Riluzole in Huntington's disease: A 3-year, randomized controlled study
Riluzole in Huntington's disease: A 3-year, randomized controlled study
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DOI：
10.1002/ana.21181
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发表时间：
2007-09-01
影响因子：
11.2
通讯作者：
Ludolph, Albert C.
中科院分区：
文献类型：
--
作者：
Landwehrmeyer, G. Bernhard;Dubois, Bruno;Ludolph, Albert C.
目的：我们进行了一项随机双盲试验利鲁唑在亨廷顿舞蹈病，以探讨这种抗兴奋毒性药物在减缓疾病progress.Methods的疗效：该研究包括537例成人患者的临床诊断亨廷顿舞蹈病基因分型证实。患者随机（2：1）接受利鲁唑（50 mg，每日两次）或安慰剂治疗3年。禁止合并使用抗舞蹈病药物，引入此类药物是预定义的终点。主要结果测量是来自统一亨廷顿病评定量表的运动和总功能能力分项评分的综合评分的变化。结果：共有379例患者完成了研究（平均年龄47 [标准差9.5]岁，50%为女性患者）。停药的主要原因是引入抗舞蹈病药物。安慰剂组和利鲁唑组“符合方案”人群的综合评分（主要结局）较基线的中位变化分别为13.7（95%置信区间，11.1-17.2）和14.3（95%置信区间，11.7-16.6）。因此，未证实组间结局差异（P = 0.93，Mann-Whitney U检验）。除了安慰剂组更频繁地求助于抗龋齿药物外，未观察到次要疗效结局变量的差异。没有意外的不良事件的报告，耐受性是acceptable.Interpretation：没有神经保护或利鲁唑在亨廷顿氏病的有益症状的影响被证明。
Objective: We conducted a randomized double-blind trial of riluzole in Huntington's disease to investigate the efficacy of this antiexcitotoxic drug in slowing disease progression.Methods: The study included 537 adult patients with a clinical diagnosis of Huntington's disease confirmed by genotyping. Patients were randomized (2:1) to treatment with riluzole (50mg twice daily) or placebo for 3 years. Concomitant use of antichoreic medication was forbidden, and introduction of such medication was a predefined end point. The primary outcome measure was change in a combined score derived from the motor and total functional capacity subscores of the Unified Huntington's Disease Rating Scale. Safety was also evaluated.Results: A total of 379 patients completed the study (mean age, 47 [standard deviation, 9.5] years; 50% female patients). The principal reason for discontinuation was introduction of antichoreic medication. The median change from baseline in the combined score (primary outcome) for the "Per protocol" population was 13.7 (95% confidence interval, 11.1-17.2) in the placebo group and 14.3 (95% confidence interval, 11.7-16.6) in the riluzole group. No intergroup difference in outcome could thus be demonstrated (P = 0.93, Mann-Whitney U test). No differences in secondary efficacy outcome variables were observed except for more frequent recourse to anticboreic medication in the placebo group. No unexpected adverse events were reported, and tolerability was acceptable.Interpretation: No neuroprotective or beneficial symptomatic effects of riluzole in Huntington's disease were demonstrated.