PHOENIX — Black women who develop high blood pressure before age 35 have a threefold increased risk of having a midlife stroke, new observational data suggest. The Black Women’s Health Study, which has followed 59,000 participants in the United States since 1990s, also showed that those who develop hypertension before age 45 have twice the risk of suffering a stroke.

“The really concerning thing about this data is the high proportion of young Black women who had high blood pressure and are suffering strokes relatively early in life,” said the study’s lead author, Hugo J. Aparicio, MD, associate professor of neurology at Boston University Chobanian & Avedisian School of Medicine, Boston. “This can lead to a burden of disability in relatively young women who may be at the prime of their life, pursuing careers, and looking after family.”

Dr. Aparicio presented the data at the International Stroke Conference presented by the American Stroke Association, a division of the American Heart Association.

He explained that while there has been good progress in reducing stroke rates in older people over the past decades, there is a concerning observation from multiple datasets showing that stroke rates in midlife have been plateauing or even increasing in recent years.

“For Black women specifically, there is a concern, as we know this group has higher rates of raised blood pressure and stroke overall,” said Dr. Aparicio. “We were interested in looking at whether the onset of hypertension at an earlier age in this group is one of the reasons for the increased stroke risk in midlife.”
 

A Large Study Cohort

The researchers analyzed data from the Black Women’s Health Study; the baseline year for this analysis, which included 46,754 stroke-free participants younger than age 65 (mean age, 42 years), was the 1999 questionnaire.

Both history of hypertension — defined as physician-diagnosed hypertension with the use of an antihypertensive medication — and stroke occurrence were determined by self-report. It has been shown in previous studies that these self-reported data on incidence of hypertension in this dataset are highly reliable, Dr. Aparicio noted.

At baseline, 10.5% of participants aged 45-64 years had hypertension. Stroke occurred in 3.2% of individuals over a mean follow-up of 17 years.

Black women with hypertension before age 45 had a higher risk for midlife stroke (hazard ratio [HR], 2.23; 95% confidence interval [CI], 1.79-2.78), after adjustment for age, neighborhood socioeconomic status, residence in the Stroke Belt, smoking, body mass index, and diabetes than women with no history of hypertension.

The risk was also increased with hypertension at midlife ages 45-64 years (HR, 1.69; 95% CI, 1.47-1.95) and was highest among those with hypertension at ages 24-34 years (HR, 3.15; 95% CI, 1.92-5.16).

“Our results show that among young Black women, those with hypertension have a much higher stroke risk than those without hypertension, even if they are taking antihypertensive medication,” Dr. Aparicio said. “This underscores how potent hypertension is as a risk factor for stroke.”

He concluded that both individuals and doctors need to realize that hypertension and stroke are not problems of the elderly exclusively.

“These are conditions that need to be addressed very early in life. This is even more important for Black women, as they are a high-risk group. They need to pay attention to blood pressure numbers early in life — ideally from adolescence — to catch levels before they become too elevated,” Dr. Aparicio said.

“We also need to address lifestyle changes including diet, physical activity, sleep habits, and address other cardiovascular risk factors such as cholesterol and body mass index, so we can prevent strokes from occurring,” he added. “At the policy level, we need to advocate, provide and fund primary prevention measures, and enable earlier screening and better treatment.”
 

The Role of Psychosocial Stressors

Commenting on the study, the American Heart Association immediate past president, Michelle A. Albert, MD, professor of medicine at the University of California, San Francisco, emphasized the importance of regular primary care appointments to screen for high blood pressure and other cardiovascular risk factors.

She pointed out that one of the contributing factors that may increase the risk for Black women is their disproportionate experience of psychosocial stressors and chronic cumulative stress.

This could include stress related to financial issues, racism and other forms of bias, the neighborhood environment, and having to take care of multiple generations of family with limited resources.

“These are some of the things that are less talked about as going into the heightened risk for many cardiovascular risk factors, including hypertension, very early in life for Black women that we need to bring to the forefront of conversations,” Dr. Albert said.

“These stressors not only impact hypertension onset but also they impact one’s ability to be able to seek help, and once the help is sought, to be able to sustain the therapies recommended and the interventions recommended,” she added.

The authors reported no relevant disclosures.
 

A version of this article appeared on Medscape.com.

PHOENIX — Black women who develop high blood pressure before age 35 have a threefold increased risk of having a midlife stroke, new observational data suggest. The Black Women’s Health Study, which has followed 59,000 participants in the United States since 1990s, also showed that those who develop hypertension before age 45 have twice the risk of suffering a stroke.

“The really concerning thing about this data is the high proportion of young Black women who had high blood pressure and are suffering strokes relatively early in life,” said the study’s lead author, Hugo J. Aparicio, MD, associate professor of neurology at Boston University Chobanian & Avedisian School of Medicine, Boston. “This can lead to a burden of disability in relatively young women who may be at the prime of their life, pursuing careers, and looking after family.”

Dr. Aparicio presented the data at the International Stroke Conference presented by the American Stroke Association, a division of the American Heart Association.

He explained that while there has been good progress in reducing stroke rates in older people over the past decades, there is a concerning observation from multiple datasets showing that stroke rates in midlife have been plateauing or even increasing in recent years.

“For Black women specifically, there is a concern, as we know this group has higher rates of raised blood pressure and stroke overall,” said Dr. Aparicio. “We were interested in looking at whether the onset of hypertension at an earlier age in this group is one of the reasons for the increased stroke risk in midlife.”
 

A Large Study Cohort

The researchers analyzed data from the Black Women’s Health Study; the baseline year for this analysis, which included 46,754 stroke-free participants younger than age 65 (mean age, 42 years), was the 1999 questionnaire.

Both history of hypertension — defined as physician-diagnosed hypertension with the use of an antihypertensive medication — and stroke occurrence were determined by self-report. It has been shown in previous studies that these self-reported data on incidence of hypertension in this dataset are highly reliable, Dr. Aparicio noted.

At baseline, 10.5% of participants aged 45-64 years had hypertension. Stroke occurred in 3.2% of individuals over a mean follow-up of 17 years.

Black women with hypertension before age 45 had a higher risk for midlife stroke (hazard ratio [HR], 2.23; 95% confidence interval [CI], 1.79-2.78), after adjustment for age, neighborhood socioeconomic status, residence in the Stroke Belt, smoking, body mass index, and diabetes than women with no history of hypertension.

The risk was also increased with hypertension at midlife ages 45-64 years (HR, 1.69; 95% CI, 1.47-1.95) and was highest among those with hypertension at ages 24-34 years (HR, 3.15; 95% CI, 1.92-5.16).

“Our results show that among young Black women, those with hypertension have a much higher stroke risk than those without hypertension, even if they are taking antihypertensive medication,” Dr. Aparicio said. “This underscores how potent hypertension is as a risk factor for stroke.”

He concluded that both individuals and doctors need to realize that hypertension and stroke are not problems of the elderly exclusively.

“These are conditions that need to be addressed very early in life. This is even more important for Black women, as they are a high-risk group. They need to pay attention to blood pressure numbers early in life — ideally from adolescence — to catch levels before they become too elevated,” Dr. Aparicio said.

“We also need to address lifestyle changes including diet, physical activity, sleep habits, and address other cardiovascular risk factors such as cholesterol and body mass index, so we can prevent strokes from occurring,” he added. “At the policy level, we need to advocate, provide and fund primary prevention measures, and enable earlier screening and better treatment.”
 

The Role of Psychosocial Stressors

Commenting on the study, the American Heart Association immediate past president, Michelle A. Albert, MD, professor of medicine at the University of California, San Francisco, emphasized the importance of regular primary care appointments to screen for high blood pressure and other cardiovascular risk factors.

She pointed out that one of the contributing factors that may increase the risk for Black women is their disproportionate experience of psychosocial stressors and chronic cumulative stress.

This could include stress related to financial issues, racism and other forms of bias, the neighborhood environment, and having to take care of multiple generations of family with limited resources.

“These are some of the things that are less talked about as going into the heightened risk for many cardiovascular risk factors, including hypertension, very early in life for Black women that we need to bring to the forefront of conversations,” Dr. Albert said.

“These stressors not only impact hypertension onset but also they impact one’s ability to be able to seek help, and once the help is sought, to be able to sustain the therapies recommended and the interventions recommended,” she added.

The authors reported no relevant disclosures.
 

A version of this article appeared on Medscape.com.

PHOENIX — Black women who develop high blood pressure before age 35 have a threefold increased risk of having a midlife stroke, new observational data suggest. The Black Women’s Health Study, which has followed 59,000 participants in the United States since 1990s, also showed that those who develop hypertension before age 45 have twice the risk of suffering a stroke.

“The really concerning thing about this data is the high proportion of young Black women who had high blood pressure and are suffering strokes relatively early in life,” said the study’s lead author, Hugo J. Aparicio, MD, associate professor of neurology at Boston University Chobanian & Avedisian School of Medicine, Boston. “This can lead to a burden of disability in relatively young women who may be at the prime of their life, pursuing careers, and looking after family.”

Dr. Aparicio presented the data at the International Stroke Conference presented by the American Stroke Association, a division of the American Heart Association.

He explained that while there has been good progress in reducing stroke rates in older people over the past decades, there is a concerning observation from multiple datasets showing that stroke rates in midlife have been plateauing or even increasing in recent years.

“For Black women specifically, there is a concern, as we know this group has higher rates of raised blood pressure and stroke overall,” said Dr. Aparicio. “We were interested in looking at whether the onset of hypertension at an earlier age in this group is one of the reasons for the increased stroke risk in midlife.”
 

A Large Study Cohort

The researchers analyzed data from the Black Women’s Health Study; the baseline year for this analysis, which included 46,754 stroke-free participants younger than age 65 (mean age, 42 years), was the 1999 questionnaire.

Both history of hypertension — defined as physician-diagnosed hypertension with the use of an antihypertensive medication — and stroke occurrence were determined by self-report. It has been shown in previous studies that these self-reported data on incidence of hypertension in this dataset are highly reliable, Dr. Aparicio noted.

At baseline, 10.5% of participants aged 45-64 years had hypertension. Stroke occurred in 3.2% of individuals over a mean follow-up of 17 years.

Black women with hypertension before age 45 had a higher risk for midlife stroke (hazard ratio [HR], 2.23; 95% confidence interval [CI], 1.79-2.78), after adjustment for age, neighborhood socioeconomic status, residence in the Stroke Belt, smoking, body mass index, and diabetes than women with no history of hypertension.

The risk was also increased with hypertension at midlife ages 45-64 years (HR, 1.69; 95% CI, 1.47-1.95) and was highest among those with hypertension at ages 24-34 years (HR, 3.15; 95% CI, 1.92-5.16).

“Our results show that among young Black women, those with hypertension have a much higher stroke risk than those without hypertension, even if they are taking antihypertensive medication,” Dr. Aparicio said. “This underscores how potent hypertension is as a risk factor for stroke.”

He concluded that both individuals and doctors need to realize that hypertension and stroke are not problems of the elderly exclusively.

“These are conditions that need to be addressed very early in life. This is even more important for Black women, as they are a high-risk group. They need to pay attention to blood pressure numbers early in life — ideally from adolescence — to catch levels before they become too elevated,” Dr. Aparicio said.

“We also need to address lifestyle changes including diet, physical activity, sleep habits, and address other cardiovascular risk factors such as cholesterol and body mass index, so we can prevent strokes from occurring,” he added. “At the policy level, we need to advocate, provide and fund primary prevention measures, and enable earlier screening and better treatment.”
 

The Role of Psychosocial Stressors

Commenting on the study, the American Heart Association immediate past president, Michelle A. Albert, MD, professor of medicine at the University of California, San Francisco, emphasized the importance of regular primary care appointments to screen for high blood pressure and other cardiovascular risk factors.

She pointed out that one of the contributing factors that may increase the risk for Black women is their disproportionate experience of psychosocial stressors and chronic cumulative stress.

This could include stress related to financial issues, racism and other forms of bias, the neighborhood environment, and having to take care of multiple generations of family with limited resources.

“These are some of the things that are less talked about as going into the heightened risk for many cardiovascular risk factors, including hypertension, very early in life for Black women that we need to bring to the forefront of conversations,” Dr. Albert said.

“These stressors not only impact hypertension onset but also they impact one’s ability to be able to seek help, and once the help is sought, to be able to sustain the therapies recommended and the interventions recommended,” she added.

The authors reported no relevant disclosures.
 

A version of this article appeared on Medscape.com.

In 2013, a prostate-specific antigen (PSA) blood test revealed that Richard LaFrate’s levels had jumped. 

Previously in a normal range, his PSA was now above 6 ng/mL, indicating an elevated likelihood for prostate cancer. The jazz guitarist from Leesburg, Florida, then 70 years old, underwent a biopsy, which found two Gleason 6 lesions. 

Mr. LaFrate had low-risk prostate cancer.

Guidelines now recommend active surveillance for patients like Mr. LaFrate, who have low-risk disease. This strategy would mean monitoring the cancer until LaFrate required treatment, with the upside being he might never need therapy.

Mr. LaFrate’s urologist, however, was pushing whole gland surgery — an invasive and unnecessary procedure given his diagnosis and age. 

Mr. LaFrate decided to look for another doctor. He filled out a form online that pointed him to a new urologist who offered him one option: An investigational procedure known as high-intensity focused ultrasound.

At the time, high-intensity focused ultrasound — a form of focal therapy — was being studied in the United States to treat men with low or intermediate-risk prostate cancer, but it was still relatively early days.

Mr. LaFrate’s urologist asked him to pay $25,000 out of pocket to undergo the focal procedure at a clinic in the Bahamas. He refused and, ultimately, landed on active surveillance as the best strategy to manage for his low-risk disease.

That urologist was “a shyster in my opinion,” Mr. LaFrate said. 

Over the past 10 years, the popularity of focal therapy has grown among men with intermediate-risk prostate cancer — Gleason 3+4 (grade group 2) tumors — as an alternative to invasive surgery and active surveillance. Prestigious medical centers, such as Cleveland Clinic, Mayo Clinic, Memorial Sloan Kettering, UCLA, and the University of Chicago, routinely offer focal therapy. 

But use of the techniques remains controversial and costly.

As the Cleveland Clinic’s website acknowledges, although “the use of focal therapy for localized prostate cancer appears to be a promising development in a number of ways, it is still considered investigational and not yet part of standard therapy.” Major caveats to focal therapy include unknown long-term effectiveness, the possibility of leaving behind untreated cancer, and higher overall costs. 

No major national guidelines endorse the use of focal therapy, unless offered in a research or clinical trial setting. Insurance companies, such as Aetna, Blue Cross Blue Shield, and United, also consider focal therapy for prostate cancer investigational and don’t cover it.

Without a stamp of approval from guideline bodies and insurance companies, patients, like Mr. LaFrate, remain vulnerable to the high out-of-pocket costs for these focal techniques. 

“Almost every place charges $15,000-$30,000 in cash,” said Daniel Spratt, MD, radiation oncology chair at University Hospitals Seidman Cancer Center and Case Western Reserve University in Cleveland. 

Dr. Spratt has seen hundreds of patients after focal therapy, some from prominent centers, who have emptied their bank accounts to undergo treatment with the promise of great results and ultimately felt misled when the cancer has recurred.

“It pains me that there are doctors willing to ignore the Hippocratic oath of ‘Do No Harm’ simply to jump on this fad to bring in revenue,” Dr. Spratt said. 
 

Evidence-Based or Oversold?

Focal therapy gained a foothold in the United Kingdom well before the United States.

Hashim Ahmed, FRCS, urology chair at Imperial College London, has used focal therapy for 15 years, treated over 1000 patients, and taught dozens of surgeons how to use the leading focal therapies — focal cryoablation, in which surgeons use a needle-thin probe to target, freeze, and kill prostate tumors, as well as high-intensity focused ultrasound, which uses sound wave energy to superheat and kill tumors.

“Certainly, in the United Kingdom, focal therapy has been prime time in a number of centers for a number of years,” Dr. Ahmed said. 

In the United States, focal therapy has become an attractive option for men with prostate cancer who want to avoid radiation or radical prostatectomy but don’t feel comfortable simply monitoring their disease with active surveillance. Experts from specialized focal therapy centers touting the promise of this “innovative technique” predict its routine use in the next few years.

But the excitement surrounding the use of focal therapy in prostate cancer has outpaced broader acceptance.

In 2015, the FDA approved high-intensity focused ultrasound to treat prostatic disease, but not prostate cancer specifically. Although the approval language “means that companies cannot advertise that their devices can be used for prostate cancer,” physicians can still determine how to use the technology, which includes treating prostate cancer, Dr. Ahmed said. 

The evidence is starting to catch up to the demand. The latest research suggests that the partial-gland techniques may stand up well to radical prostatectomy.

A 2022 prospective database study comparing radical prostatectomies to focal therapy — mostly high-intensity focused ultrasound — in more than 800 men found similar rates of failure-free survival in the two groups at the 8-year follow-up. A 2019 registry study found that failure-free survival at 3 years was just over 90% in high and intermediate-risk patients receiving focal cryotherapy, with the rate rising to about 93% for the intermediate-risk group. And a 2018 prospective study of 625 patients with intermediate or high-risk prostate cancer who underwent high-intensity focused ultrasound had 5-year metastasis-free survival of 98% and overall survival rates of 100%.

One of the biggest draws of focal therapy vs more aggressive treatments is the “massive differences in side-effect profiles,” said Dr. Ahmed.

In a 2021 meta-analysis, researchers found that 6 months after high-intensity focused ultrasound, 98% of patients remained continent and 80% retained erectile function, while erectile dysfunction can occur in 30% to as many as 85% of patients following prostatectomy or radiotherapy and urinary incontinence can occur in as many as 40% of patients.

Despite these potential advantages of focal therapy, the long-term efficacy of the techniques remains uncertain.

A recent study from a team at MSK, for instance, reported that 40% of men with intermediate (grade 2) or high-risk (grade 3) disease had residual cancer following MRI-guided focused ultrasound. A 2020 prospective registry study found that almost 20% of patients undergoing high-intensity focal ultrasound required a second round following a recurrence. 

Dr. Spratt worries that patients who recur after focal therapy may go on to receive a second round — often offered at half price — and will still ultimately need surgery or radiation therapy later. By that point, however, patients may have spent as much as $45,000 — ie, $30,000 on the initial and another $15,000 on the follow-up procedure.

When patients see Dr. Spratt after a recurrence, he informs them that their side effects will be worse if he gives them radiation or surgery now vs if he had given them curative therapy upfront. “But this is what we’re left with,” he tells them.

Another big concern in the field is “the quality of data for focal therapy is overwhelmingly poor,” said Jonathan Shoag, MD, a urologic oncologist at University Hospitals and an associate professor of urology at Case Western Reserve University School of Medicine in Cleveland. “Essentially, the bulk of the data is from single-institution retrospective series without defined follow-up protocols or endpoints.”

The American Urological Association (AUA) has even cautioned experts and patients about the lack of high-quality data comparing focal therapy techniques to radiation therapy, surgery, and active surveillance. According to the AUA, focal options should only be considered in intermediate-risk prostate cancer in a clinical trial setting.

“The lack of randomized clinical trials poses a major stumbling block for the field,” said Dr. Ahmed.

Although randomized trials would be ideal, the results would take many years to mature, and growing patient demand for these less invasive focal procedures has made randomized trials difficult to complete, explained Arvin George, MD, associate professor at Johns Hopkins School of Medicine in Baltimore. Several randomized trials attempted in Norway and the United Kingdom, for instance, fell apart when patients refused to be randomized between focal and radical therapy, Dr. George said.

Focal therapy is now in the same position that active surveillance was a few years ago, according to Dr. George.

“We are hearing the same concerns about focal therapy now as we did about active surveillance,” he said. The initial evidence supporting active surveillance largely came from real-world experience and retrospective studies. The randomized data came later, and skeptics of active surveillance “were proven wrong,” he added.

But Dr. Shoag has a different take on the trajectory of focal therapy research and care in the United States. 

“I think there’s this emerging kind of tragedy happening in our field now, where you have even academic institutions offering focal therapy to patients off-trial with essentially no data to suggest it is oncologically effective,” Dr. Shoag said.

William Catalona, MD, Northwestern University Feinberg School of Medicine, Chicago, agreed, noting that too many low-risk patients are undergoing focal treatment who should be on active surveillance. “Many men are attracted to focal because they just are uncomfortable having a cancer in their body that’s not treated,” Dr. Catalona said. But “giving these patients focal therapy is really overtreatment.”

Patients with higher-risk disease who want to avoid aggressive treatment are also being lured into focal without guidelines or clear evidence to back up that option, Dr. Catalona explained.

Although it’s not clear how many men in the United States are receiving focal therapy who shouldn’t, even proponents of focal therapy, like George, have expressed concern.

Dr. George agreed that focal therapy marketing geared towards patients is drawing in some men who are not good candidates for these techniques, and feels there’s not enough objective material from medical societies or academic centers giving patients a realistic picture of focal therapy. 

“There is concern that patients may be receiving biased information,” Dr. George said, adding that it’s ultimately up to the physician to reconcile the best available evidence, understand the outcomes, and discuss these options with the patient to guide them to what’s best.

At the end of the day, Dr. Spratt said, physicians giving focal therapy off a clinical trial need to pause and ask themselves “why are they giving a treatment that remains investigational by payers, not recommended by any major guideline, and that lacks any randomized evidence?” 

Mr. LaFrate does not regret his decision to forgo focal therapy in 2013. He has been on active surveillance for about a decade now.

Following an MRI in 2022, Mr. LaFrate’s radiology report found that “clinically significant cancer is very unlikely to be present.”

Still, his PSA has risen two points in the past year to 14. His current urologist feels that the PSA is going up because there’s cancer present and is suggesting focal therapy for Mr. LaFrate.

Mr. LaFrate, who has prostate enlargement issues, remains skeptical of focal therapy and is still resisting the sales pitch.

“My doctor is not aggressively pushing it. He’s just giving me that as one of my options,” he said. “I just have a hunch I don’t need it at this point.”

A version of this article appeared on Medscape.com.

In 2013, a prostate-specific antigen (PSA) blood test revealed that Richard LaFrate’s levels had jumped. 

Previously in a normal range, his PSA was now above 6 ng/mL, indicating an elevated likelihood for prostate cancer. The jazz guitarist from Leesburg, Florida, then 70 years old, underwent a biopsy, which found two Gleason 6 lesions. 

Mr. LaFrate had low-risk prostate cancer.

Guidelines now recommend active surveillance for patients like Mr. LaFrate, who have low-risk disease. This strategy would mean monitoring the cancer until LaFrate required treatment, with the upside being he might never need therapy.

Mr. LaFrate’s urologist, however, was pushing whole gland surgery — an invasive and unnecessary procedure given his diagnosis and age. 

Mr. LaFrate decided to look for another doctor. He filled out a form online that pointed him to a new urologist who offered him one option: An investigational procedure known as high-intensity focused ultrasound.

At the time, high-intensity focused ultrasound — a form of focal therapy — was being studied in the United States to treat men with low or intermediate-risk prostate cancer, but it was still relatively early days.

Mr. LaFrate’s urologist asked him to pay $25,000 out of pocket to undergo the focal procedure at a clinic in the Bahamas. He refused and, ultimately, landed on active surveillance as the best strategy to manage for his low-risk disease.

That urologist was “a shyster in my opinion,” Mr. LaFrate said. 

Over the past 10 years, the popularity of focal therapy has grown among men with intermediate-risk prostate cancer — Gleason 3+4 (grade group 2) tumors — as an alternative to invasive surgery and active surveillance. Prestigious medical centers, such as Cleveland Clinic, Mayo Clinic, Memorial Sloan Kettering, UCLA, and the University of Chicago, routinely offer focal therapy. 

But use of the techniques remains controversial and costly.

As the Cleveland Clinic’s website acknowledges, although “the use of focal therapy for localized prostate cancer appears to be a promising development in a number of ways, it is still considered investigational and not yet part of standard therapy.” Major caveats to focal therapy include unknown long-term effectiveness, the possibility of leaving behind untreated cancer, and higher overall costs. 

No major national guidelines endorse the use of focal therapy, unless offered in a research or clinical trial setting. Insurance companies, such as Aetna, Blue Cross Blue Shield, and United, also consider focal therapy for prostate cancer investigational and don’t cover it.

Without a stamp of approval from guideline bodies and insurance companies, patients, like Mr. LaFrate, remain vulnerable to the high out-of-pocket costs for these focal techniques. 

“Almost every place charges $15,000-$30,000 in cash,” said Daniel Spratt, MD, radiation oncology chair at University Hospitals Seidman Cancer Center and Case Western Reserve University in Cleveland. 

Dr. Spratt has seen hundreds of patients after focal therapy, some from prominent centers, who have emptied their bank accounts to undergo treatment with the promise of great results and ultimately felt misled when the cancer has recurred.

“It pains me that there are doctors willing to ignore the Hippocratic oath of ‘Do No Harm’ simply to jump on this fad to bring in revenue,” Dr. Spratt said. 
 

Evidence-Based or Oversold?

Focal therapy gained a foothold in the United Kingdom well before the United States.

Hashim Ahmed, FRCS, urology chair at Imperial College London, has used focal therapy for 15 years, treated over 1000 patients, and taught dozens of surgeons how to use the leading focal therapies — focal cryoablation, in which surgeons use a needle-thin probe to target, freeze, and kill prostate tumors, as well as high-intensity focused ultrasound, which uses sound wave energy to superheat and kill tumors.

“Certainly, in the United Kingdom, focal therapy has been prime time in a number of centers for a number of years,” Dr. Ahmed said. 

In the United States, focal therapy has become an attractive option for men with prostate cancer who want to avoid radiation or radical prostatectomy but don’t feel comfortable simply monitoring their disease with active surveillance. Experts from specialized focal therapy centers touting the promise of this “innovative technique” predict its routine use in the next few years.

But the excitement surrounding the use of focal therapy in prostate cancer has outpaced broader acceptance.

In 2015, the FDA approved high-intensity focused ultrasound to treat prostatic disease, but not prostate cancer specifically. Although the approval language “means that companies cannot advertise that their devices can be used for prostate cancer,” physicians can still determine how to use the technology, which includes treating prostate cancer, Dr. Ahmed said. 

The evidence is starting to catch up to the demand. The latest research suggests that the partial-gland techniques may stand up well to radical prostatectomy.

A 2022 prospective database study comparing radical prostatectomies to focal therapy — mostly high-intensity focused ultrasound — in more than 800 men found similar rates of failure-free survival in the two groups at the 8-year follow-up. A 2019 registry study found that failure-free survival at 3 years was just over 90% in high and intermediate-risk patients receiving focal cryotherapy, with the rate rising to about 93% for the intermediate-risk group. And a 2018 prospective study of 625 patients with intermediate or high-risk prostate cancer who underwent high-intensity focused ultrasound had 5-year metastasis-free survival of 98% and overall survival rates of 100%.

One of the biggest draws of focal therapy vs more aggressive treatments is the “massive differences in side-effect profiles,” said Dr. Ahmed.

In a 2021 meta-analysis, researchers found that 6 months after high-intensity focused ultrasound, 98% of patients remained continent and 80% retained erectile function, while erectile dysfunction can occur in 30% to as many as 85% of patients following prostatectomy or radiotherapy and urinary incontinence can occur in as many as 40% of patients.

Despite these potential advantages of focal therapy, the long-term efficacy of the techniques remains uncertain.

A recent study from a team at MSK, for instance, reported that 40% of men with intermediate (grade 2) or high-risk (grade 3) disease had residual cancer following MRI-guided focused ultrasound. A 2020 prospective registry study found that almost 20% of patients undergoing high-intensity focal ultrasound required a second round following a recurrence. 

Dr. Spratt worries that patients who recur after focal therapy may go on to receive a second round — often offered at half price — and will still ultimately need surgery or radiation therapy later. By that point, however, patients may have spent as much as $45,000 — ie, $30,000 on the initial and another $15,000 on the follow-up procedure.

When patients see Dr. Spratt after a recurrence, he informs them that their side effects will be worse if he gives them radiation or surgery now vs if he had given them curative therapy upfront. “But this is what we’re left with,” he tells them.

Another big concern in the field is “the quality of data for focal therapy is overwhelmingly poor,” said Jonathan Shoag, MD, a urologic oncologist at University Hospitals and an associate professor of urology at Case Western Reserve University School of Medicine in Cleveland. “Essentially, the bulk of the data is from single-institution retrospective series without defined follow-up protocols or endpoints.”

The American Urological Association (AUA) has even cautioned experts and patients about the lack of high-quality data comparing focal therapy techniques to radiation therapy, surgery, and active surveillance. According to the AUA, focal options should only be considered in intermediate-risk prostate cancer in a clinical trial setting.

“The lack of randomized clinical trials poses a major stumbling block for the field,” said Dr. Ahmed.

Although randomized trials would be ideal, the results would take many years to mature, and growing patient demand for these less invasive focal procedures has made randomized trials difficult to complete, explained Arvin George, MD, associate professor at Johns Hopkins School of Medicine in Baltimore. Several randomized trials attempted in Norway and the United Kingdom, for instance, fell apart when patients refused to be randomized between focal and radical therapy, Dr. George said.

Focal therapy is now in the same position that active surveillance was a few years ago, according to Dr. George.

“We are hearing the same concerns about focal therapy now as we did about active surveillance,” he said. The initial evidence supporting active surveillance largely came from real-world experience and retrospective studies. The randomized data came later, and skeptics of active surveillance “were proven wrong,” he added.

But Dr. Shoag has a different take on the trajectory of focal therapy research and care in the United States. 

“I think there’s this emerging kind of tragedy happening in our field now, where you have even academic institutions offering focal therapy to patients off-trial with essentially no data to suggest it is oncologically effective,” Dr. Shoag said.

William Catalona, MD, Northwestern University Feinberg School of Medicine, Chicago, agreed, noting that too many low-risk patients are undergoing focal treatment who should be on active surveillance. “Many men are attracted to focal because they just are uncomfortable having a cancer in their body that’s not treated,” Dr. Catalona said. But “giving these patients focal therapy is really overtreatment.”

Patients with higher-risk disease who want to avoid aggressive treatment are also being lured into focal without guidelines or clear evidence to back up that option, Dr. Catalona explained.

Although it’s not clear how many men in the United States are receiving focal therapy who shouldn’t, even proponents of focal therapy, like George, have expressed concern.

Dr. George agreed that focal therapy marketing geared towards patients is drawing in some men who are not good candidates for these techniques, and feels there’s not enough objective material from medical societies or academic centers giving patients a realistic picture of focal therapy. 

“There is concern that patients may be receiving biased information,” Dr. George said, adding that it’s ultimately up to the physician to reconcile the best available evidence, understand the outcomes, and discuss these options with the patient to guide them to what’s best.

At the end of the day, Dr. Spratt said, physicians giving focal therapy off a clinical trial need to pause and ask themselves “why are they giving a treatment that remains investigational by payers, not recommended by any major guideline, and that lacks any randomized evidence?” 

Mr. LaFrate does not regret his decision to forgo focal therapy in 2013. He has been on active surveillance for about a decade now.

Following an MRI in 2022, Mr. LaFrate’s radiology report found that “clinically significant cancer is very unlikely to be present.”

Still, his PSA has risen two points in the past year to 14. His current urologist feels that the PSA is going up because there’s cancer present and is suggesting focal therapy for Mr. LaFrate.

Mr. LaFrate, who has prostate enlargement issues, remains skeptical of focal therapy and is still resisting the sales pitch.

“My doctor is not aggressively pushing it. He’s just giving me that as one of my options,” he said. “I just have a hunch I don’t need it at this point.”

A version of this article appeared on Medscape.com.

In 2013, a prostate-specific antigen (PSA) blood test revealed that Richard LaFrate’s levels had jumped. 

Previously in a normal range, his PSA was now above 6 ng/mL, indicating an elevated likelihood for prostate cancer. The jazz guitarist from Leesburg, Florida, then 70 years old, underwent a biopsy, which found two Gleason 6 lesions. 

Mr. LaFrate had low-risk prostate cancer.

Guidelines now recommend active surveillance for patients like Mr. LaFrate, who have low-risk disease. This strategy would mean monitoring the cancer until LaFrate required treatment, with the upside being he might never need therapy.

Mr. LaFrate’s urologist, however, was pushing whole gland surgery — an invasive and unnecessary procedure given his diagnosis and age. 

Mr. LaFrate decided to look for another doctor. He filled out a form online that pointed him to a new urologist who offered him one option: An investigational procedure known as high-intensity focused ultrasound.

At the time, high-intensity focused ultrasound — a form of focal therapy — was being studied in the United States to treat men with low or intermediate-risk prostate cancer, but it was still relatively early days.

Mr. LaFrate’s urologist asked him to pay $25,000 out of pocket to undergo the focal procedure at a clinic in the Bahamas. He refused and, ultimately, landed on active surveillance as the best strategy to manage for his low-risk disease.

That urologist was “a shyster in my opinion,” Mr. LaFrate said. 

Over the past 10 years, the popularity of focal therapy has grown among men with intermediate-risk prostate cancer — Gleason 3+4 (grade group 2) tumors — as an alternative to invasive surgery and active surveillance. Prestigious medical centers, such as Cleveland Clinic, Mayo Clinic, Memorial Sloan Kettering, UCLA, and the University of Chicago, routinely offer focal therapy. 

But use of the techniques remains controversial and costly.

As the Cleveland Clinic’s website acknowledges, although “the use of focal therapy for localized prostate cancer appears to be a promising development in a number of ways, it is still considered investigational and not yet part of standard therapy.” Major caveats to focal therapy include unknown long-term effectiveness, the possibility of leaving behind untreated cancer, and higher overall costs. 

No major national guidelines endorse the use of focal therapy, unless offered in a research or clinical trial setting. Insurance companies, such as Aetna, Blue Cross Blue Shield, and United, also consider focal therapy for prostate cancer investigational and don’t cover it.

Without a stamp of approval from guideline bodies and insurance companies, patients, like Mr. LaFrate, remain vulnerable to the high out-of-pocket costs for these focal techniques. 

“Almost every place charges $15,000-$30,000 in cash,” said Daniel Spratt, MD, radiation oncology chair at University Hospitals Seidman Cancer Center and Case Western Reserve University in Cleveland. 

Dr. Spratt has seen hundreds of patients after focal therapy, some from prominent centers, who have emptied their bank accounts to undergo treatment with the promise of great results and ultimately felt misled when the cancer has recurred.

“It pains me that there are doctors willing to ignore the Hippocratic oath of ‘Do No Harm’ simply to jump on this fad to bring in revenue,” Dr. Spratt said. 
 

Evidence-Based or Oversold?

Focal therapy gained a foothold in the United Kingdom well before the United States.

Hashim Ahmed, FRCS, urology chair at Imperial College London, has used focal therapy for 15 years, treated over 1000 patients, and taught dozens of surgeons how to use the leading focal therapies — focal cryoablation, in which surgeons use a needle-thin probe to target, freeze, and kill prostate tumors, as well as high-intensity focused ultrasound, which uses sound wave energy to superheat and kill tumors.

“Certainly, in the United Kingdom, focal therapy has been prime time in a number of centers for a number of years,” Dr. Ahmed said. 

In the United States, focal therapy has become an attractive option for men with prostate cancer who want to avoid radiation or radical prostatectomy but don’t feel comfortable simply monitoring their disease with active surveillance. Experts from specialized focal therapy centers touting the promise of this “innovative technique” predict its routine use in the next few years.

But the excitement surrounding the use of focal therapy in prostate cancer has outpaced broader acceptance.

In 2015, the FDA approved high-intensity focused ultrasound to treat prostatic disease, but not prostate cancer specifically. Although the approval language “means that companies cannot advertise that their devices can be used for prostate cancer,” physicians can still determine how to use the technology, which includes treating prostate cancer, Dr. Ahmed said. 

The evidence is starting to catch up to the demand. The latest research suggests that the partial-gland techniques may stand up well to radical prostatectomy.

A 2022 prospective database study comparing radical prostatectomies to focal therapy — mostly high-intensity focused ultrasound — in more than 800 men found similar rates of failure-free survival in the two groups at the 8-year follow-up. A 2019 registry study found that failure-free survival at 3 years was just over 90% in high and intermediate-risk patients receiving focal cryotherapy, with the rate rising to about 93% for the intermediate-risk group. And a 2018 prospective study of 625 patients with intermediate or high-risk prostate cancer who underwent high-intensity focused ultrasound had 5-year metastasis-free survival of 98% and overall survival rates of 100%.

One of the biggest draws of focal therapy vs more aggressive treatments is the “massive differences in side-effect profiles,” said Dr. Ahmed.

In a 2021 meta-analysis, researchers found that 6 months after high-intensity focused ultrasound, 98% of patients remained continent and 80% retained erectile function, while erectile dysfunction can occur in 30% to as many as 85% of patients following prostatectomy or radiotherapy and urinary incontinence can occur in as many as 40% of patients.

Despite these potential advantages of focal therapy, the long-term efficacy of the techniques remains uncertain.

A recent study from a team at MSK, for instance, reported that 40% of men with intermediate (grade 2) or high-risk (grade 3) disease had residual cancer following MRI-guided focused ultrasound. A 2020 prospective registry study found that almost 20% of patients undergoing high-intensity focal ultrasound required a second round following a recurrence. 

Dr. Spratt worries that patients who recur after focal therapy may go on to receive a second round — often offered at half price — and will still ultimately need surgery or radiation therapy later. By that point, however, patients may have spent as much as $45,000 — ie, $30,000 on the initial and another $15,000 on the follow-up procedure.

When patients see Dr. Spratt after a recurrence, he informs them that their side effects will be worse if he gives them radiation or surgery now vs if he had given them curative therapy upfront. “But this is what we’re left with,” he tells them.

Another big concern in the field is “the quality of data for focal therapy is overwhelmingly poor,” said Jonathan Shoag, MD, a urologic oncologist at University Hospitals and an associate professor of urology at Case Western Reserve University School of Medicine in Cleveland. “Essentially, the bulk of the data is from single-institution retrospective series without defined follow-up protocols or endpoints.”

The American Urological Association (AUA) has even cautioned experts and patients about the lack of high-quality data comparing focal therapy techniques to radiation therapy, surgery, and active surveillance. According to the AUA, focal options should only be considered in intermediate-risk prostate cancer in a clinical trial setting.

“The lack of randomized clinical trials poses a major stumbling block for the field,” said Dr. Ahmed.

Although randomized trials would be ideal, the results would take many years to mature, and growing patient demand for these less invasive focal procedures has made randomized trials difficult to complete, explained Arvin George, MD, associate professor at Johns Hopkins School of Medicine in Baltimore. Several randomized trials attempted in Norway and the United Kingdom, for instance, fell apart when patients refused to be randomized between focal and radical therapy, Dr. George said.

Focal therapy is now in the same position that active surveillance was a few years ago, according to Dr. George.

“We are hearing the same concerns about focal therapy now as we did about active surveillance,” he said. The initial evidence supporting active surveillance largely came from real-world experience and retrospective studies. The randomized data came later, and skeptics of active surveillance “were proven wrong,” he added.

But Dr. Shoag has a different take on the trajectory of focal therapy research and care in the United States. 

“I think there’s this emerging kind of tragedy happening in our field now, where you have even academic institutions offering focal therapy to patients off-trial with essentially no data to suggest it is oncologically effective,” Dr. Shoag said.

William Catalona, MD, Northwestern University Feinberg School of Medicine, Chicago, agreed, noting that too many low-risk patients are undergoing focal treatment who should be on active surveillance. “Many men are attracted to focal because they just are uncomfortable having a cancer in their body that’s not treated,” Dr. Catalona said. But “giving these patients focal therapy is really overtreatment.”

Patients with higher-risk disease who want to avoid aggressive treatment are also being lured into focal without guidelines or clear evidence to back up that option, Dr. Catalona explained.

Although it’s not clear how many men in the United States are receiving focal therapy who shouldn’t, even proponents of focal therapy, like George, have expressed concern.

Dr. George agreed that focal therapy marketing geared towards patients is drawing in some men who are not good candidates for these techniques, and feels there’s not enough objective material from medical societies or academic centers giving patients a realistic picture of focal therapy. 

“There is concern that patients may be receiving biased information,” Dr. George said, adding that it’s ultimately up to the physician to reconcile the best available evidence, understand the outcomes, and discuss these options with the patient to guide them to what’s best.

At the end of the day, Dr. Spratt said, physicians giving focal therapy off a clinical trial need to pause and ask themselves “why are they giving a treatment that remains investigational by payers, not recommended by any major guideline, and that lacks any randomized evidence?” 

Mr. LaFrate does not regret his decision to forgo focal therapy in 2013. He has been on active surveillance for about a decade now.

Following an MRI in 2022, Mr. LaFrate’s radiology report found that “clinically significant cancer is very unlikely to be present.”

Still, his PSA has risen two points in the past year to 14. His current urologist feels that the PSA is going up because there’s cancer present and is suggesting focal therapy for Mr. LaFrate.

Mr. LaFrate, who has prostate enlargement issues, remains skeptical of focal therapy and is still resisting the sales pitch.

“My doctor is not aggressively pushing it. He’s just giving me that as one of my options,” he said. “I just have a hunch I don’t need it at this point.”

A version of this article appeared on Medscape.com.

TOPLINE:

Transcatheter arterial chemoembolization (TACE), a standard treatment for liver metastases from colorectal cancer, shows promise for treating locally advanced rectal tumors.

METHODOLOGY:

• The combination of neoadjuvant chemoradiotherapy, total mesorectal excision, and postoperative adjuvant chemotherapy is the current standard of care for locally advanced rectal cancer. But with pathological complete response rates of only 10%-15% and more than 30% of patients developing distant metastases within 3 years, outcomes remain suboptimal.
• Chinese investigators took a step to improve the situation, applying TACE — a standard treatment for colorectal liver metastases — to rectal tumors, dubbing the approach transcatheter rectal arterial chemoembolization (TRACE).
• As in TACE, TRACE uses precisely injected chemotherapeutic and vaso-occlusive agents to shut down blood flow to tumors, starving them of oxygen and nutrients.
• The research team tried the approach in 111 patients with stage II or III rectal tumors and performance status scores of 0-1.
• TRACE was delivered with oxaliplatin and followed by radiotherapy and S1 chemotherapy (tegafur, gimeracil, and potassium oteracil). Total mesorectal excisions were performed 4-8 weeks later, followed by mFOLFOX6 (5-fluorouracil, leucovorin, and oxaliplatin) or CAPOX (oxaliplatin and capecitabine) chemotherapy for 4-6 months.

TAKEAWAY:

• Overall, 20.7% of patients undergoing TRACE had a pathological complete response, and almost half (48.65%) had a major pathological response.
• Nearly 62% of patients were disease-free at 5 years, and almost 75% were alive at 5 years.
• No serious surgical complications occurred, but 21.6% of patients had postoperative complications. Overall, about 26% of patients (29 of 111) had grade 3/4 toxicities.

IN PRACTICE:

“The addition of transcatheter rectal arterial chemoembolisation to the neoadjuvant therapy can improve the pathological remission rate and prognosis in patients with locally advanced rectal cancer, without increasing the incidence of preoperative adverse events and postoperative complications,” the researchers concluded. “Given its promising effectiveness and safe profile, incorporating TRACE into the standard treatment strategy for patients with [locally advanced rectal cancer] should be considered.”

SOURCE:

The work, led by W. Yang of the Army Medical University in Chongqing, China, was published in Clinical Oncology.

LIMITATIONS:

The study was performed at a single center with no control arm in a Chinese population.

DISCLOSURES:

The work was funded by the Third Military Medical University in China. The investigators had no conflicts of interest.

A version of this article appeared on Medscape.com.

TOPLINE:

Transcatheter arterial chemoembolization (TACE), a standard treatment for liver metastases from colorectal cancer, shows promise for treating locally advanced rectal tumors.

METHODOLOGY:

• The combination of neoadjuvant chemoradiotherapy, total mesorectal excision, and postoperative adjuvant chemotherapy is the current standard of care for locally advanced rectal cancer. But with pathological complete response rates of only 10%-15% and more than 30% of patients developing distant metastases within 3 years, outcomes remain suboptimal.
• Chinese investigators took a step to improve the situation, applying TACE — a standard treatment for colorectal liver metastases — to rectal tumors, dubbing the approach transcatheter rectal arterial chemoembolization (TRACE).
• As in TACE, TRACE uses precisely injected chemotherapeutic and vaso-occlusive agents to shut down blood flow to tumors, starving them of oxygen and nutrients.
• The research team tried the approach in 111 patients with stage II or III rectal tumors and performance status scores of 0-1.
• TRACE was delivered with oxaliplatin and followed by radiotherapy and S1 chemotherapy (tegafur, gimeracil, and potassium oteracil). Total mesorectal excisions were performed 4-8 weeks later, followed by mFOLFOX6 (5-fluorouracil, leucovorin, and oxaliplatin) or CAPOX (oxaliplatin and capecitabine) chemotherapy for 4-6 months.

TAKEAWAY:

• Overall, 20.7% of patients undergoing TRACE had a pathological complete response, and almost half (48.65%) had a major pathological response.
• Nearly 62% of patients were disease-free at 5 years, and almost 75% were alive at 5 years.
• No serious surgical complications occurred, but 21.6% of patients had postoperative complications. Overall, about 26% of patients (29 of 111) had grade 3/4 toxicities.

IN PRACTICE:

“The addition of transcatheter rectal arterial chemoembolisation to the neoadjuvant therapy can improve the pathological remission rate and prognosis in patients with locally advanced rectal cancer, without increasing the incidence of preoperative adverse events and postoperative complications,” the researchers concluded. “Given its promising effectiveness and safe profile, incorporating TRACE into the standard treatment strategy for patients with [locally advanced rectal cancer] should be considered.”

SOURCE:

The work, led by W. Yang of the Army Medical University in Chongqing, China, was published in Clinical Oncology.

LIMITATIONS:

The study was performed at a single center with no control arm in a Chinese population.

DISCLOSURES:

The work was funded by the Third Military Medical University in China. The investigators had no conflicts of interest.

A version of this article appeared on Medscape.com.

TOPLINE:

Transcatheter arterial chemoembolization (TACE), a standard treatment for liver metastases from colorectal cancer, shows promise for treating locally advanced rectal tumors.

METHODOLOGY:

• The combination of neoadjuvant chemoradiotherapy, total mesorectal excision, and postoperative adjuvant chemotherapy is the current standard of care for locally advanced rectal cancer. But with pathological complete response rates of only 10%-15% and more than 30% of patients developing distant metastases within 3 years, outcomes remain suboptimal.
• Chinese investigators took a step to improve the situation, applying TACE — a standard treatment for colorectal liver metastases — to rectal tumors, dubbing the approach transcatheter rectal arterial chemoembolization (TRACE).
• As in TACE, TRACE uses precisely injected chemotherapeutic and vaso-occlusive agents to shut down blood flow to tumors, starving them of oxygen and nutrients.
• The research team tried the approach in 111 patients with stage II or III rectal tumors and performance status scores of 0-1.
• TRACE was delivered with oxaliplatin and followed by radiotherapy and S1 chemotherapy (tegafur, gimeracil, and potassium oteracil). Total mesorectal excisions were performed 4-8 weeks later, followed by mFOLFOX6 (5-fluorouracil, leucovorin, and oxaliplatin) or CAPOX (oxaliplatin and capecitabine) chemotherapy for 4-6 months.

TAKEAWAY:

• Overall, 20.7% of patients undergoing TRACE had a pathological complete response, and almost half (48.65%) had a major pathological response.
• Nearly 62% of patients were disease-free at 5 years, and almost 75% were alive at 5 years.
• No serious surgical complications occurred, but 21.6% of patients had postoperative complications. Overall, about 26% of patients (29 of 111) had grade 3/4 toxicities.

IN PRACTICE:

“The addition of transcatheter rectal arterial chemoembolisation to the neoadjuvant therapy can improve the pathological remission rate and prognosis in patients with locally advanced rectal cancer, without increasing the incidence of preoperative adverse events and postoperative complications,” the researchers concluded. “Given its promising effectiveness and safe profile, incorporating TRACE into the standard treatment strategy for patients with [locally advanced rectal cancer] should be considered.”

SOURCE:

The work, led by W. Yang of the Army Medical University in Chongqing, China, was published in Clinical Oncology.

LIMITATIONS:

The study was performed at a single center with no control arm in a Chinese population.

DISCLOSURES:

The work was funded by the Third Military Medical University in China. The investigators had no conflicts of interest.

A version of this article appeared on Medscape.com.

On February 14, 2024, Galderma announced that the Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for nemolizumab for the treatment of patients with prurigo nodularis and for adolescents and adults with moderate to severe atopic dermatitis.

A first-in-class investigational monoclonal antibody specifically designed to inhibit interleukin (IL) IL-31 signaling, nemolizumab has also been granted FDA Priority Review for prurigo nodularis, according to a press release from the company. The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab for both prurigo nodularis and atopic dermatitis.

The regulatory developments follow data from the phase III OLYMPIA clinical trial program, which evaluated the efficacy and safety of nemolizumab administered subcutaneously every 4 weeks in patients with prurigo nodularis (NCT04501679 and NCT04501666). According to the press release, in OLYMPIA 1 and 2, 58% and 56% of patients, respectively, achieved at least a least four-point reduction in itch intensity as measured by the peak-pruritus numerical rating scale (PP-NRS), compared with 17% and 21% in the placebo groups (P < .0001). At the same time, 26% and 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions on the investigator’s global assessment (IGA) score, compared with 7% and 11% in the placebo groups (P < .0001).

On February 14, 2024, Galderma announced that the Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for nemolizumab for the treatment of patients with prurigo nodularis and for adolescents and adults with moderate to severe atopic dermatitis.

A first-in-class investigational monoclonal antibody specifically designed to inhibit interleukin (IL) IL-31 signaling, nemolizumab has also been granted FDA Priority Review for prurigo nodularis, according to a press release from the company. The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab for both prurigo nodularis and atopic dermatitis.

The regulatory developments follow data from the phase III OLYMPIA clinical trial program, which evaluated the efficacy and safety of nemolizumab administered subcutaneously every 4 weeks in patients with prurigo nodularis (NCT04501679 and NCT04501666). According to the press release, in OLYMPIA 1 and 2, 58% and 56% of patients, respectively, achieved at least a least four-point reduction in itch intensity as measured by the peak-pruritus numerical rating scale (PP-NRS), compared with 17% and 21% in the placebo groups (P < .0001). At the same time, 26% and 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions on the investigator’s global assessment (IGA) score, compared with 7% and 11% in the placebo groups (P < .0001).

On February 14, 2024, Galderma announced that the Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for nemolizumab for the treatment of patients with prurigo nodularis and for adolescents and adults with moderate to severe atopic dermatitis.

A first-in-class investigational monoclonal antibody specifically designed to inhibit interleukin (IL) IL-31 signaling, nemolizumab has also been granted FDA Priority Review for prurigo nodularis, according to a press release from the company. The European Medicines Agency has also accepted Galderma’s Marketing Authorization Applications for nemolizumab for both prurigo nodularis and atopic dermatitis.

The regulatory developments follow data from the phase III OLYMPIA clinical trial program, which evaluated the efficacy and safety of nemolizumab administered subcutaneously every 4 weeks in patients with prurigo nodularis (NCT04501679 and NCT04501666). According to the press release, in OLYMPIA 1 and 2, 58% and 56% of patients, respectively, achieved at least a least four-point reduction in itch intensity as measured by the peak-pruritus numerical rating scale (PP-NRS), compared with 17% and 21% in the placebo groups (P < .0001). At the same time, 26% and 38% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions on the investigator’s global assessment (IGA) score, compared with 7% and 11% in the placebo groups (P < .0001).

TOPLINE:

ChatGPT (version 3.5) provides relatively poor and inconsistent responses when asked about appropriate colorectal cancer (CRC) screening and surveillance, a new study showed.

METHODOLOGY:

• Three board-certified gastroenterologists with 10+ years of clinical experience developed five CRC screening and five CRC surveillance clinical vignettes (with multiple choice answers), which were fed to ChatGPT version 3.5.
• ChatGPT’s responses were recorded over four separate sessions and screened for accuracy to determine reliability of the tool.
• The average number of correct answers was compared to that of 238 gastroenterologists and colorectal surgeons answering the same questions with and without the help of a previously validated CRC screening mobile app.

TAKEAWAY:

• ChatGPT’s average overall performance was 45%; the average number of correct answers was 2.75 for screening and 1.75 for surveillance.
• ChatGPT’s responses were inconsistent in a large proportion of questions; the tool gave a different answer in four questions among the different sessions.
• The average number of total correct answers of ChatGPT was significantly lower (P < .001) than that of physicians with and without the mobile app (7.71 and 5.62 correct answers, respectively).

IN PRACTICE:

“The use of validated mobile apps with decision-making algorithms could serve as more reliable assistants until large language models developed with AI are further refined,” the authors concluded.

SOURCE:

The study, with first author Lisandro Pereyra, MD, Department of Gastroenterology, Hospital Alemán of Buenos Aires, Argentina, was published online on February 7, 2024, in the Journal of Clinical Gastroenterology.

LIMITATIONS:

The 10 clinical vignettes represented a relatively small sample size to assess accuracy. The study did not use the latest version of ChatGPT. No “fine-tuning” attempts with inputs of diverse prompts, instructions, or relevant data were performed, which could potentially improve the performance of the chatbot.

DISCLOSURES:

The study had no specific funding. The authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

ChatGPT (version 3.5) provides relatively poor and inconsistent responses when asked about appropriate colorectal cancer (CRC) screening and surveillance, a new study showed.

METHODOLOGY:

• Three board-certified gastroenterologists with 10+ years of clinical experience developed five CRC screening and five CRC surveillance clinical vignettes (with multiple choice answers), which were fed to ChatGPT version 3.5.
• ChatGPT’s responses were recorded over four separate sessions and screened for accuracy to determine reliability of the tool.
• The average number of correct answers was compared to that of 238 gastroenterologists and colorectal surgeons answering the same questions with and without the help of a previously validated CRC screening mobile app.

TAKEAWAY:

• ChatGPT’s average overall performance was 45%; the average number of correct answers was 2.75 for screening and 1.75 for surveillance.
• ChatGPT’s responses were inconsistent in a large proportion of questions; the tool gave a different answer in four questions among the different sessions.
• The average number of total correct answers of ChatGPT was significantly lower (P < .001) than that of physicians with and without the mobile app (7.71 and 5.62 correct answers, respectively).

IN PRACTICE:

“The use of validated mobile apps with decision-making algorithms could serve as more reliable assistants until large language models developed with AI are further refined,” the authors concluded.

SOURCE:

The study, with first author Lisandro Pereyra, MD, Department of Gastroenterology, Hospital Alemán of Buenos Aires, Argentina, was published online on February 7, 2024, in the Journal of Clinical Gastroenterology.

LIMITATIONS:

The 10 clinical vignettes represented a relatively small sample size to assess accuracy. The study did not use the latest version of ChatGPT. No “fine-tuning” attempts with inputs of diverse prompts, instructions, or relevant data were performed, which could potentially improve the performance of the chatbot.

DISCLOSURES:

The study had no specific funding. The authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

TOPLINE:

ChatGPT (version 3.5) provides relatively poor and inconsistent responses when asked about appropriate colorectal cancer (CRC) screening and surveillance, a new study showed.

METHODOLOGY:

• Three board-certified gastroenterologists with 10+ years of clinical experience developed five CRC screening and five CRC surveillance clinical vignettes (with multiple choice answers), which were fed to ChatGPT version 3.5.
• ChatGPT’s responses were recorded over four separate sessions and screened for accuracy to determine reliability of the tool.
• The average number of correct answers was compared to that of 238 gastroenterologists and colorectal surgeons answering the same questions with and without the help of a previously validated CRC screening mobile app.

TAKEAWAY:

• ChatGPT’s average overall performance was 45%; the average number of correct answers was 2.75 for screening and 1.75 for surveillance.
• ChatGPT’s responses were inconsistent in a large proportion of questions; the tool gave a different answer in four questions among the different sessions.
• The average number of total correct answers of ChatGPT was significantly lower (P < .001) than that of physicians with and without the mobile app (7.71 and 5.62 correct answers, respectively).

IN PRACTICE:

“The use of validated mobile apps with decision-making algorithms could serve as more reliable assistants until large language models developed with AI are further refined,” the authors concluded.

SOURCE:

The study, with first author Lisandro Pereyra, MD, Department of Gastroenterology, Hospital Alemán of Buenos Aires, Argentina, was published online on February 7, 2024, in the Journal of Clinical Gastroenterology.

LIMITATIONS:

The 10 clinical vignettes represented a relatively small sample size to assess accuracy. The study did not use the latest version of ChatGPT. No “fine-tuning” attempts with inputs of diverse prompts, instructions, or relevant data were performed, which could potentially improve the performance of the chatbot.

DISCLOSURES:

The study had no specific funding. The authors declared no conflicts of interest.
 

A version of this article appeared on Medscape.com.

A significant quantity of dysfunctional monocytes appears to indicate poor cardiovascular prognosis in patients with type 2 diabetes, according to a new publication. Nicolas Venteclef, PhD, director of an Inserm institute for diabetes research at Necker Enfants Malades Hospital in Paris, France, led the research.

Quantifying Inflammation

Patients with type 2 diabetes have about twice the risk for a cardiovascular event associated with atherosclerosis, such as a heart attack or stroke, during their lifetimes. “Predicting these complications in diabetic patients is usually very difficult,” Dr. Venteclef told this news organization.

“They are strongly associated with inflammation in these patients. Therefore, we sought to quantify this inflammation in the blood.” To do this, his team focused on monocytes, a category of white blood cells circulating in the blood. They measured the blood concentration of monocytes and the subtypes present in patients with type 2 diabetes.

The results were published in Circulation Research.
 

Dysfunctional Monocytes

The team worked with three cohorts of patients. The first, named AngioSafe-2, consisting of 672 patients with type 2 diabetes, was recruited from the diabetology departments of Lariboisière and Bichat Claude Bernard hospitals in France. This cohort allowed researchers to demonstrate that the higher the number of circulating monocytes, the greater the risk for cardiovascular events, independent of age and duration of diabetes. This observation was confirmed through a second cohort, GLUTADIAB, that comprised 279 patients with type 2 diabetes. Scientists complemented their work with molecular analysis of circulating monocytes in these two cohorts, which revealed certain predominant monocyte subtypes in patients with type 2 diabetes at high cardiovascular risk. “These monocytes are dysfunctional because they have a mitochondrial problem,” Dr. Venteclef explained.

To better understand how these results could be used to predict cardiovascular risk, the team collaborated with colleagues from the University Hospital of Nantes on a cohort called SURDIAGENE, which included 757 patients with type 2 diabetes. “We conducted a longitudinal study by following these patients for 10 years and quantifying cardiovascular events and deaths,” said Dr. Venteclef. Circulating monocyte levels were correlated with the occurrence of heart attacks or strokes. The researchers observed that patients with type 2 diabetes with a monocyte count above a certain threshold (0.5 × 109/L) had a five- to seven-times higher risk for cardiovascular events over 10 years than those with a monocyte count below this threshold.

A patent was filed at the end of 2023 to protect this discovery. “Our next step is to develop a sensor to quantify monocytes more easily and avoid blood draws,” said Dr. Venteclef. “As part of a European project, we will also launch a trial with an anti-inflammatory drug in diabetics, with the hope of interrupting the inflammatory trajectory and preventing complications.”
 

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

A significant quantity of dysfunctional monocytes appears to indicate poor cardiovascular prognosis in patients with type 2 diabetes, according to a new publication. Nicolas Venteclef, PhD, director of an Inserm institute for diabetes research at Necker Enfants Malades Hospital in Paris, France, led the research.

Quantifying Inflammation

Patients with type 2 diabetes have about twice the risk for a cardiovascular event associated with atherosclerosis, such as a heart attack or stroke, during their lifetimes. “Predicting these complications in diabetic patients is usually very difficult,” Dr. Venteclef told this news organization.

“They are strongly associated with inflammation in these patients. Therefore, we sought to quantify this inflammation in the blood.” To do this, his team focused on monocytes, a category of white blood cells circulating in the blood. They measured the blood concentration of monocytes and the subtypes present in patients with type 2 diabetes.

The results were published in Circulation Research.
 

Dysfunctional Monocytes

The team worked with three cohorts of patients. The first, named AngioSafe-2, consisting of 672 patients with type 2 diabetes, was recruited from the diabetology departments of Lariboisière and Bichat Claude Bernard hospitals in France. This cohort allowed researchers to demonstrate that the higher the number of circulating monocytes, the greater the risk for cardiovascular events, independent of age and duration of diabetes. This observation was confirmed through a second cohort, GLUTADIAB, that comprised 279 patients with type 2 diabetes. Scientists complemented their work with molecular analysis of circulating monocytes in these two cohorts, which revealed certain predominant monocyte subtypes in patients with type 2 diabetes at high cardiovascular risk. “These monocytes are dysfunctional because they have a mitochondrial problem,” Dr. Venteclef explained.

To better understand how these results could be used to predict cardiovascular risk, the team collaborated with colleagues from the University Hospital of Nantes on a cohort called SURDIAGENE, which included 757 patients with type 2 diabetes. “We conducted a longitudinal study by following these patients for 10 years and quantifying cardiovascular events and deaths,” said Dr. Venteclef. Circulating monocyte levels were correlated with the occurrence of heart attacks or strokes. The researchers observed that patients with type 2 diabetes with a monocyte count above a certain threshold (0.5 × 109/L) had a five- to seven-times higher risk for cardiovascular events over 10 years than those with a monocyte count below this threshold.

A patent was filed at the end of 2023 to protect this discovery. “Our next step is to develop a sensor to quantify monocytes more easily and avoid blood draws,” said Dr. Venteclef. “As part of a European project, we will also launch a trial with an anti-inflammatory drug in diabetics, with the hope of interrupting the inflammatory trajectory and preventing complications.”
 

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

A significant quantity of dysfunctional monocytes appears to indicate poor cardiovascular prognosis in patients with type 2 diabetes, according to a new publication. Nicolas Venteclef, PhD, director of an Inserm institute for diabetes research at Necker Enfants Malades Hospital in Paris, France, led the research.

Quantifying Inflammation

Patients with type 2 diabetes have about twice the risk for a cardiovascular event associated with atherosclerosis, such as a heart attack or stroke, during their lifetimes. “Predicting these complications in diabetic patients is usually very difficult,” Dr. Venteclef told this news organization.

“They are strongly associated with inflammation in these patients. Therefore, we sought to quantify this inflammation in the blood.” To do this, his team focused on monocytes, a category of white blood cells circulating in the blood. They measured the blood concentration of monocytes and the subtypes present in patients with type 2 diabetes.

The results were published in Circulation Research.
 

Dysfunctional Monocytes

The team worked with three cohorts of patients. The first, named AngioSafe-2, consisting of 672 patients with type 2 diabetes, was recruited from the diabetology departments of Lariboisière and Bichat Claude Bernard hospitals in France. This cohort allowed researchers to demonstrate that the higher the number of circulating monocytes, the greater the risk for cardiovascular events, independent of age and duration of diabetes. This observation was confirmed through a second cohort, GLUTADIAB, that comprised 279 patients with type 2 diabetes. Scientists complemented their work with molecular analysis of circulating monocytes in these two cohorts, which revealed certain predominant monocyte subtypes in patients with type 2 diabetes at high cardiovascular risk. “These monocytes are dysfunctional because they have a mitochondrial problem,” Dr. Venteclef explained.

To better understand how these results could be used to predict cardiovascular risk, the team collaborated with colleagues from the University Hospital of Nantes on a cohort called SURDIAGENE, which included 757 patients with type 2 diabetes. “We conducted a longitudinal study by following these patients for 10 years and quantifying cardiovascular events and deaths,” said Dr. Venteclef. Circulating monocyte levels were correlated with the occurrence of heart attacks or strokes. The researchers observed that patients with type 2 diabetes with a monocyte count above a certain threshold (0.5 × 109/L) had a five- to seven-times higher risk for cardiovascular events over 10 years than those with a monocyte count below this threshold.

A patent was filed at the end of 2023 to protect this discovery. “Our next step is to develop a sensor to quantify monocytes more easily and avoid blood draws,” said Dr. Venteclef. “As part of a European project, we will also launch a trial with an anti-inflammatory drug in diabetics, with the hope of interrupting the inflammatory trajectory and preventing complications.”
 

This story was translated from the Medscape French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

TOPLINE:

Habitual consumption of caffeine is not associated with frequency, duration, or intensity of episodic migraines, a new study showed. Investigators said the findings suggest caffeine restrictions in migraineurs may not be necessary.

METHODOLOGY:

• The secondary analysis of a prospective cohort study on sleep in adults with episodic migraine (with or without aura) included a group of 97 people (median age, 31 years; 82% White) with an average of 5.0 ± 3.6  days per month at baseline.
• Participants provided sociodemographic information, medical history, habitual caffeinated beverage consumption, alcohol intake, and lifestyle and psychosocial factors and completed the Center for Epidemiologic Studies- scale, the Perceived Stress Scale, and the Pittsburgh Sleep Quality Index.
• Additionally, they completed twice-daily electronic diaries for the subsequent 6 weeks, reporting headache activity and the use of medications to treat the headache.

TAKEAWAY:

• A total of 67% of participants reported one to two servings of caffeinated beverages per day, 12% reported three to four servings per day, and 21% reported no habitual caffeine consumption.
• After adjusting for age, sex, oral  use, and other confounders, mean headache frequency was similar among groups (7.1 days for no caffeine, 7.4 days for one to two servings, and 5.9 days for three to four servings).
• Similarly, adjusted mean headache duration did not differ across levels of caffeinated beverage intake (8.6 hours for no caffeine, 8.5 hours for one to two servings, and 8.8 hours for three to four servings).
• Adjusted mean headache intensity also did not differ among groups.

IN PRACTICE:

“Our findings do not support a recommendation for people with episodic migraine to avoid habitual caffeinated beverage intake,” the authors wrote. However, they noted that habitual caffeine intake may affect systems involved in pain modulation via adenosine signaling. «Therefore, it is possible that habitual caffeine use in those with migraine does not significantly alter adenosine signaling, but significant changes above or below usual consumption may serve as a trigger or contribute to lowering the threshold for an attack to occur along with other triggers,» they added.

SOURCE:

Suzanne M. Bertisch, MD, MPH, assistant professor of medicine, Division of Sleep Medicine, Harvard Medical School, Boston, Massachusetts, was the senior and corresponding author of the study. It was published online in Headache.

LIMITATIONS:

Serving size was not standardized, and there was no information on the type of caffeinated beverage consumed or about other sources of caffeine. Moreover, the population consisted of relatively healthy participants with episodic migraine and generally low levels of habitual caffeinated beverage intake, which limited the statistical power to detect an association between migraine frequency, duration, and intensity with higher levels of caffeine intake.

DISCLOSURES:

The study was funded by the National Institute of Neurological Disorders and Stroke, the American Sleep Medicine Foundation, and the Harvard Catalyst/Harvard Clinical and Translational Science Center. Dr. Bertisch has done consulting work with Idorsia and ResMed. The other authors’ disclosures are listed in the original paper.

A version of this article appeared on Medscape.com.

TOPLINE:

Habitual consumption of caffeine is not associated with frequency, duration, or intensity of episodic migraines, a new study showed. Investigators said the findings suggest caffeine restrictions in migraineurs may not be necessary.

METHODOLOGY:

• The secondary analysis of a prospective cohort study on sleep in adults with episodic migraine (with or without aura) included a group of 97 people (median age, 31 years; 82% White) with an average of 5.0 ± 3.6  days per month at baseline.
• Participants provided sociodemographic information, medical history, habitual caffeinated beverage consumption, alcohol intake, and lifestyle and psychosocial factors and completed the Center for Epidemiologic Studies- scale, the Perceived Stress Scale, and the Pittsburgh Sleep Quality Index.
• Additionally, they completed twice-daily electronic diaries for the subsequent 6 weeks, reporting headache activity and the use of medications to treat the headache.

TAKEAWAY:

• A total of 67% of participants reported one to two servings of caffeinated beverages per day, 12% reported three to four servings per day, and 21% reported no habitual caffeine consumption.
• After adjusting for age, sex, oral  use, and other confounders, mean headache frequency was similar among groups (7.1 days for no caffeine, 7.4 days for one to two servings, and 5.9 days for three to four servings).
• Similarly, adjusted mean headache duration did not differ across levels of caffeinated beverage intake (8.6 hours for no caffeine, 8.5 hours for one to two servings, and 8.8 hours for three to four servings).
• Adjusted mean headache intensity also did not differ among groups.

IN PRACTICE:

“Our findings do not support a recommendation for people with episodic migraine to avoid habitual caffeinated beverage intake,” the authors wrote. However, they noted that habitual caffeine intake may affect systems involved in pain modulation via adenosine signaling. «Therefore, it is possible that habitual caffeine use in those with migraine does not significantly alter adenosine signaling, but significant changes above or below usual consumption may serve as a trigger or contribute to lowering the threshold for an attack to occur along with other triggers,» they added.

SOURCE:

Suzanne M. Bertisch, MD, MPH, assistant professor of medicine, Division of Sleep Medicine, Harvard Medical School, Boston, Massachusetts, was the senior and corresponding author of the study. It was published online in Headache.

LIMITATIONS:

Serving size was not standardized, and there was no information on the type of caffeinated beverage consumed or about other sources of caffeine. Moreover, the population consisted of relatively healthy participants with episodic migraine and generally low levels of habitual caffeinated beverage intake, which limited the statistical power to detect an association between migraine frequency, duration, and intensity with higher levels of caffeine intake.

DISCLOSURES:

The study was funded by the National Institute of Neurological Disorders and Stroke, the American Sleep Medicine Foundation, and the Harvard Catalyst/Harvard Clinical and Translational Science Center. Dr. Bertisch has done consulting work with Idorsia and ResMed. The other authors’ disclosures are listed in the original paper.

A version of this article appeared on Medscape.com.

TOPLINE:

Habitual consumption of caffeine is not associated with frequency, duration, or intensity of episodic migraines, a new study showed. Investigators said the findings suggest caffeine restrictions in migraineurs may not be necessary.

METHODOLOGY:

• The secondary analysis of a prospective cohort study on sleep in adults with episodic migraine (with or without aura) included a group of 97 people (median age, 31 years; 82% White) with an average of 5.0 ± 3.6  days per month at baseline.
• Participants provided sociodemographic information, medical history, habitual caffeinated beverage consumption, alcohol intake, and lifestyle and psychosocial factors and completed the Center for Epidemiologic Studies- scale, the Perceived Stress Scale, and the Pittsburgh Sleep Quality Index.
• Additionally, they completed twice-daily electronic diaries for the subsequent 6 weeks, reporting headache activity and the use of medications to treat the headache.

TAKEAWAY:

• A total of 67% of participants reported one to two servings of caffeinated beverages per day, 12% reported three to four servings per day, and 21% reported no habitual caffeine consumption.
• After adjusting for age, sex, oral  use, and other confounders, mean headache frequency was similar among groups (7.1 days for no caffeine, 7.4 days for one to two servings, and 5.9 days for three to four servings).
• Similarly, adjusted mean headache duration did not differ across levels of caffeinated beverage intake (8.6 hours for no caffeine, 8.5 hours for one to two servings, and 8.8 hours for three to four servings).
• Adjusted mean headache intensity also did not differ among groups.

IN PRACTICE:

“Our findings do not support a recommendation for people with episodic migraine to avoid habitual caffeinated beverage intake,” the authors wrote. However, they noted that habitual caffeine intake may affect systems involved in pain modulation via adenosine signaling. «Therefore, it is possible that habitual caffeine use in those with migraine does not significantly alter adenosine signaling, but significant changes above or below usual consumption may serve as a trigger or contribute to lowering the threshold for an attack to occur along with other triggers,» they added.

SOURCE:

Suzanne M. Bertisch, MD, MPH, assistant professor of medicine, Division of Sleep Medicine, Harvard Medical School, Boston, Massachusetts, was the senior and corresponding author of the study. It was published online in Headache.

LIMITATIONS:

Serving size was not standardized, and there was no information on the type of caffeinated beverage consumed or about other sources of caffeine. Moreover, the population consisted of relatively healthy participants with episodic migraine and generally low levels of habitual caffeinated beverage intake, which limited the statistical power to detect an association between migraine frequency, duration, and intensity with higher levels of caffeine intake.

DISCLOSURES:

The study was funded by the National Institute of Neurological Disorders and Stroke, the American Sleep Medicine Foundation, and the Harvard Catalyst/Harvard Clinical and Translational Science Center. Dr. Bertisch has done consulting work with Idorsia and ResMed. The other authors’ disclosures are listed in the original paper.

A version of this article appeared on Medscape.com.

TOPLINE:

Selenium supplementation is associated with improvements in key thyroid measures in patients with Hashimoto thyroiditis who are not treated with thyroid hormone replacement therapy, research from a new meta-analysis showed.

METHODOLOGY:

• For the systematic review and meta-analysis, 35 randomized controlled trials were identified that included evaluation of selenium supplementation’s effects on thyroid function.
• The studies focused on a variety of key thyroid function measures, including thyroid-stimulating hormone (TSH), free and total thyroxine (fT4, T4), free and total triiodothyronine (fT3, T3), thyroid antibodies, safety, and other factors.
• Stratified analyses were conducted to evaluate key factors including the dose and duration of selenium supplementation; patients’ thyroid status, age, gender, treatment with hormone replacement, and selenium status, such as deficiency or sufficiency; and other factors.
• While patients’ selenium levels at baseline were reported in only about half of the studies, among those that did have the data, the vast majority — 89% of cohorts — were selenium deficient.
• The study populations ranged from 31 to 364 and included children, adolescents, and adults.

TAKEAWAY:

• The analysis showed selenium supplementation to be significantly associated with decreased TSH in patients who were not treated with thyroid hormone replacement therapy (standardized mean difference [SMD], −0.21 in seven cohorts, involving 869 participants).
• Improvements associated with selenium replacement were also observed regardless of whether patients were on thyroid hormone replacement therapy in terms of decreases in thyroid peroxidase antibodies (TPOAb) (SMD, −0.96 in 29 cohorts, involving 2358 participants) and malondialdehyde (SMD, −1.16 in three cohorts, involving 248 participants).
• Overall, selenium supplementation had no significant effects on other notable thyroid measures, including fT4, T4, fT3, T3, thyroglobulin antibody (TGAb), thyroid volume, interleukin 2, or interleukin 10. However, when the analysis only included adults aged 18 and older, the selenium supplementation was linked to reductions in TSH and TPOAb, as well as increases in fT4 levels.
• Importantly, no significant differences were observed in terms of adverse effects between the studies’ intervention and control groups at selenium supplementation doses ranging from 80 to 400 μg/d for up to 12 months (odds ratio, 0.89 in 16 cohorts, involving 1339 participants).
• The authors determined that the certainty of evidence, overall, was moderate.

IN PRACTICE:

The results regarding effects of selenium on TSH “add to the existing knowledge in this field by demonstrating an effect of selenium supplementation on lowering TSH levels exclusively in Hashimoto thyroiditis patients without thyroid hormone replacement therapy,” the authors wrote. Furthermore, “our study reaffirmed the results of six prior meta-analyses reporting an effect of selenium in reducing TPOAb levels,” they added. “The inclusion of 31 cohorts enhanced statistical power compared to the previous meta-analyses, which included a maximum of nine cohorts.” “Our study suggests that selenium supplementation is safe and holds potential as a disease-modifying factor for Hashimoto thyroiditis–associated hypothyroidism,” the authors reported. “Further research is needed to confirm its efficacy, fully understand its mechanism of action, and elucidate its cost-effectiveness.”

SOURCE:

The study’s first author was Valentina V. Huwiler, MSc, of the Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland. The study was published in Thyroid.

LIMITATIONS:

Due to variations in assays used in the different studies for measures including TPOAb and TGAb, the authors used SMD instead of the mean difference typically recommended when varying assays are used; however, only the effect size can be interpreted and not the clinical significance, the authors noted. Serum selenium concentrations may vary based on the analytical technique. Data on participants’ dietary habits and compliance with study regimens were not available.

DISCLOSURES:

The authors had no disclosures to report.

A version of this article appeared on Medscape.com.

TOPLINE:

Selenium supplementation is associated with improvements in key thyroid measures in patients with Hashimoto thyroiditis who are not treated with thyroid hormone replacement therapy, research from a new meta-analysis showed.

METHODOLOGY:

• For the systematic review and meta-analysis, 35 randomized controlled trials were identified that included evaluation of selenium supplementation’s effects on thyroid function.
• The studies focused on a variety of key thyroid function measures, including thyroid-stimulating hormone (TSH), free and total thyroxine (fT4, T4), free and total triiodothyronine (fT3, T3), thyroid antibodies, safety, and other factors.
• Stratified analyses were conducted to evaluate key factors including the dose and duration of selenium supplementation; patients’ thyroid status, age, gender, treatment with hormone replacement, and selenium status, such as deficiency or sufficiency; and other factors.
• While patients’ selenium levels at baseline were reported in only about half of the studies, among those that did have the data, the vast majority — 89% of cohorts — were selenium deficient.
• The study populations ranged from 31 to 364 and included children, adolescents, and adults.

TAKEAWAY:

• The analysis showed selenium supplementation to be significantly associated with decreased TSH in patients who were not treated with thyroid hormone replacement therapy (standardized mean difference [SMD], −0.21 in seven cohorts, involving 869 participants).
• Improvements associated with selenium replacement were also observed regardless of whether patients were on thyroid hormone replacement therapy in terms of decreases in thyroid peroxidase antibodies (TPOAb) (SMD, −0.96 in 29 cohorts, involving 2358 participants) and malondialdehyde (SMD, −1.16 in three cohorts, involving 248 participants).
• Overall, selenium supplementation had no significant effects on other notable thyroid measures, including fT4, T4, fT3, T3, thyroglobulin antibody (TGAb), thyroid volume, interleukin 2, or interleukin 10. However, when the analysis only included adults aged 18 and older, the selenium supplementation was linked to reductions in TSH and TPOAb, as well as increases in fT4 levels.
• Importantly, no significant differences were observed in terms of adverse effects between the studies’ intervention and control groups at selenium supplementation doses ranging from 80 to 400 μg/d for up to 12 months (odds ratio, 0.89 in 16 cohorts, involving 1339 participants).
• The authors determined that the certainty of evidence, overall, was moderate.

IN PRACTICE:

The results regarding effects of selenium on TSH “add to the existing knowledge in this field by demonstrating an effect of selenium supplementation on lowering TSH levels exclusively in Hashimoto thyroiditis patients without thyroid hormone replacement therapy,” the authors wrote. Furthermore, “our study reaffirmed the results of six prior meta-analyses reporting an effect of selenium in reducing TPOAb levels,” they added. “The inclusion of 31 cohorts enhanced statistical power compared to the previous meta-analyses, which included a maximum of nine cohorts.” “Our study suggests that selenium supplementation is safe and holds potential as a disease-modifying factor for Hashimoto thyroiditis–associated hypothyroidism,” the authors reported. “Further research is needed to confirm its efficacy, fully understand its mechanism of action, and elucidate its cost-effectiveness.”

SOURCE:

The study’s first author was Valentina V. Huwiler, MSc, of the Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland. The study was published in Thyroid.

LIMITATIONS:

Due to variations in assays used in the different studies for measures including TPOAb and TGAb, the authors used SMD instead of the mean difference typically recommended when varying assays are used; however, only the effect size can be interpreted and not the clinical significance, the authors noted. Serum selenium concentrations may vary based on the analytical technique. Data on participants’ dietary habits and compliance with study regimens were not available.

DISCLOSURES:

The authors had no disclosures to report.

A version of this article appeared on Medscape.com.

TOPLINE:

Selenium supplementation is associated with improvements in key thyroid measures in patients with Hashimoto thyroiditis who are not treated with thyroid hormone replacement therapy, research from a new meta-analysis showed.

METHODOLOGY:

• For the systematic review and meta-analysis, 35 randomized controlled trials were identified that included evaluation of selenium supplementation’s effects on thyroid function.
• The studies focused on a variety of key thyroid function measures, including thyroid-stimulating hormone (TSH), free and total thyroxine (fT4, T4), free and total triiodothyronine (fT3, T3), thyroid antibodies, safety, and other factors.
• Stratified analyses were conducted to evaluate key factors including the dose and duration of selenium supplementation; patients’ thyroid status, age, gender, treatment with hormone replacement, and selenium status, such as deficiency or sufficiency; and other factors.
• While patients’ selenium levels at baseline were reported in only about half of the studies, among those that did have the data, the vast majority — 89% of cohorts — were selenium deficient.
• The study populations ranged from 31 to 364 and included children, adolescents, and adults.

TAKEAWAY:

• The analysis showed selenium supplementation to be significantly associated with decreased TSH in patients who were not treated with thyroid hormone replacement therapy (standardized mean difference [SMD], −0.21 in seven cohorts, involving 869 participants).
• Improvements associated with selenium replacement were also observed regardless of whether patients were on thyroid hormone replacement therapy in terms of decreases in thyroid peroxidase antibodies (TPOAb) (SMD, −0.96 in 29 cohorts, involving 2358 participants) and malondialdehyde (SMD, −1.16 in three cohorts, involving 248 participants).
• Overall, selenium supplementation had no significant effects on other notable thyroid measures, including fT4, T4, fT3, T3, thyroglobulin antibody (TGAb), thyroid volume, interleukin 2, or interleukin 10. However, when the analysis only included adults aged 18 and older, the selenium supplementation was linked to reductions in TSH and TPOAb, as well as increases in fT4 levels.
• Importantly, no significant differences were observed in terms of adverse effects between the studies’ intervention and control groups at selenium supplementation doses ranging from 80 to 400 μg/d for up to 12 months (odds ratio, 0.89 in 16 cohorts, involving 1339 participants).
• The authors determined that the certainty of evidence, overall, was moderate.

IN PRACTICE:

The results regarding effects of selenium on TSH “add to the existing knowledge in this field by demonstrating an effect of selenium supplementation on lowering TSH levels exclusively in Hashimoto thyroiditis patients without thyroid hormone replacement therapy,” the authors wrote. Furthermore, “our study reaffirmed the results of six prior meta-analyses reporting an effect of selenium in reducing TPOAb levels,” they added. “The inclusion of 31 cohorts enhanced statistical power compared to the previous meta-analyses, which included a maximum of nine cohorts.” “Our study suggests that selenium supplementation is safe and holds potential as a disease-modifying factor for Hashimoto thyroiditis–associated hypothyroidism,” the authors reported. “Further research is needed to confirm its efficacy, fully understand its mechanism of action, and elucidate its cost-effectiveness.”

SOURCE:

The study’s first author was Valentina V. Huwiler, MSc, of the Department of Diabetes, Endocrinology, Nutritional Medicine and Metabolism, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland. The study was published in Thyroid.

LIMITATIONS:

Due to variations in assays used in the different studies for measures including TPOAb and TGAb, the authors used SMD instead of the mean difference typically recommended when varying assays are used; however, only the effect size can be interpreted and not the clinical significance, the authors noted. Serum selenium concentrations may vary based on the analytical technique. Data on participants’ dietary habits and compliance with study regimens were not available.

DISCLOSURES:

The authors had no disclosures to report.

A version of this article appeared on Medscape.com.

TOPLINE:

Greater adherence to a plant-based dietary pattern was associated with a lower risk of developing type 2 diabetes (T2D) among middle-aged US adults. Greater intake of healthful plant foods, rather than lower intake of non-red meat animal foods, was the main factor underlying the inverse associations.

METHODOLOGY:

• The study population was 11,965 adults aged 45-64 years from the Atherosclerosis Risk in Communities (ARIC) study who didn›t have diabetes at baseline and who completed food-frequency questionnaires.
• Plant-based diet adherence was classified overall with the plant-based diet index (PDI) and also with higher healthful PDI (hPDI) and higher unhealthful PDI (uPDI) indexes.

TAKEAWAY:

• Mean daily total plant and animal food intakes for the highest quintile (5) were 15.1 and 3.4 servings per day, respectively, whereas average consumption for the lowest quintile (1) was 9.9 and 5.8 servings per day, respectively.
• During a median 22 years’ follow-up, 35% (n = 4208) of the participants developed T2D.
• After controlling for age, sex, race center, energy intake, education, income, smoking, alcohol intake, physical activity, and margarine intake, those in PDI quintile 5 had a significantly lower risk of developing T2D than in quintile 1 (hazard ratio, 0.89; P = .01).
• As a continuous score, each 10-point higher PDI score was associated with a significant 6% lower risk for T2D (P = .01).
• Higher hPDI scores were also inversely associated with T2D risk (hazard ratio, 0.85 for quintiles 5 vs 1; P < .001), and (0.90 per each 10 units higher; P < .001).
• Higher uPDI scores were not significantly associated with diabetes risk, regardless of adjustments (P > .05).
• Associations between plant-based diet scores and diabetes did not differ by sex, age, race, or body mass index (BMI) after accounting for multiple comparisons (all P interaction > .05).
• Further adjustment for BMI attenuated the associations between overall and healthy plant-based diets and diabetes risk, suggesting that lower adiposity may partly explain the favorable association.

IN PRACTICE:

“Emphasizing plant foods may be an effective dietary strategy to delay or prevent the onset of diabetes.”

SOURCE:

The study conducted by Valerie K. Sullivan, PhD, RD, of the Welch Center for Prevention, Epidemiology, and Clinical Research, Johns Hopkins University, Baltimore, Maryland, and colleagues was published online in Diabetes Care.

LIMITATIONS:

The limitations were self-reported dietary intake, diets assessed decades ago, possible food misclassification, possible selection bias, and residual confounding.

DISCLOSURES:

The ARIC study was funded by the US National Institutes of Health. The authors had no further disclosures.

A version of this article appeared on Medscape.com.

TOPLINE:

Greater adherence to a plant-based dietary pattern was associated with a lower risk of developing type 2 diabetes (T2D) among middle-aged US adults. Greater intake of healthful plant foods, rather than lower intake of non-red meat animal foods, was the main factor underlying the inverse associations.

METHODOLOGY:

• The study population was 11,965 adults aged 45-64 years from the Atherosclerosis Risk in Communities (ARIC) study who didn›t have diabetes at baseline and who completed food-frequency questionnaires.
• Plant-based diet adherence was classified overall with the plant-based diet index (PDI) and also with higher healthful PDI (hPDI) and higher unhealthful PDI (uPDI) indexes.

TAKEAWAY:

• Mean daily total plant and animal food intakes for the highest quintile (5) were 15.1 and 3.4 servings per day, respectively, whereas average consumption for the lowest quintile (1) was 9.9 and 5.8 servings per day, respectively.
• During a median 22 years’ follow-up, 35% (n = 4208) of the participants developed T2D.
• After controlling for age, sex, race center, energy intake, education, income, smoking, alcohol intake, physical activity, and margarine intake, those in PDI quintile 5 had a significantly lower risk of developing T2D than in quintile 1 (hazard ratio, 0.89; P = .01).
• As a continuous score, each 10-point higher PDI score was associated with a significant 6% lower risk for T2D (P = .01).
• Higher hPDI scores were also inversely associated with T2D risk (hazard ratio, 0.85 for quintiles 5 vs 1; P < .001), and (0.90 per each 10 units higher; P < .001).
• Higher uPDI scores were not significantly associated with diabetes risk, regardless of adjustments (P > .05).
• Associations between plant-based diet scores and diabetes did not differ by sex, age, race, or body mass index (BMI) after accounting for multiple comparisons (all P interaction > .05).
• Further adjustment for BMI attenuated the associations between overall and healthy plant-based diets and diabetes risk, suggesting that lower adiposity may partly explain the favorable association.

IN PRACTICE:

“Emphasizing plant foods may be an effective dietary strategy to delay or prevent the onset of diabetes.”

SOURCE:

The study conducted by Valerie K. Sullivan, PhD, RD, of the Welch Center for Prevention, Epidemiology, and Clinical Research, Johns Hopkins University, Baltimore, Maryland, and colleagues was published online in Diabetes Care.

LIMITATIONS:

The limitations were self-reported dietary intake, diets assessed decades ago, possible food misclassification, possible selection bias, and residual confounding.

DISCLOSURES:

The ARIC study was funded by the US National Institutes of Health. The authors had no further disclosures.

A version of this article appeared on Medscape.com.

TOPLINE:

Greater adherence to a plant-based dietary pattern was associated with a lower risk of developing type 2 diabetes (T2D) among middle-aged US adults. Greater intake of healthful plant foods, rather than lower intake of non-red meat animal foods, was the main factor underlying the inverse associations.

METHODOLOGY:

• The study population was 11,965 adults aged 45-64 years from the Atherosclerosis Risk in Communities (ARIC) study who didn›t have diabetes at baseline and who completed food-frequency questionnaires.
• Plant-based diet adherence was classified overall with the plant-based diet index (PDI) and also with higher healthful PDI (hPDI) and higher unhealthful PDI (uPDI) indexes.

TAKEAWAY:

• Mean daily total plant and animal food intakes for the highest quintile (5) were 15.1 and 3.4 servings per day, respectively, whereas average consumption for the lowest quintile (1) was 9.9 and 5.8 servings per day, respectively.
• During a median 22 years’ follow-up, 35% (n = 4208) of the participants developed T2D.
• After controlling for age, sex, race center, energy intake, education, income, smoking, alcohol intake, physical activity, and margarine intake, those in PDI quintile 5 had a significantly lower risk of developing T2D than in quintile 1 (hazard ratio, 0.89; P = .01).
• As a continuous score, each 10-point higher PDI score was associated with a significant 6% lower risk for T2D (P = .01).
• Higher hPDI scores were also inversely associated with T2D risk (hazard ratio, 0.85 for quintiles 5 vs 1; P < .001), and (0.90 per each 10 units higher; P < .001).
• Higher uPDI scores were not significantly associated with diabetes risk, regardless of adjustments (P > .05).
• Associations between plant-based diet scores and diabetes did not differ by sex, age, race, or body mass index (BMI) after accounting for multiple comparisons (all P interaction > .05).
• Further adjustment for BMI attenuated the associations between overall and healthy plant-based diets and diabetes risk, suggesting that lower adiposity may partly explain the favorable association.

IN PRACTICE:

“Emphasizing plant foods may be an effective dietary strategy to delay or prevent the onset of diabetes.”

SOURCE:

The study conducted by Valerie K. Sullivan, PhD, RD, of the Welch Center for Prevention, Epidemiology, and Clinical Research, Johns Hopkins University, Baltimore, Maryland, and colleagues was published online in Diabetes Care.

LIMITATIONS:

The limitations were self-reported dietary intake, diets assessed decades ago, possible food misclassification, possible selection bias, and residual confounding.

DISCLOSURES:

The ARIC study was funded by the US National Institutes of Health. The authors had no further disclosures.

A version of this article appeared on Medscape.com.

TOPLINE:

Cefepime-taniborbactam was 22% more effective than meropenem, which is a current treatment for complicated urinary tract infections (UTIs) and acute pyelonephritis, according to a study published in The New England Journal of Medicine.

METHODOLOGY:

• Cefepime-taniborbactam is an antibiotic currently being explored as a treatment for antibiotic-resistant bacteria.
• The phase 3, double-blind, randomized trial included participants from 15 countries, including a safety group of 657 patients who were studied for adverse events and 436 in the micro intention-to-treat group who were studied for drug effectiveness.
• Each drug’s efficacy was measured as a combination of reduced bacteria levels and a resolution of symptoms and signs of infection.
• Patients in the study were over age 18; had a diagnosis of either complicated UTI or acute pyelonephritis; and had pyuria, at least one systemic sign, and at least one local sign or symptom. People were excluded if they had already received antibacterial drug therapy for more than 24 hours before randomization or had an infection with a meropenem-resistant pathogen.

TAKEAWAY:

• At days 19-23, 70.6% of patients in the cefepime-taniborbactam group showed a successful reduction in bacteria and symptoms compared with 58.0% in the meropenem group.
• Cefepime-taniborbactam was more effective than meropenem during follow-up, with 89.1% efficacy less than 24 hours after the last dose, compared to meropenem’s 86%. Cefepime-taniborbactam continued to have 63.8% efficacy up to 35 days after starting treatment, while meropenem was 51.7% during that timeframe.
• In the cefepime-taniborbactam group, 35.5% of patients experienced adverse effects that were mild to moderate, including headache, diarrhea, constipation, hypertension, and nausea, compared to 29% in the meropenem group.
• Overall, 3% of participants discontinued cefepime-taniborbactam and 1.8% discontinued meropenem, but reasons were heterogeneous.

IN PRACTICE:

“Cefepime-taniborbactam was superior to meropenem for the treatment of complicated UTI that included acute pyelonephritis, with a safety profile similar to that of meropenem,” the study authors wrote.

SOURCE:

Paul McGovern, MD, infectious disease specialist and senior vice president of Venatorx Pharmaceuticals, was the corresponding author of the study.

LIMITATIONS:

The authors reported no limitations.

DISCLOSURES:

The study was funded by Venatorx Pharmaceuticals, which received funding from the US Department of Health and Human Services, the Administration for Strategic Preparedness and Response, the Biomedical Advanced Research and Development Authority, the Global Antibiotic Research and Development Partnership, and Everest Medicines.

A version of this article appeared on Medscape.com.

TOPLINE:

Cefepime-taniborbactam was 22% more effective than meropenem, which is a current treatment for complicated urinary tract infections (UTIs) and acute pyelonephritis, according to a study published in The New England Journal of Medicine.

METHODOLOGY:

• Cefepime-taniborbactam is an antibiotic currently being explored as a treatment for antibiotic-resistant bacteria.
• The phase 3, double-blind, randomized trial included participants from 15 countries, including a safety group of 657 patients who were studied for adverse events and 436 in the micro intention-to-treat group who were studied for drug effectiveness.
• Each drug’s efficacy was measured as a combination of reduced bacteria levels and a resolution of symptoms and signs of infection.
• Patients in the study were over age 18; had a diagnosis of either complicated UTI or acute pyelonephritis; and had pyuria, at least one systemic sign, and at least one local sign or symptom. People were excluded if they had already received antibacterial drug therapy for more than 24 hours before randomization or had an infection with a meropenem-resistant pathogen.

TAKEAWAY:

• At days 19-23, 70.6% of patients in the cefepime-taniborbactam group showed a successful reduction in bacteria and symptoms compared with 58.0% in the meropenem group.
• Cefepime-taniborbactam was more effective than meropenem during follow-up, with 89.1% efficacy less than 24 hours after the last dose, compared to meropenem’s 86%. Cefepime-taniborbactam continued to have 63.8% efficacy up to 35 days after starting treatment, while meropenem was 51.7% during that timeframe.
• In the cefepime-taniborbactam group, 35.5% of patients experienced adverse effects that were mild to moderate, including headache, diarrhea, constipation, hypertension, and nausea, compared to 29% in the meropenem group.
• Overall, 3% of participants discontinued cefepime-taniborbactam and 1.8% discontinued meropenem, but reasons were heterogeneous.

IN PRACTICE:

“Cefepime-taniborbactam was superior to meropenem for the treatment of complicated UTI that included acute pyelonephritis, with a safety profile similar to that of meropenem,” the study authors wrote.

SOURCE:

Paul McGovern, MD, infectious disease specialist and senior vice president of Venatorx Pharmaceuticals, was the corresponding author of the study.

LIMITATIONS:

The authors reported no limitations.

DISCLOSURES:

The study was funded by Venatorx Pharmaceuticals, which received funding from the US Department of Health and Human Services, the Administration for Strategic Preparedness and Response, the Biomedical Advanced Research and Development Authority, the Global Antibiotic Research and Development Partnership, and Everest Medicines.

A version of this article appeared on Medscape.com.

TOPLINE:

Cefepime-taniborbactam was 22% more effective than meropenem, which is a current treatment for complicated urinary tract infections (UTIs) and acute pyelonephritis, according to a study published in The New England Journal of Medicine.

METHODOLOGY:

• Cefepime-taniborbactam is an antibiotic currently being explored as a treatment for antibiotic-resistant bacteria.
• The phase 3, double-blind, randomized trial included participants from 15 countries, including a safety group of 657 patients who were studied for adverse events and 436 in the micro intention-to-treat group who were studied for drug effectiveness.
• Each drug’s efficacy was measured as a combination of reduced bacteria levels and a resolution of symptoms and signs of infection.
• Patients in the study were over age 18; had a diagnosis of either complicated UTI or acute pyelonephritis; and had pyuria, at least one systemic sign, and at least one local sign or symptom. People were excluded if they had already received antibacterial drug therapy for more than 24 hours before randomization or had an infection with a meropenem-resistant pathogen.

TAKEAWAY:

• At days 19-23, 70.6% of patients in the cefepime-taniborbactam group showed a successful reduction in bacteria and symptoms compared with 58.0% in the meropenem group.
• Cefepime-taniborbactam was more effective than meropenem during follow-up, with 89.1% efficacy less than 24 hours after the last dose, compared to meropenem’s 86%. Cefepime-taniborbactam continued to have 63.8% efficacy up to 35 days after starting treatment, while meropenem was 51.7% during that timeframe.
• In the cefepime-taniborbactam group, 35.5% of patients experienced adverse effects that were mild to moderate, including headache, diarrhea, constipation, hypertension, and nausea, compared to 29% in the meropenem group.
• Overall, 3% of participants discontinued cefepime-taniborbactam and 1.8% discontinued meropenem, but reasons were heterogeneous.

IN PRACTICE:

“Cefepime-taniborbactam was superior to meropenem for the treatment of complicated UTI that included acute pyelonephritis, with a safety profile similar to that of meropenem,” the study authors wrote.

SOURCE:

Paul McGovern, MD, infectious disease specialist and senior vice president of Venatorx Pharmaceuticals, was the corresponding author of the study.

LIMITATIONS:

The authors reported no limitations.

DISCLOSURES:

The study was funded by Venatorx Pharmaceuticals, which received funding from the US Department of Health and Human Services, the Administration for Strategic Preparedness and Response, the Biomedical Advanced Research and Development Authority, the Global Antibiotic Research and Development Partnership, and Everest Medicines.

A version of this article appeared on Medscape.com.