INTRODUCTION: In 2004, a Cochrane Review and AAN practice parameter concluded that prednisone 0.75 mg/kg/day is of short-term efficacy in Duchenne muscular dystrophy (DMD). Subsequent efforts to standardize care for DMD indicated wide variation in corticosteroid use. METHODS: We surveyed physicians who follow patients with DMD, including: (1) clinics in the TREAT-NMD (Translational Research in Europe-Assessment and Treatment of Neuromuscular Diseases) network (predominantly Europe) and (2) U.S. MDA clinic directors. We also documented the co-administered corticosteroids in a trial of a putative treatment (ataluren) for DMD. RESULTS: Of 105 Treat-NMD clinicians, corticosteroids were not used in 10 clinics, and 29 different regimens were used--the most frequent 0.75 mg/kg/day prednisone (61 centers); 10 days on/10 days off (36 centers); 0.9 mg/kg/day deflazacort (32 centers); and 5 mg/kg/day on weekends (10 centers). Similar diversity was identified in MDA clinics and in the ataluren trial. CONCLUSIONS: Variability in corticosteroid use suggests uncertainty about risks/benefits of corticosteroid regimens for DMD.
INTRODUCTION: In 2004, a Cochrane Review and AAN practice parameter concluded that prednisone 0.75 mg/kg/day is of short-term efficacy in Duchenne muscular dystrophy (DMD). Subsequent efforts to standardize care for DMD indicated wide variation in corticosteroid use. METHODS: We surveyed physicians who follow patients with DMD, including: (1) clinics in the TREAT-NMD (Translational Research in Europe-Assessment and Treatment of Neuromuscular Diseases) network (predominantly Europe) and (2) U.S. MDA clinic directors. We also documented the co-administered corticosteroids in a trial of a putative treatment (ataluren) for DMD. RESULTS: Of 105 Treat-NMD clinicians, corticosteroids were not used in 10 clinics, and 29 different regimens were used--the most frequent 0.75 mg/kg/day prednisone (61 centers); 10 days on/10 days off (36 centers); 0.9 mg/kg/day deflazacort (32 centers); and 5 mg/kg/day on weekends (10 centers). Similar diversity was identified in MDA clinics and in the ataluren trial. CONCLUSIONS: Variability in corticosteroid use suggests uncertainty about risks/benefits of corticosteroid regimens for DMD.
Authors: Pushpa Narayanaswami; Richard Dubinsky; David Wang; Gina Gjorvad; William David; Jonathan Finder; Benn Smith; Jianguo Cheng; Frederic Shapiro; Michelle Mellion; Christopher Spurney; Jodi Wolff; John England Journal: Neurology Date: 2015-09-08 Impact factor: 9.910
Authors: Luca Bello; Heather Gordish-Dressman; Lauren P Morgenroth; Erik K Henricson; Tina Duong; Eric P Hoffman; Avital Cnaan; Craig M McDonald Journal: Neurology Date: 2015-08-26 Impact factor: 9.910
Authors: Maria C Vila; James S Novak; Margaret Benny Klimek; Ning Li; Melissa Morales; Alexander G Fritz; Katie Edwards; Jessica F Boehler; Marshall W Hogarth; Travis B Kinder; Aiping Zhang; Davi Mazala; Alyson A Fiorillo; Bonnie Douglas; Yi-Wen Chen; John van den Anker; Qi L Lu; Yetrib Hathout; Eric P Hoffman; Terence A Partridge; Kanneboyina Nagaraju Journal: J Pathol Date: 2019-04-16 Impact factor: 7.996
Authors: David J Birnkrant; Katharine Bushby; Carla M Bann; Susan D Apkon; Angela Blackwell; David Brumbaugh; Laura E Case; Paula R Clemens; Stasia Hadjiyannakis; Shree Pandya; Natalie Street; Jean Tomezsko; Kathryn R Wagner; Leanne M Ward; David R Weber Journal: Lancet Neurol Date: 2018-02-03 Impact factor: 44.182