Center for Experimental Neurological Therapies, S. Andrea Hospital, II Faculty of Medicine, Sapienza University of Rome, Via di Grottarossa 1035-1039, 00189, Rome, Italy.
Neurology. 2010 Mar 9;74(10):839-45. doi: 10.1212/WNL.0b013e3181d31e23.
The pleiotropic effects of riluzole may antagonize common mechanisms underlying chronic cerebellar ataxia, a debilitating and untreatable consequence of various diseases.
In a randomized, double-blind, placebo-controlled pilot trial, 40 patients presenting with cerebellar ataxias of different etiologies were randomly assigned to riluzole (100 mg/day) or placebo for 8 weeks. The following outcome measures were compared: proportion of patients with a decrease of at least 5 points in the International Cooperative Ataxia Rating Scale (ICARS) total score after 4 and 8 weeks compared with the baseline score; mean changes from the baseline to posttreatment ICARS (total score and subscores at 8 weeks); and occurrence of adverse events.
Riluzole and placebo groups did not differ in baseline characteristics. The number of patients with a 5-point ICARS drop was significantly higher in the riluzole group than in the placebo group after 4 weeks (9/19 vs 1/19; odds ratio [OR] = 16.2; 95% confidence interval [CI ] 1.8-147.1) and 8 weeks (13/19 vs 1/19; OR = 39.0; 95% CI 4.2-364.2). The mean change in the riluzole group ICARS after treatment revealed a decrease (p < 0.001) in the total score (-7.05 [4.96] vs 0.16 [2.65]) and major subscores (-2.11 [2.75] vs 0.68 [1.94] for static function, -4.11 [2.96] vs 0.37 [2.0] for kinetic function, and -0.74 [0.81] vs 0.05 [0.40] for dysarthria). Sporadic, mild adverse events occurred.
These findings indicate the potential effectiveness of riluzole as symptomatic therapy in diverse forms of cerebellar ataxia.
This study provides Class I evidence that riluzole reduces, by at least 5 points, the ICARS score in patients with a wide range of disorders that cause cerebellar ataxia (risk difference 63.2%, 95% CI 33.5%-79.9%).
利鲁唑的多效作用可能拮抗慢性小脑共济失调的共同机制，这是多种疾病导致的一种使人虚弱且无法治疗的后果。
在一项随机、双盲、安慰剂对照的试验中，40 名患有不同病因小脑共济失调的患者被随机分配接受利鲁唑（每天 100 毫克）或安慰剂治疗 8 周。比较以下结局指标：与基线评分相比，4 周和 8 周后国际合作共济失调评分量表（ICARS）总分至少下降 5 分的患者比例；从基线到治疗后 8 周 ICARS（总分和子评分）的平均变化；以及不良事件的发生。
利鲁唑组和安慰剂组在基线特征上无差异。4 周后，利鲁唑组 ICARS 下降 5 分的患者人数明显多于安慰剂组（9/19 比 1/19；比值比[OR] = 16.2；95%置信区间[CI] 1.8-147.1）和 8 周（13/19 比 1/19；OR = 39.0；95%CI 4.2-364.2）。治疗后利鲁唑组 ICARS 的平均变化显示总分（p < 0.001）下降（-7.05 [4.96] 比 0.16 [2.65]）和主要子评分下降（静态功能-2.11 [2.75] 比 0.68 [1.94]，运动功能-4.11 [2.96] 比 0.37 [2.0]，构音障碍-0.74 [0.81] 比 0.05 [0.40]）。偶发、轻度不良事件发生。
这些发现表明利鲁唑作为多种形式小脑共济失调的症状治疗具有潜在疗效。
本研究提供了 I 级证据，表明利鲁唑可使广泛引起小脑共济失调的疾病患者的 ICARS 评分至少降低 5 分（风险差异 63.2%，95%CI 33.5%-79.9%）。
