RT期刊文章SR电子T1 deflazacort vs强的松和安慰剂的疗效和安全性杜氏肌萎缩症摩根富林明神经病学神经学乔FD Lippincott Williams &威尔金斯SP 2123 OP 2131 10.1212 / WNL。首页87签证官0000000000003217是20 A1 A1菲利普j .罗伯特·c·格里戈斯米勒A1谢丽尔·r·格林伯格A1达西l .费林A1 Alan Pestronk A1杰瑞·r·Mendell说A1理查德·t·莫克斯利三世A1温迪A1国王约翰·t·Kissel A1瓦莱丽·库克A1米歇尔Vanasse A1 Julaine m .佛罗伦萨A1 Shree迪亚A1约旦s Dubow A1詹姆斯·m·迈耶年2016 UL //www.ez-admanager.com/content/87/20/2123.abstract AB目的:评估安全性和有效性的deflazacort (DFZ)和强的松(PRED)和安慰剂在杜氏肌肉营养不良症(DMD)。首页方法:这第三阶段,双盲、随机、安慰剂对照、多中心研究评估肌肉力量在5 - 15岁的196个男孩DMD 52周期间。在第一阶段中,参与者被随机分配到接受治疗与DFZ 0.9毫克/公斤/天,DFZ 1.2毫克/公斤/天,PRED 0.75毫克/公斤/天,12周或安慰剂。在阶段2中,安慰剂的参与者被随机分配到治疗组1的3活跃。分配给一个活跃的参与者最初治疗持续治疗一个额外的40周。主要疗效终点是肌肉力量的平均变化与安慰剂比较,从基线到第12周。这项研究是在1995年完成。结果:治疗组(DFZ 0.9毫克/公斤/天,DFZ 1.2毫克/公斤/天,和PRED 0.75毫克/公斤/天)证明显著改善肌肉力量与安慰剂比较在12周。参与者服用PRED有明显的体重增加比安慰剂或两个剂量的DFZ 12周;在52周,参与者服用PRED有明显的体重增加比DFZ剂量。 The most frequent adverse events in all 3 active treatment arms were Cushingoid appearance, erythema, hirsutism, increased weight, headache, and nasopharyngitis.Conclusions: After 12 weeks of treatment, PRED and both doses of DFZ improved muscle strength compared with placebo. Deflazacort was associated with less weight gain than PRED.Classification of evidence: This study provides Class I evidence that for boys with DMD, daily use of either DFZ and PRED is effective in preserving muscle strength over a 12-week period.AE=adverse event; BMD=Becker muscular dystrophy; BMI=body mass index; CI=confidence interval; DFZ=deflazacort; DMD=Duchenne muscular dystrophy; FVC=forced vital capacity; GC=glucocorticoid; ITT=intent-to-treat; LS=least squares; MRC=Medical Research Council; PRED=prednisone; SAE=serious adverse event