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. (C) 1994 John Wiley and Sons, Inc.