Corticosteroids in Duchenne muscular dystrophy: major variations in practice

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


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.

MeSH terms

  • Adrenal Cortex Hormones / therapeutic use*
  • Clinical Trials, Phase II as Topic / methods
  • Data Collection* / methods
  • Europe / epidemiology
  • Humans
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / epidemiology*
  • Physicians / standards*
  • Practice Guidelines as Topic / standards
  • Randomized Controlled Trials as Topic / methods
  • United States / epidemiology


  • Adrenal Cortex Hormones