在临床试验中,逆概率审查加权比按方案分析更安全吗?

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES
Jingyi Xuan, Shahrul Mt-Isa, Nicholas Latimer, Helen Bell Gorrod, William Malbecq, Kristel Vandormael, Victoria Yorke-Edwards, Ian R White
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引用次数: 0

摘要

在许多临床试验中,与研究中的治疗策略发生偏差。我们称之为干预偏差。在不发生干预偏差的情况下,普遍采用按方案分析来估计假设估计。当干预偏差与影响反事实结果的时变混杂因素相关时,通过审查按协议容易产生选择偏差。这可以通过审查权的逆概率来纠正,这给了与审查个体具有相似预后特征的未审查个体额外的权重。这些权重是通过对选定的协变量建模来计算的。审查权的逆概率依赖于不可测量的混杂假设,其合理性不能进行统计检验。不符合假设的逆概率滤波加权的次优实现将导致偏差。在仿真研究中,我们评估了不同实现下的每协议和审查权的逆概率的性能,以探索审查权的逆概率是否为每协议的安全替代方案。设计了不同的方案,以改变不同患病率的单组或双组的干预偏差、两个混杂因素之间的相关性、每个混杂因素的影响和样本量。结果表明,在大多数情况下,使用不同协变量组合的审查权重的逆概率优于每个协议,除了由两个混杂因素引起的选择偏差在两个方向上并且“抵消”的不寻常情况。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Is inverse probability of censoring weighting a safer choice than per-protocol analysis in clinical trials?

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.

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来源期刊
Statistical Methods in Medical Research
Statistical Methods in Medical Research 医学-数学与计算生物学
CiteScore
4.10
自引率
4.30%
发文量
127
审稿时长
>12 weeks
期刊介绍: Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)
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