Estimation of the proportion of treatment effect explained by a high-dimensional surrogate.
Estimation of the proportion of treatment effect explained by a high-dimensional surrogate.
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DOI：
10.1002/sim.9352
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发表时间：
2022-05-30
影响因子：
2
通讯作者：
Parast, Layla
中科院分区：
文献类型：
--
作者：
Zhou, Ruixuan Rachel;Zhao, Sihai Dave;Parast, Layla
就某些主要结果而言，检验治疗有效性的临床研究通常需要对患者进行长期随访，和/或对主要结果进行昂贵或繁重的测量。确定感兴趣的主要结果的替代标记物可能允许人们以更少的随访时间、更少的成本或更少的负担来评估治疗效果。虽然许多临床和统计工作都集中在识别和验证代理标记上，但现有的方法往往集中在只有一个代理标记感兴趣的环境中。已经做了有限的工作来适应高维代理标记设置，其中潜在代理的数量大于样本大小。在这篇文章中，我们发展了一些方法来估计高维替代者解释的治疗效果的比例。我们研究了我们所提出的估计量的渐近性质，提出了推断步骤，并通过模拟研究检验了有限样本的性能。我们使用了一项随机研究的数据来说明我们提出的方法，该研究比较了一种新的乳清口服营养补充剂和一种标准补充剂在12周内体脂百分比的变化，其中感兴趣的替代标记是基因表达研究。
Clinical studies examining the effectiveness of a treatment with respect to some primary outcome often require long-term follow-up of patients and/or costly or burdensome measurements of the primary outcome of interest. Identifying a surrogate marker for the primary outcome of interest may allow one to evaluate a treatment effect with less follow-up time, less cost, or less burden. While much clinical and statistical work has focused on identifying and validating surrogate markers, available approaches tend to focus on settings in which only a single surrogate marker is of interest. Limited work has been done to accommodate the high-dimensional surrogate marker setting where the number of potential surrogates is greater than the sample size. In this paper, we develop methods to estimate the proportion of treatment effect explained by high-dimensional surrogates. We study the asymptotic properties of our proposed estimator, propose inference procedures, and examine finite sample performance via a simulation study. We illustrate our proposed methods using data from a randomized study comparing a novel whey-based oral nutrition supplement with a standard supplement with respect to change in body fat percentage over 12 weeks, where the surrogate markers of interest are gene expression probesets.
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影响因子：
1.9
作者：
Parast L;Cai T;Tian L
通讯作者：
Tian L
DOI：
10.18632/aging.202912
发表时间：
2021-04-02
期刊：
Aging
影响因子：
--
作者：
Grootswagers P;Smeets E;Oteng AB;Groot L
通讯作者：
Groot L
影响因子：
1.9
作者：
Gilbert, Peter B.;Hudgens, Michael G.
通讯作者：
Hudgens, Michael G.
影响因子：
2
作者：
Parast L;McDermott MM;Tian L
通讯作者：
Tian L
影响因子：
2.5
作者：
Oh JH;Jung SH;Hong SJ;Rhyu MG
通讯作者：
Rhyu MG