Supporters of home phototherapy for patients with plaque and guttate psoriasis had plenty to cheer about at the annual meeting of the American Academy of Dermatology (AAD) in March. There, Joel M. Gelfand, MD, professor of dermatology and epidemiology at the University of Pennsylvania in Philadelphia, presented results from the LITE study, a trial that tested the hypothesis that narrowband ultraviolet B phototherapy of psoriasis at home is noninferior to office treatment, based on outcomes that matter to patients, clinicians, and payers. While smaller studies have drawn similar conclusions, LITE was the largest trial of its kind to show what many dermatologists have suspected for years: Home phototherapy can be an effective first-line treatment for patients with moderate to severe psoriasis.

The co-primary outcomes in the LITE study were a Physician’s Global Assessment (PGA) score of 0/1 (clear, almost clear) and a Dermatology Life Quality Index (DLQI) score of 5 or less (small, no effect on health-related quality of life).

Dr. Gelfand and colleagues at 42 sites in the United States enrolled 783 patients aged 12 years and older who had plaque or guttate psoriasis and were candidates for phototherapy at home or in an office setting. Following 12 weeks of treatment, 25.6% of patients in the office-based phototherapy group achieved a PGA score of 0/1 compared with 32.8% of patients in the home-based phototherapy group (P > .0001 for noninferiority, non-response imputation for missing data). Similarly, 33.6% of patients in the office-based phototherapy group achieved a DLQI score of 5 or less compared with 52.4% of patients in the home-based phototherapy group (P > .0001 for noninferiority, non-response imputation for missing data).

Courtesy Dr. Gelfand

A Safe and Effective Option

“I think that it’s important for physicians, insurance companies, and patients with psoriasis to understand that this is a very safe and effective form of therapy,” Craig A. Elmets, MD, professor of dermatology at The University of Alabama at Birmingham, said in an interview. “For people who are not interested in systemic medications or who have contraindications to systemic medications, phototherapy would be ideal,” added Dr. Elmets, first author of the joint AAD–National Psoriasis Foundation (NPF) guidelines for the management and treatment of psoriasis with phototherapy, published in 2019.

Factors beyond efficacy support the role of home phototherapy, Dr. Gelfand said, including the fact that it costs 10-100 times less than biologics for psoriasis and that office-based phototherapy is not available in 90% of counties in the United States. However, insurance coverage of home phototherapy “is highly variable because until the LITE study, there was no large-scale US data to support its use,” he told this news organization.

“Also, insurance companies are broken up into two parts: Durable medical goods and the medical side such as pharmacy costs, and they are siloed. The durable medical goods side views phototherapy as expensive, while the pharmacy side views it as dirt cheap. This is part of the problem with our health system. A lot of things are siloed and don’t make any sense,” said Dr. Gelfand, director of the Psoriasis and Phototherapy Treatment Center at the University of Pennsylvania. By working with the NPF and payers, he added, “we’re hoping ... to transform the way insurance companies think about covering home phototherapy.”

In the meantime, he and Dr. Elmets shared practical ways to optimize access to home phototherapy for psoriasis patients:

Have the discussion. Patients “rarely bring this up as an option,” Dr. Elmets said, so the onus is on clinicians to talk about it. In his view, the ideal candidate “is averse to using systemic agents but whose disease is beyond the point where topical medicines alone will work. One of the advantages of phototherapy is that it doesn’t have immunosuppressive effects.”

Clinicians and patients can learn about the efficacy and safety of phototherapy for psoriasis, including home-based options, on the NPF’s web site and by reading the 2019 joint AAD-NPF guidelines.

Shared decision-making is key. “When a patient comes in, I’ll discuss what their treatment options are and [we] will decide upon a course of action based on their unique needs and preferences [and] if it’s medically appropriate, meaning they have the type of psoriasis likely to respond to phototherapy,” Dr. Gelfand said. A patient with psoriasis mainly on the fingernails or genitals “is not a good candidate for phototherapy. If it’s on the trunk or extremities, that patient would be a good candidate.”

Home phototherapy candidates also must be willing and able to operate a machine and have dedicated space in their dwelling for it (most units are about the size of a door). Patients also have to be reliable, follow directions, and come back in person for follow-up appointments “so we can assess their response to treatment and fine-tune things as necessary and make sure they’re not developing any skin damage,” Dr. Gelfand said.

Educate yourself about existing options. Home phototherapy units from manufacturers such as Daavlin, National Biological Corporation, and SolRx range between $1200 and $6000 in cost, Dr. Gelfand said. He and his colleagues used the Daavlin 7 series in the LITE study. That unit features an integrated dosimetry system that delivers the correct dose of energy based on parameters that the prescribing clinician recommends. Settings are based on the patient’s skin type and how much the prescriber wants to increase the dose for each treatment. “The machine does the rest,” he said. “It knows what dose to give, so they get the same dosing as they would in an office situation.”

Smaller home-based phototherapy units designed to treat the hands and feet are available. So are handheld units to treat the scalp. “These can be a nice option for patients who have a few spots, but if the disease is moderate to severe, then it’s going to be pretty laborious to [use them],” Dr. Elmets said.

Remember that phototherapy is not a cure-all. According to the joint AAD-NPF guidelines, most phototherapy regimens require treatments two to three times per week for 10-14 weeks. Once patients achieve their home phototherapy treatment goal, Dr. Elmets often recommends treatments one to two times per week for maintenance.

“Patients with psoriasis have a lifetime condition,” he noted. “There are certainly cases where people have gone on phototherapy, cleared, and then stopped for a period of time. If they flare up, they can always go back to phototherapy. Usually, people who are on phototherapy use some type of topical agents to touch up areas that are resistant.”

Expect pushback from insurers on coverage. While Medicare and some integrated health plans cover home phototherapy, expect to spend time writing letters or placing phone calls to insurance companies to convince them why they should cover home phototherapy for candidate psoriasis patients. “Usually there’s a lot of letter writing and a long delay in getting approval,” Dr. Elmets said.

Dr. Elmets and Dr. Gelfand reported no relevant financial relationships. The LITE study was funded by the Patient-Centered Outcomes Research Institute. Research partners included the National Psoriasis Foundation and Daavlin, which provided the home phototherapy machines and covered the cost of shipping the devices.

A version of this article appeared on Medscape.com.

Supporters of home phototherapy for patients with plaque and guttate psoriasis had plenty to cheer about at the annual meeting of the American Academy of Dermatology (AAD) in March. There, Joel M. Gelfand, MD, professor of dermatology and epidemiology at the University of Pennsylvania in Philadelphia, presented results from the LITE study, a trial that tested the hypothesis that narrowband ultraviolet B phototherapy of psoriasis at home is noninferior to office treatment, based on outcomes that matter to patients, clinicians, and payers. While smaller studies have drawn similar conclusions, LITE was the largest trial of its kind to show what many dermatologists have suspected for years: Home phototherapy can be an effective first-line treatment for patients with moderate to severe psoriasis.

The co-primary outcomes in the LITE study were a Physician’s Global Assessment (PGA) score of 0/1 (clear, almost clear) and a Dermatology Life Quality Index (DLQI) score of 5 or less (small, no effect on health-related quality of life).

Dr. Gelfand and colleagues at 42 sites in the United States enrolled 783 patients aged 12 years and older who had plaque or guttate psoriasis and were candidates for phototherapy at home or in an office setting. Following 12 weeks of treatment, 25.6% of patients in the office-based phototherapy group achieved a PGA score of 0/1 compared with 32.8% of patients in the home-based phototherapy group (P > .0001 for noninferiority, non-response imputation for missing data). Similarly, 33.6% of patients in the office-based phototherapy group achieved a DLQI score of 5 or less compared with 52.4% of patients in the home-based phototherapy group (P > .0001 for noninferiority, non-response imputation for missing data).

Courtesy Dr. Gelfand

A Safe and Effective Option

“I think that it’s important for physicians, insurance companies, and patients with psoriasis to understand that this is a very safe and effective form of therapy,” Craig A. Elmets, MD, professor of dermatology at The University of Alabama at Birmingham, said in an interview. “For people who are not interested in systemic medications or who have contraindications to systemic medications, phototherapy would be ideal,” added Dr. Elmets, first author of the joint AAD–National Psoriasis Foundation (NPF) guidelines for the management and treatment of psoriasis with phototherapy, published in 2019.

Factors beyond efficacy support the role of home phototherapy, Dr. Gelfand said, including the fact that it costs 10-100 times less than biologics for psoriasis and that office-based phototherapy is not available in 90% of counties in the United States. However, insurance coverage of home phototherapy “is highly variable because until the LITE study, there was no large-scale US data to support its use,” he told this news organization.

“Also, insurance companies are broken up into two parts: Durable medical goods and the medical side such as pharmacy costs, and they are siloed. The durable medical goods side views phototherapy as expensive, while the pharmacy side views it as dirt cheap. This is part of the problem with our health system. A lot of things are siloed and don’t make any sense,” said Dr. Gelfand, director of the Psoriasis and Phototherapy Treatment Center at the University of Pennsylvania. By working with the NPF and payers, he added, “we’re hoping ... to transform the way insurance companies think about covering home phototherapy.”

In the meantime, he and Dr. Elmets shared practical ways to optimize access to home phototherapy for psoriasis patients:

Have the discussion. Patients “rarely bring this up as an option,” Dr. Elmets said, so the onus is on clinicians to talk about it. In his view, the ideal candidate “is averse to using systemic agents but whose disease is beyond the point where topical medicines alone will work. One of the advantages of phototherapy is that it doesn’t have immunosuppressive effects.”

Clinicians and patients can learn about the efficacy and safety of phototherapy for psoriasis, including home-based options, on the NPF’s web site and by reading the 2019 joint AAD-NPF guidelines.

Shared decision-making is key. “When a patient comes in, I’ll discuss what their treatment options are and [we] will decide upon a course of action based on their unique needs and preferences [and] if it’s medically appropriate, meaning they have the type of psoriasis likely to respond to phototherapy,” Dr. Gelfand said. A patient with psoriasis mainly on the fingernails or genitals “is not a good candidate for phototherapy. If it’s on the trunk or extremities, that patient would be a good candidate.”

Home phototherapy candidates also must be willing and able to operate a machine and have dedicated space in their dwelling for it (most units are about the size of a door). Patients also have to be reliable, follow directions, and come back in person for follow-up appointments “so we can assess their response to treatment and fine-tune things as necessary and make sure they’re not developing any skin damage,” Dr. Gelfand said.

Educate yourself about existing options. Home phototherapy units from manufacturers such as Daavlin, National Biological Corporation, and SolRx range between $1200 and $6000 in cost, Dr. Gelfand said. He and his colleagues used the Daavlin 7 series in the LITE study. That unit features an integrated dosimetry system that delivers the correct dose of energy based on parameters that the prescribing clinician recommends. Settings are based on the patient’s skin type and how much the prescriber wants to increase the dose for each treatment. “The machine does the rest,” he said. “It knows what dose to give, so they get the same dosing as they would in an office situation.”

Smaller home-based phototherapy units designed to treat the hands and feet are available. So are handheld units to treat the scalp. “These can be a nice option for patients who have a few spots, but if the disease is moderate to severe, then it’s going to be pretty laborious to [use them],” Dr. Elmets said.

Remember that phototherapy is not a cure-all. According to the joint AAD-NPF guidelines, most phototherapy regimens require treatments two to three times per week for 10-14 weeks. Once patients achieve their home phototherapy treatment goal, Dr. Elmets often recommends treatments one to two times per week for maintenance.

“Patients with psoriasis have a lifetime condition,” he noted. “There are certainly cases where people have gone on phototherapy, cleared, and then stopped for a period of time. If they flare up, they can always go back to phototherapy. Usually, people who are on phototherapy use some type of topical agents to touch up areas that are resistant.”

Expect pushback from insurers on coverage. While Medicare and some integrated health plans cover home phototherapy, expect to spend time writing letters or placing phone calls to insurance companies to convince them why they should cover home phototherapy for candidate psoriasis patients. “Usually there’s a lot of letter writing and a long delay in getting approval,” Dr. Elmets said.

Dr. Elmets and Dr. Gelfand reported no relevant financial relationships. The LITE study was funded by the Patient-Centered Outcomes Research Institute. Research partners included the National Psoriasis Foundation and Daavlin, which provided the home phototherapy machines and covered the cost of shipping the devices.

A version of this article appeared on Medscape.com.

Supporters of home phototherapy for patients with plaque and guttate psoriasis had plenty to cheer about at the annual meeting of the American Academy of Dermatology (AAD) in March. There, Joel M. Gelfand, MD, professor of dermatology and epidemiology at the University of Pennsylvania in Philadelphia, presented results from the LITE study, a trial that tested the hypothesis that narrowband ultraviolet B phototherapy of psoriasis at home is noninferior to office treatment, based on outcomes that matter to patients, clinicians, and payers. While smaller studies have drawn similar conclusions, LITE was the largest trial of its kind to show what many dermatologists have suspected for years: Home phototherapy can be an effective first-line treatment for patients with moderate to severe psoriasis.

The co-primary outcomes in the LITE study were a Physician’s Global Assessment (PGA) score of 0/1 (clear, almost clear) and a Dermatology Life Quality Index (DLQI) score of 5 or less (small, no effect on health-related quality of life).

Dr. Gelfand and colleagues at 42 sites in the United States enrolled 783 patients aged 12 years and older who had plaque or guttate psoriasis and were candidates for phototherapy at home or in an office setting. Following 12 weeks of treatment, 25.6% of patients in the office-based phototherapy group achieved a PGA score of 0/1 compared with 32.8% of patients in the home-based phototherapy group (P > .0001 for noninferiority, non-response imputation for missing data). Similarly, 33.6% of patients in the office-based phototherapy group achieved a DLQI score of 5 or less compared with 52.4% of patients in the home-based phototherapy group (P > .0001 for noninferiority, non-response imputation for missing data).

Courtesy Dr. Gelfand

A Safe and Effective Option

“I think that it’s important for physicians, insurance companies, and patients with psoriasis to understand that this is a very safe and effective form of therapy,” Craig A. Elmets, MD, professor of dermatology at The University of Alabama at Birmingham, said in an interview. “For people who are not interested in systemic medications or who have contraindications to systemic medications, phototherapy would be ideal,” added Dr. Elmets, first author of the joint AAD–National Psoriasis Foundation (NPF) guidelines for the management and treatment of psoriasis with phototherapy, published in 2019.

Factors beyond efficacy support the role of home phototherapy, Dr. Gelfand said, including the fact that it costs 10-100 times less than biologics for psoriasis and that office-based phototherapy is not available in 90% of counties in the United States. However, insurance coverage of home phototherapy “is highly variable because until the LITE study, there was no large-scale US data to support its use,” he told this news organization.

“Also, insurance companies are broken up into two parts: Durable medical goods and the medical side such as pharmacy costs, and they are siloed. The durable medical goods side views phototherapy as expensive, while the pharmacy side views it as dirt cheap. This is part of the problem with our health system. A lot of things are siloed and don’t make any sense,” said Dr. Gelfand, director of the Psoriasis and Phototherapy Treatment Center at the University of Pennsylvania. By working with the NPF and payers, he added, “we’re hoping ... to transform the way insurance companies think about covering home phototherapy.”

In the meantime, he and Dr. Elmets shared practical ways to optimize access to home phototherapy for psoriasis patients:

Have the discussion. Patients “rarely bring this up as an option,” Dr. Elmets said, so the onus is on clinicians to talk about it. In his view, the ideal candidate “is averse to using systemic agents but whose disease is beyond the point where topical medicines alone will work. One of the advantages of phototherapy is that it doesn’t have immunosuppressive effects.”

Clinicians and patients can learn about the efficacy and safety of phototherapy for psoriasis, including home-based options, on the NPF’s web site and by reading the 2019 joint AAD-NPF guidelines.

Shared decision-making is key. “When a patient comes in, I’ll discuss what their treatment options are and [we] will decide upon a course of action based on their unique needs and preferences [and] if it’s medically appropriate, meaning they have the type of psoriasis likely to respond to phototherapy,” Dr. Gelfand said. A patient with psoriasis mainly on the fingernails or genitals “is not a good candidate for phototherapy. If it’s on the trunk or extremities, that patient would be a good candidate.”

Home phototherapy candidates also must be willing and able to operate a machine and have dedicated space in their dwelling for it (most units are about the size of a door). Patients also have to be reliable, follow directions, and come back in person for follow-up appointments “so we can assess their response to treatment and fine-tune things as necessary and make sure they’re not developing any skin damage,” Dr. Gelfand said.

Educate yourself about existing options. Home phototherapy units from manufacturers such as Daavlin, National Biological Corporation, and SolRx range between $1200 and $6000 in cost, Dr. Gelfand said. He and his colleagues used the Daavlin 7 series in the LITE study. That unit features an integrated dosimetry system that delivers the correct dose of energy based on parameters that the prescribing clinician recommends. Settings are based on the patient’s skin type and how much the prescriber wants to increase the dose for each treatment. “The machine does the rest,” he said. “It knows what dose to give, so they get the same dosing as they would in an office situation.”

Smaller home-based phototherapy units designed to treat the hands and feet are available. So are handheld units to treat the scalp. “These can be a nice option for patients who have a few spots, but if the disease is moderate to severe, then it’s going to be pretty laborious to [use them],” Dr. Elmets said.

Remember that phototherapy is not a cure-all. According to the joint AAD-NPF guidelines, most phototherapy regimens require treatments two to three times per week for 10-14 weeks. Once patients achieve their home phototherapy treatment goal, Dr. Elmets often recommends treatments one to two times per week for maintenance.

“Patients with psoriasis have a lifetime condition,” he noted. “There are certainly cases where people have gone on phototherapy, cleared, and then stopped for a period of time. If they flare up, they can always go back to phototherapy. Usually, people who are on phototherapy use some type of topical agents to touch up areas that are resistant.”

Expect pushback from insurers on coverage. While Medicare and some integrated health plans cover home phototherapy, expect to spend time writing letters or placing phone calls to insurance companies to convince them why they should cover home phototherapy for candidate psoriasis patients. “Usually there’s a lot of letter writing and a long delay in getting approval,” Dr. Elmets said.

Dr. Elmets and Dr. Gelfand reported no relevant financial relationships. The LITE study was funded by the Patient-Centered Outcomes Research Institute. Research partners included the National Psoriasis Foundation and Daavlin, which provided the home phototherapy machines and covered the cost of shipping the devices.

A version of this article appeared on Medscape.com.

NEW ORLEANS — Postmenopausal women with osteoporosis may not be receiving all the recommended tests to rule out secondary causes of bone loss prior to treatment initiation, new research found.

In a single-center chart review of 150 postmenopausal women who had been diagnosed and treated for osteoporosis, most had received a complete blood cell count, basic metabolic panel, thyroid screening, and vitamin D testing. However, one in four had not been tested for a parathyroid hormone (PTH) level, and in nearly two thirds, a 24-hour urine calcium collection had not been ordered.

Overall, less than a third had received the complete workup for secondary osteoporosis causes as recommended by the American Association of Clinical Endocrinologists (AACE) and the Endocrine Society.

“An appropriate evaluation for secondary causes of osteoporosis is essential because it impacts different treatment options and modalities. We discovered low rates of complete testing for secondary causes of osteoporosis in our patient population prior to treatment initiation,” said Kajol Manglani, MD, an internal medicine resident at Georgetown University/MedStar Washington Hospital Center, Washington, DC, and colleagues, in a poster at the American Association of Clinical Endocrinology (AACE) annual meeting held on May 9-12, 2024.

First author Sheetal Bulchandani, MD, said in an interview, “It depends a lot on clinical judgment, but there are certain things that everybody with osteoporosis should be evaluated for. We looked for the things that all the guidelines recommend.”

Studies have suggested that up to 30% of postmenopausal women with osteoporosis have secondary causes, noted Dr. Bulchandani, who conducted the study as a postdoctoral fellow with colleagues at Georgetown University/MedStar Washington Hospital and is now in private endocrine practice in Petersburg, Virginia.

“It’s important not to assume that every woman who walks in with osteoporosis has postmenopausal osteoporosis. I think it would be appropriate to at least discuss with the patients what would warrant certain kinds of clinical workup. … If you don’t figure out if there is an underlying cause, you may end up using an unnecessary medication,” Dr. Bulchandani said.
 

Are You Missing Something Treatable?

For example, she said, if the patient has underlying hyperparathyroidism and is treated with osteoporosis medications, “you might not see the desired or expected outcome in their bone density.”

Asked to comment, Rachel Pessah-Pollack, MD, clinical associate professor at the Holman Division of Endocrinology, Diabetes, and Metabolism at New York University School of Medicine, New York City, told this news organization, “Certainly, if you have patients who have osteoporosis, it’s important to take a good history and consider secondary causes of bone loss because you may find a treatable etiology that actually can improve their bone density without even starting on a medication.”

Dr. Pessah-Pollack, who was an author of the 2020 AACE/American College of Endocrinology 2020 Clinical Practice Guidelines for the Diagnosis and Treatment of Osteoporosis, said a 24-hour urine calcium collection, not a spot calcium check, is “super important because you’re looking to see if there’s any evidence of hypercalciuria or malabsorption that may be associated with higher rates of bone loss. … These may be a little more cumbersome and harder to get patients to do and more logistics to arrange. But clearly, if you pick up hypercalciuria, that is a potentially treatable etiology and can improve bone density as well.”

Another example, Dr. Pessah-Pollack said, is “if they have a low serum calcium level and high PTH, that would be a real reason to look for celiac disease. By not getting that PTH level, you may be missing that potential diagnosis. There is a wide range of additional causes of osteoporosis ranging from common conditions such as hyperthyroidism to rare conditions such as Cushing disease.”
 

Differences in Ordering Found Across Specialties

The 150 postmenopausal women were all receiving treatment with either alendronate, denosumab, or zoledronic acid. Their average age was 64.7 years, and 63% were seeing an endocrinologist.

Complete workups as per AACE and Endocrine Society guidelines had been performed in just 28% of those who saw an endocrinologist and 12.5% of patients seen by a rheumatologist, in contrast to 84% of those who saw the head of the hospital’s fracture prevention program.

Overall, across all specialties, just 28.67% had the complete recommended workup for secondary osteoporosis causes.

The most missed test was a 24-hour urine calcium collection, ordered for just 38% of the patients, while PTH was ordered for 73% and phosphorus for 80%. The rest were more commonly ordered: Thyroid-stimulating hormone level for 92.7%, complete blood cell count for 91.3%, basic metabolic panel for 100%, and vitamin D level for 96%.

The high rate of vitamin D testing is noteworthy, Dr. Pessah-Pollack said. “The fact that 96% of women are having vitamin D levels checked as part of an osteoporosis evaluation means that everybody’s aware about vitamin D deficiency, and people want to know what their vitamin D levels are. … That’s good because we want to identify vitamin D deficiency in our osteoporosis patients.”

But the low rate of complete secondary screening even by endocrinologists is concerning. “I look at this study as an opportunity for education that we can reinforce the importance of a secondary evaluation for our osteoporosis patients and really tailor which additional tests should be ordered for the individual patient,” Dr. Pessah-Pollack said.

In the poster, Dr. Bulchandani and colleagues wrote, “Further intervention will be aimed to ensure physicians undertake adequate evaluation before considering further treatment directions.” Possibilities that have been discussed include electronic health record alerts and educational materials for primary care providers, she told this news organization.

Dr. Manglani and Dr. Bulchandani had no disclosures. Dr. Pessah-Pollack is an advisor for Boehringer Ingelheim and Eli Lilly.

A version of this article appeared on Medscape.com.

NEW ORLEANS — Postmenopausal women with osteoporosis may not be receiving all the recommended tests to rule out secondary causes of bone loss prior to treatment initiation, new research found.

In a single-center chart review of 150 postmenopausal women who had been diagnosed and treated for osteoporosis, most had received a complete blood cell count, basic metabolic panel, thyroid screening, and vitamin D testing. However, one in four had not been tested for a parathyroid hormone (PTH) level, and in nearly two thirds, a 24-hour urine calcium collection had not been ordered.

Overall, less than a third had received the complete workup for secondary osteoporosis causes as recommended by the American Association of Clinical Endocrinologists (AACE) and the Endocrine Society.

“An appropriate evaluation for secondary causes of osteoporosis is essential because it impacts different treatment options and modalities. We discovered low rates of complete testing for secondary causes of osteoporosis in our patient population prior to treatment initiation,” said Kajol Manglani, MD, an internal medicine resident at Georgetown University/MedStar Washington Hospital Center, Washington, DC, and colleagues, in a poster at the American Association of Clinical Endocrinology (AACE) annual meeting held on May 9-12, 2024.

First author Sheetal Bulchandani, MD, said in an interview, “It depends a lot on clinical judgment, but there are certain things that everybody with osteoporosis should be evaluated for. We looked for the things that all the guidelines recommend.”

Studies have suggested that up to 30% of postmenopausal women with osteoporosis have secondary causes, noted Dr. Bulchandani, who conducted the study as a postdoctoral fellow with colleagues at Georgetown University/MedStar Washington Hospital and is now in private endocrine practice in Petersburg, Virginia.

“It’s important not to assume that every woman who walks in with osteoporosis has postmenopausal osteoporosis. I think it would be appropriate to at least discuss with the patients what would warrant certain kinds of clinical workup. … If you don’t figure out if there is an underlying cause, you may end up using an unnecessary medication,” Dr. Bulchandani said.
 

Are You Missing Something Treatable?

For example, she said, if the patient has underlying hyperparathyroidism and is treated with osteoporosis medications, “you might not see the desired or expected outcome in their bone density.”

Asked to comment, Rachel Pessah-Pollack, MD, clinical associate professor at the Holman Division of Endocrinology, Diabetes, and Metabolism at New York University School of Medicine, New York City, told this news organization, “Certainly, if you have patients who have osteoporosis, it’s important to take a good history and consider secondary causes of bone loss because you may find a treatable etiology that actually can improve their bone density without even starting on a medication.”

Dr. Pessah-Pollack, who was an author of the 2020 AACE/American College of Endocrinology 2020 Clinical Practice Guidelines for the Diagnosis and Treatment of Osteoporosis, said a 24-hour urine calcium collection, not a spot calcium check, is “super important because you’re looking to see if there’s any evidence of hypercalciuria or malabsorption that may be associated with higher rates of bone loss. … These may be a little more cumbersome and harder to get patients to do and more logistics to arrange. But clearly, if you pick up hypercalciuria, that is a potentially treatable etiology and can improve bone density as well.”

Another example, Dr. Pessah-Pollack said, is “if they have a low serum calcium level and high PTH, that would be a real reason to look for celiac disease. By not getting that PTH level, you may be missing that potential diagnosis. There is a wide range of additional causes of osteoporosis ranging from common conditions such as hyperthyroidism to rare conditions such as Cushing disease.”
 

Differences in Ordering Found Across Specialties

The 150 postmenopausal women were all receiving treatment with either alendronate, denosumab, or zoledronic acid. Their average age was 64.7 years, and 63% were seeing an endocrinologist.

Complete workups as per AACE and Endocrine Society guidelines had been performed in just 28% of those who saw an endocrinologist and 12.5% of patients seen by a rheumatologist, in contrast to 84% of those who saw the head of the hospital’s fracture prevention program.

Overall, across all specialties, just 28.67% had the complete recommended workup for secondary osteoporosis causes.

The most missed test was a 24-hour urine calcium collection, ordered for just 38% of the patients, while PTH was ordered for 73% and phosphorus for 80%. The rest were more commonly ordered: Thyroid-stimulating hormone level for 92.7%, complete blood cell count for 91.3%, basic metabolic panel for 100%, and vitamin D level for 96%.

The high rate of vitamin D testing is noteworthy, Dr. Pessah-Pollack said. “The fact that 96% of women are having vitamin D levels checked as part of an osteoporosis evaluation means that everybody’s aware about vitamin D deficiency, and people want to know what their vitamin D levels are. … That’s good because we want to identify vitamin D deficiency in our osteoporosis patients.”

But the low rate of complete secondary screening even by endocrinologists is concerning. “I look at this study as an opportunity for education that we can reinforce the importance of a secondary evaluation for our osteoporosis patients and really tailor which additional tests should be ordered for the individual patient,” Dr. Pessah-Pollack said.

In the poster, Dr. Bulchandani and colleagues wrote, “Further intervention will be aimed to ensure physicians undertake adequate evaluation before considering further treatment directions.” Possibilities that have been discussed include electronic health record alerts and educational materials for primary care providers, she told this news organization.

Dr. Manglani and Dr. Bulchandani had no disclosures. Dr. Pessah-Pollack is an advisor for Boehringer Ingelheim and Eli Lilly.

A version of this article appeared on Medscape.com.

NEW ORLEANS — Postmenopausal women with osteoporosis may not be receiving all the recommended tests to rule out secondary causes of bone loss prior to treatment initiation, new research found.

In a single-center chart review of 150 postmenopausal women who had been diagnosed and treated for osteoporosis, most had received a complete blood cell count, basic metabolic panel, thyroid screening, and vitamin D testing. However, one in four had not been tested for a parathyroid hormone (PTH) level, and in nearly two thirds, a 24-hour urine calcium collection had not been ordered.

Overall, less than a third had received the complete workup for secondary osteoporosis causes as recommended by the American Association of Clinical Endocrinologists (AACE) and the Endocrine Society.

“An appropriate evaluation for secondary causes of osteoporosis is essential because it impacts different treatment options and modalities. We discovered low rates of complete testing for secondary causes of osteoporosis in our patient population prior to treatment initiation,” said Kajol Manglani, MD, an internal medicine resident at Georgetown University/MedStar Washington Hospital Center, Washington, DC, and colleagues, in a poster at the American Association of Clinical Endocrinology (AACE) annual meeting held on May 9-12, 2024.

First author Sheetal Bulchandani, MD, said in an interview, “It depends a lot on clinical judgment, but there are certain things that everybody with osteoporosis should be evaluated for. We looked for the things that all the guidelines recommend.”

Studies have suggested that up to 30% of postmenopausal women with osteoporosis have secondary causes, noted Dr. Bulchandani, who conducted the study as a postdoctoral fellow with colleagues at Georgetown University/MedStar Washington Hospital and is now in private endocrine practice in Petersburg, Virginia.

“It’s important not to assume that every woman who walks in with osteoporosis has postmenopausal osteoporosis. I think it would be appropriate to at least discuss with the patients what would warrant certain kinds of clinical workup. … If you don’t figure out if there is an underlying cause, you may end up using an unnecessary medication,” Dr. Bulchandani said.
 

Are You Missing Something Treatable?

For example, she said, if the patient has underlying hyperparathyroidism and is treated with osteoporosis medications, “you might not see the desired or expected outcome in their bone density.”

Asked to comment, Rachel Pessah-Pollack, MD, clinical associate professor at the Holman Division of Endocrinology, Diabetes, and Metabolism at New York University School of Medicine, New York City, told this news organization, “Certainly, if you have patients who have osteoporosis, it’s important to take a good history and consider secondary causes of bone loss because you may find a treatable etiology that actually can improve their bone density without even starting on a medication.”

Dr. Pessah-Pollack, who was an author of the 2020 AACE/American College of Endocrinology 2020 Clinical Practice Guidelines for the Diagnosis and Treatment of Osteoporosis, said a 24-hour urine calcium collection, not a spot calcium check, is “super important because you’re looking to see if there’s any evidence of hypercalciuria or malabsorption that may be associated with higher rates of bone loss. … These may be a little more cumbersome and harder to get patients to do and more logistics to arrange. But clearly, if you pick up hypercalciuria, that is a potentially treatable etiology and can improve bone density as well.”

Another example, Dr. Pessah-Pollack said, is “if they have a low serum calcium level and high PTH, that would be a real reason to look for celiac disease. By not getting that PTH level, you may be missing that potential diagnosis. There is a wide range of additional causes of osteoporosis ranging from common conditions such as hyperthyroidism to rare conditions such as Cushing disease.”
 

Differences in Ordering Found Across Specialties

The 150 postmenopausal women were all receiving treatment with either alendronate, denosumab, or zoledronic acid. Their average age was 64.7 years, and 63% were seeing an endocrinologist.

Complete workups as per AACE and Endocrine Society guidelines had been performed in just 28% of those who saw an endocrinologist and 12.5% of patients seen by a rheumatologist, in contrast to 84% of those who saw the head of the hospital’s fracture prevention program.

Overall, across all specialties, just 28.67% had the complete recommended workup for secondary osteoporosis causes.

The most missed test was a 24-hour urine calcium collection, ordered for just 38% of the patients, while PTH was ordered for 73% and phosphorus for 80%. The rest were more commonly ordered: Thyroid-stimulating hormone level for 92.7%, complete blood cell count for 91.3%, basic metabolic panel for 100%, and vitamin D level for 96%.

The high rate of vitamin D testing is noteworthy, Dr. Pessah-Pollack said. “The fact that 96% of women are having vitamin D levels checked as part of an osteoporosis evaluation means that everybody’s aware about vitamin D deficiency, and people want to know what their vitamin D levels are. … That’s good because we want to identify vitamin D deficiency in our osteoporosis patients.”

But the low rate of complete secondary screening even by endocrinologists is concerning. “I look at this study as an opportunity for education that we can reinforce the importance of a secondary evaluation for our osteoporosis patients and really tailor which additional tests should be ordered for the individual patient,” Dr. Pessah-Pollack said.

In the poster, Dr. Bulchandani and colleagues wrote, “Further intervention will be aimed to ensure physicians undertake adequate evaluation before considering further treatment directions.” Possibilities that have been discussed include electronic health record alerts and educational materials for primary care providers, she told this news organization.

Dr. Manglani and Dr. Bulchandani had no disclosures. Dr. Pessah-Pollack is an advisor for Boehringer Ingelheim and Eli Lilly.

A version of this article appeared on Medscape.com.

SAN DIEGO — While there is currently no smoking gun definitively showing that indoor nitrogen dioxide (NO₂) concentrations from gas appliances are a cause of pulmonary diseases, the circumstantial evidence of the baleful effects of gas stoves on lung function is pretty compelling, said participants in a pro-con debate.

In what the moderator called “one of the most agreeable debates yet,” experts presented their views on the risks that cooking with natural gas pose on pulmonary health, and discussed ways for mitigating that harm. The debate was held at the American Thoracic Society’s international conference.
 

PRO: Gas stoves cause lung disease

Arguing for the “pro” side, John R. Balmes, MD of the University of California, San Francisco, and a physician member of the California Air Resources Board, began by admitting that “I would never have said gas stoves cause lung disease, but that’s what they assigned me.”

Gamely proceeding anyway, Dr. Balmes noted that natural gas — methane — is a potent greenhouse gas, and that cooking with natural gas leads to generation of NO₂ with high peak concentrations in the home, especially in the kitchen, but in other rooms as well.

Neil Osterweil/MDedge News

Con: More evidence needed

Arguing for the “con” side of the question, Meredith C. McCormack, MD, MHS, professor of medicine in the pulmonary and critical care division at Johns Hopkins University in Baltimore, said that “more definitive evidence is needed to define whether gas stoves cause lung disease.”

But Dr. McCormack didn’t let the natural gas industry off the hook, noting that a systematic review and meta-analysis of cooking with gas in high-, middle-, and low-income countries showed that domestic use of gas fuels vs. electric was associated with increased risk of asthma (1.11 overall), COPD (1.15), and pneumonia (1.26).

Neil Osterweil/MDedge News

On common ground

Environmental interventions that can benefit all members of a household — not just those with obstructive pulmonary disease — include smoking cessation, charcoal filter-equipped air cleaners, stove hoods that vent outdoors, integrated pest management, hypoallergenic pillow and mattress covers, high efficiency particulate air (HEPA) vacuums, and mold and radon abatement.

Both Dr. Balmes and Dr. McCormack agreed in the end that gas stoves contribute to respiratory morbidity, and that both state and national policy changes are needed to support transition to cleaner indoor air, with financial incentives available for households with more modest incomes.

“For everyone, there is a climate-change mitigation imperative to transition away from gas appliances if we want to tackle the climate emergency,” Dr. Balmes said.
 

End indoor combustion

George D. Thurston, ScD, professor of medicine and population health at the NYU Grossman School of Medicine, who attended the debate, told Chest Physician that the participants talked about NO₂ but didn’t touch on particulate pollution generated by gas stoves.

Burning natural gas produces particles that are very similar in composition to those produced by burning coal, oil, or diesel fuel, Dr. Thurston said, and he pointed out that interventions such as range hoods work only if they actually vent outdoors, and aren’t simply fans that recirculate the air within the home. And even when ventilation works as it should to move air out of the house, it only pumps it back into the atmosphere, where it contributes to climate change.

“We need combustion-free homes. That’s the unifying principle. We have to keep our eyes on that prize,” he said.

Dr. Balmes, Dr. McCormack, and Dr. Thurston all reported having no relevant disclosures.

SAN DIEGO — While there is currently no smoking gun definitively showing that indoor nitrogen dioxide (NO₂) concentrations from gas appliances are a cause of pulmonary diseases, the circumstantial evidence of the baleful effects of gas stoves on lung function is pretty compelling, said participants in a pro-con debate.

In what the moderator called “one of the most agreeable debates yet,” experts presented their views on the risks that cooking with natural gas pose on pulmonary health, and discussed ways for mitigating that harm. The debate was held at the American Thoracic Society’s international conference.
 

PRO: Gas stoves cause lung disease

Arguing for the “pro” side, John R. Balmes, MD of the University of California, San Francisco, and a physician member of the California Air Resources Board, began by admitting that “I would never have said gas stoves cause lung disease, but that’s what they assigned me.”

Gamely proceeding anyway, Dr. Balmes noted that natural gas — methane — is a potent greenhouse gas, and that cooking with natural gas leads to generation of NO₂ with high peak concentrations in the home, especially in the kitchen, but in other rooms as well.

Neil Osterweil/MDedge News

Con: More evidence needed

Arguing for the “con” side of the question, Meredith C. McCormack, MD, MHS, professor of medicine in the pulmonary and critical care division at Johns Hopkins University in Baltimore, said that “more definitive evidence is needed to define whether gas stoves cause lung disease.”

But Dr. McCormack didn’t let the natural gas industry off the hook, noting that a systematic review and meta-analysis of cooking with gas in high-, middle-, and low-income countries showed that domestic use of gas fuels vs. electric was associated with increased risk of asthma (1.11 overall), COPD (1.15), and pneumonia (1.26).

Neil Osterweil/MDedge News

On common ground

Environmental interventions that can benefit all members of a household — not just those with obstructive pulmonary disease — include smoking cessation, charcoal filter-equipped air cleaners, stove hoods that vent outdoors, integrated pest management, hypoallergenic pillow and mattress covers, high efficiency particulate air (HEPA) vacuums, and mold and radon abatement.

Both Dr. Balmes and Dr. McCormack agreed in the end that gas stoves contribute to respiratory morbidity, and that both state and national policy changes are needed to support transition to cleaner indoor air, with financial incentives available for households with more modest incomes.

“For everyone, there is a climate-change mitigation imperative to transition away from gas appliances if we want to tackle the climate emergency,” Dr. Balmes said.
 

End indoor combustion

George D. Thurston, ScD, professor of medicine and population health at the NYU Grossman School of Medicine, who attended the debate, told Chest Physician that the participants talked about NO₂ but didn’t touch on particulate pollution generated by gas stoves.

Burning natural gas produces particles that are very similar in composition to those produced by burning coal, oil, or diesel fuel, Dr. Thurston said, and he pointed out that interventions such as range hoods work only if they actually vent outdoors, and aren’t simply fans that recirculate the air within the home. And even when ventilation works as it should to move air out of the house, it only pumps it back into the atmosphere, where it contributes to climate change.

“We need combustion-free homes. That’s the unifying principle. We have to keep our eyes on that prize,” he said.

Dr. Balmes, Dr. McCormack, and Dr. Thurston all reported having no relevant disclosures.

SAN DIEGO — While there is currently no smoking gun definitively showing that indoor nitrogen dioxide (NO₂) concentrations from gas appliances are a cause of pulmonary diseases, the circumstantial evidence of the baleful effects of gas stoves on lung function is pretty compelling, said participants in a pro-con debate.

In what the moderator called “one of the most agreeable debates yet,” experts presented their views on the risks that cooking with natural gas pose on pulmonary health, and discussed ways for mitigating that harm. The debate was held at the American Thoracic Society’s international conference.
 

PRO: Gas stoves cause lung disease

Arguing for the “pro” side, John R. Balmes, MD of the University of California, San Francisco, and a physician member of the California Air Resources Board, began by admitting that “I would never have said gas stoves cause lung disease, but that’s what they assigned me.”

Gamely proceeding anyway, Dr. Balmes noted that natural gas — methane — is a potent greenhouse gas, and that cooking with natural gas leads to generation of NO₂ with high peak concentrations in the home, especially in the kitchen, but in other rooms as well.

Neil Osterweil/MDedge News

Con: More evidence needed

Arguing for the “con” side of the question, Meredith C. McCormack, MD, MHS, professor of medicine in the pulmonary and critical care division at Johns Hopkins University in Baltimore, said that “more definitive evidence is needed to define whether gas stoves cause lung disease.”

But Dr. McCormack didn’t let the natural gas industry off the hook, noting that a systematic review and meta-analysis of cooking with gas in high-, middle-, and low-income countries showed that domestic use of gas fuels vs. electric was associated with increased risk of asthma (1.11 overall), COPD (1.15), and pneumonia (1.26).

Neil Osterweil/MDedge News

On common ground

Environmental interventions that can benefit all members of a household — not just those with obstructive pulmonary disease — include smoking cessation, charcoal filter-equipped air cleaners, stove hoods that vent outdoors, integrated pest management, hypoallergenic pillow and mattress covers, high efficiency particulate air (HEPA) vacuums, and mold and radon abatement.

Both Dr. Balmes and Dr. McCormack agreed in the end that gas stoves contribute to respiratory morbidity, and that both state and national policy changes are needed to support transition to cleaner indoor air, with financial incentives available for households with more modest incomes.

“For everyone, there is a climate-change mitigation imperative to transition away from gas appliances if we want to tackle the climate emergency,” Dr. Balmes said.
 

End indoor combustion

George D. Thurston, ScD, professor of medicine and population health at the NYU Grossman School of Medicine, who attended the debate, told Chest Physician that the participants talked about NO₂ but didn’t touch on particulate pollution generated by gas stoves.

Burning natural gas produces particles that are very similar in composition to those produced by burning coal, oil, or diesel fuel, Dr. Thurston said, and he pointed out that interventions such as range hoods work only if they actually vent outdoors, and aren’t simply fans that recirculate the air within the home. And even when ventilation works as it should to move air out of the house, it only pumps it back into the atmosphere, where it contributes to climate change.

“We need combustion-free homes. That’s the unifying principle. We have to keep our eyes on that prize,” he said.

Dr. Balmes, Dr. McCormack, and Dr. Thurston all reported having no relevant disclosures.

Older patients prescribed hydroxychloroquine (HCQ) have a higher risk of developing retinal damage from taking the medication, according to a new analysis.

In addition to known risk factors such as a higher weight-based HCQ dose and higher cumulative dose, researchers also found that female sex, chronic kidney disease stage III, and tamoxifen use were associated with HCQ retinopathy.

The findings provide “evidence for other key risk factors for hydroxychloroquine retinopathy beyond hydroxychloroquine exposure itself,” wrote April M. Jorge, MD, of the Division of Rheumatology, Allergy, and Immunology at Massachusetts General Hospital, Boston, and colleagues.

Baylor College of Medicine

Study Details

Researchers identified patients in the Kaiser Permanente Northern California (KPNC), Oakland, California, health system who began taking HCQ between July 1, 1997, and December 14, 2014. To be included, patients needed to have at least 5 years of continuous enrollment in the KPNC system and at least one prescription for HCQ after more than 5 years of starting the drug. Patients were followed from HCQ initiation to their last retinopathy screening study, up to December 31, 2020.

The study was published May 9 in JAMA Network Open.

Of the 4677 users followed for the study, 83% were women, and the average age starting HCQ was 52. Most patients were White (58.1%), while 13.7% were Asian, 10.5% were Black, and 17.7% were Hispanic.

More than 60% of patients had an initial dose > 5 mg/kg/d, though the mean initial dose of HCQ was 4.4 mg/kg/d. After 5 years, only 34.4% of patients were using a daily dose over 5 mg/kg.

Of the entire cohort, 125 patients (2.7%) developed HCQ retinopathy. As expected, cumulative HCQ exposure was associated with a higher retinopathy risk: For every 100 g of HCQ cumulative exposure, risk rose by 64% (hazard ratio [HR], 1.64; 95% CI, 1.44-1.87).

Age was a significant risk factor for retinal damage from HCQ use. Individuals who began taking the drug at 65 years or older were nearly six times more likely to develop retinopathy than those who started HCQ when they were younger than 45. In people aged 55-64 years, this risk was nearly four times higher, and individuals aged 45-54 years when starting the drug were 2.5 times more likely to have retinal damage than those younger than 45.

Other risk factors were female sex (HR, 3.83; 95% CI, 1.86-7.89), chronic kidney disease stage III (HR, 1.95; 95% CI, 1.25-3.04), and tamoxifen use (HR, 3.43; 95% CI, 1.08-10.89), although only 17 patients were taking tamoxifen during the study.

Researchers also found that the type of HCQ retinopathy varied by race. Of the 125 cases in the cohort, 102 had a parafoveal pattern, and 23 had a pericentral pattern. Asian individuals were 15 times more likely, and Black individuals were more than 5 times more likely to develop this pericentral type than were White patients.

This association in Asian patients has also been found in previous studies, Dr. Weng said, and many eye practices now screen their Asian patients with a 30-2 Humphrey visual field — rather than the more commonly used 10-2 — to examine areas farther outside the center.

This study also found this association in Black patients, though only five Black patients developed HCQ retinopathy over the study period.

“More studies and larger studies will be very helpful in strengthening or dispelling some of the associations that have been seen here,” Dr. Weng said.
 

‘More Room for Personalized Medicine’

The team found a “relatively linear” relationship between HCQ dose and retinopathy risk, with higher daily doses correlating with higher incidence. While 2016 guidelines from the American Academy of Ophthalmology advise using < 5 mg/kg, “what we found is it’s not that straightforward [where there’s] just this one cutoff,” Dr. Jorge told this news organization. “It does seem like the higher the dose of medication per bodyweight and the longer duration of use, there is a higher risk of retinopathy.”

These findings leave “a bit more room for personalized medicine” with patients, she explained. “For an elderly female patient with CKD, aiming for a dose < 5 mg/kg might be more appropriate; however, a young male patient without any additional risk factors may be able to exceed 5 mg/kg and continue to have a low risk for HCQ retinopathy,” she said.

“For anyone, I think it really is more of an individual risk-benefit evaluation,” rather than strict cutoffs, she continued.

“Guidelines are just that: They’re guidelines,” added Dr. Weng, “and treatment plans should be tailored to each individual patient.”

As the study authors also discussed, “the goal is to treat the patient with the lowest dose that is still effective and also be mindful of the duration that a patient is left at higher doses,” Dr. Weng said. “But ultimately, we need to control these diseases, which can cause damage across multiple organ systems in the body. While it’s important to be aware of the potential retinopathy adverse events, we also don’t want physicians to feel restricted in their use of this very effective drug.”

The work of three coauthors on the current study was supported by a grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Dr. Jorge’s work on the study was supported by an award from the Rheumatology Research Foundation and a grant from NIAMS. Dr. Jorge reported clinical trial agreements with Bristol Myers Squibb and Cabaletta Bio outside of this study. Dr. Weng has served as a consultant for Allergan/AbbVie, Alcon, Apellis Pharmaceuticals, Alimera Sciences, DORC, Novartis, Genentech, Regeneron, RegenxBio, Iveric Bio, and EyePoint Pharmaceuticals. Dr. Weng disclosed financial relationships with Springer Publishers (royalties) and DRCR Retina Network, Alimera Sciences, and Applied Genetic Technologies Corporation (research).

A version of this article appeared on Medscape.com.

Older patients prescribed hydroxychloroquine (HCQ) have a higher risk of developing retinal damage from taking the medication, according to a new analysis.

In addition to known risk factors such as a higher weight-based HCQ dose and higher cumulative dose, researchers also found that female sex, chronic kidney disease stage III, and tamoxifen use were associated with HCQ retinopathy.

The findings provide “evidence for other key risk factors for hydroxychloroquine retinopathy beyond hydroxychloroquine exposure itself,” wrote April M. Jorge, MD, of the Division of Rheumatology, Allergy, and Immunology at Massachusetts General Hospital, Boston, and colleagues.

Baylor College of Medicine

Study Details

Researchers identified patients in the Kaiser Permanente Northern California (KPNC), Oakland, California, health system who began taking HCQ between July 1, 1997, and December 14, 2014. To be included, patients needed to have at least 5 years of continuous enrollment in the KPNC system and at least one prescription for HCQ after more than 5 years of starting the drug. Patients were followed from HCQ initiation to their last retinopathy screening study, up to December 31, 2020.

The study was published May 9 in JAMA Network Open.

Of the 4677 users followed for the study, 83% were women, and the average age starting HCQ was 52. Most patients were White (58.1%), while 13.7% were Asian, 10.5% were Black, and 17.7% were Hispanic.

More than 60% of patients had an initial dose > 5 mg/kg/d, though the mean initial dose of HCQ was 4.4 mg/kg/d. After 5 years, only 34.4% of patients were using a daily dose over 5 mg/kg.

Of the entire cohort, 125 patients (2.7%) developed HCQ retinopathy. As expected, cumulative HCQ exposure was associated with a higher retinopathy risk: For every 100 g of HCQ cumulative exposure, risk rose by 64% (hazard ratio [HR], 1.64; 95% CI, 1.44-1.87).

Age was a significant risk factor for retinal damage from HCQ use. Individuals who began taking the drug at 65 years or older were nearly six times more likely to develop retinopathy than those who started HCQ when they were younger than 45. In people aged 55-64 years, this risk was nearly four times higher, and individuals aged 45-54 years when starting the drug were 2.5 times more likely to have retinal damage than those younger than 45.

Other risk factors were female sex (HR, 3.83; 95% CI, 1.86-7.89), chronic kidney disease stage III (HR, 1.95; 95% CI, 1.25-3.04), and tamoxifen use (HR, 3.43; 95% CI, 1.08-10.89), although only 17 patients were taking tamoxifen during the study.

Researchers also found that the type of HCQ retinopathy varied by race. Of the 125 cases in the cohort, 102 had a parafoveal pattern, and 23 had a pericentral pattern. Asian individuals were 15 times more likely, and Black individuals were more than 5 times more likely to develop this pericentral type than were White patients.

This association in Asian patients has also been found in previous studies, Dr. Weng said, and many eye practices now screen their Asian patients with a 30-2 Humphrey visual field — rather than the more commonly used 10-2 — to examine areas farther outside the center.

This study also found this association in Black patients, though only five Black patients developed HCQ retinopathy over the study period.

“More studies and larger studies will be very helpful in strengthening or dispelling some of the associations that have been seen here,” Dr. Weng said.
 

‘More Room for Personalized Medicine’

The team found a “relatively linear” relationship between HCQ dose and retinopathy risk, with higher daily doses correlating with higher incidence. While 2016 guidelines from the American Academy of Ophthalmology advise using < 5 mg/kg, “what we found is it’s not that straightforward [where there’s] just this one cutoff,” Dr. Jorge told this news organization. “It does seem like the higher the dose of medication per bodyweight and the longer duration of use, there is a higher risk of retinopathy.”

These findings leave “a bit more room for personalized medicine” with patients, she explained. “For an elderly female patient with CKD, aiming for a dose < 5 mg/kg might be more appropriate; however, a young male patient without any additional risk factors may be able to exceed 5 mg/kg and continue to have a low risk for HCQ retinopathy,” she said.

“For anyone, I think it really is more of an individual risk-benefit evaluation,” rather than strict cutoffs, she continued.

“Guidelines are just that: They’re guidelines,” added Dr. Weng, “and treatment plans should be tailored to each individual patient.”

As the study authors also discussed, “the goal is to treat the patient with the lowest dose that is still effective and also be mindful of the duration that a patient is left at higher doses,” Dr. Weng said. “But ultimately, we need to control these diseases, which can cause damage across multiple organ systems in the body. While it’s important to be aware of the potential retinopathy adverse events, we also don’t want physicians to feel restricted in their use of this very effective drug.”

The work of three coauthors on the current study was supported by a grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Dr. Jorge’s work on the study was supported by an award from the Rheumatology Research Foundation and a grant from NIAMS. Dr. Jorge reported clinical trial agreements with Bristol Myers Squibb and Cabaletta Bio outside of this study. Dr. Weng has served as a consultant for Allergan/AbbVie, Alcon, Apellis Pharmaceuticals, Alimera Sciences, DORC, Novartis, Genentech, Regeneron, RegenxBio, Iveric Bio, and EyePoint Pharmaceuticals. Dr. Weng disclosed financial relationships with Springer Publishers (royalties) and DRCR Retina Network, Alimera Sciences, and Applied Genetic Technologies Corporation (research).

A version of this article appeared on Medscape.com.

Older patients prescribed hydroxychloroquine (HCQ) have a higher risk of developing retinal damage from taking the medication, according to a new analysis.

In addition to known risk factors such as a higher weight-based HCQ dose and higher cumulative dose, researchers also found that female sex, chronic kidney disease stage III, and tamoxifen use were associated with HCQ retinopathy.

The findings provide “evidence for other key risk factors for hydroxychloroquine retinopathy beyond hydroxychloroquine exposure itself,” wrote April M. Jorge, MD, of the Division of Rheumatology, Allergy, and Immunology at Massachusetts General Hospital, Boston, and colleagues.

Baylor College of Medicine

Study Details

Researchers identified patients in the Kaiser Permanente Northern California (KPNC), Oakland, California, health system who began taking HCQ between July 1, 1997, and December 14, 2014. To be included, patients needed to have at least 5 years of continuous enrollment in the KPNC system and at least one prescription for HCQ after more than 5 years of starting the drug. Patients were followed from HCQ initiation to their last retinopathy screening study, up to December 31, 2020.

The study was published May 9 in JAMA Network Open.

Of the 4677 users followed for the study, 83% were women, and the average age starting HCQ was 52. Most patients were White (58.1%), while 13.7% were Asian, 10.5% were Black, and 17.7% were Hispanic.

More than 60% of patients had an initial dose > 5 mg/kg/d, though the mean initial dose of HCQ was 4.4 mg/kg/d. After 5 years, only 34.4% of patients were using a daily dose over 5 mg/kg.

Of the entire cohort, 125 patients (2.7%) developed HCQ retinopathy. As expected, cumulative HCQ exposure was associated with a higher retinopathy risk: For every 100 g of HCQ cumulative exposure, risk rose by 64% (hazard ratio [HR], 1.64; 95% CI, 1.44-1.87).

Age was a significant risk factor for retinal damage from HCQ use. Individuals who began taking the drug at 65 years or older were nearly six times more likely to develop retinopathy than those who started HCQ when they were younger than 45. In people aged 55-64 years, this risk was nearly four times higher, and individuals aged 45-54 years when starting the drug were 2.5 times more likely to have retinal damage than those younger than 45.

Other risk factors were female sex (HR, 3.83; 95% CI, 1.86-7.89), chronic kidney disease stage III (HR, 1.95; 95% CI, 1.25-3.04), and tamoxifen use (HR, 3.43; 95% CI, 1.08-10.89), although only 17 patients were taking tamoxifen during the study.

Researchers also found that the type of HCQ retinopathy varied by race. Of the 125 cases in the cohort, 102 had a parafoveal pattern, and 23 had a pericentral pattern. Asian individuals were 15 times more likely, and Black individuals were more than 5 times more likely to develop this pericentral type than were White patients.

This association in Asian patients has also been found in previous studies, Dr. Weng said, and many eye practices now screen their Asian patients with a 30-2 Humphrey visual field — rather than the more commonly used 10-2 — to examine areas farther outside the center.

This study also found this association in Black patients, though only five Black patients developed HCQ retinopathy over the study period.

“More studies and larger studies will be very helpful in strengthening or dispelling some of the associations that have been seen here,” Dr. Weng said.
 

‘More Room for Personalized Medicine’

The team found a “relatively linear” relationship between HCQ dose and retinopathy risk, with higher daily doses correlating with higher incidence. While 2016 guidelines from the American Academy of Ophthalmology advise using < 5 mg/kg, “what we found is it’s not that straightforward [where there’s] just this one cutoff,” Dr. Jorge told this news organization. “It does seem like the higher the dose of medication per bodyweight and the longer duration of use, there is a higher risk of retinopathy.”

These findings leave “a bit more room for personalized medicine” with patients, she explained. “For an elderly female patient with CKD, aiming for a dose < 5 mg/kg might be more appropriate; however, a young male patient without any additional risk factors may be able to exceed 5 mg/kg and continue to have a low risk for HCQ retinopathy,” she said.

“For anyone, I think it really is more of an individual risk-benefit evaluation,” rather than strict cutoffs, she continued.

“Guidelines are just that: They’re guidelines,” added Dr. Weng, “and treatment plans should be tailored to each individual patient.”

As the study authors also discussed, “the goal is to treat the patient with the lowest dose that is still effective and also be mindful of the duration that a patient is left at higher doses,” Dr. Weng said. “But ultimately, we need to control these diseases, which can cause damage across multiple organ systems in the body. While it’s important to be aware of the potential retinopathy adverse events, we also don’t want physicians to feel restricted in their use of this very effective drug.”

The work of three coauthors on the current study was supported by a grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Dr. Jorge’s work on the study was supported by an award from the Rheumatology Research Foundation and a grant from NIAMS. Dr. Jorge reported clinical trial agreements with Bristol Myers Squibb and Cabaletta Bio outside of this study. Dr. Weng has served as a consultant for Allergan/AbbVie, Alcon, Apellis Pharmaceuticals, Alimera Sciences, DORC, Novartis, Genentech, Regeneron, RegenxBio, Iveric Bio, and EyePoint Pharmaceuticals. Dr. Weng disclosed financial relationships with Springer Publishers (royalties) and DRCR Retina Network, Alimera Sciences, and Applied Genetic Technologies Corporation (research).

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved Benlysta (belimumab) autoinjector for patients aged 5 years or older with active systemic lupus erythematosus (SLE) on standard therapy. This is the first time that children with SLE can receive this treatment at home, according to a GSK press release.

Prior to this approval, pediatric patients aged 5 years or older could receive belimumab only intravenously via a 1-hour infusion in a hospital or clinic setting.

Wikimedia Commons/FitzColinGerald/Creative Commons License

“Going to the doctor’s office once every 4 weeks can be a logistical hurdle for some children and their caregivers, so having the option to administer Benlysta in the comfort of their home provides much-needed flexibility,” Mary Crimmings, the interim CEO and senior vice president for marketing and communications at the Lupus Foundation of America, said in a statement. 

An estimated 5000-10,000 children in the United States are living with SLE.

Belimumab is a B-lymphocyte stimulator–specific inhibitor approved for the treatment of active SLE and active lupus nephritis in patients aged 5 years or older receiving standard therapy. This approval of the subcutaneous administration of belimumab applies only to pediatric patients with SLE.

The 200-mg injection can be administered once every week for children who weigh ≥ 40 kg and should be given once every 2 weeks for children weighing between 15 and 40 kg. 

The autoinjector “will be available immediately” for caregivers, the company announcement said.

“Patients are our top priority, and we are always working to innovate solutions that can improve lives and address unmet needs,” Court Horncastle, senior vice president and head of US specialty at GSK, said in the press release. “This approval for an at-home treatment is the first and only of its kind for children with lupus and is a testament to our continued commitment to the lupus community.”

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved Benlysta (belimumab) autoinjector for patients aged 5 years or older with active systemic lupus erythematosus (SLE) on standard therapy. This is the first time that children with SLE can receive this treatment at home, according to a GSK press release.

Prior to this approval, pediatric patients aged 5 years or older could receive belimumab only intravenously via a 1-hour infusion in a hospital or clinic setting.

Wikimedia Commons/FitzColinGerald/Creative Commons License

“Going to the doctor’s office once every 4 weeks can be a logistical hurdle for some children and their caregivers, so having the option to administer Benlysta in the comfort of their home provides much-needed flexibility,” Mary Crimmings, the interim CEO and senior vice president for marketing and communications at the Lupus Foundation of America, said in a statement. 

An estimated 5000-10,000 children in the United States are living with SLE.

Belimumab is a B-lymphocyte stimulator–specific inhibitor approved for the treatment of active SLE and active lupus nephritis in patients aged 5 years or older receiving standard therapy. This approval of the subcutaneous administration of belimumab applies only to pediatric patients with SLE.

The 200-mg injection can be administered once every week for children who weigh ≥ 40 kg and should be given once every 2 weeks for children weighing between 15 and 40 kg. 

The autoinjector “will be available immediately” for caregivers, the company announcement said.

“Patients are our top priority, and we are always working to innovate solutions that can improve lives and address unmet needs,” Court Horncastle, senior vice president and head of US specialty at GSK, said in the press release. “This approval for an at-home treatment is the first and only of its kind for children with lupus and is a testament to our continued commitment to the lupus community.”

A version of this article appeared on Medscape.com.

The US Food and Drug Administration (FDA) has approved Benlysta (belimumab) autoinjector for patients aged 5 years or older with active systemic lupus erythematosus (SLE) on standard therapy. This is the first time that children with SLE can receive this treatment at home, according to a GSK press release.

Prior to this approval, pediatric patients aged 5 years or older could receive belimumab only intravenously via a 1-hour infusion in a hospital or clinic setting.

Wikimedia Commons/FitzColinGerald/Creative Commons License

“Going to the doctor’s office once every 4 weeks can be a logistical hurdle for some children and their caregivers, so having the option to administer Benlysta in the comfort of their home provides much-needed flexibility,” Mary Crimmings, the interim CEO and senior vice president for marketing and communications at the Lupus Foundation of America, said in a statement. 

An estimated 5000-10,000 children in the United States are living with SLE.

Belimumab is a B-lymphocyte stimulator–specific inhibitor approved for the treatment of active SLE and active lupus nephritis in patients aged 5 years or older receiving standard therapy. This approval of the subcutaneous administration of belimumab applies only to pediatric patients with SLE.

The 200-mg injection can be administered once every week for children who weigh ≥ 40 kg and should be given once every 2 weeks for children weighing between 15 and 40 kg. 

The autoinjector “will be available immediately” for caregivers, the company announcement said.

“Patients are our top priority, and we are always working to innovate solutions that can improve lives and address unmet needs,” Court Horncastle, senior vice president and head of US specialty at GSK, said in the press release. “This approval for an at-home treatment is the first and only of its kind for children with lupus and is a testament to our continued commitment to the lupus community.”

A version of this article appeared on Medscape.com.

TOPLINE:

A commercially available test for 40 genetic variants that identifies people with the “hungry gut” obesity phenotype can predict who will respond most to semaglutide for weight management, new evidence reveals.

METHODOLOGY:

• A machine learning genetic risk score can identify people with the hungry gut obesity phenotype, which has been found to be associated with greater weight loss with the glucagon-like peptide 1 receptor agonists (GLP-1 RA) liraglutide and exenatide. 
• For this study, researchers calculated the genetic risk score for 84 adults undergoing weight loss interventions at Mayo Clinic who were prescribed the GLP-1 RA semaglutide.
• Study participants were classified as the obesity phenotype hungry gut positive (n = 51) or hungry gut negative (n = 33).
• The researchers measured total body weight loss at 3, 6, 9, and 12 months and assessed the ability of the score to predict the response to semaglutide (defined as ≥ 5% of total body weight loss measured at 12 months).

TAKEAWAY:

• At 3 and 6 months, there were no significant differences in weight loss between the hungry gut positive and hungry gut negative groups.
• By 9 months, participants in the positive group lost 14.4% of their total body weight compared with 10.3% in case of participants in the negative group (P = .045).
• After a total of 12 months, the positive group lost 19.5% of their total body weight compared with 10.0% in case of participants in the negative group (P = .01).
• When used to predict the response to semaglutide, the area under the curve for the machine-learning genetic risk score was 0.76 (95% CI, 0.57-0.94; P = .04).

IN PRACTICE:

We can now tell with confidence who is going to respond to semaglutide, said Andres Acosta, MD, PhD, associate professor of medicine at Mayo Clinic. “For nonresponders, we can think about other interventions or medications that we have available.”

SOURCE:

This study was presented on May 20, 2024, at the annual Digestive Disease Week® (DDW) (Abstract 638).

LIMITATIONS:

Further prospective studies are needed to assess the validity of the test in a more diverse population and with different weight loss interventions.

DISCLOSURES:

This study was supported by a partnership between Mayo Clinic and Phenomix Sciences. Dr. Acosta is a cofounder of Phenomix Sciences.

A version of this article appeared on Medscape.com.

TOPLINE:

A commercially available test for 40 genetic variants that identifies people with the “hungry gut” obesity phenotype can predict who will respond most to semaglutide for weight management, new evidence reveals.

METHODOLOGY:

• A machine learning genetic risk score can identify people with the hungry gut obesity phenotype, which has been found to be associated with greater weight loss with the glucagon-like peptide 1 receptor agonists (GLP-1 RA) liraglutide and exenatide. 
• For this study, researchers calculated the genetic risk score for 84 adults undergoing weight loss interventions at Mayo Clinic who were prescribed the GLP-1 RA semaglutide.
• Study participants were classified as the obesity phenotype hungry gut positive (n = 51) or hungry gut negative (n = 33).
• The researchers measured total body weight loss at 3, 6, 9, and 12 months and assessed the ability of the score to predict the response to semaglutide (defined as ≥ 5% of total body weight loss measured at 12 months).

TAKEAWAY:

• At 3 and 6 months, there were no significant differences in weight loss between the hungry gut positive and hungry gut negative groups.
• By 9 months, participants in the positive group lost 14.4% of their total body weight compared with 10.3% in case of participants in the negative group (P = .045).
• After a total of 12 months, the positive group lost 19.5% of their total body weight compared with 10.0% in case of participants in the negative group (P = .01).
• When used to predict the response to semaglutide, the area under the curve for the machine-learning genetic risk score was 0.76 (95% CI, 0.57-0.94; P = .04).

IN PRACTICE:

We can now tell with confidence who is going to respond to semaglutide, said Andres Acosta, MD, PhD, associate professor of medicine at Mayo Clinic. “For nonresponders, we can think about other interventions or medications that we have available.”

SOURCE:

This study was presented on May 20, 2024, at the annual Digestive Disease Week® (DDW) (Abstract 638).

LIMITATIONS:

Further prospective studies are needed to assess the validity of the test in a more diverse population and with different weight loss interventions.

DISCLOSURES:

This study was supported by a partnership between Mayo Clinic and Phenomix Sciences. Dr. Acosta is a cofounder of Phenomix Sciences.

A version of this article appeared on Medscape.com.

TOPLINE:

A commercially available test for 40 genetic variants that identifies people with the “hungry gut” obesity phenotype can predict who will respond most to semaglutide for weight management, new evidence reveals.

METHODOLOGY:

• A machine learning genetic risk score can identify people with the hungry gut obesity phenotype, which has been found to be associated with greater weight loss with the glucagon-like peptide 1 receptor agonists (GLP-1 RA) liraglutide and exenatide. 
• For this study, researchers calculated the genetic risk score for 84 adults undergoing weight loss interventions at Mayo Clinic who were prescribed the GLP-1 RA semaglutide.
• Study participants were classified as the obesity phenotype hungry gut positive (n = 51) or hungry gut negative (n = 33).
• The researchers measured total body weight loss at 3, 6, 9, and 12 months and assessed the ability of the score to predict the response to semaglutide (defined as ≥ 5% of total body weight loss measured at 12 months).

TAKEAWAY:

• At 3 and 6 months, there were no significant differences in weight loss between the hungry gut positive and hungry gut negative groups.
• By 9 months, participants in the positive group lost 14.4% of their total body weight compared with 10.3% in case of participants in the negative group (P = .045).
• After a total of 12 months, the positive group lost 19.5% of their total body weight compared with 10.0% in case of participants in the negative group (P = .01).
• When used to predict the response to semaglutide, the area under the curve for the machine-learning genetic risk score was 0.76 (95% CI, 0.57-0.94; P = .04).

IN PRACTICE:

We can now tell with confidence who is going to respond to semaglutide, said Andres Acosta, MD, PhD, associate professor of medicine at Mayo Clinic. “For nonresponders, we can think about other interventions or medications that we have available.”

SOURCE:

This study was presented on May 20, 2024, at the annual Digestive Disease Week® (DDW) (Abstract 638).

LIMITATIONS:

Further prospective studies are needed to assess the validity of the test in a more diverse population and with different weight loss interventions.

DISCLOSURES:

This study was supported by a partnership between Mayo Clinic and Phenomix Sciences. Dr. Acosta is a cofounder of Phenomix Sciences.

A version of this article appeared on Medscape.com.

Five experts discussed research that they considered to be highlights of the European Society of Medical Oncology (ESMO) Breast Cancer annual congress during a “Key Takeaways” session at the meeting.

Among the topics the speakers addressed were breast cancer prevention, early breast cancer, advanced breast cancer, and supportive care.

In recent years, the way clinicians look at carcinogenesis in breast cancer has changed, and many new targets for potential early detection and prevention have emerged, said Suzette Delaloge, MD, of Gustave Roussy, Paris, France, in her presentation at the meeting.

Instant risk assessment at different time points could potentially intercept cancer among high-risk individuals, she said.

A study by Mikael Eriksson, PhD, and colleagues focused on external validation of the Profound AI tool to identify breast cancer risk in the general population. The researchers showed an area under the curve of 0.72 in their AI risk model, which has the potential to be clinically meaningful, although it must be prospectively validated, Dr. Delaloge said in her presentation.

She also reviewed two studies on the use of genes to further refine breast cancer risk among carriers. One of these, a prospective study presented in a session by Kelly-Anne Phillips, MD, of Peter MacCallum Cancer Center, Melbourne, Australia, used the CANRISK online risk assessment tool and validated increased breast cancer risk in BRCA1 and BRCA2 carriers, with AUCs of 0.79 and 0.78, respectively. The other study, which was by Maria Rezqallah Aron, MD, and colleagues examined polygenic scores as a way to refine breast cancer risk stratification among carriers of the ALM and PALB2 genes as well. These genes might be useful in identifying individuals who could benefit from early intervention, including surgery, Dr. Delaloge said.
 

Translational Research

“Preparing my talk, I felt like a kid in a candy store,” because of the amount of new translational research presented, including several studies of endocrine treatment–based approaches to therapy, said Marleen Kok, MD, of the Netherlands Cancer Institute, Amsterdam.

In her presentation, Dr. Kok highlighted findings from an analysis of patients in the monarchE study (a trial of high-risk patients) showing a consistent improvement in invasive disease-free survival for the subset of patients with germline BRCA1 and BRCA2 mutations who received abemaciclib plus endocrine therapy.

The value of tumor-infiltrating lymphocytes (TILs) on patients who are not receiving chemotherapy is important because of the focus on prognosis, and prospective trials are underway, she said.

A poster on the impact of chemotherapy and stromal tumor-infiltrating lymphocytes (sTILs) in stage I triple-negative breast cancer showed no association between chemotherapy and better outcomes regardless of sTILs in patients who did and did not receive chemotherapy, which has implications for potential treatment sparing in this population, Dr. Kok noted.

Artificial Intelligence (AI) was the subject of several posters at the meeting, and Dr. Kok identified a multisite European study of an automated HER2 scoring system as notable for its size and accuracy. In the study, the accuracy among pathologists was much higher with the assistance of AI, she said. Using AI for more complex analysis has shown success, she said.

Dr. Kok ended her talk with a poster that surveyed breast cancer patients about their understanding of their disease. The results showed that less than half (44%) of patients reported that their healthcare providers had given them enough information to learn about their breast cancer type, and less than one third could recall terminology about biomarkers; the study is important because it shows that clinicians need to do better in explaining these terms to patients, Dr. Kok said.
 

Early Breast Cancer

Right-sizing therapy, meaning identifying the right treatment for every patient, is a key element of new research in early breast cancer, said Erika Hamilton, MD, of the Sarah Cannon Research Institute, Nashville, Tenn.

She highlighted safety and treatment duration updates from the NATALEE study, which compared adjuvant ribociclib plus nonsteroidal aromatase inhibitor (NSAI) to NSAI alone for ER+/HER2- breast cancer. The current analysis presented at the meeting showed significant benefits with the addition of ribociclib and no evidence of new safety signals or adverse event exacerbations at 3 years, she said. Dose modifications had no significant impact on efficacy, she added.

The findings of no impact of dose reduction on efficacy in both the NATALEE and monarchE studies provide important information on whether dosage can be reduced in patients, which will increase the odds that patients will tolerate extended therapy with good outcomes and stay on their prescribed therapies, Dr. Hamilton emphasized.

The CARABELA study, a phase 2 trial of neoadjuvant letrozole plus abemaciclib vs adriamycin and cyclophosphamide (AC), showed clinically similar response rates but did not meet its endpoint for residual cancer burden (RCB) scores. These data add to results from other studies and show that it is too soon to universally replace neoadjuvant chemotherapy as first-line treatment for highly proliferative ER+ breast cancer, Dr. Hamilton said in her presentation.
 

Advanced Breast Cancer

Take-home messages about advanced breast cancer include growing evidence for the potential benefits of antibody drug conjugates (ADCs), said Eva Ciruelos, MD, of University Hospital, Madrid, Spain. The TROPION-BREAST01 study, a phase 3 randomized trial, showed significant and clinically meaningful improvement in progression-free survival in patients with previously treated, inoperable, or metastatic HR+/HER2- breast cancer who received datopotamab deruxtecan (Dato-DXd) compared with those who received chemotherapy.

Data from an additional safety analysis were presented at the meeting; although Dato-DXd, a trophoblast cell-surface antigen 2 (TROP2)–directed antibody-drug conjugate, was well-tolerated, it is important to remain aware of toxicities, notably oral mucositis, which occurred in 55.6% of the patients in the study across all grades, and ocular surface toxicity, which occurred in 40% of patients across all grades, Dr. Ciruelos emphasized.

Key research in the area of advanced triple-negative breast cancer included data from the IMPASSION 132 study. This study is “specifically centered on early relapsers,” a population often excluded from other trials, Dr. Ciruelos said. In this study, patients with advanced triple-negative breast cancer were randomized to chemotherapy with or without atezolizumab, and the study showed no benefits with atezolizumab for overall survival, progression-free survival, or overall response rate, she said. “This is something to work with, because this is a very refractory population,” Dr. Ciruelos noted.

New immunotherapy combinations are needed to improve survival in advanced breast cancer patients, Dr. Ciruelos said. At the meeting, researchers presented interim data from a subset of patients in the MORPHEUS-pan breast cancer trial, a phase 1B/2 study involving multiple treatment combinations in locally advanced/metastatic breast cancer patients.

The interim analysis included 18-week data from triple-negative breast cancer patients and compared outcomes for patients randomized to atezolizumab with or without sacituzumab govitecan (SG).

The study was small, with only 31 patients in the combination arm and 11 controls, but the results were promising, with an overall response rate of 76.7% in the combination arm vs 66.7% in the control arm, Dr. Ciruelos said.

Supportive Care

Key supportive care takeaways included data on pregnancy in young breast cancer survivors and the safety of vaginal estrogen therapy in breast cancer patients with genitourinary symptoms, said Anne May, MD, of the University Medical Center Utrecht, Utrecht, Netherlands.

A study previously published in JAMA including nearly 5000 BRCA carriers who were diagnosed with invasive breast cancer at age 40 years or younger showed no association between pregnancy after breast cancer and adverse maternal or fetal outcomes, and pregnancy had no significant impact on overall survival. The authors presented new data on the safety of assisted reproductive techniques (ART) based on the 543 pregnancies in the original study, at the meeting. Of these, 436 conceived naturally, and 107 used ART. After a median of 9.1 years, ART had no effect on disease-free survival compared to natural conception (hazard ratio [HR], 0.64). Based on these findings, fertility preservation should be offered to all women who receive a breast cancer diagnosis and are interested in future fertility, Dr. May said.

Conceiving after breast cancer treatment and follow-up should not be contraindicated for young BRCA carriers, she added.No trial data are available for the effects of vaginal estrogen therapy (VET) on disease-free survival in breast cancer survivors with genitourinary symptoms caused by declining estrogen levels, Dr. May said. However, researchers in France and Switzerland conducted an emulation of a hypothetical target trial using data from the French National social security system for more than 130,000 individuals. Although VET therapy had no impact on disease-free survival in most breast cancer survivors overall, it did have a negative impact in a subset of patients with HR-positive and HR-negative tumors who were treated with aromatase inhibitors. The study was hypothetical, but important because the results suggest that clinicians can safely propose VTE to patients who report genitourinary symptoms after treatment for early-stage breast cancer with tamoxifen, but VTE should be avoided in patients treated with aromatase inhibitors, Dr. May said.

Dr. Delaloge disclosed research support to her institution from AstraZeneca, MSD, Bristol Myers Squibb, Sanofi, Taiho, Novartis, European Commission, INCa, Banque des Territoires, and Fondation Philanthropia. She also disclosed honoraria to her institution from AstraZeneca, Gilead, Novartis, Elsan, Besins, Sanofi, Exact Sciences, and Lilly, as well as travel support from Novartis.

Dr. Kok disclosed research funding from AstraZeneca, Bristol Myers Squibb, Daichi, and Roche, and advisory board membership/speaker’s fees from Alderaan Biotechnology, BIONTECH, Domain Therapeutics, AstraZeneca, Daichi, Bristol Myers Squibb, Gilead, Medscape, MSD, and Roche.

Dr. Hamilton disclosed a consulting advisory role (to her institution) for Accutar Biotechology, AstraZeneca, Daiichi Sankyo, Ellipses Pharma, Entos, Forsum Pharma, Gilead Sciences, Greenwich LifeSciences, Jazz Pharmaceuticals, Lilly, Medical Pharma Services, Mersana, Novartis, Olema Pharmaceuticals, Orum Therapeutics, Roche/Genentech, Stemline Therapeutics, ands others. She also disclosed contracted research/grant support to her institution only from Abbvie, Acerta Pharma, Accutar Biotechnology , ADC Therapeutics, AKESOBIO Australia , Amgen, Aravive, ArQule, Artios, Arvinas, AstraZeneca, AtlasMedx, BeiGene, Black Diamond and others.

Dr. Ciruelos disclosed serving as an external advisor for Roche, MSD, Gilead, AstraZeneca, Daichii Sankyo, Reveal Genomics, Pfizer, Novartis, and Lilly, as well as serving as a speaker for Roche, MSD, Gilead, AstraZeneca, Daichii Sankyo, Reveal Genomics, Pfizer, Novartis, Lilly, and Pierre Fabre. She also disclosed travel grants from Roche, Pfizer, and AstraZeneca, and research grants from Seagen and Roche.

Dr. May had no financial conflicts to disclose.

Five experts discussed research that they considered to be highlights of the European Society of Medical Oncology (ESMO) Breast Cancer annual congress during a “Key Takeaways” session at the meeting.

Among the topics the speakers addressed were breast cancer prevention, early breast cancer, advanced breast cancer, and supportive care.

In recent years, the way clinicians look at carcinogenesis in breast cancer has changed, and many new targets for potential early detection and prevention have emerged, said Suzette Delaloge, MD, of Gustave Roussy, Paris, France, in her presentation at the meeting.

Instant risk assessment at different time points could potentially intercept cancer among high-risk individuals, she said.

A study by Mikael Eriksson, PhD, and colleagues focused on external validation of the Profound AI tool to identify breast cancer risk in the general population. The researchers showed an area under the curve of 0.72 in their AI risk model, which has the potential to be clinically meaningful, although it must be prospectively validated, Dr. Delaloge said in her presentation.

She also reviewed two studies on the use of genes to further refine breast cancer risk among carriers. One of these, a prospective study presented in a session by Kelly-Anne Phillips, MD, of Peter MacCallum Cancer Center, Melbourne, Australia, used the CANRISK online risk assessment tool and validated increased breast cancer risk in BRCA1 and BRCA2 carriers, with AUCs of 0.79 and 0.78, respectively. The other study, which was by Maria Rezqallah Aron, MD, and colleagues examined polygenic scores as a way to refine breast cancer risk stratification among carriers of the ALM and PALB2 genes as well. These genes might be useful in identifying individuals who could benefit from early intervention, including surgery, Dr. Delaloge said.
 

Translational Research

“Preparing my talk, I felt like a kid in a candy store,” because of the amount of new translational research presented, including several studies of endocrine treatment–based approaches to therapy, said Marleen Kok, MD, of the Netherlands Cancer Institute, Amsterdam.

In her presentation, Dr. Kok highlighted findings from an analysis of patients in the monarchE study (a trial of high-risk patients) showing a consistent improvement in invasive disease-free survival for the subset of patients with germline BRCA1 and BRCA2 mutations who received abemaciclib plus endocrine therapy.

The value of tumor-infiltrating lymphocytes (TILs) on patients who are not receiving chemotherapy is important because of the focus on prognosis, and prospective trials are underway, she said.

A poster on the impact of chemotherapy and stromal tumor-infiltrating lymphocytes (sTILs) in stage I triple-negative breast cancer showed no association between chemotherapy and better outcomes regardless of sTILs in patients who did and did not receive chemotherapy, which has implications for potential treatment sparing in this population, Dr. Kok noted.

Artificial Intelligence (AI) was the subject of several posters at the meeting, and Dr. Kok identified a multisite European study of an automated HER2 scoring system as notable for its size and accuracy. In the study, the accuracy among pathologists was much higher with the assistance of AI, she said. Using AI for more complex analysis has shown success, she said.

Dr. Kok ended her talk with a poster that surveyed breast cancer patients about their understanding of their disease. The results showed that less than half (44%) of patients reported that their healthcare providers had given them enough information to learn about their breast cancer type, and less than one third could recall terminology about biomarkers; the study is important because it shows that clinicians need to do better in explaining these terms to patients, Dr. Kok said.
 

Early Breast Cancer

Right-sizing therapy, meaning identifying the right treatment for every patient, is a key element of new research in early breast cancer, said Erika Hamilton, MD, of the Sarah Cannon Research Institute, Nashville, Tenn.

She highlighted safety and treatment duration updates from the NATALEE study, which compared adjuvant ribociclib plus nonsteroidal aromatase inhibitor (NSAI) to NSAI alone for ER+/HER2- breast cancer. The current analysis presented at the meeting showed significant benefits with the addition of ribociclib and no evidence of new safety signals or adverse event exacerbations at 3 years, she said. Dose modifications had no significant impact on efficacy, she added.

The findings of no impact of dose reduction on efficacy in both the NATALEE and monarchE studies provide important information on whether dosage can be reduced in patients, which will increase the odds that patients will tolerate extended therapy with good outcomes and stay on their prescribed therapies, Dr. Hamilton emphasized.

The CARABELA study, a phase 2 trial of neoadjuvant letrozole plus abemaciclib vs adriamycin and cyclophosphamide (AC), showed clinically similar response rates but did not meet its endpoint for residual cancer burden (RCB) scores. These data add to results from other studies and show that it is too soon to universally replace neoadjuvant chemotherapy as first-line treatment for highly proliferative ER+ breast cancer, Dr. Hamilton said in her presentation.
 

Advanced Breast Cancer

Take-home messages about advanced breast cancer include growing evidence for the potential benefits of antibody drug conjugates (ADCs), said Eva Ciruelos, MD, of University Hospital, Madrid, Spain. The TROPION-BREAST01 study, a phase 3 randomized trial, showed significant and clinically meaningful improvement in progression-free survival in patients with previously treated, inoperable, or metastatic HR+/HER2- breast cancer who received datopotamab deruxtecan (Dato-DXd) compared with those who received chemotherapy.

Data from an additional safety analysis were presented at the meeting; although Dato-DXd, a trophoblast cell-surface antigen 2 (TROP2)–directed antibody-drug conjugate, was well-tolerated, it is important to remain aware of toxicities, notably oral mucositis, which occurred in 55.6% of the patients in the study across all grades, and ocular surface toxicity, which occurred in 40% of patients across all grades, Dr. Ciruelos emphasized.

Key research in the area of advanced triple-negative breast cancer included data from the IMPASSION 132 study. This study is “specifically centered on early relapsers,” a population often excluded from other trials, Dr. Ciruelos said. In this study, patients with advanced triple-negative breast cancer were randomized to chemotherapy with or without atezolizumab, and the study showed no benefits with atezolizumab for overall survival, progression-free survival, or overall response rate, she said. “This is something to work with, because this is a very refractory population,” Dr. Ciruelos noted.

New immunotherapy combinations are needed to improve survival in advanced breast cancer patients, Dr. Ciruelos said. At the meeting, researchers presented interim data from a subset of patients in the MORPHEUS-pan breast cancer trial, a phase 1B/2 study involving multiple treatment combinations in locally advanced/metastatic breast cancer patients.

The interim analysis included 18-week data from triple-negative breast cancer patients and compared outcomes for patients randomized to atezolizumab with or without sacituzumab govitecan (SG).

The study was small, with only 31 patients in the combination arm and 11 controls, but the results were promising, with an overall response rate of 76.7% in the combination arm vs 66.7% in the control arm, Dr. Ciruelos said.

Supportive Care

Key supportive care takeaways included data on pregnancy in young breast cancer survivors and the safety of vaginal estrogen therapy in breast cancer patients with genitourinary symptoms, said Anne May, MD, of the University Medical Center Utrecht, Utrecht, Netherlands.

A study previously published in JAMA including nearly 5000 BRCA carriers who were diagnosed with invasive breast cancer at age 40 years or younger showed no association between pregnancy after breast cancer and adverse maternal or fetal outcomes, and pregnancy had no significant impact on overall survival. The authors presented new data on the safety of assisted reproductive techniques (ART) based on the 543 pregnancies in the original study, at the meeting. Of these, 436 conceived naturally, and 107 used ART. After a median of 9.1 years, ART had no effect on disease-free survival compared to natural conception (hazard ratio [HR], 0.64). Based on these findings, fertility preservation should be offered to all women who receive a breast cancer diagnosis and are interested in future fertility, Dr. May said.

Conceiving after breast cancer treatment and follow-up should not be contraindicated for young BRCA carriers, she added.No trial data are available for the effects of vaginal estrogen therapy (VET) on disease-free survival in breast cancer survivors with genitourinary symptoms caused by declining estrogen levels, Dr. May said. However, researchers in France and Switzerland conducted an emulation of a hypothetical target trial using data from the French National social security system for more than 130,000 individuals. Although VET therapy had no impact on disease-free survival in most breast cancer survivors overall, it did have a negative impact in a subset of patients with HR-positive and HR-negative tumors who were treated with aromatase inhibitors. The study was hypothetical, but important because the results suggest that clinicians can safely propose VTE to patients who report genitourinary symptoms after treatment for early-stage breast cancer with tamoxifen, but VTE should be avoided in patients treated with aromatase inhibitors, Dr. May said.

Dr. Delaloge disclosed research support to her institution from AstraZeneca, MSD, Bristol Myers Squibb, Sanofi, Taiho, Novartis, European Commission, INCa, Banque des Territoires, and Fondation Philanthropia. She also disclosed honoraria to her institution from AstraZeneca, Gilead, Novartis, Elsan, Besins, Sanofi, Exact Sciences, and Lilly, as well as travel support from Novartis.

Dr. Kok disclosed research funding from AstraZeneca, Bristol Myers Squibb, Daichi, and Roche, and advisory board membership/speaker’s fees from Alderaan Biotechnology, BIONTECH, Domain Therapeutics, AstraZeneca, Daichi, Bristol Myers Squibb, Gilead, Medscape, MSD, and Roche.

Dr. Hamilton disclosed a consulting advisory role (to her institution) for Accutar Biotechology, AstraZeneca, Daiichi Sankyo, Ellipses Pharma, Entos, Forsum Pharma, Gilead Sciences, Greenwich LifeSciences, Jazz Pharmaceuticals, Lilly, Medical Pharma Services, Mersana, Novartis, Olema Pharmaceuticals, Orum Therapeutics, Roche/Genentech, Stemline Therapeutics, ands others. She also disclosed contracted research/grant support to her institution only from Abbvie, Acerta Pharma, Accutar Biotechnology , ADC Therapeutics, AKESOBIO Australia , Amgen, Aravive, ArQule, Artios, Arvinas, AstraZeneca, AtlasMedx, BeiGene, Black Diamond and others.

Dr. Ciruelos disclosed serving as an external advisor for Roche, MSD, Gilead, AstraZeneca, Daichii Sankyo, Reveal Genomics, Pfizer, Novartis, and Lilly, as well as serving as a speaker for Roche, MSD, Gilead, AstraZeneca, Daichii Sankyo, Reveal Genomics, Pfizer, Novartis, Lilly, and Pierre Fabre. She also disclosed travel grants from Roche, Pfizer, and AstraZeneca, and research grants from Seagen and Roche.

Dr. May had no financial conflicts to disclose.

Five experts discussed research that they considered to be highlights of the European Society of Medical Oncology (ESMO) Breast Cancer annual congress during a “Key Takeaways” session at the meeting.

Among the topics the speakers addressed were breast cancer prevention, early breast cancer, advanced breast cancer, and supportive care.

In recent years, the way clinicians look at carcinogenesis in breast cancer has changed, and many new targets for potential early detection and prevention have emerged, said Suzette Delaloge, MD, of Gustave Roussy, Paris, France, in her presentation at the meeting.

Instant risk assessment at different time points could potentially intercept cancer among high-risk individuals, she said.

A study by Mikael Eriksson, PhD, and colleagues focused on external validation of the Profound AI tool to identify breast cancer risk in the general population. The researchers showed an area under the curve of 0.72 in their AI risk model, which has the potential to be clinically meaningful, although it must be prospectively validated, Dr. Delaloge said in her presentation.

She also reviewed two studies on the use of genes to further refine breast cancer risk among carriers. One of these, a prospective study presented in a session by Kelly-Anne Phillips, MD, of Peter MacCallum Cancer Center, Melbourne, Australia, used the CANRISK online risk assessment tool and validated increased breast cancer risk in BRCA1 and BRCA2 carriers, with AUCs of 0.79 and 0.78, respectively. The other study, which was by Maria Rezqallah Aron, MD, and colleagues examined polygenic scores as a way to refine breast cancer risk stratification among carriers of the ALM and PALB2 genes as well. These genes might be useful in identifying individuals who could benefit from early intervention, including surgery, Dr. Delaloge said.
 

Translational Research

“Preparing my talk, I felt like a kid in a candy store,” because of the amount of new translational research presented, including several studies of endocrine treatment–based approaches to therapy, said Marleen Kok, MD, of the Netherlands Cancer Institute, Amsterdam.

In her presentation, Dr. Kok highlighted findings from an analysis of patients in the monarchE study (a trial of high-risk patients) showing a consistent improvement in invasive disease-free survival for the subset of patients with germline BRCA1 and BRCA2 mutations who received abemaciclib plus endocrine therapy.

The value of tumor-infiltrating lymphocytes (TILs) on patients who are not receiving chemotherapy is important because of the focus on prognosis, and prospective trials are underway, she said.

A poster on the impact of chemotherapy and stromal tumor-infiltrating lymphocytes (sTILs) in stage I triple-negative breast cancer showed no association between chemotherapy and better outcomes regardless of sTILs in patients who did and did not receive chemotherapy, which has implications for potential treatment sparing in this population, Dr. Kok noted.

Artificial Intelligence (AI) was the subject of several posters at the meeting, and Dr. Kok identified a multisite European study of an automated HER2 scoring system as notable for its size and accuracy. In the study, the accuracy among pathologists was much higher with the assistance of AI, she said. Using AI for more complex analysis has shown success, she said.

Dr. Kok ended her talk with a poster that surveyed breast cancer patients about their understanding of their disease. The results showed that less than half (44%) of patients reported that their healthcare providers had given them enough information to learn about their breast cancer type, and less than one third could recall terminology about biomarkers; the study is important because it shows that clinicians need to do better in explaining these terms to patients, Dr. Kok said.
 

Early Breast Cancer

Right-sizing therapy, meaning identifying the right treatment for every patient, is a key element of new research in early breast cancer, said Erika Hamilton, MD, of the Sarah Cannon Research Institute, Nashville, Tenn.

She highlighted safety and treatment duration updates from the NATALEE study, which compared adjuvant ribociclib plus nonsteroidal aromatase inhibitor (NSAI) to NSAI alone for ER+/HER2- breast cancer. The current analysis presented at the meeting showed significant benefits with the addition of ribociclib and no evidence of new safety signals or adverse event exacerbations at 3 years, she said. Dose modifications had no significant impact on efficacy, she added.

The findings of no impact of dose reduction on efficacy in both the NATALEE and monarchE studies provide important information on whether dosage can be reduced in patients, which will increase the odds that patients will tolerate extended therapy with good outcomes and stay on their prescribed therapies, Dr. Hamilton emphasized.

The CARABELA study, a phase 2 trial of neoadjuvant letrozole plus abemaciclib vs adriamycin and cyclophosphamide (AC), showed clinically similar response rates but did not meet its endpoint for residual cancer burden (RCB) scores. These data add to results from other studies and show that it is too soon to universally replace neoadjuvant chemotherapy as first-line treatment for highly proliferative ER+ breast cancer, Dr. Hamilton said in her presentation.
 

Advanced Breast Cancer

Take-home messages about advanced breast cancer include growing evidence for the potential benefits of antibody drug conjugates (ADCs), said Eva Ciruelos, MD, of University Hospital, Madrid, Spain. The TROPION-BREAST01 study, a phase 3 randomized trial, showed significant and clinically meaningful improvement in progression-free survival in patients with previously treated, inoperable, or metastatic HR+/HER2- breast cancer who received datopotamab deruxtecan (Dato-DXd) compared with those who received chemotherapy.

Data from an additional safety analysis were presented at the meeting; although Dato-DXd, a trophoblast cell-surface antigen 2 (TROP2)–directed antibody-drug conjugate, was well-tolerated, it is important to remain aware of toxicities, notably oral mucositis, which occurred in 55.6% of the patients in the study across all grades, and ocular surface toxicity, which occurred in 40% of patients across all grades, Dr. Ciruelos emphasized.

Key research in the area of advanced triple-negative breast cancer included data from the IMPASSION 132 study. This study is “specifically centered on early relapsers,” a population often excluded from other trials, Dr. Ciruelos said. In this study, patients with advanced triple-negative breast cancer were randomized to chemotherapy with or without atezolizumab, and the study showed no benefits with atezolizumab for overall survival, progression-free survival, or overall response rate, she said. “This is something to work with, because this is a very refractory population,” Dr. Ciruelos noted.

New immunotherapy combinations are needed to improve survival in advanced breast cancer patients, Dr. Ciruelos said. At the meeting, researchers presented interim data from a subset of patients in the MORPHEUS-pan breast cancer trial, a phase 1B/2 study involving multiple treatment combinations in locally advanced/metastatic breast cancer patients.

The interim analysis included 18-week data from triple-negative breast cancer patients and compared outcomes for patients randomized to atezolizumab with or without sacituzumab govitecan (SG).

The study was small, with only 31 patients in the combination arm and 11 controls, but the results were promising, with an overall response rate of 76.7% in the combination arm vs 66.7% in the control arm, Dr. Ciruelos said.

Supportive Care

Key supportive care takeaways included data on pregnancy in young breast cancer survivors and the safety of vaginal estrogen therapy in breast cancer patients with genitourinary symptoms, said Anne May, MD, of the University Medical Center Utrecht, Utrecht, Netherlands.

A study previously published in JAMA including nearly 5000 BRCA carriers who were diagnosed with invasive breast cancer at age 40 years or younger showed no association between pregnancy after breast cancer and adverse maternal or fetal outcomes, and pregnancy had no significant impact on overall survival. The authors presented new data on the safety of assisted reproductive techniques (ART) based on the 543 pregnancies in the original study, at the meeting. Of these, 436 conceived naturally, and 107 used ART. After a median of 9.1 years, ART had no effect on disease-free survival compared to natural conception (hazard ratio [HR], 0.64). Based on these findings, fertility preservation should be offered to all women who receive a breast cancer diagnosis and are interested in future fertility, Dr. May said.

Conceiving after breast cancer treatment and follow-up should not be contraindicated for young BRCA carriers, she added.No trial data are available for the effects of vaginal estrogen therapy (VET) on disease-free survival in breast cancer survivors with genitourinary symptoms caused by declining estrogen levels, Dr. May said. However, researchers in France and Switzerland conducted an emulation of a hypothetical target trial using data from the French National social security system for more than 130,000 individuals. Although VET therapy had no impact on disease-free survival in most breast cancer survivors overall, it did have a negative impact in a subset of patients with HR-positive and HR-negative tumors who were treated with aromatase inhibitors. The study was hypothetical, but important because the results suggest that clinicians can safely propose VTE to patients who report genitourinary symptoms after treatment for early-stage breast cancer with tamoxifen, but VTE should be avoided in patients treated with aromatase inhibitors, Dr. May said.

Dr. Delaloge disclosed research support to her institution from AstraZeneca, MSD, Bristol Myers Squibb, Sanofi, Taiho, Novartis, European Commission, INCa, Banque des Territoires, and Fondation Philanthropia. She also disclosed honoraria to her institution from AstraZeneca, Gilead, Novartis, Elsan, Besins, Sanofi, Exact Sciences, and Lilly, as well as travel support from Novartis.

Dr. Kok disclosed research funding from AstraZeneca, Bristol Myers Squibb, Daichi, and Roche, and advisory board membership/speaker’s fees from Alderaan Biotechnology, BIONTECH, Domain Therapeutics, AstraZeneca, Daichi, Bristol Myers Squibb, Gilead, Medscape, MSD, and Roche.

Dr. Hamilton disclosed a consulting advisory role (to her institution) for Accutar Biotechology, AstraZeneca, Daiichi Sankyo, Ellipses Pharma, Entos, Forsum Pharma, Gilead Sciences, Greenwich LifeSciences, Jazz Pharmaceuticals, Lilly, Medical Pharma Services, Mersana, Novartis, Olema Pharmaceuticals, Orum Therapeutics, Roche/Genentech, Stemline Therapeutics, ands others. She also disclosed contracted research/grant support to her institution only from Abbvie, Acerta Pharma, Accutar Biotechnology , ADC Therapeutics, AKESOBIO Australia , Amgen, Aravive, ArQule, Artios, Arvinas, AstraZeneca, AtlasMedx, BeiGene, Black Diamond and others.

Dr. Ciruelos disclosed serving as an external advisor for Roche, MSD, Gilead, AstraZeneca, Daichii Sankyo, Reveal Genomics, Pfizer, Novartis, and Lilly, as well as serving as a speaker for Roche, MSD, Gilead, AstraZeneca, Daichii Sankyo, Reveal Genomics, Pfizer, Novartis, Lilly, and Pierre Fabre. She also disclosed travel grants from Roche, Pfizer, and AstraZeneca, and research grants from Seagen and Roche.

Dr. May had no financial conflicts to disclose.

Patients with metastatic or advanced cancer treated in practices that have high rates of giving systemic care in the last two weeks of life do not have longer survival rates than patients in practices that have low rates of such care.

This conclusion of a new study published online May 16 in JAMA Oncology may help reassure oncologists that giving systemic anticancer therapy (SACT) at the most advanced stages of cancer will not improve the patient’s life, the authors wrote. It also may encourage them to instead focus more on honest communication with patients about their choices, Maureen E. Canavan, PhD, at the Cancer and Outcomes, Public Policy and Effectiveness Research (COPPER) Center at the Yale School of Medicine in New Haven, Connecticut, and colleagues, wrote in their paper.
 

How Was the Study Conducted?

Researchers used Flatiron Health, a nationwide electronic health records database of academic and community practices throughout the United State. They identified 78,446 adults with advanced or metastatic stages of one of six common cancers (breast, colorectal, urothelial, non–small cell lung cancer [NSCLC], pancreatic and renal cell carcinoma) who were treated at healthcare practices from 2015 to 2019. They then stratified practices into quintiles based on how often the practices treated patients with any systemic therapy, including chemotherapy and immunotherapy, in their last 14 days of life. They compared whether patients in practices with greater use of systemic treatment at very advanced stages had longer overall survival.

What Were the Main Findings?

“We saw that there were absolutely no survival differences between the practices that used more systemic therapy for very advanced cancer than the practices that use less,” said senior author Kerin Adelson, MD, chief quality and value officer at MD Anderson Cancer Center in Houston, Texas. In some cancers, those in the lowest quintile (those with the lowest rates of systemic end-of-life care) lived fewer years compared with those in the highest quintiles. In other cancers, those in the lowest quintiles lived more years than those in the highest quintiles.

“What’s important is that none of those differences, after you control for other factors, was statistically significant,” Dr. Adelson said. “That was the same in every cancer type we looked at.”

An example is seen in advanced urothelial cancer. Those in the first quintile (lowest rates of systemic care at end of life) had an SACT rate range of 4.0-9.1. The SACT rate range in the highest quintile was 19.8-42.6. But the median overall survival (OS) rate for those in the lowest quintile was 12.7 months, not statistically different from the median OS in the highest quintile (11 months.)
 

How Does This Study Add to the Literature?

The American Society of Clinical Oncology (ASCO) and the National Quality Forum (NQF) developed a cancer quality metric to reduce SACT at the end of life. The NQF 0210 is a ratio of patients who get systemic treatment within 14 days of death over all patients who die of cancer. The quality metric has been widely adopted and used in value-based care reporting.

But the metric has been criticized because it focuses only on people who died and not people who lived longer because they benefited from the systemic therapy, the authors wrote.

Dr. Canavan’s team focused on all patients treated in the practice, not just those who died, Dr. Adelson said. This may put that criticism to rest, Dr. Adelson said.

“I personally believed the ASCO and NQF metric was appropriate and the criticisms were off base,” said Otis Brawley, MD, associate director of community outreach and engagement at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University School of Medicine in Baltimore. “Canavan’s study is evidence suggesting the metrics were appropriate.”

This study included not just chemotherapy, as some other studies have, but targeted therapies and immunotherapies as well. Dr. Adelson said some think that the newer drugs might change the prognosis at end of life. But this study shows “even those drugs are not helping patients to survive with very advanced cancer,” she said.

 

Could This Change Practice?

The authors noted that end-of life SACT has been linked with more acute care use, delays in conversations about care goals, late enrollment in hospice, higher costs, and potentially shorter and poorer quality life.

Dr. Adelson said she’s hoping that the knowledge that there’s no survival benefit for use of SACT for patients with advanced solid tumors who are nearing the end of life will lead instead to more conversations about prognosis with patients and transitions to palliative care.

“Palliative care has actually been shown to improve quality of life and, in some studies, even survival,” she said.

“I doubt it will change practice, but it should,” Dr. Brawley said. “The study suggests that doctors and patients have too much hope for chemotherapy as patients’ disease progresses. In the US especially, there is a tendency to believe we have better therapies than we truly do and we have difficulty accepting that the patient is dying. Many patients get third- and fourth-line chemotherapy that is highly likely to increase suffering without realistic hope of prolonging life and especially no hope of prolonging life with good quality.”

Dr. Adelson disclosed ties with AbbVie, Quantum Health, Gilead, ParetoHealth, and Carrum Health. Various coauthors disclosed ties with Roche, AbbVie, Johnson & Johnson, Genentech, the National Comprehensive Cancer Network, and AstraZeneca. The study was funded by Flatiron Health, an independent member of the Roche group. Dr. Brawley reports no relevant financial disclosures.

Patients with metastatic or advanced cancer treated in practices that have high rates of giving systemic care in the last two weeks of life do not have longer survival rates than patients in practices that have low rates of such care.

This conclusion of a new study published online May 16 in JAMA Oncology may help reassure oncologists that giving systemic anticancer therapy (SACT) at the most advanced stages of cancer will not improve the patient’s life, the authors wrote. It also may encourage them to instead focus more on honest communication with patients about their choices, Maureen E. Canavan, PhD, at the Cancer and Outcomes, Public Policy and Effectiveness Research (COPPER) Center at the Yale School of Medicine in New Haven, Connecticut, and colleagues, wrote in their paper.
 

How Was the Study Conducted?

Researchers used Flatiron Health, a nationwide electronic health records database of academic and community practices throughout the United State. They identified 78,446 adults with advanced or metastatic stages of one of six common cancers (breast, colorectal, urothelial, non–small cell lung cancer [NSCLC], pancreatic and renal cell carcinoma) who were treated at healthcare practices from 2015 to 2019. They then stratified practices into quintiles based on how often the practices treated patients with any systemic therapy, including chemotherapy and immunotherapy, in their last 14 days of life. They compared whether patients in practices with greater use of systemic treatment at very advanced stages had longer overall survival.

What Were the Main Findings?

“We saw that there were absolutely no survival differences between the practices that used more systemic therapy for very advanced cancer than the practices that use less,” said senior author Kerin Adelson, MD, chief quality and value officer at MD Anderson Cancer Center in Houston, Texas. In some cancers, those in the lowest quintile (those with the lowest rates of systemic end-of-life care) lived fewer years compared with those in the highest quintiles. In other cancers, those in the lowest quintiles lived more years than those in the highest quintiles.

“What’s important is that none of those differences, after you control for other factors, was statistically significant,” Dr. Adelson said. “That was the same in every cancer type we looked at.”

An example is seen in advanced urothelial cancer. Those in the first quintile (lowest rates of systemic care at end of life) had an SACT rate range of 4.0-9.1. The SACT rate range in the highest quintile was 19.8-42.6. But the median overall survival (OS) rate for those in the lowest quintile was 12.7 months, not statistically different from the median OS in the highest quintile (11 months.)
 

How Does This Study Add to the Literature?

The American Society of Clinical Oncology (ASCO) and the National Quality Forum (NQF) developed a cancer quality metric to reduce SACT at the end of life. The NQF 0210 is a ratio of patients who get systemic treatment within 14 days of death over all patients who die of cancer. The quality metric has been widely adopted and used in value-based care reporting.

But the metric has been criticized because it focuses only on people who died and not people who lived longer because they benefited from the systemic therapy, the authors wrote.

Dr. Canavan’s team focused on all patients treated in the practice, not just those who died, Dr. Adelson said. This may put that criticism to rest, Dr. Adelson said.

“I personally believed the ASCO and NQF metric was appropriate and the criticisms were off base,” said Otis Brawley, MD, associate director of community outreach and engagement at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University School of Medicine in Baltimore. “Canavan’s study is evidence suggesting the metrics were appropriate.”

This study included not just chemotherapy, as some other studies have, but targeted therapies and immunotherapies as well. Dr. Adelson said some think that the newer drugs might change the prognosis at end of life. But this study shows “even those drugs are not helping patients to survive with very advanced cancer,” she said.

 

Could This Change Practice?

The authors noted that end-of life SACT has been linked with more acute care use, delays in conversations about care goals, late enrollment in hospice, higher costs, and potentially shorter and poorer quality life.

Dr. Adelson said she’s hoping that the knowledge that there’s no survival benefit for use of SACT for patients with advanced solid tumors who are nearing the end of life will lead instead to more conversations about prognosis with patients and transitions to palliative care.

“Palliative care has actually been shown to improve quality of life and, in some studies, even survival,” she said.

“I doubt it will change practice, but it should,” Dr. Brawley said. “The study suggests that doctors and patients have too much hope for chemotherapy as patients’ disease progresses. In the US especially, there is a tendency to believe we have better therapies than we truly do and we have difficulty accepting that the patient is dying. Many patients get third- and fourth-line chemotherapy that is highly likely to increase suffering without realistic hope of prolonging life and especially no hope of prolonging life with good quality.”

Dr. Adelson disclosed ties with AbbVie, Quantum Health, Gilead, ParetoHealth, and Carrum Health. Various coauthors disclosed ties with Roche, AbbVie, Johnson & Johnson, Genentech, the National Comprehensive Cancer Network, and AstraZeneca. The study was funded by Flatiron Health, an independent member of the Roche group. Dr. Brawley reports no relevant financial disclosures.

Patients with metastatic or advanced cancer treated in practices that have high rates of giving systemic care in the last two weeks of life do not have longer survival rates than patients in practices that have low rates of such care.

This conclusion of a new study published online May 16 in JAMA Oncology may help reassure oncologists that giving systemic anticancer therapy (SACT) at the most advanced stages of cancer will not improve the patient’s life, the authors wrote. It also may encourage them to instead focus more on honest communication with patients about their choices, Maureen E. Canavan, PhD, at the Cancer and Outcomes, Public Policy and Effectiveness Research (COPPER) Center at the Yale School of Medicine in New Haven, Connecticut, and colleagues, wrote in their paper.
 

How Was the Study Conducted?

Researchers used Flatiron Health, a nationwide electronic health records database of academic and community practices throughout the United State. They identified 78,446 adults with advanced or metastatic stages of one of six common cancers (breast, colorectal, urothelial, non–small cell lung cancer [NSCLC], pancreatic and renal cell carcinoma) who were treated at healthcare practices from 2015 to 2019. They then stratified practices into quintiles based on how often the practices treated patients with any systemic therapy, including chemotherapy and immunotherapy, in their last 14 days of life. They compared whether patients in practices with greater use of systemic treatment at very advanced stages had longer overall survival.

What Were the Main Findings?

“We saw that there were absolutely no survival differences between the practices that used more systemic therapy for very advanced cancer than the practices that use less,” said senior author Kerin Adelson, MD, chief quality and value officer at MD Anderson Cancer Center in Houston, Texas. In some cancers, those in the lowest quintile (those with the lowest rates of systemic end-of-life care) lived fewer years compared with those in the highest quintiles. In other cancers, those in the lowest quintiles lived more years than those in the highest quintiles.

“What’s important is that none of those differences, after you control for other factors, was statistically significant,” Dr. Adelson said. “That was the same in every cancer type we looked at.”

An example is seen in advanced urothelial cancer. Those in the first quintile (lowest rates of systemic care at end of life) had an SACT rate range of 4.0-9.1. The SACT rate range in the highest quintile was 19.8-42.6. But the median overall survival (OS) rate for those in the lowest quintile was 12.7 months, not statistically different from the median OS in the highest quintile (11 months.)
 

How Does This Study Add to the Literature?

The American Society of Clinical Oncology (ASCO) and the National Quality Forum (NQF) developed a cancer quality metric to reduce SACT at the end of life. The NQF 0210 is a ratio of patients who get systemic treatment within 14 days of death over all patients who die of cancer. The quality metric has been widely adopted and used in value-based care reporting.

But the metric has been criticized because it focuses only on people who died and not people who lived longer because they benefited from the systemic therapy, the authors wrote.

Dr. Canavan’s team focused on all patients treated in the practice, not just those who died, Dr. Adelson said. This may put that criticism to rest, Dr. Adelson said.

“I personally believed the ASCO and NQF metric was appropriate and the criticisms were off base,” said Otis Brawley, MD, associate director of community outreach and engagement at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University School of Medicine in Baltimore. “Canavan’s study is evidence suggesting the metrics were appropriate.”

This study included not just chemotherapy, as some other studies have, but targeted therapies and immunotherapies as well. Dr. Adelson said some think that the newer drugs might change the prognosis at end of life. But this study shows “even those drugs are not helping patients to survive with very advanced cancer,” she said.

 

Could This Change Practice?

The authors noted that end-of life SACT has been linked with more acute care use, delays in conversations about care goals, late enrollment in hospice, higher costs, and potentially shorter and poorer quality life.

Dr. Adelson said she’s hoping that the knowledge that there’s no survival benefit for use of SACT for patients with advanced solid tumors who are nearing the end of life will lead instead to more conversations about prognosis with patients and transitions to palliative care.

“Palliative care has actually been shown to improve quality of life and, in some studies, even survival,” she said.

“I doubt it will change practice, but it should,” Dr. Brawley said. “The study suggests that doctors and patients have too much hope for chemotherapy as patients’ disease progresses. In the US especially, there is a tendency to believe we have better therapies than we truly do and we have difficulty accepting that the patient is dying. Many patients get third- and fourth-line chemotherapy that is highly likely to increase suffering without realistic hope of prolonging life and especially no hope of prolonging life with good quality.”

Dr. Adelson disclosed ties with AbbVie, Quantum Health, Gilead, ParetoHealth, and Carrum Health. Various coauthors disclosed ties with Roche, AbbVie, Johnson & Johnson, Genentech, the National Comprehensive Cancer Network, and AstraZeneca. The study was funded by Flatiron Health, an independent member of the Roche group. Dr. Brawley reports no relevant financial disclosures.

PHOENIX — Immunotherapy has revolutionized outcomes for patients with metastatic Merkel cell carcinoma (MCC). However, findings from a new study suggest that many patients who are eligible for immunotherapy are not receiving this treatment, despite guideline recommendations, and survival outcomes are better at high-volume centers.

The study has important implications, said study author Shayan Cheraghlou, MD, an incoming fellow in Mohs surgery at New York University, New York City. “We can see that in a real-world setting, these agents have an impact on survival,” he said. “We also found high-volume centers were significantly more likely to use the agents than low-volume centers.” He presented the findings at the annual meeting of the American College of Mohs Surgery.

MCC is a neuroendocrine skin cancer with a high rate of mortality, and even though it remains relatively rare, its incidence has been rising rapidly since the late 1990s and continues to increase. There were no approved treatments available until 2017, when the US Food and Drug Administration (FDA) approved the immunotherapy drug avelumab (Bavencio) to treat advanced MCC. Two years later, pembrolizumab (Keytruda) also received regulatory approval for MCC, and these two agents have revolutionized outcomes.

“In clinical trial settings, these agents led to significant and durable responses, and they are now the recommended treatments in guidelines for metastatic Merkel cell carcinoma,” said Dr. Cheraghlou. “However, we don’t have data as to how they are being used in the real-world setting and if survival outcomes are similar.”

Real World vs Clinical Trials

Real-world outcomes can differ from clinical trial data, and the adoption of novel therapeutics can be gradual. The goal of this study was to see if clinical trial data matched what was being observed in actual clinical use and if the agents were being used uniformly in centers across the United States.

The authors used data from the National Cancer Database that included patients diagnosed with cancer from 2004 to 2019 and identified 1017 adult cases of metastatic MCC. They then looked at the association of a variety of patient characteristics, tumors, and system factors with the likelihood of receiving systemic treatment for their disease.

“Our first finding was maybe the least surprising,” he said. “Patients who received these therapeutic agents had significantly improved survival compared to those who have not.”

Those who received immunotherapy had a 35% decrease in the risk for death per year compared with those who did not. The 1-, 3-, and 5-year survival rates were 47.2%, 21.8%, and 16.5%, respectively, for patients who did not receive immunotherapy compared with 62.7%, 34.4%, and 23.6%, respectively, for those who were treated with these agents.

Dr. Cheraghlou noted that they started to get some “surprising” findings when they looked at utilization data. “While it has been increasing over time, it is not as high as it should be,” he emphasized.

From 2017 to 2019, 54.2% of patients with metastatic MCC received immunotherapy. The data also showed an increase in use from 45.1% in 2017 to 63.0% in 2019. “This is an effective treatment for aggressive malignancy, so we have to ask why more patients aren’t getting them,” said Dr. Cheraghlou.

Their findings did suggest one possible reason, and that was that high-volume centers were significantly more likely to use the agents than low-volume centers. Centers that were in the top percentile for MCC case volume were three times as likely to use immunotherapy for MCC compared with other institutions. “So, if you have metastatic Merkel cell carcinoma and go to a low volume center, you may be less likely to get potential lifesaving treatment,” he noted.
 

Implications Going Forward

Dr. Cheraghlou concluded his presentation by pointing out that this study has important implications. The data showed that in a real-world setting, these agents have an impact on survival, but all eligible patients do not have access. “In other countries, there are established referral patterns for all patients with aggressive rare malignancies and really all cancers,” he added. “But in the US, cancer care is more decentralized. Studies like this and others show that high-volume centers have much better outcomes for aggressive rare malignancies, and we should be looking at why this is the case and mitigating these disparities and outcomes.”

Commenting on the study results, Jeffrey M. Farma, MD, co-director of the Melanoma and Skin Cancer Program and professor of surgical oncology at Fox Chase Cancer Center, Philadelphia, referred to the two immunotherapies that have been approved for MCC since 2017, which have demonstrated a survival benefit and improved outcomes in patients with metastatic MCC.

Fox Chase Cancer Center

“In their study, immunotherapy was associated with improved outcomes,” said Dr. Farma. “This study highlights the continued lag of implementation of guidelines when new therapies are approved, and that for rare cancers like Merkel cell carcinoma, being treated at high-volume centers and the regionalization of care can lead to improved outcomes for patients.”

Dr. Cheraghlou and Dr. Farma had no disclosures.

A version of this article appeared on Medscape.com.

PHOENIX — Immunotherapy has revolutionized outcomes for patients with metastatic Merkel cell carcinoma (MCC). However, findings from a new study suggest that many patients who are eligible for immunotherapy are not receiving this treatment, despite guideline recommendations, and survival outcomes are better at high-volume centers.

The study has important implications, said study author Shayan Cheraghlou, MD, an incoming fellow in Mohs surgery at New York University, New York City. “We can see that in a real-world setting, these agents have an impact on survival,” he said. “We also found high-volume centers were significantly more likely to use the agents than low-volume centers.” He presented the findings at the annual meeting of the American College of Mohs Surgery.

MCC is a neuroendocrine skin cancer with a high rate of mortality, and even though it remains relatively rare, its incidence has been rising rapidly since the late 1990s and continues to increase. There were no approved treatments available until 2017, when the US Food and Drug Administration (FDA) approved the immunotherapy drug avelumab (Bavencio) to treat advanced MCC. Two years later, pembrolizumab (Keytruda) also received regulatory approval for MCC, and these two agents have revolutionized outcomes.

“In clinical trial settings, these agents led to significant and durable responses, and they are now the recommended treatments in guidelines for metastatic Merkel cell carcinoma,” said Dr. Cheraghlou. “However, we don’t have data as to how they are being used in the real-world setting and if survival outcomes are similar.”

Real World vs Clinical Trials

Real-world outcomes can differ from clinical trial data, and the adoption of novel therapeutics can be gradual. The goal of this study was to see if clinical trial data matched what was being observed in actual clinical use and if the agents were being used uniformly in centers across the United States.

The authors used data from the National Cancer Database that included patients diagnosed with cancer from 2004 to 2019 and identified 1017 adult cases of metastatic MCC. They then looked at the association of a variety of patient characteristics, tumors, and system factors with the likelihood of receiving systemic treatment for their disease.

“Our first finding was maybe the least surprising,” he said. “Patients who received these therapeutic agents had significantly improved survival compared to those who have not.”

Those who received immunotherapy had a 35% decrease in the risk for death per year compared with those who did not. The 1-, 3-, and 5-year survival rates were 47.2%, 21.8%, and 16.5%, respectively, for patients who did not receive immunotherapy compared with 62.7%, 34.4%, and 23.6%, respectively, for those who were treated with these agents.

Dr. Cheraghlou noted that they started to get some “surprising” findings when they looked at utilization data. “While it has been increasing over time, it is not as high as it should be,” he emphasized.

From 2017 to 2019, 54.2% of patients with metastatic MCC received immunotherapy. The data also showed an increase in use from 45.1% in 2017 to 63.0% in 2019. “This is an effective treatment for aggressive malignancy, so we have to ask why more patients aren’t getting them,” said Dr. Cheraghlou.

Their findings did suggest one possible reason, and that was that high-volume centers were significantly more likely to use the agents than low-volume centers. Centers that were in the top percentile for MCC case volume were three times as likely to use immunotherapy for MCC compared with other institutions. “So, if you have metastatic Merkel cell carcinoma and go to a low volume center, you may be less likely to get potential lifesaving treatment,” he noted.
 

Implications Going Forward

Dr. Cheraghlou concluded his presentation by pointing out that this study has important implications. The data showed that in a real-world setting, these agents have an impact on survival, but all eligible patients do not have access. “In other countries, there are established referral patterns for all patients with aggressive rare malignancies and really all cancers,” he added. “But in the US, cancer care is more decentralized. Studies like this and others show that high-volume centers have much better outcomes for aggressive rare malignancies, and we should be looking at why this is the case and mitigating these disparities and outcomes.”

Commenting on the study results, Jeffrey M. Farma, MD, co-director of the Melanoma and Skin Cancer Program and professor of surgical oncology at Fox Chase Cancer Center, Philadelphia, referred to the two immunotherapies that have been approved for MCC since 2017, which have demonstrated a survival benefit and improved outcomes in patients with metastatic MCC.

Fox Chase Cancer Center

“In their study, immunotherapy was associated with improved outcomes,” said Dr. Farma. “This study highlights the continued lag of implementation of guidelines when new therapies are approved, and that for rare cancers like Merkel cell carcinoma, being treated at high-volume centers and the regionalization of care can lead to improved outcomes for patients.”

Dr. Cheraghlou and Dr. Farma had no disclosures.

A version of this article appeared on Medscape.com.

PHOENIX — Immunotherapy has revolutionized outcomes for patients with metastatic Merkel cell carcinoma (MCC). However, findings from a new study suggest that many patients who are eligible for immunotherapy are not receiving this treatment, despite guideline recommendations, and survival outcomes are better at high-volume centers.

The study has important implications, said study author Shayan Cheraghlou, MD, an incoming fellow in Mohs surgery at New York University, New York City. “We can see that in a real-world setting, these agents have an impact on survival,” he said. “We also found high-volume centers were significantly more likely to use the agents than low-volume centers.” He presented the findings at the annual meeting of the American College of Mohs Surgery.

MCC is a neuroendocrine skin cancer with a high rate of mortality, and even though it remains relatively rare, its incidence has been rising rapidly since the late 1990s and continues to increase. There were no approved treatments available until 2017, when the US Food and Drug Administration (FDA) approved the immunotherapy drug avelumab (Bavencio) to treat advanced MCC. Two years later, pembrolizumab (Keytruda) also received regulatory approval for MCC, and these two agents have revolutionized outcomes.

“In clinical trial settings, these agents led to significant and durable responses, and they are now the recommended treatments in guidelines for metastatic Merkel cell carcinoma,” said Dr. Cheraghlou. “However, we don’t have data as to how they are being used in the real-world setting and if survival outcomes are similar.”

Real World vs Clinical Trials

Real-world outcomes can differ from clinical trial data, and the adoption of novel therapeutics can be gradual. The goal of this study was to see if clinical trial data matched what was being observed in actual clinical use and if the agents were being used uniformly in centers across the United States.

The authors used data from the National Cancer Database that included patients diagnosed with cancer from 2004 to 2019 and identified 1017 adult cases of metastatic MCC. They then looked at the association of a variety of patient characteristics, tumors, and system factors with the likelihood of receiving systemic treatment for their disease.

“Our first finding was maybe the least surprising,” he said. “Patients who received these therapeutic agents had significantly improved survival compared to those who have not.”

Those who received immunotherapy had a 35% decrease in the risk for death per year compared with those who did not. The 1-, 3-, and 5-year survival rates were 47.2%, 21.8%, and 16.5%, respectively, for patients who did not receive immunotherapy compared with 62.7%, 34.4%, and 23.6%, respectively, for those who were treated with these agents.

Dr. Cheraghlou noted that they started to get some “surprising” findings when they looked at utilization data. “While it has been increasing over time, it is not as high as it should be,” he emphasized.

From 2017 to 2019, 54.2% of patients with metastatic MCC received immunotherapy. The data also showed an increase in use from 45.1% in 2017 to 63.0% in 2019. “This is an effective treatment for aggressive malignancy, so we have to ask why more patients aren’t getting them,” said Dr. Cheraghlou.

Their findings did suggest one possible reason, and that was that high-volume centers were significantly more likely to use the agents than low-volume centers. Centers that were in the top percentile for MCC case volume were three times as likely to use immunotherapy for MCC compared with other institutions. “So, if you have metastatic Merkel cell carcinoma and go to a low volume center, you may be less likely to get potential lifesaving treatment,” he noted.
 

Implications Going Forward

Dr. Cheraghlou concluded his presentation by pointing out that this study has important implications. The data showed that in a real-world setting, these agents have an impact on survival, but all eligible patients do not have access. “In other countries, there are established referral patterns for all patients with aggressive rare malignancies and really all cancers,” he added. “But in the US, cancer care is more decentralized. Studies like this and others show that high-volume centers have much better outcomes for aggressive rare malignancies, and we should be looking at why this is the case and mitigating these disparities and outcomes.”

Commenting on the study results, Jeffrey M. Farma, MD, co-director of the Melanoma and Skin Cancer Program and professor of surgical oncology at Fox Chase Cancer Center, Philadelphia, referred to the two immunotherapies that have been approved for MCC since 2017, which have demonstrated a survival benefit and improved outcomes in patients with metastatic MCC.

Fox Chase Cancer Center

“In their study, immunotherapy was associated with improved outcomes,” said Dr. Farma. “This study highlights the continued lag of implementation of guidelines when new therapies are approved, and that for rare cancers like Merkel cell carcinoma, being treated at high-volume centers and the regionalization of care can lead to improved outcomes for patients.”

Dr. Cheraghlou and Dr. Farma had no disclosures.

A version of this article appeared on Medscape.com.

Among patients with irritable bowel syndrome (IBS), a low-carbohydrate diet or a diet low in fermentable oligosaccharides, disaccharides, monosaccharides, and polyols (FODMAP) plus traditional IBS dietary advice outperformed pharmacological treatment.

According to a new study, evidence was found that these dietary interventions were more efficacious at 4 weeks, suggesting their potential as first-line treatments.

“IBS is a disorder that may have different underlying causes, and it can manifest in different ways among patients. It is also likely that the most effective treatment option can differ in patients,” said lead author Sanna Nybacka, RD, PhD, a postdoctoral researcher in molecular and clinical medicine at the University of Gothenburg’s Sahlgrenska Academy, Gothenburg, Sweden.

“Up to 80% of patients with IBS report that their symptoms are exacerbated by dietary factors, and dietary modifications are considered a promising avenue for alleviating IBS symptoms,” she said. “However, as not all patients respond to dietary modifications, we need studies comparing the effectiveness of dietary vs pharmacological treatments in IBS to better understand which patients are more likely to benefit from which treatment.”

The study was published online in The Lancet Gastroenterology and Hepatology.
 

Treatment Comparison

Dr. Nybacka and colleagues conducted a single-blind randomized controlled trial at a specialized outpatient clinic at Sahlgrenska University Hospital in Gothenburg, Sweden, between January 2017 and September 2021. They included adults with moderate to severe IBS, which was defined as ≥ 175 points on the IBS Severity Scoring System (IBS-SSS), and who had no other serious diseases or food allergies.

The participants were assigned 1:1:1 to receive a low-FODMAP diet plus traditional dietary advice (50% carbohydrates, 33% fat, 17% protein), a fiber-optimized diet with low carbohydrates and high protein and fat (10% carbohydrates, 67% fat, 23% protein), or optimized medical treatment based on predominant IBS symptoms. Participants were masked to the names of the diets, but the pharmacological treatment was open-label.

After 4 weeks, participants were unmasked and encouraged to continue their diets.

During 6 months of follow-up, those in the low-FODMAP group were instructed on how to reintroduce FODMAPs, and those in the pharmacological treatment group were offered personalized diet counseling and to continue their medication.

Among 1104 participants assessed for eligibility, 304 were randomly assigned. However, 10 participants did not receive their intervention after randomization, so only 294 participants were included in the modified intention-to-treat population: 96 in the low-FODMAP group, 97 in the low-carbohydrate group, and 101 in the optimized medical treatment group. Overall, 82% were women, and the mean age was 38 years.

Following the 4-week intervention, 73 of 96 participants (76%) in the low-FODMAP group, 69 of 97 participants (71%) in the low-carbohydrate group, and 59 of 101 participants (58%) in the optimized medical treatment group had a reduction of ≥ 50 points in the IBS-SSS compared with baseline.

A stricter score reduction of ≥ 100 points was observed in 61% of the low-FODMAP group, 58% of the low-carbohydrate group, and 39% of the optimized medical treatment group.

In both the low-FODMAP group and the low-carbohydrate group, 95% of participants completed the 4-week intervention compared with 90% among the pharmacological group. Two people in each group said adverse events prompted their discontinuation, and five in the medical treatment group stopped prematurely due to side effects. No serious adverse events or treatment-related deaths occurred.

“We were surprised by the effectiveness of the fiber-optimized low-carbohydrate diet, which demonstrated comparable efficacy to the combined low-FODMAP and traditional IBS diet,” Dr. Nybacka said. “While previous knowledge suggested that high-fat intake could worsen symptoms in some individuals, the synergy with low-carbohydrate intake appeared to render the diet more tolerable for these patients.”

The authors noted that since all three treatment options showed significant and clinically meaningful efficacy, patient preference, ease of implementation, compliance, cost-effectiveness, and long-term effects, including those on nutritional status and gut microbiota, should be considered in personalized plans.
 

Future Practice Considerations

Dr. Nybacka and colleagues recommended additional trials before implementing the low-carbohydrate diet in clinical practice. “Worse blood lipid levels among some participants in the low-carbohydrate group point to an area for caution,” she said.

The research team also plans to evaluate changes in microbiota composition and metabolomic profiles among participants to further understand factors associated with positive treatment outcomes.

“Approximately two thirds of patients with IBS report that certain foods trigger symptoms of IBS, which is why many patients are interested in exploring dietary interventions for their symptoms,” said Brian Lacy, MD, professor of medicine and program director of the GI fellowship program at the Mayo Clinic in Jacksonville, Florida. “One of the most commonly employed diets for the treatment of IBS is the low-FODMAP diet.”

Dr. Lacy, who wasn’t involved with this study, co-authored the 2021 American College of Gastroenterology clinical guideline for the management of IBS.

He and his colleagues recommended a limited trial of a low-FODMAP diet to improve symptoms, as well as targeted use of medications for IBS subtypes with constipation or diarrhea and gut-directed psychotherapy for overall IBS symptoms.

“However, there are problems with the low-FODMAP diet, as it can be difficult to institute, it can be fairly restrictive, and long-term use has the potential to lead to micronutrient deficiencies,” he said. “Importantly, large studies comparing dietary interventions directly to medical therapies are absent, which led to the study by Nybacka and colleagues.”

Dr. Lacy noted several limitations, including the single-center focus, short-term intervention, and variety of therapies used among the medical arm of the study. In addition, some therapies available in the United States aren’t available in Europe, so the varying approaches to medical management in the former may lead to different results. At the same time, he said, the study is important and will be widely discussed among patients and clinicians.

“I think it will likely stand the test of time,” Dr. Lacy said. “An easy-to-use diet with common sense advice that improves symptoms will likely eventually translate into first-line therapy for IBS patients.”

The study was funded by grants from the Healthcare Board Region Västra Götaland, Swedish Research Council, Swedish Research Council for Health, Working Life and Welfare, and AFA Insurance; the ALF agreement between the Swedish government and county councils; Wilhelm and Martina Lundgren Science Foundation; Skandia; Dietary Science Foundation; and Nanna Swartz Foundation. Several authors declared grants, consulting fees, and advisory board roles with various pharmaceutical companies. Dr. Lacy reported no relevant disclosures.

A version of this article appeared on Medscape.com.

Among patients with irritable bowel syndrome (IBS), a low-carbohydrate diet or a diet low in fermentable oligosaccharides, disaccharides, monosaccharides, and polyols (FODMAP) plus traditional IBS dietary advice outperformed pharmacological treatment.

According to a new study, evidence was found that these dietary interventions were more efficacious at 4 weeks, suggesting their potential as first-line treatments.

“IBS is a disorder that may have different underlying causes, and it can manifest in different ways among patients. It is also likely that the most effective treatment option can differ in patients,” said lead author Sanna Nybacka, RD, PhD, a postdoctoral researcher in molecular and clinical medicine at the University of Gothenburg’s Sahlgrenska Academy, Gothenburg, Sweden.

“Up to 80% of patients with IBS report that their symptoms are exacerbated by dietary factors, and dietary modifications are considered a promising avenue for alleviating IBS symptoms,” she said. “However, as not all patients respond to dietary modifications, we need studies comparing the effectiveness of dietary vs pharmacological treatments in IBS to better understand which patients are more likely to benefit from which treatment.”

The study was published online in The Lancet Gastroenterology and Hepatology.
 

Treatment Comparison

Dr. Nybacka and colleagues conducted a single-blind randomized controlled trial at a specialized outpatient clinic at Sahlgrenska University Hospital in Gothenburg, Sweden, between January 2017 and September 2021. They included adults with moderate to severe IBS, which was defined as ≥ 175 points on the IBS Severity Scoring System (IBS-SSS), and who had no other serious diseases or food allergies.

The participants were assigned 1:1:1 to receive a low-FODMAP diet plus traditional dietary advice (50% carbohydrates, 33% fat, 17% protein), a fiber-optimized diet with low carbohydrates and high protein and fat (10% carbohydrates, 67% fat, 23% protein), or optimized medical treatment based on predominant IBS symptoms. Participants were masked to the names of the diets, but the pharmacological treatment was open-label.

After 4 weeks, participants were unmasked and encouraged to continue their diets.

During 6 months of follow-up, those in the low-FODMAP group were instructed on how to reintroduce FODMAPs, and those in the pharmacological treatment group were offered personalized diet counseling and to continue their medication.

Among 1104 participants assessed for eligibility, 304 were randomly assigned. However, 10 participants did not receive their intervention after randomization, so only 294 participants were included in the modified intention-to-treat population: 96 in the low-FODMAP group, 97 in the low-carbohydrate group, and 101 in the optimized medical treatment group. Overall, 82% were women, and the mean age was 38 years.

Following the 4-week intervention, 73 of 96 participants (76%) in the low-FODMAP group, 69 of 97 participants (71%) in the low-carbohydrate group, and 59 of 101 participants (58%) in the optimized medical treatment group had a reduction of ≥ 50 points in the IBS-SSS compared with baseline.

A stricter score reduction of ≥ 100 points was observed in 61% of the low-FODMAP group, 58% of the low-carbohydrate group, and 39% of the optimized medical treatment group.

In both the low-FODMAP group and the low-carbohydrate group, 95% of participants completed the 4-week intervention compared with 90% among the pharmacological group. Two people in each group said adverse events prompted their discontinuation, and five in the medical treatment group stopped prematurely due to side effects. No serious adverse events or treatment-related deaths occurred.

“We were surprised by the effectiveness of the fiber-optimized low-carbohydrate diet, which demonstrated comparable efficacy to the combined low-FODMAP and traditional IBS diet,” Dr. Nybacka said. “While previous knowledge suggested that high-fat intake could worsen symptoms in some individuals, the synergy with low-carbohydrate intake appeared to render the diet more tolerable for these patients.”

The authors noted that since all three treatment options showed significant and clinically meaningful efficacy, patient preference, ease of implementation, compliance, cost-effectiveness, and long-term effects, including those on nutritional status and gut microbiota, should be considered in personalized plans.
 

Future Practice Considerations

Dr. Nybacka and colleagues recommended additional trials before implementing the low-carbohydrate diet in clinical practice. “Worse blood lipid levels among some participants in the low-carbohydrate group point to an area for caution,” she said.

The research team also plans to evaluate changes in microbiota composition and metabolomic profiles among participants to further understand factors associated with positive treatment outcomes.

“Approximately two thirds of patients with IBS report that certain foods trigger symptoms of IBS, which is why many patients are interested in exploring dietary interventions for their symptoms,” said Brian Lacy, MD, professor of medicine and program director of the GI fellowship program at the Mayo Clinic in Jacksonville, Florida. “One of the most commonly employed diets for the treatment of IBS is the low-FODMAP diet.”

Dr. Lacy, who wasn’t involved with this study, co-authored the 2021 American College of Gastroenterology clinical guideline for the management of IBS.

He and his colleagues recommended a limited trial of a low-FODMAP diet to improve symptoms, as well as targeted use of medications for IBS subtypes with constipation or diarrhea and gut-directed psychotherapy for overall IBS symptoms.

“However, there are problems with the low-FODMAP diet, as it can be difficult to institute, it can be fairly restrictive, and long-term use has the potential to lead to micronutrient deficiencies,” he said. “Importantly, large studies comparing dietary interventions directly to medical therapies are absent, which led to the study by Nybacka and colleagues.”

Dr. Lacy noted several limitations, including the single-center focus, short-term intervention, and variety of therapies used among the medical arm of the study. In addition, some therapies available in the United States aren’t available in Europe, so the varying approaches to medical management in the former may lead to different results. At the same time, he said, the study is important and will be widely discussed among patients and clinicians.

“I think it will likely stand the test of time,” Dr. Lacy said. “An easy-to-use diet with common sense advice that improves symptoms will likely eventually translate into first-line therapy for IBS patients.”

The study was funded by grants from the Healthcare Board Region Västra Götaland, Swedish Research Council, Swedish Research Council for Health, Working Life and Welfare, and AFA Insurance; the ALF agreement between the Swedish government and county councils; Wilhelm and Martina Lundgren Science Foundation; Skandia; Dietary Science Foundation; and Nanna Swartz Foundation. Several authors declared grants, consulting fees, and advisory board roles with various pharmaceutical companies. Dr. Lacy reported no relevant disclosures.

A version of this article appeared on Medscape.com.

Among patients with irritable bowel syndrome (IBS), a low-carbohydrate diet or a diet low in fermentable oligosaccharides, disaccharides, monosaccharides, and polyols (FODMAP) plus traditional IBS dietary advice outperformed pharmacological treatment.

According to a new study, evidence was found that these dietary interventions were more efficacious at 4 weeks, suggesting their potential as first-line treatments.

“IBS is a disorder that may have different underlying causes, and it can manifest in different ways among patients. It is also likely that the most effective treatment option can differ in patients,” said lead author Sanna Nybacka, RD, PhD, a postdoctoral researcher in molecular and clinical medicine at the University of Gothenburg’s Sahlgrenska Academy, Gothenburg, Sweden.

“Up to 80% of patients with IBS report that their symptoms are exacerbated by dietary factors, and dietary modifications are considered a promising avenue for alleviating IBS symptoms,” she said. “However, as not all patients respond to dietary modifications, we need studies comparing the effectiveness of dietary vs pharmacological treatments in IBS to better understand which patients are more likely to benefit from which treatment.”

The study was published online in The Lancet Gastroenterology and Hepatology.
 

Treatment Comparison

Dr. Nybacka and colleagues conducted a single-blind randomized controlled trial at a specialized outpatient clinic at Sahlgrenska University Hospital in Gothenburg, Sweden, between January 2017 and September 2021. They included adults with moderate to severe IBS, which was defined as ≥ 175 points on the IBS Severity Scoring System (IBS-SSS), and who had no other serious diseases or food allergies.

The participants were assigned 1:1:1 to receive a low-FODMAP diet plus traditional dietary advice (50% carbohydrates, 33% fat, 17% protein), a fiber-optimized diet with low carbohydrates and high protein and fat (10% carbohydrates, 67% fat, 23% protein), or optimized medical treatment based on predominant IBS symptoms. Participants were masked to the names of the diets, but the pharmacological treatment was open-label.

After 4 weeks, participants were unmasked and encouraged to continue their diets.

During 6 months of follow-up, those in the low-FODMAP group were instructed on how to reintroduce FODMAPs, and those in the pharmacological treatment group were offered personalized diet counseling and to continue their medication.

Among 1104 participants assessed for eligibility, 304 were randomly assigned. However, 10 participants did not receive their intervention after randomization, so only 294 participants were included in the modified intention-to-treat population: 96 in the low-FODMAP group, 97 in the low-carbohydrate group, and 101 in the optimized medical treatment group. Overall, 82% were women, and the mean age was 38 years.

Following the 4-week intervention, 73 of 96 participants (76%) in the low-FODMAP group, 69 of 97 participants (71%) in the low-carbohydrate group, and 59 of 101 participants (58%) in the optimized medical treatment group had a reduction of ≥ 50 points in the IBS-SSS compared with baseline.

A stricter score reduction of ≥ 100 points was observed in 61% of the low-FODMAP group, 58% of the low-carbohydrate group, and 39% of the optimized medical treatment group.

In both the low-FODMAP group and the low-carbohydrate group, 95% of participants completed the 4-week intervention compared with 90% among the pharmacological group. Two people in each group said adverse events prompted their discontinuation, and five in the medical treatment group stopped prematurely due to side effects. No serious adverse events or treatment-related deaths occurred.

“We were surprised by the effectiveness of the fiber-optimized low-carbohydrate diet, which demonstrated comparable efficacy to the combined low-FODMAP and traditional IBS diet,” Dr. Nybacka said. “While previous knowledge suggested that high-fat intake could worsen symptoms in some individuals, the synergy with low-carbohydrate intake appeared to render the diet more tolerable for these patients.”

The authors noted that since all three treatment options showed significant and clinically meaningful efficacy, patient preference, ease of implementation, compliance, cost-effectiveness, and long-term effects, including those on nutritional status and gut microbiota, should be considered in personalized plans.
 

Future Practice Considerations

Dr. Nybacka and colleagues recommended additional trials before implementing the low-carbohydrate diet in clinical practice. “Worse blood lipid levels among some participants in the low-carbohydrate group point to an area for caution,” she said.

The research team also plans to evaluate changes in microbiota composition and metabolomic profiles among participants to further understand factors associated with positive treatment outcomes.

“Approximately two thirds of patients with IBS report that certain foods trigger symptoms of IBS, which is why many patients are interested in exploring dietary interventions for their symptoms,” said Brian Lacy, MD, professor of medicine and program director of the GI fellowship program at the Mayo Clinic in Jacksonville, Florida. “One of the most commonly employed diets for the treatment of IBS is the low-FODMAP diet.”

Dr. Lacy, who wasn’t involved with this study, co-authored the 2021 American College of Gastroenterology clinical guideline for the management of IBS.

He and his colleagues recommended a limited trial of a low-FODMAP diet to improve symptoms, as well as targeted use of medications for IBS subtypes with constipation or diarrhea and gut-directed psychotherapy for overall IBS symptoms.

“However, there are problems with the low-FODMAP diet, as it can be difficult to institute, it can be fairly restrictive, and long-term use has the potential to lead to micronutrient deficiencies,” he said. “Importantly, large studies comparing dietary interventions directly to medical therapies are absent, which led to the study by Nybacka and colleagues.”

Dr. Lacy noted several limitations, including the single-center focus, short-term intervention, and variety of therapies used among the medical arm of the study. In addition, some therapies available in the United States aren’t available in Europe, so the varying approaches to medical management in the former may lead to different results. At the same time, he said, the study is important and will be widely discussed among patients and clinicians.

“I think it will likely stand the test of time,” Dr. Lacy said. “An easy-to-use diet with common sense advice that improves symptoms will likely eventually translate into first-line therapy for IBS patients.”

The study was funded by grants from the Healthcare Board Region Västra Götaland, Swedish Research Council, Swedish Research Council for Health, Working Life and Welfare, and AFA Insurance; the ALF agreement between the Swedish government and county councils; Wilhelm and Martina Lundgren Science Foundation; Skandia; Dietary Science Foundation; and Nanna Swartz Foundation. Several authors declared grants, consulting fees, and advisory board roles with various pharmaceutical companies. Dr. Lacy reported no relevant disclosures.

A version of this article appeared on Medscape.com.