LONG-TERM BENEFIT FROM PREDNISONE THERAPY IN DUCHENNE MUSCULAR-DYSTROPHY
LONG-TERM BENEFIT FROM PREDNISONE THERAPY IN DUCHENNE MUSCULAR-DYSTROPHY
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DOI：
10.1212/wnl.41.12.1874
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发表时间：
1991-12-01
期刊：
影响因子：
9.9
通讯作者：
WILSON, B
中科院分区：
文献类型：
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作者：
FENICHEL, GM;FLORENCE, JM;WILSON, B
两项连续6个月、随机、双盲、对照的强的松试验表明，0.75 mg/kg/d是改善男孩杜氏肌营养不良症（DMD）力量的最佳剂量。我们试图让93名男孩继续服用这种剂量2年。在3年的观察中，所有服用强的松的男孩平均肌力评分下降0.072单位/年，而自然史对照组的预期下降0.341单位/年。在一些男孩中，副作用的发生阻止了维持全剂量，这可能降低了反应。在最后一次访问时，剂量范围为0.15 mg/kg至0.75 mg/kg。除了保持他们的力量外，一些男孩在举重和一些计时功能测试中的表现也有所提高。用强的松治疗DMD可显著减缓虚弱和功能丧失的进展，至少持续3年。
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.