Is inverse probability of censoring weighting a safer choice than per-protocol analysis in clinical trials?

被引:0
|
作者
Xuan, Jingyi [1 ]
Mt-Isa, Shahrul [2 ]
Latimer, Nicholas [3 ,4 ]
Gorrod, Helen Bell [3 ]
Malbecq, William [5 ,8 ]
Vandormael, Kristel [6 ]
Yorke-Edwards, Victoria [1 ,7 ]
White, Ian R. [1 ]
机构
[1] UCL, MRC Clin Trials Unit UCL, London, England
[2] MSD, Biostat & Res Decis Sci BARDS, Hlth Technol Assessment HTA Stat, Zurich, Switzerland
[3] Univ Sheffield, Sheffield Ctr Hlth & Related Res, Sch Med & Populat Hlth, Sheffield, England
[4] Delta Hat Ltd, Nottingham, England
[5] Univ Brussels, Dept Math, Brussels, Belgium
[6] MSD, Biostat & Res Decis Sci BARDS, Hlth Technol Assessment HTA Stat, Brussels, Belgium
[7] UCL, Ctr Adv Res Comp, London, England
[8] MSD, Brussels, Belgium
基金
英国医学研究理事会;
关键词
Inverse probability of censoring weight; per-protocol; non-adherence; treatment switching; dependent censoring; MARGINAL STRUCTURAL MODELS; DIRECTED ACYCLIC GRAPHS; 2ND-LINE TREATMENT; NONCOMPLIANCE; AIDS; INTENTION; SURVIVAL; THERAPY; ISSUES; TREAT;
D O I
10.1177/09622802241289559
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
Deviation from the treatment strategy under investigation occurs in many clinical trials. We term this intervention deviation. Per-protocol analyses are widely adopted to estimate a hypothetical estimand without the occurrence of intervention deviation. Per-protocol by censoring is prone to selection bias when intervention deviation is associated with time-varying confounders that also influence counterfactual outcomes. This can be corrected by inverse probability of censoring weighting, which gives extra weight to uncensored individuals who had similar prognostic characteristics to censored individuals. Such weights are computed by modelling selected covariates. Inverse probability of censoring weighting relies on the no unmeasured confounding assumption whose plausibility is not statistically testable. Suboptimal implementation of inverse probability of censoring weighting which violates the assumption will lead to bias. In a simulation study, we evaluated the performance of per-protocol and inverse probability of censoring weighting with different implementations to explore whether inverse probability of censoring weighting is a safe alternative to per-protocol. Scenarios were designed to vary intervention deviation in one or both arms with different prevalences, correlation between two confounders, effect of each confounder, and sample size. Results show that inverse probability of censoring weighting with different combinations of covariates outperforms per-protocol in most scenarios, except for an unusual case where selection bias caused by two confounders is in two directions, and 'cancels' out.
引用
收藏
页码:286 / 306
页数:21
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