Corticosteroids in Duchenne muscular dystrophy: Major variations in practice. Issue 1 (25th April 2013)

• Record Type: Journal Article
• Authors: Griggs, Robert C.; Herr, Barbara E.; Reha, Allen; Elfring, Gary; Atkinson, Leone; Cwik, Valerie; Mccoll, Elaine; Tawil, Rabi; Pandya, Shree; McDermott, Michael P.; Bushby, Kate
• Is Part Of: Muscle & nerve. Volume 48:Issue 1(2013:Jul.)
• Page Start: 27
• Page End: 31
• Publication Date: 2013-04-25
• Subjects: Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.23831
• Languages: English
• ISSNs: 0148-639X

Abstract

<abstract abstract-type="main"> <title>ABSTRACT</title> <p>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. Muscle Nerve, 2013</p> </abstract>