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A multicenter, double‐blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy
Author(s) -
Bonifati Marco Domenico,
Ruzza Giampietro,
Bonometto Piero,
Berardinelli Angela,
Gorni Kseniya,
Orcesi Simona,
Lanzi Giovanni,
Angelini Corrado
Publication year - 2000
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 - 10.1002/1097-4598(200009)23:9<1344::aid-mus4>3.0.co;2-f
Subject(s) - deflazacort , prednisone , duchenne muscular dystrophy , medicine , randomized controlled trial , physical therapy , muscular dystrophy , pediatrics
We randomized 18 Duchenne muscular dystrophy (DMD) boys whose age ranged from 5.2 to 14.6 years (mean, 7.3 years) for treatment with either deflazacort (0.9 mg/kg/day) or prednisone (0.75 mg/kg/day) on the basis of age and functional score at the onset of treatment. We followed the patients every 3 months for 1 year, evaluating four limb muscles with the Medical Research Council scale and performance of four functions (walking, climbing stairs, Gowers' maneuver, and rising from a chair). Side effects were monitored by a questionnaire and by routine blood examination, and weight and height were recorded at each visit. At 12 months, the effect of both steroids was examined by comparing the status of the treated patients with another group of untreated DMD patients that served as natural history control. The two steroids were equally effective in improving motor function and functional performances. At 9 months, the average weight increase with respect to baseline value was 5% (2 kg) in the deflazacort group but 18% in the prednisone group ( P < 0.005), and the change remained significant after 12 months ( P < 0.05). Other minor but nonsignificant side effects were observed. Steroid treatment with deflazacort appears to cause fewer side effects than with prednisone, particularly weight gain, which could be important to maximize motor performances. © 2000 John Wiley & Sons, Inc. Muscle Nerve 23: 1344–1347, 2000.