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利鲁唑对中国肌萎缩侧索硬化症队列患者生存率及肌萎缩侧索硬化功能评分变化影响的真实世界证据

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

作者信息

Jiang JingSi, Li XiaoGang, Mi YuXin, Wang YiYing, Heng YanXi, Li ZhiWen, Deng Min

机构信息

Institute of Medical Innovation and Research, Peking University Third Hospital, Beijing, China.

Department of Neurology, Peking University Third Hospital, Beijing, China.

出版信息

Neurodegener Dis Manag. 2025 Apr-Jun;15(2-3):77-87. doi: 10.1080/17582024.2025.2488235. Epub 2025 Apr 4.

DOI:10.1080/17582024.2025.2488235
PMID:40183433
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12118432/
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 ( = 238) and control group ( = 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 ( = 0.478) or ALSFRS changes at 6 months ( = 0.380) or 12 months ( = 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.

摘要

目的

本研究旨在评估利鲁唑对中国肌萎缩侧索硬化症(ALS)患者生存率及肌萎缩侧索硬化功能评定量表(ALSFRS)变化的影响。

患者与方法

采用倾向得分匹配法平衡利鲁唑组(n = 238)和对照组(n = 454)之间的基线变量。使用Kaplan - Meier曲线和Cox回归分析生存率,同时采用多变量线性回归评估6个月和12个月时ALSFRS的变化。进行亚组分析以确定潜在的反应者。

结果

利鲁唑未显著改善生存率(P = 0.478),也未显著改善6个月时(P = 0.380)或12个月时(P = 0.175)的ALSFRS变化。亚组分析显示任何亚组均未获得生存益处,进一步分层显示对ALSFRS评分的不良反应不一致。

结论

利鲁唑既未延长中国ALS患者的生存期,也未减缓其功能衰退,没有亚组显示出更好的反应。

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Front Nutr. 2024 Oct 31;11:1437521. doi: 10.3389/fnut.2024.1437521. eCollection 2024.
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Differential cortical gray matter changes in early- and late-onset patients with amyotrophic lateral sclerosis.早发性和晚发性肌萎缩侧索硬化症患者的皮质灰质差异变化。
Cereb Cortex. 2024 Jan 14;34(1). doi: 10.1093/cercor/bhad426.
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Effect of sodium phenylbutyrate and taurursodiol on plasma concentrations of neuroinflammatory biomarkers in amyotrophic lateral sclerosis: results from the CENTAUR trial.苯丁酸钠和牛磺酸熊脱氧胆酸对肌萎缩侧索硬化症患者神经炎症生物标志物血浆浓度的影响:CENTAUR 试验结果。
J Neurol Neurosurg Psychiatry. 2024 Jun 17;95(7):605-608. doi: 10.1136/jnnp-2023-332106.
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The amyotrophic lateral sclerosis exposome: recent advances and future directions.肌萎缩侧索硬化症外显子组:最新进展与未来方向。
Nat Rev Neurol. 2023 Oct;19(10):617-634. doi: 10.1038/s41582-023-00867-2. Epub 2023 Sep 14.
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