TY -的T1 deflazacort vs强的松和安慰剂的疗效和安全性杜氏肌萎缩症JF -神经学乔-神经病学SP - 2123 LP - 2131 - 10.1212 / WNL。首页0000000000003217六世- 87 - 20盟盟罗伯特·c·格里戈斯- j·菲利普·米勒盟谢丽尔·r·格林伯格AU -达西l .费林AU -艾伦Pestronk盟-杰里·r·Mendell说非盟-理查德·t·莫克斯利三世盟温迪王盟——约翰·t·Kissel AU -瓦莱丽·库克盟-米歇尔Vanasse盟Julaine m .佛罗伦萨盟Shree迪亚AU -乔丹s Dubow盟詹姆斯·m·迈耶Y1 - 2016/11/15 UR - //www.ez-admanager.com/content/87/20/2123.abstract N2 -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 ER -