Rank-based principal stratum sensitivity analyses.

Rank-based principal stratum sensitivity analyses.
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基于等级的主要阶层敏感性分析。

DOI:
10.1002/sim.5849
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发表时间:
2013
影响因子:
2
通讯作者:
Shepherd,BE
Shepherd,BE
中科院分区:
医学3区
文献类型:
--
作者:
Lu,X;Mehrotra,DV;Shepherd,BE

文献摘要

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我们描述了基于秩的方法,以评估研究中的主要分层治疗效应,其中仅在随机化后选择的亚组中明确定义了关注的结局。我们的方法是敏感性分析,在该estimands确定通过固定一个参数,然后我们通过改变这个参数在一个合理的值范围内的结果的敏感性进行调查。我们提出了三种基于秩的检验统计量,并通过模拟比较了它们的性能,并提供了建议。我们还研究了三种不同的自举方法来确定显着性水平。最后,我们将我们的方法应用于两项研究:艾滋病毒疫苗试验和前列腺癌预防试验。版权所有© 2013约翰威利父子有限公司.
We describe rank‐based approaches to assess principal stratification treatment effects in studies where the outcome of interest is only well‐defined in a subgroup selected after randomization. Our methods are sensitivity analyses, in that estimands are identified by fixing a parameter and then we investigate the sensitivity of results by varying this parameter over a range of plausible values. We present three rank‐based test statistics and compare their performance through simulations, and provide recommendations. We also study three different bootstrap approaches for determining levels of significance. Finally, we apply our methods to two studies: an HIV vaccine trial and a prostate cancer prevention trial. Copyright © 2013 John Wiley & Sons, Ltd.