Real-world evidence of riluzole on survival and ALSFRS change in a Chinese ALS cohort

This study aimed to evaluate the effects of riluzole on survival and changes in ALS Functional Rating Scale (ALSFRS) among Chinese patients with Amyotrophic Lateral Sclerosis (ALS).
Propensity score matching was used to balance baseline variables between the riluzole group (n = 238) and control group (n = 454). Survival was analyzed using Kaplan – Meier curves and Cox regression, while multivariable linear regression assessed ALSFRS changes at 6 and 12 months. Subgroup analyses were conducted to identify potential responders.
Riluzole did not significantly improve survival (p = 0.478) or ALSFRS changes at 6 months (p = 0.380) or 12 months (p = 0.175). Subgroup analyses revealed no survival benefit in any subgroup, and further stratification showed inconsistent adverse effects on ALSFRS scores.
Riluzole neither prolonged survival nor slowed functional decline in Chinese ALS patients, with no subgroup demonstrating a better response.

本研究旨在评估利鲁唑（riluzole）对中国肌萎缩侧索硬化症（Amyotrophic Lateral Sclerosis, ALS）患者生存率及肌萎缩侧索硬化功能评定量表（Amyotrophic Lateral Sclerosis Functional Rating Scale, ALSFRS）评分变化的影响。本研究采用倾向性评分匹配法平衡利鲁唑组（n=238）与对照组（n=454）的基线变量。生存率分析采用卡普兰-迈耶（Kaplan–Meier）曲线与Cox回归模型，多变量线性回归则用于评估患者治疗6个月及12个月时的ALSFRS评分变化；此外本研究开展亚组分析以识别潜在的应答人群。结果显示，利鲁唑并未显著改善患者生存率（p=0.478），亦未在治疗6个月（p=0.380）或12个月（p=0.175）时对ALSFRS评分变化产生积极影响；亚组分析同样未发现任何亚组存在生存率获益，进一步分层分析则表明利鲁唑对ALSFRS评分存在不一致的不良影响。综上，利鲁唑既未延长中国ALS患者的生存时长，亦未延缓其功能衰退，且未发现任何亚组能从中获得更优的治疗应答。