RANDOMIZED, DOUBLE-BLIND 6-MONTH TRIAL OF PREDNISONE IN DUCHENNES MUSCULAR-DYSTROPHY
RANDOMIZED, DOUBLE-BLIND 6-MONTH TRIAL OF PREDNISONE IN DUCHENNES MUSCULAR-DYSTROPHY
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DOI：
10.1056/nejm198906153202405
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发表时间：
1989-06-15
影响因子：
158.5
通讯作者：
GILDER, B
中科院分区：
文献类型：
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作者：
MENDELL, JR;MOXLEY, RT;GILDER, B
我们对103名患有杜氏肌营养不良症的男孩（5 - 15岁）进行了一项为期6个月的随机、双盲、对照强的松试验。患者被分配到三种方案中的一种：强的松，每天每公斤体重0.75 mg (n = 33)；强的松，每公斤每天1.5毫克（n = 34）；或安慰剂（n = 36）。最初，两组在肌肉功能的所有指标上都具有可比性。强的松组和强的松组在肌肉力量和功能综合评分上均有相似程度的显著改善。改善最早开始于一个月，并在三个月时达到顶峰。在6个月时，与安慰剂组相比，高剂量强的松组在从仰卧位上升到站立位所需的时间（3.5秒对6.2秒）、步行9米（7.0秒对9.7秒）、爬4级楼梯（4.0秒对7.1秒）、举重（2.1公斤对1.2公斤）和用力肺活量（1.7升对1.5升）（所有比较P < 0.001）方面都有所改善。尿肌酐排泄量增加（261毫克/ 24小时vs 190毫克/ 24小时），这表明总肌肉量增加。然而，需要长腿支架（n = 5）或轮椅（n = 11）的强的松治疗患者继续需要它们。最常见的副作用是体重增加，库欣样外观和毛发过度生长。我们从这项为期六个月的研究中得出结论，强的松可以改善杜氏肌萎缩症患者的力量和功能。然而，需要进一步的研究来确定这些改善的机制，并确定长期使用皮质类固醇治疗是否有必要，尽管它们有副作用。
We performed a randomized, double-blind, controlled six-month trial of prednisone in 103 boys with Duchenne''s muscular dystrophy (age, 5 to 15 years). The patients were assigned to one of three regimens: prednisone, 0.75 mg per kilogram of body weight per day (n = 33); prednisone, 1.5 mg per kilogram per day (n = 34); or placebo (n = 36). The groups were initially comparable in all measures of muscle function. Both prednisone groups had significant improvement of similar degree in the summary scores of muscle strength and function. Improvement began as early as one month and peaked by three months. At six months the high-dose prednisone group, as compared with the placebo group, had improvement in the time needed to rise from a supine to a standing position (3.5 vs. 6.2 seconds), to walk 9 m (7.0 vs. 9.7 seconds), and to climb four stairs (4.0 vs. 7.1 seconds), in lifting a weight (2.1 vs 1.2 kg), and in forced vital capacity (1.7 vs. 1.5 liters) (P < 0.001 for all comparisons). There was an increase in urinary creatinine excretion (261 vs. 190 mg per 24 hours), which suggested an increase in total muscle mass. However, the prednisone-treated patients who had required long-leg braces (n = 5) or wheelchairs (n = 11) continued to require them. The most frequent side effects were weight gain, cushingoid appearance, and excessive hair growth. We conclude from this six-month study that prednisone improves the strength and function of patients with Duchenne''s muscular dystrophy. However, further research is required to identify the mechanisms responsible for these improvements and to determine whether prolonged treatment with corticosteroids may be warranted despite their side effects.