VHA warns of a ‘second epidemic’ of carbapenem-resistant E. cloacae complex

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Tue, 12/04/2018 - 13:31

 

Veterans Health Administration monitoring of carbapenem-resistant Enterobacteriaceae (CRE) trends from 2006 to 2015 shows a rise in resistance rates of E. cloacae complex nationwide.

The first major CRE outbreak, Klebsiella pneumoniae, occurred in the eastern United States in the early 2000s and has since spread across the country. K. pneumoniae has recently shown a decrease in resistance rates in the region including New York, both in the current VHA-based study and in a 2016 study of three New York City hospitals.

“CRE trends during 2006-2015 in the VHA recapitulate the epidemic of carbapenem-resistant K. pneumoniae in the United States and indicate that a ‘second epidemic’ of carbapenem-resistant E. cloacae complex appears to be unfolding,” wrote Brigid M. Wilson, PhD, of Louis Stokes Cleveland Department of Veterans Affairs Medical Center, and her coauthors.

The researchers used VHA network data to identify 128,431 K. pneumoniae and 38,219 E. cloacae complex (which refers to the species E. cloacae, E. asburiae, E. kobei, E. hormaechei, and E. xiafangensis) isolates from patients hospitalized in 140 facilities in 40 states, the District of Columbia, and Puerto Rico from 2006 to 2015. These isolates, paired with their carbapenem susceptibility test results, show the rise and geographic concentration of the CRE cases over the decade.

The increased E. cloacae complex resistance in 2014-2015 was centered around the Pacific Coast and Southwest regions. The researchers noted that E. cloacae complex has a less well defined genetic makeup, compared with K. pneumoniae.

“We hypothesize that E. cloacae complex contains genotypes with epidemic potential associated with increasing rates of carbapenem resistance observed in the VHA,” they wrote, concluding that “the VHA may serve as a vantage point for detecting nationwide trends in antimicrobial drug resistance” (Emerg Infect Dis. 2017 Mar. doi: 10.3201/eid2305.162034).

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Veterans Health Administration monitoring of carbapenem-resistant Enterobacteriaceae (CRE) trends from 2006 to 2015 shows a rise in resistance rates of E. cloacae complex nationwide.

The first major CRE outbreak, Klebsiella pneumoniae, occurred in the eastern United States in the early 2000s and has since spread across the country. K. pneumoniae has recently shown a decrease in resistance rates in the region including New York, both in the current VHA-based study and in a 2016 study of three New York City hospitals.

“CRE trends during 2006-2015 in the VHA recapitulate the epidemic of carbapenem-resistant K. pneumoniae in the United States and indicate that a ‘second epidemic’ of carbapenem-resistant E. cloacae complex appears to be unfolding,” wrote Brigid M. Wilson, PhD, of Louis Stokes Cleveland Department of Veterans Affairs Medical Center, and her coauthors.

The researchers used VHA network data to identify 128,431 K. pneumoniae and 38,219 E. cloacae complex (which refers to the species E. cloacae, E. asburiae, E. kobei, E. hormaechei, and E. xiafangensis) isolates from patients hospitalized in 140 facilities in 40 states, the District of Columbia, and Puerto Rico from 2006 to 2015. These isolates, paired with their carbapenem susceptibility test results, show the rise and geographic concentration of the CRE cases over the decade.

The increased E. cloacae complex resistance in 2014-2015 was centered around the Pacific Coast and Southwest regions. The researchers noted that E. cloacae complex has a less well defined genetic makeup, compared with K. pneumoniae.

“We hypothesize that E. cloacae complex contains genotypes with epidemic potential associated with increasing rates of carbapenem resistance observed in the VHA,” they wrote, concluding that “the VHA may serve as a vantage point for detecting nationwide trends in antimicrobial drug resistance” (Emerg Infect Dis. 2017 Mar. doi: 10.3201/eid2305.162034).

 

Veterans Health Administration monitoring of carbapenem-resistant Enterobacteriaceae (CRE) trends from 2006 to 2015 shows a rise in resistance rates of E. cloacae complex nationwide.

The first major CRE outbreak, Klebsiella pneumoniae, occurred in the eastern United States in the early 2000s and has since spread across the country. K. pneumoniae has recently shown a decrease in resistance rates in the region including New York, both in the current VHA-based study and in a 2016 study of three New York City hospitals.

“CRE trends during 2006-2015 in the VHA recapitulate the epidemic of carbapenem-resistant K. pneumoniae in the United States and indicate that a ‘second epidemic’ of carbapenem-resistant E. cloacae complex appears to be unfolding,” wrote Brigid M. Wilson, PhD, of Louis Stokes Cleveland Department of Veterans Affairs Medical Center, and her coauthors.

The researchers used VHA network data to identify 128,431 K. pneumoniae and 38,219 E. cloacae complex (which refers to the species E. cloacae, E. asburiae, E. kobei, E. hormaechei, and E. xiafangensis) isolates from patients hospitalized in 140 facilities in 40 states, the District of Columbia, and Puerto Rico from 2006 to 2015. These isolates, paired with their carbapenem susceptibility test results, show the rise and geographic concentration of the CRE cases over the decade.

The increased E. cloacae complex resistance in 2014-2015 was centered around the Pacific Coast and Southwest regions. The researchers noted that E. cloacae complex has a less well defined genetic makeup, compared with K. pneumoniae.

“We hypothesize that E. cloacae complex contains genotypes with epidemic potential associated with increasing rates of carbapenem resistance observed in the VHA,” they wrote, concluding that “the VHA may serve as a vantage point for detecting nationwide trends in antimicrobial drug resistance” (Emerg Infect Dis. 2017 Mar. doi: 10.3201/eid2305.162034).

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Physical frailty may lengthen late-life depression

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Fri, 01/18/2019 - 16:42

 

Physical frailty negatively affects the course of late-life depression and requires multifaceted interventions, according to a new analysis from a study of depression in seniors.

Researchers in the Netherlands worked with participants from the Netherlands Study of Depression in Older Persons, an ongoing cohort study of people aged 60-93 years who have been diagnosed with a depressive disorder.

From the cohort, the researchers examined 285 participants for a 2-year period with physical tests (such as a walking test and a hand grip strength test) and questionnaires aimed at assessing the course of participants’ depression and their physical health.

“The present study, to our knowledge, is the first to examine the longitudinal association between physical frailty and the course of depression in a sample of clinically depressed older persons,” wrote Rose M. Collard, PhD, of the department of psychiatry at Radboud University Medical Center, Nijmegen, the Netherlands, and her coauthors (Eur Psychiatry. 2017 Jan 24;43:66-72). “Our results confirm that late-life depression is a highly persisting disorder, with half of the patients not achieving remission at 2-year follow-up.”

More “performance-based” frailty was associated with nonremission of depression, while “vitality-based” frailty was associated with remission.

The authors commented that “this latter result puzzled us, but a possible explanation might be that the vitality-based dimension of frailty reflects a classic, uncomplicated clinical depression,” which is more treatable.

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Physical frailty negatively affects the course of late-life depression and requires multifaceted interventions, according to a new analysis from a study of depression in seniors.

Researchers in the Netherlands worked with participants from the Netherlands Study of Depression in Older Persons, an ongoing cohort study of people aged 60-93 years who have been diagnosed with a depressive disorder.

From the cohort, the researchers examined 285 participants for a 2-year period with physical tests (such as a walking test and a hand grip strength test) and questionnaires aimed at assessing the course of participants’ depression and their physical health.

“The present study, to our knowledge, is the first to examine the longitudinal association between physical frailty and the course of depression in a sample of clinically depressed older persons,” wrote Rose M. Collard, PhD, of the department of psychiatry at Radboud University Medical Center, Nijmegen, the Netherlands, and her coauthors (Eur Psychiatry. 2017 Jan 24;43:66-72). “Our results confirm that late-life depression is a highly persisting disorder, with half of the patients not achieving remission at 2-year follow-up.”

More “performance-based” frailty was associated with nonremission of depression, while “vitality-based” frailty was associated with remission.

The authors commented that “this latter result puzzled us, but a possible explanation might be that the vitality-based dimension of frailty reflects a classic, uncomplicated clinical depression,” which is more treatable.

 

Physical frailty negatively affects the course of late-life depression and requires multifaceted interventions, according to a new analysis from a study of depression in seniors.

Researchers in the Netherlands worked with participants from the Netherlands Study of Depression in Older Persons, an ongoing cohort study of people aged 60-93 years who have been diagnosed with a depressive disorder.

From the cohort, the researchers examined 285 participants for a 2-year period with physical tests (such as a walking test and a hand grip strength test) and questionnaires aimed at assessing the course of participants’ depression and their physical health.

“The present study, to our knowledge, is the first to examine the longitudinal association between physical frailty and the course of depression in a sample of clinically depressed older persons,” wrote Rose M. Collard, PhD, of the department of psychiatry at Radboud University Medical Center, Nijmegen, the Netherlands, and her coauthors (Eur Psychiatry. 2017 Jan 24;43:66-72). “Our results confirm that late-life depression is a highly persisting disorder, with half of the patients not achieving remission at 2-year follow-up.”

More “performance-based” frailty was associated with nonremission of depression, while “vitality-based” frailty was associated with remission.

The authors commented that “this latter result puzzled us, but a possible explanation might be that the vitality-based dimension of frailty reflects a classic, uncomplicated clinical depression,” which is more treatable.

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Vaccination reduces risk of flu-associated pediatric deaths

Comment by Dr. Susan Millard, FCCP
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Fri, 01/18/2019 - 16:39

 

Influenza vaccination was associated with reduced risk of laboratory-confirmed influenza-associated death in children, a case-cohort analysis found.

“These results support current recommendations for annual influenza vaccination for all children 6 months of age” and older, wrote Brendan Flannery, PhD, and his coauthors at the Centers for Disease Control and Prevention, Atlanta. “To our knowledge, this is the first study to use laboratory-confirmed outcomes to investigate influenza vaccine effectiveness against influenza-associated deaths.”

A doctor vaccinates a toddler
KatarzynaBialasiewicz/Thinkstock


“Best estimates based on [National Health Interview Survey] data suggested that vaccination reduced the risk of influenza-associated death by half among children with high-risk conditions and by nearly two-thirds among children without high-risk conditions,” Dr. Flannery and his coauthors reported.

Of 358 cases of pediatric death (aged 6 months to 17 years) confirmed to be associated with influenza, 75 (26%) had been vaccinated prior to their disease onset. The case-cohort analysis compared the 358 cases against three cohorts of U.S. children and adolescents: a telephone survey, a household survey, and a health insurance claims database.

The researchers had examined cases that were reported to the U.S. Influenza-Associated Pediatric Mortality Surveillance System from July 2010 to June 2014. They excluded cases of children not yet eligible to be vaccinated or whose disease onset may have occurred before their vaccine had 14 days to take full effect (Pediatrics. 2017 Apr. doi: 10.1542/peds.2016-4244).

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Dr. Susan Millard
Dr. Susan Millard
This information screams at all providers and parents regarding the critical importance of yearly influenza vaccinations for all children 6 months of age and older!

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Dr. Susan Millard
Dr. Susan Millard
This information screams at all providers and parents regarding the critical importance of yearly influenza vaccinations for all children 6 months of age and older!

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Dr. Susan Millard
Dr. Susan Millard
This information screams at all providers and parents regarding the critical importance of yearly influenza vaccinations for all children 6 months of age and older!

Title
Comment by Dr. Susan Millard, FCCP
Comment by Dr. Susan Millard, FCCP

 

Influenza vaccination was associated with reduced risk of laboratory-confirmed influenza-associated death in children, a case-cohort analysis found.

“These results support current recommendations for annual influenza vaccination for all children 6 months of age” and older, wrote Brendan Flannery, PhD, and his coauthors at the Centers for Disease Control and Prevention, Atlanta. “To our knowledge, this is the first study to use laboratory-confirmed outcomes to investigate influenza vaccine effectiveness against influenza-associated deaths.”

A doctor vaccinates a toddler
KatarzynaBialasiewicz/Thinkstock


“Best estimates based on [National Health Interview Survey] data suggested that vaccination reduced the risk of influenza-associated death by half among children with high-risk conditions and by nearly two-thirds among children without high-risk conditions,” Dr. Flannery and his coauthors reported.

Of 358 cases of pediatric death (aged 6 months to 17 years) confirmed to be associated with influenza, 75 (26%) had been vaccinated prior to their disease onset. The case-cohort analysis compared the 358 cases against three cohorts of U.S. children and adolescents: a telephone survey, a household survey, and a health insurance claims database.

The researchers had examined cases that were reported to the U.S. Influenza-Associated Pediatric Mortality Surveillance System from July 2010 to June 2014. They excluded cases of children not yet eligible to be vaccinated or whose disease onset may have occurred before their vaccine had 14 days to take full effect (Pediatrics. 2017 Apr. doi: 10.1542/peds.2016-4244).

 

Influenza vaccination was associated with reduced risk of laboratory-confirmed influenza-associated death in children, a case-cohort analysis found.

“These results support current recommendations for annual influenza vaccination for all children 6 months of age” and older, wrote Brendan Flannery, PhD, and his coauthors at the Centers for Disease Control and Prevention, Atlanta. “To our knowledge, this is the first study to use laboratory-confirmed outcomes to investigate influenza vaccine effectiveness against influenza-associated deaths.”

A doctor vaccinates a toddler
KatarzynaBialasiewicz/Thinkstock


“Best estimates based on [National Health Interview Survey] data suggested that vaccination reduced the risk of influenza-associated death by half among children with high-risk conditions and by nearly two-thirds among children without high-risk conditions,” Dr. Flannery and his coauthors reported.

Of 358 cases of pediatric death (aged 6 months to 17 years) confirmed to be associated with influenza, 75 (26%) had been vaccinated prior to their disease onset. The case-cohort analysis compared the 358 cases against three cohorts of U.S. children and adolescents: a telephone survey, a household survey, and a health insurance claims database.

The researchers had examined cases that were reported to the U.S. Influenza-Associated Pediatric Mortality Surveillance System from July 2010 to June 2014. They excluded cases of children not yet eligible to be vaccinated or whose disease onset may have occurred before their vaccine had 14 days to take full effect (Pediatrics. 2017 Apr. doi: 10.1542/peds.2016-4244).

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FDA grants breakthrough therapy status to rituximab for pemphigus vulgaris

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Fri, 01/18/2019 - 16:39

 

The Food and Drug Administration has granted breakthrough therapy status to rituximab (Rituxan) for treating pemphigus vulgaris, according to the manufacturer.

Rituximab, a CD20-directed cytolytic antibody approved in 1997, is currently in a phase III study evaluating its efficacy for the pemphigus indication. It is approved in the United States for treating non-Hodgkin lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis (with methotrexate), granulomatosis with polyangiitis (Wegener’s granulomatosis), and microscopic polyangiitis (with glucocorticoids).

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The FDA granted breakthrough therapy status based on the results of a prospective, multicenter, open label trial of 90 patients conducted in France evaluating rituximab for pemphigus, according to Genentech. The researchers found that first-line treatment for pemphigus with rituximab and oral prednisone was likely more effective than prednisone alone, with fewer side effects. The study was published in March in the Lancet (doi: 10.1016/S0140-6736[17]30070-3).

The patients, who were experiencing their first episode of pemphigus vulgaris, were randomized to daily oral prednisone, tapered over a 12- to 18-month period, or rituximab administered intravenously (at days 0 and 14, and months 12 and 18), plus daily oral prednisone, tapered over 3 or 6 months. At 2 years, when they were no longer on therapy, 89% of those treated with rituximab and prednisone were in complete remission, compared with 34% of those treated with prednisone alone (P less than .0001).

The breakthrough therapy process is “designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s),” according to the FDA.

The study was supported by the French Ministry of Health, the French Society of Dermatology, and Roche, which owns Genentech. Genentech markets rituximab in the United States with Biogen and is conducting the phase III study.
 

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The Food and Drug Administration has granted breakthrough therapy status to rituximab (Rituxan) for treating pemphigus vulgaris, according to the manufacturer.

Rituximab, a CD20-directed cytolytic antibody approved in 1997, is currently in a phase III study evaluating its efficacy for the pemphigus indication. It is approved in the United States for treating non-Hodgkin lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis (with methotrexate), granulomatosis with polyangiitis (Wegener’s granulomatosis), and microscopic polyangiitis (with glucocorticoids).

FDA icon
The FDA granted breakthrough therapy status based on the results of a prospective, multicenter, open label trial of 90 patients conducted in France evaluating rituximab for pemphigus, according to Genentech. The researchers found that first-line treatment for pemphigus with rituximab and oral prednisone was likely more effective than prednisone alone, with fewer side effects. The study was published in March in the Lancet (doi: 10.1016/S0140-6736[17]30070-3).

The patients, who were experiencing their first episode of pemphigus vulgaris, were randomized to daily oral prednisone, tapered over a 12- to 18-month period, or rituximab administered intravenously (at days 0 and 14, and months 12 and 18), plus daily oral prednisone, tapered over 3 or 6 months. At 2 years, when they were no longer on therapy, 89% of those treated with rituximab and prednisone were in complete remission, compared with 34% of those treated with prednisone alone (P less than .0001).

The breakthrough therapy process is “designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s),” according to the FDA.

The study was supported by the French Ministry of Health, the French Society of Dermatology, and Roche, which owns Genentech. Genentech markets rituximab in the United States with Biogen and is conducting the phase III study.
 

 

The Food and Drug Administration has granted breakthrough therapy status to rituximab (Rituxan) for treating pemphigus vulgaris, according to the manufacturer.

Rituximab, a CD20-directed cytolytic antibody approved in 1997, is currently in a phase III study evaluating its efficacy for the pemphigus indication. It is approved in the United States for treating non-Hodgkin lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis (with methotrexate), granulomatosis with polyangiitis (Wegener’s granulomatosis), and microscopic polyangiitis (with glucocorticoids).

FDA icon
The FDA granted breakthrough therapy status based on the results of a prospective, multicenter, open label trial of 90 patients conducted in France evaluating rituximab for pemphigus, according to Genentech. The researchers found that first-line treatment for pemphigus with rituximab and oral prednisone was likely more effective than prednisone alone, with fewer side effects. The study was published in March in the Lancet (doi: 10.1016/S0140-6736[17]30070-3).

The patients, who were experiencing their first episode of pemphigus vulgaris, were randomized to daily oral prednisone, tapered over a 12- to 18-month period, or rituximab administered intravenously (at days 0 and 14, and months 12 and 18), plus daily oral prednisone, tapered over 3 or 6 months. At 2 years, when they were no longer on therapy, 89% of those treated with rituximab and prednisone were in complete remission, compared with 34% of those treated with prednisone alone (P less than .0001).

The breakthrough therapy process is “designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s),” according to the FDA.

The study was supported by the French Ministry of Health, the French Society of Dermatology, and Roche, which owns Genentech. Genentech markets rituximab in the United States with Biogen and is conducting the phase III study.
 

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Childhood adiposity tied to NAFLD and elevated ALT in adulthood

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Fri, 01/18/2019 - 16:39

 

Overweight or obese children in a cohort study were more likely to have adult nonalcoholic fatty liver disease (NAFLD) and elevated levels of the liver enzyme alanine aminotransferase than were healthy weight children of both sexes, but this association can be mitigated by weight loss in adulthood.

“These findings underscore the importance of both early prevention and lifelong treatment of overweight and obesity to reduce the risk of adverse liver outcome in adulthood,” Yinkun Yan, PhD, an epidemiologist at the Capital Institute of Pediatrics, Beijing, and associates wrote (Pediatrics. 2017;139[4]:e20162738).

Overweight child eating lunch
©SolStock/iStock
The investigators examined data for 1,350 children (aged 6-18 years) from the Beijing Blood Pressure Cohort Study, begun in 1987 and 1988 to learn about precursors of adult cardiovascular diseases. The participants underwent health examinations between 2010 and 2014, at ages 28-45 years, after 23 years of follow-up.

The adiposity of the children was determined by caliper measurements and body mass index. ALT elevation was diagnosed via blood tests and NAFLD from ultrasonography. ALT is considered to be the most specific marker of liver damage, and NAFLD is one of the most common causes of liver disease worldwide.

Children who were overweight or obese were more likely to grow up to have elevated ALT levels (40% vs. 30% in men and 23% vs. 12% in women) or NAFLD (62% vs. 39% in men and 39% vs. 15% in women) than were healthy weight children. Obesity in adulthood was a higher risk whether a participant was obese as a child or not, but the researchers noted that risks could be mitigated by acquiring normal weight in adulthood.

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Overweight or obese children in a cohort study were more likely to have adult nonalcoholic fatty liver disease (NAFLD) and elevated levels of the liver enzyme alanine aminotransferase than were healthy weight children of both sexes, but this association can be mitigated by weight loss in adulthood.

“These findings underscore the importance of both early prevention and lifelong treatment of overweight and obesity to reduce the risk of adverse liver outcome in adulthood,” Yinkun Yan, PhD, an epidemiologist at the Capital Institute of Pediatrics, Beijing, and associates wrote (Pediatrics. 2017;139[4]:e20162738).

Overweight child eating lunch
©SolStock/iStock
The investigators examined data for 1,350 children (aged 6-18 years) from the Beijing Blood Pressure Cohort Study, begun in 1987 and 1988 to learn about precursors of adult cardiovascular diseases. The participants underwent health examinations between 2010 and 2014, at ages 28-45 years, after 23 years of follow-up.

The adiposity of the children was determined by caliper measurements and body mass index. ALT elevation was diagnosed via blood tests and NAFLD from ultrasonography. ALT is considered to be the most specific marker of liver damage, and NAFLD is one of the most common causes of liver disease worldwide.

Children who were overweight or obese were more likely to grow up to have elevated ALT levels (40% vs. 30% in men and 23% vs. 12% in women) or NAFLD (62% vs. 39% in men and 39% vs. 15% in women) than were healthy weight children. Obesity in adulthood was a higher risk whether a participant was obese as a child or not, but the researchers noted that risks could be mitigated by acquiring normal weight in adulthood.

 

Overweight or obese children in a cohort study were more likely to have adult nonalcoholic fatty liver disease (NAFLD) and elevated levels of the liver enzyme alanine aminotransferase than were healthy weight children of both sexes, but this association can be mitigated by weight loss in adulthood.

“These findings underscore the importance of both early prevention and lifelong treatment of overweight and obesity to reduce the risk of adverse liver outcome in adulthood,” Yinkun Yan, PhD, an epidemiologist at the Capital Institute of Pediatrics, Beijing, and associates wrote (Pediatrics. 2017;139[4]:e20162738).

Overweight child eating lunch
©SolStock/iStock
The investigators examined data for 1,350 children (aged 6-18 years) from the Beijing Blood Pressure Cohort Study, begun in 1987 and 1988 to learn about precursors of adult cardiovascular diseases. The participants underwent health examinations between 2010 and 2014, at ages 28-45 years, after 23 years of follow-up.

The adiposity of the children was determined by caliper measurements and body mass index. ALT elevation was diagnosed via blood tests and NAFLD from ultrasonography. ALT is considered to be the most specific marker of liver damage, and NAFLD is one of the most common causes of liver disease worldwide.

Children who were overweight or obese were more likely to grow up to have elevated ALT levels (40% vs. 30% in men and 23% vs. 12% in women) or NAFLD (62% vs. 39% in men and 39% vs. 15% in women) than were healthy weight children. Obesity in adulthood was a higher risk whether a participant was obese as a child or not, but the researchers noted that risks could be mitigated by acquiring normal weight in adulthood.

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Rotavirus vaccine and PCV reduce hospital burden of young children

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Fri, 01/18/2019 - 16:35

 

Vaccination programs targeting rotavirus and pneumonia in children younger than 2 years both contributed to a “rapid and considerable” decline in the hospital burden of pediatric patients, both in relation to those diseases and overall, according to an observational study.

Three vaccines were added to the National Immunization Plan in Israel within a 1.5-year interval, between July 2009 and January 2011: rotavirus vaccine and the 7-valent and 13-valent pneumococcal conjugate vaccines (PCV). Researchers studied the population at the Soroka University Medical Center in Beer Sheva, Israel, which was split roughly 50/50 between Jewish children and Bedouin Muslim children.

©Rawpixel Ltd/Thinkstock
“The socioeconomic conditions and lifestyles of the two populations differ and social contacts between them, especially between children, are uncommon. However, both have access to the same medical services,” wrote Shalom Ben-Shimol, MD, of Ben-Gurion University of the Negev, Beer Sheva, and coauthors (J Pediatr. 2017 Mar;182:253-9.e2).

The rates of rotavirus gastroenteritis, nonrotavirus gastroenteritis, alveolar pneumonia, and nonalveolar lower respiratory tract infections in the 37,591 hospitalized children younger than 2 years declined by 78%, 21%, 46%, and 7%, respectively, over the course of the study period. Outpatient ED visits for the same diseases declined 80%, 16%, 67%, and 13%, respectively.

The results are more evidence that rotavirus vaccine can help prevent diarrhea not caused by rotavirus and, similarly, that PCV can help prevent lower respiratory tract infections not caused by pneumococci.

Overall, hospitalizations and outpatient ED visits also declined significantly, by 11% and 12%, respectively.

“The impact of [rotavirus vaccine] and PCV may not be limited to prevention of diarrhea and respiratory disease, respectively. In one study, it was suggested that diarrhea may increase the risk of subsequent pneumonia in young children, pointing to potential synergistic benefits” of the vaccines, the authors wrote (Am J Epidemiol. 2005;162[10]:999-1007).

The study was supported by Merck Sharp & Dohme and Pfizer. Authors received speaker fees, research support, and consulting fees from those companies and from GlaxoSmithKline.

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Vaccination programs targeting rotavirus and pneumonia in children younger than 2 years both contributed to a “rapid and considerable” decline in the hospital burden of pediatric patients, both in relation to those diseases and overall, according to an observational study.

Three vaccines were added to the National Immunization Plan in Israel within a 1.5-year interval, between July 2009 and January 2011: rotavirus vaccine and the 7-valent and 13-valent pneumococcal conjugate vaccines (PCV). Researchers studied the population at the Soroka University Medical Center in Beer Sheva, Israel, which was split roughly 50/50 between Jewish children and Bedouin Muslim children.

©Rawpixel Ltd/Thinkstock
“The socioeconomic conditions and lifestyles of the two populations differ and social contacts between them, especially between children, are uncommon. However, both have access to the same medical services,” wrote Shalom Ben-Shimol, MD, of Ben-Gurion University of the Negev, Beer Sheva, and coauthors (J Pediatr. 2017 Mar;182:253-9.e2).

The rates of rotavirus gastroenteritis, nonrotavirus gastroenteritis, alveolar pneumonia, and nonalveolar lower respiratory tract infections in the 37,591 hospitalized children younger than 2 years declined by 78%, 21%, 46%, and 7%, respectively, over the course of the study period. Outpatient ED visits for the same diseases declined 80%, 16%, 67%, and 13%, respectively.

The results are more evidence that rotavirus vaccine can help prevent diarrhea not caused by rotavirus and, similarly, that PCV can help prevent lower respiratory tract infections not caused by pneumococci.

Overall, hospitalizations and outpatient ED visits also declined significantly, by 11% and 12%, respectively.

“The impact of [rotavirus vaccine] and PCV may not be limited to prevention of diarrhea and respiratory disease, respectively. In one study, it was suggested that diarrhea may increase the risk of subsequent pneumonia in young children, pointing to potential synergistic benefits” of the vaccines, the authors wrote (Am J Epidemiol. 2005;162[10]:999-1007).

The study was supported by Merck Sharp & Dohme and Pfizer. Authors received speaker fees, research support, and consulting fees from those companies and from GlaxoSmithKline.

 

Vaccination programs targeting rotavirus and pneumonia in children younger than 2 years both contributed to a “rapid and considerable” decline in the hospital burden of pediatric patients, both in relation to those diseases and overall, according to an observational study.

Three vaccines were added to the National Immunization Plan in Israel within a 1.5-year interval, between July 2009 and January 2011: rotavirus vaccine and the 7-valent and 13-valent pneumococcal conjugate vaccines (PCV). Researchers studied the population at the Soroka University Medical Center in Beer Sheva, Israel, which was split roughly 50/50 between Jewish children and Bedouin Muslim children.

©Rawpixel Ltd/Thinkstock
“The socioeconomic conditions and lifestyles of the two populations differ and social contacts between them, especially between children, are uncommon. However, both have access to the same medical services,” wrote Shalom Ben-Shimol, MD, of Ben-Gurion University of the Negev, Beer Sheva, and coauthors (J Pediatr. 2017 Mar;182:253-9.e2).

The rates of rotavirus gastroenteritis, nonrotavirus gastroenteritis, alveolar pneumonia, and nonalveolar lower respiratory tract infections in the 37,591 hospitalized children younger than 2 years declined by 78%, 21%, 46%, and 7%, respectively, over the course of the study period. Outpatient ED visits for the same diseases declined 80%, 16%, 67%, and 13%, respectively.

The results are more evidence that rotavirus vaccine can help prevent diarrhea not caused by rotavirus and, similarly, that PCV can help prevent lower respiratory tract infections not caused by pneumococci.

Overall, hospitalizations and outpatient ED visits also declined significantly, by 11% and 12%, respectively.

“The impact of [rotavirus vaccine] and PCV may not be limited to prevention of diarrhea and respiratory disease, respectively. In one study, it was suggested that diarrhea may increase the risk of subsequent pneumonia in young children, pointing to potential synergistic benefits” of the vaccines, the authors wrote (Am J Epidemiol. 2005;162[10]:999-1007).

The study was supported by Merck Sharp & Dohme and Pfizer. Authors received speaker fees, research support, and consulting fees from those companies and from GlaxoSmithKline.

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FROM THE JOURNAL OF PEDIATRICS

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Key clinical point: Vaccination programs targeting rotavirus and pneumonia in children younger than 2 years both contributed to a “rapid and considerable” decline in the hospital burden of pediatric patients.

Major finding: The rates of rotavirus gastroenteritis, nonrotavirus gastroenteritis, alveolar pneumonia, and nonalveolar lower respiratory tract infections in hospitalized children younger than 2 years declined by 78%, 21%, 46%, and 7%, respectively, over the course of the study period.

Data source: A prospective, population-based observational study of one hospital in southern Israel.

Disclosures: The study was supported by Merck Sharp & Dohme and Pfizer. Authors received speaker fees, research support, and consulting fees from those companies and from GlaxoSmithKline.

Tissue, peripheral eosinophilia correlate with UC activity, severity

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Fri, 01/18/2019 - 16:34

 

Evidence linking tissue and peripheral eosinophilia with ulcerative colitis (UC) activity and severity was found in a retrospective chart review of pediatric UC cases.

Further, the review found both types of eosinophilia linked with the need for step-up therapy or corticosteroid therapy in the first year following UC diagnosis.

Sara Morgenstern, MD, of Tel Aviv University, and her coauthors reviewed all pediatric UC cases diagnosed between ages 0 and 17 years at the Schneider Children’s Hospital of Israel, Petah Tikva, between 1990 and 2015. Of 96 children diagnosed with UC by colonoscopy and followed for a median of 13 years, 31 had severe eosinophilia at the time of diagnosis, compared with 40 who had mild eosinophilia, and 25 who had a normal tissue eosinophil count. After remission, 77 had a normal eosinophilia and 19 had mild eosinophilia.

“At diagnosis, at follow-up with histologic activity and at follow-up with histologic remission, peripheral eosinophilia was demonstrated in 27%, 30% and 8%, respectively,” Dr. Morgenstern and her coauthors wrote. In the control group, 5% had peripheral eosinophilia, a significant difference (Dig Liver Dis. 2017 Feb;49[2]:170-4).

Disease activity and severity, as measured using the Pediatric UC Activity Index score, correlated significantly with tissue and blood eosinophil counts at diagnosis (P = .02 and P = .01, respectively). Disease activity and severity also correlated significantly with corticosteroid therapy, immunomodulatory therapy, and biologic therapy during the first year following diagnosis (P = .018, .04, and .05 for tissue eosinophilia; P = .013, .01, and .04 for peripheral eosinophilia, respectively).

“These findings may suggest that both tissue and peripheral eosinophilia may serve as a diagnostic marker for disease activity, severity, and short-term outcomes also in the pediatric population,” they wrote.

Dr. Morgenstern and her coauthors had no relevant financial disclosures.

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Evidence linking tissue and peripheral eosinophilia with ulcerative colitis (UC) activity and severity was found in a retrospective chart review of pediatric UC cases.

Further, the review found both types of eosinophilia linked with the need for step-up therapy or corticosteroid therapy in the first year following UC diagnosis.

Sara Morgenstern, MD, of Tel Aviv University, and her coauthors reviewed all pediatric UC cases diagnosed between ages 0 and 17 years at the Schneider Children’s Hospital of Israel, Petah Tikva, between 1990 and 2015. Of 96 children diagnosed with UC by colonoscopy and followed for a median of 13 years, 31 had severe eosinophilia at the time of diagnosis, compared with 40 who had mild eosinophilia, and 25 who had a normal tissue eosinophil count. After remission, 77 had a normal eosinophilia and 19 had mild eosinophilia.

“At diagnosis, at follow-up with histologic activity and at follow-up with histologic remission, peripheral eosinophilia was demonstrated in 27%, 30% and 8%, respectively,” Dr. Morgenstern and her coauthors wrote. In the control group, 5% had peripheral eosinophilia, a significant difference (Dig Liver Dis. 2017 Feb;49[2]:170-4).

Disease activity and severity, as measured using the Pediatric UC Activity Index score, correlated significantly with tissue and blood eosinophil counts at diagnosis (P = .02 and P = .01, respectively). Disease activity and severity also correlated significantly with corticosteroid therapy, immunomodulatory therapy, and biologic therapy during the first year following diagnosis (P = .018, .04, and .05 for tissue eosinophilia; P = .013, .01, and .04 for peripheral eosinophilia, respectively).

“These findings may suggest that both tissue and peripheral eosinophilia may serve as a diagnostic marker for disease activity, severity, and short-term outcomes also in the pediatric population,” they wrote.

Dr. Morgenstern and her coauthors had no relevant financial disclosures.

 

Evidence linking tissue and peripheral eosinophilia with ulcerative colitis (UC) activity and severity was found in a retrospective chart review of pediatric UC cases.

Further, the review found both types of eosinophilia linked with the need for step-up therapy or corticosteroid therapy in the first year following UC diagnosis.

Sara Morgenstern, MD, of Tel Aviv University, and her coauthors reviewed all pediatric UC cases diagnosed between ages 0 and 17 years at the Schneider Children’s Hospital of Israel, Petah Tikva, between 1990 and 2015. Of 96 children diagnosed with UC by colonoscopy and followed for a median of 13 years, 31 had severe eosinophilia at the time of diagnosis, compared with 40 who had mild eosinophilia, and 25 who had a normal tissue eosinophil count. After remission, 77 had a normal eosinophilia and 19 had mild eosinophilia.

“At diagnosis, at follow-up with histologic activity and at follow-up with histologic remission, peripheral eosinophilia was demonstrated in 27%, 30% and 8%, respectively,” Dr. Morgenstern and her coauthors wrote. In the control group, 5% had peripheral eosinophilia, a significant difference (Dig Liver Dis. 2017 Feb;49[2]:170-4).

Disease activity and severity, as measured using the Pediatric UC Activity Index score, correlated significantly with tissue and blood eosinophil counts at diagnosis (P = .02 and P = .01, respectively). Disease activity and severity also correlated significantly with corticosteroid therapy, immunomodulatory therapy, and biologic therapy during the first year following diagnosis (P = .018, .04, and .05 for tissue eosinophilia; P = .013, .01, and .04 for peripheral eosinophilia, respectively).

“These findings may suggest that both tissue and peripheral eosinophilia may serve as a diagnostic marker for disease activity, severity, and short-term outcomes also in the pediatric population,” they wrote.

Dr. Morgenstern and her coauthors had no relevant financial disclosures.

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FROM DIGESTIVE AND LIVER DISEASE

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Key clinical point: Tissue and peripheral eosinophilia may serve as a diagnostic marker for disease activity, severity, and short-term outcomes in pediatric ulcerative colitis.

Major finding: At diagnosis, at follow-up with histologic activity, and at follow-up with histologic remission, peripheral eosinophilia was demonstrated in 27%, 30%, and 8%, respectively.

Data source: Ninety-six children diagnosed with UC by colonoscopy.

Disclosures: Dr. Morgenstern and her coauthors had no relevant financial disclosures.

Increased schizophrenia, affective disorder risks associated with infections

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Fri, 01/18/2019 - 16:33

 

Increased risks of schizophrenia and affective disorders were found to be associated with infections treated with anti-infective agents and with infections requiring hospitalization, a large-scale study shows.

Researchers examined health records of all 1,015,447 individuals born in Denmark from 1985 to 2002, their history of treatment of infection, and the risk of schizophrenia and affective disorders.

Infections previously have been shown to be associated with increased risks of mental disorders. The goal of the current study was to investigate whether the use of anti-infective agents in primary care settings had a similar association.

And the researchers did find such an association: an increased risk of schizophrenia (hazard ratio, 1.37; 95% confidence interval, 1.2-1.57) and an increased risk of affective disorders (HR, 1.64; 95% CI, 1.48-1.82) associated with the use of anti-infective agents.

“The excess risk was primarily driven by infections treated with antibiotics, whereas infections treated with antivirals, antimycotics, and antiparasitic agents were not significant after mutual adjustment,” wrote Ole Köhler, MD, of Aarhus University Hospital, Risskov, Denmark, and his associates (Acta Psychiatr Scand. 2017 Feb;135[2]:97-105).

An even higher risk of schizophrenia and affective disorders was found to be associated with individuals with infections requiring hospitalization (HR, 2.05; 95% CI, 1.77-2.38; and HR, 2.59; 95% CI, 2.31-2.89; respectively). Find more details about the study here.

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Increased risks of schizophrenia and affective disorders were found to be associated with infections treated with anti-infective agents and with infections requiring hospitalization, a large-scale study shows.

Researchers examined health records of all 1,015,447 individuals born in Denmark from 1985 to 2002, their history of treatment of infection, and the risk of schizophrenia and affective disorders.

Infections previously have been shown to be associated with increased risks of mental disorders. The goal of the current study was to investigate whether the use of anti-infective agents in primary care settings had a similar association.

And the researchers did find such an association: an increased risk of schizophrenia (hazard ratio, 1.37; 95% confidence interval, 1.2-1.57) and an increased risk of affective disorders (HR, 1.64; 95% CI, 1.48-1.82) associated with the use of anti-infective agents.

“The excess risk was primarily driven by infections treated with antibiotics, whereas infections treated with antivirals, antimycotics, and antiparasitic agents were not significant after mutual adjustment,” wrote Ole Köhler, MD, of Aarhus University Hospital, Risskov, Denmark, and his associates (Acta Psychiatr Scand. 2017 Feb;135[2]:97-105).

An even higher risk of schizophrenia and affective disorders was found to be associated with individuals with infections requiring hospitalization (HR, 2.05; 95% CI, 1.77-2.38; and HR, 2.59; 95% CI, 2.31-2.89; respectively). Find more details about the study here.

 

Increased risks of schizophrenia and affective disorders were found to be associated with infections treated with anti-infective agents and with infections requiring hospitalization, a large-scale study shows.

Researchers examined health records of all 1,015,447 individuals born in Denmark from 1985 to 2002, their history of treatment of infection, and the risk of schizophrenia and affective disorders.

Infections previously have been shown to be associated with increased risks of mental disorders. The goal of the current study was to investigate whether the use of anti-infective agents in primary care settings had a similar association.

And the researchers did find such an association: an increased risk of schizophrenia (hazard ratio, 1.37; 95% confidence interval, 1.2-1.57) and an increased risk of affective disorders (HR, 1.64; 95% CI, 1.48-1.82) associated with the use of anti-infective agents.

“The excess risk was primarily driven by infections treated with antibiotics, whereas infections treated with antivirals, antimycotics, and antiparasitic agents were not significant after mutual adjustment,” wrote Ole Köhler, MD, of Aarhus University Hospital, Risskov, Denmark, and his associates (Acta Psychiatr Scand. 2017 Feb;135[2]:97-105).

An even higher risk of schizophrenia and affective disorders was found to be associated with individuals with infections requiring hospitalization (HR, 2.05; 95% CI, 1.77-2.38; and HR, 2.59; 95% CI, 2.31-2.89; respectively). Find more details about the study here.

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ERCP testing guidelines accurate, but could be improved

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Sat, 12/08/2018 - 03:19

 

Though current American Society for Gastrointestinal Endoscopy guidelines for whether a patient suspected of choledocholithiasis should undergo endoscopic retrograde cholangiopancreatography (ERCP) are accurate, more restrictive criteria would improve specificity and positive predictive value, researchers have determined.

ERCP is a highly effective procedure but is associated with serious, potentially life-threatening risks. Researchers also noted that “adverse events associated with ERCP are a frequent cause of litigation against gastroenterologists,” highlighting the need for decisions driven by data rather than expert opinion (Gastrointest Endosc. 2017 Feb 4. doi: 10.1016/j.gie.2017.01.039).

Society guidelines recommend less invasive testing – such as baseline liver tests and abdominal ultrasound – prior to ERCP to rule out patients who do not need the riskier procedure. From there, the guidelines stratify patients by high, intermediate, or low risk, recommending higher-risk patients (greater than 50% probability of choledocholithiasis) to proceed to ERCP, and lower-risk patients to take more tests first.

Researchers at Sir Run Run Shaw Hospital, Hangzhou, China, studied 2,724 patients with suspected choledocholithiasis. The patients received biochemical testing, abdominal ultrasound, and definitive testing.

The researchers found that the American Society for Gastrointestinal Endoscopy high-risk criteria were able to provide greater than 50% probability that a patient had choledocholithiasis, but that the criteria would still lead to more than a third of patients undergoing unnecessary ERCP.

Two of the tests – abdominal ultrasound, and determining the level of bilirubin to be greater than 4 mg/dL combined with a dilated common bile duct – provided greater specificity and positive predictive value than did the broader society guidelines. Patients who are negative for both of these tests, the researchers recommend, should receive a less invasive magnetic resonance cholangiopancreatography prior to ERCP.

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Though current American Society for Gastrointestinal Endoscopy guidelines for whether a patient suspected of choledocholithiasis should undergo endoscopic retrograde cholangiopancreatography (ERCP) are accurate, more restrictive criteria would improve specificity and positive predictive value, researchers have determined.

ERCP is a highly effective procedure but is associated with serious, potentially life-threatening risks. Researchers also noted that “adverse events associated with ERCP are a frequent cause of litigation against gastroenterologists,” highlighting the need for decisions driven by data rather than expert opinion (Gastrointest Endosc. 2017 Feb 4. doi: 10.1016/j.gie.2017.01.039).

Society guidelines recommend less invasive testing – such as baseline liver tests and abdominal ultrasound – prior to ERCP to rule out patients who do not need the riskier procedure. From there, the guidelines stratify patients by high, intermediate, or low risk, recommending higher-risk patients (greater than 50% probability of choledocholithiasis) to proceed to ERCP, and lower-risk patients to take more tests first.

Researchers at Sir Run Run Shaw Hospital, Hangzhou, China, studied 2,724 patients with suspected choledocholithiasis. The patients received biochemical testing, abdominal ultrasound, and definitive testing.

The researchers found that the American Society for Gastrointestinal Endoscopy high-risk criteria were able to provide greater than 50% probability that a patient had choledocholithiasis, but that the criteria would still lead to more than a third of patients undergoing unnecessary ERCP.

Two of the tests – abdominal ultrasound, and determining the level of bilirubin to be greater than 4 mg/dL combined with a dilated common bile duct – provided greater specificity and positive predictive value than did the broader society guidelines. Patients who are negative for both of these tests, the researchers recommend, should receive a less invasive magnetic resonance cholangiopancreatography prior to ERCP.

 

Though current American Society for Gastrointestinal Endoscopy guidelines for whether a patient suspected of choledocholithiasis should undergo endoscopic retrograde cholangiopancreatography (ERCP) are accurate, more restrictive criteria would improve specificity and positive predictive value, researchers have determined.

ERCP is a highly effective procedure but is associated with serious, potentially life-threatening risks. Researchers also noted that “adverse events associated with ERCP are a frequent cause of litigation against gastroenterologists,” highlighting the need for decisions driven by data rather than expert opinion (Gastrointest Endosc. 2017 Feb 4. doi: 10.1016/j.gie.2017.01.039).

Society guidelines recommend less invasive testing – such as baseline liver tests and abdominal ultrasound – prior to ERCP to rule out patients who do not need the riskier procedure. From there, the guidelines stratify patients by high, intermediate, or low risk, recommending higher-risk patients (greater than 50% probability of choledocholithiasis) to proceed to ERCP, and lower-risk patients to take more tests first.

Researchers at Sir Run Run Shaw Hospital, Hangzhou, China, studied 2,724 patients with suspected choledocholithiasis. The patients received biochemical testing, abdominal ultrasound, and definitive testing.

The researchers found that the American Society for Gastrointestinal Endoscopy high-risk criteria were able to provide greater than 50% probability that a patient had choledocholithiasis, but that the criteria would still lead to more than a third of patients undergoing unnecessary ERCP.

Two of the tests – abdominal ultrasound, and determining the level of bilirubin to be greater than 4 mg/dL combined with a dilated common bile duct – provided greater specificity and positive predictive value than did the broader society guidelines. Patients who are negative for both of these tests, the researchers recommend, should receive a less invasive magnetic resonance cholangiopancreatography prior to ERCP.

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FROM GASTROINTESTINAL ENDOSCOPY

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FDA approves Emflaza for Duchenne muscular dystrophy

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Fri, 01/18/2019 - 16:31

 

Emflaza, a tablet and oral suspension corticosteroid, has been approved by the Food and Drug Administration for the treatment of Duchenne muscular dystrophy in patients aged 5 years and older.

The agency’s Feb. 9 announcement notes that similar corticosteroids have been used around the world to treat Duchenne muscular dystrophy (DMD), but this is the first to gain approval in the United States. Emflaza (deflazacort) works by decreasing inflammation and immune system activity.

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The drug received fast track status, priority review, and orphan drug designation from the FDA. It will be marketed in the United States by Marathon Pharmaceuticals.

DMD is the most common form of muscular dystrophy but is still rare, occurring in about 1 in 3,600 male infants worldwide. One study found that patients taking deflazacort had some improvements in muscle strength at 12 weeks, compared with those taking placebo, and maintained muscle strength stability through 52 weeks. A longer-term study showed that patients who took deflazacort had better average muscle strength than did those taking placebo and suggested that deflazacort helped prolong patients’ ability to walk.

Side effects experienced by patients taking Emflaza are similar to those associated with other corticosteroids, such as facial puffiness (cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism), and excessive fat around the stomach (central obesity).

In the FDA’s announcement, Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said, “We hope that this treatment option will benefit many patients with DMD.”

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Emflaza, a tablet and oral suspension corticosteroid, has been approved by the Food and Drug Administration for the treatment of Duchenne muscular dystrophy in patients aged 5 years and older.

The agency’s Feb. 9 announcement notes that similar corticosteroids have been used around the world to treat Duchenne muscular dystrophy (DMD), but this is the first to gain approval in the United States. Emflaza (deflazacort) works by decreasing inflammation and immune system activity.

FDA icon
The drug received fast track status, priority review, and orphan drug designation from the FDA. It will be marketed in the United States by Marathon Pharmaceuticals.

DMD is the most common form of muscular dystrophy but is still rare, occurring in about 1 in 3,600 male infants worldwide. One study found that patients taking deflazacort had some improvements in muscle strength at 12 weeks, compared with those taking placebo, and maintained muscle strength stability through 52 weeks. A longer-term study showed that patients who took deflazacort had better average muscle strength than did those taking placebo and suggested that deflazacort helped prolong patients’ ability to walk.

Side effects experienced by patients taking Emflaza are similar to those associated with other corticosteroids, such as facial puffiness (cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism), and excessive fat around the stomach (central obesity).

In the FDA’s announcement, Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said, “We hope that this treatment option will benefit many patients with DMD.”

 

Emflaza, a tablet and oral suspension corticosteroid, has been approved by the Food and Drug Administration for the treatment of Duchenne muscular dystrophy in patients aged 5 years and older.

The agency’s Feb. 9 announcement notes that similar corticosteroids have been used around the world to treat Duchenne muscular dystrophy (DMD), but this is the first to gain approval in the United States. Emflaza (deflazacort) works by decreasing inflammation and immune system activity.

FDA icon
The drug received fast track status, priority review, and orphan drug designation from the FDA. It will be marketed in the United States by Marathon Pharmaceuticals.

DMD is the most common form of muscular dystrophy but is still rare, occurring in about 1 in 3,600 male infants worldwide. One study found that patients taking deflazacort had some improvements in muscle strength at 12 weeks, compared with those taking placebo, and maintained muscle strength stability through 52 weeks. A longer-term study showed that patients who took deflazacort had better average muscle strength than did those taking placebo and suggested that deflazacort helped prolong patients’ ability to walk.

Side effects experienced by patients taking Emflaza are similar to those associated with other corticosteroids, such as facial puffiness (cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism), and excessive fat around the stomach (central obesity).

In the FDA’s announcement, Billy Dunn, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said, “We hope that this treatment option will benefit many patients with DMD.”

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