Deflazacort in Duchenne dystrophy: Study of long-term effect

A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo. The deflazacort group showed significant improvement in climbing stairs (P <0.01), in rising from a chair, Gower's maneuver, and walking (P <0.0025) after 6 months of treatment. After 1 year, all the above changes remained significantly improved and the MRC index was significantly better (P <0.05) in the treated group. After 2 years, a significant change was found in the MRC index: higher scores in walking, chair rising (P <0.02), and grade and time of Gower's maneuver (P <0.05) were found. The mean time for loss of ambulation for the treated group after we started the trial was 20.5 ± 11 months; for the placebo group it was 33.2 ± 9 months (placebo vs. deflazacort group, P <0.05). Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group. Side effects were mild, consisting of moderate weight gain and slight behavioral changes.