Fenichel G M, Florence J M, Pestronk A, Mendell J R, Moxley R T, Griggs R C, Brooke M H, Miller J P, Robison J, King W
Department of Neurology, Vanderbilt University Medical Center, Nashville, TN 37212.
Neurology. 1991 Dec;41(12):1874-7. doi: 10.1212/wnl.41.12.1874.
Two successive, 6-month, randomized, double-blind, controlled trials of prednisone showed that 0.75 mg/kg/d was the optimal dose to improve strength in boys with Duchenne muscular dystrophy (DMD). We attempted to maintain 93 boys on that dose for an additional 2 years. During the 3 years of observation, the decline in average muscle strength scores of all boys taking prednisone was 0.072 units/yr, as compared with an expected decline of 0.341 units/yr from natural history controls. The occurrence of side effects in some boys prevented maintenance of the full dose, which may have lessened the response. At the time of last visit, dosages ranged from 0.15 mg/kg to 0.75 mg/kg. In addition to maintaining their strength, several of the boys actually improved their performance in lifting kilogram weights and in some timed function tests. Treatment of DMD with prednisone significantly slows the progression of weakness and loss of function for at least 3 years.
两项连续的、为期6个月的泼尼松随机双盲对照试验表明，对于患有杜氏肌营养不良症（DMD）的男孩，0.75毫克/千克/天是增强肌力的最佳剂量。我们试图让93名男孩维持该剂量再治疗2年。在3年的观察期内，所有服用泼尼松的男孩平均肌肉力量评分每年下降0.072个单位，而自然史对照预期每年下降0.341个单位。一些男孩出现的副作用使得无法维持全剂量，这可能减弱了治疗反应。在最后一次随访时，剂量范围为0.15毫克/千克至0.75毫克/千克。除了维持肌力外，一些男孩在举重和一些定时功能测试中的表现实际上有所改善。用泼尼松治疗DMD至少3年可显著减缓肌无力和功能丧失的进展。
