Testing for heterogeneity in the utility of a surrogate marker.
Testing for heterogeneity in the utility of a surrogate marker.
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DOI：
10.1111/biom.13600
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发表时间：
2023-06
期刊：
影响因子：
1.9
通讯作者：
Tian, Lu
中科院分区：
文献类型：
--
作者：
Parast, Layla;Cai, Tianxi;Tian, Lu
关键词：
在需要对参与者进行长期和/或昂贵的随访以评估治疗的研究中，通常有兴趣识别和使用替代标记物来评估治疗效果。虽然已经提出了几种统计方法来评估潜在的替代标记，但可用的方法通常没有考虑或解决替代在效用或强度上因患者特征而变化的可能性。先前研究替代标记的工作已经表明，可能存在这种异质性，即，替代标记可能对某些亚组有用(就捕捉对主要结果的治疗效果而言)，但对其他亚组无效。了解这种异质性很重要，特别是如果要在未来的试验中使用替代物来取代主要结果。在这篇文章中，我们提出了一种方法和估计程序来衡量替代强度作为基线协变量W的函数，从而检验替代标记相对于W的效用的潜在异质性。在潜在的结果框架内，我们使用治疗对主要结果的影响的比例来量化替代强度/效用，这由对替代的治疗效果来解释。我们提出了测试程序来测试异质性的证据，通过模拟来检验这些方法的有限样本性能，并使用AIDS临床试验数据来说明方法。
In studies that require long-term and/or costly follow-up of participants to evaluate a treatment, there is often interest in identifying and using a surrogate marker to evaluate the treatment effect. While several statistical methods have been proposed to evaluate potential surrogate markers, available methods generally do not account for or address the potential for a surrogate to vary in utility or strength by patient characteristics. Previous work examining surrogate markers has indicated that there may be such heterogeneity i.e., that a surrogate marker may be useful (with respect to capturing the treatment effect on the primary outcome) for some subgroups, but not for others. This heterogeneity is important to understand, particularly if the surrogate is to be used in a future trial to replace the primary outcome. In this paper, we propose an approach and estimation procedures to measure the surrogate strength as a function of a baseline covariate W and thus, examine potential heterogeneity in the utility of the surrogate marker with respect to W. Within a potential outcome framework, we quantify the surrogate strength/utility using the proportion of treatment effect on the primary outcome that is explained by the treatment effect on the surrogate. We propose testing procedures to test for evidence of heterogeneity, examine finite sample performance of these methods via simulation, and illustrate the methods using AIDS clinical trial data.
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