在托弗森对肌缩侧面硬化症的临床试验中发生了严重的神经学不良事件
Alexandra Lovett1, Sowmya Chary1, Suma Babu2
1Biogen, Cambridge, Massachusetts, USA.
Muscle & nerve
|February 28, 2025
概括
严重的神经学不良事件 (AEs) 发生在对超氧化物脱酶1突变异位侧硬化症 (SOD1-ALS) 的tofersen试验中. 大多数事件都得到解决,但有些人导致停止治疗,告知药物的益处/风险概况.
科学领域:
- 神经学 神经学
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
背景情况:
- 托弗森已被批准用于治疗与超氧化物失突酶1突变 (SOD1-ALS) 相关的肌缩性侧面硬化症 (ALS).
- 反感性寡核酸 (ASOs) 可能表现出促炎性质,需要安全监测.
研究的目的:
- 报告和描述在对SOD1-ALS的tofersen临床试验中观察到的严重神经系统不良事件 (AEs).
- 为在SOD1-ALS患者中对托弗森的益处/风险概况的持续评估提供信息.
主要方法:
- 严重的神经系统性AE的系统描述,包括骨髓炎,激素炎,无菌性脑膜炎和乳头炎.
- 副作用是根据国际协调会议的指导方针定义的,并且由研究人员根据临床表现和工作情况来诊断.
主要成果:
- 十个参与者 (大约. 7%) 经历了12种严重的神经病变:4种髓炎,2种根炎,2种无菌性脑膜炎,4种内高血压/乳头炎.
- 大多数事件自发或随着治疗修改而消失;三个导致停止治疗,但大多数参与者继续接受治疗.
- 没有严重的神经学AE是危及生命或致命的.
结论:
- 神经系统的副作用,如骨髓炎,根性炎和乳头炎,以前没有在ASO治疗中报告过,发生在tofersen.
- 这些发现对于评估托弗森对SOD1-ALS的整体益处/风险至关重要.
- 持续收集安全数据将进一步完善tofersen在SOD1-ALS.中的安全概况.
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