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Deflazacort in Duchenne dystrophy: Study of long‐term effect
Author(s) -
Angelini C.,
Pegoraro E.,
Turella E.,
Intino M. T.,
Pini A.,
Costa C.
Publication year - 1994
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.880170405
Subject(s) - deflazacort , placebo , duchenne muscular dystrophy , medicine , placebo group , physical therapy , anesthesia , surgery , alternative medicine , pathology
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 croup. 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. © 1994 John Wiley & Sons, Inc.