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[美西吲哚与安慰剂治疗杜氏肌营养不良症的疗效试验。对14名儿童进行的为期一年的随访研究]

[A therapeutic trial of mazindol versus placebo in Duchenne muscular dystrophy. A one-year follow-up study of 14 children].

作者信息

Desnuelle C, Serratrice G, N'guyen V K, Richelme C, Pinsard N

机构信息

Clinique des Maladies du Système nerveux, CHU La Timone, Marseille.

出版信息

Arch Fr Pediatr. 1989 Dec;46(10):759-65.

PMID:2697199
Abstract

A simple-blind therapeutic trial of mazindol (2 mg/d) versus placebo in 14 boys with Duchenne muscular dystrophy, 5 to 13 years old, for 12 months was conducted in order to analyse the efficacy of this drug on the natural history of the disease. Evaluation of muscle strength by manual testing, motor ability by functional testing and timed tests, weight, height, serum CK and pulmonary function were performed every two months. The differences in evolution between groups were significant only for weight and height. However comparison of muscle strength and of vital capacity expressed as percent of predicted at the beginning and at the end of the study suggested a beneficial trend in the mazindol-treated group.

摘要

为分析马吲哚(2毫克/天)对杜氏肌营养不良症自然病程的疗效,对14名5至13岁的患杜氏肌营养不良症男孩进行了一项为期12个月的马吲哚(2毫克/天)与安慰剂的单盲治疗试验。每两个月通过手动测试评估肌肉力量,通过功能测试和定时测试评估运动能力,测量体重、身高、血清肌酸激酶(CK)以及肺功能。两组之间仅在体重和身高的变化上存在显著差异。然而,在研究开始和结束时,以预测值的百分比表示的肌肉力量和肺活量的比较表明,马吲哚治疗组有有益的趋势。

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