Deflazacort in Duchenne dystrophy: study of long-term effect

Muscle Nerve. 1994 Apr;17(4):386-91. doi: 10.1002/mus.880170405.

Abstract

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 33.2 +/- 9 months; for the placebo group it was 20.5 +/- 11 months (deflazacort vs. placebo group, P < 0.05) [corrected]. 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.

Publication types

  • Clinical Trial
  • Comparative Study
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anti-Inflammatory Agents, Non-Steroidal / therapeutic use*
  • Body Weight / drug effects
  • Child
  • Double-Blind Method
  • Follow-Up Studies
  • Gait
  • Humans
  • Motor Activity
  • Muscles / physiopathology*
  • Muscular Dystrophies / drug therapy*
  • Muscular Dystrophies / physiopathology
  • Patient Dropouts
  • Pregnenediones / adverse effects
  • Pregnenediones / therapeutic use*
  • Time Factors

Substances

  • Anti-Inflammatory Agents, Non-Steroidal
  • Pregnenediones
  • deflazacort

Grant support