使用反事实框架来估计随机对照试验中的非预期治疗估计:一个方法论范围审查
Ellie Medcalf1, Fiona Stanaway1, Robin M Turner2
1Sydney School of Public Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
Contemporary clinical trials
|April 13, 2025
概括
随机对照试验 (RCT) 经常使用治疗意图 (ITT) 估计,当治疗坚持变化时,这些估计就不那么有用了. 本次审查强调了对于临床决策需要更好的非ITT估计,以及每个协议的估计.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 流行病学 流行病学
背景情况:
- 随机对照试验 (RCT) 通常使用治疗意向 (ITT) 估计.
- 当治疗不坚持发生时,ITT估值反映了治疗分配的影响而不是坚持,限制了临床效用.
研究的目的:
- 总结现有的关于RCT中非ITT估值的文献.
- 审查用于推导这些非ITT估计的反事实框架估计器.
主要方法:
- 进行了对MEDLINE和EMBASE数据库的方法范围审查.
- 搜索包括向前和向后引用搜索到2024年3月.
- 符合条件的记录集中在非ITT估计或相关模拟研究的反事实估计器上.
主要成果:
- 从746个选的记录中,发现了56个符合条件的记录.
- 47记录了不遵守的详细具体估计,报告了51个非ITT估计.
- 通常报告的非ITT估计包括定义不佳 (41%),符合平均因果效应 (33%),切换调整 (14%),每个协议 (12%).
- 使用的关键估计是反向概率权重 (27%) 和仪器变量 (IVs) (18%用于时间变化的治疗,17%用于标准IVs).
结论:
- 在RCT中,非ITT估值通常定义不佳,缺乏临床相关性.
- 需要对反事实框架估计器进行进一步的研究,以获得明确的估计值.
- 强调每种方案的估计值对于改善临床和政策决策至关重要.
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