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Bariatric surgery shows metabolic benefits in lower-BMI patients
LAS VEGAS – It’s time to take bariatric out of bariatric surgery.
“The way forward is to not call it bariatric surgery or weight-loss surgery but surgery to treat diabetes, hypertension, hyperlipidemia, and other metabolic diseases,” said Oliver A. Varban, MD, at a meeting presented by the Obesity Society and the American Society for Metabolic and Bariatric Surgery. “We need to reframe the conversation with patients about what success [with bariatric surgery] looks like. Weight loss can be a side effect of the operation if patients have surgery to resolve their diabetes. It’s not about BMI; it’s about treating metabolic disease.”
Dr. Varban, a bariatric surgeon at the University of Michigan in Ann Arbor, reported data showing that bariatric surgery with sleeve gastrectomy in patients with baseline body mass index (BMI) levels below 35 kg/m2 was as effective at normalizing a range of metabolically associated disorders as it was in more obese patients in an observational study of more than 45,000 patients who underwent surgery in Michigan.
The findings add to an already extensive pool of evidence for loosening current guidelines that restrict bariatric surgery to patients with a BMI of 35 kg/m2 or greater, Dr. Varban said. But an influential bariatric surgery consensus statement from the National Institutes of Health that dates from 1991 and remains in place, recommends this surgery only for people with a BMI of at least 35 kg/m2, and this guidance often limits access to the surgery for patients at lower BMI, he noted.
A more inclusive assessment of patients as potential candidates for bariatric surgery should include a range of considerations in addition to weight and height, he explained in an interview. “Even if people have a BMI of less than 30 kg/m2 but have, or are at high risk for developing, metabolic disease, they should also be offered the operation.”
The guidance from the NIH results in a U.S. bariatric surgery population that effectively centers mainly on women with a BMI of 40 kg/m2 or greater and makes procedures like sleeve gastrectomy unavailable to many other types of patients who could benefit from it, Dr. Varban said. In 2018, the American Society for Metabolic and Bariatric Surgery released a position statement that summarized the evidence for the safety and efficacy of bariatric surgery in people with a BMI of 30-34 kg/m2, and cited the lingering and restrictive impact of the 1991 NIH consensus statement.
The study run by Dr. Varban and his associates used data collected by 43 programs in the Michigan Bariatric Surgery Collaborative during 2006-2018 that included 1,073 patients who had a BMI of less than 35 kg/m2 on the day they underwent sleeve gastrectomy, and 44,511 patients who had the same procedure and had a BMI of at least 35 kg/m2. The operations were performed by any one of 81 surgeons who worked at the centers during this time.
The patients with lower BMIs were older, with an average age of 51 years, compared with 45 years in the higher-BMI group, and they had higher prevalences of certain metabolic disorders. Diabetes affected 37% of those in the lower-BMI group and 31% of those with higher BMIs; hyperlipidemia affected 57% and 45%, respectively; and gastroesophageal reflux disease affected 56% and 49%, respectively. Obstructive sleep apnea was more common in the group with higher BMIs, at 47%, compared with 41% of those with lower BMIs.
The average BMI in the lower group was 33.7 kg/m2; in the higher group it was 46.7 kg/m2. Dr. Varban did not have data on whether any patients in the lower-BMI group had a BMI below 30 kg/m2. Roughly a third of the patients in the lower-BMI group had a BMI of less than 35 kg/m2 at the time of their initial examination, whereas the other two-thirds had a BMI that low only on the day of their surgery.At follow-up 1 year after their surgery, patients who started with lower BMIs had, in general, a very similar pattern of responses as those who started with higher BMIs, with rates of discontinuation of treatments for diabetes, hypertension, hyperlipidemia, obstructive sleep apnea, and gastroesophageal reflux of about 50%-80% and similar in both treatment arms. For example, discontinuation of oral diabetes drugs occurred in 79% and 78% of those with low and high BMIs, respectively, and discontinuation of hypertension medications occurred in 60% and 54%, respectively. Although the average absolute weight loss in the patients with lower BMIs was nearly half that of patients with higher starting BMIs, a much greater percentage of patients in the lower-BMI group achieved a BMI of less than 25 kg/m2, compared with the higher-BMI group (36% vs. 6%, respectively).
Patients from the lower-BMI group also showed high levels of satisfaction with their surgery and its results after 1 year. Questionnaire results from roughly half the patients in each treatment group showed that 90% were very satisfied in the lower-BMI group, compared with 84% of those who began with higher BMIs, with a dissatisfaction rate of 1% and 2%, respectively. The average body-image score at 1 year follow-up was significantly higher in those who started with lower BMIs. The rate of complications was low and similar in the two groups, with a 6% rate in the lower-BMI group and 5% in those with higher BMIs.
The study received no commercial funding. Dr. Varban receives salary support from Blue Cross Blue Shield of Michigan.
SOURCE: Varban et al. Obesity Week 2019, Abstract A105.
This article was updated 11/8/2020.
This is a very important topic and study, and its findings are very positive and reinforcing for more liberal use of bariatric surgery. Results from several prior studies had documented the safety and efficacy of bariatric surgery in patients with lower body mass index, and its fantastic to now have additional data that show the same outcomes. A major challenge is making patients and more physicians aware of the range of comorbidities that can be effectively managed with bariatric surgery, even in patients with lower body mass index.
Mona Misra, MD, is associate director of the Bariatric Program at Cedars-Sinai Marina Del Rey Hospital in Los Angeles. She had no relevant disclosures. She made these comments as designated discussant for the study.
This is a very important topic and study, and its findings are very positive and reinforcing for more liberal use of bariatric surgery. Results from several prior studies had documented the safety and efficacy of bariatric surgery in patients with lower body mass index, and its fantastic to now have additional data that show the same outcomes. A major challenge is making patients and more physicians aware of the range of comorbidities that can be effectively managed with bariatric surgery, even in patients with lower body mass index.
Mona Misra, MD, is associate director of the Bariatric Program at Cedars-Sinai Marina Del Rey Hospital in Los Angeles. She had no relevant disclosures. She made these comments as designated discussant for the study.
This is a very important topic and study, and its findings are very positive and reinforcing for more liberal use of bariatric surgery. Results from several prior studies had documented the safety and efficacy of bariatric surgery in patients with lower body mass index, and its fantastic to now have additional data that show the same outcomes. A major challenge is making patients and more physicians aware of the range of comorbidities that can be effectively managed with bariatric surgery, even in patients with lower body mass index.
Mona Misra, MD, is associate director of the Bariatric Program at Cedars-Sinai Marina Del Rey Hospital in Los Angeles. She had no relevant disclosures. She made these comments as designated discussant for the study.
LAS VEGAS – It’s time to take bariatric out of bariatric surgery.
“The way forward is to not call it bariatric surgery or weight-loss surgery but surgery to treat diabetes, hypertension, hyperlipidemia, and other metabolic diseases,” said Oliver A. Varban, MD, at a meeting presented by the Obesity Society and the American Society for Metabolic and Bariatric Surgery. “We need to reframe the conversation with patients about what success [with bariatric surgery] looks like. Weight loss can be a side effect of the operation if patients have surgery to resolve their diabetes. It’s not about BMI; it’s about treating metabolic disease.”
Dr. Varban, a bariatric surgeon at the University of Michigan in Ann Arbor, reported data showing that bariatric surgery with sleeve gastrectomy in patients with baseline body mass index (BMI) levels below 35 kg/m2 was as effective at normalizing a range of metabolically associated disorders as it was in more obese patients in an observational study of more than 45,000 patients who underwent surgery in Michigan.
The findings add to an already extensive pool of evidence for loosening current guidelines that restrict bariatric surgery to patients with a BMI of 35 kg/m2 or greater, Dr. Varban said. But an influential bariatric surgery consensus statement from the National Institutes of Health that dates from 1991 and remains in place, recommends this surgery only for people with a BMI of at least 35 kg/m2, and this guidance often limits access to the surgery for patients at lower BMI, he noted.
A more inclusive assessment of patients as potential candidates for bariatric surgery should include a range of considerations in addition to weight and height, he explained in an interview. “Even if people have a BMI of less than 30 kg/m2 but have, or are at high risk for developing, metabolic disease, they should also be offered the operation.”
The guidance from the NIH results in a U.S. bariatric surgery population that effectively centers mainly on women with a BMI of 40 kg/m2 or greater and makes procedures like sleeve gastrectomy unavailable to many other types of patients who could benefit from it, Dr. Varban said. In 2018, the American Society for Metabolic and Bariatric Surgery released a position statement that summarized the evidence for the safety and efficacy of bariatric surgery in people with a BMI of 30-34 kg/m2, and cited the lingering and restrictive impact of the 1991 NIH consensus statement.
The study run by Dr. Varban and his associates used data collected by 43 programs in the Michigan Bariatric Surgery Collaborative during 2006-2018 that included 1,073 patients who had a BMI of less than 35 kg/m2 on the day they underwent sleeve gastrectomy, and 44,511 patients who had the same procedure and had a BMI of at least 35 kg/m2. The operations were performed by any one of 81 surgeons who worked at the centers during this time.
The patients with lower BMIs were older, with an average age of 51 years, compared with 45 years in the higher-BMI group, and they had higher prevalences of certain metabolic disorders. Diabetes affected 37% of those in the lower-BMI group and 31% of those with higher BMIs; hyperlipidemia affected 57% and 45%, respectively; and gastroesophageal reflux disease affected 56% and 49%, respectively. Obstructive sleep apnea was more common in the group with higher BMIs, at 47%, compared with 41% of those with lower BMIs.
The average BMI in the lower group was 33.7 kg/m2; in the higher group it was 46.7 kg/m2. Dr. Varban did not have data on whether any patients in the lower-BMI group had a BMI below 30 kg/m2. Roughly a third of the patients in the lower-BMI group had a BMI of less than 35 kg/m2 at the time of their initial examination, whereas the other two-thirds had a BMI that low only on the day of their surgery.At follow-up 1 year after their surgery, patients who started with lower BMIs had, in general, a very similar pattern of responses as those who started with higher BMIs, with rates of discontinuation of treatments for diabetes, hypertension, hyperlipidemia, obstructive sleep apnea, and gastroesophageal reflux of about 50%-80% and similar in both treatment arms. For example, discontinuation of oral diabetes drugs occurred in 79% and 78% of those with low and high BMIs, respectively, and discontinuation of hypertension medications occurred in 60% and 54%, respectively. Although the average absolute weight loss in the patients with lower BMIs was nearly half that of patients with higher starting BMIs, a much greater percentage of patients in the lower-BMI group achieved a BMI of less than 25 kg/m2, compared with the higher-BMI group (36% vs. 6%, respectively).
Patients from the lower-BMI group also showed high levels of satisfaction with their surgery and its results after 1 year. Questionnaire results from roughly half the patients in each treatment group showed that 90% were very satisfied in the lower-BMI group, compared with 84% of those who began with higher BMIs, with a dissatisfaction rate of 1% and 2%, respectively. The average body-image score at 1 year follow-up was significantly higher in those who started with lower BMIs. The rate of complications was low and similar in the two groups, with a 6% rate in the lower-BMI group and 5% in those with higher BMIs.
The study received no commercial funding. Dr. Varban receives salary support from Blue Cross Blue Shield of Michigan.
SOURCE: Varban et al. Obesity Week 2019, Abstract A105.
This article was updated 11/8/2020.
LAS VEGAS – It’s time to take bariatric out of bariatric surgery.
“The way forward is to not call it bariatric surgery or weight-loss surgery but surgery to treat diabetes, hypertension, hyperlipidemia, and other metabolic diseases,” said Oliver A. Varban, MD, at a meeting presented by the Obesity Society and the American Society for Metabolic and Bariatric Surgery. “We need to reframe the conversation with patients about what success [with bariatric surgery] looks like. Weight loss can be a side effect of the operation if patients have surgery to resolve their diabetes. It’s not about BMI; it’s about treating metabolic disease.”
Dr. Varban, a bariatric surgeon at the University of Michigan in Ann Arbor, reported data showing that bariatric surgery with sleeve gastrectomy in patients with baseline body mass index (BMI) levels below 35 kg/m2 was as effective at normalizing a range of metabolically associated disorders as it was in more obese patients in an observational study of more than 45,000 patients who underwent surgery in Michigan.
The findings add to an already extensive pool of evidence for loosening current guidelines that restrict bariatric surgery to patients with a BMI of 35 kg/m2 or greater, Dr. Varban said. But an influential bariatric surgery consensus statement from the National Institutes of Health that dates from 1991 and remains in place, recommends this surgery only for people with a BMI of at least 35 kg/m2, and this guidance often limits access to the surgery for patients at lower BMI, he noted.
A more inclusive assessment of patients as potential candidates for bariatric surgery should include a range of considerations in addition to weight and height, he explained in an interview. “Even if people have a BMI of less than 30 kg/m2 but have, or are at high risk for developing, metabolic disease, they should also be offered the operation.”
The guidance from the NIH results in a U.S. bariatric surgery population that effectively centers mainly on women with a BMI of 40 kg/m2 or greater and makes procedures like sleeve gastrectomy unavailable to many other types of patients who could benefit from it, Dr. Varban said. In 2018, the American Society for Metabolic and Bariatric Surgery released a position statement that summarized the evidence for the safety and efficacy of bariatric surgery in people with a BMI of 30-34 kg/m2, and cited the lingering and restrictive impact of the 1991 NIH consensus statement.
The study run by Dr. Varban and his associates used data collected by 43 programs in the Michigan Bariatric Surgery Collaborative during 2006-2018 that included 1,073 patients who had a BMI of less than 35 kg/m2 on the day they underwent sleeve gastrectomy, and 44,511 patients who had the same procedure and had a BMI of at least 35 kg/m2. The operations were performed by any one of 81 surgeons who worked at the centers during this time.
The patients with lower BMIs were older, with an average age of 51 years, compared with 45 years in the higher-BMI group, and they had higher prevalences of certain metabolic disorders. Diabetes affected 37% of those in the lower-BMI group and 31% of those with higher BMIs; hyperlipidemia affected 57% and 45%, respectively; and gastroesophageal reflux disease affected 56% and 49%, respectively. Obstructive sleep apnea was more common in the group with higher BMIs, at 47%, compared with 41% of those with lower BMIs.
The average BMI in the lower group was 33.7 kg/m2; in the higher group it was 46.7 kg/m2. Dr. Varban did not have data on whether any patients in the lower-BMI group had a BMI below 30 kg/m2. Roughly a third of the patients in the lower-BMI group had a BMI of less than 35 kg/m2 at the time of their initial examination, whereas the other two-thirds had a BMI that low only on the day of their surgery.At follow-up 1 year after their surgery, patients who started with lower BMIs had, in general, a very similar pattern of responses as those who started with higher BMIs, with rates of discontinuation of treatments for diabetes, hypertension, hyperlipidemia, obstructive sleep apnea, and gastroesophageal reflux of about 50%-80% and similar in both treatment arms. For example, discontinuation of oral diabetes drugs occurred in 79% and 78% of those with low and high BMIs, respectively, and discontinuation of hypertension medications occurred in 60% and 54%, respectively. Although the average absolute weight loss in the patients with lower BMIs was nearly half that of patients with higher starting BMIs, a much greater percentage of patients in the lower-BMI group achieved a BMI of less than 25 kg/m2, compared with the higher-BMI group (36% vs. 6%, respectively).
Patients from the lower-BMI group also showed high levels of satisfaction with their surgery and its results after 1 year. Questionnaire results from roughly half the patients in each treatment group showed that 90% were very satisfied in the lower-BMI group, compared with 84% of those who began with higher BMIs, with a dissatisfaction rate of 1% and 2%, respectively. The average body-image score at 1 year follow-up was significantly higher in those who started with lower BMIs. The rate of complications was low and similar in the two groups, with a 6% rate in the lower-BMI group and 5% in those with higher BMIs.
The study received no commercial funding. Dr. Varban receives salary support from Blue Cross Blue Shield of Michigan.
SOURCE: Varban et al. Obesity Week 2019, Abstract A105.
This article was updated 11/8/2020.
REPORTING FROM OBESITY WEEK 2019
Key clinical point:
Major finding: Discontinuation of hypertension drugs occurred in 60% of patients with lower BMIs at baseline and 54% of those with higher BMIs.
Study details: Review of prospectively collected data from 45,584 patients who underwent sleeve gastrectomy in Michigan during 2006-2018.
Disclosures: The study received no commercial funding. Dr. Varban receives salary support from Blue Cross Blue Shield of Michigan. Dr. Misra had no relevant disclosures.
Source: Varban OA et al. Obesity Week 2019, Abstract A105.
Ataluren shows real-world benefit for nonsense mutation Duchenne muscular dystrophy
AUSTIN, TEX. – , according to new data.
“Participants in the STRIDE Registry [real-world patients] showed a reduction in functional decline over 48 weeks, compared with patients receiving placebo” in the trial, reported Abdallah Delage of PTC Therapeutics in Zug, Switzerland, and his associates.
Duchenne muscular dystrophy affects an estimated 1 in 3,600-6,000 male births globally, about 10%-15% of whom have nonsense mutation DMD. This mutation causes a truncated, nonfunctional dystrophin protein due to a premature stop codon, the authors explained. Ataluren “promotes ribosomal read-through of the premature stop codon to produce a full-length dystrophin protein,” they explained.
Ataluren is currently approved for ambulatory patients age 2 and older with nonsense mutation DMD in the European Union and several other European countries. Israel, Korea, Chile, and Ukraine have approved it for patients aged 5 and older.
The Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry contains real-world data from patients using ataluren as part of an ongoing multicenter observational postapproval safety study. The investigators are tracking patients for at least 5 years after enrollment in 14 countries where ataluren is approved or commercially available through early-access programs. Patients take 40 mg/kg daily: 10 mg/kg in the morning, 10 mg/kg midday, and 20 mg/kg in the evening.
The researchers compared outcomes in 216 patients in the STRIDE Registry with participants in a randomized controlled phase 3 study of ataluren involving 228 boys, aged 7-16, who received ataluren (n = 114) or placebo (n = 114) for 48 weeks. Patients were an average 9 years old in STRIDE and in both arms of the randomized controlled trial.
The STRIDE Registry participants, comprising 184 ambulatory and 26 nonambulatory patients at enrollment, had at least 48 weeks between their first and last assessment. All of the patients in the phase 3 study and 88.6% of the STRIDE Registry patients were receiving corticosteroids along with ataluren. The researchers compared the 184 ambulatory STRIDE participants with the participants of the randomized controlled trial for one primary and four secondary endpoints from baseline to 48 weeks.
For the primary endpoint, 6-minute walk distance, average distance was 35 meters shorter than baseline in STRIDE Registry participants (n = 66), 42.2 meters shorter in the patients receiving ataluren in the phase 3 study (n = 109), and 57.6 meters shorter in RCT patients receiving placebo in the phase 3 trial (n = 109).
A secondary endpoint, the time it took patients to walk or run 10 meters, increased 1.6 seconds from baseline to 48 weeks in STRIDE Registry participants (n = 61), 2.3 seconds in participants receiving ataluren in the phase 3 trial (n = 109), and 3.5 seconds in study participants receiving placebo (n = 110).
Another secondary endpoint, the change in time it took for patients to stand from supine position from baseline to 48 weeks, was 2.9 additional seconds for STRIDE participants (n = 55), 3.8 additional seconds in study participants receiving ataluren (n = 101), and 3.9 additional seconds in study participants receiving placebo (n = 96).
Two final secondary endpoints were the changes in time to climb four stairs and to descend four stairs from baseline to 48 weeks. STRIDE participants (n = 47) climbed four stairs 1.2 seconds more slowly at 48 weeks, compared with 2.7 seconds more slowly in the participants who received ataluren in the phase 3 trial (n = 105) and 4.5 seconds more slowly in those who received placebo. Descending four stairs took 0.5 more seconds at 48 weeks in STRIDE participants (n = 40), 2.2 more seconds in participants who received ataluren in the phase 3 trial (n = 106), and 4.0 more seconds in those who received placebo (n = 100).
At least one adverse event occurred in 20.7% of registry participants; seven of these were considered treatment related. Treatment-related side effects included abdominal pain, vomiting, headache, stomach ache, diarrhea, and increased serum lipids.
The study and STRIDE Registry is funded by PTC Therapeutics with TREAT-NMD and the Cooperative International Neuromuscular Research Group. Mr. Delage and five other authors are employees of PTC Therapeutics, and six authors had received speaker or consultancy fees or served on the advisory board of a variety of companies.
SOURCE: Delage A et al. AANEM 2019, Abstract 115.
AUSTIN, TEX. – , according to new data.
“Participants in the STRIDE Registry [real-world patients] showed a reduction in functional decline over 48 weeks, compared with patients receiving placebo” in the trial, reported Abdallah Delage of PTC Therapeutics in Zug, Switzerland, and his associates.
Duchenne muscular dystrophy affects an estimated 1 in 3,600-6,000 male births globally, about 10%-15% of whom have nonsense mutation DMD. This mutation causes a truncated, nonfunctional dystrophin protein due to a premature stop codon, the authors explained. Ataluren “promotes ribosomal read-through of the premature stop codon to produce a full-length dystrophin protein,” they explained.
Ataluren is currently approved for ambulatory patients age 2 and older with nonsense mutation DMD in the European Union and several other European countries. Israel, Korea, Chile, and Ukraine have approved it for patients aged 5 and older.
The Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry contains real-world data from patients using ataluren as part of an ongoing multicenter observational postapproval safety study. The investigators are tracking patients for at least 5 years after enrollment in 14 countries where ataluren is approved or commercially available through early-access programs. Patients take 40 mg/kg daily: 10 mg/kg in the morning, 10 mg/kg midday, and 20 mg/kg in the evening.
The researchers compared outcomes in 216 patients in the STRIDE Registry with participants in a randomized controlled phase 3 study of ataluren involving 228 boys, aged 7-16, who received ataluren (n = 114) or placebo (n = 114) for 48 weeks. Patients were an average 9 years old in STRIDE and in both arms of the randomized controlled trial.
The STRIDE Registry participants, comprising 184 ambulatory and 26 nonambulatory patients at enrollment, had at least 48 weeks between their first and last assessment. All of the patients in the phase 3 study and 88.6% of the STRIDE Registry patients were receiving corticosteroids along with ataluren. The researchers compared the 184 ambulatory STRIDE participants with the participants of the randomized controlled trial for one primary and four secondary endpoints from baseline to 48 weeks.
For the primary endpoint, 6-minute walk distance, average distance was 35 meters shorter than baseline in STRIDE Registry participants (n = 66), 42.2 meters shorter in the patients receiving ataluren in the phase 3 study (n = 109), and 57.6 meters shorter in RCT patients receiving placebo in the phase 3 trial (n = 109).
A secondary endpoint, the time it took patients to walk or run 10 meters, increased 1.6 seconds from baseline to 48 weeks in STRIDE Registry participants (n = 61), 2.3 seconds in participants receiving ataluren in the phase 3 trial (n = 109), and 3.5 seconds in study participants receiving placebo (n = 110).
Another secondary endpoint, the change in time it took for patients to stand from supine position from baseline to 48 weeks, was 2.9 additional seconds for STRIDE participants (n = 55), 3.8 additional seconds in study participants receiving ataluren (n = 101), and 3.9 additional seconds in study participants receiving placebo (n = 96).
Two final secondary endpoints were the changes in time to climb four stairs and to descend four stairs from baseline to 48 weeks. STRIDE participants (n = 47) climbed four stairs 1.2 seconds more slowly at 48 weeks, compared with 2.7 seconds more slowly in the participants who received ataluren in the phase 3 trial (n = 105) and 4.5 seconds more slowly in those who received placebo. Descending four stairs took 0.5 more seconds at 48 weeks in STRIDE participants (n = 40), 2.2 more seconds in participants who received ataluren in the phase 3 trial (n = 106), and 4.0 more seconds in those who received placebo (n = 100).
At least one adverse event occurred in 20.7% of registry participants; seven of these were considered treatment related. Treatment-related side effects included abdominal pain, vomiting, headache, stomach ache, diarrhea, and increased serum lipids.
The study and STRIDE Registry is funded by PTC Therapeutics with TREAT-NMD and the Cooperative International Neuromuscular Research Group. Mr. Delage and five other authors are employees of PTC Therapeutics, and six authors had received speaker or consultancy fees or served on the advisory board of a variety of companies.
SOURCE: Delage A et al. AANEM 2019, Abstract 115.
AUSTIN, TEX. – , according to new data.
“Participants in the STRIDE Registry [real-world patients] showed a reduction in functional decline over 48 weeks, compared with patients receiving placebo” in the trial, reported Abdallah Delage of PTC Therapeutics in Zug, Switzerland, and his associates.
Duchenne muscular dystrophy affects an estimated 1 in 3,600-6,000 male births globally, about 10%-15% of whom have nonsense mutation DMD. This mutation causes a truncated, nonfunctional dystrophin protein due to a premature stop codon, the authors explained. Ataluren “promotes ribosomal read-through of the premature stop codon to produce a full-length dystrophin protein,” they explained.
Ataluren is currently approved for ambulatory patients age 2 and older with nonsense mutation DMD in the European Union and several other European countries. Israel, Korea, Chile, and Ukraine have approved it for patients aged 5 and older.
The Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry contains real-world data from patients using ataluren as part of an ongoing multicenter observational postapproval safety study. The investigators are tracking patients for at least 5 years after enrollment in 14 countries where ataluren is approved or commercially available through early-access programs. Patients take 40 mg/kg daily: 10 mg/kg in the morning, 10 mg/kg midday, and 20 mg/kg in the evening.
The researchers compared outcomes in 216 patients in the STRIDE Registry with participants in a randomized controlled phase 3 study of ataluren involving 228 boys, aged 7-16, who received ataluren (n = 114) or placebo (n = 114) for 48 weeks. Patients were an average 9 years old in STRIDE and in both arms of the randomized controlled trial.
The STRIDE Registry participants, comprising 184 ambulatory and 26 nonambulatory patients at enrollment, had at least 48 weeks between their first and last assessment. All of the patients in the phase 3 study and 88.6% of the STRIDE Registry patients were receiving corticosteroids along with ataluren. The researchers compared the 184 ambulatory STRIDE participants with the participants of the randomized controlled trial for one primary and four secondary endpoints from baseline to 48 weeks.
For the primary endpoint, 6-minute walk distance, average distance was 35 meters shorter than baseline in STRIDE Registry participants (n = 66), 42.2 meters shorter in the patients receiving ataluren in the phase 3 study (n = 109), and 57.6 meters shorter in RCT patients receiving placebo in the phase 3 trial (n = 109).
A secondary endpoint, the time it took patients to walk or run 10 meters, increased 1.6 seconds from baseline to 48 weeks in STRIDE Registry participants (n = 61), 2.3 seconds in participants receiving ataluren in the phase 3 trial (n = 109), and 3.5 seconds in study participants receiving placebo (n = 110).
Another secondary endpoint, the change in time it took for patients to stand from supine position from baseline to 48 weeks, was 2.9 additional seconds for STRIDE participants (n = 55), 3.8 additional seconds in study participants receiving ataluren (n = 101), and 3.9 additional seconds in study participants receiving placebo (n = 96).
Two final secondary endpoints were the changes in time to climb four stairs and to descend four stairs from baseline to 48 weeks. STRIDE participants (n = 47) climbed four stairs 1.2 seconds more slowly at 48 weeks, compared with 2.7 seconds more slowly in the participants who received ataluren in the phase 3 trial (n = 105) and 4.5 seconds more slowly in those who received placebo. Descending four stairs took 0.5 more seconds at 48 weeks in STRIDE participants (n = 40), 2.2 more seconds in participants who received ataluren in the phase 3 trial (n = 106), and 4.0 more seconds in those who received placebo (n = 100).
At least one adverse event occurred in 20.7% of registry participants; seven of these were considered treatment related. Treatment-related side effects included abdominal pain, vomiting, headache, stomach ache, diarrhea, and increased serum lipids.
The study and STRIDE Registry is funded by PTC Therapeutics with TREAT-NMD and the Cooperative International Neuromuscular Research Group. Mr. Delage and five other authors are employees of PTC Therapeutics, and six authors had received speaker or consultancy fees or served on the advisory board of a variety of companies.
SOURCE: Delage A et al. AANEM 2019, Abstract 115.
REPORTING FROM AANEM 2019
Geriatric assessments improve communication during elderly patient visits
Integrating geriatric assessments into oncology consultations for older patients with cancer could help address aging-related concerns about their disease and treatment, a study has found.
In a study published in JAMA Oncology, researchers reported the outcomes of a cluster-randomized clinical trial involving 541 cancer patients aged 70 years and older, from 31 community oncology practices.
The trial examined the effects of an intervention in which the patient’s oncologist was provided with a geriatric assessment summary of that patient’s age-related impairments, and guided recommendations on how to manage these.
“As an example, the summary would include information that a patient recently fell, that falls increase the risk of chemotherapy toxic effects, and a recommendation for physical therapy to prevent falls,” wrote Supriya G. Mohile, MD, of the University of Rochester (N.Y.), and coauthors. The usual care control arm provided oncologists with alerts only if their patients had scored abnormally on depression and cognitive tests.
The study found that over 6 months, patients in the intervention group reported significantly greater satisfaction with communication about aging-related concerns and greater satisfaction with their overall care.
The intervention group had an adjusted mean of 8.02 conversations about aging-related concerns compared with 4.43 conversations in the usual care group, and an adjusted mean of 4.6 high-quality conversations compared with 2.59 in the usual care group. There were also significantly more conversations about the assessment recommendations in the intervention group compared with the usual care group.
There were no significant differences between the two groups in health-related quality of life for patients or their caregivers. However, caregivers in the intervention group did report significantly greater satisfaction with communication about aging-related concerns and the patient’s condition. They also reported significantly greater satisfaction with their own communications with oncologists about overall care.
“Evidence increasingly supports the use of GA for evaluation and management of older patients with cancer to guide shared decision-making between older patients, caregivers, and oncologists,” the authors wrote. “Despite patient and caregiver concerns and preferences for maintaining function and cognition, oncologists often do not discuss implications of aging-related conditions or inform older patients and caregivers of heightened risk of adverse events from treatment.”
Overall, 90% of patients had at least three geriatric assessment domain impairments; 93.7% had impairments in physical performance, 25.1% reported impairments in psychological status, and 33.3% had possible cognitive impairments.
“These patients represent less-fit individuals for whom there is limited evidence for the risks and benefits of cancer treatment, yet these patients are commonly seen in real-world community practices,” the authors wrote.
The study was supported by funding and individual research grants from the Patient-Centered Outcomes Research Institute, the University of Rochester, the National Institute of Aging, and the National Cancer Institute. Two authors also declared funding from the pharmaceutical sector outside the study.
SOURCE: Mohile S et al. JAMA Oncol. 2019 Nov 7. doi: 10.1001/jamaoncol.2019.4728.
Around half of all cancer cases and 70% of all cancer deaths occur in individuals aged over 65 years; this is a very heterogeneous population with a range of impairments, comorbidities, polypharmacy, geriatric syndromes, and other issues. There is limited evidence to guide management of cancer in this group.
This study provides further support for a worldwide consensus on the importance of incorporating geriatric assessments into oncology practice, although it did not find an improvement in quality of life associated with this. This may be because improved communication doesn’t necessarily translate into implementation of the management recommendations that might have benefited quality of life in these patients. There is a need for multidisciplinary care in the older population, not just at assessment but throughout the entire care pathway.
Laura Biganzoli, MD, and Amelia McCartney, MBBS, are from the “Sandro Pitigliani” medical oncology department, Hospital of Prato (Italy), and Nicolò Matteo Luca Battisti, MD, is from the department of medicine–breast unit, The Royal Marsden National Health Service Foundation Trust, Sutton, England. These comments are adapted from their editorial (JAMA Oncol. 2019 Nov 7. doi: 10.1001/jamaoncol.2019.4708). Dr Battisti declared personal fees and nonfinancial support from the pharmaceutical sector unrelated to the submitted work. No other conflicts of interest were declared.
Around half of all cancer cases and 70% of all cancer deaths occur in individuals aged over 65 years; this is a very heterogeneous population with a range of impairments, comorbidities, polypharmacy, geriatric syndromes, and other issues. There is limited evidence to guide management of cancer in this group.
This study provides further support for a worldwide consensus on the importance of incorporating geriatric assessments into oncology practice, although it did not find an improvement in quality of life associated with this. This may be because improved communication doesn’t necessarily translate into implementation of the management recommendations that might have benefited quality of life in these patients. There is a need for multidisciplinary care in the older population, not just at assessment but throughout the entire care pathway.
Laura Biganzoli, MD, and Amelia McCartney, MBBS, are from the “Sandro Pitigliani” medical oncology department, Hospital of Prato (Italy), and Nicolò Matteo Luca Battisti, MD, is from the department of medicine–breast unit, The Royal Marsden National Health Service Foundation Trust, Sutton, England. These comments are adapted from their editorial (JAMA Oncol. 2019 Nov 7. doi: 10.1001/jamaoncol.2019.4708). Dr Battisti declared personal fees and nonfinancial support from the pharmaceutical sector unrelated to the submitted work. No other conflicts of interest were declared.
Around half of all cancer cases and 70% of all cancer deaths occur in individuals aged over 65 years; this is a very heterogeneous population with a range of impairments, comorbidities, polypharmacy, geriatric syndromes, and other issues. There is limited evidence to guide management of cancer in this group.
This study provides further support for a worldwide consensus on the importance of incorporating geriatric assessments into oncology practice, although it did not find an improvement in quality of life associated with this. This may be because improved communication doesn’t necessarily translate into implementation of the management recommendations that might have benefited quality of life in these patients. There is a need for multidisciplinary care in the older population, not just at assessment but throughout the entire care pathway.
Laura Biganzoli, MD, and Amelia McCartney, MBBS, are from the “Sandro Pitigliani” medical oncology department, Hospital of Prato (Italy), and Nicolò Matteo Luca Battisti, MD, is from the department of medicine–breast unit, The Royal Marsden National Health Service Foundation Trust, Sutton, England. These comments are adapted from their editorial (JAMA Oncol. 2019 Nov 7. doi: 10.1001/jamaoncol.2019.4708). Dr Battisti declared personal fees and nonfinancial support from the pharmaceutical sector unrelated to the submitted work. No other conflicts of interest were declared.
Integrating geriatric assessments into oncology consultations for older patients with cancer could help address aging-related concerns about their disease and treatment, a study has found.
In a study published in JAMA Oncology, researchers reported the outcomes of a cluster-randomized clinical trial involving 541 cancer patients aged 70 years and older, from 31 community oncology practices.
The trial examined the effects of an intervention in which the patient’s oncologist was provided with a geriatric assessment summary of that patient’s age-related impairments, and guided recommendations on how to manage these.
“As an example, the summary would include information that a patient recently fell, that falls increase the risk of chemotherapy toxic effects, and a recommendation for physical therapy to prevent falls,” wrote Supriya G. Mohile, MD, of the University of Rochester (N.Y.), and coauthors. The usual care control arm provided oncologists with alerts only if their patients had scored abnormally on depression and cognitive tests.
The study found that over 6 months, patients in the intervention group reported significantly greater satisfaction with communication about aging-related concerns and greater satisfaction with their overall care.
The intervention group had an adjusted mean of 8.02 conversations about aging-related concerns compared with 4.43 conversations in the usual care group, and an adjusted mean of 4.6 high-quality conversations compared with 2.59 in the usual care group. There were also significantly more conversations about the assessment recommendations in the intervention group compared with the usual care group.
There were no significant differences between the two groups in health-related quality of life for patients or their caregivers. However, caregivers in the intervention group did report significantly greater satisfaction with communication about aging-related concerns and the patient’s condition. They also reported significantly greater satisfaction with their own communications with oncologists about overall care.
“Evidence increasingly supports the use of GA for evaluation and management of older patients with cancer to guide shared decision-making between older patients, caregivers, and oncologists,” the authors wrote. “Despite patient and caregiver concerns and preferences for maintaining function and cognition, oncologists often do not discuss implications of aging-related conditions or inform older patients and caregivers of heightened risk of adverse events from treatment.”
Overall, 90% of patients had at least three geriatric assessment domain impairments; 93.7% had impairments in physical performance, 25.1% reported impairments in psychological status, and 33.3% had possible cognitive impairments.
“These patients represent less-fit individuals for whom there is limited evidence for the risks and benefits of cancer treatment, yet these patients are commonly seen in real-world community practices,” the authors wrote.
The study was supported by funding and individual research grants from the Patient-Centered Outcomes Research Institute, the University of Rochester, the National Institute of Aging, and the National Cancer Institute. Two authors also declared funding from the pharmaceutical sector outside the study.
SOURCE: Mohile S et al. JAMA Oncol. 2019 Nov 7. doi: 10.1001/jamaoncol.2019.4728.
Integrating geriatric assessments into oncology consultations for older patients with cancer could help address aging-related concerns about their disease and treatment, a study has found.
In a study published in JAMA Oncology, researchers reported the outcomes of a cluster-randomized clinical trial involving 541 cancer patients aged 70 years and older, from 31 community oncology practices.
The trial examined the effects of an intervention in which the patient’s oncologist was provided with a geriatric assessment summary of that patient’s age-related impairments, and guided recommendations on how to manage these.
“As an example, the summary would include information that a patient recently fell, that falls increase the risk of chemotherapy toxic effects, and a recommendation for physical therapy to prevent falls,” wrote Supriya G. Mohile, MD, of the University of Rochester (N.Y.), and coauthors. The usual care control arm provided oncologists with alerts only if their patients had scored abnormally on depression and cognitive tests.
The study found that over 6 months, patients in the intervention group reported significantly greater satisfaction with communication about aging-related concerns and greater satisfaction with their overall care.
The intervention group had an adjusted mean of 8.02 conversations about aging-related concerns compared with 4.43 conversations in the usual care group, and an adjusted mean of 4.6 high-quality conversations compared with 2.59 in the usual care group. There were also significantly more conversations about the assessment recommendations in the intervention group compared with the usual care group.
There were no significant differences between the two groups in health-related quality of life for patients or their caregivers. However, caregivers in the intervention group did report significantly greater satisfaction with communication about aging-related concerns and the patient’s condition. They also reported significantly greater satisfaction with their own communications with oncologists about overall care.
“Evidence increasingly supports the use of GA for evaluation and management of older patients with cancer to guide shared decision-making between older patients, caregivers, and oncologists,” the authors wrote. “Despite patient and caregiver concerns and preferences for maintaining function and cognition, oncologists often do not discuss implications of aging-related conditions or inform older patients and caregivers of heightened risk of adverse events from treatment.”
Overall, 90% of patients had at least three geriatric assessment domain impairments; 93.7% had impairments in physical performance, 25.1% reported impairments in psychological status, and 33.3% had possible cognitive impairments.
“These patients represent less-fit individuals for whom there is limited evidence for the risks and benefits of cancer treatment, yet these patients are commonly seen in real-world community practices,” the authors wrote.
The study was supported by funding and individual research grants from the Patient-Centered Outcomes Research Institute, the University of Rochester, the National Institute of Aging, and the National Cancer Institute. Two authors also declared funding from the pharmaceutical sector outside the study.
SOURCE: Mohile S et al. JAMA Oncol. 2019 Nov 7. doi: 10.1001/jamaoncol.2019.4728.
FROM JAMA ONCOLOGY
U.S. deaths from preventable causes occur more often in rural areas
compared with the most urban counties during 2010-2017, according to study published in CDC’s Morbidity and Mortality Weekly Report.
These leading causes of death comprised heart disease, cancer, unintentional injury, chronic lower respiratory disease, and stroke and accounted for approximately 1.7 million deaths or 61% of all deaths in 2017.
The study presents estimates, percentages, and annual percent changes for potentially excess deaths by urban-rural classification from heart disease, cancer, unintentional injury, chronic lower respiratory disease, and stroke. Urban-rural categories were identified using the National Center for Health Statistics 2013 urban-rural classification scheme for counties.
The report’s main findings include the following statistics:
- In 2010, 28.7% of deaths from cancer in the most rural counties were potentially preventable, compared with 17.9% in the most urban counties. By 2017, 21.7% of cancer deaths in the most rural counties were potentially preventable, compared with 3.2% in the most urban counties.
- In 2010, 45.1% of deaths from heart disease in the most rural counties were potentially preventable, compared with 33.5% in the most urban counties. By 2017, 44.9% of deaths from heart disease in the most rural counties were potentially preventable, compared with 28.0% in the most urban counties.
- In 2010, 60.9% of deaths from unintentional injury in the most rural counties were potentially preventable, compared with 25.4% in the most urban counties. By 2017, 64.1% of deaths from unintentional injury in the most rural counties were potentially preventable, compared with 47.8% in the most urban counties.
- In 2010, 54.3% of deaths from chronic lower respiratory disease (such as COPD) in the most rural counties were potentially preventable, compared with 23.4% in the most urban counties. By 2017, 57.1% of deaths from chronic lower respiratory disease in the most rural counties were potentially preventable, compared with 13% in the most urban counties.
- In 2010, 41.6% of deaths from stroke in the most rural counties were potentially preventable, compared with 31.7% in most urban areas. By 2017, 37.8% of deaths from stroke in the most rural counties were potentially preventable, compared with 27.4% most urban counties.
“This report demonstrates the value of analyzing potentially excess deaths according to the six 2013 [National Center for Health Statistics] urban-rural county classifications. Reporting trends in potentially excess deaths over an 8-year period highlights differences over time, independent of traditional underlying structural, environmental, and genetic factors,” wrote Macarena C. Garcia, DrPH, and coauthors.
“Because of increasing percentages of potentially excess deaths in recent years for certain causes of death and certain demographic groups, these data can be used, with traditional rate comparisons, by public health practitioners who are involved in planning interventions. Comparing the findings in this report with data from tools such as the CDC Interactive Atlas of Heart Disease and Stroke might help identify the social determinants, health care infrastructures, and public policies that could increase or decrease numbers of deaths in specific nonmetropolitan areas,” they added.
The study authors did not disclose any potential conflicts of interest.
SOURCE: Garcia MC et al. MMWR Morb Mortal Wkly Rep. 2019 Nov 8: 68(10);1-11.
compared with the most urban counties during 2010-2017, according to study published in CDC’s Morbidity and Mortality Weekly Report.
These leading causes of death comprised heart disease, cancer, unintentional injury, chronic lower respiratory disease, and stroke and accounted for approximately 1.7 million deaths or 61% of all deaths in 2017.
The study presents estimates, percentages, and annual percent changes for potentially excess deaths by urban-rural classification from heart disease, cancer, unintentional injury, chronic lower respiratory disease, and stroke. Urban-rural categories were identified using the National Center for Health Statistics 2013 urban-rural classification scheme for counties.
The report’s main findings include the following statistics:
- In 2010, 28.7% of deaths from cancer in the most rural counties were potentially preventable, compared with 17.9% in the most urban counties. By 2017, 21.7% of cancer deaths in the most rural counties were potentially preventable, compared with 3.2% in the most urban counties.
- In 2010, 45.1% of deaths from heart disease in the most rural counties were potentially preventable, compared with 33.5% in the most urban counties. By 2017, 44.9% of deaths from heart disease in the most rural counties were potentially preventable, compared with 28.0% in the most urban counties.
- In 2010, 60.9% of deaths from unintentional injury in the most rural counties were potentially preventable, compared with 25.4% in the most urban counties. By 2017, 64.1% of deaths from unintentional injury in the most rural counties were potentially preventable, compared with 47.8% in the most urban counties.
- In 2010, 54.3% of deaths from chronic lower respiratory disease (such as COPD) in the most rural counties were potentially preventable, compared with 23.4% in the most urban counties. By 2017, 57.1% of deaths from chronic lower respiratory disease in the most rural counties were potentially preventable, compared with 13% in the most urban counties.
- In 2010, 41.6% of deaths from stroke in the most rural counties were potentially preventable, compared with 31.7% in most urban areas. By 2017, 37.8% of deaths from stroke in the most rural counties were potentially preventable, compared with 27.4% most urban counties.
“This report demonstrates the value of analyzing potentially excess deaths according to the six 2013 [National Center for Health Statistics] urban-rural county classifications. Reporting trends in potentially excess deaths over an 8-year period highlights differences over time, independent of traditional underlying structural, environmental, and genetic factors,” wrote Macarena C. Garcia, DrPH, and coauthors.
“Because of increasing percentages of potentially excess deaths in recent years for certain causes of death and certain demographic groups, these data can be used, with traditional rate comparisons, by public health practitioners who are involved in planning interventions. Comparing the findings in this report with data from tools such as the CDC Interactive Atlas of Heart Disease and Stroke might help identify the social determinants, health care infrastructures, and public policies that could increase or decrease numbers of deaths in specific nonmetropolitan areas,” they added.
The study authors did not disclose any potential conflicts of interest.
SOURCE: Garcia MC et al. MMWR Morb Mortal Wkly Rep. 2019 Nov 8: 68(10);1-11.
compared with the most urban counties during 2010-2017, according to study published in CDC’s Morbidity and Mortality Weekly Report.
These leading causes of death comprised heart disease, cancer, unintentional injury, chronic lower respiratory disease, and stroke and accounted for approximately 1.7 million deaths or 61% of all deaths in 2017.
The study presents estimates, percentages, and annual percent changes for potentially excess deaths by urban-rural classification from heart disease, cancer, unintentional injury, chronic lower respiratory disease, and stroke. Urban-rural categories were identified using the National Center for Health Statistics 2013 urban-rural classification scheme for counties.
The report’s main findings include the following statistics:
- In 2010, 28.7% of deaths from cancer in the most rural counties were potentially preventable, compared with 17.9% in the most urban counties. By 2017, 21.7% of cancer deaths in the most rural counties were potentially preventable, compared with 3.2% in the most urban counties.
- In 2010, 45.1% of deaths from heart disease in the most rural counties were potentially preventable, compared with 33.5% in the most urban counties. By 2017, 44.9% of deaths from heart disease in the most rural counties were potentially preventable, compared with 28.0% in the most urban counties.
- In 2010, 60.9% of deaths from unintentional injury in the most rural counties were potentially preventable, compared with 25.4% in the most urban counties. By 2017, 64.1% of deaths from unintentional injury in the most rural counties were potentially preventable, compared with 47.8% in the most urban counties.
- In 2010, 54.3% of deaths from chronic lower respiratory disease (such as COPD) in the most rural counties were potentially preventable, compared with 23.4% in the most urban counties. By 2017, 57.1% of deaths from chronic lower respiratory disease in the most rural counties were potentially preventable, compared with 13% in the most urban counties.
- In 2010, 41.6% of deaths from stroke in the most rural counties were potentially preventable, compared with 31.7% in most urban areas. By 2017, 37.8% of deaths from stroke in the most rural counties were potentially preventable, compared with 27.4% most urban counties.
“This report demonstrates the value of analyzing potentially excess deaths according to the six 2013 [National Center for Health Statistics] urban-rural county classifications. Reporting trends in potentially excess deaths over an 8-year period highlights differences over time, independent of traditional underlying structural, environmental, and genetic factors,” wrote Macarena C. Garcia, DrPH, and coauthors.
“Because of increasing percentages of potentially excess deaths in recent years for certain causes of death and certain demographic groups, these data can be used, with traditional rate comparisons, by public health practitioners who are involved in planning interventions. Comparing the findings in this report with data from tools such as the CDC Interactive Atlas of Heart Disease and Stroke might help identify the social determinants, health care infrastructures, and public policies that could increase or decrease numbers of deaths in specific nonmetropolitan areas,” they added.
The study authors did not disclose any potential conflicts of interest.
SOURCE: Garcia MC et al. MMWR Morb Mortal Wkly Rep. 2019 Nov 8: 68(10);1-11.
In Oregon, ‘war on melanoma’ takes flight
LAS VEGAS – with the goal of dramatically reducing melanoma deaths in the state of 4.2 million people.
Research shows that “early detection works in melanoma. And awareness seems to be important for the public in detecting melanoma early,” said Sancy Leachman, MD, PhD, professor and chair of the department of dermatology at Oregon Health & Science University, Portland, said at the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.
Dr. Leachman, who is also the John D. Gray chair in melanoma research at OHSU, directs the “War on Melanoma” project, which was inspired by a project in the German state of Schleswig-Holstein that aimed to screen all residents aged over 21 years for melanoma. The project featured an education campaign and population-wide skin cancer screening, and mandated that certain patients – those at high risk and those who needed biopsies – would be referred to dermatologists (Br J Cancer. 2012 Feb 28;106[5]:970-4).
According to Dr. Leachman, the German project was initially a success, and was linked to a 50% decrease in melanoma mortality.
“In Oregon, we thought ‘that sounds very good, so we’re going to try that.’ ” But when it went national, the German project failed, she said, providing lessons for dermatologists in Oregon. “We’re going to try to improve upon the first [German] experiment by making ours controlled with a defined baseline. If it works, the plan is to extend it to select states nationwide.”
The War on Melanoma project was launched earlier this year. According to the university, the program is featuring or will feature the following elements:
- A media campaign called “Start Seeing Melanoma” that’s devoted to educating the public about the early detection of melanoma.
- The release of an iPhone app called MoleMapper that allows users to monitor moles over time.
- Education of medical professionals and partnerships with state-licensed skin care professionals such as massage therapists, cosmetologists, and tattoo artists.
In an interview at the meeting, Dr. Leachman said the project is expected to cost $1 million to $1.5 million over the first 18 months. At that time, she said, researchers will survey residents of Oregon and two control states – Washington and Utah– to see if their knowledge of melanoma has improved, compared with baseline survey results.
In 5 years, researchers plan to begin analyzing melanoma mortality in Oregon and the other states. “We hope to see a decline,” and to link it to increased awareness of melanoma, she said.
Dr. Leachman reported no relevant disclosures. She spoke during a forum on cutaneous malignancies at the meeting.
SDEF and this news organization are owned by the same parent company.
LAS VEGAS – with the goal of dramatically reducing melanoma deaths in the state of 4.2 million people.
Research shows that “early detection works in melanoma. And awareness seems to be important for the public in detecting melanoma early,” said Sancy Leachman, MD, PhD, professor and chair of the department of dermatology at Oregon Health & Science University, Portland, said at the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.
Dr. Leachman, who is also the John D. Gray chair in melanoma research at OHSU, directs the “War on Melanoma” project, which was inspired by a project in the German state of Schleswig-Holstein that aimed to screen all residents aged over 21 years for melanoma. The project featured an education campaign and population-wide skin cancer screening, and mandated that certain patients – those at high risk and those who needed biopsies – would be referred to dermatologists (Br J Cancer. 2012 Feb 28;106[5]:970-4).
According to Dr. Leachman, the German project was initially a success, and was linked to a 50% decrease in melanoma mortality.
“In Oregon, we thought ‘that sounds very good, so we’re going to try that.’ ” But when it went national, the German project failed, she said, providing lessons for dermatologists in Oregon. “We’re going to try to improve upon the first [German] experiment by making ours controlled with a defined baseline. If it works, the plan is to extend it to select states nationwide.”
The War on Melanoma project was launched earlier this year. According to the university, the program is featuring or will feature the following elements:
- A media campaign called “Start Seeing Melanoma” that’s devoted to educating the public about the early detection of melanoma.
- The release of an iPhone app called MoleMapper that allows users to monitor moles over time.
- Education of medical professionals and partnerships with state-licensed skin care professionals such as massage therapists, cosmetologists, and tattoo artists.
In an interview at the meeting, Dr. Leachman said the project is expected to cost $1 million to $1.5 million over the first 18 months. At that time, she said, researchers will survey residents of Oregon and two control states – Washington and Utah– to see if their knowledge of melanoma has improved, compared with baseline survey results.
In 5 years, researchers plan to begin analyzing melanoma mortality in Oregon and the other states. “We hope to see a decline,” and to link it to increased awareness of melanoma, she said.
Dr. Leachman reported no relevant disclosures. She spoke during a forum on cutaneous malignancies at the meeting.
SDEF and this news organization are owned by the same parent company.
LAS VEGAS – with the goal of dramatically reducing melanoma deaths in the state of 4.2 million people.
Research shows that “early detection works in melanoma. And awareness seems to be important for the public in detecting melanoma early,” said Sancy Leachman, MD, PhD, professor and chair of the department of dermatology at Oregon Health & Science University, Portland, said at the Skin Disease Education Foundation’s annual Las Vegas Dermatology Seminar.
Dr. Leachman, who is also the John D. Gray chair in melanoma research at OHSU, directs the “War on Melanoma” project, which was inspired by a project in the German state of Schleswig-Holstein that aimed to screen all residents aged over 21 years for melanoma. The project featured an education campaign and population-wide skin cancer screening, and mandated that certain patients – those at high risk and those who needed biopsies – would be referred to dermatologists (Br J Cancer. 2012 Feb 28;106[5]:970-4).
According to Dr. Leachman, the German project was initially a success, and was linked to a 50% decrease in melanoma mortality.
“In Oregon, we thought ‘that sounds very good, so we’re going to try that.’ ” But when it went national, the German project failed, she said, providing lessons for dermatologists in Oregon. “We’re going to try to improve upon the first [German] experiment by making ours controlled with a defined baseline. If it works, the plan is to extend it to select states nationwide.”
The War on Melanoma project was launched earlier this year. According to the university, the program is featuring or will feature the following elements:
- A media campaign called “Start Seeing Melanoma” that’s devoted to educating the public about the early detection of melanoma.
- The release of an iPhone app called MoleMapper that allows users to monitor moles over time.
- Education of medical professionals and partnerships with state-licensed skin care professionals such as massage therapists, cosmetologists, and tattoo artists.
In an interview at the meeting, Dr. Leachman said the project is expected to cost $1 million to $1.5 million over the first 18 months. At that time, she said, researchers will survey residents of Oregon and two control states – Washington and Utah– to see if their knowledge of melanoma has improved, compared with baseline survey results.
In 5 years, researchers plan to begin analyzing melanoma mortality in Oregon and the other states. “We hope to see a decline,” and to link it to increased awareness of melanoma, she said.
Dr. Leachman reported no relevant disclosures. She spoke during a forum on cutaneous malignancies at the meeting.
SDEF and this news organization are owned by the same parent company.
EXPERT ANALYSIS FROM SDEF LAS VEGAS DERMATOLOGY SEMINAR
Mandatory OSA treatment for truckers lowers insurance costs
“Among the 1.87 million U.S. commercial drivers estimated by the Bureau of Labor Statistics to be operating non–farm-based heavy trucks (gross weight at least 26,000 pounds), 17%-28%, or 318,00 to 524,000, are expected to have at least mild [obstructive sleep apnea] based on prevalence studies on commercial drivers,” wrote Stephen V. Burks, PhD, of the University of Minnesota, Morris, and colleagues.
“If the larger population of the 4.0 million drivers estimated by the Federal Motor Carrier Safety Administration to be using commercial driver’s licenses in interstate and intrastate transportation is considered, 0.68 to 1.1 million drivers may have OSA. The majority of these drivers are thought to be undiagnosed and untreated. There is thus considerable scope for healthcare cost savings through treatment of OSA among commercial drivers,” they noted.
The study was published in Sleep.
In 2017, after industry complaints about costs, the federal government withdrew its proposal to mandate testing truckers for OSA. Yet some commercial fleets do require testing, and a new study looking at retrospective data from a large fleet’s health insurance program shows that drivers diagnosed and treated for OSA saw medical cost savings, compared with those considered likely to have OSA who had neither diagnosis nor treatment.
Dr. Burks and his colleagues looked at records for 1,516 drivers tested for OSA, of whom 1,224 were positive, and compared these with an equal number of controls flagged through the same screening program as likely to have OSA, but who had never received a diagnosis in a sleep clinic or treatment with auto-adjusting positive airway pressure (APAP). All cases received auto-adjusting positive airway pressure (APAP) treatment. The investigators then looked at insurance costs for diagnosed drivers, compared with screen-positive controls before and after the polysomnogram date, over an 18-month period.
Most of the diagnosed cases in the cohort (n = 932) were deemed compliant with treatment. For every pair of subjects and controls, the researchers looked at per-member insurance costs over 18 months, though not all drivers were observed for a full period of 18 months, mostly because of turnover. Dr. Burks and colleagues found that non-OSA related medical claim costs savings after diagnosis and treatment of every 100 screen-positive controls was $153,042 (95% confidence interval, –$5,352, $330,525, P = .06.) Subjects adhering to treatment with APAP saw mean non-OSA related savings of $441 per member per month (95% CI, –$861, –$21, P = .035).
“Taken together, this is substantial evidence that OSA treatment is associated with savings in non-OSA–program medical insurance claim costs,” the authors wrote, adding that such savings could be expected to help offset expenses related to mandatory OSA programs. The authors acknowledged as a limitation of their study that it did not capture costs of pharmaceutical treatment or those of the OSA program itself. Nor did the findings capture “the value of injuries, lost work time, or disability days associated with untreated OSA, nor the savings from avoided preventable crashes.”
The study received funding from Harvard University and the National Surface Transportation Safety Center for Excellence. Two coauthors disclosed funding from pharmaceutical and device manufacturers and hold patents on sleep therapies. Another coauthor disclosed being an expert witness in trucking-related cases and one received funds from the study’s sponsor, a trucking safety research group.
SOURCE: Burks SV et al. Sleep 2019 Oct 24. doi: 10.1093/sleep/zsz262.
“Among the 1.87 million U.S. commercial drivers estimated by the Bureau of Labor Statistics to be operating non–farm-based heavy trucks (gross weight at least 26,000 pounds), 17%-28%, or 318,00 to 524,000, are expected to have at least mild [obstructive sleep apnea] based on prevalence studies on commercial drivers,” wrote Stephen V. Burks, PhD, of the University of Minnesota, Morris, and colleagues.
“If the larger population of the 4.0 million drivers estimated by the Federal Motor Carrier Safety Administration to be using commercial driver’s licenses in interstate and intrastate transportation is considered, 0.68 to 1.1 million drivers may have OSA. The majority of these drivers are thought to be undiagnosed and untreated. There is thus considerable scope for healthcare cost savings through treatment of OSA among commercial drivers,” they noted.
The study was published in Sleep.
In 2017, after industry complaints about costs, the federal government withdrew its proposal to mandate testing truckers for OSA. Yet some commercial fleets do require testing, and a new study looking at retrospective data from a large fleet’s health insurance program shows that drivers diagnosed and treated for OSA saw medical cost savings, compared with those considered likely to have OSA who had neither diagnosis nor treatment.
Dr. Burks and his colleagues looked at records for 1,516 drivers tested for OSA, of whom 1,224 were positive, and compared these with an equal number of controls flagged through the same screening program as likely to have OSA, but who had never received a diagnosis in a sleep clinic or treatment with auto-adjusting positive airway pressure (APAP). All cases received auto-adjusting positive airway pressure (APAP) treatment. The investigators then looked at insurance costs for diagnosed drivers, compared with screen-positive controls before and after the polysomnogram date, over an 18-month period.
Most of the diagnosed cases in the cohort (n = 932) were deemed compliant with treatment. For every pair of subjects and controls, the researchers looked at per-member insurance costs over 18 months, though not all drivers were observed for a full period of 18 months, mostly because of turnover. Dr. Burks and colleagues found that non-OSA related medical claim costs savings after diagnosis and treatment of every 100 screen-positive controls was $153,042 (95% confidence interval, –$5,352, $330,525, P = .06.) Subjects adhering to treatment with APAP saw mean non-OSA related savings of $441 per member per month (95% CI, –$861, –$21, P = .035).
“Taken together, this is substantial evidence that OSA treatment is associated with savings in non-OSA–program medical insurance claim costs,” the authors wrote, adding that such savings could be expected to help offset expenses related to mandatory OSA programs. The authors acknowledged as a limitation of their study that it did not capture costs of pharmaceutical treatment or those of the OSA program itself. Nor did the findings capture “the value of injuries, lost work time, or disability days associated with untreated OSA, nor the savings from avoided preventable crashes.”
The study received funding from Harvard University and the National Surface Transportation Safety Center for Excellence. Two coauthors disclosed funding from pharmaceutical and device manufacturers and hold patents on sleep therapies. Another coauthor disclosed being an expert witness in trucking-related cases and one received funds from the study’s sponsor, a trucking safety research group.
SOURCE: Burks SV et al. Sleep 2019 Oct 24. doi: 10.1093/sleep/zsz262.
“Among the 1.87 million U.S. commercial drivers estimated by the Bureau of Labor Statistics to be operating non–farm-based heavy trucks (gross weight at least 26,000 pounds), 17%-28%, or 318,00 to 524,000, are expected to have at least mild [obstructive sleep apnea] based on prevalence studies on commercial drivers,” wrote Stephen V. Burks, PhD, of the University of Minnesota, Morris, and colleagues.
“If the larger population of the 4.0 million drivers estimated by the Federal Motor Carrier Safety Administration to be using commercial driver’s licenses in interstate and intrastate transportation is considered, 0.68 to 1.1 million drivers may have OSA. The majority of these drivers are thought to be undiagnosed and untreated. There is thus considerable scope for healthcare cost savings through treatment of OSA among commercial drivers,” they noted.
The study was published in Sleep.
In 2017, after industry complaints about costs, the federal government withdrew its proposal to mandate testing truckers for OSA. Yet some commercial fleets do require testing, and a new study looking at retrospective data from a large fleet’s health insurance program shows that drivers diagnosed and treated for OSA saw medical cost savings, compared with those considered likely to have OSA who had neither diagnosis nor treatment.
Dr. Burks and his colleagues looked at records for 1,516 drivers tested for OSA, of whom 1,224 were positive, and compared these with an equal number of controls flagged through the same screening program as likely to have OSA, but who had never received a diagnosis in a sleep clinic or treatment with auto-adjusting positive airway pressure (APAP). All cases received auto-adjusting positive airway pressure (APAP) treatment. The investigators then looked at insurance costs for diagnosed drivers, compared with screen-positive controls before and after the polysomnogram date, over an 18-month period.
Most of the diagnosed cases in the cohort (n = 932) were deemed compliant with treatment. For every pair of subjects and controls, the researchers looked at per-member insurance costs over 18 months, though not all drivers were observed for a full period of 18 months, mostly because of turnover. Dr. Burks and colleagues found that non-OSA related medical claim costs savings after diagnosis and treatment of every 100 screen-positive controls was $153,042 (95% confidence interval, –$5,352, $330,525, P = .06.) Subjects adhering to treatment with APAP saw mean non-OSA related savings of $441 per member per month (95% CI, –$861, –$21, P = .035).
“Taken together, this is substantial evidence that OSA treatment is associated with savings in non-OSA–program medical insurance claim costs,” the authors wrote, adding that such savings could be expected to help offset expenses related to mandatory OSA programs. The authors acknowledged as a limitation of their study that it did not capture costs of pharmaceutical treatment or those of the OSA program itself. Nor did the findings capture “the value of injuries, lost work time, or disability days associated with untreated OSA, nor the savings from avoided preventable crashes.”
The study received funding from Harvard University and the National Surface Transportation Safety Center for Excellence. Two coauthors disclosed funding from pharmaceutical and device manufacturers and hold patents on sleep therapies. Another coauthor disclosed being an expert witness in trucking-related cases and one received funds from the study’s sponsor, a trucking safety research group.
SOURCE: Burks SV et al. Sleep 2019 Oct 24. doi: 10.1093/sleep/zsz262.
FROM SLEEP
Dupilumab effective in early- and late-onset asthma
NEW ORLEANS – A new analysis suggests
Dupilumab may be more effective in reducing severe asthma exacerbations in patients with late-onset asthma, but the drug’s effect on lung function appeared the same regardless of asthma onset. Nicola Hanania, MD, of Baylor College of Medicine in Houston presented these results at the annual meeting of the American College of Chest Physicians.
Dr. Hanania and colleagues conducted a subanalysis of the LIBERTY ASTHMA QUEST study (NCT02414854). Previous data from this study showed that patients with uncontrolled, moderate to severe asthma who received dupilumab had fewer exacerbations and better lung function than did patients who received placebo (N Engl J Med. 2018;378:2486-96).
In their subanalysis, Dr. Hanania and his colleagues evaluated the efficacy of dupilumab, given at 200 mg or 300 mg every 2 weeks, in patients with early-onset asthma (at 40 years of age or younger) and late-onset asthma (at 41 years or older). The analysis included 919 patients with early-onset asthma who received dupilumab and 450 early-onset patients who received placebo. There were 345 patients with late-onset asthma who received dupilumab and 188 late-onset patients who received placebo.
Exacerbations
Dupilumab significantly reduced the adjusted annualized severe exacerbation rates during the 52-week treatment period. Significant reductions occurred in both early- and late-onset patients, though reductions were greater in the late-onset group.
In early-onset patients, dupilumab reduced severe exacerbations by 38% when given at 200 mg and by 37% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients, dupilumab reduced exacerbations by 64% and 69%, respectively (P less than .001 vs. placebo).
Dr. Hanania said it isn’t clear why late-onset patients appear to derive more benefit with regard to exacerbations. It may be because these patients have more comorbidities or because they aren’t using their inhalers correctly. The researchers are investigating these possibilities.
Dr. Hanania went on to note that reductions in exacerbation rates were greatest in patients with elevated blood eosinophils (150 cells/mcL or greater) or fractional exhaled nitric oxide (FeNO; 25 ppb or greater).
In patients with early-onset asthma and elevated eosinophils, dupilumab reduced severe exacerbations by 50% when given at 200 mg and by 55% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients with elevated eosinophils, dupilumab reduced exacerbations by 65% and 73%, respectively (P less than .001 vs. placebo).
In patients with early-onset asthma and elevated FeNO, dupilumab reduced severe exacerbations by 56% when given at 200 mg and by 52% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients with elevated FeNO, dupilumab reduced exacerbations by 79% and 71%, respectively (P less than .001 vs. placebo).
Lung function
Dupilumab also improved prebronchodilator forced expiratory volume in 1 second (pre-BD FEV1), compared with placebo, with similar results in early- and late-onset patients.
In early-onset patients, the P values were less than .001 for both doses of dupilumab at weeks 12 and 52. In late-onset patients, the P values were less than .001 for the 300-mg dose at week 12 and the 200-mg dose at week 52, less than .01 for the 200-mg dose at week 12, and less than .05 for the 300-mg dose at week 52.
The effects of dupilumab on pre-BD FEV1 were greatest in patients with elevated eosinophils or FeNO. At week 12, the P value was less than .001 for both doses of dupilumab in early-onset patients with elevated eosinophils or FeNO. The P value was less than .01 for both doses in late-onset patients with elevated eosinophils. And the P value was less than .001 for both doses in late-onset patients with elevated FeNO.
This research was sponsored by Sanofi and Regeneron. Dr. Hanania disclosed relationships with Genentech, Novartis, AstraZeneca, Boehringer Ingelheim, GSK, Regeneron, and Sanofi.
SOURCE: Hanania N et al. CHEST 2019. Abstract, doi: 10.1016/j.chest.2019.08.870.
NEW ORLEANS – A new analysis suggests
Dupilumab may be more effective in reducing severe asthma exacerbations in patients with late-onset asthma, but the drug’s effect on lung function appeared the same regardless of asthma onset. Nicola Hanania, MD, of Baylor College of Medicine in Houston presented these results at the annual meeting of the American College of Chest Physicians.
Dr. Hanania and colleagues conducted a subanalysis of the LIBERTY ASTHMA QUEST study (NCT02414854). Previous data from this study showed that patients with uncontrolled, moderate to severe asthma who received dupilumab had fewer exacerbations and better lung function than did patients who received placebo (N Engl J Med. 2018;378:2486-96).
In their subanalysis, Dr. Hanania and his colleagues evaluated the efficacy of dupilumab, given at 200 mg or 300 mg every 2 weeks, in patients with early-onset asthma (at 40 years of age or younger) and late-onset asthma (at 41 years or older). The analysis included 919 patients with early-onset asthma who received dupilumab and 450 early-onset patients who received placebo. There were 345 patients with late-onset asthma who received dupilumab and 188 late-onset patients who received placebo.
Exacerbations
Dupilumab significantly reduced the adjusted annualized severe exacerbation rates during the 52-week treatment period. Significant reductions occurred in both early- and late-onset patients, though reductions were greater in the late-onset group.
In early-onset patients, dupilumab reduced severe exacerbations by 38% when given at 200 mg and by 37% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients, dupilumab reduced exacerbations by 64% and 69%, respectively (P less than .001 vs. placebo).
Dr. Hanania said it isn’t clear why late-onset patients appear to derive more benefit with regard to exacerbations. It may be because these patients have more comorbidities or because they aren’t using their inhalers correctly. The researchers are investigating these possibilities.
Dr. Hanania went on to note that reductions in exacerbation rates were greatest in patients with elevated blood eosinophils (150 cells/mcL or greater) or fractional exhaled nitric oxide (FeNO; 25 ppb or greater).
In patients with early-onset asthma and elevated eosinophils, dupilumab reduced severe exacerbations by 50% when given at 200 mg and by 55% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients with elevated eosinophils, dupilumab reduced exacerbations by 65% and 73%, respectively (P less than .001 vs. placebo).
In patients with early-onset asthma and elevated FeNO, dupilumab reduced severe exacerbations by 56% when given at 200 mg and by 52% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients with elevated FeNO, dupilumab reduced exacerbations by 79% and 71%, respectively (P less than .001 vs. placebo).
Lung function
Dupilumab also improved prebronchodilator forced expiratory volume in 1 second (pre-BD FEV1), compared with placebo, with similar results in early- and late-onset patients.
In early-onset patients, the P values were less than .001 for both doses of dupilumab at weeks 12 and 52. In late-onset patients, the P values were less than .001 for the 300-mg dose at week 12 and the 200-mg dose at week 52, less than .01 for the 200-mg dose at week 12, and less than .05 for the 300-mg dose at week 52.
The effects of dupilumab on pre-BD FEV1 were greatest in patients with elevated eosinophils or FeNO. At week 12, the P value was less than .001 for both doses of dupilumab in early-onset patients with elevated eosinophils or FeNO. The P value was less than .01 for both doses in late-onset patients with elevated eosinophils. And the P value was less than .001 for both doses in late-onset patients with elevated FeNO.
This research was sponsored by Sanofi and Regeneron. Dr. Hanania disclosed relationships with Genentech, Novartis, AstraZeneca, Boehringer Ingelheim, GSK, Regeneron, and Sanofi.
SOURCE: Hanania N et al. CHEST 2019. Abstract, doi: 10.1016/j.chest.2019.08.870.
NEW ORLEANS – A new analysis suggests
Dupilumab may be more effective in reducing severe asthma exacerbations in patients with late-onset asthma, but the drug’s effect on lung function appeared the same regardless of asthma onset. Nicola Hanania, MD, of Baylor College of Medicine in Houston presented these results at the annual meeting of the American College of Chest Physicians.
Dr. Hanania and colleagues conducted a subanalysis of the LIBERTY ASTHMA QUEST study (NCT02414854). Previous data from this study showed that patients with uncontrolled, moderate to severe asthma who received dupilumab had fewer exacerbations and better lung function than did patients who received placebo (N Engl J Med. 2018;378:2486-96).
In their subanalysis, Dr. Hanania and his colleagues evaluated the efficacy of dupilumab, given at 200 mg or 300 mg every 2 weeks, in patients with early-onset asthma (at 40 years of age or younger) and late-onset asthma (at 41 years or older). The analysis included 919 patients with early-onset asthma who received dupilumab and 450 early-onset patients who received placebo. There were 345 patients with late-onset asthma who received dupilumab and 188 late-onset patients who received placebo.
Exacerbations
Dupilumab significantly reduced the adjusted annualized severe exacerbation rates during the 52-week treatment period. Significant reductions occurred in both early- and late-onset patients, though reductions were greater in the late-onset group.
In early-onset patients, dupilumab reduced severe exacerbations by 38% when given at 200 mg and by 37% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients, dupilumab reduced exacerbations by 64% and 69%, respectively (P less than .001 vs. placebo).
Dr. Hanania said it isn’t clear why late-onset patients appear to derive more benefit with regard to exacerbations. It may be because these patients have more comorbidities or because they aren’t using their inhalers correctly. The researchers are investigating these possibilities.
Dr. Hanania went on to note that reductions in exacerbation rates were greatest in patients with elevated blood eosinophils (150 cells/mcL or greater) or fractional exhaled nitric oxide (FeNO; 25 ppb or greater).
In patients with early-onset asthma and elevated eosinophils, dupilumab reduced severe exacerbations by 50% when given at 200 mg and by 55% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients with elevated eosinophils, dupilumab reduced exacerbations by 65% and 73%, respectively (P less than .001 vs. placebo).
In patients with early-onset asthma and elevated FeNO, dupilumab reduced severe exacerbations by 56% when given at 200 mg and by 52% when given at 300 mg (P less than .001 vs. placebo). In late-onset patients with elevated FeNO, dupilumab reduced exacerbations by 79% and 71%, respectively (P less than .001 vs. placebo).
Lung function
Dupilumab also improved prebronchodilator forced expiratory volume in 1 second (pre-BD FEV1), compared with placebo, with similar results in early- and late-onset patients.
In early-onset patients, the P values were less than .001 for both doses of dupilumab at weeks 12 and 52. In late-onset patients, the P values were less than .001 for the 300-mg dose at week 12 and the 200-mg dose at week 52, less than .01 for the 200-mg dose at week 12, and less than .05 for the 300-mg dose at week 52.
The effects of dupilumab on pre-BD FEV1 were greatest in patients with elevated eosinophils or FeNO. At week 12, the P value was less than .001 for both doses of dupilumab in early-onset patients with elevated eosinophils or FeNO. The P value was less than .01 for both doses in late-onset patients with elevated eosinophils. And the P value was less than .001 for both doses in late-onset patients with elevated FeNO.
This research was sponsored by Sanofi and Regeneron. Dr. Hanania disclosed relationships with Genentech, Novartis, AstraZeneca, Boehringer Ingelheim, GSK, Regeneron, and Sanofi.
SOURCE: Hanania N et al. CHEST 2019. Abstract, doi: 10.1016/j.chest.2019.08.870.
REPORTING FROM CHEST 2019
Collaborating in the Care of Spinal Muscular Atrophy: A Multidisciplinary Approach to Timely Screening, Diagnosis, and Management
Learn more about spinal muscular atrophy in this supplement to Pediatric News.
Click here to read.
Supplement Faculty
Claudia A. Chiriboga, MD, MPH, FAAN
(Program Chair)
Professor of Neurology and Pediatrics at CUIMC
Division of Pediatric Neurology
Columbia University Irving Medical Center
New York, NY
Diana P. Castro, MD
Assistant Professor
Pediatrics and Neurology
Neurotherapeutics
UT Southwestern Medical Center
Neurologist
Children’s Health
Dallas, TX
Mary Schroth, MD
Chief Medical Officer
Cure SMA
Elk Grove Village, IL
Charlotte J. Sumner, MD
Professor of Neurology and Neuroscience
Johns Hopkins University School of Medicine
Baltimore, MD
Kathryn J. Swoboda, MD
Katherine B. Sims, MD, Endowed Chair in Neurogenetics
Director, Neurogenetics Program
Mass General Hospital for Children
Boston, MA
Learn more about spinal muscular atrophy in this supplement to Pediatric News.
Click here to read.
Supplement Faculty
Claudia A. Chiriboga, MD, MPH, FAAN
(Program Chair)
Professor of Neurology and Pediatrics at CUIMC
Division of Pediatric Neurology
Columbia University Irving Medical Center
New York, NY
Diana P. Castro, MD
Assistant Professor
Pediatrics and Neurology
Neurotherapeutics
UT Southwestern Medical Center
Neurologist
Children’s Health
Dallas, TX
Mary Schroth, MD
Chief Medical Officer
Cure SMA
Elk Grove Village, IL
Charlotte J. Sumner, MD
Professor of Neurology and Neuroscience
Johns Hopkins University School of Medicine
Baltimore, MD
Kathryn J. Swoboda, MD
Katherine B. Sims, MD, Endowed Chair in Neurogenetics
Director, Neurogenetics Program
Mass General Hospital for Children
Boston, MA
Learn more about spinal muscular atrophy in this supplement to Pediatric News.
Click here to read.
Supplement Faculty
Claudia A. Chiriboga, MD, MPH, FAAN
(Program Chair)
Professor of Neurology and Pediatrics at CUIMC
Division of Pediatric Neurology
Columbia University Irving Medical Center
New York, NY
Diana P. Castro, MD
Assistant Professor
Pediatrics and Neurology
Neurotherapeutics
UT Southwestern Medical Center
Neurologist
Children’s Health
Dallas, TX
Mary Schroth, MD
Chief Medical Officer
Cure SMA
Elk Grove Village, IL
Charlotte J. Sumner, MD
Professor of Neurology and Neuroscience
Johns Hopkins University School of Medicine
Baltimore, MD
Kathryn J. Swoboda, MD
Katherine B. Sims, MD, Endowed Chair in Neurogenetics
Director, Neurogenetics Program
Mass General Hospital for Children
Boston, MA
Spanish risk score predicts 30-day mortality in acute HF in ED patients
Background: The MEESSI-AHF (Multiple Estimation of Risk based on the Emergency Department Spanish Score In patients with Acute Heart Failure) score is a risk-stratification tool that includes systolic blood pressure, age, NT-proBNP, potassium, cardiac troponin T, New York Heart Association class 4 disease, respiratory rate, low-output symptoms, oxygen saturation, episode associated with acute coronary syndrome, signs of left ventricular hypertrophy on EKG, creatinine, and Barthel Index Score. Prior research has shown that it accurately risk-stratified ED patients with AHF in Spain. It has not been studied in other populations.
Study design: Prospective multicenter cohort study.
Setting: Adult ED patients with acute dyspnea in four hospitals in Switzerland.
Synopsis: The study included 1,247 nonhemodialysis patients who presented to the ED with acute dyspnea, were found to have all the necessary variables to calculate the MEESSI-AHF score, and were adjudicated to have acute heart failure. The authors calculated a modified MEESSI-AHF score, excluding the Barthel Index for all patients. The authors found that a six-group modified MEESSI-AHF risk-stratification model could predict 30-day mortality with excellent discrimination (C-Statistic, 0.80). Limitations of the study include the exclusion of all hemodynamically unstable patients and those on hemodialysis.
Bottom line: The MEESSI-AHF score effectively predicts 30-day mortality in AHF in Swiss and Spanish ED patients.
Citation: Wussler D et al. External validation of the MEESSI acute heart failure risk score: A cohort study. Ann Intern Med. 2019;170:248-56.
Dr. Radhakrishnan is a hospitalist at Beth Israel Deaconess Medical Center.
Background: The MEESSI-AHF (Multiple Estimation of Risk based on the Emergency Department Spanish Score In patients with Acute Heart Failure) score is a risk-stratification tool that includes systolic blood pressure, age, NT-proBNP, potassium, cardiac troponin T, New York Heart Association class 4 disease, respiratory rate, low-output symptoms, oxygen saturation, episode associated with acute coronary syndrome, signs of left ventricular hypertrophy on EKG, creatinine, and Barthel Index Score. Prior research has shown that it accurately risk-stratified ED patients with AHF in Spain. It has not been studied in other populations.
Study design: Prospective multicenter cohort study.
Setting: Adult ED patients with acute dyspnea in four hospitals in Switzerland.
Synopsis: The study included 1,247 nonhemodialysis patients who presented to the ED with acute dyspnea, were found to have all the necessary variables to calculate the MEESSI-AHF score, and were adjudicated to have acute heart failure. The authors calculated a modified MEESSI-AHF score, excluding the Barthel Index for all patients. The authors found that a six-group modified MEESSI-AHF risk-stratification model could predict 30-day mortality with excellent discrimination (C-Statistic, 0.80). Limitations of the study include the exclusion of all hemodynamically unstable patients and those on hemodialysis.
Bottom line: The MEESSI-AHF score effectively predicts 30-day mortality in AHF in Swiss and Spanish ED patients.
Citation: Wussler D et al. External validation of the MEESSI acute heart failure risk score: A cohort study. Ann Intern Med. 2019;170:248-56.
Dr. Radhakrishnan is a hospitalist at Beth Israel Deaconess Medical Center.
Background: The MEESSI-AHF (Multiple Estimation of Risk based on the Emergency Department Spanish Score In patients with Acute Heart Failure) score is a risk-stratification tool that includes systolic blood pressure, age, NT-proBNP, potassium, cardiac troponin T, New York Heart Association class 4 disease, respiratory rate, low-output symptoms, oxygen saturation, episode associated with acute coronary syndrome, signs of left ventricular hypertrophy on EKG, creatinine, and Barthel Index Score. Prior research has shown that it accurately risk-stratified ED patients with AHF in Spain. It has not been studied in other populations.
Study design: Prospective multicenter cohort study.
Setting: Adult ED patients with acute dyspnea in four hospitals in Switzerland.
Synopsis: The study included 1,247 nonhemodialysis patients who presented to the ED with acute dyspnea, were found to have all the necessary variables to calculate the MEESSI-AHF score, and were adjudicated to have acute heart failure. The authors calculated a modified MEESSI-AHF score, excluding the Barthel Index for all patients. The authors found that a six-group modified MEESSI-AHF risk-stratification model could predict 30-day mortality with excellent discrimination (C-Statistic, 0.80). Limitations of the study include the exclusion of all hemodynamically unstable patients and those on hemodialysis.
Bottom line: The MEESSI-AHF score effectively predicts 30-day mortality in AHF in Swiss and Spanish ED patients.
Citation: Wussler D et al. External validation of the MEESSI acute heart failure risk score: A cohort study. Ann Intern Med. 2019;170:248-56.
Dr. Radhakrishnan is a hospitalist at Beth Israel Deaconess Medical Center.
Study: Pediatricians support alternative MOC assessment
two recent surveys show.
Lead author Laurel K. Leslie, MD, of Tufts University, Boston, and colleagues analyzed the perceptions of 4,238 pediatricians who participated in a pilot MOC program in 2017 and completed a fourth-quarter survey, a voluntary end-of-year survey, or both surveys. The new MOC option was developed by the American Board of Pediatrics between 2015 and 2016 as an alternative to the current proctored, closed-book general pediatrics (GP) MOC examination. The Maintenance of Certification Assessment for Pediatrics (MOCA-Peds) includes timed, multiple-choice questions delivered quarterly through a Web-based interface, provides resources to help answer questions, and yields immediate feedback, rationale, and references pertaining to answers.
Of the pediatricians surveyed, 93% considered the MOCA-Peds to be a feasible and acceptable alternative to the traditional MOC GP examination, and 89% of pediatricians indicated they would chose the MOCA-Peds model over the proctored examination, according to the study, published in Pediatrics. Of doctors who completed the quarter 4 survey, 88% agreed or strongly agreed that assessment questions aligned with the stated learning objectives, 82% agreed the questions assessed clinical judgment, 82% agreed the questions were relevant to the practice of GP, and 59% agreed the questions were relevant to their specific practice setting, according to the study. For most pediatricians surveyed, the time to answer questions was sufficient (78%).
Regarding potential anxiety associated with MOC, 89% of doctors surveyed agreed that they felt less anxiety participating in the MOCA-Peds compared with the proctored examination, and that any anxiety they felt about the MOCA-Peds lessened during the pilot as their comfort with the assessment grew (81%). When asked whether they would partake in MOCA-Peds in the future, 97% of general pediatricians and 95% of subspecialists planned to participate in MOCA-Peds to maintain their GP certification; 95% of subspecialists said they would participate in MOCA-Peds to maintain their subspecialty certification.
In a second analysis of the same study population led by Adam L. Turner, MPH, of the American Board of Pediatrics, participants were asked whether the MOCA-Peds led to any practice or knowledge changes. Nearly all (98%) respondents reported they had “learned, refreshed, or enhanced their medical knowledge” because of the assessment and 62% of pediatricians reported a practice change associated with pilot participation, according to the study, published in Pediatrics. Of those who made a practice change, 70% were general pediatricians and 41% were subspecialists.
Of the 1,727 pediatricians who positively indicated a practice change, 84% reported details about the specific change made. The most common content domains associated with a practice change included ear, nose, and throat; preventive and well-child care; and mental and behavioral health.
Within the content domains, the three most frequently cited instances in which knowledge or a practice change took place included planning the management of a child with otitis media, managing a child with an acute asthma exacerbation, and planning the management of a child with influenza. Of 400 additional responses related to practice changes beyond answers associated with a specific content domain, the three most common subthemes were using evidence-based medicine, expanding review of materials or resource use, and improving differential diagnosis. Pediatricians reported that participation in MOCA-Peds exposed them to new material and encouraged them to review guidelines or standard care protocols and their application. Respondents also reported increased review of materials or resources in practice, updating the types of resources used, and/or an increased effort to stay more updated.
Dr. Turner and associates concluded that the results “support the notion that assessment and learning can be integrated into a single platform and need not be considered mutually exclusive activities, in turn adding value for practicing physicians.”
The studies were funded by the American Board of Pediatrics Foundation. Dr. Leslie, Dr. Linda Althouse, Mr. Andrew Bradford, and Mr. Adam L. Turner are employees of The American Board of Pediatrics. Dr. Victoria Dounoucos, Dr. Murrey G. Olmsted, and Ms. Amanda C. Smith are employees of RTI International, an international nonprofit research firm with whom the board contracted to conduct this evaluation.
SOURCE: Leslie LK et al. Pediatrics. Nov. 2019 Nov. doi: 10.1542/peds.2019-2303; Turner AL et al. Pediatrics. 2019 Nov. doi: 10.1542/peds.2019-2305.
two recent surveys show.
Lead author Laurel K. Leslie, MD, of Tufts University, Boston, and colleagues analyzed the perceptions of 4,238 pediatricians who participated in a pilot MOC program in 2017 and completed a fourth-quarter survey, a voluntary end-of-year survey, or both surveys. The new MOC option was developed by the American Board of Pediatrics between 2015 and 2016 as an alternative to the current proctored, closed-book general pediatrics (GP) MOC examination. The Maintenance of Certification Assessment for Pediatrics (MOCA-Peds) includes timed, multiple-choice questions delivered quarterly through a Web-based interface, provides resources to help answer questions, and yields immediate feedback, rationale, and references pertaining to answers.
Of the pediatricians surveyed, 93% considered the MOCA-Peds to be a feasible and acceptable alternative to the traditional MOC GP examination, and 89% of pediatricians indicated they would chose the MOCA-Peds model over the proctored examination, according to the study, published in Pediatrics. Of doctors who completed the quarter 4 survey, 88% agreed or strongly agreed that assessment questions aligned with the stated learning objectives, 82% agreed the questions assessed clinical judgment, 82% agreed the questions were relevant to the practice of GP, and 59% agreed the questions were relevant to their specific practice setting, according to the study. For most pediatricians surveyed, the time to answer questions was sufficient (78%).
Regarding potential anxiety associated with MOC, 89% of doctors surveyed agreed that they felt less anxiety participating in the MOCA-Peds compared with the proctored examination, and that any anxiety they felt about the MOCA-Peds lessened during the pilot as their comfort with the assessment grew (81%). When asked whether they would partake in MOCA-Peds in the future, 97% of general pediatricians and 95% of subspecialists planned to participate in MOCA-Peds to maintain their GP certification; 95% of subspecialists said they would participate in MOCA-Peds to maintain their subspecialty certification.
In a second analysis of the same study population led by Adam L. Turner, MPH, of the American Board of Pediatrics, participants were asked whether the MOCA-Peds led to any practice or knowledge changes. Nearly all (98%) respondents reported they had “learned, refreshed, or enhanced their medical knowledge” because of the assessment and 62% of pediatricians reported a practice change associated with pilot participation, according to the study, published in Pediatrics. Of those who made a practice change, 70% were general pediatricians and 41% were subspecialists.
Of the 1,727 pediatricians who positively indicated a practice change, 84% reported details about the specific change made. The most common content domains associated with a practice change included ear, nose, and throat; preventive and well-child care; and mental and behavioral health.
Within the content domains, the three most frequently cited instances in which knowledge or a practice change took place included planning the management of a child with otitis media, managing a child with an acute asthma exacerbation, and planning the management of a child with influenza. Of 400 additional responses related to practice changes beyond answers associated with a specific content domain, the three most common subthemes were using evidence-based medicine, expanding review of materials or resource use, and improving differential diagnosis. Pediatricians reported that participation in MOCA-Peds exposed them to new material and encouraged them to review guidelines or standard care protocols and their application. Respondents also reported increased review of materials or resources in practice, updating the types of resources used, and/or an increased effort to stay more updated.
Dr. Turner and associates concluded that the results “support the notion that assessment and learning can be integrated into a single platform and need not be considered mutually exclusive activities, in turn adding value for practicing physicians.”
The studies were funded by the American Board of Pediatrics Foundation. Dr. Leslie, Dr. Linda Althouse, Mr. Andrew Bradford, and Mr. Adam L. Turner are employees of The American Board of Pediatrics. Dr. Victoria Dounoucos, Dr. Murrey G. Olmsted, and Ms. Amanda C. Smith are employees of RTI International, an international nonprofit research firm with whom the board contracted to conduct this evaluation.
SOURCE: Leslie LK et al. Pediatrics. Nov. 2019 Nov. doi: 10.1542/peds.2019-2303; Turner AL et al. Pediatrics. 2019 Nov. doi: 10.1542/peds.2019-2305.
two recent surveys show.
Lead author Laurel K. Leslie, MD, of Tufts University, Boston, and colleagues analyzed the perceptions of 4,238 pediatricians who participated in a pilot MOC program in 2017 and completed a fourth-quarter survey, a voluntary end-of-year survey, or both surveys. The new MOC option was developed by the American Board of Pediatrics between 2015 and 2016 as an alternative to the current proctored, closed-book general pediatrics (GP) MOC examination. The Maintenance of Certification Assessment for Pediatrics (MOCA-Peds) includes timed, multiple-choice questions delivered quarterly through a Web-based interface, provides resources to help answer questions, and yields immediate feedback, rationale, and references pertaining to answers.
Of the pediatricians surveyed, 93% considered the MOCA-Peds to be a feasible and acceptable alternative to the traditional MOC GP examination, and 89% of pediatricians indicated they would chose the MOCA-Peds model over the proctored examination, according to the study, published in Pediatrics. Of doctors who completed the quarter 4 survey, 88% agreed or strongly agreed that assessment questions aligned with the stated learning objectives, 82% agreed the questions assessed clinical judgment, 82% agreed the questions were relevant to the practice of GP, and 59% agreed the questions were relevant to their specific practice setting, according to the study. For most pediatricians surveyed, the time to answer questions was sufficient (78%).
Regarding potential anxiety associated with MOC, 89% of doctors surveyed agreed that they felt less anxiety participating in the MOCA-Peds compared with the proctored examination, and that any anxiety they felt about the MOCA-Peds lessened during the pilot as their comfort with the assessment grew (81%). When asked whether they would partake in MOCA-Peds in the future, 97% of general pediatricians and 95% of subspecialists planned to participate in MOCA-Peds to maintain their GP certification; 95% of subspecialists said they would participate in MOCA-Peds to maintain their subspecialty certification.
In a second analysis of the same study population led by Adam L. Turner, MPH, of the American Board of Pediatrics, participants were asked whether the MOCA-Peds led to any practice or knowledge changes. Nearly all (98%) respondents reported they had “learned, refreshed, or enhanced their medical knowledge” because of the assessment and 62% of pediatricians reported a practice change associated with pilot participation, according to the study, published in Pediatrics. Of those who made a practice change, 70% were general pediatricians and 41% were subspecialists.
Of the 1,727 pediatricians who positively indicated a practice change, 84% reported details about the specific change made. The most common content domains associated with a practice change included ear, nose, and throat; preventive and well-child care; and mental and behavioral health.
Within the content domains, the three most frequently cited instances in which knowledge or a practice change took place included planning the management of a child with otitis media, managing a child with an acute asthma exacerbation, and planning the management of a child with influenza. Of 400 additional responses related to practice changes beyond answers associated with a specific content domain, the three most common subthemes were using evidence-based medicine, expanding review of materials or resource use, and improving differential diagnosis. Pediatricians reported that participation in MOCA-Peds exposed them to new material and encouraged them to review guidelines or standard care protocols and their application. Respondents also reported increased review of materials or resources in practice, updating the types of resources used, and/or an increased effort to stay more updated.
Dr. Turner and associates concluded that the results “support the notion that assessment and learning can be integrated into a single platform and need not be considered mutually exclusive activities, in turn adding value for practicing physicians.”
The studies were funded by the American Board of Pediatrics Foundation. Dr. Leslie, Dr. Linda Althouse, Mr. Andrew Bradford, and Mr. Adam L. Turner are employees of The American Board of Pediatrics. Dr. Victoria Dounoucos, Dr. Murrey G. Olmsted, and Ms. Amanda C. Smith are employees of RTI International, an international nonprofit research firm with whom the board contracted to conduct this evaluation.
SOURCE: Leslie LK et al. Pediatrics. Nov. 2019 Nov. doi: 10.1542/peds.2019-2303; Turner AL et al. Pediatrics. 2019 Nov. doi: 10.1542/peds.2019-2305.