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

利鲁唑对中国ALS患者生存率和ALSFRS评分变化的真实世界证据

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Abstract

AIMS: 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). PATIENTS & METHODS: 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. RESULTS: 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. CONCLUSIONS: Riluzole neither prolonged survival nor slowed functional decline in Chinese ALS patients, with no subgroup demonstrating a better response.

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