Federal Practitioner

Berberine, a plant-derived compound historically used in traditional Chinese medicine, is experiencing increased popularity thanks to social media, especially TikTok, where the hashtag #berberine has more than 75 million views at the time of this writing. Social media influencers are promoting the compound, calling it “nature’s Ozempic,” saying they lost weight taking the supplement.

Off-the-shelf berberine comes as a yellow-orange powder usually encased in a capsule or mixed into tablet form. It’s extracted from the roots, stems, and leaves of various plants, including goldenseal and barberry.

Its use is additionally promoted for insulin resistance, polycystic ovary syndrome, and even cancer, but medical experts are warning potential users that it lacks robust evidence to support its use.

“There’s not that much data on it,” says Reshmi Srinath, MD, director of the Mount Sinai weight and metabolism management program, New York. “It’s sort of shocking now that it’s popped up into the media, to be frank.”

In response to berberine’s online popularity, the National Center for Complementary and Integrative Health issued a warning, stating that “there isn’t enough rigorous scientific evidence to determine whether it is effective.”
 

Overstated claims, lack of scientific research?

Other endocrinologists and weight management experts agree. “The claims are pretty overstated when it comes to the impact on weight loss, based on the evidence in the literature that’s currently available,” says Jaime Almandoz, MD, medical director of the UT Southwestern Medical Center, Dallas, weight wellness program.

A review of 12 randomized controlled trials evaluating berberine’s effects on obesity concluded that the treatment moderately decreased body weight. The trials included were conducted over only a few months and had small numbers of participants, and weight loss was not the primary outcome measure.

“There are few randomized controlled trials,” says Ivania Rizo, MD, an endocrinologist at Boston University. “It appears that they all have some low quality of methods which essentially can lead to an increased risk of bias.”

Another review, of 35 studies – most of them on animals and human cells and similarly underpowered – concluded that berberine showed promise for reducing blood glucose. A separate study found that berberine treatment actually increased the body weight and appetite of rats.

How exactly berberine elicits these effects is not entirely clear. Several studies point to its activation of AMP-activated protein kinase, which improves glucose tolerance in rats, as the mechanism for weight loss. Metformin, a drug used to improve glycemic control in people with type 2 diabetes, works in a similar way. Other researchers have hypothesized a link between berberine and the gut microbiome to explain its effect on type 2 diabetes and weight loss, though the clinical data to substantiate this link are shaky.

“I caution my patients about dietary supplements for weight management because we do not have high-quality data demonstrating efficacy,” Katherine Saunders, MD, DABOM, an obesity expert and cofounder of Intellihealth, a platform for obesity management, said in an email.
 

Experimenting with berberine

Despite the lack of substantial evidence supporting berberine’s use for weight management and obesity, interest in the supplement seems to be increasing. One reason could be that lifestyle interventions aren’t sufficient for most people with obesity to lose a significant amount of weight, with many requiring medical intervention, according to Dr. Saunders.

But access to treatment providers is limited. “As a result, it is not uncommon for individuals with obesity to experiment with dietary supplements like berberine,” she observed.

Dr. Srinath, the Mount Sinai doctor, says many patients have asked for her thoughts on berberine as a weight loss supplement. “I say, you know, it’s something you’re welcome to try, but we don’t have enough data at this time to recommend it.”

The hype surrounding the supplement isn’t all that surprising. About 42% of adults in the United States have obesity, according to 2019-2023 National Health and Nutrition Examination Survey data, pointing to a serious need for accessible drugs to address the condition. Berberine is available over the counter and is far cheaper than most of the newer U.S. Food and Drug Administration–approved drugs for weight loss.

Wegovy, semaglutide approved to treat obesity, can cost as much as $1,300 per package; and Ozempic, semaglutide approved to treat type 2 diabetes, can cost more than $1,000 per month. “That’s a very steep price to pay,” says Dr. Srinath.

Many insurance companies won’t cover the drugs, curbing access to Americans who need them, says Dr. Almandoz. Federally sponsored programs such as Medicare and Medicaid also don’t cover the drugs, which are approved for obesity and weight management. “That’s been a huge hole in our health care system,” says Dr. Srinath. “That’s sort of what’s been driving interest in supplements and things like that.”

Among adults trying to lose weight, only about 3% said they took prescription medication for weight loss, according to a report from the U.S. Government Accountability Office. This report includes 2013-2016 data, predating Wegovy’s approval for chronic weight management.

“These classes are notorious for being quite pricey and not well covered by insurance,” says Dr. Almandoz. “It’s easy to see why someone would promote something that someone may have more access to.”

Comparing Ozempic or Wegovy with berberine can be misleading. Those drugs work by mimicking the effect of the hormone GLP-1 to help reduce appetite.

A clinical trial assessing the efficacy of semaglutide found that adults with obesity who took the drug for 68 weeks lost approximately 15% of their body weight in combination with lifestyle changes. The FDA approval was based on this trial and three others that showed similarly substantial reductions in weight.

The trials also document the many side effects of taking the drugs, primarily gastrointestinal in nature. The short- and long-term effects of berberine, on the other hand, are less clear. Some of the clinical trials reported diarrhea and stomach upset as the most common adverse effects.

Its perception as a naturally derived option for weight loss, though, might encourage people to overlook the potential interactions that berberine could have with other drugs, according to Dr. Almandoz.

He says clinicians considering natural products or nutraceuticals for patients should check for potential side effects and find reliable database sources to determine any potential medication interactions for patients. But the unregulated nature of berberine makes this challenging, Dr. Almandoz adds.

The dosage, formulations, and quality of berberine vary in each study and each product because supplements don’t need to pass through the checks and balances of the FDA to land on shelves.

The lack of regulation could incentivize some companies to add stimulants to enhance any weight loss effect that the supplement may have. Those additives might interact with other health conditions or cause side effects like anxiety, says Dr. Almandoz.

Berberine should also not be taken during pregnancy or while breastfeeding, and it is unsafe for young children; in newborns and children, the supplement can cause higher levels of bilirubin in the blood, worsening any jaundice at birth and posing a greater risk for kernicterus.

Dr. Rizo urges patients, before they ask for berberine, to first ask for safe and effective interventions they can access. “I don’t want to have people not use effective interventions that are currently available to them, and instead use something that needs to be better studied and needs to be better regulated,” she says.

While the “nature’s Ozempic” catchphrase could be drawing in potential users with its dubious comparison, berberine’s escalating popularity might also be a symptom of people seeking a quick fix, the experts worry.

“That’s my fear,” says Dr. Srinath. “ ‘Let me get this medicine, let me lose the weight fast,’ but at the end of the day, weight management is a long-term journey. It takes time, it takes effort, it is not easy, and there is no quick fix.”

This is another concern for doctors; for people who’ve struggled with losing weight for years, not seeing results from berberine could feel like another failure.

“It will give them another opportunity to feel like they are being unsuccessful or that they are failing at weight loss again,” says Dr. Almandoz. “It feeds into the hopelessness that many people with obesity have around their weight management.”

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

Berberine, a plant-derived compound historically used in traditional Chinese medicine, is experiencing increased popularity thanks to social media, especially TikTok, where the hashtag #berberine has more than 75 million views at the time of this writing. Social media influencers are promoting the compound, calling it “nature’s Ozempic,” saying they lost weight taking the supplement.

Off-the-shelf berberine comes as a yellow-orange powder usually encased in a capsule or mixed into tablet form. It’s extracted from the roots, stems, and leaves of various plants, including goldenseal and barberry.

Its use is additionally promoted for insulin resistance, polycystic ovary syndrome, and even cancer, but medical experts are warning potential users that it lacks robust evidence to support its use.

“There’s not that much data on it,” says Reshmi Srinath, MD, director of the Mount Sinai weight and metabolism management program, New York. “It’s sort of shocking now that it’s popped up into the media, to be frank.”

In response to berberine’s online popularity, the National Center for Complementary and Integrative Health issued a warning, stating that “there isn’t enough rigorous scientific evidence to determine whether it is effective.”
 

Overstated claims, lack of scientific research?

Other endocrinologists and weight management experts agree. “The claims are pretty overstated when it comes to the impact on weight loss, based on the evidence in the literature that’s currently available,” says Jaime Almandoz, MD, medical director of the UT Southwestern Medical Center, Dallas, weight wellness program.

A review of 12 randomized controlled trials evaluating berberine’s effects on obesity concluded that the treatment moderately decreased body weight. The trials included were conducted over only a few months and had small numbers of participants, and weight loss was not the primary outcome measure.

“There are few randomized controlled trials,” says Ivania Rizo, MD, an endocrinologist at Boston University. “It appears that they all have some low quality of methods which essentially can lead to an increased risk of bias.”

Another review, of 35 studies – most of them on animals and human cells and similarly underpowered – concluded that berberine showed promise for reducing blood glucose. A separate study found that berberine treatment actually increased the body weight and appetite of rats.

How exactly berberine elicits these effects is not entirely clear. Several studies point to its activation of AMP-activated protein kinase, which improves glucose tolerance in rats, as the mechanism for weight loss. Metformin, a drug used to improve glycemic control in people with type 2 diabetes, works in a similar way. Other researchers have hypothesized a link between berberine and the gut microbiome to explain its effect on type 2 diabetes and weight loss, though the clinical data to substantiate this link are shaky.

“I caution my patients about dietary supplements for weight management because we do not have high-quality data demonstrating efficacy,” Katherine Saunders, MD, DABOM, an obesity expert and cofounder of Intellihealth, a platform for obesity management, said in an email.
 

Experimenting with berberine

Despite the lack of substantial evidence supporting berberine’s use for weight management and obesity, interest in the supplement seems to be increasing. One reason could be that lifestyle interventions aren’t sufficient for most people with obesity to lose a significant amount of weight, with many requiring medical intervention, according to Dr. Saunders.

But access to treatment providers is limited. “As a result, it is not uncommon for individuals with obesity to experiment with dietary supplements like berberine,” she observed.

Dr. Srinath, the Mount Sinai doctor, says many patients have asked for her thoughts on berberine as a weight loss supplement. “I say, you know, it’s something you’re welcome to try, but we don’t have enough data at this time to recommend it.”

The hype surrounding the supplement isn’t all that surprising. About 42% of adults in the United States have obesity, according to 2019-2023 National Health and Nutrition Examination Survey data, pointing to a serious need for accessible drugs to address the condition. Berberine is available over the counter and is far cheaper than most of the newer U.S. Food and Drug Administration–approved drugs for weight loss.

Wegovy, semaglutide approved to treat obesity, can cost as much as $1,300 per package; and Ozempic, semaglutide approved to treat type 2 diabetes, can cost more than $1,000 per month. “That’s a very steep price to pay,” says Dr. Srinath.

Many insurance companies won’t cover the drugs, curbing access to Americans who need them, says Dr. Almandoz. Federally sponsored programs such as Medicare and Medicaid also don’t cover the drugs, which are approved for obesity and weight management. “That’s been a huge hole in our health care system,” says Dr. Srinath. “That’s sort of what’s been driving interest in supplements and things like that.”

Among adults trying to lose weight, only about 3% said they took prescription medication for weight loss, according to a report from the U.S. Government Accountability Office. This report includes 2013-2016 data, predating Wegovy’s approval for chronic weight management.

“These classes are notorious for being quite pricey and not well covered by insurance,” says Dr. Almandoz. “It’s easy to see why someone would promote something that someone may have more access to.”

Comparing Ozempic or Wegovy with berberine can be misleading. Those drugs work by mimicking the effect of the hormone GLP-1 to help reduce appetite.

A clinical trial assessing the efficacy of semaglutide found that adults with obesity who took the drug for 68 weeks lost approximately 15% of their body weight in combination with lifestyle changes. The FDA approval was based on this trial and three others that showed similarly substantial reductions in weight.

The trials also document the many side effects of taking the drugs, primarily gastrointestinal in nature. The short- and long-term effects of berberine, on the other hand, are less clear. Some of the clinical trials reported diarrhea and stomach upset as the most common adverse effects.

Its perception as a naturally derived option for weight loss, though, might encourage people to overlook the potential interactions that berberine could have with other drugs, according to Dr. Almandoz.

He says clinicians considering natural products or nutraceuticals for patients should check for potential side effects and find reliable database sources to determine any potential medication interactions for patients. But the unregulated nature of berberine makes this challenging, Dr. Almandoz adds.

The dosage, formulations, and quality of berberine vary in each study and each product because supplements don’t need to pass through the checks and balances of the FDA to land on shelves.

The lack of regulation could incentivize some companies to add stimulants to enhance any weight loss effect that the supplement may have. Those additives might interact with other health conditions or cause side effects like anxiety, says Dr. Almandoz.

Berberine should also not be taken during pregnancy or while breastfeeding, and it is unsafe for young children; in newborns and children, the supplement can cause higher levels of bilirubin in the blood, worsening any jaundice at birth and posing a greater risk for kernicterus.

Dr. Rizo urges patients, before they ask for berberine, to first ask for safe and effective interventions they can access. “I don’t want to have people not use effective interventions that are currently available to them, and instead use something that needs to be better studied and needs to be better regulated,” she says.

While the “nature’s Ozempic” catchphrase could be drawing in potential users with its dubious comparison, berberine’s escalating popularity might also be a symptom of people seeking a quick fix, the experts worry.

“That’s my fear,” says Dr. Srinath. “ ‘Let me get this medicine, let me lose the weight fast,’ but at the end of the day, weight management is a long-term journey. It takes time, it takes effort, it is not easy, and there is no quick fix.”

This is another concern for doctors; for people who’ve struggled with losing weight for years, not seeing results from berberine could feel like another failure.

“It will give them another opportunity to feel like they are being unsuccessful or that they are failing at weight loss again,” says Dr. Almandoz. “It feeds into the hopelessness that many people with obesity have around their weight management.”

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

Berberine, a plant-derived compound historically used in traditional Chinese medicine, is experiencing increased popularity thanks to social media, especially TikTok, where the hashtag #berberine has more than 75 million views at the time of this writing. Social media influencers are promoting the compound, calling it “nature’s Ozempic,” saying they lost weight taking the supplement.

Off-the-shelf berberine comes as a yellow-orange powder usually encased in a capsule or mixed into tablet form. It’s extracted from the roots, stems, and leaves of various plants, including goldenseal and barberry.

Its use is additionally promoted for insulin resistance, polycystic ovary syndrome, and even cancer, but medical experts are warning potential users that it lacks robust evidence to support its use.

“There’s not that much data on it,” says Reshmi Srinath, MD, director of the Mount Sinai weight and metabolism management program, New York. “It’s sort of shocking now that it’s popped up into the media, to be frank.”

In response to berberine’s online popularity, the National Center for Complementary and Integrative Health issued a warning, stating that “there isn’t enough rigorous scientific evidence to determine whether it is effective.”
 

Overstated claims, lack of scientific research?

Other endocrinologists and weight management experts agree. “The claims are pretty overstated when it comes to the impact on weight loss, based on the evidence in the literature that’s currently available,” says Jaime Almandoz, MD, medical director of the UT Southwestern Medical Center, Dallas, weight wellness program.

A review of 12 randomized controlled trials evaluating berberine’s effects on obesity concluded that the treatment moderately decreased body weight. The trials included were conducted over only a few months and had small numbers of participants, and weight loss was not the primary outcome measure.

“There are few randomized controlled trials,” says Ivania Rizo, MD, an endocrinologist at Boston University. “It appears that they all have some low quality of methods which essentially can lead to an increased risk of bias.”

Another review, of 35 studies – most of them on animals and human cells and similarly underpowered – concluded that berberine showed promise for reducing blood glucose. A separate study found that berberine treatment actually increased the body weight and appetite of rats.

How exactly berberine elicits these effects is not entirely clear. Several studies point to its activation of AMP-activated protein kinase, which improves glucose tolerance in rats, as the mechanism for weight loss. Metformin, a drug used to improve glycemic control in people with type 2 diabetes, works in a similar way. Other researchers have hypothesized a link between berberine and the gut microbiome to explain its effect on type 2 diabetes and weight loss, though the clinical data to substantiate this link are shaky.

“I caution my patients about dietary supplements for weight management because we do not have high-quality data demonstrating efficacy,” Katherine Saunders, MD, DABOM, an obesity expert and cofounder of Intellihealth, a platform for obesity management, said in an email.
 

Experimenting with berberine

Despite the lack of substantial evidence supporting berberine’s use for weight management and obesity, interest in the supplement seems to be increasing. One reason could be that lifestyle interventions aren’t sufficient for most people with obesity to lose a significant amount of weight, with many requiring medical intervention, according to Dr. Saunders.

But access to treatment providers is limited. “As a result, it is not uncommon for individuals with obesity to experiment with dietary supplements like berberine,” she observed.

Dr. Srinath, the Mount Sinai doctor, says many patients have asked for her thoughts on berberine as a weight loss supplement. “I say, you know, it’s something you’re welcome to try, but we don’t have enough data at this time to recommend it.”

The hype surrounding the supplement isn’t all that surprising. About 42% of adults in the United States have obesity, according to 2019-2023 National Health and Nutrition Examination Survey data, pointing to a serious need for accessible drugs to address the condition. Berberine is available over the counter and is far cheaper than most of the newer U.S. Food and Drug Administration–approved drugs for weight loss.

Wegovy, semaglutide approved to treat obesity, can cost as much as $1,300 per package; and Ozempic, semaglutide approved to treat type 2 diabetes, can cost more than $1,000 per month. “That’s a very steep price to pay,” says Dr. Srinath.

Many insurance companies won’t cover the drugs, curbing access to Americans who need them, says Dr. Almandoz. Federally sponsored programs such as Medicare and Medicaid also don’t cover the drugs, which are approved for obesity and weight management. “That’s been a huge hole in our health care system,” says Dr. Srinath. “That’s sort of what’s been driving interest in supplements and things like that.”

Among adults trying to lose weight, only about 3% said they took prescription medication for weight loss, according to a report from the U.S. Government Accountability Office. This report includes 2013-2016 data, predating Wegovy’s approval for chronic weight management.

“These classes are notorious for being quite pricey and not well covered by insurance,” says Dr. Almandoz. “It’s easy to see why someone would promote something that someone may have more access to.”

Comparing Ozempic or Wegovy with berberine can be misleading. Those drugs work by mimicking the effect of the hormone GLP-1 to help reduce appetite.

A clinical trial assessing the efficacy of semaglutide found that adults with obesity who took the drug for 68 weeks lost approximately 15% of their body weight in combination with lifestyle changes. The FDA approval was based on this trial and three others that showed similarly substantial reductions in weight.

The trials also document the many side effects of taking the drugs, primarily gastrointestinal in nature. The short- and long-term effects of berberine, on the other hand, are less clear. Some of the clinical trials reported diarrhea and stomach upset as the most common adverse effects.

Its perception as a naturally derived option for weight loss, though, might encourage people to overlook the potential interactions that berberine could have with other drugs, according to Dr. Almandoz.

He says clinicians considering natural products or nutraceuticals for patients should check for potential side effects and find reliable database sources to determine any potential medication interactions for patients. But the unregulated nature of berberine makes this challenging, Dr. Almandoz adds.

The dosage, formulations, and quality of berberine vary in each study and each product because supplements don’t need to pass through the checks and balances of the FDA to land on shelves.

The lack of regulation could incentivize some companies to add stimulants to enhance any weight loss effect that the supplement may have. Those additives might interact with other health conditions or cause side effects like anxiety, says Dr. Almandoz.

Berberine should also not be taken during pregnancy or while breastfeeding, and it is unsafe for young children; in newborns and children, the supplement can cause higher levels of bilirubin in the blood, worsening any jaundice at birth and posing a greater risk for kernicterus.

Dr. Rizo urges patients, before they ask for berberine, to first ask for safe and effective interventions they can access. “I don’t want to have people not use effective interventions that are currently available to them, and instead use something that needs to be better studied and needs to be better regulated,” she says.

While the “nature’s Ozempic” catchphrase could be drawing in potential users with its dubious comparison, berberine’s escalating popularity might also be a symptom of people seeking a quick fix, the experts worry.

“That’s my fear,” says Dr. Srinath. “ ‘Let me get this medicine, let me lose the weight fast,’ but at the end of the day, weight management is a long-term journey. It takes time, it takes effort, it is not easy, and there is no quick fix.”

This is another concern for doctors; for people who’ve struggled with losing weight for years, not seeing results from berberine could feel like another failure.

“It will give them another opportunity to feel like they are being unsuccessful or that they are failing at weight loss again,” says Dr. Almandoz. “It feeds into the hopelessness that many people with obesity have around their weight management.”

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

Asthma has long been associated with the use of inhalers to control symptoms. The new S2K guideline on the management of asthma, compiled by experts and published in March 2023, aims to change this. “For decades, we have known about medication that can be used to put asthma into remission. The patient can go out or travel on vacation without an inhaler. This is possible. This is a symptom-prevention approach,” said the guideline coordinator Marek Lommatzsch, MD, PhD, head senior physician of the pulmonology department at the University Medicine Rostock, Germany, in an interview.

The guideline was created by the German Respiratory Society, and a further 11 professional societies from Germany and Austria were involved in the update. The authors comprehensively revised the guideline from 2017, and the evidence-based national disease management guideline (NVL) for general asthma care from 2020 was amended.

Erika von Mutius, MD, PhD, pediatrician and professor of pediatric allergology and pulmonology at the Dr. Von Hauner Children’s Hospital, Munich, and director of the Institute of Asthma and Allergy Prevention at Helmholtz Munich, was not directly involved in the guideline. She said, “This guideline is an informed statement that takes the development over recent years into account. It had been anticipated for some time now. For me, the input from pediatricians should be particularly noted.”
 

Anti-inflammatory therapy

The significance of anti-inflammatory therapy was stressed in the NVL from 2020. The new guideline holds that anti-inflammatory therapy should be considered the primary therapeutic option. “We are making a U-turn: only treat the respiratory inflammation. Salbutamol should still only be given in exceptional cases as required,” according to Dr. Lommatzsch.

In the guideline, asthma therapy is described using an updated step-by-step plan. Inhaled glucocorticoids (ICS) represent the most important pillar of therapy. ICS can be used as permanent therapy or as as-needed therapy in fixed combination with formoterol, which rapidly dilates the airways.

Allergen immunotherapy, also known as hyposensitization, and biologics are also effective anti-inflammatory treatments, Dr. Lommatzsch added. “We must ensure that these anti-inflammatory medicines are also used effectively. Mild to moderate forms of asthma can be treated easily by a primary care physician,” he said. Basic diagnostics in the form of a blood sample are required. A somewhat more comprehensive medical history is also needed. “It takes a little more time and involves more than just taking the inhaler out of the cupboard.”

The situation regarding children, however, is a little different with regard to anti-inflammatory therapy, Dr. Von Mutius explained. “Childhood asthma has many forms, and confirming the diagnosis is not always straightforward, especially in infancy. If needed, salbutamol can be prescribed. However, the anti-inflammatory medication should usually also be administered.”

She emphasized that the guideline has been designed in a sophisticated way that offers the option of “using medical experience to see what is suitable for this family or better for this patient. This is still always subject to medical judgment and responsibility. I find this really successful.”
 

Diagnostics using biomarkers

The previous guideline concentrated on measuring lung function as a way of diagnosing asthmatic illness. Three biomarkers were brought to the fore:

• Eosinophils in the blood.
• IgE levels.
• The FeNO test (proportion of nitrogen monoxide in exhaled air).

Slightly amended, the guideline now states that the FeNO test is implemented as “an integral component of specialist diagnosis.”

The test measures the nitrogen monoxide content of exhaled air as an indicator of inflammation in the airways. However, this test must often be paid for by the patient. “In this respect, we want to give a nudge in the direction of the political decision-makers,” emphasized Dr. Lommatzsch.

Dr. Von Mutius added that use of the FeNO test has not been established in many practices and outpatient clinics. The inflammatory marker is also subject to fluctuations. “This is an update to the guideline where we must wait to see the political response.”
 

Which biologic?

Despite treatment with the established therapies, the symptoms of asthma can persist in some people with severe forms of the condition. Biologics are highly effective for these patients and are preferable in the last stage of therapy to long-term therapy with oral steroids, which have numerous side effects. The current guideline provides an overview diagram to help decide which biologic is suitable for which patient.

“There are six biologics that can be used to treat severe asthma. Officially, almost any biologic can be taken into consideration for a patient, since the approvals overlap. Nevertheless, we know that certain patients benefit hugely from certain biologics. A targeted choice should therefore be made,” explained Dr. Lommatzsch.

Biologics were mentioned in the 2020 NVL but not to the great extent that they are in the latest version. “For the first time, we have created an overview diagram for the individual choice of biologic. With it, we have now set a standard,” said Dr. Lommatzsch.

Therapy with biologics has brought about rapid progress for adults. Dr. Von Mutius anticipates challenges in approving such therapeutics for pediatric treatment. “As is often the case, these therapies are not approved for young children. Meanwhile, dupilumab is approved for children aged 6 months and older; unfortunately, the indication for this is actually atopic dermatitis,” she explains.

When using this therapy for pediatric patients, it is therefore important to explain the options to parents and to inform them of side effects. Severe forms of asthma are rare in children; they are uncommon in adults but are more prevalent than in children.
 

Children and adolescents

One new chapter in the guideline describes giving medical advice to adolescents choosing a career. A table has been compiled that contains information regarding jobs and their respective allergy and asthma risk. The table is designed to be displayed in a medical practice.

Another chapter characterizes the interrelation between asthma and mental health. It differentiates between psychiatric comorbidities for which the patient requires professional help and the stress caused by the asthmatic illness itself. Many patients do not have a mental illness but do suffer under the everyday strain of having asthma, said Dr. Lommatzsch. Therefore, it is important to educate patients and their relatives on how to make a strength out of this supposed weakness – the asthmatic illness. “We have established a procedure for this and have summarized its key points in the guideline,” said Dr. Lommatzsch.

Other updates to the guideline cover asthma in different contexts, such as in pregnant women. The updates address adrenal insufficiency as a side effect of the use of steroids over many years. In addition, the guideline contains a chapter on digital apps that can help with diagnostics and medical history.

Dr. Lommatzsch highlighted a new tool. “By using 15 key points summarized in a table, the guideline displays the essential differences between COPD [chronic obstructive pulmonary disease] and asthma in terms of the symptoms and the findings. It is the most modern table available in Germany that differentiates between the two diseases.”

This article was translated from the Medscape German Edition and a version appeared on Medscape.com.

Asthma has long been associated with the use of inhalers to control symptoms. The new S2K guideline on the management of asthma, compiled by experts and published in March 2023, aims to change this. “For decades, we have known about medication that can be used to put asthma into remission. The patient can go out or travel on vacation without an inhaler. This is possible. This is a symptom-prevention approach,” said the guideline coordinator Marek Lommatzsch, MD, PhD, head senior physician of the pulmonology department at the University Medicine Rostock, Germany, in an interview.

The guideline was created by the German Respiratory Society, and a further 11 professional societies from Germany and Austria were involved in the update. The authors comprehensively revised the guideline from 2017, and the evidence-based national disease management guideline (NVL) for general asthma care from 2020 was amended.

Erika von Mutius, MD, PhD, pediatrician and professor of pediatric allergology and pulmonology at the Dr. Von Hauner Children’s Hospital, Munich, and director of the Institute of Asthma and Allergy Prevention at Helmholtz Munich, was not directly involved in the guideline. She said, “This guideline is an informed statement that takes the development over recent years into account. It had been anticipated for some time now. For me, the input from pediatricians should be particularly noted.”
 

Anti-inflammatory therapy

The significance of anti-inflammatory therapy was stressed in the NVL from 2020. The new guideline holds that anti-inflammatory therapy should be considered the primary therapeutic option. “We are making a U-turn: only treat the respiratory inflammation. Salbutamol should still only be given in exceptional cases as required,” according to Dr. Lommatzsch.

In the guideline, asthma therapy is described using an updated step-by-step plan. Inhaled glucocorticoids (ICS) represent the most important pillar of therapy. ICS can be used as permanent therapy or as as-needed therapy in fixed combination with formoterol, which rapidly dilates the airways.

Allergen immunotherapy, also known as hyposensitization, and biologics are also effective anti-inflammatory treatments, Dr. Lommatzsch added. “We must ensure that these anti-inflammatory medicines are also used effectively. Mild to moderate forms of asthma can be treated easily by a primary care physician,” he said. Basic diagnostics in the form of a blood sample are required. A somewhat more comprehensive medical history is also needed. “It takes a little more time and involves more than just taking the inhaler out of the cupboard.”

The situation regarding children, however, is a little different with regard to anti-inflammatory therapy, Dr. Von Mutius explained. “Childhood asthma has many forms, and confirming the diagnosis is not always straightforward, especially in infancy. If needed, salbutamol can be prescribed. However, the anti-inflammatory medication should usually also be administered.”

She emphasized that the guideline has been designed in a sophisticated way that offers the option of “using medical experience to see what is suitable for this family or better for this patient. This is still always subject to medical judgment and responsibility. I find this really successful.”
 

Diagnostics using biomarkers

The previous guideline concentrated on measuring lung function as a way of diagnosing asthmatic illness. Three biomarkers were brought to the fore:

• Eosinophils in the blood.
• IgE levels.
• The FeNO test (proportion of nitrogen monoxide in exhaled air).

Slightly amended, the guideline now states that the FeNO test is implemented as “an integral component of specialist diagnosis.”

The test measures the nitrogen monoxide content of exhaled air as an indicator of inflammation in the airways. However, this test must often be paid for by the patient. “In this respect, we want to give a nudge in the direction of the political decision-makers,” emphasized Dr. Lommatzsch.

Dr. Von Mutius added that use of the FeNO test has not been established in many practices and outpatient clinics. The inflammatory marker is also subject to fluctuations. “This is an update to the guideline where we must wait to see the political response.”
 

Which biologic?

Despite treatment with the established therapies, the symptoms of asthma can persist in some people with severe forms of the condition. Biologics are highly effective for these patients and are preferable in the last stage of therapy to long-term therapy with oral steroids, which have numerous side effects. The current guideline provides an overview diagram to help decide which biologic is suitable for which patient.

“There are six biologics that can be used to treat severe asthma. Officially, almost any biologic can be taken into consideration for a patient, since the approvals overlap. Nevertheless, we know that certain patients benefit hugely from certain biologics. A targeted choice should therefore be made,” explained Dr. Lommatzsch.

Biologics were mentioned in the 2020 NVL but not to the great extent that they are in the latest version. “For the first time, we have created an overview diagram for the individual choice of biologic. With it, we have now set a standard,” said Dr. Lommatzsch.

Therapy with biologics has brought about rapid progress for adults. Dr. Von Mutius anticipates challenges in approving such therapeutics for pediatric treatment. “As is often the case, these therapies are not approved for young children. Meanwhile, dupilumab is approved for children aged 6 months and older; unfortunately, the indication for this is actually atopic dermatitis,” she explains.

When using this therapy for pediatric patients, it is therefore important to explain the options to parents and to inform them of side effects. Severe forms of asthma are rare in children; they are uncommon in adults but are more prevalent than in children.
 

Children and adolescents

One new chapter in the guideline describes giving medical advice to adolescents choosing a career. A table has been compiled that contains information regarding jobs and their respective allergy and asthma risk. The table is designed to be displayed in a medical practice.

Another chapter characterizes the interrelation between asthma and mental health. It differentiates between psychiatric comorbidities for which the patient requires professional help and the stress caused by the asthmatic illness itself. Many patients do not have a mental illness but do suffer under the everyday strain of having asthma, said Dr. Lommatzsch. Therefore, it is important to educate patients and their relatives on how to make a strength out of this supposed weakness – the asthmatic illness. “We have established a procedure for this and have summarized its key points in the guideline,” said Dr. Lommatzsch.

Other updates to the guideline cover asthma in different contexts, such as in pregnant women. The updates address adrenal insufficiency as a side effect of the use of steroids over many years. In addition, the guideline contains a chapter on digital apps that can help with diagnostics and medical history.

Dr. Lommatzsch highlighted a new tool. “By using 15 key points summarized in a table, the guideline displays the essential differences between COPD [chronic obstructive pulmonary disease] and asthma in terms of the symptoms and the findings. It is the most modern table available in Germany that differentiates between the two diseases.”

This article was translated from the Medscape German Edition and a version appeared on Medscape.com.

Asthma has long been associated with the use of inhalers to control symptoms. The new S2K guideline on the management of asthma, compiled by experts and published in March 2023, aims to change this. “For decades, we have known about medication that can be used to put asthma into remission. The patient can go out or travel on vacation without an inhaler. This is possible. This is a symptom-prevention approach,” said the guideline coordinator Marek Lommatzsch, MD, PhD, head senior physician of the pulmonology department at the University Medicine Rostock, Germany, in an interview.

The guideline was created by the German Respiratory Society, and a further 11 professional societies from Germany and Austria were involved in the update. The authors comprehensively revised the guideline from 2017, and the evidence-based national disease management guideline (NVL) for general asthma care from 2020 was amended.

Erika von Mutius, MD, PhD, pediatrician and professor of pediatric allergology and pulmonology at the Dr. Von Hauner Children’s Hospital, Munich, and director of the Institute of Asthma and Allergy Prevention at Helmholtz Munich, was not directly involved in the guideline. She said, “This guideline is an informed statement that takes the development over recent years into account. It had been anticipated for some time now. For me, the input from pediatricians should be particularly noted.”
 

Anti-inflammatory therapy

The significance of anti-inflammatory therapy was stressed in the NVL from 2020. The new guideline holds that anti-inflammatory therapy should be considered the primary therapeutic option. “We are making a U-turn: only treat the respiratory inflammation. Salbutamol should still only be given in exceptional cases as required,” according to Dr. Lommatzsch.

In the guideline, asthma therapy is described using an updated step-by-step plan. Inhaled glucocorticoids (ICS) represent the most important pillar of therapy. ICS can be used as permanent therapy or as as-needed therapy in fixed combination with formoterol, which rapidly dilates the airways.

Allergen immunotherapy, also known as hyposensitization, and biologics are also effective anti-inflammatory treatments, Dr. Lommatzsch added. “We must ensure that these anti-inflammatory medicines are also used effectively. Mild to moderate forms of asthma can be treated easily by a primary care physician,” he said. Basic diagnostics in the form of a blood sample are required. A somewhat more comprehensive medical history is also needed. “It takes a little more time and involves more than just taking the inhaler out of the cupboard.”

The situation regarding children, however, is a little different with regard to anti-inflammatory therapy, Dr. Von Mutius explained. “Childhood asthma has many forms, and confirming the diagnosis is not always straightforward, especially in infancy. If needed, salbutamol can be prescribed. However, the anti-inflammatory medication should usually also be administered.”

She emphasized that the guideline has been designed in a sophisticated way that offers the option of “using medical experience to see what is suitable for this family or better for this patient. This is still always subject to medical judgment and responsibility. I find this really successful.”
 

Diagnostics using biomarkers

The previous guideline concentrated on measuring lung function as a way of diagnosing asthmatic illness. Three biomarkers were brought to the fore:

• Eosinophils in the blood.
• IgE levels.
• The FeNO test (proportion of nitrogen monoxide in exhaled air).

Slightly amended, the guideline now states that the FeNO test is implemented as “an integral component of specialist diagnosis.”

The test measures the nitrogen monoxide content of exhaled air as an indicator of inflammation in the airways. However, this test must often be paid for by the patient. “In this respect, we want to give a nudge in the direction of the political decision-makers,” emphasized Dr. Lommatzsch.

Dr. Von Mutius added that use of the FeNO test has not been established in many practices and outpatient clinics. The inflammatory marker is also subject to fluctuations. “This is an update to the guideline where we must wait to see the political response.”
 

Which biologic?

Despite treatment with the established therapies, the symptoms of asthma can persist in some people with severe forms of the condition. Biologics are highly effective for these patients and are preferable in the last stage of therapy to long-term therapy with oral steroids, which have numerous side effects. The current guideline provides an overview diagram to help decide which biologic is suitable for which patient.

“There are six biologics that can be used to treat severe asthma. Officially, almost any biologic can be taken into consideration for a patient, since the approvals overlap. Nevertheless, we know that certain patients benefit hugely from certain biologics. A targeted choice should therefore be made,” explained Dr. Lommatzsch.

Biologics were mentioned in the 2020 NVL but not to the great extent that they are in the latest version. “For the first time, we have created an overview diagram for the individual choice of biologic. With it, we have now set a standard,” said Dr. Lommatzsch.

Therapy with biologics has brought about rapid progress for adults. Dr. Von Mutius anticipates challenges in approving such therapeutics for pediatric treatment. “As is often the case, these therapies are not approved for young children. Meanwhile, dupilumab is approved for children aged 6 months and older; unfortunately, the indication for this is actually atopic dermatitis,” she explains.

When using this therapy for pediatric patients, it is therefore important to explain the options to parents and to inform them of side effects. Severe forms of asthma are rare in children; they are uncommon in adults but are more prevalent than in children.
 

Children and adolescents

One new chapter in the guideline describes giving medical advice to adolescents choosing a career. A table has been compiled that contains information regarding jobs and their respective allergy and asthma risk. The table is designed to be displayed in a medical practice.

Another chapter characterizes the interrelation between asthma and mental health. It differentiates between psychiatric comorbidities for which the patient requires professional help and the stress caused by the asthmatic illness itself. Many patients do not have a mental illness but do suffer under the everyday strain of having asthma, said Dr. Lommatzsch. Therefore, it is important to educate patients and their relatives on how to make a strength out of this supposed weakness – the asthmatic illness. “We have established a procedure for this and have summarized its key points in the guideline,” said Dr. Lommatzsch.

Other updates to the guideline cover asthma in different contexts, such as in pregnant women. The updates address adrenal insufficiency as a side effect of the use of steroids over many years. In addition, the guideline contains a chapter on digital apps that can help with diagnostics and medical history.

Dr. Lommatzsch highlighted a new tool. “By using 15 key points summarized in a table, the guideline displays the essential differences between COPD [chronic obstructive pulmonary disease] and asthma in terms of the symptoms and the findings. It is the most modern table available in Germany that differentiates between the two diseases.”

This article was translated from the Medscape German Edition and a version appeared on Medscape.com.

An estimated 3 in every 10 white-tailed deer in the United States have had COVID-19, and new research suggests deer populations could be a source of virus mutations that may be passed to humans.

According to the U.S. Department of Agriculture, which led the research project, humans transmitted the virus to deer at least 100 times. The virus then spread widely among free-ranging deer populations, and there were three possible cases of the deer transmitting the virus to humans.

The data comes from tests done between November 2021 and April 2022 on more than 12,000 deer found across half of the United States. Sequencing of the virus found in the deer showed that deer had been exposed to all of the prominent variants, including Alpha, Gamma, Delta, and Omicron.

Some of the findings about transmission were published in the journal Nature Communications, in which researchers noted that in addition to being identified in deer, the virus has been found in wild and domestic animals, including mink, rats, otters, ferrets, hamsters, gorillas, cats, dogs, lions, and tigers. Animal-to-human transmission has been documented or suspected in mink and domestic cats, in addition to white-tailed deer.

The findings are important because the animal populations can become “reservoirs ... in which the virus circulates covertly, persisting in the population and can be transmitted to other animals or humans potentially causing disease outbreaks,” according to the paper, which was a collaboration among scientists from the U.S. Department of Agriculture, CDC, and the University of Missouri–Columbia.

In the three cases of possible deer-to-human transmission, researchers said that mutated versions of the virus previously found only in deer had been found in COVID test samples taken from one person in North Carolina and two people in Massachusetts. Those deer-specific mutated versions of the virus have not been found in any other human samples, lending evidence that the mutations occurred within deer.

“Deer regularly interact with humans and are commonly found in human environments – near our homes, pets, wastewater, and trash,” researcher and University of Missouri–Columbia professor Xiu-Feng “Henry” Wan, PhD, said in a statement. “The potential for SARS-CoV-2, or any zoonotic disease, to persist and evolve in wildlife populations can pose unique public health risks.”

In the Nature Communications paper, the researchers suggested that deer may be exposed to the virus from human food waste, masks, or other waste products. The authors concluded that further study is needed to determine how virus transmission occurs between deer and humans.

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

An estimated 3 in every 10 white-tailed deer in the United States have had COVID-19, and new research suggests deer populations could be a source of virus mutations that may be passed to humans.

According to the U.S. Department of Agriculture, which led the research project, humans transmitted the virus to deer at least 100 times. The virus then spread widely among free-ranging deer populations, and there were three possible cases of the deer transmitting the virus to humans.

The data comes from tests done between November 2021 and April 2022 on more than 12,000 deer found across half of the United States. Sequencing of the virus found in the deer showed that deer had been exposed to all of the prominent variants, including Alpha, Gamma, Delta, and Omicron.

Some of the findings about transmission were published in the journal Nature Communications, in which researchers noted that in addition to being identified in deer, the virus has been found in wild and domestic animals, including mink, rats, otters, ferrets, hamsters, gorillas, cats, dogs, lions, and tigers. Animal-to-human transmission has been documented or suspected in mink and domestic cats, in addition to white-tailed deer.

The findings are important because the animal populations can become “reservoirs ... in which the virus circulates covertly, persisting in the population and can be transmitted to other animals or humans potentially causing disease outbreaks,” according to the paper, which was a collaboration among scientists from the U.S. Department of Agriculture, CDC, and the University of Missouri–Columbia.

In the three cases of possible deer-to-human transmission, researchers said that mutated versions of the virus previously found only in deer had been found in COVID test samples taken from one person in North Carolina and two people in Massachusetts. Those deer-specific mutated versions of the virus have not been found in any other human samples, lending evidence that the mutations occurred within deer.

“Deer regularly interact with humans and are commonly found in human environments – near our homes, pets, wastewater, and trash,” researcher and University of Missouri–Columbia professor Xiu-Feng “Henry” Wan, PhD, said in a statement. “The potential for SARS-CoV-2, or any zoonotic disease, to persist and evolve in wildlife populations can pose unique public health risks.”

In the Nature Communications paper, the researchers suggested that deer may be exposed to the virus from human food waste, masks, or other waste products. The authors concluded that further study is needed to determine how virus transmission occurs between deer and humans.

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

An estimated 3 in every 10 white-tailed deer in the United States have had COVID-19, and new research suggests deer populations could be a source of virus mutations that may be passed to humans.

According to the U.S. Department of Agriculture, which led the research project, humans transmitted the virus to deer at least 100 times. The virus then spread widely among free-ranging deer populations, and there were three possible cases of the deer transmitting the virus to humans.

The data comes from tests done between November 2021 and April 2022 on more than 12,000 deer found across half of the United States. Sequencing of the virus found in the deer showed that deer had been exposed to all of the prominent variants, including Alpha, Gamma, Delta, and Omicron.

Some of the findings about transmission were published in the journal Nature Communications, in which researchers noted that in addition to being identified in deer, the virus has been found in wild and domestic animals, including mink, rats, otters, ferrets, hamsters, gorillas, cats, dogs, lions, and tigers. Animal-to-human transmission has been documented or suspected in mink and domestic cats, in addition to white-tailed deer.

The findings are important because the animal populations can become “reservoirs ... in which the virus circulates covertly, persisting in the population and can be transmitted to other animals or humans potentially causing disease outbreaks,” according to the paper, which was a collaboration among scientists from the U.S. Department of Agriculture, CDC, and the University of Missouri–Columbia.

In the three cases of possible deer-to-human transmission, researchers said that mutated versions of the virus previously found only in deer had been found in COVID test samples taken from one person in North Carolina and two people in Massachusetts. Those deer-specific mutated versions of the virus have not been found in any other human samples, lending evidence that the mutations occurred within deer.

“Deer regularly interact with humans and are commonly found in human environments – near our homes, pets, wastewater, and trash,” researcher and University of Missouri–Columbia professor Xiu-Feng “Henry” Wan, PhD, said in a statement. “The potential for SARS-CoV-2, or any zoonotic disease, to persist and evolve in wildlife populations can pose unique public health risks.”

In the Nature Communications paper, the researchers suggested that deer may be exposed to the virus from human food waste, masks, or other waste products. The authors concluded that further study is needed to determine how virus transmission occurs between deer and humans.

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

The Food and Drug Administration has approved a new catheter designed to treat urinary tract symptoms of an enlarged prostate, also known as benign prostate hyperplasia (BPH).

Designed and marketed by Urotronic (Plymouth, Minn.), the Optilume BPH Catheter System employs mechanical dilation to relieve obstruction of the prostate and then delivers paclitaxel to aid in prostate healing. The device is used in an outpatient setting and is less invasive than other procedures.

Olivier Le Moal/Getty Images

“There’s nothing else like Optilume BPH that’s currently available, it’s the only treatment option that requires no cutting, burning, steaming, or implants,” said Urotronic President and CEO David Perry in a press release.

Two randomized trials, EVEREST-1 and PINNACLE, both showed that the Optilume BPH system improved urinary flow rate and decreased the amount of urine stored in the bladder following urination. Men who used the OPTILUME device were able to ejaculate normally and reported no sexual difficulties.

“Optilume BPH is the next generation of minimally invasive technology, creating a new drug device space among BPH therapies,” Steven A. Kaplan, MD, professor of urology at the Icahn School of Medicine at Mount Sinai, New York, said in the press release. Dr. Kaplan led the EVEREST-1 and PINNACLE studies, which Urotronic funded.

More than 80% of men older than age 70 have an enlarged prostate, based on autopsy analyses.

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

The Food and Drug Administration has approved a new catheter designed to treat urinary tract symptoms of an enlarged prostate, also known as benign prostate hyperplasia (BPH).

Designed and marketed by Urotronic (Plymouth, Minn.), the Optilume BPH Catheter System employs mechanical dilation to relieve obstruction of the prostate and then delivers paclitaxel to aid in prostate healing. The device is used in an outpatient setting and is less invasive than other procedures.

Olivier Le Moal/Getty Images

“There’s nothing else like Optilume BPH that’s currently available, it’s the only treatment option that requires no cutting, burning, steaming, or implants,” said Urotronic President and CEO David Perry in a press release.

Two randomized trials, EVEREST-1 and PINNACLE, both showed that the Optilume BPH system improved urinary flow rate and decreased the amount of urine stored in the bladder following urination. Men who used the OPTILUME device were able to ejaculate normally and reported no sexual difficulties.

“Optilume BPH is the next generation of minimally invasive technology, creating a new drug device space among BPH therapies,” Steven A. Kaplan, MD, professor of urology at the Icahn School of Medicine at Mount Sinai, New York, said in the press release. Dr. Kaplan led the EVEREST-1 and PINNACLE studies, which Urotronic funded.

More than 80% of men older than age 70 have an enlarged prostate, based on autopsy analyses.

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

The Food and Drug Administration has approved a new catheter designed to treat urinary tract symptoms of an enlarged prostate, also known as benign prostate hyperplasia (BPH).

Designed and marketed by Urotronic (Plymouth, Minn.), the Optilume BPH Catheter System employs mechanical dilation to relieve obstruction of the prostate and then delivers paclitaxel to aid in prostate healing. The device is used in an outpatient setting and is less invasive than other procedures.

Olivier Le Moal/Getty Images

“There’s nothing else like Optilume BPH that’s currently available, it’s the only treatment option that requires no cutting, burning, steaming, or implants,” said Urotronic President and CEO David Perry in a press release.

Two randomized trials, EVEREST-1 and PINNACLE, both showed that the Optilume BPH system improved urinary flow rate and decreased the amount of urine stored in the bladder following urination. Men who used the OPTILUME device were able to ejaculate normally and reported no sexual difficulties.

“Optilume BPH is the next generation of minimally invasive technology, creating a new drug device space among BPH therapies,” Steven A. Kaplan, MD, professor of urology at the Icahn School of Medicine at Mount Sinai, New York, said in the press release. Dr. Kaplan led the EVEREST-1 and PINNACLE studies, which Urotronic funded.

More than 80% of men older than age 70 have an enlarged prostate, based on autopsy analyses.

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

TOPLINE:

Although practice patterns vary widely, modified, reduced-dose FOLFIRINOX is as effective as standard, full-dose regimens for patients with metastatic pancreatic cancer in the first-line setting, and it is less likely to cause febrile neutropenia.

METHODOLOGY:

• No randomized controlled trials have directly compared modified FOLFIRINOX to standard FOLFIRINOX; this meta-analysis aims to fill the evidence gap.
• The investigators winnowed hundreds of first-line FOLFIRINOX studies down to 37 – 11 prospective and 26 retrospective analyses – to assess practice patterns and clinical outcomes.
• Dose information was grouped into four categories: planned dose in the standard FOLFIRINOX group; actual administered dose in the standard group; planned dose in the modified group; actual administered dose in the modified group.

TAKEAWAY:

• There were 12 types of “planned” dose reductions in FOLFIRINOX: 75%-100% oxaliplatin, 75%-100% irinotecan, 0%-100% 5-fluorouracil (5-FU) bolus, and 75%-133% 5-FU continuous injection.
• Doses actually delivered fell further to 54%-96% for oxaliplatin, 61%-88% for irinotecan, 0%-92% for 5-FU bolus, and 63%-98% 5-FU continuous injection.
• Despite the variations in dosing, reduced doses of FOLFIRINOX were associated with a slightly but not significantly higher objective response rate: 33.8% versus 28.2% for standard dosing (P = .1).
• The incidence of febrile neutropenia was significantly lower in the reduced-dose groups: 5.5% with modified FOLFIRINOX versus 11.6% with standard (P = .03).

IN PRACTICE:

Although the study supports reduced-dose regimens, it also shows that there is “still no consensus” on appropriate dose modification, the authors said. “The best dose modification protocol” remains to be determined and standardized for metastatic pancreatic cancer.

SOURCE:

The study was led by Kwangrok Jung at Seoul (South Korea) National University, and was published June 29 in Therapeutic Advances in Medical Oncology.

LIMITATIONS:

• Only 11 of the 37 studies were prospective.
• The studies often lacked key information, including the reason for dose reductions or detailed dose reduction protocols.
• Studies were also inconsistent in how they reported FOLFIRINOX dose modifications.

DISCLOSURES:

There was no funding for the study, and the investigators had no disclosures.

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

TOPLINE:

Although practice patterns vary widely, modified, reduced-dose FOLFIRINOX is as effective as standard, full-dose regimens for patients with metastatic pancreatic cancer in the first-line setting, and it is less likely to cause febrile neutropenia.

METHODOLOGY:

• No randomized controlled trials have directly compared modified FOLFIRINOX to standard FOLFIRINOX; this meta-analysis aims to fill the evidence gap.
• The investigators winnowed hundreds of first-line FOLFIRINOX studies down to 37 – 11 prospective and 26 retrospective analyses – to assess practice patterns and clinical outcomes.
• Dose information was grouped into four categories: planned dose in the standard FOLFIRINOX group; actual administered dose in the standard group; planned dose in the modified group; actual administered dose in the modified group.

TAKEAWAY:

• There were 12 types of “planned” dose reductions in FOLFIRINOX: 75%-100% oxaliplatin, 75%-100% irinotecan, 0%-100% 5-fluorouracil (5-FU) bolus, and 75%-133% 5-FU continuous injection.
• Doses actually delivered fell further to 54%-96% for oxaliplatin, 61%-88% for irinotecan, 0%-92% for 5-FU bolus, and 63%-98% 5-FU continuous injection.
• Despite the variations in dosing, reduced doses of FOLFIRINOX were associated with a slightly but not significantly higher objective response rate: 33.8% versus 28.2% for standard dosing (P = .1).
• The incidence of febrile neutropenia was significantly lower in the reduced-dose groups: 5.5% with modified FOLFIRINOX versus 11.6% with standard (P = .03).

IN PRACTICE:

Although the study supports reduced-dose regimens, it also shows that there is “still no consensus” on appropriate dose modification, the authors said. “The best dose modification protocol” remains to be determined and standardized for metastatic pancreatic cancer.

SOURCE:

The study was led by Kwangrok Jung at Seoul (South Korea) National University, and was published June 29 in Therapeutic Advances in Medical Oncology.

LIMITATIONS:

• Only 11 of the 37 studies were prospective.
• The studies often lacked key information, including the reason for dose reductions or detailed dose reduction protocols.
• Studies were also inconsistent in how they reported FOLFIRINOX dose modifications.

DISCLOSURES:

There was no funding for the study, and the investigators had no disclosures.

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

TOPLINE:

Although practice patterns vary widely, modified, reduced-dose FOLFIRINOX is as effective as standard, full-dose regimens for patients with metastatic pancreatic cancer in the first-line setting, and it is less likely to cause febrile neutropenia.

METHODOLOGY:

• No randomized controlled trials have directly compared modified FOLFIRINOX to standard FOLFIRINOX; this meta-analysis aims to fill the evidence gap.
• The investigators winnowed hundreds of first-line FOLFIRINOX studies down to 37 – 11 prospective and 26 retrospective analyses – to assess practice patterns and clinical outcomes.
• Dose information was grouped into four categories: planned dose in the standard FOLFIRINOX group; actual administered dose in the standard group; planned dose in the modified group; actual administered dose in the modified group.

TAKEAWAY:

• There were 12 types of “planned” dose reductions in FOLFIRINOX: 75%-100% oxaliplatin, 75%-100% irinotecan, 0%-100% 5-fluorouracil (5-FU) bolus, and 75%-133% 5-FU continuous injection.
• Doses actually delivered fell further to 54%-96% for oxaliplatin, 61%-88% for irinotecan, 0%-92% for 5-FU bolus, and 63%-98% 5-FU continuous injection.
• Despite the variations in dosing, reduced doses of FOLFIRINOX were associated with a slightly but not significantly higher objective response rate: 33.8% versus 28.2% for standard dosing (P = .1).
• The incidence of febrile neutropenia was significantly lower in the reduced-dose groups: 5.5% with modified FOLFIRINOX versus 11.6% with standard (P = .03).

IN PRACTICE:

Although the study supports reduced-dose regimens, it also shows that there is “still no consensus” on appropriate dose modification, the authors said. “The best dose modification protocol” remains to be determined and standardized for metastatic pancreatic cancer.

SOURCE:

The study was led by Kwangrok Jung at Seoul (South Korea) National University, and was published June 29 in Therapeutic Advances in Medical Oncology.

LIMITATIONS:

• Only 11 of the 37 studies were prospective.
• The studies often lacked key information, including the reason for dose reductions or detailed dose reduction protocols.
• Studies were also inconsistent in how they reported FOLFIRINOX dose modifications.

DISCLOSURES:

There was no funding for the study, and the investigators had no disclosures.

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

Nonalcoholic fatty liver disease will now be called metabolic dysfunction–associated steatotic liver disease, or MASLD, according to new nomenclature adopted by a global consensus panel composed mostly of hepatology researchers and clinicians.

The new nomenclature, published in the journal Hepatology, includes the umbrella term steatotic liver disease, or SLD, which will cover MASLD and MetALD, a term describing people with MASLD who consume more than 140 grams of alcohol per week for women and 210 grams per week for men.

Metabolic dysfunction–associated steatohepatitis, or MASH, will replace the term nonalcoholic steatohepatitis, or NASH.

Mary E. Rinella, MD, of University of Chicago Medicine led the consensus group. The changes were needed, Dr. Rinella and her colleagues argued, because the terms “fatty liver disease” “and nonalcoholic” could be considered to confer stigma, and to better reflect the metabolic dysfunction occurring in the disease. Under the new nomenclature, people with MASLD must have a cardiometabolic risk factor, such as type 2 diabetes. People without metabolic parameters and no known cause will be classed as having cryptogenic SLD.

While the new nomenclature largely conserves existing disease definitions, it allows for alcohol consumption beyond current parameters for nonalcoholic forms of the disease. “There are individuals with risk factors for NAFLD, such as type 2 diabetes, who consume more alcohol than the relatively strict thresholds used to define the nonalcoholic nature of the disease [and] are excluded from trials and consideration for treatments,” the authors wrote.

Moreover, they wrote, “within MetALD there is a continuum where conceptually the condition can be seen to be MASLD or ALD predominant. This may vary over time within a given individual.”

Respondents overwhelmingly agreed, however, that even moderate alcohol use alters the natural history of the disease and that patients with more than minimal alcohol consumption should be analyzed separately in clinical trials.

The new nomenclature reflects a 3-year effort involving some 236 panelists from 56 countries who participated in several rounds of online surveys using a Delphi process. Pediatricians, gastroenterologists, and endocrinologists also participated as well as some patient advocates. Changes were based on a super-majority of opinion (67% or higher), though the consensus on whether the term “fatty” was stigmatizing never reached that threshold. In early rounds of surveys only 44% of respondents considered the word “fatty” to be stigmatizing, while more considered “nonalcoholic” to be problematic.

“Substantial proportions of the respondents deemed terms such as ‘fatty’ stigmatizing, hence its exclusion as part of any new name,” Dr. Rinella and her colleagues wrote. “Although health care professionals may contend that patients have not reported this previously, this likely reflects in part a failure to ask the question in the first place and the power imbalance in the doctor-patient relationship.” The authors noted that the new terminology may help raise awareness at a time when new therapeutics are in sight and it becomes more important to identify at-risk individuals.

Of concern was whether the new definitions would alter the utility of earlier data from registries and trials. However, the authors determined that some 98% of people registered in a European NAFLD cohort would meet the new criteria for MASLD. “Maintenance of the term, and clinical definition, of steatohepatitis ensures retention and validity of prior data from clinical trials and biomarker discovery studies of patients with NASH to be generalizable to individuals classified as MASLD or MASH under the new nomenclature, without impeding the efficiency of research,” they stated.

The effort was spearheaded by three international liver societies: La Asociación Latinoamericana para el Estudio del Hígado, the American Association for the Study of Liver Diseases, and the European Association for the Study of the Liver, as well as the cochairs of the NAFLD Nomenclature Initiative.

Each of the authors disclosed a number of potential conflicts of interest.

Nonalcoholic fatty liver disease will now be called metabolic dysfunction–associated steatotic liver disease, or MASLD, according to new nomenclature adopted by a global consensus panel composed mostly of hepatology researchers and clinicians.

The new nomenclature, published in the journal Hepatology, includes the umbrella term steatotic liver disease, or SLD, which will cover MASLD and MetALD, a term describing people with MASLD who consume more than 140 grams of alcohol per week for women and 210 grams per week for men.

Metabolic dysfunction–associated steatohepatitis, or MASH, will replace the term nonalcoholic steatohepatitis, or NASH.

Mary E. Rinella, MD, of University of Chicago Medicine led the consensus group. The changes were needed, Dr. Rinella and her colleagues argued, because the terms “fatty liver disease” “and nonalcoholic” could be considered to confer stigma, and to better reflect the metabolic dysfunction occurring in the disease. Under the new nomenclature, people with MASLD must have a cardiometabolic risk factor, such as type 2 diabetes. People without metabolic parameters and no known cause will be classed as having cryptogenic SLD.

While the new nomenclature largely conserves existing disease definitions, it allows for alcohol consumption beyond current parameters for nonalcoholic forms of the disease. “There are individuals with risk factors for NAFLD, such as type 2 diabetes, who consume more alcohol than the relatively strict thresholds used to define the nonalcoholic nature of the disease [and] are excluded from trials and consideration for treatments,” the authors wrote.

Moreover, they wrote, “within MetALD there is a continuum where conceptually the condition can be seen to be MASLD or ALD predominant. This may vary over time within a given individual.”

Respondents overwhelmingly agreed, however, that even moderate alcohol use alters the natural history of the disease and that patients with more than minimal alcohol consumption should be analyzed separately in clinical trials.

The new nomenclature reflects a 3-year effort involving some 236 panelists from 56 countries who participated in several rounds of online surveys using a Delphi process. Pediatricians, gastroenterologists, and endocrinologists also participated as well as some patient advocates. Changes were based on a super-majority of opinion (67% or higher), though the consensus on whether the term “fatty” was stigmatizing never reached that threshold. In early rounds of surveys only 44% of respondents considered the word “fatty” to be stigmatizing, while more considered “nonalcoholic” to be problematic.

“Substantial proportions of the respondents deemed terms such as ‘fatty’ stigmatizing, hence its exclusion as part of any new name,” Dr. Rinella and her colleagues wrote. “Although health care professionals may contend that patients have not reported this previously, this likely reflects in part a failure to ask the question in the first place and the power imbalance in the doctor-patient relationship.” The authors noted that the new terminology may help raise awareness at a time when new therapeutics are in sight and it becomes more important to identify at-risk individuals.

Of concern was whether the new definitions would alter the utility of earlier data from registries and trials. However, the authors determined that some 98% of people registered in a European NAFLD cohort would meet the new criteria for MASLD. “Maintenance of the term, and clinical definition, of steatohepatitis ensures retention and validity of prior data from clinical trials and biomarker discovery studies of patients with NASH to be generalizable to individuals classified as MASLD or MASH under the new nomenclature, without impeding the efficiency of research,” they stated.

The effort was spearheaded by three international liver societies: La Asociación Latinoamericana para el Estudio del Hígado, the American Association for the Study of Liver Diseases, and the European Association for the Study of the Liver, as well as the cochairs of the NAFLD Nomenclature Initiative.

Each of the authors disclosed a number of potential conflicts of interest.

Nonalcoholic fatty liver disease will now be called metabolic dysfunction–associated steatotic liver disease, or MASLD, according to new nomenclature adopted by a global consensus panel composed mostly of hepatology researchers and clinicians.

The new nomenclature, published in the journal Hepatology, includes the umbrella term steatotic liver disease, or SLD, which will cover MASLD and MetALD, a term describing people with MASLD who consume more than 140 grams of alcohol per week for women and 210 grams per week for men.

Metabolic dysfunction–associated steatohepatitis, or MASH, will replace the term nonalcoholic steatohepatitis, or NASH.

Mary E. Rinella, MD, of University of Chicago Medicine led the consensus group. The changes were needed, Dr. Rinella and her colleagues argued, because the terms “fatty liver disease” “and nonalcoholic” could be considered to confer stigma, and to better reflect the metabolic dysfunction occurring in the disease. Under the new nomenclature, people with MASLD must have a cardiometabolic risk factor, such as type 2 diabetes. People without metabolic parameters and no known cause will be classed as having cryptogenic SLD.

While the new nomenclature largely conserves existing disease definitions, it allows for alcohol consumption beyond current parameters for nonalcoholic forms of the disease. “There are individuals with risk factors for NAFLD, such as type 2 diabetes, who consume more alcohol than the relatively strict thresholds used to define the nonalcoholic nature of the disease [and] are excluded from trials and consideration for treatments,” the authors wrote.

Moreover, they wrote, “within MetALD there is a continuum where conceptually the condition can be seen to be MASLD or ALD predominant. This may vary over time within a given individual.”

Respondents overwhelmingly agreed, however, that even moderate alcohol use alters the natural history of the disease and that patients with more than minimal alcohol consumption should be analyzed separately in clinical trials.

The new nomenclature reflects a 3-year effort involving some 236 panelists from 56 countries who participated in several rounds of online surveys using a Delphi process. Pediatricians, gastroenterologists, and endocrinologists also participated as well as some patient advocates. Changes were based on a super-majority of opinion (67% or higher), though the consensus on whether the term “fatty” was stigmatizing never reached that threshold. In early rounds of surveys only 44% of respondents considered the word “fatty” to be stigmatizing, while more considered “nonalcoholic” to be problematic.

“Substantial proportions of the respondents deemed terms such as ‘fatty’ stigmatizing, hence its exclusion as part of any new name,” Dr. Rinella and her colleagues wrote. “Although health care professionals may contend that patients have not reported this previously, this likely reflects in part a failure to ask the question in the first place and the power imbalance in the doctor-patient relationship.” The authors noted that the new terminology may help raise awareness at a time when new therapeutics are in sight and it becomes more important to identify at-risk individuals.

Of concern was whether the new definitions would alter the utility of earlier data from registries and trials. However, the authors determined that some 98% of people registered in a European NAFLD cohort would meet the new criteria for MASLD. “Maintenance of the term, and clinical definition, of steatohepatitis ensures retention and validity of prior data from clinical trials and biomarker discovery studies of patients with NASH to be generalizable to individuals classified as MASLD or MASH under the new nomenclature, without impeding the efficiency of research,” they stated.

The effort was spearheaded by three international liver societies: La Asociación Latinoamericana para el Estudio del Hígado, the American Association for the Study of Liver Diseases, and the European Association for the Study of the Liver, as well as the cochairs of the NAFLD Nomenclature Initiative.

Each of the authors disclosed a number of potential conflicts of interest.

TOPLINE:

Irradiating a small number of metastatic lesions does not appear to improve progression-free or overall survival in patients receiving immune checkpoint inhibitor monotherapy for advanced cancer.

METHODOLOGY:

• In the phase 2 CHEERS trial, 52 patients with advanced solid tumors were randomized to anti-PD-1/PD-L1 monotherapy and 47 patients to the same treatment plus stereotactic body radiotherapy (3 x 8 Gy) to a maximum of three lesions before the second or third cycle of an immune checkpoint inhibitor.
• Patients had locally advanced or metastatic melanoma, renal cell carcinoma, urothelial carcinoma, non-small cell lung carcinoma, or head and neck squamous cell carcinoma and were treated at five Belgian hospitals.
• Most patients had more than three lesions.
• Seven patients in the experimental group did not complete radiotherapy because of early progression or intercurrent illness.

TAKEAWAY:

• Over a median follow-up of 12.5 months, median progression-free survival was 4.4 months in the radiotherapy group versus 2.8 months in the control group (hazard ratio, 0.95; P = .82).
• Median overall survival was not significantly better with radiotherapy, compared with the control group (14.3 vs. 11 months; HR, 0.82; P = .47), nor was the objective response rate (27% vs. 22%; P = .56).
• However, a post hoc analysis demonstrated a significant association between the number of irradiated lesions and overall survival among patients receiving radiotherapy (HR, 0.31; P = .002).
• The incidence of grade 3 or worse treatment-related adverse events was 18% in both groups.

IN PRACTICE:

Although the study was negative overall, the post hoc analysis coupled with “recent evidence suggests that treating all active disease sites with higher radiation doses ... may be a more promising strategy to optimize systemic disease control,” the authors concluded.
 

SOURCE:

The study was led by Mathieu Spaas, MD, department of radiation oncology, Ghent (Bellgium) University, and published online in JAMA Oncology.

LIMITATIONS:

• There was insufficient power to detect if certain cancers benefited more from add-on radiation because of the small sample size.
• More than half of patients in the control group had already received some form of radiotherapy before study inclusion, which may mean the study underestimated the benefit of radiotherapy.

DISCLOSURES:

The work was funded by Kom Op Tegen Kanker and Varian Medical Systems.

Investigators disclosed numerous industry ties, including Merck, Novartis, and Bristol Myers Squibb.

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

TOPLINE:

Irradiating a small number of metastatic lesions does not appear to improve progression-free or overall survival in patients receiving immune checkpoint inhibitor monotherapy for advanced cancer.

METHODOLOGY:

• In the phase 2 CHEERS trial, 52 patients with advanced solid tumors were randomized to anti-PD-1/PD-L1 monotherapy and 47 patients to the same treatment plus stereotactic body radiotherapy (3 x 8 Gy) to a maximum of three lesions before the second or third cycle of an immune checkpoint inhibitor.
• Patients had locally advanced or metastatic melanoma, renal cell carcinoma, urothelial carcinoma, non-small cell lung carcinoma, or head and neck squamous cell carcinoma and were treated at five Belgian hospitals.
• Most patients had more than three lesions.
• Seven patients in the experimental group did not complete radiotherapy because of early progression or intercurrent illness.

TAKEAWAY:

• Over a median follow-up of 12.5 months, median progression-free survival was 4.4 months in the radiotherapy group versus 2.8 months in the control group (hazard ratio, 0.95; P = .82).
• Median overall survival was not significantly better with radiotherapy, compared with the control group (14.3 vs. 11 months; HR, 0.82; P = .47), nor was the objective response rate (27% vs. 22%; P = .56).
• However, a post hoc analysis demonstrated a significant association between the number of irradiated lesions and overall survival among patients receiving radiotherapy (HR, 0.31; P = .002).
• The incidence of grade 3 or worse treatment-related adverse events was 18% in both groups.

IN PRACTICE:

Although the study was negative overall, the post hoc analysis coupled with “recent evidence suggests that treating all active disease sites with higher radiation doses ... may be a more promising strategy to optimize systemic disease control,” the authors concluded.
 

SOURCE:

The study was led by Mathieu Spaas, MD, department of radiation oncology, Ghent (Bellgium) University, and published online in JAMA Oncology.

LIMITATIONS:

• There was insufficient power to detect if certain cancers benefited more from add-on radiation because of the small sample size.
• More than half of patients in the control group had already received some form of radiotherapy before study inclusion, which may mean the study underestimated the benefit of radiotherapy.

DISCLOSURES:

The work was funded by Kom Op Tegen Kanker and Varian Medical Systems.

Investigators disclosed numerous industry ties, including Merck, Novartis, and Bristol Myers Squibb.

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

TOPLINE:

Irradiating a small number of metastatic lesions does not appear to improve progression-free or overall survival in patients receiving immune checkpoint inhibitor monotherapy for advanced cancer.

METHODOLOGY:

• In the phase 2 CHEERS trial, 52 patients with advanced solid tumors were randomized to anti-PD-1/PD-L1 monotherapy and 47 patients to the same treatment plus stereotactic body radiotherapy (3 x 8 Gy) to a maximum of three lesions before the second or third cycle of an immune checkpoint inhibitor.
• Patients had locally advanced or metastatic melanoma, renal cell carcinoma, urothelial carcinoma, non-small cell lung carcinoma, or head and neck squamous cell carcinoma and were treated at five Belgian hospitals.
• Most patients had more than three lesions.
• Seven patients in the experimental group did not complete radiotherapy because of early progression or intercurrent illness.

TAKEAWAY:

• Over a median follow-up of 12.5 months, median progression-free survival was 4.4 months in the radiotherapy group versus 2.8 months in the control group (hazard ratio, 0.95; P = .82).
• Median overall survival was not significantly better with radiotherapy, compared with the control group (14.3 vs. 11 months; HR, 0.82; P = .47), nor was the objective response rate (27% vs. 22%; P = .56).
• However, a post hoc analysis demonstrated a significant association between the number of irradiated lesions and overall survival among patients receiving radiotherapy (HR, 0.31; P = .002).
• The incidence of grade 3 or worse treatment-related adverse events was 18% in both groups.

IN PRACTICE:

Although the study was negative overall, the post hoc analysis coupled with “recent evidence suggests that treating all active disease sites with higher radiation doses ... may be a more promising strategy to optimize systemic disease control,” the authors concluded.
 

SOURCE:

The study was led by Mathieu Spaas, MD, department of radiation oncology, Ghent (Bellgium) University, and published online in JAMA Oncology.

LIMITATIONS:

• There was insufficient power to detect if certain cancers benefited more from add-on radiation because of the small sample size.
• More than half of patients in the control group had already received some form of radiotherapy before study inclusion, which may mean the study underestimated the benefit of radiotherapy.

DISCLOSURES:

The work was funded by Kom Op Tegen Kanker and Varian Medical Systems.

Investigators disclosed numerous industry ties, including Merck, Novartis, and Bristol Myers Squibb.

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

Diets containing higher amounts of certain food categories appear to be protective against cardiovascular (CV) disease and premature death, suggests a new study with a broad international scope. Most of the protective food categories are in line with standard dietary guidelines for good health, but one that may be heart-protective is not usually included in such recommendations.

fcafotodigital/Getty Images

The food categories that were found to be protective include fruit, vegetables, nuts, legumes, and fish but also dairy, “mainly whole-fat,” in an analysis based on the international Prospective Urban and Rural Epidemiological (PURE) study and data from five other international trials that encompassed more than 240,000 people.

A healthy diet scoring system was derived from dietary patterns and clinical events observed in the PURE study and was applied to the populations of the other trials. Higher scores, corresponding to greater consumption of the six food categories, tracked with significantly reduced risks for death, myocardial infarction (MI), and stroke.

Reductions in mortality and CV-disease risk that were linked to the higher scores were especially pronounced in lower-income countries in the study published onlinein the European Heart Journal with lead author Andrew Mente, PhD, Population Health Research Institute, McMaster University, Hamilton, Ont.

The study in part refutes the frequent preference for low-fat or no-fat dairy foods over whole-fat dairy in healthy-diet recommendations. But it is consistent with earlier findings from PURE of reduced mortality risk with increased consumption of dietary fat, including saturated fat.

Whereas healthy-diet recommendations tend to emphasize reduced intake of fat, especially saturated fat, the report notes that “there are almost no national or international strategies and policies to increase a number of protective foods,” such as nuts, fish, and dairy.

“Therefore, while the findings from PURE are largely consistent with the nutrition science and modern dietary recommendations to focus on protective foods, the public’s understanding of healthy eating and relevant global policies have not yet caught up to this science,” it states.

“Guidelines and policy actions need to be updated with this newer evidence,” Dr. Mente said in an interview. “For example, the World Health Organization remains mainly focused on reducing certain nutrients, such as fat, saturated fat, added sugar, and salt,” he said. “These recommendations are echoed by government policy actions and industry, as evident by the continued focus on the usual nutrients in food labels of many countries.”

The current findings, Dr. Mente said, “can be used to ensure that the public’s understanding of healthy eating and relevant global policies are able to catch up to the science.”
 

Healthy diet score

PURE investigators developed their healthy diet score using data from 147,642 people from the general population in 21 countries. The investigators compared self-reported dietary intakes with long-term clinical outcomes.

The scoring system assigned a value of 1 for each of the six health-food categories when individuals’ intake exceeded the entire cohort’s median intake. It assigned a 0 when intake was below the median. The total PURE healthy diet score consisted of the sum of the six values, with higher scores corresponding to a healthier diet. The mean score for cohort was 2.95.

There were 15,707 deaths and 40,764 CV events during a median follow-up of 9.3 years. A score of at least 5 points, compared with 0 or 1 point, was associated with significantly reduced hazard ratios for mortality, MI, and stroke in multivariable analysis:

• Mortality: HR, 0.70 (95% CI, 0.63-0.77; P < .0001).
• Major CV disease: HR, 0.82 (95% CI, 0.75-0.91; P < .0001).
• MI: HR, 0.86 (95% CI, 0.75-0.99; P = .0014).
• Stroke: HR, 0.81 (95% CI, 0.71-0.93; P = .0034).

The healthy diet score’s relationship to clinical outcomes was explored in five other large independent studies, including three prospective trials of patients with CV disease that spanned 50 countries, a case-control study with MI patients in 52 countries, and a case-control study with stroke patients in 33 countries.

In the three prospective trials, higher scores were associated with reduced mortality, CV disease events, and MI:

• Mortality: HR, 0.73 (95% CI, 0.66-0.81).
• Major CV disease: HR, 0.79 (95% CI, 0.72-0.87).
• MI: HR, 0.85 (95% CI, 0.71-0.99).

In the two case-control studies, a higher diet score was associated with reduced odds ratios for first MI and for stroke:

• MI: OR, 0.72 (95% CI, 0.65-0.80).
• Stroke: OR, 0.57 (95% CI, 0.50-0.65).

In an analysis based on the PURE cohort, incorporation of unprocessed red meat or whole grains into the health diet score produced similar results, suggesting that a “modest amount” of meat or whole grains can be part of a healthy diet, the authors contend.

The results were similar in a combined analysis of all the prospective studies. In particular, improvement in diet score by one quintile was associated with significantly reduced risks for the following:

• Mortality: HR, 0.92 (95% CI, 0.90-0.93).
• Major CV disease: HR, 0.94 (95% CI, 0.93-0.95).
• MI: HR, 0.94 (95% CI, 0.92-0.96).
• Stroke: HR, 0.94 (95% CI, 0.89-0.99).
• Death or CV disease: HR, 0.93 (95% CI, 0.92-0.94).

“This strongly indicates that the take-home message for patients is the same as for general populations,” Dr. Mente said. “Eat plenty of fruits, vegetables, nuts, legumes, and a moderate amount of fish and whole-fat dairy to lower risk of CV disease and mortality.”

Dairy foods are not widely consumed in some cultures, he said, “but availability and cost are also factors in determining consumption.” Nonetheless, a high-quality diet can be achieved without including or excluding dairy foods. Context-specific policies and priorities are needed for different populations, “rather than a one-size-fits-all global policy.”

Food labels in many countries mainly focus on “reducing certain nutrients as the end-all, be-all,” Dr. Mente observed. “Our findings can be used as a basis for recommendations regarding what a healthy diet should be globally and then modified for each region based on the specific types of foods that are available and affordable in each region.”

Moreover, he said, “targeted food policies are needed to increase the availability and affordability of healthy foods, especially in lower-income countries where intakes are low.”
 

Common human biology

The current results from PURE “confirm prior observations from mostly Western nations that low intakes of fruits, vegetables, nuts, legumes, and fish are major risk factors for poor health,” observes Dariush Mozaffarian, MD, DrPH, MPH, Tufts University, Boston, in an accompanying editorial. “This suggests that common human biology, not merely confounding, explains these observed diet–disease relationships, strengthening causal inference on the power of nutrition.”

Moreover, “These findings provide further support that dairy foods, including whole-fat dairy, can be part of a healthy diet,” Dr. Mozaffarian writes. “The new results in PURE, in combination with prior reports, call for a re-evaluation of unrelenting guidelines to avoid whole-fat dairy products.”

Such studies “remind us of the continuing and devastating rise in diet-related chronic diseases globally, and of the power of protective foods to help address these burdens,” the editorial continues. “It is time for national nutrition guidelines, private sector innovations, government tax policy and agricultural incentives, food procurement policies, labeling and other regulatory priorities, and food-based health care interventions to catch up to the science.”
 

Not automatically superior

“I do not believe guidelines should be changed based on this single study,” contends Howard D. Sesso, ScD, MPH, associate director of the division of preventive medicine at Brigham and Women’s Hospital, Boston, who isn’t part of PURE. “But I welcome the scientific dialog that should come out of any study that challenges what we think we know,” he told this news organization.

“Many other dietary patterns have been identified over the years that also do a great job in predicting disease risk in observational studies,” observed Dr. Sesso. “Is PURE that much better? Maybe, maybe not. But not enough to dismiss other dietary patterns that are already the basis of dietary recommendations in the U.S., Europe, and worldwide.”

The PURE healthy diet score, he said, “appears to work well within the confines of their large pooling of studies around the world, but that doesn’t automatically make it superior to other dietary patterns.” The score “was only modestly, but not greatly, better than existing dietary patterns evaluated.”

Randomized controlled trials are needed, Dr. Sesso said, to “delve into more specific dietary components,” including unprocessed red meat, whole grains, and high-fat dairy foods. And, he said, more observational studies are needed to examine the score’s association with other cardiometabolic outcomes.

The PURE study is funded by the Population Health Research Institute, the Hamilton Health Sciences Research Institute, the Canadian Institutes of Health Research, the Heart and Stroke Foundation of Ontario; with support from Canadian Institutes of Health Research’s Strategy for Patient Oriented Research through the Ontario SPOR Support Unit, as well as the Ontario Ministry of Health and Long-Term Care; and through unrestricted grants from several pharmaceutical companies, with major contributions from AstraZeneca, Sanofi-Aventis, Boehringer Ingelheim, Servier, and GlaxoSmithKline. Additional contributions are from Novartis and King Pharma. Dr. Mente, Dr. Mozaffarian, and Dr. Sesso have disclosed no relevant financial relationships.

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

Diets containing higher amounts of certain food categories appear to be protective against cardiovascular (CV) disease and premature death, suggests a new study with a broad international scope. Most of the protective food categories are in line with standard dietary guidelines for good health, but one that may be heart-protective is not usually included in such recommendations.

fcafotodigital/Getty Images

The food categories that were found to be protective include fruit, vegetables, nuts, legumes, and fish but also dairy, “mainly whole-fat,” in an analysis based on the international Prospective Urban and Rural Epidemiological (PURE) study and data from five other international trials that encompassed more than 240,000 people.

A healthy diet scoring system was derived from dietary patterns and clinical events observed in the PURE study and was applied to the populations of the other trials. Higher scores, corresponding to greater consumption of the six food categories, tracked with significantly reduced risks for death, myocardial infarction (MI), and stroke.

Reductions in mortality and CV-disease risk that were linked to the higher scores were especially pronounced in lower-income countries in the study published onlinein the European Heart Journal with lead author Andrew Mente, PhD, Population Health Research Institute, McMaster University, Hamilton, Ont.

The study in part refutes the frequent preference for low-fat or no-fat dairy foods over whole-fat dairy in healthy-diet recommendations. But it is consistent with earlier findings from PURE of reduced mortality risk with increased consumption of dietary fat, including saturated fat.

Whereas healthy-diet recommendations tend to emphasize reduced intake of fat, especially saturated fat, the report notes that “there are almost no national or international strategies and policies to increase a number of protective foods,” such as nuts, fish, and dairy.

“Therefore, while the findings from PURE are largely consistent with the nutrition science and modern dietary recommendations to focus on protective foods, the public’s understanding of healthy eating and relevant global policies have not yet caught up to this science,” it states.

“Guidelines and policy actions need to be updated with this newer evidence,” Dr. Mente said in an interview. “For example, the World Health Organization remains mainly focused on reducing certain nutrients, such as fat, saturated fat, added sugar, and salt,” he said. “These recommendations are echoed by government policy actions and industry, as evident by the continued focus on the usual nutrients in food labels of many countries.”

The current findings, Dr. Mente said, “can be used to ensure that the public’s understanding of healthy eating and relevant global policies are able to catch up to the science.”
 

Healthy diet score

PURE investigators developed their healthy diet score using data from 147,642 people from the general population in 21 countries. The investigators compared self-reported dietary intakes with long-term clinical outcomes.

The scoring system assigned a value of 1 for each of the six health-food categories when individuals’ intake exceeded the entire cohort’s median intake. It assigned a 0 when intake was below the median. The total PURE healthy diet score consisted of the sum of the six values, with higher scores corresponding to a healthier diet. The mean score for cohort was 2.95.

There were 15,707 deaths and 40,764 CV events during a median follow-up of 9.3 years. A score of at least 5 points, compared with 0 or 1 point, was associated with significantly reduced hazard ratios for mortality, MI, and stroke in multivariable analysis:

• Mortality: HR, 0.70 (95% CI, 0.63-0.77; P < .0001).
• Major CV disease: HR, 0.82 (95% CI, 0.75-0.91; P < .0001).
• MI: HR, 0.86 (95% CI, 0.75-0.99; P = .0014).
• Stroke: HR, 0.81 (95% CI, 0.71-0.93; P = .0034).

The healthy diet score’s relationship to clinical outcomes was explored in five other large independent studies, including three prospective trials of patients with CV disease that spanned 50 countries, a case-control study with MI patients in 52 countries, and a case-control study with stroke patients in 33 countries.

In the three prospective trials, higher scores were associated with reduced mortality, CV disease events, and MI:

• Mortality: HR, 0.73 (95% CI, 0.66-0.81).
• Major CV disease: HR, 0.79 (95% CI, 0.72-0.87).
• MI: HR, 0.85 (95% CI, 0.71-0.99).

In the two case-control studies, a higher diet score was associated with reduced odds ratios for first MI and for stroke:

• MI: OR, 0.72 (95% CI, 0.65-0.80).
• Stroke: OR, 0.57 (95% CI, 0.50-0.65).

In an analysis based on the PURE cohort, incorporation of unprocessed red meat or whole grains into the health diet score produced similar results, suggesting that a “modest amount” of meat or whole grains can be part of a healthy diet, the authors contend.

The results were similar in a combined analysis of all the prospective studies. In particular, improvement in diet score by one quintile was associated with significantly reduced risks for the following:

• Mortality: HR, 0.92 (95% CI, 0.90-0.93).
• Major CV disease: HR, 0.94 (95% CI, 0.93-0.95).
• MI: HR, 0.94 (95% CI, 0.92-0.96).
• Stroke: HR, 0.94 (95% CI, 0.89-0.99).
• Death or CV disease: HR, 0.93 (95% CI, 0.92-0.94).

“This strongly indicates that the take-home message for patients is the same as for general populations,” Dr. Mente said. “Eat plenty of fruits, vegetables, nuts, legumes, and a moderate amount of fish and whole-fat dairy to lower risk of CV disease and mortality.”

Dairy foods are not widely consumed in some cultures, he said, “but availability and cost are also factors in determining consumption.” Nonetheless, a high-quality diet can be achieved without including or excluding dairy foods. Context-specific policies and priorities are needed for different populations, “rather than a one-size-fits-all global policy.”

Food labels in many countries mainly focus on “reducing certain nutrients as the end-all, be-all,” Dr. Mente observed. “Our findings can be used as a basis for recommendations regarding what a healthy diet should be globally and then modified for each region based on the specific types of foods that are available and affordable in each region.”

Moreover, he said, “targeted food policies are needed to increase the availability and affordability of healthy foods, especially in lower-income countries where intakes are low.”
 

Common human biology

The current results from PURE “confirm prior observations from mostly Western nations that low intakes of fruits, vegetables, nuts, legumes, and fish are major risk factors for poor health,” observes Dariush Mozaffarian, MD, DrPH, MPH, Tufts University, Boston, in an accompanying editorial. “This suggests that common human biology, not merely confounding, explains these observed diet–disease relationships, strengthening causal inference on the power of nutrition.”

Moreover, “These findings provide further support that dairy foods, including whole-fat dairy, can be part of a healthy diet,” Dr. Mozaffarian writes. “The new results in PURE, in combination with prior reports, call for a re-evaluation of unrelenting guidelines to avoid whole-fat dairy products.”

Such studies “remind us of the continuing and devastating rise in diet-related chronic diseases globally, and of the power of protective foods to help address these burdens,” the editorial continues. “It is time for national nutrition guidelines, private sector innovations, government tax policy and agricultural incentives, food procurement policies, labeling and other regulatory priorities, and food-based health care interventions to catch up to the science.”
 

Not automatically superior

“I do not believe guidelines should be changed based on this single study,” contends Howard D. Sesso, ScD, MPH, associate director of the division of preventive medicine at Brigham and Women’s Hospital, Boston, who isn’t part of PURE. “But I welcome the scientific dialog that should come out of any study that challenges what we think we know,” he told this news organization.

“Many other dietary patterns have been identified over the years that also do a great job in predicting disease risk in observational studies,” observed Dr. Sesso. “Is PURE that much better? Maybe, maybe not. But not enough to dismiss other dietary patterns that are already the basis of dietary recommendations in the U.S., Europe, and worldwide.”

The PURE healthy diet score, he said, “appears to work well within the confines of their large pooling of studies around the world, but that doesn’t automatically make it superior to other dietary patterns.” The score “was only modestly, but not greatly, better than existing dietary patterns evaluated.”

Randomized controlled trials are needed, Dr. Sesso said, to “delve into more specific dietary components,” including unprocessed red meat, whole grains, and high-fat dairy foods. And, he said, more observational studies are needed to examine the score’s association with other cardiometabolic outcomes.

The PURE study is funded by the Population Health Research Institute, the Hamilton Health Sciences Research Institute, the Canadian Institutes of Health Research, the Heart and Stroke Foundation of Ontario; with support from Canadian Institutes of Health Research’s Strategy for Patient Oriented Research through the Ontario SPOR Support Unit, as well as the Ontario Ministry of Health and Long-Term Care; and through unrestricted grants from several pharmaceutical companies, with major contributions from AstraZeneca, Sanofi-Aventis, Boehringer Ingelheim, Servier, and GlaxoSmithKline. Additional contributions are from Novartis and King Pharma. Dr. Mente, Dr. Mozaffarian, and Dr. Sesso have disclosed no relevant financial relationships.

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

Diets containing higher amounts of certain food categories appear to be protective against cardiovascular (CV) disease and premature death, suggests a new study with a broad international scope. Most of the protective food categories are in line with standard dietary guidelines for good health, but one that may be heart-protective is not usually included in such recommendations.

fcafotodigital/Getty Images

The food categories that were found to be protective include fruit, vegetables, nuts, legumes, and fish but also dairy, “mainly whole-fat,” in an analysis based on the international Prospective Urban and Rural Epidemiological (PURE) study and data from five other international trials that encompassed more than 240,000 people.

A healthy diet scoring system was derived from dietary patterns and clinical events observed in the PURE study and was applied to the populations of the other trials. Higher scores, corresponding to greater consumption of the six food categories, tracked with significantly reduced risks for death, myocardial infarction (MI), and stroke.

Reductions in mortality and CV-disease risk that were linked to the higher scores were especially pronounced in lower-income countries in the study published onlinein the European Heart Journal with lead author Andrew Mente, PhD, Population Health Research Institute, McMaster University, Hamilton, Ont.

The study in part refutes the frequent preference for low-fat or no-fat dairy foods over whole-fat dairy in healthy-diet recommendations. But it is consistent with earlier findings from PURE of reduced mortality risk with increased consumption of dietary fat, including saturated fat.

Whereas healthy-diet recommendations tend to emphasize reduced intake of fat, especially saturated fat, the report notes that “there are almost no national or international strategies and policies to increase a number of protective foods,” such as nuts, fish, and dairy.

“Therefore, while the findings from PURE are largely consistent with the nutrition science and modern dietary recommendations to focus on protective foods, the public’s understanding of healthy eating and relevant global policies have not yet caught up to this science,” it states.

“Guidelines and policy actions need to be updated with this newer evidence,” Dr. Mente said in an interview. “For example, the World Health Organization remains mainly focused on reducing certain nutrients, such as fat, saturated fat, added sugar, and salt,” he said. “These recommendations are echoed by government policy actions and industry, as evident by the continued focus on the usual nutrients in food labels of many countries.”

The current findings, Dr. Mente said, “can be used to ensure that the public’s understanding of healthy eating and relevant global policies are able to catch up to the science.”
 

Healthy diet score

PURE investigators developed their healthy diet score using data from 147,642 people from the general population in 21 countries. The investigators compared self-reported dietary intakes with long-term clinical outcomes.

The scoring system assigned a value of 1 for each of the six health-food categories when individuals’ intake exceeded the entire cohort’s median intake. It assigned a 0 when intake was below the median. The total PURE healthy diet score consisted of the sum of the six values, with higher scores corresponding to a healthier diet. The mean score for cohort was 2.95.

There were 15,707 deaths and 40,764 CV events during a median follow-up of 9.3 years. A score of at least 5 points, compared with 0 or 1 point, was associated with significantly reduced hazard ratios for mortality, MI, and stroke in multivariable analysis:

• Mortality: HR, 0.70 (95% CI, 0.63-0.77; P < .0001).
• Major CV disease: HR, 0.82 (95% CI, 0.75-0.91; P < .0001).
• MI: HR, 0.86 (95% CI, 0.75-0.99; P = .0014).
• Stroke: HR, 0.81 (95% CI, 0.71-0.93; P = .0034).

The healthy diet score’s relationship to clinical outcomes was explored in five other large independent studies, including three prospective trials of patients with CV disease that spanned 50 countries, a case-control study with MI patients in 52 countries, and a case-control study with stroke patients in 33 countries.

In the three prospective trials, higher scores were associated with reduced mortality, CV disease events, and MI:

• Mortality: HR, 0.73 (95% CI, 0.66-0.81).
• Major CV disease: HR, 0.79 (95% CI, 0.72-0.87).
• MI: HR, 0.85 (95% CI, 0.71-0.99).

In the two case-control studies, a higher diet score was associated with reduced odds ratios for first MI and for stroke:

• MI: OR, 0.72 (95% CI, 0.65-0.80).
• Stroke: OR, 0.57 (95% CI, 0.50-0.65).

In an analysis based on the PURE cohort, incorporation of unprocessed red meat or whole grains into the health diet score produced similar results, suggesting that a “modest amount” of meat or whole grains can be part of a healthy diet, the authors contend.

The results were similar in a combined analysis of all the prospective studies. In particular, improvement in diet score by one quintile was associated with significantly reduced risks for the following:

• Mortality: HR, 0.92 (95% CI, 0.90-0.93).
• Major CV disease: HR, 0.94 (95% CI, 0.93-0.95).
• MI: HR, 0.94 (95% CI, 0.92-0.96).
• Stroke: HR, 0.94 (95% CI, 0.89-0.99).
• Death or CV disease: HR, 0.93 (95% CI, 0.92-0.94).

“This strongly indicates that the take-home message for patients is the same as for general populations,” Dr. Mente said. “Eat plenty of fruits, vegetables, nuts, legumes, and a moderate amount of fish and whole-fat dairy to lower risk of CV disease and mortality.”

Dairy foods are not widely consumed in some cultures, he said, “but availability and cost are also factors in determining consumption.” Nonetheless, a high-quality diet can be achieved without including or excluding dairy foods. Context-specific policies and priorities are needed for different populations, “rather than a one-size-fits-all global policy.”

Food labels in many countries mainly focus on “reducing certain nutrients as the end-all, be-all,” Dr. Mente observed. “Our findings can be used as a basis for recommendations regarding what a healthy diet should be globally and then modified for each region based on the specific types of foods that are available and affordable in each region.”

Moreover, he said, “targeted food policies are needed to increase the availability and affordability of healthy foods, especially in lower-income countries where intakes are low.”
 

Common human biology

The current results from PURE “confirm prior observations from mostly Western nations that low intakes of fruits, vegetables, nuts, legumes, and fish are major risk factors for poor health,” observes Dariush Mozaffarian, MD, DrPH, MPH, Tufts University, Boston, in an accompanying editorial. “This suggests that common human biology, not merely confounding, explains these observed diet–disease relationships, strengthening causal inference on the power of nutrition.”

Moreover, “These findings provide further support that dairy foods, including whole-fat dairy, can be part of a healthy diet,” Dr. Mozaffarian writes. “The new results in PURE, in combination with prior reports, call for a re-evaluation of unrelenting guidelines to avoid whole-fat dairy products.”

Such studies “remind us of the continuing and devastating rise in diet-related chronic diseases globally, and of the power of protective foods to help address these burdens,” the editorial continues. “It is time for national nutrition guidelines, private sector innovations, government tax policy and agricultural incentives, food procurement policies, labeling and other regulatory priorities, and food-based health care interventions to catch up to the science.”
 

Not automatically superior

“I do not believe guidelines should be changed based on this single study,” contends Howard D. Sesso, ScD, MPH, associate director of the division of preventive medicine at Brigham and Women’s Hospital, Boston, who isn’t part of PURE. “But I welcome the scientific dialog that should come out of any study that challenges what we think we know,” he told this news organization.

“Many other dietary patterns have been identified over the years that also do a great job in predicting disease risk in observational studies,” observed Dr. Sesso. “Is PURE that much better? Maybe, maybe not. But not enough to dismiss other dietary patterns that are already the basis of dietary recommendations in the U.S., Europe, and worldwide.”

The PURE healthy diet score, he said, “appears to work well within the confines of their large pooling of studies around the world, but that doesn’t automatically make it superior to other dietary patterns.” The score “was only modestly, but not greatly, better than existing dietary patterns evaluated.”

Randomized controlled trials are needed, Dr. Sesso said, to “delve into more specific dietary components,” including unprocessed red meat, whole grains, and high-fat dairy foods. And, he said, more observational studies are needed to examine the score’s association with other cardiometabolic outcomes.

The PURE study is funded by the Population Health Research Institute, the Hamilton Health Sciences Research Institute, the Canadian Institutes of Health Research, the Heart and Stroke Foundation of Ontario; with support from Canadian Institutes of Health Research’s Strategy for Patient Oriented Research through the Ontario SPOR Support Unit, as well as the Ontario Ministry of Health and Long-Term Care; and through unrestricted grants from several pharmaceutical companies, with major contributions from AstraZeneca, Sanofi-Aventis, Boehringer Ingelheim, Servier, and GlaxoSmithKline. Additional contributions are from Novartis and King Pharma. Dr. Mente, Dr. Mozaffarian, and Dr. Sesso have disclosed no relevant financial relationships.

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

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

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

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

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

This transcript has been edited for clarity.

Hi. I’m Art Caplan, PhD. I’m director of the division of medical ethics at the New York University Grossman School of Medicine.

Every once in a while at my school, I get referrals about interesting or difficult clinical cases where doctors would like some input or advice that they can consider in managing a patient. Sometimes those requests come from other hospitals to me. I’ve been doing that kind of ethics consulting, both as a member of various ethics committees and sometimes individually, when, for various reasons, doctors don’t want to go to the Ethics Committee as a first stop.

There was a very interesting case recently involving a young woman I’m going to call Tinslee. She was 17 years old and she suffered, sadly, from recurrent metastatic osteogenic sarcoma. She had bone cancer. It had first been diagnosed at the age of 9. She had received chemotherapy and been under that treatment for a while.

If osteosarcoma is treated before it spreads outside the area where it began, the 5-year survival rate for people like her is about 75%. If the cancer spreads outside of the bones and gets into surrounding tissues, organs, or – worse – into the lymph nodes and starts traveling around, the 5-year survival rate drops to about 60%. The two approaches are chemotherapy and amputation. That’s what we have to offer patients like Tinslee.

Initially, her chemotherapy worked. She went to school and enjoyed sports. She was a real fan of softball and tried to manage the team and be involved. At the time I learned about her, she was planning to go to college. Her love of softball remained, but given the recurrence of the cancer, she had no chance to pursue her athletic interests, not only as a player, but also as a manager or even as a coach for younger players. That was all off the table.

She’d been very compliant up until this time with her chemotherapy. When the recommendation came in that she undergo nonstandard chemotherapy because of the reoccurrence, with experimental drugs using an experimental protocol, she said to her family and the doctors that she didn’t want to do it. She would rather die. She couldn’t take any more chemotherapy and she certainly didn’t want to do it if it was experimental, with the outcomes of this intervention being uncertain.

Her dad said, “She can’t decide. She’s a minor. She’s only 17. I want you to do it – administer this novel form of chemotherapy and try to save her.” Her mother said, “Her input matters. I want to listen to her.” Her mom wasn’t as adamant about doing it or not, but she really felt that Tinslee should be heard loudly because she felt she was mature enough or old enough, even though a minor, to really have a position about what it is to undergo chemotherapy.

Time matters in trying to control the spread, and the doctors were pushing for experimental intervention. I should add, by the way, that although it didn’t really drive the decision about whether to do it or not do it, experimental care like this is not covered by most insurance, and it wasn’t covered by their insurance, so they were facing a big bill if the experimental intervention was administered.

There was some money in a grant to cover some of it, but they were going to face some big financial costs. It never came up in my discussions with the doctors about what to do. I’m not sure whether it ever came up with the family’s discussion with the doctors about what to do, or even whether Tinslee was worrying and didn’t want her family to face a financial burden.

I suggested that we bring the family in. We did some counseling. We had a social worker and we brought in a pastor because these people were fairly religious. We talked about all scenarios, including accepting death, knowing that this disease was not likely to go into remission with the experimental effort; maybe it would, but the doctors were not optimistic.

We tried to talk about how much we should listen to what this young woman wanted. We knew there was the possibility of going to court and having a judge decide this, but in my experience, I do not like going to judges and courts because I know what they’re going to say. They almost always say “administer the intervention.” They don’t want to be in a position of saying don’t do something. They’re a little less willing to do that if something is experimental, but generally speaking, if you’re headed to court, it’s because you’ve decided that you want this to happen.

I felt, in all honesty, that this young woman should have some real respect of her position because the treatment was experimental. She is approaching the age of competency and consent, and she’s been through many interventions. She knows what’s involved. I think you really have to listen hard to what she’s saying.

By the way, after this case, I looked and there have been some surveys of residents in pediatrics. A large number of them said that they hadn’t received any training about what to do when mature minors refuse experimental treatments. The study I saw said that 30% had not undergone any training about this, so we certainly want to introduce that into the appropriate areas of medicine and talk about this with residents and fellows.

Long story short, we had the family meeting, we had another meeting with dad and mom and Tinslee, and the dad began to come around and he began to listen hard. Tinslee said what she wanted was to go to her prom. She wanted to get to her sister’s junior high school softball championship game. If you will, setting some smaller goals that seemed to make her very, very happy began to satisfy mom and dad and they could accept her refusal.

Ultimately, an agreement was reached that she would not undergo the experimental intervention. We agreed on a course of palliative care, recommended that as what the doctors follow, and they decided to do so. Sadly, Tinslee died. She died at home. She did make it to her prom.

I think the outcome, while difficult, sad, tragic, and a close call, was correct. Mature minors who have been through a rough life of interventions and know the price to pay – and for those who have recurrent disease and now face only experimental options – if they say no, that’s something we really have to listen to very hard.



Dr. Kaplan is director, division of medical ethics, New York University Langone Medical Center, New York. He reported a conflict of interest with Johnson & Johnson’s Panel for Compassionate Drug Use.
 

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

Scientists have developed a biodegradable implant that helps chemotherapy drugs penetrate the blood-brain barrier in mice and deliver a direct hit on brain tumors.

It’s the latest advance in a rapidly growing field using ultrasound – high-frequency sound waves undetectable to humans – to fight cancer and other diseases.

The problem addressed by the researchers is the blood-brain barrier, a nearly impenetrable blood vessel lining that keeps harmful molecules from passing into the brain from the blood. But this lining can also block chemo drugs from reaching cancer cells.

So the scientists implanted 1-cm² devices into the skulls of mice, directly behind the tumor site. The implants generate ultrasound waves, loosening the barrier and allowing the drugs to reach the tumor. The sound waves leave healthy tissue undamaged.

“You inject the drug into the body and turn on the ultrasound at the same time. You’re going to hit precisely at the tumor area every single time you use it,” said lead study author Thanh Nguyen, PhD, an associate professor of mechanical engineering at the University of Connecticut, Storrs.

The drug used in the study was paclitaxel, which normally struggles to get through the blood-brain barrier. The tumors shrank, and the mice doubled their lifetime, compared with untreated mice. The mice showed no bad health effects 6 months later. 
 

Breaking through the blood-brain barrier 

The biodegradable implant is made of glycine, an amino acid that’s also strongly piezoelectric, meaning it vibrates when subjected to an electrical current. To make it, researchers cultivated glycine crystals, shattered them into pieces, and finally used a process called electrospinning, which applies a high electrical voltage to the nanocrystals. 

Voltage flows to the implant via an external device. The resulting ultrasound causes the tightly adhered cells of the blood-brain barrier to vibrate, stretching them out and creating space for pores to form. 

“That allows in very tiny particles, including chemo drugs,” said Dr. Nguyen. 

His earlier biodegradable implant broke apart from the force, but the new glycine implant is more flexible, stable, and highly piezoelectric. It could be implanted after a patient has surgery to remove a brain tumor, to continue treating residual cancer cells. The implant dissolves harmlessly in the body over time, and doctors can control its lifespan. 
 

A new wave of uses for ultrasound 

Dr. Nguyen’s study builds on similar efforts, including a recent clinical trial of a nonbiodegradable implant for treating brain tumors. Ultrasound can focus energy on precise targets in the body.

It’s like “using a magnifying glass to focus multiple beams of light on a point and burn a hole in a leaf,” said Neal Kassell, MD, founder and chairman of the Focused Ultrasound Foundation. This approach spares adjacent normal tissue.  

Doctors now understand more than 30 ways that ultrasound interacts with tissue – from destroying abnormal tissue to delivering drugs more effectively to stimulating an immune response. A decade ago, only five such interactions were known.

This opens the door for treating “a wide spectrum of medical disorders,” from neurodegenerative diseases like Alzheimer’s and Parkinson’s to difficult-to-treat cancers of the prostate and pancreas, and even addiction, said Dr. Kassell. 

Dr. Kassell envisions using focused ultrasound to treat brain tumors as an alternative (or complement) to surgery, chemotherapy, immunotherapy, or radiation therapy. In the meantime, implants have helped show “the effectiveness of opening the blood-brain barrier.”

Dr. Nguyen’s team plans on testing the safety and efficacy of their implant in pigs next. Eventually, Dr. Nguyen hopes to develop a patch with an array of implants to target different areas of the brain. 

One study coauthor is cofounder of PiezoBioMembrane and SingleTimeMicroneedles. The other study authors reported no conflicts of interest.

A version of this article originally appeared on WebMD.com.

Scientists have developed a biodegradable implant that helps chemotherapy drugs penetrate the blood-brain barrier in mice and deliver a direct hit on brain tumors.

It’s the latest advance in a rapidly growing field using ultrasound – high-frequency sound waves undetectable to humans – to fight cancer and other diseases.

The problem addressed by the researchers is the blood-brain barrier, a nearly impenetrable blood vessel lining that keeps harmful molecules from passing into the brain from the blood. But this lining can also block chemo drugs from reaching cancer cells.

So the scientists implanted 1-cm² devices into the skulls of mice, directly behind the tumor site. The implants generate ultrasound waves, loosening the barrier and allowing the drugs to reach the tumor. The sound waves leave healthy tissue undamaged.

“You inject the drug into the body and turn on the ultrasound at the same time. You’re going to hit precisely at the tumor area every single time you use it,” said lead study author Thanh Nguyen, PhD, an associate professor of mechanical engineering at the University of Connecticut, Storrs.

The drug used in the study was paclitaxel, which normally struggles to get through the blood-brain barrier. The tumors shrank, and the mice doubled their lifetime, compared with untreated mice. The mice showed no bad health effects 6 months later. 
 

Breaking through the blood-brain barrier 

The biodegradable implant is made of glycine, an amino acid that’s also strongly piezoelectric, meaning it vibrates when subjected to an electrical current. To make it, researchers cultivated glycine crystals, shattered them into pieces, and finally used a process called electrospinning, which applies a high electrical voltage to the nanocrystals. 

Voltage flows to the implant via an external device. The resulting ultrasound causes the tightly adhered cells of the blood-brain barrier to vibrate, stretching them out and creating space for pores to form. 

“That allows in very tiny particles, including chemo drugs,” said Dr. Nguyen. 

His earlier biodegradable implant broke apart from the force, but the new glycine implant is more flexible, stable, and highly piezoelectric. It could be implanted after a patient has surgery to remove a brain tumor, to continue treating residual cancer cells. The implant dissolves harmlessly in the body over time, and doctors can control its lifespan. 
 

A new wave of uses for ultrasound 

Dr. Nguyen’s study builds on similar efforts, including a recent clinical trial of a nonbiodegradable implant for treating brain tumors. Ultrasound can focus energy on precise targets in the body.

It’s like “using a magnifying glass to focus multiple beams of light on a point and burn a hole in a leaf,” said Neal Kassell, MD, founder and chairman of the Focused Ultrasound Foundation. This approach spares adjacent normal tissue.  

Doctors now understand more than 30 ways that ultrasound interacts with tissue – from destroying abnormal tissue to delivering drugs more effectively to stimulating an immune response. A decade ago, only five such interactions were known.

This opens the door for treating “a wide spectrum of medical disorders,” from neurodegenerative diseases like Alzheimer’s and Parkinson’s to difficult-to-treat cancers of the prostate and pancreas, and even addiction, said Dr. Kassell. 

Dr. Kassell envisions using focused ultrasound to treat brain tumors as an alternative (or complement) to surgery, chemotherapy, immunotherapy, or radiation therapy. In the meantime, implants have helped show “the effectiveness of opening the blood-brain barrier.”

Dr. Nguyen’s team plans on testing the safety and efficacy of their implant in pigs next. Eventually, Dr. Nguyen hopes to develop a patch with an array of implants to target different areas of the brain. 

One study coauthor is cofounder of PiezoBioMembrane and SingleTimeMicroneedles. The other study authors reported no conflicts of interest.

A version of this article originally appeared on WebMD.com.

Scientists have developed a biodegradable implant that helps chemotherapy drugs penetrate the blood-brain barrier in mice and deliver a direct hit on brain tumors.

It’s the latest advance in a rapidly growing field using ultrasound – high-frequency sound waves undetectable to humans – to fight cancer and other diseases.

The problem addressed by the researchers is the blood-brain barrier, a nearly impenetrable blood vessel lining that keeps harmful molecules from passing into the brain from the blood. But this lining can also block chemo drugs from reaching cancer cells.

So the scientists implanted 1-cm² devices into the skulls of mice, directly behind the tumor site. The implants generate ultrasound waves, loosening the barrier and allowing the drugs to reach the tumor. The sound waves leave healthy tissue undamaged.

“You inject the drug into the body and turn on the ultrasound at the same time. You’re going to hit precisely at the tumor area every single time you use it,” said lead study author Thanh Nguyen, PhD, an associate professor of mechanical engineering at the University of Connecticut, Storrs.

The drug used in the study was paclitaxel, which normally struggles to get through the blood-brain barrier. The tumors shrank, and the mice doubled their lifetime, compared with untreated mice. The mice showed no bad health effects 6 months later. 
 

Breaking through the blood-brain barrier 

The biodegradable implant is made of glycine, an amino acid that’s also strongly piezoelectric, meaning it vibrates when subjected to an electrical current. To make it, researchers cultivated glycine crystals, shattered them into pieces, and finally used a process called electrospinning, which applies a high electrical voltage to the nanocrystals. 

Voltage flows to the implant via an external device. The resulting ultrasound causes the tightly adhered cells of the blood-brain barrier to vibrate, stretching them out and creating space for pores to form. 

“That allows in very tiny particles, including chemo drugs,” said Dr. Nguyen. 

His earlier biodegradable implant broke apart from the force, but the new glycine implant is more flexible, stable, and highly piezoelectric. It could be implanted after a patient has surgery to remove a brain tumor, to continue treating residual cancer cells. The implant dissolves harmlessly in the body over time, and doctors can control its lifespan. 
 

A new wave of uses for ultrasound 

Dr. Nguyen’s study builds on similar efforts, including a recent clinical trial of a nonbiodegradable implant for treating brain tumors. Ultrasound can focus energy on precise targets in the body.

It’s like “using a magnifying glass to focus multiple beams of light on a point and burn a hole in a leaf,” said Neal Kassell, MD, founder and chairman of the Focused Ultrasound Foundation. This approach spares adjacent normal tissue.  

Doctors now understand more than 30 ways that ultrasound interacts with tissue – from destroying abnormal tissue to delivering drugs more effectively to stimulating an immune response. A decade ago, only five such interactions were known.

This opens the door for treating “a wide spectrum of medical disorders,” from neurodegenerative diseases like Alzheimer’s and Parkinson’s to difficult-to-treat cancers of the prostate and pancreas, and even addiction, said Dr. Kassell. 

Dr. Kassell envisions using focused ultrasound to treat brain tumors as an alternative (or complement) to surgery, chemotherapy, immunotherapy, or radiation therapy. In the meantime, implants have helped show “the effectiveness of opening the blood-brain barrier.”

Dr. Nguyen’s team plans on testing the safety and efficacy of their implant in pigs next. Eventually, Dr. Nguyen hopes to develop a patch with an array of implants to target different areas of the brain. 

One study coauthor is cofounder of PiezoBioMembrane and SingleTimeMicroneedles. The other study authors reported no conflicts of interest.

A version of this article originally appeared on WebMD.com.