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Muscle Nerve. 2013 Jul;48(1):27-31. doi: 10.1002/mus.23831. Epub 2013 Apr 25.

Corticosteroids in Duchenne muscular dystrophy: major variations in practice.

Author information

1
University of Rochester, Departments of Neurology and Biostatistics, 265 Crittenden Boulevard, CU 420669, Rochester, New York, 14642, USA. robert_griggs@urmc.rochester.edu

Abstract

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.

PMID:
23483575
DOI:
10.1002/mus.23831
[Indexed for MEDLINE]

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