变异性侧面硬化症的疾病修饰疗法:随机临床试验的网络元分析
Mario Prado1, Karen Joy Adiao2
1Department of Physiology, University of the Philippines-College of Medicinehttps://ror.org/01rrczv41, Manila, Philippines.
概括
利鲁在肌缩性侧面硬化症 (ALS) 患者中显示出生存益处,但网络元分析 (NMA) 发现治疗方法之间没有显著差异. 需要进一步的研究来有效地比较这些ALS疗法.
科学领域:
- 神经学 神经学
- 临床试验 临床试验
- 药理学 药理学是指药理学的学科.
背景情况:
- 目前还没有对比的试验,比较了riluzole,edaravone,sodium phenylbutyrate和taurursodiol (SPT) 和tofersen用于肌缩侧面硬化症 (ALS).
- 现有的ALS治疗方法缺乏直接的比较疗效数据.
研究的目的:
- 进行一个网络元分析 (NMA),比较5种对肌缩侧面硬化症 (ALS) 的干预措施.
- 为了评估瑞卢,埃达拉,SPT和托弗森的相对疗效,与安慰剂相比.
主要方法:
- 对至少有两项研究的干预措施进行了常规的元分析.
- 对随机临床试验进行了网络元分析 (NMA),比较了五种ALS干预措施.
- 包含的研究:3项用于riluzole,3项用于edaravone,1项用于SPT,1项用于tofersen.
主要成果:
- 分析了1601名ALS患者 (1185名干预,416名控制).
- 利卢的生存概率比安慰剂高36% (OR:1.36,p=0.03).
- 在NMA的任何干预措施之间,没有发现死亡率或ALSFRS-R得分的显著差异.
结论:
- 在传统的元分析中指出了瑞卢的生存益处,但NMA的发现是无意义的.
- 对ALS干预措施之间的结果测量和患者异质性极限比较的差异.
- 由于局限性,基于当前的NMA无法对ALS治疗做出强有力的建议.
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