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A randomized,controlled trial of daily prednisone was conducted in 99 boys (aged 5 to 15 years)with Duchenne dystrophy to define the time course of improvement and the dose response to treatment.Prednisone at 0.3 mg/kg(n= 33),prednisone at 0.75 mg/kg(n=34),and placebo(n=32)were administered for 6 months.Patients were examined using manual muscle and myometry testing, timed functional testing,pulmonary function testing,and laboratory measurements at 10 days,1 month,2 months,3 months,and 6 months of treatment.Boys treated with prednisone had stronger average muscle strength scores,than did boys treated with placebo as early as 10 days after starting therapy.At the 3-month visit,the boys in the group given 0. 75 mg/kg of prednisone were significantly stronger than those in the group given 0.3 mg/kg of prednisone,indicating a dose response.At 6 months, significant side effects occurred in the group treated with 0.75 mg/kg of prednisone,including weight gain,cushingold appearance,and excessive hair growth.Only weight gain was observed in the group taking prednisone at a dose of 0.3 mg/kg.Importantly,no side effects were evident at 10 days or 1 month of treatment,despite improvement in muscle strength and function.We conclude that prednisone produces a rapid increase in muscle strength in patients with Duchenne dystrophy and that this improvement is maximal at a prednisone dosage of 0.75 mg/kg or less. |
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