A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments.

A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments.
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DOI:
10.1093/biostatistics/kxq053
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发表时间:
2011-04
期刊:
Biostatistics (Oxford, England)
影响因子:
--
通讯作者:
White IR
White IR
中科院分区:
其他
文献类型:
--
作者:
Fischer K;Goetghebeur E;Vrijens B;White IR

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我们提出了一种结构平均建模方法,以获得在随机对照试验中比较2种活性治疗的依从性调整估计的治疗效果。该模型将个体的观察结果与他或她通过观察到的积极治疗的接受而产生的反事实的未治疗结果联系起来。我们提出的估计程序利用基线协变量,预测每个arm.We的依从性水平给出了一个封闭的形式估计,允许差分和无法解释的选择性(即非因果依从性,由于未观察到的混杂结果的关联)以及非参数误差分布。在一个简单的线性模型的2臂试验中,我们表明,不同的因果参数被确定,除非协变量特定的预期依从性水平是成比例的两个治疗组。在后一种情况下,只有两种治疗效果之间的线性对比是可估计的,并且可能是关键的关注点。我们在一项临床试验中比较了2种抗抑郁药。
We propose a structural mean modeling approach to obtain compliance-adjusted estimates for treatment effects in a randomized-controlled trial comparing 2 active treatments. The model relates an individual's observed outcome to his or her counterfactual untreated outcome through the observed receipt of active treatments. Our proposed estimation procedure exploits baseline covariates that predict compliance levels on each arm. We give a closed-form estimator which allows for differential and unexplained selectivity (i.e. noncausal compliance-outcome association due to unobserved confounding) as well as a nonparametric error distribution. In a simple linear model for a 2-arm trial, we show that the distinct causal parameters are identified unless covariate-specific expected compliance levels are proportional on both treatment arms. In the latter case, only a linear contrast between the 2 treatment effects is estimable and may well be of key interest. We demonstrate the method in a clinical trial comparing 2 antidepressants.
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