In 2004 a Cochrane review and AAN practice parameter concluded that prednisone 0.75 mg/kg/d is of short-term efficacy in Duchenne muscular dystrophy (DMD). Subsequent efforts to standardize care for DMD found wide variation in corticosteroid use. In this study, the authors surveyed physicians who follow patients with DMD: (1) Clinics in the TREAT-NMD (Translational Research in Europe – Assessment and Treatment of Neuromuscular Diseases) network (predominantly Europe); (2) US MDA clinic directors. They also documented the co-administered corticosteroids in a trial of a putative treatment (ataluren) for DMD. Of 105 Treat-NMD clinicians, corticosteroids were not used in 10 clinics and 29 different regimens were used, the most frequent was 0.75 mg/kg/d prednisone in 61 centres; 10 days on/10 days off in 36 centres; 0.9 mg/kg/d deflazacort in 32 centres; and 5 mg/kg/d weekends in 10 centres. Similar diversity was identified in MDA clinics and in the ataluren trial. Variability in corticosteroid use suggests uncertainty about risks/benefits of corticosteroid regimens for DMD.
