多重疾病修饰疗法在复发性缓解性多发性硬化症中的有效性:因果推理模拟多臂随机试验
Ibrahima Diouf1,2, Charles B Malpas1,3, Sifat Sharmin1
1CORe, Department of Medicine, The University of Melbourne, Melbourne, Victoria, Australia.
Journal of neurology, neurosurgery, and psychiatry
|July 6, 2023
概括
这项研究比较了复发性复发性硬化症 (RRMS) 的多种疾病修饰疗法. 与其他治疗方法相比,纳塔利祖马布和芬戈利莫德在减少复发和残疾方面表现出更高的有效性.
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:
- 对复发性复发性硬化症 (RRMS) 的多种疾病修饰疗法 (DMT) 的同时长期比较是有限的.
- 这项研究模拟了一项随机试验,在五年内比较六种常见的DMT.
研究的目的:
- 在RRMS患者中,同时比较纳塔利祖马布,芬戈利莫德,二甲基烟酸盐,特里弗卢诺米德,干扰素β,格拉蒂拉默酸盐和没有治疗的有效性.
- 评估这些疗法对复发发病率,残疾恶化和残疾改善的影响.
主要方法:
- 通过MSBase利用来自35个国家的74个中心的数据,分析了23,236名符合条件的患者.
- 采用边际结构模型 (MSM) 来估计平均治疗效应 (ATE) 和治疗者中的平均治疗效应 (ATT).
- 在6个月间调整为多个共变量,以考虑治疗变化和患者特征.
主要成果:
- 与格拉蒂拉默酸盐相比,纳塔利祖马布,芬戈利莫德和二甲基 fumarate 在减少复发方面显示出优异的ATE.
- 纳塔利祖马布在减少残疾恶化和改善残疾方面表现出优异的ATE.
- 双对ATT比较证实了纳塔利祖马布和芬戈利莫德对复发和残疾的优越作用.
结论:
- 在活性RRMS中,纳塔利祖马布和芬戈利莫德比二甲基烟酸,特里弗卢诺米德,格拉提拉默酸和干扰素β更有效.
- 边缘结构模型 (MSM) 是模拟试验和同时比较多个干预措施的宝贵工具.
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