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 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.
Angelini
C
C
Regional Neuromuscular Center, University of Padova, Italy.
Pegoraro
E
E
Turella
E
E
Intino
M T
MT
Pini
A
A
Costa
C
C
D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000627:therapeutic use*; D001835:Body Weight / Q000187:drug effects; D002648:Child; D004311:Double-Blind Method; D005500:Follow-Up Studies; D005684:Gait; D006801:Humans; D009043:Motor Activity; D009132:Muscles / Q000503:physiopathology*; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000503:physiopathology; D010352:Patient Dropouts; D011282:Pregnenediones / Q000009:adverse effects / Q000627:therapeutic use*; D013997:Time Factors
D000894:Anti-Inflammatory Agents, Non-Steroidal; D011282:Pregnenediones; C021988:deflazacort