatitis A, which is most commonly spread through contaminated food and water, was next, discovered in 1973 by researchers Stephen Mark Feinstone, MD, Albert Kapikian, MD, and Robert Purcell, MD.

Screening tests for those two types of viruses paved the way to discovering a third. In the 1970s, hematologist Harvey J. Alter, MD, examined unexplained cases of hepatitis in patients after blood transfusions and found that only 25% of such cases were caused by the hepatitis B virus, and none were linked to the hepatitis A virus. The rest were caused by an unidentified transmissible agent that could persist in the body as a chronic infection and lead to liver cirrhosis and liver cancer.

The agent behind this disease, named non-A, non-B hepatitis, remained a mystery for a decade until Michael Houghton, PhD, a microbiologist working at the biotechnology company Chiron Corporation, and his team sequenced the agent’s genome in 1989 after years of intensive investigation. They identified it as a novel virus of the family to which yellow fever virus belongs: the flaviviruses, a group of RNA viruses often transmitted through the bite of infected arthropods.

But there was more to the story. Scientists needed to show that this new virus could, indeed, cause hepatitis C on its own – a feat achieved in 1997, when Charles M. Rice, PhD, then a virologist at Washington University in St. Louis, and others succeeded in creating a form of the virus in the lab that could replicate in the only animal model for hepatitis C, the chimpanzee. When they injected the virus into the liver of chimpanzees, it triggered clinical hepatitis, demonstrating the direct connection between hepatitis C and non-A, non-B hepatitis.

The findings led to lifesaving hepatitis C tests to avert infections through transfusions with contaminated blood, as well as for the development of effective antiviral medications to treat the disease. In 2020, in the thick of the SARS-CoV-2 pandemic, Dr. Alter, Dr. Houghton, and Dr. Rice received a Nobel Prize in Medicine for their work on identifying the virus.

To learn more about hepatitis C history and the treatment and prevention challenges that remain, Knowable Magazine spoke with Dr. Rice, now at the Rockefeller University, at the 72nd Lindau Nobel Laureate Meeting in Germany in June 2023. This conversation has been edited for length and clarity.
 

What were the challenges at the time you began your research on hepatitis C?

The realization that an agent was behind non-A, non-B hepatitis had initiated a virus hunt to try to figure out what the causative agent was. Dr. Houghton and his group at Chiron won that race and reported the partial sequence of the virus in 1989 in Science.

It was an interesting kind of a dilemma for me as an early-stage assistant professor at Washington University in St. Louis, where I’d been working on yellow fever. All of a sudden, we had this new human virus that dropped into our laps and joined the flavivirus family; we had to decide if we were going to shift some of our attention to work on this virus. Initially, people in the viral hepatitis field invited us to meetings, but because we were doing work on the related virus, yellow fever, not because we were considered majors player in the field.

The main challenge was that we could not grow the virus in cell culture. And the only experimental model was the chimpanzee, so it was really difficult for laboratories to study this virus.

There were two major goals. One was to establish a cell culture system where you could replicate the virus and study it. And the other was try to create a system where we could do genetics on the virus. It was shown to be an RNA virus, and the collection of tools available for modifying RNA at that time, in the early 1990s, was not the same as it was for DNA. Now that’s changed to some extent, with modern editing technologies.

If there’s one lesson to be learned from this hepatitis C story, it’s that persistence pays off.
 

This journey started with an unknown virus and ended up with treatment in a relatively short period of time.

I don’t think it was a short period of time, between all of the failures to actually get a cell culture system and to show that we had a functional clone. From 1989, when the virus sequence was reported, to 2011, when the first antiviral compounds were produced, was 22 years.

And then, that initial generation of treatment compounds was not the greatest, and they were combined with the treatment that we were trying to get rid of – interferon – that made people quite ill and didn’t always cure them. They only had about a 50% cure rate.

It was 2014 when the interferon-free cocktails came about. And that was really amazing.

There were people who thought, “You are not going to be able to develop a drug cocktail that can eliminate this virus.” It was presumptuous to think that one could, but it was accomplished by biotech and the pharmaceutical industry. So it is really quite a success story, but I wish it could have been faster.

Over the last 50 years, researchers have identified five types of viruses that cause different forms of viral hepatitis. Each virus has its own mode of transmission and health impacts. Scientists around the world have worked to develop treatments and vaccines.
 

What are the current challenges in combating hepatitis C?

One thing that was a little sobering and disappointing for me was that when these medical advances are made and shown to be efficacious, it is not possible to get these drugs to everybody who needs them and successfully treat them. It’s a lot more complicated, in part because of the economics – how much the companies decide to charge for the drugs.

Also, it’s difficult to identify people who are infected with hepatitis C, because it’s often asymptomatic. Even when identified, getting people into treatment is challenging given differences in public health capabilities which vary at the local, national, and global levels. So we have wonderful drugs that can basically cure anybody, but I think we still could use a vaccine for hepatitis C.
 

During the first year of the COVID-19 pandemic, you won the Nobel Prize for the discovery of the hepatitis C virus. What was that experience like?

It was December 2020, and we were working on SARS-CoV-2 in the peak of the pandemic in New York City. My spouse and the dogs were off at our house in Connecticut, and I was living in the apartment in Manhattan. And I got this call at 4:30 in the morning. It was pretty shocking.

The pandemic made people more aware of what a highly infectious, disease-causing virus can do to our world. It encouraged the rapid dissemination of research results and more open publications. It also really made us appreciate how the same virus does different things depending upon who’s infected: In the case of COVID-19, it’s not good to be old, for example.
 

After many decades working with viruses, what would you say is the next frontier in virology?

There’s a lot that we don’t understand about these viruses. The more we study them, the more we understand about ourselves, our cells and our antiviral defense systems.

And there’s also great power in terms of being able to diagnose new viruses. The sequencing technology, the functional genomics technologies, all of those things, when applied to virology, give us a much richer picture of how these viruses interact with cells. I think it’s a golden age.

The five known types of viral hepatitis afflict hundreds of millions of people around the world, causing both acute and chronic liver diseases. Among them, types B and C are the most severe, and diagnosis often remains a challenge.
 

You have been working with flaviviruses (dengue, Zika, yellow fever, and hepatitis C) for many decades. Zika and dengue pose an ongoing threat worldwide and, in particular, Latin America. Based on the successful example of hepatitis C, what can scientific research do to mitigate the impact of these viruses?

For viruses like Zika, developing a vaccine is probably going to be fairly straightforward – except that since Zika is so transient, it makes it hard to prove that your vaccine works. You would have to do a human challenge study, in which volunteers are deliberately exposed to an infection in a safe way with health-care support.

For dengue, it’s much more difficult, because there are four different serotypes – different versions of the same virus – and infection with one serotype can put you at increased risk of more severe disease if you get infected with a second serotype. Eliciting a balanced response that would protect you against all four dengue serotypes is the holy grail of trying to develop a dengue vaccine.

People are using various approaches to accomplish that. The classic one is to take live attenuated versions – weakened forms of viruses that have been modified so they can’t cause severe illness but can still stimulate the immune system – of each of the four serotypes and mix them together. Another is to make chimeric viruses: a combination of genetic material from different viruses, resulting in new viruses that have features of each of the four dengue serotypes, engineered into the backbone of the yellow fever vaccine. But this hasn’t worked as well as people have hoped. I think the cocktail of live, attenuated dengue variants is probably the most advanced approach. But I would guess that given the success of COVID-19 mRNA vaccines, the mRNA approach will also be tried out.

These diseases are not going to go away. You can’t eradicate every mosquito. And you can’t really immunize every susceptible vertebrate host. So occasionally there’s going to be spillover into the human population. We need to keep working on these because they are big problems.
 

You began your career at the California Institute of Technology studying RNA viruses, such as the mosquito-borne Sindbis virus, and then flaviviruses that cause encephalitis, polyarthritis, yellow fever, and dengue fever. Later on, you also studied hepatitis C virus. Is there any advantage for virologists in changing the viruses they study throughout their careers?

They’re all interesting, right? And they are all different in their own ways. I say that my career has been a downward spiral of tackling increasingly intricate viruses. Initially, the alphaviruses – a viral family that includes chikungunya virus, for example – were easy. The classical flaviviruses – like yellow fever, dengue fever, West Nile viruses, and Zika virus, among others – were a little more difficult, but the hepatitis C virus was impossible for 15 years, until we, and others, finally achieved a complete replication system in the laboratory.

We coexist daily with viruses, but the pandemic may have given people the idea that all these microorganisms are invariably life-threatening.

We have to treat them with respect. We’ve seen what can happen with the emergence of a novel coronavirus that can spread during an asymptomatic phase of infection. You can’t be prepared for everything, but in some respects our response was a lot slower and less effective than it could have been.

If there’s anything that we’ve learned over the last 10 years with the new nucleic acid sequencing technologies, it’s that our past view of the virosphere was very narrow. And if you really look at what’s out there, the estimated virus diversity is a staggering number, like 1,031 types. Although most of them are not pathogenic to humans, some are. We have to take this threat seriously.
 

Is science prepared?

I think so, but there has to be an investment, a societal investment. And that investment has to not only be an investment in infrastructure that can react quickly to something new, but also to establish a repository of protective antibodies and small molecules against viruses that we know could be future threats.

Often, these things go in cycles. There’s a disaster, like the COVID-19 pandemic, people are changed by the experience, but then they think “Oh, well, the virus has faded into the background, the threat is over.” And that’s just not the case. We need a more sustained plan rather than a reactive stance. And that’s hard to do when resources and money are limited.
 

What is the effect of science illiteracy, conspiracy theories, and lack of science information on the battle against viruses?

These are huge issues, and I don’t know the best way to combat them and educate people. Any combative, confrontational kind of response – it’s just not going to work. People will get more resolute in their entrenched beliefs and not hear or believe compelling evidence to the contrary.

It’s frustrating. I think that we have amazing tools and the power to make really significant advances to help people. It is more than a little discouraging for scientists when there’s a substantial fraction of people who don’t believe in things that are well-supported by facts.

It’s in large part an educational problem. I think we don’t put enough money into education, particularly early education. A lot of people don’t understand how much of what we take for granted today is underpinned by science. All this technology – good, bad or ugly – is all science.
 

This article originally appeared in Knowable Magazine on Oct. 30, 2023. Knowable Magazine is an independent journalistic endeavor from Annual Reviews, a nonprofit publisher dedicated to synthesizing and integrating knowledge for the progress of science and the benefit of society. Sign up for Knowable Magazine’s newsletter.

A, B, C, D, E: It’s a short, menacing alphabet representing the five types of virus causing viral hepatitis, a sickness afflicting some 400 million people around the world today.

Hepatitis viruses are a set of very different pathogens that kill 1.4 million people annually and infect more than HIV and the malaria pathogen do combined. Most of the deaths are from cirrhosis of the liver or hepatic cancer due to chronic infections with hepatitis viruses B or C, picked up through contact with contaminated blood.

Hepatitis B was the first of the five to be discovered, in the 1960s, by biochemist Baruch S. Blumberg, MD. Hepatitis A, which is most commonly spread through contaminated food and water, was next, discovered in 1973 by researchers Stephen Mark Feinstone, MD, Albert Kapikian, MD, and Robert Purcell, MD.

Screening tests for those two types of viruses paved the way to discovering a third. In the 1970s, hematologist Harvey J. Alter, MD, examined unexplained cases of hepatitis in patients after blood transfusions and found that only 25% of such cases were caused by the hepatitis B virus, and none were linked to the hepatitis A virus. The rest were caused by an unidentified transmissible agent that could persist in the body as a chronic infection and lead to liver cirrhosis and liver cancer.

The agent behind this disease, named non-A, non-B hepatitis, remained a mystery for a decade until Michael Houghton, PhD, a microbiologist working at the biotechnology company Chiron Corporation, and his team sequenced the agent’s genome in 1989 after years of intensive investigation. They identified it as a novel virus of the family to which yellow fever virus belongs: the flaviviruses, a group of RNA viruses often transmitted through the bite of infected arthropods.

But there was more to the story. Scientists needed to show that this new virus could, indeed, cause hepatitis C on its own – a feat achieved in 1997, when Charles M. Rice, PhD, then a virologist at Washington University in St. Louis, and others succeeded in creating a form of the virus in the lab that could replicate in the only animal model for hepatitis C, the chimpanzee. When they injected the virus into the liver of chimpanzees, it triggered clinical hepatitis, demonstrating the direct connection between hepatitis C and non-A, non-B hepatitis.

The findings led to lifesaving hepatitis C tests to avert infections through transfusions with contaminated blood, as well as for the development of effective antiviral medications to treat the disease. In 2020, in the thick of the SARS-CoV-2 pandemic, Dr. Alter, Dr. Houghton, and Dr. Rice received a Nobel Prize in Medicine for their work on identifying the virus.

To learn more about hepatitis C history and the treatment and prevention challenges that remain, Knowable Magazine spoke with Dr. Rice, now at the Rockefeller University, at the 72nd Lindau Nobel Laureate Meeting in Germany in June 2023. This conversation has been edited for length and clarity.
 

What were the challenges at the time you began your research on hepatitis C?

The realization that an agent was behind non-A, non-B hepatitis had initiated a virus hunt to try to figure out what the causative agent was. Dr. Houghton and his group at Chiron won that race and reported the partial sequence of the virus in 1989 in Science.

It was an interesting kind of a dilemma for me as an early-stage assistant professor at Washington University in St. Louis, where I’d been working on yellow fever. All of a sudden, we had this new human virus that dropped into our laps and joined the flavivirus family; we had to decide if we were going to shift some of our attention to work on this virus. Initially, people in the viral hepatitis field invited us to meetings, but because we were doing work on the related virus, yellow fever, not because we were considered majors player in the field.

The main challenge was that we could not grow the virus in cell culture. And the only experimental model was the chimpanzee, so it was really difficult for laboratories to study this virus.

There were two major goals. One was to establish a cell culture system where you could replicate the virus and study it. And the other was try to create a system where we could do genetics on the virus. It was shown to be an RNA virus, and the collection of tools available for modifying RNA at that time, in the early 1990s, was not the same as it was for DNA. Now that’s changed to some extent, with modern editing technologies.

If there’s one lesson to be learned from this hepatitis C story, it’s that persistence pays off.
 

This journey started with an unknown virus and ended up with treatment in a relatively short period of time.

I don’t think it was a short period of time, between all of the failures to actually get a cell culture system and to show that we had a functional clone. From 1989, when the virus sequence was reported, to 2011, when the first antiviral compounds were produced, was 22 years.

And then, that initial generation of treatment compounds was not the greatest, and they were combined with the treatment that we were trying to get rid of – interferon – that made people quite ill and didn’t always cure them. They only had about a 50% cure rate.

It was 2014 when the interferon-free cocktails came about. And that was really amazing.

There were people who thought, “You are not going to be able to develop a drug cocktail that can eliminate this virus.” It was presumptuous to think that one could, but it was accomplished by biotech and the pharmaceutical industry. So it is really quite a success story, but I wish it could have been faster.

Over the last 50 years, researchers have identified five types of viruses that cause different forms of viral hepatitis. Each virus has its own mode of transmission and health impacts. Scientists around the world have worked to develop treatments and vaccines.
 

What are the current challenges in combating hepatitis C?

One thing that was a little sobering and disappointing for me was that when these medical advances are made and shown to be efficacious, it is not possible to get these drugs to everybody who needs them and successfully treat them. It’s a lot more complicated, in part because of the economics – how much the companies decide to charge for the drugs.

Also, it’s difficult to identify people who are infected with hepatitis C, because it’s often asymptomatic. Even when identified, getting people into treatment is challenging given differences in public health capabilities which vary at the local, national, and global levels. So we have wonderful drugs that can basically cure anybody, but I think we still could use a vaccine for hepatitis C.
 

During the first year of the COVID-19 pandemic, you won the Nobel Prize for the discovery of the hepatitis C virus. What was that experience like?

It was December 2020, and we were working on SARS-CoV-2 in the peak of the pandemic in New York City. My spouse and the dogs were off at our house in Connecticut, and I was living in the apartment in Manhattan. And I got this call at 4:30 in the morning. It was pretty shocking.

The pandemic made people more aware of what a highly infectious, disease-causing virus can do to our world. It encouraged the rapid dissemination of research results and more open publications. It also really made us appreciate how the same virus does different things depending upon who’s infected: In the case of COVID-19, it’s not good to be old, for example.
 

After many decades working with viruses, what would you say is the next frontier in virology?

There’s a lot that we don’t understand about these viruses. The more we study them, the more we understand about ourselves, our cells and our antiviral defense systems.

And there’s also great power in terms of being able to diagnose new viruses. The sequencing technology, the functional genomics technologies, all of those things, when applied to virology, give us a much richer picture of how these viruses interact with cells. I think it’s a golden age.

The five known types of viral hepatitis afflict hundreds of millions of people around the world, causing both acute and chronic liver diseases. Among them, types B and C are the most severe, and diagnosis often remains a challenge.
 

You have been working with flaviviruses (dengue, Zika, yellow fever, and hepatitis C) for many decades. Zika and dengue pose an ongoing threat worldwide and, in particular, Latin America. Based on the successful example of hepatitis C, what can scientific research do to mitigate the impact of these viruses?

For viruses like Zika, developing a vaccine is probably going to be fairly straightforward – except that since Zika is so transient, it makes it hard to prove that your vaccine works. You would have to do a human challenge study, in which volunteers are deliberately exposed to an infection in a safe way with health-care support.

For dengue, it’s much more difficult, because there are four different serotypes – different versions of the same virus – and infection with one serotype can put you at increased risk of more severe disease if you get infected with a second serotype. Eliciting a balanced response that would protect you against all four dengue serotypes is the holy grail of trying to develop a dengue vaccine.

People are using various approaches to accomplish that. The classic one is to take live attenuated versions – weakened forms of viruses that have been modified so they can’t cause severe illness but can still stimulate the immune system – of each of the four serotypes and mix them together. Another is to make chimeric viruses: a combination of genetic material from different viruses, resulting in new viruses that have features of each of the four dengue serotypes, engineered into the backbone of the yellow fever vaccine. But this hasn’t worked as well as people have hoped. I think the cocktail of live, attenuated dengue variants is probably the most advanced approach. But I would guess that given the success of COVID-19 mRNA vaccines, the mRNA approach will also be tried out.

These diseases are not going to go away. You can’t eradicate every mosquito. And you can’t really immunize every susceptible vertebrate host. So occasionally there’s going to be spillover into the human population. We need to keep working on these because they are big problems.
 

You began your career at the California Institute of Technology studying RNA viruses, such as the mosquito-borne Sindbis virus, and then flaviviruses that cause encephalitis, polyarthritis, yellow fever, and dengue fever. Later on, you also studied hepatitis C virus. Is there any advantage for virologists in changing the viruses they study throughout their careers?

They’re all interesting, right? And they are all different in their own ways. I say that my career has been a downward spiral of tackling increasingly intricate viruses. Initially, the alphaviruses – a viral family that includes chikungunya virus, for example – were easy. The classical flaviviruses – like yellow fever, dengue fever, West Nile viruses, and Zika virus, among others – were a little more difficult, but the hepatitis C virus was impossible for 15 years, until we, and others, finally achieved a complete replication system in the laboratory.

We coexist daily with viruses, but the pandemic may have given people the idea that all these microorganisms are invariably life-threatening.

We have to treat them with respect. We’ve seen what can happen with the emergence of a novel coronavirus that can spread during an asymptomatic phase of infection. You can’t be prepared for everything, but in some respects our response was a lot slower and less effective than it could have been.

If there’s anything that we’ve learned over the last 10 years with the new nucleic acid sequencing technologies, it’s that our past view of the virosphere was very narrow. And if you really look at what’s out there, the estimated virus diversity is a staggering number, like 1,031 types. Although most of them are not pathogenic to humans, some are. We have to take this threat seriously.
 

Is science prepared?

I think so, but there has to be an investment, a societal investment. And that investment has to not only be an investment in infrastructure that can react quickly to something new, but also to establish a repository of protective antibodies and small molecules against viruses that we know could be future threats.

Often, these things go in cycles. There’s a disaster, like the COVID-19 pandemic, people are changed by the experience, but then they think “Oh, well, the virus has faded into the background, the threat is over.” And that’s just not the case. We need a more sustained plan rather than a reactive stance. And that’s hard to do when resources and money are limited.
 

What is the effect of science illiteracy, conspiracy theories, and lack of science information on the battle against viruses?

These are huge issues, and I don’t know the best way to combat them and educate people. Any combative, confrontational kind of response – it’s just not going to work. People will get more resolute in their entrenched beliefs and not hear or believe compelling evidence to the contrary.

It’s frustrating. I think that we have amazing tools and the power to make really significant advances to help people. It is more than a little discouraging for scientists when there’s a substantial fraction of people who don’t believe in things that are well-supported by facts.

It’s in large part an educational problem. I think we don’t put enough money into education, particularly early education. A lot of people don’t understand how much of what we take for granted today is underpinned by science. All this technology – good, bad or ugly – is all science.
 

This article originally appeared in Knowable Magazine on Oct. 30, 2023. Knowable Magazine is an independent journalistic endeavor from Annual Reviews, a nonprofit publisher dedicated to synthesizing and integrating knowledge for the progress of science and the benefit of society. Sign up for Knowable Magazine’s newsletter.

A, B, C, D, E: It’s a short, menacing alphabet representing the five types of virus causing viral hepatitis, a sickness afflicting some 400 million people around the world today.

Hepatitis viruses are a set of very different pathogens that kill 1.4 million people annually and infect more than HIV and the malaria pathogen do combined. Most of the deaths are from cirrhosis of the liver or hepatic cancer due to chronic infections with hepatitis viruses B or C, picked up through contact with contaminated blood.

Hepatitis B was the first of the five to be discovered, in the 1960s, by biochemist Baruch S. Blumberg, MD. Hepatitis A, which is most commonly spread through contaminated food and water, was next, discovered in 1973 by researchers Stephen Mark Feinstone, MD, Albert Kapikian, MD, and Robert Purcell, MD.

Screening tests for those two types of viruses paved the way to discovering a third. In the 1970s, hematologist Harvey J. Alter, MD, examined unexplained cases of hepatitis in patients after blood transfusions and found that only 25% of such cases were caused by the hepatitis B virus, and none were linked to the hepatitis A virus. The rest were caused by an unidentified transmissible agent that could persist in the body as a chronic infection and lead to liver cirrhosis and liver cancer.

The agent behind this disease, named non-A, non-B hepatitis, remained a mystery for a decade until Michael Houghton, PhD, a microbiologist working at the biotechnology company Chiron Corporation, and his team sequenced the agent’s genome in 1989 after years of intensive investigation. They identified it as a novel virus of the family to which yellow fever virus belongs: the flaviviruses, a group of RNA viruses often transmitted through the bite of infected arthropods.

But there was more to the story. Scientists needed to show that this new virus could, indeed, cause hepatitis C on its own – a feat achieved in 1997, when Charles M. Rice, PhD, then a virologist at Washington University in St. Louis, and others succeeded in creating a form of the virus in the lab that could replicate in the only animal model for hepatitis C, the chimpanzee. When they injected the virus into the liver of chimpanzees, it triggered clinical hepatitis, demonstrating the direct connection between hepatitis C and non-A, non-B hepatitis.

The findings led to lifesaving hepatitis C tests to avert infections through transfusions with contaminated blood, as well as for the development of effective antiviral medications to treat the disease. In 2020, in the thick of the SARS-CoV-2 pandemic, Dr. Alter, Dr. Houghton, and Dr. Rice received a Nobel Prize in Medicine for their work on identifying the virus.

To learn more about hepatitis C history and the treatment and prevention challenges that remain, Knowable Magazine spoke with Dr. Rice, now at the Rockefeller University, at the 72nd Lindau Nobel Laureate Meeting in Germany in June 2023. This conversation has been edited for length and clarity.
 

What were the challenges at the time you began your research on hepatitis C?

The realization that an agent was behind non-A, non-B hepatitis had initiated a virus hunt to try to figure out what the causative agent was. Dr. Houghton and his group at Chiron won that race and reported the partial sequence of the virus in 1989 in Science.

It was an interesting kind of a dilemma for me as an early-stage assistant professor at Washington University in St. Louis, where I’d been working on yellow fever. All of a sudden, we had this new human virus that dropped into our laps and joined the flavivirus family; we had to decide if we were going to shift some of our attention to work on this virus. Initially, people in the viral hepatitis field invited us to meetings, but because we were doing work on the related virus, yellow fever, not because we were considered majors player in the field.

The main challenge was that we could not grow the virus in cell culture. And the only experimental model was the chimpanzee, so it was really difficult for laboratories to study this virus.

There were two major goals. One was to establish a cell culture system where you could replicate the virus and study it. And the other was try to create a system where we could do genetics on the virus. It was shown to be an RNA virus, and the collection of tools available for modifying RNA at that time, in the early 1990s, was not the same as it was for DNA. Now that’s changed to some extent, with modern editing technologies.

If there’s one lesson to be learned from this hepatitis C story, it’s that persistence pays off.
 

This journey started with an unknown virus and ended up with treatment in a relatively short period of time.

I don’t think it was a short period of time, between all of the failures to actually get a cell culture system and to show that we had a functional clone. From 1989, when the virus sequence was reported, to 2011, when the first antiviral compounds were produced, was 22 years.

And then, that initial generation of treatment compounds was not the greatest, and they were combined with the treatment that we were trying to get rid of – interferon – that made people quite ill and didn’t always cure them. They only had about a 50% cure rate.

It was 2014 when the interferon-free cocktails came about. And that was really amazing.

There were people who thought, “You are not going to be able to develop a drug cocktail that can eliminate this virus.” It was presumptuous to think that one could, but it was accomplished by biotech and the pharmaceutical industry. So it is really quite a success story, but I wish it could have been faster.

Over the last 50 years, researchers have identified five types of viruses that cause different forms of viral hepatitis. Each virus has its own mode of transmission and health impacts. Scientists around the world have worked to develop treatments and vaccines.
 

What are the current challenges in combating hepatitis C?

One thing that was a little sobering and disappointing for me was that when these medical advances are made and shown to be efficacious, it is not possible to get these drugs to everybody who needs them and successfully treat them. It’s a lot more complicated, in part because of the economics – how much the companies decide to charge for the drugs.

Also, it’s difficult to identify people who are infected with hepatitis C, because it’s often asymptomatic. Even when identified, getting people into treatment is challenging given differences in public health capabilities which vary at the local, national, and global levels. So we have wonderful drugs that can basically cure anybody, but I think we still could use a vaccine for hepatitis C.
 

During the first year of the COVID-19 pandemic, you won the Nobel Prize for the discovery of the hepatitis C virus. What was that experience like?

It was December 2020, and we were working on SARS-CoV-2 in the peak of the pandemic in New York City. My spouse and the dogs were off at our house in Connecticut, and I was living in the apartment in Manhattan. And I got this call at 4:30 in the morning. It was pretty shocking.

The pandemic made people more aware of what a highly infectious, disease-causing virus can do to our world. It encouraged the rapid dissemination of research results and more open publications. It also really made us appreciate how the same virus does different things depending upon who’s infected: In the case of COVID-19, it’s not good to be old, for example.
 

After many decades working with viruses, what would you say is the next frontier in virology?

There’s a lot that we don’t understand about these viruses. The more we study them, the more we understand about ourselves, our cells and our antiviral defense systems.

And there’s also great power in terms of being able to diagnose new viruses. The sequencing technology, the functional genomics technologies, all of those things, when applied to virology, give us a much richer picture of how these viruses interact with cells. I think it’s a golden age.

The five known types of viral hepatitis afflict hundreds of millions of people around the world, causing both acute and chronic liver diseases. Among them, types B and C are the most severe, and diagnosis often remains a challenge.
 

You have been working with flaviviruses (dengue, Zika, yellow fever, and hepatitis C) for many decades. Zika and dengue pose an ongoing threat worldwide and, in particular, Latin America. Based on the successful example of hepatitis C, what can scientific research do to mitigate the impact of these viruses?

For viruses like Zika, developing a vaccine is probably going to be fairly straightforward – except that since Zika is so transient, it makes it hard to prove that your vaccine works. You would have to do a human challenge study, in which volunteers are deliberately exposed to an infection in a safe way with health-care support.

For dengue, it’s much more difficult, because there are four different serotypes – different versions of the same virus – and infection with one serotype can put you at increased risk of more severe disease if you get infected with a second serotype. Eliciting a balanced response that would protect you against all four dengue serotypes is the holy grail of trying to develop a dengue vaccine.

People are using various approaches to accomplish that. The classic one is to take live attenuated versions – weakened forms of viruses that have been modified so they can’t cause severe illness but can still stimulate the immune system – of each of the four serotypes and mix them together. Another is to make chimeric viruses: a combination of genetic material from different viruses, resulting in new viruses that have features of each of the four dengue serotypes, engineered into the backbone of the yellow fever vaccine. But this hasn’t worked as well as people have hoped. I think the cocktail of live, attenuated dengue variants is probably the most advanced approach. But I would guess that given the success of COVID-19 mRNA vaccines, the mRNA approach will also be tried out.

These diseases are not going to go away. You can’t eradicate every mosquito. And you can’t really immunize every susceptible vertebrate host. So occasionally there’s going to be spillover into the human population. We need to keep working on these because they are big problems.
 

You began your career at the California Institute of Technology studying RNA viruses, such as the mosquito-borne Sindbis virus, and then flaviviruses that cause encephalitis, polyarthritis, yellow fever, and dengue fever. Later on, you also studied hepatitis C virus. Is there any advantage for virologists in changing the viruses they study throughout their careers?

They’re all interesting, right? And they are all different in their own ways. I say that my career has been a downward spiral of tackling increasingly intricate viruses. Initially, the alphaviruses – a viral family that includes chikungunya virus, for example – were easy. The classical flaviviruses – like yellow fever, dengue fever, West Nile viruses, and Zika virus, among others – were a little more difficult, but the hepatitis C virus was impossible for 15 years, until we, and others, finally achieved a complete replication system in the laboratory.

We coexist daily with viruses, but the pandemic may have given people the idea that all these microorganisms are invariably life-threatening.

We have to treat them with respect. We’ve seen what can happen with the emergence of a novel coronavirus that can spread during an asymptomatic phase of infection. You can’t be prepared for everything, but in some respects our response was a lot slower and less effective than it could have been.

If there’s anything that we’ve learned over the last 10 years with the new nucleic acid sequencing technologies, it’s that our past view of the virosphere was very narrow. And if you really look at what’s out there, the estimated virus diversity is a staggering number, like 1,031 types. Although most of them are not pathogenic to humans, some are. We have to take this threat seriously.
 

Is science prepared?

I think so, but there has to be an investment, a societal investment. And that investment has to not only be an investment in infrastructure that can react quickly to something new, but also to establish a repository of protective antibodies and small molecules against viruses that we know could be future threats.

Often, these things go in cycles. There’s a disaster, like the COVID-19 pandemic, people are changed by the experience, but then they think “Oh, well, the virus has faded into the background, the threat is over.” And that’s just not the case. We need a more sustained plan rather than a reactive stance. And that’s hard to do when resources and money are limited.
 

What is the effect of science illiteracy, conspiracy theories, and lack of science information on the battle against viruses?

These are huge issues, and I don’t know the best way to combat them and educate people. Any combative, confrontational kind of response – it’s just not going to work. People will get more resolute in their entrenched beliefs and not hear or believe compelling evidence to the contrary.

It’s frustrating. I think that we have amazing tools and the power to make really significant advances to help people. It is more than a little discouraging for scientists when there’s a substantial fraction of people who don’t believe in things that are well-supported by facts.

It’s in large part an educational problem. I think we don’t put enough money into education, particularly early education. A lot of people don’t understand how much of what we take for granted today is underpinned by science. All this technology – good, bad or ugly – is all science.
 

This article originally appeared in Knowable Magazine on Oct. 30, 2023. Knowable Magazine is an independent journalistic endeavor from Annual Reviews, a nonprofit publisher dedicated to synthesizing and integrating knowledge for the progress of science and the benefit of society. Sign up for Knowable Magazine’s newsletter.

As the aging population grows and telehealth expands in the wake of the COVID-19 pandemic, an emerging trend of in-home care is reshaping how patients access and receive physical therapy services.

Partnerships between hospitals and home health companies are increasing access to rehabilitation services not only for older adults but also for people in rural areas, those without reliable transportation, and patients with injuries that hinder their driving abilities.

“We find more and more that physical therapy at their home, instead of coming to an outpatient facility, is something more and more folks are requesting,” said Bill Benoit, MBA, chief operating officer of University Hospitals, Cleveland. “In this post-COVID environment, people are getting all different types of services in their home when they’re available, and this is one of them. The pandemic sped up the process of us moving away from the traditional brick and mortar hospital.”

UH recently announced a partnership with Luna Physical Therapy, a company founded in 2018 that provides home services. Luna has teamed up with more than two dozen other hospitals in the United States to offer home-based rehabilitation, according to the company.

The process for arranging in-home therapies through hospital-clinic partnerships is like any other inpatient or outpatient rehabilitation, Mr. Benoit said: A patient meets with a specialist or primary care practitioner, they discuss options, and eventually the clinician recommends physical therapy. The only difference here, he said, is rather than going to a separate facility or a hospital, the patient logs onto a mobile app that matches them with a physical therapist on the basis of their location, needs, and the times they are available.

The prescribing physician oversees the patient’s progress through notes provided by the therapist.

“For the primary care physician or surgeon, they’re not going to see much of a difference,” Mr. Benoit said. “This just adds to that list of options for patients.”
 

Safer, more productive PT

A study, published in the journal Family Practice, found that 76% of patients who are prescribed physical therapy do not initiate the services after it has been recommended.

Aside from the convenience and expanded accessibility for patients, the home therapy option can be more productive, said Denise Wagner, PT, DPT, a physical therapist with Johns Hopkins, Baltimore.

“Home is safer for many patients, but home is also more engaging and motivating,” she said. “Home health clinicians are experts in using whatever they find in the home environment as equipment; many people have stairs in their home, so we can use the rail as something to hold. If patient likes to walk their dog, we can use putting a leash on dog as balance activity.”

Therapy in the home setting helps physical therapists customize programs to fit each patient’s lifestyle, said Gira Shah, PT, a physical therapist with Providence Home Services in Seattle.

For example, patients generally want to know how to function within their own space – navigate their kitchens to make food or get in and out of their bathtubs. Staying in that space allows therapists to focus on those specific goals, Ms. Shah said. “It’s more of a functional therapy. The beauty of this [is that] as therapists we’re trying to assess, ‘what does the patient need to be independent?’ ”

The consulting firm McKinsey predicts that as much as $265 billion in health care services for Medicare recipients will be provided within the home by 2025.

The obvious question is: Why would hospitals partner with clinics rather than offer in-home services on their own?

The answer, like most things in health care, boils down to money.

The billing and documentation system that they use is more efficient than anything hospitals have, said John Brickley, PT, MA, vice president and physical therapist at MedStar Health, a health care system in Maryland and the Washington, D.C., area. MedStar and Luna announced a partnership last June.

“We would financially fall on our face if we tried to use our own billing systems; it would take too much time,” Mr. Brickley said. “Do we need them from a quality-of-care standpoint? No. They have the type of technology that’s not at our disposal.”

Patients should be aware of the difference between home-based PT and other health services for homebound patients, Mr. Brickley said. Medicare considers a patient homebound if they need the help of another person or medical equipment to leave their home or if their doctor believes their condition would worsen with greater mobility.

From the perspective of an insurance company, a home therapy session arranged by a hospital-clinic partnership is an ambulatory appointment and uses the same charging mechanism as most other visits. For a home health care visit, patients must qualify as homebound.

Home-based PT can be used for conditions including neurologic issues, bone and joint problems, balance, and fall deconditioning and prevention. But if a patient needs heavy equipment that cannot be transported, outpatient services are more practical.

That should be determined by the primary care practitioner or specialist evaluating each patient, said Palak Shah, PT, cofounder and head of clinical services at Luna.

“Primary care physicians play a huge role – that’s where patients express their initial concerns,” she said. “It’s up to them to make patients aware about all the options.”

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

As the aging population grows and telehealth expands in the wake of the COVID-19 pandemic, an emerging trend of in-home care is reshaping how patients access and receive physical therapy services.

Partnerships between hospitals and home health companies are increasing access to rehabilitation services not only for older adults but also for people in rural areas, those without reliable transportation, and patients with injuries that hinder their driving abilities.

“We find more and more that physical therapy at their home, instead of coming to an outpatient facility, is something more and more folks are requesting,” said Bill Benoit, MBA, chief operating officer of University Hospitals, Cleveland. “In this post-COVID environment, people are getting all different types of services in their home when they’re available, and this is one of them. The pandemic sped up the process of us moving away from the traditional brick and mortar hospital.”

UH recently announced a partnership with Luna Physical Therapy, a company founded in 2018 that provides home services. Luna has teamed up with more than two dozen other hospitals in the United States to offer home-based rehabilitation, according to the company.

The process for arranging in-home therapies through hospital-clinic partnerships is like any other inpatient or outpatient rehabilitation, Mr. Benoit said: A patient meets with a specialist or primary care practitioner, they discuss options, and eventually the clinician recommends physical therapy. The only difference here, he said, is rather than going to a separate facility or a hospital, the patient logs onto a mobile app that matches them with a physical therapist on the basis of their location, needs, and the times they are available.

The prescribing physician oversees the patient’s progress through notes provided by the therapist.

“For the primary care physician or surgeon, they’re not going to see much of a difference,” Mr. Benoit said. “This just adds to that list of options for patients.”
 

Safer, more productive PT

A study, published in the journal Family Practice, found that 76% of patients who are prescribed physical therapy do not initiate the services after it has been recommended.

Aside from the convenience and expanded accessibility for patients, the home therapy option can be more productive, said Denise Wagner, PT, DPT, a physical therapist with Johns Hopkins, Baltimore.

“Home is safer for many patients, but home is also more engaging and motivating,” she said. “Home health clinicians are experts in using whatever they find in the home environment as equipment; many people have stairs in their home, so we can use the rail as something to hold. If patient likes to walk their dog, we can use putting a leash on dog as balance activity.”

Therapy in the home setting helps physical therapists customize programs to fit each patient’s lifestyle, said Gira Shah, PT, a physical therapist with Providence Home Services in Seattle.

For example, patients generally want to know how to function within their own space – navigate their kitchens to make food or get in and out of their bathtubs. Staying in that space allows therapists to focus on those specific goals, Ms. Shah said. “It’s more of a functional therapy. The beauty of this [is that] as therapists we’re trying to assess, ‘what does the patient need to be independent?’ ”

The consulting firm McKinsey predicts that as much as $265 billion in health care services for Medicare recipients will be provided within the home by 2025.

The obvious question is: Why would hospitals partner with clinics rather than offer in-home services on their own?

The answer, like most things in health care, boils down to money.

The billing and documentation system that they use is more efficient than anything hospitals have, said John Brickley, PT, MA, vice president and physical therapist at MedStar Health, a health care system in Maryland and the Washington, D.C., area. MedStar and Luna announced a partnership last June.

“We would financially fall on our face if we tried to use our own billing systems; it would take too much time,” Mr. Brickley said. “Do we need them from a quality-of-care standpoint? No. They have the type of technology that’s not at our disposal.”

Patients should be aware of the difference between home-based PT and other health services for homebound patients, Mr. Brickley said. Medicare considers a patient homebound if they need the help of another person or medical equipment to leave their home or if their doctor believes their condition would worsen with greater mobility.

From the perspective of an insurance company, a home therapy session arranged by a hospital-clinic partnership is an ambulatory appointment and uses the same charging mechanism as most other visits. For a home health care visit, patients must qualify as homebound.

Home-based PT can be used for conditions including neurologic issues, bone and joint problems, balance, and fall deconditioning and prevention. But if a patient needs heavy equipment that cannot be transported, outpatient services are more practical.

That should be determined by the primary care practitioner or specialist evaluating each patient, said Palak Shah, PT, cofounder and head of clinical services at Luna.

“Primary care physicians play a huge role – that’s where patients express their initial concerns,” she said. “It’s up to them to make patients aware about all the options.”

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

As the aging population grows and telehealth expands in the wake of the COVID-19 pandemic, an emerging trend of in-home care is reshaping how patients access and receive physical therapy services.

Partnerships between hospitals and home health companies are increasing access to rehabilitation services not only for older adults but also for people in rural areas, those without reliable transportation, and patients with injuries that hinder their driving abilities.

“We find more and more that physical therapy at their home, instead of coming to an outpatient facility, is something more and more folks are requesting,” said Bill Benoit, MBA, chief operating officer of University Hospitals, Cleveland. “In this post-COVID environment, people are getting all different types of services in their home when they’re available, and this is one of them. The pandemic sped up the process of us moving away from the traditional brick and mortar hospital.”

UH recently announced a partnership with Luna Physical Therapy, a company founded in 2018 that provides home services. Luna has teamed up with more than two dozen other hospitals in the United States to offer home-based rehabilitation, according to the company.

The process for arranging in-home therapies through hospital-clinic partnerships is like any other inpatient or outpatient rehabilitation, Mr. Benoit said: A patient meets with a specialist or primary care practitioner, they discuss options, and eventually the clinician recommends physical therapy. The only difference here, he said, is rather than going to a separate facility or a hospital, the patient logs onto a mobile app that matches them with a physical therapist on the basis of their location, needs, and the times they are available.

The prescribing physician oversees the patient’s progress through notes provided by the therapist.

“For the primary care physician or surgeon, they’re not going to see much of a difference,” Mr. Benoit said. “This just adds to that list of options for patients.”
 

Safer, more productive PT

A study, published in the journal Family Practice, found that 76% of patients who are prescribed physical therapy do not initiate the services after it has been recommended.

Aside from the convenience and expanded accessibility for patients, the home therapy option can be more productive, said Denise Wagner, PT, DPT, a physical therapist with Johns Hopkins, Baltimore.

“Home is safer for many patients, but home is also more engaging and motivating,” she said. “Home health clinicians are experts in using whatever they find in the home environment as equipment; many people have stairs in their home, so we can use the rail as something to hold. If patient likes to walk their dog, we can use putting a leash on dog as balance activity.”

Therapy in the home setting helps physical therapists customize programs to fit each patient’s lifestyle, said Gira Shah, PT, a physical therapist with Providence Home Services in Seattle.

For example, patients generally want to know how to function within their own space – navigate their kitchens to make food or get in and out of their bathtubs. Staying in that space allows therapists to focus on those specific goals, Ms. Shah said. “It’s more of a functional therapy. The beauty of this [is that] as therapists we’re trying to assess, ‘what does the patient need to be independent?’ ”

The consulting firm McKinsey predicts that as much as $265 billion in health care services for Medicare recipients will be provided within the home by 2025.

The obvious question is: Why would hospitals partner with clinics rather than offer in-home services on their own?

The answer, like most things in health care, boils down to money.

The billing and documentation system that they use is more efficient than anything hospitals have, said John Brickley, PT, MA, vice president and physical therapist at MedStar Health, a health care system in Maryland and the Washington, D.C., area. MedStar and Luna announced a partnership last June.

“We would financially fall on our face if we tried to use our own billing systems; it would take too much time,” Mr. Brickley said. “Do we need them from a quality-of-care standpoint? No. They have the type of technology that’s not at our disposal.”

Patients should be aware of the difference between home-based PT and other health services for homebound patients, Mr. Brickley said. Medicare considers a patient homebound if they need the help of another person or medical equipment to leave their home or if their doctor believes their condition would worsen with greater mobility.

From the perspective of an insurance company, a home therapy session arranged by a hospital-clinic partnership is an ambulatory appointment and uses the same charging mechanism as most other visits. For a home health care visit, patients must qualify as homebound.

Home-based PT can be used for conditions including neurologic issues, bone and joint problems, balance, and fall deconditioning and prevention. But if a patient needs heavy equipment that cannot be transported, outpatient services are more practical.

That should be determined by the primary care practitioner or specialist evaluating each patient, said Palak Shah, PT, cofounder and head of clinical services at Luna.

“Primary care physicians play a huge role – that’s where patients express their initial concerns,” she said. “It’s up to them to make patients aware about all the options.”

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