A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy

• Published in: Muscle & nerve Vol. 23; no. 9; pp. 1344 - 1347
• Main Authors: Bonifati, Marco Domenico, Ruzza, Giampietro, Bonometto, Piero, Berardinelli, Angela, Gorni, Kseniya, Orcesi, Simona, Lanzi, Giovanni, Angelini, Corrado
• Format: Journal Article
• Language: English
• Published: New York John Wiley & Sons, Inc 01-09-2000; Wiley
• Subjects: Adolescent; Biological and medical sciences; Child; Child, Preschool; deflazacort; Double-Blind Method; Duchenne muscular dystrophy; Humans; Male; Medical sciences; Muscle; Muscular Dystrophy, Duchenne - drug therapy; Pharmacology. Drug treatments; prednisone; Prednisone - adverse effects; Prednisone - therapeutic use; Pregnenediones - adverse effects; Pregnenediones - therapeutic use; steroid; trial; Human; Steroid; Corticosteroid; Neuromuscular diseases; Nervous system diseases; Deflazacort; Questionnaire; Treatment efficiency; Prednisone; Genetic disease; Walking; Chemotherapy; Treatment; Duchenne muscular dystrophy
• ISSN: 0148-639X; 1097-4598
• DOI: 10.1002/1097-4598(200009)23:9<1344::AID-MUS4>3.0.CO;2-F

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.