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Corticosteroids in Duchenne muscular dystrophy: Major variations in practice

Lookup NU author(s): Emerita Professor Elaine McCollORCiD, Emerita Professor Katherine Bushby


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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 EuropeAssessment 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 usedthe 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

Publication metadata

Author(s): Griggs RC, Herr BE, Reha A, Elfring G, Atkinson L, Cwik V, McColl E, Tawil R, Pandya S, McDermott MP, Bushby K

Publication type: Article

Publication status: Published

Journal: Muscle & Nerve

Year: 2013

Volume: 48

Issue: 1

Pages: 27-31

Print publication date: 01/07/2013

Online publication date: 24/04/2013

Acceptance date: 09/03/2013

ISSN (print): 0148-639X

ISSN (electronic): 1097-4598

Publisher: John Wiley & Sons, Inc.


DOI: 10.1002/mus.23831


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Funder referenceFunder name
MR/K000608/1Medical Research Council