Pharmacology

Physicians may be missing opportunities to reduce harmful polypharmacy in elderly patients with newly diagnosed dementia, investigators for a large study of Medicare beneficiaries reported.

They found that those with an incident dementia diagnosis were somewhat more likely to initiate central nervous system–active medications and slightly more likely to discontinue cardiometabolic and anticholinergic medications, compared with controls.

According to the authors, time of diagnosis can be a potential inflexion point for deprescribing long-term medications with high safety risks, limited likelihood of benefit, or possible association with impaired cognition.

“Understanding the chronology of medication changes following a first dementia diagnosis may identify targets for deprescribing interventions to reduce preventable medication-related harms, said Timothy S. Anderson, MD, MAS, of the division of general medicine at Beth Israel Deaconess Medical Center, Boston, and colleagues in JAMA Internal Medicine.

“Our results provide a baseline to inform efforts to rethink the clinical approach to medication use at the time of a new dementia diagnosis.”

Hundreds of thousands of Americans are diagnosed annually with Alzheimer’s and related dementias, the authors pointed out, and the majority have multiple other chronic conditions. Worsening cognitive impairment may alter the risk-benefit balance of medications taken for these conditions.

Matched cohort study

The sample consisted of adults 67 years or older enrolled in traditional Medicare and Medicare Part D. Patients with an initial incident dementia diagnosis between January 2012 and December 2018 were matched with controls (as of last doctor’s office visit) based on demographics, geographic location, and baseline medication count. Data were analyzed from 2021 to June 2023.

The study included 266,675 adults with incident dementia and 266,675 controls. In both groups, 65.1% were 80 years or older (mean age, 82.2) and 67.8% were female. At baseline, patients with incident dementia were more likely than controls to use CNS-active medications (54.32% vs. 48.39%) and anticholinergic medications (17.79% vs. 15.96%) and less likely to use most cardiometabolic medications (for example, antidiabetics, 31.19% vs. 36.45%).

Immediately following the index diagnosis, the dementia cohort had greater increases in the mean number of medications used: 0.41 vs. –0.06 (95% confidence interval, 0.27-0.66) and in the proportion using CNS-active medications (absolute change, 3.44% vs. 0.79%; 95% CI, 0.85%-4.45%). The rise was because of an increased use of antipsychotics, antidepressants, and antiepileptics.

The affected cohort showed a modestly greater decline in anticholinergic medications: quarterly change in use: −0.53% vs. −0.21% (95% CI, −0.55% to −0.08%); and in most cardiometabolic medications: for example, quarterly change in antihypertensive use: –0.84% vs. –0.40% (95% CI, –0.64% to –0.25%). Still, a year post diagnosis, 75.2% of dementia patients were using five or more medications, for a 2.8% increase.

The drug classes with the steepest rate of discontinuation – such as lipid-lowering and antihypertensive medications – had low risks for adverse drug events, while higher-risk classes – such as insulins and antiplatelet and anticoagulant agents – had smaller or no reductions in use.

While the findings point to opportunities to reduce polypharmacy by deprescribing long-term medications of dubious benefit, interventions to reduce polypharmacy and inappropriate medications have been modestly successful for patients without dementia, the authors said. But the recent OPTIMIZE trial, an educational effort aimed at primary care clinicians and patients with cognitive impairment, reduced neither polypharmacy nor potentially inappropriate medications.

Luke D. Kim, MD, a geriatrician at the Cleveland Clinic in Ohio, agreed that seniors with dementia can benefit from reassessment of their pharmacologic therapies. “Older adults in general are more prone to have side effects from medications as their renal and hepatic clearance and metabolism are different and lower than those of younger individuals. But they tend to take multiple medications owing to more comorbidities,” said Dr. Kim, who was not involved in the study. “While all older adults need to be more careful about medication management, those with dementia need an even more careful approach as they have diminished cognitive reserve and risk more potential harm from medications.” 

The authors noted that since decision-making models aligned with patient priorities for older adults without dementia led to reductions in overall medication use, that may be a path forward in populations with dementia.

The study was supported by grants from the National Institute on Aging, National Institutes of Health. The authors had no competing interests to disclose. Dr. Kim disclosed no competing interests relevant to his comments.

Physicians may be missing opportunities to reduce harmful polypharmacy in elderly patients with newly diagnosed dementia, investigators for a large study of Medicare beneficiaries reported.

They found that those with an incident dementia diagnosis were somewhat more likely to initiate central nervous system–active medications and slightly more likely to discontinue cardiometabolic and anticholinergic medications, compared with controls.

According to the authors, time of diagnosis can be a potential inflexion point for deprescribing long-term medications with high safety risks, limited likelihood of benefit, or possible association with impaired cognition.

“Understanding the chronology of medication changes following a first dementia diagnosis may identify targets for deprescribing interventions to reduce preventable medication-related harms, said Timothy S. Anderson, MD, MAS, of the division of general medicine at Beth Israel Deaconess Medical Center, Boston, and colleagues in JAMA Internal Medicine.

“Our results provide a baseline to inform efforts to rethink the clinical approach to medication use at the time of a new dementia diagnosis.”

Hundreds of thousands of Americans are diagnosed annually with Alzheimer’s and related dementias, the authors pointed out, and the majority have multiple other chronic conditions. Worsening cognitive impairment may alter the risk-benefit balance of medications taken for these conditions.

Matched cohort study

The sample consisted of adults 67 years or older enrolled in traditional Medicare and Medicare Part D. Patients with an initial incident dementia diagnosis between January 2012 and December 2018 were matched with controls (as of last doctor’s office visit) based on demographics, geographic location, and baseline medication count. Data were analyzed from 2021 to June 2023.

The study included 266,675 adults with incident dementia and 266,675 controls. In both groups, 65.1% were 80 years or older (mean age, 82.2) and 67.8% were female. At baseline, patients with incident dementia were more likely than controls to use CNS-active medications (54.32% vs. 48.39%) and anticholinergic medications (17.79% vs. 15.96%) and less likely to use most cardiometabolic medications (for example, antidiabetics, 31.19% vs. 36.45%).

Immediately following the index diagnosis, the dementia cohort had greater increases in the mean number of medications used: 0.41 vs. –0.06 (95% confidence interval, 0.27-0.66) and in the proportion using CNS-active medications (absolute change, 3.44% vs. 0.79%; 95% CI, 0.85%-4.45%). The rise was because of an increased use of antipsychotics, antidepressants, and antiepileptics.

The affected cohort showed a modestly greater decline in anticholinergic medications: quarterly change in use: −0.53% vs. −0.21% (95% CI, −0.55% to −0.08%); and in most cardiometabolic medications: for example, quarterly change in antihypertensive use: –0.84% vs. –0.40% (95% CI, –0.64% to –0.25%). Still, a year post diagnosis, 75.2% of dementia patients were using five or more medications, for a 2.8% increase.

The drug classes with the steepest rate of discontinuation – such as lipid-lowering and antihypertensive medications – had low risks for adverse drug events, while higher-risk classes – such as insulins and antiplatelet and anticoagulant agents – had smaller or no reductions in use.

While the findings point to opportunities to reduce polypharmacy by deprescribing long-term medications of dubious benefit, interventions to reduce polypharmacy and inappropriate medications have been modestly successful for patients without dementia, the authors said. But the recent OPTIMIZE trial, an educational effort aimed at primary care clinicians and patients with cognitive impairment, reduced neither polypharmacy nor potentially inappropriate medications.

Luke D. Kim, MD, a geriatrician at the Cleveland Clinic in Ohio, agreed that seniors with dementia can benefit from reassessment of their pharmacologic therapies. “Older adults in general are more prone to have side effects from medications as their renal and hepatic clearance and metabolism are different and lower than those of younger individuals. But they tend to take multiple medications owing to more comorbidities,” said Dr. Kim, who was not involved in the study. “While all older adults need to be more careful about medication management, those with dementia need an even more careful approach as they have diminished cognitive reserve and risk more potential harm from medications.” 

The authors noted that since decision-making models aligned with patient priorities for older adults without dementia led to reductions in overall medication use, that may be a path forward in populations with dementia.

The study was supported by grants from the National Institute on Aging, National Institutes of Health. The authors had no competing interests to disclose. Dr. Kim disclosed no competing interests relevant to his comments.

Physicians may be missing opportunities to reduce harmful polypharmacy in elderly patients with newly diagnosed dementia, investigators for a large study of Medicare beneficiaries reported.

They found that those with an incident dementia diagnosis were somewhat more likely to initiate central nervous system–active medications and slightly more likely to discontinue cardiometabolic and anticholinergic medications, compared with controls.

According to the authors, time of diagnosis can be a potential inflexion point for deprescribing long-term medications with high safety risks, limited likelihood of benefit, or possible association with impaired cognition.

“Understanding the chronology of medication changes following a first dementia diagnosis may identify targets for deprescribing interventions to reduce preventable medication-related harms, said Timothy S. Anderson, MD, MAS, of the division of general medicine at Beth Israel Deaconess Medical Center, Boston, and colleagues in JAMA Internal Medicine.

“Our results provide a baseline to inform efforts to rethink the clinical approach to medication use at the time of a new dementia diagnosis.”

Hundreds of thousands of Americans are diagnosed annually with Alzheimer’s and related dementias, the authors pointed out, and the majority have multiple other chronic conditions. Worsening cognitive impairment may alter the risk-benefit balance of medications taken for these conditions.

Matched cohort study

The sample consisted of adults 67 years or older enrolled in traditional Medicare and Medicare Part D. Patients with an initial incident dementia diagnosis between January 2012 and December 2018 were matched with controls (as of last doctor’s office visit) based on demographics, geographic location, and baseline medication count. Data were analyzed from 2021 to June 2023.

The study included 266,675 adults with incident dementia and 266,675 controls. In both groups, 65.1% were 80 years or older (mean age, 82.2) and 67.8% were female. At baseline, patients with incident dementia were more likely than controls to use CNS-active medications (54.32% vs. 48.39%) and anticholinergic medications (17.79% vs. 15.96%) and less likely to use most cardiometabolic medications (for example, antidiabetics, 31.19% vs. 36.45%).

Immediately following the index diagnosis, the dementia cohort had greater increases in the mean number of medications used: 0.41 vs. –0.06 (95% confidence interval, 0.27-0.66) and in the proportion using CNS-active medications (absolute change, 3.44% vs. 0.79%; 95% CI, 0.85%-4.45%). The rise was because of an increased use of antipsychotics, antidepressants, and antiepileptics.

The affected cohort showed a modestly greater decline in anticholinergic medications: quarterly change in use: −0.53% vs. −0.21% (95% CI, −0.55% to −0.08%); and in most cardiometabolic medications: for example, quarterly change in antihypertensive use: –0.84% vs. –0.40% (95% CI, –0.64% to –0.25%). Still, a year post diagnosis, 75.2% of dementia patients were using five or more medications, for a 2.8% increase.

The drug classes with the steepest rate of discontinuation – such as lipid-lowering and antihypertensive medications – had low risks for adverse drug events, while higher-risk classes – such as insulins and antiplatelet and anticoagulant agents – had smaller or no reductions in use.

While the findings point to opportunities to reduce polypharmacy by deprescribing long-term medications of dubious benefit, interventions to reduce polypharmacy and inappropriate medications have been modestly successful for patients without dementia, the authors said. But the recent OPTIMIZE trial, an educational effort aimed at primary care clinicians and patients with cognitive impairment, reduced neither polypharmacy nor potentially inappropriate medications.

Luke D. Kim, MD, a geriatrician at the Cleveland Clinic in Ohio, agreed that seniors with dementia can benefit from reassessment of their pharmacologic therapies. “Older adults in general are more prone to have side effects from medications as their renal and hepatic clearance and metabolism are different and lower than those of younger individuals. But they tend to take multiple medications owing to more comorbidities,” said Dr. Kim, who was not involved in the study. “While all older adults need to be more careful about medication management, those with dementia need an even more careful approach as they have diminished cognitive reserve and risk more potential harm from medications.” 

The authors noted that since decision-making models aligned with patient priorities for older adults without dementia led to reductions in overall medication use, that may be a path forward in populations with dementia.

The study was supported by grants from the National Institute on Aging, National Institutes of Health. The authors had no competing interests to disclose. Dr. Kim disclosed no competing interests relevant to his comments.

Among patients with female pattern hair loss taking low-dose oral minoxidil (LDOM) for at least 4 months, minimal changes from baseline were observed in systolic blood pressure, diastolic blood pressure, and heart rate, results from a small retrospective analysis showed.

“Additionally, few patients experienced hair loss progression while slightly over a third experienced hair regrowth,” the study’s first author, Reese Imhof, MD, a third-year resident in the department of dermatology at Mayo Clinic, Rochester, Minn., said in an interview. The results were published online in JAAD International.

At low doses, oral minoxidil, approved as an antihypertensive over 40 years ago, has become an increasingly popular treatment for hair loss, particularly since an article about its use for hair loss was published in the New York Times in August 2022. (Oral minoxidil is not approved for treating alopecia, and is used off label for this purpose.)

To evaluate the effects of LDOM in female patients with female pattern hair loss, Dr. Imhof, along with colleagues Beija Villalpando, MD, of the department of medicine and Rochelle R. Torgerson, MD, PhD, of the department of dermatology at the Mayo Clinic, reviewed the records of 25 adult women who were evaluated for female pattern hair loss at the Mayo Clinic over a 5-year period that ended on Nov. 27, 2022. Previous studies have looked at the cardiovascular effects of treatment with oral minoxidil and impact on BP in men, but “few studies have reported on female patients receiving LDOM as monotherapy for female pattern hair loss,” the authors noted.

The mean age of the women in their study was 61 years, and they took LDOM for a mean of 6.2 months. Slightly more than half (52%) took a dose of 1.25 mg daily, while 40% took 2.5 mg daily and 8% took 0.625 mg daily.

Of the 25 patients, 10 (40%) had previously tried topical minoxidil but had discontinued it because of local side effects or challenges with adherence. Also, three patients (12%) had previously tried finasteride and spironolactone but discontinued those medications because of adverse side effects.

The researchers noted disease improvement and hair regrowth was observed in nine patients who were treated with LDOM (36%), while three patients (12%) had “unaltered disease progression.” Adverse side effects observed in the cohort included four patients with facial hypertrichosis (16%) and one patient with fluid retention/lower limb edema (4%).

The patients who developed hypertrichosis did not discontinue LDOM, but the patient who developed edema did stop treatment.

At baseline, systolic BP (SBP) ranged from 107-161 mm Hg, diastolic BP (DBP) ranged from 58-88 mm Hg, and heart rate ranged from 54-114 beats per minute. Post treatment, SBP ranged from 102-152 mm Hg, DBP ranged from 63-90 mm Hg, and heart rate ranged from 56 to 105 bpm. “It was surprising how little ambulatory blood pressure and heart rate changed after an average of 6 months of treatment,” Dr. Imhof said in an interview. “On average, SBP decreased by 2.8 mm HG while DBP decreased by 1.4 mm Hg. Heart rate increased an average of 4.4 beats per minute.”

He acknowledged certain limitations of the study, including its small sample size and lack of inclusion of patients who were being treated for hypertension with concomitant antihypertensive medications. “Some unique aspects of our study are that we focused on women, and we had a slightly older cohort than prior studies (61 years old on average) as well as exposure to higher doses of LDOM, with most patients on either 1.25 mg daily or 2.5 mg daily,” Dr. Imhof said.

The researchers reported having no relevant disclosures, and there was no funding source for the study.

Among patients with female pattern hair loss taking low-dose oral minoxidil (LDOM) for at least 4 months, minimal changes from baseline were observed in systolic blood pressure, diastolic blood pressure, and heart rate, results from a small retrospective analysis showed.

“Additionally, few patients experienced hair loss progression while slightly over a third experienced hair regrowth,” the study’s first author, Reese Imhof, MD, a third-year resident in the department of dermatology at Mayo Clinic, Rochester, Minn., said in an interview. The results were published online in JAAD International.

At low doses, oral minoxidil, approved as an antihypertensive over 40 years ago, has become an increasingly popular treatment for hair loss, particularly since an article about its use for hair loss was published in the New York Times in August 2022. (Oral minoxidil is not approved for treating alopecia, and is used off label for this purpose.)

To evaluate the effects of LDOM in female patients with female pattern hair loss, Dr. Imhof, along with colleagues Beija Villalpando, MD, of the department of medicine and Rochelle R. Torgerson, MD, PhD, of the department of dermatology at the Mayo Clinic, reviewed the records of 25 adult women who were evaluated for female pattern hair loss at the Mayo Clinic over a 5-year period that ended on Nov. 27, 2022. Previous studies have looked at the cardiovascular effects of treatment with oral minoxidil and impact on BP in men, but “few studies have reported on female patients receiving LDOM as monotherapy for female pattern hair loss,” the authors noted.

The mean age of the women in their study was 61 years, and they took LDOM for a mean of 6.2 months. Slightly more than half (52%) took a dose of 1.25 mg daily, while 40% took 2.5 mg daily and 8% took 0.625 mg daily.

Of the 25 patients, 10 (40%) had previously tried topical minoxidil but had discontinued it because of local side effects or challenges with adherence. Also, three patients (12%) had previously tried finasteride and spironolactone but discontinued those medications because of adverse side effects.

The researchers noted disease improvement and hair regrowth was observed in nine patients who were treated with LDOM (36%), while three patients (12%) had “unaltered disease progression.” Adverse side effects observed in the cohort included four patients with facial hypertrichosis (16%) and one patient with fluid retention/lower limb edema (4%).

The patients who developed hypertrichosis did not discontinue LDOM, but the patient who developed edema did stop treatment.

At baseline, systolic BP (SBP) ranged from 107-161 mm Hg, diastolic BP (DBP) ranged from 58-88 mm Hg, and heart rate ranged from 54-114 beats per minute. Post treatment, SBP ranged from 102-152 mm Hg, DBP ranged from 63-90 mm Hg, and heart rate ranged from 56 to 105 bpm. “It was surprising how little ambulatory blood pressure and heart rate changed after an average of 6 months of treatment,” Dr. Imhof said in an interview. “On average, SBP decreased by 2.8 mm HG while DBP decreased by 1.4 mm Hg. Heart rate increased an average of 4.4 beats per minute.”

He acknowledged certain limitations of the study, including its small sample size and lack of inclusion of patients who were being treated for hypertension with concomitant antihypertensive medications. “Some unique aspects of our study are that we focused on women, and we had a slightly older cohort than prior studies (61 years old on average) as well as exposure to higher doses of LDOM, with most patients on either 1.25 mg daily or 2.5 mg daily,” Dr. Imhof said.

The researchers reported having no relevant disclosures, and there was no funding source for the study.

Among patients with female pattern hair loss taking low-dose oral minoxidil (LDOM) for at least 4 months, minimal changes from baseline were observed in systolic blood pressure, diastolic blood pressure, and heart rate, results from a small retrospective analysis showed.

“Additionally, few patients experienced hair loss progression while slightly over a third experienced hair regrowth,” the study’s first author, Reese Imhof, MD, a third-year resident in the department of dermatology at Mayo Clinic, Rochester, Minn., said in an interview. The results were published online in JAAD International.

At low doses, oral minoxidil, approved as an antihypertensive over 40 years ago, has become an increasingly popular treatment for hair loss, particularly since an article about its use for hair loss was published in the New York Times in August 2022. (Oral minoxidil is not approved for treating alopecia, and is used off label for this purpose.)

To evaluate the effects of LDOM in female patients with female pattern hair loss, Dr. Imhof, along with colleagues Beija Villalpando, MD, of the department of medicine and Rochelle R. Torgerson, MD, PhD, of the department of dermatology at the Mayo Clinic, reviewed the records of 25 adult women who were evaluated for female pattern hair loss at the Mayo Clinic over a 5-year period that ended on Nov. 27, 2022. Previous studies have looked at the cardiovascular effects of treatment with oral minoxidil and impact on BP in men, but “few studies have reported on female patients receiving LDOM as monotherapy for female pattern hair loss,” the authors noted.

The mean age of the women in their study was 61 years, and they took LDOM for a mean of 6.2 months. Slightly more than half (52%) took a dose of 1.25 mg daily, while 40% took 2.5 mg daily and 8% took 0.625 mg daily.

Of the 25 patients, 10 (40%) had previously tried topical minoxidil but had discontinued it because of local side effects or challenges with adherence. Also, three patients (12%) had previously tried finasteride and spironolactone but discontinued those medications because of adverse side effects.

The researchers noted disease improvement and hair regrowth was observed in nine patients who were treated with LDOM (36%), while three patients (12%) had “unaltered disease progression.” Adverse side effects observed in the cohort included four patients with facial hypertrichosis (16%) and one patient with fluid retention/lower limb edema (4%).

The patients who developed hypertrichosis did not discontinue LDOM, but the patient who developed edema did stop treatment.

At baseline, systolic BP (SBP) ranged from 107-161 mm Hg, diastolic BP (DBP) ranged from 58-88 mm Hg, and heart rate ranged from 54-114 beats per minute. Post treatment, SBP ranged from 102-152 mm Hg, DBP ranged from 63-90 mm Hg, and heart rate ranged from 56 to 105 bpm. “It was surprising how little ambulatory blood pressure and heart rate changed after an average of 6 months of treatment,” Dr. Imhof said in an interview. “On average, SBP decreased by 2.8 mm HG while DBP decreased by 1.4 mm Hg. Heart rate increased an average of 4.4 beats per minute.”

He acknowledged certain limitations of the study, including its small sample size and lack of inclusion of patients who were being treated for hypertension with concomitant antihypertensive medications. “Some unique aspects of our study are that we focused on women, and we had a slightly older cohort than prior studies (61 years old on average) as well as exposure to higher doses of LDOM, with most patients on either 1.25 mg daily or 2.5 mg daily,” Dr. Imhof said.

The researchers reported having no relevant disclosures, and there was no funding source for the study.

If you’re looking to grow hair, you might just have a solution in your kitchen cabinet – if TikTok and some dermatologists are correct.

A small study published in 2015 suggested that rosemary oil may help regrow hair in people with androgenetic alopecia. The herb might also protect hair from the sun, pollution, and other environmental elements, according to an article in Insider.

The study published in Skinmed found that rosemary oil was similar to the effectiveness of minoxidil for regrowing hair in men with androgenetic alopecia. The scalp was also less itchy after 3-6 months of use.

The study included only men.

Still, dermatologist Shilpi Khetarpal, MD, told the Cleveland Clinic that it seems to work.

“The study really prompted people to look at rosemary oil for hair growth,” she said. “It became much more common in over-the-counter products after that, too.”

The Cleveland Clinic also reports that rosemary oil might help against dandruff and premature graying.

Dr. Khetarpal suggested massaging rosemary oil into the scalp, letting it soak overnight, and then washing it out. This should be done two or three times a week. 

She also noted that only a few drops of rosemary oil are needed, and that the focus should be on the scalp rather than the hair, which rosemary oil makes look greasy.

It may take 6 months for “meaningful improvement,” Dr. Khetarpal said.

Meanwhile, TikTok users love hyping the oil’s hair care qualities. On the social media platform, videos with the hashtag #rosemaryoil have been viewed more than 2 billion times.
 

A version of this article appeared on WebMD.com.

If you’re looking to grow hair, you might just have a solution in your kitchen cabinet – if TikTok and some dermatologists are correct.

A small study published in 2015 suggested that rosemary oil may help regrow hair in people with androgenetic alopecia. The herb might also protect hair from the sun, pollution, and other environmental elements, according to an article in Insider.

The study published in Skinmed found that rosemary oil was similar to the effectiveness of minoxidil for regrowing hair in men with androgenetic alopecia. The scalp was also less itchy after 3-6 months of use.

The study included only men.

Still, dermatologist Shilpi Khetarpal, MD, told the Cleveland Clinic that it seems to work.

“The study really prompted people to look at rosemary oil for hair growth,” she said. “It became much more common in over-the-counter products after that, too.”

The Cleveland Clinic also reports that rosemary oil might help against dandruff and premature graying.

Dr. Khetarpal suggested massaging rosemary oil into the scalp, letting it soak overnight, and then washing it out. This should be done two or three times a week. 

She also noted that only a few drops of rosemary oil are needed, and that the focus should be on the scalp rather than the hair, which rosemary oil makes look greasy.

It may take 6 months for “meaningful improvement,” Dr. Khetarpal said.

Meanwhile, TikTok users love hyping the oil’s hair care qualities. On the social media platform, videos with the hashtag #rosemaryoil have been viewed more than 2 billion times.
 

A version of this article appeared on WebMD.com.

If you’re looking to grow hair, you might just have a solution in your kitchen cabinet – if TikTok and some dermatologists are correct.

A small study published in 2015 suggested that rosemary oil may help regrow hair in people with androgenetic alopecia. The herb might also protect hair from the sun, pollution, and other environmental elements, according to an article in Insider.

The study published in Skinmed found that rosemary oil was similar to the effectiveness of minoxidil for regrowing hair in men with androgenetic alopecia. The scalp was also less itchy after 3-6 months of use.

The study included only men.

Still, dermatologist Shilpi Khetarpal, MD, told the Cleveland Clinic that it seems to work.

“The study really prompted people to look at rosemary oil for hair growth,” she said. “It became much more common in over-the-counter products after that, too.”

The Cleveland Clinic also reports that rosemary oil might help against dandruff and premature graying.

Dr. Khetarpal suggested massaging rosemary oil into the scalp, letting it soak overnight, and then washing it out. This should be done two or three times a week. 

She also noted that only a few drops of rosemary oil are needed, and that the focus should be on the scalp rather than the hair, which rosemary oil makes look greasy.

It may take 6 months for “meaningful improvement,” Dr. Khetarpal said.

Meanwhile, TikTok users love hyping the oil’s hair care qualities. On the social media platform, videos with the hashtag #rosemaryoil have been viewed more than 2 billion times.
 

A version of this article appeared on WebMD.com.

Adding oral piroxicam to oral levonorgestrel significantly improved the efficacy of emergency contraception, based on data from 860 women.

Oral hormonal emergency contraception (EC) is the most widely used EC method worldwide, but the two currently available drugs, levonorgestrel and ulipristal acetate (UPA), are not effective when given after ovulation, wrote Raymond Hang Wun Li, MD, of the University of Hong Kong, and colleagues. Previous studies suggest that cyclo-oxygenase (COX) inhibitors may disrupt follicular rupture and prevent ovulation, but data on their use in combination with current oral ECs are lacking, the researchers said.

In a study published in The Lancet, the researchers randomized 430 women to receive a single oral dose of 1.5 mg levonorgestrel plus 40 mg of the COX-2 inhibitor piroxicam or 1.5 mg levonorgestrel plus a placebo. The study participants were women aged 18 years and older who requested EC within 72 hours of unprotected sex and who had regular menstrual cycles between 24 and 42 days long. The median age of the participants was 30 years; 97% were Chinese. The median time from intercourse to treatment was 18 hours for both groups.

The primary outcome was the percentage of pregnancies prevented, based on pregnancy status 1-2 weeks after treatment.

One pregnancy occurred in the piroxicam group, compared with seven pregnancies in the placebo group, which translated to a significant difference in the percentage of pregnancies prevented (94.7% vs. 63.4%, P < .0001).

No trend toward increased failure rates appeared based on the time elapsed between intercourse and EC use in either group, and no differences appeared in the return or delay of subsequent menstrual periods between the groups.

The most common adverse events (reported by more than 5% of participants in both groups) included fatigue or weakness, nausea, lower abdominal pain, dizziness, and headache.

The choice of piroxicam as the COX inhibitor in conjunction with levonorgestrel for the current study had several potential advantages, the researchers wrote in their discussion. These advantages include the widespread availability and long-acting characteristics of piroxicam, which is also true of levonorgestrel, they said.

The findings were limited by several factors including the generalizability to other settings and populations, the researchers noted. The efficacy of the levonorgestrel/piroxicam combination in women with a body mass index greater than 26 kg/m² may be lower, but the current study population did not have enough women in this category to measure the potential effect, they said. The study also did not examine the effect of piroxicam in combination with ulipristal acetate.

However, the results are the first known to demonstrate the improved effectiveness of oral piroxicam coadministered with oral levonorgestrel for EC, they said.

“The strength of this recommendation and changes in clinical guidelines may be determined upon demonstration of reproducible results in further studies,” they added.
 

Pill combination shows potential and practicality

Oral emergency contraception on demand is an unmet need on a global level, Erica P. Cahill, MD, of the department of obstetrics and gynecology and division of family planning services at Stanford (Calif.) University, wrote in an accompanying editorial.

Dr. Cahill noted the longer half-life of piroxicam compared with other COX-2 inhibitors, which made it a practical choice. Although the study was not powered to evaluate secondary outcomes, bleeding patterns consistent with use of EC pills were observed. Documentation of these patterns is worthwhile, Dr. Cahill said, “because people using emergency contraceptive pills might also be using fertility awareness methods and need to know when they can be certain they are not pregnant.”

Overall, the study supports the addition of 40 mg piroxicam to 1.5 mg levonorgestrel as emergency contraception, said Dr. Cahill. Future studies can build on the current findings by evaluating repeat dosing of the piroxicam/levonorgestrel combination and by evaluating the combination of COX-2 inhibitors and ulipristal acetate to prevent pregnancy, she said.

The study received no outside funding. The researchers and Dr. Cahill had no financial conflicts to disclose.

Adding oral piroxicam to oral levonorgestrel significantly improved the efficacy of emergency contraception, based on data from 860 women.

Oral hormonal emergency contraception (EC) is the most widely used EC method worldwide, but the two currently available drugs, levonorgestrel and ulipristal acetate (UPA), are not effective when given after ovulation, wrote Raymond Hang Wun Li, MD, of the University of Hong Kong, and colleagues. Previous studies suggest that cyclo-oxygenase (COX) inhibitors may disrupt follicular rupture and prevent ovulation, but data on their use in combination with current oral ECs are lacking, the researchers said.

In a study published in The Lancet, the researchers randomized 430 women to receive a single oral dose of 1.5 mg levonorgestrel plus 40 mg of the COX-2 inhibitor piroxicam or 1.5 mg levonorgestrel plus a placebo. The study participants were women aged 18 years and older who requested EC within 72 hours of unprotected sex and who had regular menstrual cycles between 24 and 42 days long. The median age of the participants was 30 years; 97% were Chinese. The median time from intercourse to treatment was 18 hours for both groups.

The primary outcome was the percentage of pregnancies prevented, based on pregnancy status 1-2 weeks after treatment.

One pregnancy occurred in the piroxicam group, compared with seven pregnancies in the placebo group, which translated to a significant difference in the percentage of pregnancies prevented (94.7% vs. 63.4%, P < .0001).

No trend toward increased failure rates appeared based on the time elapsed between intercourse and EC use in either group, and no differences appeared in the return or delay of subsequent menstrual periods between the groups.

The most common adverse events (reported by more than 5% of participants in both groups) included fatigue or weakness, nausea, lower abdominal pain, dizziness, and headache.

The choice of piroxicam as the COX inhibitor in conjunction with levonorgestrel for the current study had several potential advantages, the researchers wrote in their discussion. These advantages include the widespread availability and long-acting characteristics of piroxicam, which is also true of levonorgestrel, they said.

The findings were limited by several factors including the generalizability to other settings and populations, the researchers noted. The efficacy of the levonorgestrel/piroxicam combination in women with a body mass index greater than 26 kg/m² may be lower, but the current study population did not have enough women in this category to measure the potential effect, they said. The study also did not examine the effect of piroxicam in combination with ulipristal acetate.

However, the results are the first known to demonstrate the improved effectiveness of oral piroxicam coadministered with oral levonorgestrel for EC, they said.

“The strength of this recommendation and changes in clinical guidelines may be determined upon demonstration of reproducible results in further studies,” they added.
 

Pill combination shows potential and practicality

Oral emergency contraception on demand is an unmet need on a global level, Erica P. Cahill, MD, of the department of obstetrics and gynecology and division of family planning services at Stanford (Calif.) University, wrote in an accompanying editorial.

Dr. Cahill noted the longer half-life of piroxicam compared with other COX-2 inhibitors, which made it a practical choice. Although the study was not powered to evaluate secondary outcomes, bleeding patterns consistent with use of EC pills were observed. Documentation of these patterns is worthwhile, Dr. Cahill said, “because people using emergency contraceptive pills might also be using fertility awareness methods and need to know when they can be certain they are not pregnant.”

Overall, the study supports the addition of 40 mg piroxicam to 1.5 mg levonorgestrel as emergency contraception, said Dr. Cahill. Future studies can build on the current findings by evaluating repeat dosing of the piroxicam/levonorgestrel combination and by evaluating the combination of COX-2 inhibitors and ulipristal acetate to prevent pregnancy, she said.

The study received no outside funding. The researchers and Dr. Cahill had no financial conflicts to disclose.

Adding oral piroxicam to oral levonorgestrel significantly improved the efficacy of emergency contraception, based on data from 860 women.

Oral hormonal emergency contraception (EC) is the most widely used EC method worldwide, but the two currently available drugs, levonorgestrel and ulipristal acetate (UPA), are not effective when given after ovulation, wrote Raymond Hang Wun Li, MD, of the University of Hong Kong, and colleagues. Previous studies suggest that cyclo-oxygenase (COX) inhibitors may disrupt follicular rupture and prevent ovulation, but data on their use in combination with current oral ECs are lacking, the researchers said.

In a study published in The Lancet, the researchers randomized 430 women to receive a single oral dose of 1.5 mg levonorgestrel plus 40 mg of the COX-2 inhibitor piroxicam or 1.5 mg levonorgestrel plus a placebo. The study participants were women aged 18 years and older who requested EC within 72 hours of unprotected sex and who had regular menstrual cycles between 24 and 42 days long. The median age of the participants was 30 years; 97% were Chinese. The median time from intercourse to treatment was 18 hours for both groups.

The primary outcome was the percentage of pregnancies prevented, based on pregnancy status 1-2 weeks after treatment.

One pregnancy occurred in the piroxicam group, compared with seven pregnancies in the placebo group, which translated to a significant difference in the percentage of pregnancies prevented (94.7% vs. 63.4%, P < .0001).

No trend toward increased failure rates appeared based on the time elapsed between intercourse and EC use in either group, and no differences appeared in the return or delay of subsequent menstrual periods between the groups.

The most common adverse events (reported by more than 5% of participants in both groups) included fatigue or weakness, nausea, lower abdominal pain, dizziness, and headache.

The choice of piroxicam as the COX inhibitor in conjunction with levonorgestrel for the current study had several potential advantages, the researchers wrote in their discussion. These advantages include the widespread availability and long-acting characteristics of piroxicam, which is also true of levonorgestrel, they said.

The findings were limited by several factors including the generalizability to other settings and populations, the researchers noted. The efficacy of the levonorgestrel/piroxicam combination in women with a body mass index greater than 26 kg/m² may be lower, but the current study population did not have enough women in this category to measure the potential effect, they said. The study also did not examine the effect of piroxicam in combination with ulipristal acetate.

However, the results are the first known to demonstrate the improved effectiveness of oral piroxicam coadministered with oral levonorgestrel for EC, they said.

“The strength of this recommendation and changes in clinical guidelines may be determined upon demonstration of reproducible results in further studies,” they added.
 

Pill combination shows potential and practicality

Oral emergency contraception on demand is an unmet need on a global level, Erica P. Cahill, MD, of the department of obstetrics and gynecology and division of family planning services at Stanford (Calif.) University, wrote in an accompanying editorial.

Dr. Cahill noted the longer half-life of piroxicam compared with other COX-2 inhibitors, which made it a practical choice. Although the study was not powered to evaluate secondary outcomes, bleeding patterns consistent with use of EC pills were observed. Documentation of these patterns is worthwhile, Dr. Cahill said, “because people using emergency contraceptive pills might also be using fertility awareness methods and need to know when they can be certain they are not pregnant.”

Overall, the study supports the addition of 40 mg piroxicam to 1.5 mg levonorgestrel as emergency contraception, said Dr. Cahill. Future studies can build on the current findings by evaluating repeat dosing of the piroxicam/levonorgestrel combination and by evaluating the combination of COX-2 inhibitors and ulipristal acetate to prevent pregnancy, she said.

The study received no outside funding. The researchers and Dr. Cahill had no financial conflicts to disclose.

Treatment with medication often used for attention-deficit/hyperactivity disorder (ADHD) was associated with lower risk of psychiatric hospitalization, all-cause hospitalization, or death in adults with borderline personality disorder, based on data from more than 17,000 individuals.

Although most patients with borderline personality disorder (BPD) receive psychopharmacological treatment, clinical guidance and outcomes data for specific medication use in these patients are lacking, wrote Johannes Lieslehto, MD, PhD, of the University of Eastern Finland, Niuvankuja, and colleagues.

Dr. Lieslehto

In a study published in Acta Psychiatrica Scandinavica , the researchers – using national databases in Sweden – identified 17,532 adults with BPD who were treated with medications between 2006 and 2018.

Medications included benzodiazepines, antipsychotics, and antidepressants, as well as medications often used for ADHD: clozapine, lisdexamphetamine, bupropion, and methylphenidate. The mean age of the study population was 29.8 years and 2,649 were men.

The primary outcomes were psychiatric hospitalization (which served as an indication of treatment failure), all-cause hospitalization, or death.

Overall, treatment with benzodiazepines, antipsychotics, and antidepressants was associated with increased risk of psychiatric rehospitalization, with hazard ratios of 1.38, 1.19, and 1.18, respectively, and with increased risk of all-cause hospitalization or death (HR 1.37, HR 1.21, HR 1.17, respectively).

By contrast, treatment with ADHD medication was associated with decreased risk of psychiatric hospitalization (HR = 0.88), as well as a decreased risk of all-cause hospitalization or death (HR = 0.86).

Specifically, clozapine, lisdexamphetamine, bupropion, and methylphenidate were associated with decreased risk of psychiatric rehospitalization, with hazard ratios of 0.54, 0.79, 0.84, and 0.90, respectively.

Treatment with mood stabilizers had no significant impact on outcomes.

BPD patients treated with ADHD medications also may exhibit ADHD symptoms, the researchers wrote in their discussion. However, “Although BPD and ADHD partially overlap in symptoms such as impulsivity and emotion dysregulation, previous efforts to investigate the efficacy of ADHD medication treatment in BPD are scarce,” and randomized, controlled trials are needed to determine whether these medications should be given to BPD patients without comorbid ADHD symptoms, they said.

The findings were limited by several factors including the lack of clinical parameters on symptom severity, quality of life, and level of function, and premature prescribing of medication (protopathic bias) may have affected the results, the researchers noted.

The results were strengthened by the large sample size and long follow-up, which increases the generalizability to real-world patients, and suggest that many pharmacological treatments for BPD may not improve outcomes, the researchers said. However, “even in the presence of possible protopathic bias, treatment with lisdexamphetamine, bupropion, methylphenidate, and clozapine was associated with improved outcomes, encouraging further research on these treatments,” they said.

The study was supported by the Finnish Ministry of Social Affairs and Health and the Academy of Finland. Dr. Lieslehto had no financial conflicts to disclose.

Treatment with medication often used for attention-deficit/hyperactivity disorder (ADHD) was associated with lower risk of psychiatric hospitalization, all-cause hospitalization, or death in adults with borderline personality disorder, based on data from more than 17,000 individuals.

Although most patients with borderline personality disorder (BPD) receive psychopharmacological treatment, clinical guidance and outcomes data for specific medication use in these patients are lacking, wrote Johannes Lieslehto, MD, PhD, of the University of Eastern Finland, Niuvankuja, and colleagues.

Dr. Lieslehto

In a study published in Acta Psychiatrica Scandinavica , the researchers – using national databases in Sweden – identified 17,532 adults with BPD who were treated with medications between 2006 and 2018.

Medications included benzodiazepines, antipsychotics, and antidepressants, as well as medications often used for ADHD: clozapine, lisdexamphetamine, bupropion, and methylphenidate. The mean age of the study population was 29.8 years and 2,649 were men.

The primary outcomes were psychiatric hospitalization (which served as an indication of treatment failure), all-cause hospitalization, or death.

Overall, treatment with benzodiazepines, antipsychotics, and antidepressants was associated with increased risk of psychiatric rehospitalization, with hazard ratios of 1.38, 1.19, and 1.18, respectively, and with increased risk of all-cause hospitalization or death (HR 1.37, HR 1.21, HR 1.17, respectively).

By contrast, treatment with ADHD medication was associated with decreased risk of psychiatric hospitalization (HR = 0.88), as well as a decreased risk of all-cause hospitalization or death (HR = 0.86).

Specifically, clozapine, lisdexamphetamine, bupropion, and methylphenidate were associated with decreased risk of psychiatric rehospitalization, with hazard ratios of 0.54, 0.79, 0.84, and 0.90, respectively.

Treatment with mood stabilizers had no significant impact on outcomes.

BPD patients treated with ADHD medications also may exhibit ADHD symptoms, the researchers wrote in their discussion. However, “Although BPD and ADHD partially overlap in symptoms such as impulsivity and emotion dysregulation, previous efforts to investigate the efficacy of ADHD medication treatment in BPD are scarce,” and randomized, controlled trials are needed to determine whether these medications should be given to BPD patients without comorbid ADHD symptoms, they said.

The findings were limited by several factors including the lack of clinical parameters on symptom severity, quality of life, and level of function, and premature prescribing of medication (protopathic bias) may have affected the results, the researchers noted.

The results were strengthened by the large sample size and long follow-up, which increases the generalizability to real-world patients, and suggest that many pharmacological treatments for BPD may not improve outcomes, the researchers said. However, “even in the presence of possible protopathic bias, treatment with lisdexamphetamine, bupropion, methylphenidate, and clozapine was associated with improved outcomes, encouraging further research on these treatments,” they said.

The study was supported by the Finnish Ministry of Social Affairs and Health and the Academy of Finland. Dr. Lieslehto had no financial conflicts to disclose.

Treatment with medication often used for attention-deficit/hyperactivity disorder (ADHD) was associated with lower risk of psychiatric hospitalization, all-cause hospitalization, or death in adults with borderline personality disorder, based on data from more than 17,000 individuals.

Although most patients with borderline personality disorder (BPD) receive psychopharmacological treatment, clinical guidance and outcomes data for specific medication use in these patients are lacking, wrote Johannes Lieslehto, MD, PhD, of the University of Eastern Finland, Niuvankuja, and colleagues.

Dr. Lieslehto

In a study published in Acta Psychiatrica Scandinavica , the researchers – using national databases in Sweden – identified 17,532 adults with BPD who were treated with medications between 2006 and 2018.

Medications included benzodiazepines, antipsychotics, and antidepressants, as well as medications often used for ADHD: clozapine, lisdexamphetamine, bupropion, and methylphenidate. The mean age of the study population was 29.8 years and 2,649 were men.

The primary outcomes were psychiatric hospitalization (which served as an indication of treatment failure), all-cause hospitalization, or death.

Overall, treatment with benzodiazepines, antipsychotics, and antidepressants was associated with increased risk of psychiatric rehospitalization, with hazard ratios of 1.38, 1.19, and 1.18, respectively, and with increased risk of all-cause hospitalization or death (HR 1.37, HR 1.21, HR 1.17, respectively).

By contrast, treatment with ADHD medication was associated with decreased risk of psychiatric hospitalization (HR = 0.88), as well as a decreased risk of all-cause hospitalization or death (HR = 0.86).

Specifically, clozapine, lisdexamphetamine, bupropion, and methylphenidate were associated with decreased risk of psychiatric rehospitalization, with hazard ratios of 0.54, 0.79, 0.84, and 0.90, respectively.

Treatment with mood stabilizers had no significant impact on outcomes.

BPD patients treated with ADHD medications also may exhibit ADHD symptoms, the researchers wrote in their discussion. However, “Although BPD and ADHD partially overlap in symptoms such as impulsivity and emotion dysregulation, previous efforts to investigate the efficacy of ADHD medication treatment in BPD are scarce,” and randomized, controlled trials are needed to determine whether these medications should be given to BPD patients without comorbid ADHD symptoms, they said.

The findings were limited by several factors including the lack of clinical parameters on symptom severity, quality of life, and level of function, and premature prescribing of medication (protopathic bias) may have affected the results, the researchers noted.

The results were strengthened by the large sample size and long follow-up, which increases the generalizability to real-world patients, and suggest that many pharmacological treatments for BPD may not improve outcomes, the researchers said. However, “even in the presence of possible protopathic bias, treatment with lisdexamphetamine, bupropion, methylphenidate, and clozapine was associated with improved outcomes, encouraging further research on these treatments,” they said.

The study was supported by the Finnish Ministry of Social Affairs and Health and the Academy of Finland. Dr. Lieslehto had no financial conflicts to disclose.

Dual antiplatelet therapy (DAPT) consisting of aspirin plus a P2Y₁₂ inhibitor has been the standard of care to prevent thrombotic events in patients with acute coronary syndrome (ACS) undergoing percutaneous coronary intervention (PCI).

A new pilot study suggests that aspirin can be discontinued on the day after the PCI, and colchicine, an anti-inflammatory agent, could be added to reduce the risk for ischemic events in these patients, while mitigating the increased bleeding risk associated with aspirin.

Investigators conducted a pilot trial in ACS patients treated with drug-eluting stents (DES) who received low-dose colchicine the day after PCI, together with P2Y₁₂ inhibitor (ticagrelor or prasugrel) maintenance therapy. Aspirin use was discontinued.

At 3 months, only 1% of the patients experienced stent thrombosis, and only 1 patient showed high platelet reactivity. Moreover, at 1 month, high-sensitivity C-reactive protein (hs-CRP) and platelet reactivity both decreased, pointing to reduced inflammation.

“In ACS patients undergoing PCI, it is feasible to discontinue aspirin therapy and administer low-dose colchicine on the day after PCI in addition to ticagrelor or prasugrel P2Y₁₂ inhibitors,” write Seung-Yul Lee, MD, CHA Bundang Medical Center, CHA University, Seongnam, South Korea, and colleagues. “This approach is associated with favorable platelet function and inflammatory profiles.”

The study was published online in JACC: Cardiovascular Interventions.
 

Safety without compromised efficacy

The U.S. Food and Drug Administration recently approved colchicine 0.5-mg tablets (Lodoco, Agepha Pharma) as the first anti-inflammatory drug shown to reduce the risk for myocardial infarction, stroke, coronary revascularization, and cardiovascular death in adult patients with either established atherosclerotic disease or multiple risk factors for cardiovascular disease. It targets residual inflammation as an underlying cause of cardiovascular events.

Patients after PCI are generally treated using DAPT, but given the risk for increased bleeding associated with aspirin – especially when used long-term – there is a “need to identify strategies associated with a more favorable safety profile without compromising efficacy,” the authors write.

Previous research has yielded mixed results in terms of the discontinuation of aspirin therapy after 1-3 months and maintenance on P2Y₁₂ inhibitor monotherapy. But one trial found colchicine to be effective in reducing recurrent ischemia, and its benefits may be more beneficial with early initiation in the hospital.

In this new study, researchers tested a “strategy that substitutes aspirin with colchicine during the acute phase to maximize the treatment effect of reducing recurrent ischemia and bleeding,” they write. The Mono Antiplatelet and Colchicine Therapy (MACT) single-arm, open-label proof-of-concept study was designed to investigate this approach.

The researchers studied 200 patients with non–ST-segment elevation ACS and ST-segment elevation myocardial infarction (STEMI) who underwent PCI with DES (mean [SD] age, 61.4 [10.7] years; 90% male; 100% of Asian ethnicity), who were receiving either ticagrelor or prasugrel plus a loading dose of aspirin.

On the day after PCI, aspirin was discontinued, and low-dose colchicine (0.6 mg once daily) was administered in addition to the P2Y₁₂ inhibitor. In the case of staged PCI, it was performed under the maintenance of colchicine and ticagrelor or prasugrel.

No other antiplatelet or anticoagulant agents were permitted.

Patients underwent platelet function testing using the VerifyNow P2Y₁₂ assay before discharge. Levels of hs-CRP were measured at admission, at 24 and 48 hours after PCI, and at 1-month follow-up. Clinical follow-up was performed at 1 and at 3 months.

The primary outcome was stent thrombosis within 3 months of follow-up. Secondary outcomes included all-cause mortality, MI, revascularization, major bleeding, a composite of cardiac death, target vessel MI, or target lesion revascularization, P2Y₁₂ reaction units (PRUs), and change in hs-CRP levels between 24 hours post-PCI and 1-month follow-up.
 

The role of inflammation

Of the original 200 patients, 190 completed the full protocol and were available for follow-up.

The primary outcome occurred in only two patients. It turned out that one of the patients had not been adherent with antiplatelet medications.

“Although bleeding occurred in 36 patients, major bleeding occurred in only 1 patient,” the authors report.

The level of platelet reactivity at discharge was 27 ± 42 PRUs. Most patients (91%) met the criteria for low platelet reactivity, while only 0.5% met the criteria for high platelet reactivity. Platelet reactivity was similar, regardless of which P2Y₁₂ inhibitor (ticagrelor or prasugrel) the patients were taking.

In all patients, the level of inflammation was “reduced considerably” over time: After 1 month, the hs-CRP level decreased from 6.1 mg/L (interquartile range [IQR], 2.6-15.9 mg/L) at 24 hours after PCI to 0.6 mg/L (IQR, 0.4-1.2 mg/L; P < .001).

The prevalence of high-inflammation criteria, defined as hs-CRP ≥ 2 mg/L, decreased significantly, from 81.8% at 24 hours after PCI to 11.8% at 1 month (P < .001).

Major bleeding was rare, they report, with a 3-month incidence of 0.5%.

“Inflammation plays a fundamental role in the development and progression of the atherothrombotic process,” the authors explain. A series of factors also trigger “an intense inflammatory response” in the acute phase of MI, which may lead to adverse myocardial remodeling. In the present study, inflammatory levels were rapidly reduced.

They noted several limitations. For example, all enrolled patients were Asian and were at relatively low bleeding and ischemic risk. “Although ticagrelor or prasugrel is effective regardless of ethnicity, clinical data supporting this de-escalation strategy are limited,” they state. Additionally, there was no control group for comparison.

The findings warrant further investigation, they conclude.
 

Promising but preliminary

Commenting for this news organization, Francesco Costa, MD, PhD, interventional cardiologist and assistant professor, University of Messina, Sicily, Italy, said he thinks it’s “too early for extensive clinical translation of these findings.”

Rather, larger and more extensive randomized trials are “on their way to give more precise estimates regarding the risks and benefits of early aspirin withdrawal in ACS.”

However, added Dr. Costa, who was not involved with the current research, “in this setting, adding colchicine early looks very promising to mitigate potential thrombotic risk without increasing bleeding risk.”

In the meantime, the study “provides novel insights on early aspirin withdrawal and P2Y₁₂ monotherapy in an unselected population, including [those with] STEMI,” said Dr. Costa, also the coauthor of an accompanying editorial. The findings “could be of particular interest for those patients at extremely high bleeding risk or who are truly intolerant to aspirin, a scenario in which options are limited.”

This study was supported by the Cardiovascular Research Center, Seoul, South Korea. Dr. Lee reports no relevant financial relationships. The other authors’ disclosures are listed on the original paper. Dr. Costa has served on an advisory board for AstraZeneca and has received speaker fees from Chiesi Farmaceutici. His coauthor reports no relevant financial relationships.

A version of this article appeared on Medscape.com.

Dual antiplatelet therapy (DAPT) consisting of aspirin plus a P2Y₁₂ inhibitor has been the standard of care to prevent thrombotic events in patients with acute coronary syndrome (ACS) undergoing percutaneous coronary intervention (PCI).

A new pilot study suggests that aspirin can be discontinued on the day after the PCI, and colchicine, an anti-inflammatory agent, could be added to reduce the risk for ischemic events in these patients, while mitigating the increased bleeding risk associated with aspirin.

Investigators conducted a pilot trial in ACS patients treated with drug-eluting stents (DES) who received low-dose colchicine the day after PCI, together with P2Y₁₂ inhibitor (ticagrelor or prasugrel) maintenance therapy. Aspirin use was discontinued.

At 3 months, only 1% of the patients experienced stent thrombosis, and only 1 patient showed high platelet reactivity. Moreover, at 1 month, high-sensitivity C-reactive protein (hs-CRP) and platelet reactivity both decreased, pointing to reduced inflammation.

“In ACS patients undergoing PCI, it is feasible to discontinue aspirin therapy and administer low-dose colchicine on the day after PCI in addition to ticagrelor or prasugrel P2Y₁₂ inhibitors,” write Seung-Yul Lee, MD, CHA Bundang Medical Center, CHA University, Seongnam, South Korea, and colleagues. “This approach is associated with favorable platelet function and inflammatory profiles.”

The study was published online in JACC: Cardiovascular Interventions.
 

Safety without compromised efficacy

The U.S. Food and Drug Administration recently approved colchicine 0.5-mg tablets (Lodoco, Agepha Pharma) as the first anti-inflammatory drug shown to reduce the risk for myocardial infarction, stroke, coronary revascularization, and cardiovascular death in adult patients with either established atherosclerotic disease or multiple risk factors for cardiovascular disease. It targets residual inflammation as an underlying cause of cardiovascular events.

Patients after PCI are generally treated using DAPT, but given the risk for increased bleeding associated with aspirin – especially when used long-term – there is a “need to identify strategies associated with a more favorable safety profile without compromising efficacy,” the authors write.

Previous research has yielded mixed results in terms of the discontinuation of aspirin therapy after 1-3 months and maintenance on P2Y₁₂ inhibitor monotherapy. But one trial found colchicine to be effective in reducing recurrent ischemia, and its benefits may be more beneficial with early initiation in the hospital.

In this new study, researchers tested a “strategy that substitutes aspirin with colchicine during the acute phase to maximize the treatment effect of reducing recurrent ischemia and bleeding,” they write. The Mono Antiplatelet and Colchicine Therapy (MACT) single-arm, open-label proof-of-concept study was designed to investigate this approach.

The researchers studied 200 patients with non–ST-segment elevation ACS and ST-segment elevation myocardial infarction (STEMI) who underwent PCI with DES (mean [SD] age, 61.4 [10.7] years; 90% male; 100% of Asian ethnicity), who were receiving either ticagrelor or prasugrel plus a loading dose of aspirin.

On the day after PCI, aspirin was discontinued, and low-dose colchicine (0.6 mg once daily) was administered in addition to the P2Y₁₂ inhibitor. In the case of staged PCI, it was performed under the maintenance of colchicine and ticagrelor or prasugrel.

No other antiplatelet or anticoagulant agents were permitted.

Patients underwent platelet function testing using the VerifyNow P2Y₁₂ assay before discharge. Levels of hs-CRP were measured at admission, at 24 and 48 hours after PCI, and at 1-month follow-up. Clinical follow-up was performed at 1 and at 3 months.

The primary outcome was stent thrombosis within 3 months of follow-up. Secondary outcomes included all-cause mortality, MI, revascularization, major bleeding, a composite of cardiac death, target vessel MI, or target lesion revascularization, P2Y₁₂ reaction units (PRUs), and change in hs-CRP levels between 24 hours post-PCI and 1-month follow-up.
 

The role of inflammation

Of the original 200 patients, 190 completed the full protocol and were available for follow-up.

The primary outcome occurred in only two patients. It turned out that one of the patients had not been adherent with antiplatelet medications.

“Although bleeding occurred in 36 patients, major bleeding occurred in only 1 patient,” the authors report.

The level of platelet reactivity at discharge was 27 ± 42 PRUs. Most patients (91%) met the criteria for low platelet reactivity, while only 0.5% met the criteria for high platelet reactivity. Platelet reactivity was similar, regardless of which P2Y₁₂ inhibitor (ticagrelor or prasugrel) the patients were taking.

In all patients, the level of inflammation was “reduced considerably” over time: After 1 month, the hs-CRP level decreased from 6.1 mg/L (interquartile range [IQR], 2.6-15.9 mg/L) at 24 hours after PCI to 0.6 mg/L (IQR, 0.4-1.2 mg/L; P < .001).

The prevalence of high-inflammation criteria, defined as hs-CRP ≥ 2 mg/L, decreased significantly, from 81.8% at 24 hours after PCI to 11.8% at 1 month (P < .001).

Major bleeding was rare, they report, with a 3-month incidence of 0.5%.

“Inflammation plays a fundamental role in the development and progression of the atherothrombotic process,” the authors explain. A series of factors also trigger “an intense inflammatory response” in the acute phase of MI, which may lead to adverse myocardial remodeling. In the present study, inflammatory levels were rapidly reduced.

They noted several limitations. For example, all enrolled patients were Asian and were at relatively low bleeding and ischemic risk. “Although ticagrelor or prasugrel is effective regardless of ethnicity, clinical data supporting this de-escalation strategy are limited,” they state. Additionally, there was no control group for comparison.

The findings warrant further investigation, they conclude.
 

Promising but preliminary

Commenting for this news organization, Francesco Costa, MD, PhD, interventional cardiologist and assistant professor, University of Messina, Sicily, Italy, said he thinks it’s “too early for extensive clinical translation of these findings.”

Rather, larger and more extensive randomized trials are “on their way to give more precise estimates regarding the risks and benefits of early aspirin withdrawal in ACS.”

However, added Dr. Costa, who was not involved with the current research, “in this setting, adding colchicine early looks very promising to mitigate potential thrombotic risk without increasing bleeding risk.”

In the meantime, the study “provides novel insights on early aspirin withdrawal and P2Y₁₂ monotherapy in an unselected population, including [those with] STEMI,” said Dr. Costa, also the coauthor of an accompanying editorial. The findings “could be of particular interest for those patients at extremely high bleeding risk or who are truly intolerant to aspirin, a scenario in which options are limited.”

This study was supported by the Cardiovascular Research Center, Seoul, South Korea. Dr. Lee reports no relevant financial relationships. The other authors’ disclosures are listed on the original paper. Dr. Costa has served on an advisory board for AstraZeneca and has received speaker fees from Chiesi Farmaceutici. His coauthor reports no relevant financial relationships.

A version of this article appeared on Medscape.com.

Dual antiplatelet therapy (DAPT) consisting of aspirin plus a P2Y₁₂ inhibitor has been the standard of care to prevent thrombotic events in patients with acute coronary syndrome (ACS) undergoing percutaneous coronary intervention (PCI).

A new pilot study suggests that aspirin can be discontinued on the day after the PCI, and colchicine, an anti-inflammatory agent, could be added to reduce the risk for ischemic events in these patients, while mitigating the increased bleeding risk associated with aspirin.

Investigators conducted a pilot trial in ACS patients treated with drug-eluting stents (DES) who received low-dose colchicine the day after PCI, together with P2Y₁₂ inhibitor (ticagrelor or prasugrel) maintenance therapy. Aspirin use was discontinued.

At 3 months, only 1% of the patients experienced stent thrombosis, and only 1 patient showed high platelet reactivity. Moreover, at 1 month, high-sensitivity C-reactive protein (hs-CRP) and platelet reactivity both decreased, pointing to reduced inflammation.

“In ACS patients undergoing PCI, it is feasible to discontinue aspirin therapy and administer low-dose colchicine on the day after PCI in addition to ticagrelor or prasugrel P2Y₁₂ inhibitors,” write Seung-Yul Lee, MD, CHA Bundang Medical Center, CHA University, Seongnam, South Korea, and colleagues. “This approach is associated with favorable platelet function and inflammatory profiles.”

The study was published online in JACC: Cardiovascular Interventions.
 

Safety without compromised efficacy

The U.S. Food and Drug Administration recently approved colchicine 0.5-mg tablets (Lodoco, Agepha Pharma) as the first anti-inflammatory drug shown to reduce the risk for myocardial infarction, stroke, coronary revascularization, and cardiovascular death in adult patients with either established atherosclerotic disease or multiple risk factors for cardiovascular disease. It targets residual inflammation as an underlying cause of cardiovascular events.

Patients after PCI are generally treated using DAPT, but given the risk for increased bleeding associated with aspirin – especially when used long-term – there is a “need to identify strategies associated with a more favorable safety profile without compromising efficacy,” the authors write.

Previous research has yielded mixed results in terms of the discontinuation of aspirin therapy after 1-3 months and maintenance on P2Y₁₂ inhibitor monotherapy. But one trial found colchicine to be effective in reducing recurrent ischemia, and its benefits may be more beneficial with early initiation in the hospital.

In this new study, researchers tested a “strategy that substitutes aspirin with colchicine during the acute phase to maximize the treatment effect of reducing recurrent ischemia and bleeding,” they write. The Mono Antiplatelet and Colchicine Therapy (MACT) single-arm, open-label proof-of-concept study was designed to investigate this approach.

The researchers studied 200 patients with non–ST-segment elevation ACS and ST-segment elevation myocardial infarction (STEMI) who underwent PCI with DES (mean [SD] age, 61.4 [10.7] years; 90% male; 100% of Asian ethnicity), who were receiving either ticagrelor or prasugrel plus a loading dose of aspirin.

On the day after PCI, aspirin was discontinued, and low-dose colchicine (0.6 mg once daily) was administered in addition to the P2Y₁₂ inhibitor. In the case of staged PCI, it was performed under the maintenance of colchicine and ticagrelor or prasugrel.

No other antiplatelet or anticoagulant agents were permitted.

Patients underwent platelet function testing using the VerifyNow P2Y₁₂ assay before discharge. Levels of hs-CRP were measured at admission, at 24 and 48 hours after PCI, and at 1-month follow-up. Clinical follow-up was performed at 1 and at 3 months.

The primary outcome was stent thrombosis within 3 months of follow-up. Secondary outcomes included all-cause mortality, MI, revascularization, major bleeding, a composite of cardiac death, target vessel MI, or target lesion revascularization, P2Y₁₂ reaction units (PRUs), and change in hs-CRP levels between 24 hours post-PCI and 1-month follow-up.
 

The role of inflammation

Of the original 200 patients, 190 completed the full protocol and were available for follow-up.

The primary outcome occurred in only two patients. It turned out that one of the patients had not been adherent with antiplatelet medications.

“Although bleeding occurred in 36 patients, major bleeding occurred in only 1 patient,” the authors report.

The level of platelet reactivity at discharge was 27 ± 42 PRUs. Most patients (91%) met the criteria for low platelet reactivity, while only 0.5% met the criteria for high platelet reactivity. Platelet reactivity was similar, regardless of which P2Y₁₂ inhibitor (ticagrelor or prasugrel) the patients were taking.

In all patients, the level of inflammation was “reduced considerably” over time: After 1 month, the hs-CRP level decreased from 6.1 mg/L (interquartile range [IQR], 2.6-15.9 mg/L) at 24 hours after PCI to 0.6 mg/L (IQR, 0.4-1.2 mg/L; P < .001).

The prevalence of high-inflammation criteria, defined as hs-CRP ≥ 2 mg/L, decreased significantly, from 81.8% at 24 hours after PCI to 11.8% at 1 month (P < .001).

Major bleeding was rare, they report, with a 3-month incidence of 0.5%.

“Inflammation plays a fundamental role in the development and progression of the atherothrombotic process,” the authors explain. A series of factors also trigger “an intense inflammatory response” in the acute phase of MI, which may lead to adverse myocardial remodeling. In the present study, inflammatory levels were rapidly reduced.

They noted several limitations. For example, all enrolled patients were Asian and were at relatively low bleeding and ischemic risk. “Although ticagrelor or prasugrel is effective regardless of ethnicity, clinical data supporting this de-escalation strategy are limited,” they state. Additionally, there was no control group for comparison.

The findings warrant further investigation, they conclude.
 

Promising but preliminary

Commenting for this news organization, Francesco Costa, MD, PhD, interventional cardiologist and assistant professor, University of Messina, Sicily, Italy, said he thinks it’s “too early for extensive clinical translation of these findings.”

Rather, larger and more extensive randomized trials are “on their way to give more precise estimates regarding the risks and benefits of early aspirin withdrawal in ACS.”

However, added Dr. Costa, who was not involved with the current research, “in this setting, adding colchicine early looks very promising to mitigate potential thrombotic risk without increasing bleeding risk.”

In the meantime, the study “provides novel insights on early aspirin withdrawal and P2Y₁₂ monotherapy in an unselected population, including [those with] STEMI,” said Dr. Costa, also the coauthor of an accompanying editorial. The findings “could be of particular interest for those patients at extremely high bleeding risk or who are truly intolerant to aspirin, a scenario in which options are limited.”

This study was supported by the Cardiovascular Research Center, Seoul, South Korea. Dr. Lee reports no relevant financial relationships. The other authors’ disclosures are listed on the original paper. Dr. Costa has served on an advisory board for AstraZeneca and has received speaker fees from Chiesi Farmaceutici. His coauthor reports no relevant financial relationships.

A version of this article appeared on Medscape.com.

The Food and Drug Administration has approved palovarotene (Sohonos), the first-ever treatment for people with the rare and severely disabling bone condition fibrodysplasia ossificans progressiva (FOP).

Affecting roughly 400 people in the United States and 900 worldwide, FOP is an autosomal dominant condition in which bone develops in soft connective tissue areas of the body where it isn’t normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. This leads to severe restriction in mobility and function, to the point that people lose the ability to feed or care for themselves. Most are completely disabled by age 30 years and median life expectancy is 56 years, with death often caused by bone formation around the rib cage restricting respiration.

Olivier Le Moal/Getty Images

“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with ... since the accumulation of heterotopic ossification in FOP is progressive, irreversible, and life altering. This medication is an important treatment option for our FOP community,” said endocrinologist Edward Hsiao, MD, professor of medicine at the University of California, San Francisco, in a statement from Ipsen.

Taken orally, palovarotene selectively targets the gamma subtype of retinoic acid receptors that regulate skeletal development and ectopic bone in the retinoid signaling pathway. The drug mediates interactions between these receptors, growth factors, and proteins within that pathway to reduce new abnormal bone formation.

It is now FDA approved for the treatment of FOP in female patients aged 8 years or older and male patients aged 10 years or older. The recommended dosing is 5 mg daily or weight-based equivalent for pediatric patients under 14 years of age, which can be modified or increased for flare-up symptoms. It is contraindicated during pregnancy.

The FDA approval was based on 18-month data from the phase 3, multicenter, open-label MOVE trial that included 107 adult and pediatric patients, over 10% of the world’s population with FOP. All received oral palovarotene and were compared with untreated individuals from a prior natural history study of the condition. The drug reduced annualized heterotopic ossification volume by 54%.

Side effects were typical of those seen with other systemic retinoid drugs, including mucocutaneous events such as dryness of the skin and mucous membranes, alopecia, drug eruption, rash, and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure in growing children.

According to Dr. Hsiao, who was a MOVE investigator, the study “showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids.”

The FDA approval of palovarotene follows its rejection for marketing authorization in the European Union in July 2023.

Reached for comment, an Ipsen spokesperson said in an interview: “We reached the end of the regulatory process in the European Union for Sohonos and are disappointed the European Commission decided not to approved palovarotene for people with FOP in Europe.”

The company is developing another drug, fidrisertib, for treating FOP. A pivotal phase 2 trial for that drug is now recruiting patients. Asked where Ipsen might try to market fidrisertib, the spokesperson replied:“At this point, our focus is on the completion of the pivotal trial.”

Meanwhile, in the United States, the FOP community is celebrating the palovarotene approval. In a statement, Michelle Davis, executive director of the International Fibrodysplasia Ossificans Progressiva Association, said: “FOP is life altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close. ... The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”

Ipsen is offering a patient support program to assist with education, coverage, and reimbursement (1-866-435-5677).

A version of this article appeared on Medscape.com.

The Food and Drug Administration has approved palovarotene (Sohonos), the first-ever treatment for people with the rare and severely disabling bone condition fibrodysplasia ossificans progressiva (FOP).

Affecting roughly 400 people in the United States and 900 worldwide, FOP is an autosomal dominant condition in which bone develops in soft connective tissue areas of the body where it isn’t normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. This leads to severe restriction in mobility and function, to the point that people lose the ability to feed or care for themselves. Most are completely disabled by age 30 years and median life expectancy is 56 years, with death often caused by bone formation around the rib cage restricting respiration.

Olivier Le Moal/Getty Images

“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with ... since the accumulation of heterotopic ossification in FOP is progressive, irreversible, and life altering. This medication is an important treatment option for our FOP community,” said endocrinologist Edward Hsiao, MD, professor of medicine at the University of California, San Francisco, in a statement from Ipsen.

Taken orally, palovarotene selectively targets the gamma subtype of retinoic acid receptors that regulate skeletal development and ectopic bone in the retinoid signaling pathway. The drug mediates interactions between these receptors, growth factors, and proteins within that pathway to reduce new abnormal bone formation.

It is now FDA approved for the treatment of FOP in female patients aged 8 years or older and male patients aged 10 years or older. The recommended dosing is 5 mg daily or weight-based equivalent for pediatric patients under 14 years of age, which can be modified or increased for flare-up symptoms. It is contraindicated during pregnancy.

The FDA approval was based on 18-month data from the phase 3, multicenter, open-label MOVE trial that included 107 adult and pediatric patients, over 10% of the world’s population with FOP. All received oral palovarotene and were compared with untreated individuals from a prior natural history study of the condition. The drug reduced annualized heterotopic ossification volume by 54%.

Side effects were typical of those seen with other systemic retinoid drugs, including mucocutaneous events such as dryness of the skin and mucous membranes, alopecia, drug eruption, rash, and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure in growing children.

According to Dr. Hsiao, who was a MOVE investigator, the study “showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids.”

The FDA approval of palovarotene follows its rejection for marketing authorization in the European Union in July 2023.

Reached for comment, an Ipsen spokesperson said in an interview: “We reached the end of the regulatory process in the European Union for Sohonos and are disappointed the European Commission decided not to approved palovarotene for people with FOP in Europe.”

The company is developing another drug, fidrisertib, for treating FOP. A pivotal phase 2 trial for that drug is now recruiting patients. Asked where Ipsen might try to market fidrisertib, the spokesperson replied:“At this point, our focus is on the completion of the pivotal trial.”

Meanwhile, in the United States, the FOP community is celebrating the palovarotene approval. In a statement, Michelle Davis, executive director of the International Fibrodysplasia Ossificans Progressiva Association, said: “FOP is life altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close. ... The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”

Ipsen is offering a patient support program to assist with education, coverage, and reimbursement (1-866-435-5677).

A version of this article appeared on Medscape.com.

The Food and Drug Administration has approved palovarotene (Sohonos), the first-ever treatment for people with the rare and severely disabling bone condition fibrodysplasia ossificans progressiva (FOP).

Affecting roughly 400 people in the United States and 900 worldwide, FOP is an autosomal dominant condition in which bone develops in soft connective tissue areas of the body where it isn’t normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. This leads to severe restriction in mobility and function, to the point that people lose the ability to feed or care for themselves. Most are completely disabled by age 30 years and median life expectancy is 56 years, with death often caused by bone formation around the rib cage restricting respiration.

Olivier Le Moal/Getty Images

“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with ... since the accumulation of heterotopic ossification in FOP is progressive, irreversible, and life altering. This medication is an important treatment option for our FOP community,” said endocrinologist Edward Hsiao, MD, professor of medicine at the University of California, San Francisco, in a statement from Ipsen.

Taken orally, palovarotene selectively targets the gamma subtype of retinoic acid receptors that regulate skeletal development and ectopic bone in the retinoid signaling pathway. The drug mediates interactions between these receptors, growth factors, and proteins within that pathway to reduce new abnormal bone formation.

It is now FDA approved for the treatment of FOP in female patients aged 8 years or older and male patients aged 10 years or older. The recommended dosing is 5 mg daily or weight-based equivalent for pediatric patients under 14 years of age, which can be modified or increased for flare-up symptoms. It is contraindicated during pregnancy.

The FDA approval was based on 18-month data from the phase 3, multicenter, open-label MOVE trial that included 107 adult and pediatric patients, over 10% of the world’s population with FOP. All received oral palovarotene and were compared with untreated individuals from a prior natural history study of the condition. The drug reduced annualized heterotopic ossification volume by 54%.

Side effects were typical of those seen with other systemic retinoid drugs, including mucocutaneous events such as dryness of the skin and mucous membranes, alopecia, drug eruption, rash, and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure in growing children.

According to Dr. Hsiao, who was a MOVE investigator, the study “showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids.”

The FDA approval of palovarotene follows its rejection for marketing authorization in the European Union in July 2023.

Reached for comment, an Ipsen spokesperson said in an interview: “We reached the end of the regulatory process in the European Union for Sohonos and are disappointed the European Commission decided not to approved palovarotene for people with FOP in Europe.”

The company is developing another drug, fidrisertib, for treating FOP. A pivotal phase 2 trial for that drug is now recruiting patients. Asked where Ipsen might try to market fidrisertib, the spokesperson replied:“At this point, our focus is on the completion of the pivotal trial.”

Meanwhile, in the United States, the FOP community is celebrating the palovarotene approval. In a statement, Michelle Davis, executive director of the International Fibrodysplasia Ossificans Progressiva Association, said: “FOP is life altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close. ... The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”

Ipsen is offering a patient support program to assist with education, coverage, and reimbursement (1-866-435-5677).

A version of this article appeared on Medscape.com.

The Food and Drug Administration has granted accelerated approval to the off-the-shelf biologic agent elranatamab (Elrexfio) for the treatment of relapsed or refractory multiple myeloma.

The B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb) was given Priority Review in February and had previously received Breakthrough Therapy Designation for relapsed or refractory multiple myeloma (RRMM), according to Pfizer.

Olivier Le Moal/Getty Images

FDA approval was based on favorable response and duration of response rates in the single-arm, phase 2 MagnetisMM-3 trial. The trial showed meaningful responses in heavily pretreated patients with RRMM who received elranatamab as their first BCMA-directed therapy.

The overall response rate in 97 BCMA-naive patients (cohort A) who previously received at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, was 58%, with an estimated 82% maintaining the response for 9 months or longer. Median time to first response was 1.2 months.

In 63 patients who received at least four prior lines of therapy, which also included a BCMA-directed therapy, the overall response rate was 33% after median follow-up of 10.2 months. An estimated 84% maintained a response for at least 9 months.

Elranatamab was given subcutaneously at a dose of 76 mg weekly on a 28-day cycle with a step-up priming dose regimen. The priming regimen included 12 mg and 32 mg doses on days 1 and 4, respectively, during cycle 1. Patients who received at least six cycles and showed at least a partial response for 2 or more months had a biweekly dosing interval.

Elranatamab carries a boxed warning for cytokine release syndrome (CRS) and neurologic toxicity, as well as warnings and precautions for infections, neutropenia, hepatotoxicity, and embryo–fetal toxicity. Therefore, the agent is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS).

The boxed warning is included in the full prescribing information.

A confirmatory trial to gather additional safety and efficacy data was launched in 2022. Continued FDA approval is contingent on confirmed safety and efficacy data.

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

The Food and Drug Administration has granted accelerated approval to the off-the-shelf biologic agent elranatamab (Elrexfio) for the treatment of relapsed or refractory multiple myeloma.

The B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb) was given Priority Review in February and had previously received Breakthrough Therapy Designation for relapsed or refractory multiple myeloma (RRMM), according to Pfizer.

Olivier Le Moal/Getty Images

FDA approval was based on favorable response and duration of response rates in the single-arm, phase 2 MagnetisMM-3 trial. The trial showed meaningful responses in heavily pretreated patients with RRMM who received elranatamab as their first BCMA-directed therapy.

The overall response rate in 97 BCMA-naive patients (cohort A) who previously received at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, was 58%, with an estimated 82% maintaining the response for 9 months or longer. Median time to first response was 1.2 months.

In 63 patients who received at least four prior lines of therapy, which also included a BCMA-directed therapy, the overall response rate was 33% after median follow-up of 10.2 months. An estimated 84% maintained a response for at least 9 months.

Elranatamab was given subcutaneously at a dose of 76 mg weekly on a 28-day cycle with a step-up priming dose regimen. The priming regimen included 12 mg and 32 mg doses on days 1 and 4, respectively, during cycle 1. Patients who received at least six cycles and showed at least a partial response for 2 or more months had a biweekly dosing interval.

Elranatamab carries a boxed warning for cytokine release syndrome (CRS) and neurologic toxicity, as well as warnings and precautions for infections, neutropenia, hepatotoxicity, and embryo–fetal toxicity. Therefore, the agent is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS).

The boxed warning is included in the full prescribing information.

A confirmatory trial to gather additional safety and efficacy data was launched in 2022. Continued FDA approval is contingent on confirmed safety and efficacy data.

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

The Food and Drug Administration has granted accelerated approval to the off-the-shelf biologic agent elranatamab (Elrexfio) for the treatment of relapsed or refractory multiple myeloma.

The B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb) was given Priority Review in February and had previously received Breakthrough Therapy Designation for relapsed or refractory multiple myeloma (RRMM), according to Pfizer.

Olivier Le Moal/Getty Images

FDA approval was based on favorable response and duration of response rates in the single-arm, phase 2 MagnetisMM-3 trial. The trial showed meaningful responses in heavily pretreated patients with RRMM who received elranatamab as their first BCMA-directed therapy.

The overall response rate in 97 BCMA-naive patients (cohort A) who previously received at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, was 58%, with an estimated 82% maintaining the response for 9 months or longer. Median time to first response was 1.2 months.

In 63 patients who received at least four prior lines of therapy, which also included a BCMA-directed therapy, the overall response rate was 33% after median follow-up of 10.2 months. An estimated 84% maintained a response for at least 9 months.

Elranatamab was given subcutaneously at a dose of 76 mg weekly on a 28-day cycle with a step-up priming dose regimen. The priming regimen included 12 mg and 32 mg doses on days 1 and 4, respectively, during cycle 1. Patients who received at least six cycles and showed at least a partial response for 2 or more months had a biweekly dosing interval.

Elranatamab carries a boxed warning for cytokine release syndrome (CRS) and neurologic toxicity, as well as warnings and precautions for infections, neutropenia, hepatotoxicity, and embryo–fetal toxicity. Therefore, the agent is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS).

The boxed warning is included in the full prescribing information.

A confirmatory trial to gather additional safety and efficacy data was launched in 2022. Continued FDA approval is contingent on confirmed safety and efficacy data.

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

CHICAGO – At this year’s AGA Postgraduate Course in May at the annual Digestive Disease Week^®(DDW), we reviewed the latest updates in inflammatory bowel disease (IBD).

Although it had been thought that incidence rates of IBD were plateauing in high-incidence areas, a Danish study found a steady increase in incidence of Crohn’s disease and ulcerative colitis (UC).¹ The highest increase in rates occurred in children and young adults, which will have repercussions as people get older and contribute to higher compounding prevalence. We need to get better at dealing with other health conditions as patients get older. A very large prospective Spanish study found that 42% of IBD patients scanned consecutively had MAFLD (metabolic-associated fatty liver disease) – even if they didn’t have high BMI and type 2 diabetes, suggesting that systemic inflammation in IBD contributes to the development of metabolic liver disease.²

University of Miami

The AGA has recently published guidelines for using biomarkers in the management of UC. Patients with very low fecal calprotectin (FCP) are unlikely to have active disease whereas FCP over 150 with significant symptoms may warrant empiric changes in treatment.³

Intestinal ultrasound is gaining wider acceptance as a noninvasive way to monitor IBD.⁴ In a UC study, improvement in bowel wall thickness following tofacitinib treatment correlated well with endoscopic activity.⁵

Brigham and Women&#039;s Hospital

The majority of the presentation focused on the explosion of Food and Drug Administration–-approved medications for IBD in recent years. S1P receptor agonists, such as ozanimod and etrasimod, may work by trapping specific T-cell subsets in peripheral lymph nodes, preventing migration to intestinal tissues. Ozanimod is approved for UC. Etrasimod showed efficacy in UC with clinical remission rates of about 27% at week 12 and 32% at week 52.^6,7

There has been a lot of excitement about JAK inhibitors for IBD. Upadacitinib has recently been approved for both UC and Crohn’s disease. Response rates of 73% and remission rates of 26% were seen in UC patients who had been largely biologic exposed.⁸ Similar results were seen in a biologic-exposed Crohn’s disease population treated with upadacitinib including in endoscopy.⁹ Upadacitinib was effective in maintaining remission at both 15-mg and 30-mg doses; but the higher dose had a greater effect on endoscopic endpoints.¹⁰

Dr. Loftus

For Crohn’s disease, we now have risankizumab, an anti-p19/IL-23 inhibitor. Risankizumab was efficacious at inducing and maintain remission in the pivotal phase 3 studies, even with 75% of patients being biologic exposed. These studies used combined endpoints of clinical remission as well as endoscopic response.¹¹ Guselkumab (anti-p19/IL-23) is also being studied for Crohn’s disease and early trials has appears to be efficacious.¹²

A head-to-head study of naive CD patients treated with ustekinumab or adalimumab (SEAVUE) showed comparable rates of clinical remission. At 52 weeks, the rates of clinical remission were quite high: >60% and endoscopic remission >30% with either therapy.¹³

We now have phase 3 data showing that a biologic is efficacious in patients with chronic pouchitis. The EARNEST trial demonstrated that vedolizumab has efficacy in treating pouchitis with improved clinical symptoms and endoscopy.¹⁴ Future treatment strategies may involve combinations of biologic therapies. The VEGA study showed that combining an anti-TNF, golimumab, with an anti-IL23, guselkumab, was superior than either alone with respect to clinical remission and endoscopic improvement in UC.¹⁵ We will see more studies combining therapies with diverse mechanisms of action.

In summary, there have been many noteworthy advances in treatment and management of IBD in the past year.

DDW is sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and The Society for Surgery of the Alimentary Tract (SSAT).

Dr. Abreu is director of the Crohn’s and Colitis Center and professor of medicine, microbiology, and immunology at the University of Miami. She is president-elect of AGA. Dr. Allegretti is director of the Crohn’s and Colitis Center and director of the fecal microbiota transplant program at Brigham and Women’s Hospital, Boston. She is associate professor of medicine at Harvard Medical School, Boston. Dr. Loftus is the Maxine and Jack Zarrow Family Professor of Gastroenterology, codirector of the advanced IBD fellowship in the division of gastroenterology and hepatology at Mayo Clinic, Rochester, Minn. Dr. Ungaro is associate professor of medicine at the Icahn School of Medicine at Mount Sinai, New York.

References

1. Agrawal M et al. Gastroenterology. 2022;163(6):1547-54.e5.

2. Rodriguez-Duque JC et al. Clin Gastroenterol Hepatol. 2023;21(2):406-14.e7.

3. Singh S, et al. Gastroenterology. 2023;164(3):344-72.

4. de Voogd F et al. Gastroenterology. 2022;163(6):1569-81.

5. Sandborn WJ et al. N Engl J Med. 2017;376(18):1723-36.

6. Sandborn WJ et al. N Engl J Med. 2021;385(14):1280-91.

7. Sandborn WJ et al. Lancet. 2023 Mar 25;401(10381):1000]. Lancet. 2023;401(10383):1159-71.

8. Danese S et al. Lancet. 2022 Sep 24;400(10357):996]. Lancet. 2022;399(10341):2113-28.

9. Loftus EV Jr et al. N Engl J Med. 2023 May 25;388(21):1966-80.

10. Panes J et al. Am J Gastroenterol 2022;117(S10). Abstract S37.

11. D’Haens G, et al. Lancet. 2022;399(10340):2015-30

12. Sandborn WJ et al. Gastroenterology. 2022;162(6):1650-64.e8.

13. Sands BE, et al. Lancet. 2022;399(10342):2200-11.

14. Travis S et al. N Engl J Med. 2023;388(13):1191-1200.

15. Feagan BG et al. Lancet Gastroenterol Hepatol. 2023;8(4):307-20.

CHICAGO – At this year’s AGA Postgraduate Course in May at the annual Digestive Disease Week^®(DDW), we reviewed the latest updates in inflammatory bowel disease (IBD).

Although it had been thought that incidence rates of IBD were plateauing in high-incidence areas, a Danish study found a steady increase in incidence of Crohn’s disease and ulcerative colitis (UC).¹ The highest increase in rates occurred in children and young adults, which will have repercussions as people get older and contribute to higher compounding prevalence. We need to get better at dealing with other health conditions as patients get older. A very large prospective Spanish study found that 42% of IBD patients scanned consecutively had MAFLD (metabolic-associated fatty liver disease) – even if they didn’t have high BMI and type 2 diabetes, suggesting that systemic inflammation in IBD contributes to the development of metabolic liver disease.²

University of Miami

The AGA has recently published guidelines for using biomarkers in the management of UC. Patients with very low fecal calprotectin (FCP) are unlikely to have active disease whereas FCP over 150 with significant symptoms may warrant empiric changes in treatment.³

Intestinal ultrasound is gaining wider acceptance as a noninvasive way to monitor IBD.⁴ In a UC study, improvement in bowel wall thickness following tofacitinib treatment correlated well with endoscopic activity.⁵

Brigham and Women&#039;s Hospital

The majority of the presentation focused on the explosion of Food and Drug Administration–-approved medications for IBD in recent years. S1P receptor agonists, such as ozanimod and etrasimod, may work by trapping specific T-cell subsets in peripheral lymph nodes, preventing migration to intestinal tissues. Ozanimod is approved for UC. Etrasimod showed efficacy in UC with clinical remission rates of about 27% at week 12 and 32% at week 52.^6,7

There has been a lot of excitement about JAK inhibitors for IBD. Upadacitinib has recently been approved for both UC and Crohn’s disease. Response rates of 73% and remission rates of 26% were seen in UC patients who had been largely biologic exposed.⁸ Similar results were seen in a biologic-exposed Crohn’s disease population treated with upadacitinib including in endoscopy.⁹ Upadacitinib was effective in maintaining remission at both 15-mg and 30-mg doses; but the higher dose had a greater effect on endoscopic endpoints.¹⁰

Dr. Loftus

For Crohn’s disease, we now have risankizumab, an anti-p19/IL-23 inhibitor. Risankizumab was efficacious at inducing and maintain remission in the pivotal phase 3 studies, even with 75% of patients being biologic exposed. These studies used combined endpoints of clinical remission as well as endoscopic response.¹¹ Guselkumab (anti-p19/IL-23) is also being studied for Crohn’s disease and early trials has appears to be efficacious.¹²

A head-to-head study of naive CD patients treated with ustekinumab or adalimumab (SEAVUE) showed comparable rates of clinical remission. At 52 weeks, the rates of clinical remission were quite high: >60% and endoscopic remission >30% with either therapy.¹³

We now have phase 3 data showing that a biologic is efficacious in patients with chronic pouchitis. The EARNEST trial demonstrated that vedolizumab has efficacy in treating pouchitis with improved clinical symptoms and endoscopy.¹⁴ Future treatment strategies may involve combinations of biologic therapies. The VEGA study showed that combining an anti-TNF, golimumab, with an anti-IL23, guselkumab, was superior than either alone with respect to clinical remission and endoscopic improvement in UC.¹⁵ We will see more studies combining therapies with diverse mechanisms of action.

In summary, there have been many noteworthy advances in treatment and management of IBD in the past year.

DDW is sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and The Society for Surgery of the Alimentary Tract (SSAT).

Dr. Abreu is director of the Crohn’s and Colitis Center and professor of medicine, microbiology, and immunology at the University of Miami. She is president-elect of AGA. Dr. Allegretti is director of the Crohn’s and Colitis Center and director of the fecal microbiota transplant program at Brigham and Women’s Hospital, Boston. She is associate professor of medicine at Harvard Medical School, Boston. Dr. Loftus is the Maxine and Jack Zarrow Family Professor of Gastroenterology, codirector of the advanced IBD fellowship in the division of gastroenterology and hepatology at Mayo Clinic, Rochester, Minn. Dr. Ungaro is associate professor of medicine at the Icahn School of Medicine at Mount Sinai, New York.

References

1. Agrawal M et al. Gastroenterology. 2022;163(6):1547-54.e5.

2. Rodriguez-Duque JC et al. Clin Gastroenterol Hepatol. 2023;21(2):406-14.e7.

3. Singh S, et al. Gastroenterology. 2023;164(3):344-72.

4. de Voogd F et al. Gastroenterology. 2022;163(6):1569-81.

5. Sandborn WJ et al. N Engl J Med. 2017;376(18):1723-36.

6. Sandborn WJ et al. N Engl J Med. 2021;385(14):1280-91.

7. Sandborn WJ et al. Lancet. 2023 Mar 25;401(10381):1000]. Lancet. 2023;401(10383):1159-71.

8. Danese S et al. Lancet. 2022 Sep 24;400(10357):996]. Lancet. 2022;399(10341):2113-28.

9. Loftus EV Jr et al. N Engl J Med. 2023 May 25;388(21):1966-80.

10. Panes J et al. Am J Gastroenterol 2022;117(S10). Abstract S37.

11. D’Haens G, et al. Lancet. 2022;399(10340):2015-30

12. Sandborn WJ et al. Gastroenterology. 2022;162(6):1650-64.e8.

13. Sands BE, et al. Lancet. 2022;399(10342):2200-11.

14. Travis S et al. N Engl J Med. 2023;388(13):1191-1200.

15. Feagan BG et al. Lancet Gastroenterol Hepatol. 2023;8(4):307-20.

CHICAGO – At this year’s AGA Postgraduate Course in May at the annual Digestive Disease Week^®(DDW), we reviewed the latest updates in inflammatory bowel disease (IBD).

Although it had been thought that incidence rates of IBD were plateauing in high-incidence areas, a Danish study found a steady increase in incidence of Crohn’s disease and ulcerative colitis (UC).¹ The highest increase in rates occurred in children and young adults, which will have repercussions as people get older and contribute to higher compounding prevalence. We need to get better at dealing with other health conditions as patients get older. A very large prospective Spanish study found that 42% of IBD patients scanned consecutively had MAFLD (metabolic-associated fatty liver disease) – even if they didn’t have high BMI and type 2 diabetes, suggesting that systemic inflammation in IBD contributes to the development of metabolic liver disease.²

University of Miami

The AGA has recently published guidelines for using biomarkers in the management of UC. Patients with very low fecal calprotectin (FCP) are unlikely to have active disease whereas FCP over 150 with significant symptoms may warrant empiric changes in treatment.³

Intestinal ultrasound is gaining wider acceptance as a noninvasive way to monitor IBD.⁴ In a UC study, improvement in bowel wall thickness following tofacitinib treatment correlated well with endoscopic activity.⁵

Brigham and Women&#039;s Hospital

The majority of the presentation focused on the explosion of Food and Drug Administration–-approved medications for IBD in recent years. S1P receptor agonists, such as ozanimod and etrasimod, may work by trapping specific T-cell subsets in peripheral lymph nodes, preventing migration to intestinal tissues. Ozanimod is approved for UC. Etrasimod showed efficacy in UC with clinical remission rates of about 27% at week 12 and 32% at week 52.^6,7

There has been a lot of excitement about JAK inhibitors for IBD. Upadacitinib has recently been approved for both UC and Crohn’s disease. Response rates of 73% and remission rates of 26% were seen in UC patients who had been largely biologic exposed.⁸ Similar results were seen in a biologic-exposed Crohn’s disease population treated with upadacitinib including in endoscopy.⁹ Upadacitinib was effective in maintaining remission at both 15-mg and 30-mg doses; but the higher dose had a greater effect on endoscopic endpoints.¹⁰

Dr. Loftus

For Crohn’s disease, we now have risankizumab, an anti-p19/IL-23 inhibitor. Risankizumab was efficacious at inducing and maintain remission in the pivotal phase 3 studies, even with 75% of patients being biologic exposed. These studies used combined endpoints of clinical remission as well as endoscopic response.¹¹ Guselkumab (anti-p19/IL-23) is also being studied for Crohn’s disease and early trials has appears to be efficacious.¹²

A head-to-head study of naive CD patients treated with ustekinumab or adalimumab (SEAVUE) showed comparable rates of clinical remission. At 52 weeks, the rates of clinical remission were quite high: >60% and endoscopic remission >30% with either therapy.¹³

We now have phase 3 data showing that a biologic is efficacious in patients with chronic pouchitis. The EARNEST trial demonstrated that vedolizumab has efficacy in treating pouchitis with improved clinical symptoms and endoscopy.¹⁴ Future treatment strategies may involve combinations of biologic therapies. The VEGA study showed that combining an anti-TNF, golimumab, with an anti-IL23, guselkumab, was superior than either alone with respect to clinical remission and endoscopic improvement in UC.¹⁵ We will see more studies combining therapies with diverse mechanisms of action.

In summary, there have been many noteworthy advances in treatment and management of IBD in the past year.

DDW is sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and The Society for Surgery of the Alimentary Tract (SSAT).

Dr. Abreu is director of the Crohn’s and Colitis Center and professor of medicine, microbiology, and immunology at the University of Miami. She is president-elect of AGA. Dr. Allegretti is director of the Crohn’s and Colitis Center and director of the fecal microbiota transplant program at Brigham and Women’s Hospital, Boston. She is associate professor of medicine at Harvard Medical School, Boston. Dr. Loftus is the Maxine and Jack Zarrow Family Professor of Gastroenterology, codirector of the advanced IBD fellowship in the division of gastroenterology and hepatology at Mayo Clinic, Rochester, Minn. Dr. Ungaro is associate professor of medicine at the Icahn School of Medicine at Mount Sinai, New York.

References

1. Agrawal M et al. Gastroenterology. 2022;163(6):1547-54.e5.

2. Rodriguez-Duque JC et al. Clin Gastroenterol Hepatol. 2023;21(2):406-14.e7.

3. Singh S, et al. Gastroenterology. 2023;164(3):344-72.

4. de Voogd F et al. Gastroenterology. 2022;163(6):1569-81.

5. Sandborn WJ et al. N Engl J Med. 2017;376(18):1723-36.

6. Sandborn WJ et al. N Engl J Med. 2021;385(14):1280-91.

7. Sandborn WJ et al. Lancet. 2023 Mar 25;401(10381):1000]. Lancet. 2023;401(10383):1159-71.

8. Danese S et al. Lancet. 2022 Sep 24;400(10357):996]. Lancet. 2022;399(10341):2113-28.

9. Loftus EV Jr et al. N Engl J Med. 2023 May 25;388(21):1966-80.

10. Panes J et al. Am J Gastroenterol 2022;117(S10). Abstract S37.

11. D’Haens G, et al. Lancet. 2022;399(10340):2015-30

12. Sandborn WJ et al. Gastroenterology. 2022;162(6):1650-64.e8.

13. Sands BE, et al. Lancet. 2022;399(10342):2200-11.

14. Travis S et al. N Engl J Med. 2023;388(13):1191-1200.

15. Feagan BG et al. Lancet Gastroenterol Hepatol. 2023;8(4):307-20.

Nearly half of adults with a chronic hepatitis D (HDV) viral infection showed undetectable or greatly reduced viral RNA and normalized alanine aminotransferase (ALT) levels with bulevirtide at 48 weeks, shows an ongoing phase 3 study conducted in the United States and four other countries.

The findings were published in the New England Journal of Medicine.

Led by Heiner Wedemeyer, MD, of Hannover Medical School in Germany, the study included 150 patients with HDV, with and without compensated cirrhosis (mean age, 42 years; 57% male; 83% White). They were randomly assigned to receive 2 mg or 10 mg of bulevirtide subcutaneously daily for 144 weeks or, as a control group, receive no treatment for 48 weeks, followed by 10 mg of bulevirtide daily for 96 weeks. All patients were followed for 96 weeks after treatment ends.

For the primary endpoint, the combined viral and ALT response at week 48 was similar in the 2-mg (45%) and 10-mg (48%) groups, compared with 2% in the control group (one patient). Twelve percent of patients in the 2-mg group and 20% of patients in the 10-mg group had a clinical benefit, compared with none of the patients in the control group.

Among those with a combined response, normalization of the ALT level occurred in most patients by week 24, while the HDV RNA level continued to decline between week 24 and week 48, the authors wrote.

“This surrogate end point is considered to be a reasonably likely predictor of improved clinical outcomes in patients with HDV; however, longer-term data are needed to confirm the clinical benefit of bulevirtide,” the investigators wrote.

The results offer a glimmer of hope, Marc Ghany, MD, MHSc, of the National Institute of Diabetes and Digestive and Kidney Diseases wrote in an accompanying editorial. “The goal of HDV therapy is to improve patient survival by preventing progression to cirrhosis, liver failure, and liver cancer,” he wrote.

In safety results, headache, pruritus, fatigue, and eosinophilia were more common in the bulevirtide groups than in the control group. All adverse events were mild to moderate.

HDV infects about 5% of people with chronic HBV and relies on HBV surface antigen (HBsAg) for transmission and infectivity. Bulevirtide is derived from a region of the large envelope protein of HBsAg and irreversibly binds to the hepatocyte entry receptor for both HDV and HBV.

Bulevirtide has received conditional approval in the European Union. In 2022, the Food and Drug Administration declined to approve bulevirtide over concerns about production and delivery of the drug. There are no approved treatments for HDV in the United States.

The study was supported by Gilead Sciences. Dr. Wedemeyer disclosed research funding, acting as a consultant to, and giving paid lectures on behalf of Gilead Sciences. He and other coauthors disclosed financial relationships with Gilead and other pharmaceutical companies.

Nearly half of adults with a chronic hepatitis D (HDV) viral infection showed undetectable or greatly reduced viral RNA and normalized alanine aminotransferase (ALT) levels with bulevirtide at 48 weeks, shows an ongoing phase 3 study conducted in the United States and four other countries.

The findings were published in the New England Journal of Medicine.

Led by Heiner Wedemeyer, MD, of Hannover Medical School in Germany, the study included 150 patients with HDV, with and without compensated cirrhosis (mean age, 42 years; 57% male; 83% White). They were randomly assigned to receive 2 mg or 10 mg of bulevirtide subcutaneously daily for 144 weeks or, as a control group, receive no treatment for 48 weeks, followed by 10 mg of bulevirtide daily for 96 weeks. All patients were followed for 96 weeks after treatment ends.

For the primary endpoint, the combined viral and ALT response at week 48 was similar in the 2-mg (45%) and 10-mg (48%) groups, compared with 2% in the control group (one patient). Twelve percent of patients in the 2-mg group and 20% of patients in the 10-mg group had a clinical benefit, compared with none of the patients in the control group.

Among those with a combined response, normalization of the ALT level occurred in most patients by week 24, while the HDV RNA level continued to decline between week 24 and week 48, the authors wrote.

“This surrogate end point is considered to be a reasonably likely predictor of improved clinical outcomes in patients with HDV; however, longer-term data are needed to confirm the clinical benefit of bulevirtide,” the investigators wrote.

The results offer a glimmer of hope, Marc Ghany, MD, MHSc, of the National Institute of Diabetes and Digestive and Kidney Diseases wrote in an accompanying editorial. “The goal of HDV therapy is to improve patient survival by preventing progression to cirrhosis, liver failure, and liver cancer,” he wrote.

In safety results, headache, pruritus, fatigue, and eosinophilia were more common in the bulevirtide groups than in the control group. All adverse events were mild to moderate.

HDV infects about 5% of people with chronic HBV and relies on HBV surface antigen (HBsAg) for transmission and infectivity. Bulevirtide is derived from a region of the large envelope protein of HBsAg and irreversibly binds to the hepatocyte entry receptor for both HDV and HBV.

Bulevirtide has received conditional approval in the European Union. In 2022, the Food and Drug Administration declined to approve bulevirtide over concerns about production and delivery of the drug. There are no approved treatments for HDV in the United States.

The study was supported by Gilead Sciences. Dr. Wedemeyer disclosed research funding, acting as a consultant to, and giving paid lectures on behalf of Gilead Sciences. He and other coauthors disclosed financial relationships with Gilead and other pharmaceutical companies.

Nearly half of adults with a chronic hepatitis D (HDV) viral infection showed undetectable or greatly reduced viral RNA and normalized alanine aminotransferase (ALT) levels with bulevirtide at 48 weeks, shows an ongoing phase 3 study conducted in the United States and four other countries.

The findings were published in the New England Journal of Medicine.

Led by Heiner Wedemeyer, MD, of Hannover Medical School in Germany, the study included 150 patients with HDV, with and without compensated cirrhosis (mean age, 42 years; 57% male; 83% White). They were randomly assigned to receive 2 mg or 10 mg of bulevirtide subcutaneously daily for 144 weeks or, as a control group, receive no treatment for 48 weeks, followed by 10 mg of bulevirtide daily for 96 weeks. All patients were followed for 96 weeks after treatment ends.

For the primary endpoint, the combined viral and ALT response at week 48 was similar in the 2-mg (45%) and 10-mg (48%) groups, compared with 2% in the control group (one patient). Twelve percent of patients in the 2-mg group and 20% of patients in the 10-mg group had a clinical benefit, compared with none of the patients in the control group.

Among those with a combined response, normalization of the ALT level occurred in most patients by week 24, while the HDV RNA level continued to decline between week 24 and week 48, the authors wrote.

“This surrogate end point is considered to be a reasonably likely predictor of improved clinical outcomes in patients with HDV; however, longer-term data are needed to confirm the clinical benefit of bulevirtide,” the investigators wrote.

The results offer a glimmer of hope, Marc Ghany, MD, MHSc, of the National Institute of Diabetes and Digestive and Kidney Diseases wrote in an accompanying editorial. “The goal of HDV therapy is to improve patient survival by preventing progression to cirrhosis, liver failure, and liver cancer,” he wrote.

In safety results, headache, pruritus, fatigue, and eosinophilia were more common in the bulevirtide groups than in the control group. All adverse events were mild to moderate.

HDV infects about 5% of people with chronic HBV and relies on HBV surface antigen (HBsAg) for transmission and infectivity. Bulevirtide is derived from a region of the large envelope protein of HBsAg and irreversibly binds to the hepatocyte entry receptor for both HDV and HBV.

Bulevirtide has received conditional approval in the European Union. In 2022, the Food and Drug Administration declined to approve bulevirtide over concerns about production and delivery of the drug. There are no approved treatments for HDV in the United States.

The study was supported by Gilead Sciences. Dr. Wedemeyer disclosed research funding, acting as a consultant to, and giving paid lectures on behalf of Gilead Sciences. He and other coauthors disclosed financial relationships with Gilead and other pharmaceutical companies.