Rabkin Judith G, Gordon Paul H, McElhiney Martin, Rabkin Richard, Chew Sheena, Mitsumoto Hiroshi
Department of Psychiatry, Columbia University, New York, New York, USA.
Muscle Nerve. 2009 Mar;39(3):297-303. doi: 10.1002/mus.21245.
Our objective was to determine whether modafinil alleviates fatigue in patients with amyotrophic lateral sclerosis (ALS). A placebo controlled trial with a 3:1 modafinil:placebo randomization in doses up to 300 mg/day for 4 weeks was followed by 8 weeks of open maintenance treatment. The primary endpoint was the Clinical Global Impressions-Improvement Scale. Secondary endpoints were the Fatigue Severity Scale, Epworth Sleepiness Scale, Beck Depression Inventory, Role Function Scale, and visual analog scales. Analysis of covariance was used to assess change at Week 4. Thirty-two patients were randomized; 29 completed the 4-week trial. In intention to treat (ITT) analysis, the response was 76% for modafinil versus 14% for placebo. In a completer analysis, the modafinil response rate was 86%, and the placebo response rate remained 14%. The number needed to treat was 1.6 (ITT). No medically serious adverse events were reported. Modafinil may be a promising intervention for fatigue in ALS patients. Replication in a larger study is needed.
我们的目标是确定莫达非尼是否能减轻肌萎缩侧索硬化症（ALS）患者的疲劳。进行了一项安慰剂对照试验，以3:1的比例将患者随机分为莫达非尼组和安慰剂组，莫达非尼剂量最高达300毫克/天，为期4周，随后进行8周的开放维持治疗。主要终点是临床总体印象改善量表。次要终点包括疲劳严重程度量表、爱泼华嗜睡量表、贝克抑郁量表、角色功能量表和视觉模拟量表。采用协方差分析评估第4周时的变化。32名患者被随机分组；29名患者完成了为期4周的试验。在意向性治疗（ITT）分析中，莫达非尼组的缓解率为76%，而安慰剂组为14%。在完成者分析中，莫达非尼的缓解率为86%，安慰剂的缓解率仍为14%。治疗所需人数为1.6（ITT）。未报告有严重医学不良事件。莫达非尼可能是一种有前景的干预ALS患者疲劳的药物。需要在更大规模的研究中进行重复验证。
