在存在潜在随机遗漏(LMAR)结果的情况下,识别合意者和非合意者的平均因果效应:统一观点和假设选择。

IF 1.8 3区 数学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY Biostatistics Pub Date : 2024-04-05 DOI:10.1093/biostatistics/kxae011
Trang Quynh Nguyen, Michelle C Carlson, Elizabeth A Stuart
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引用次数: 0

摘要

对治疗效果的研究通常会因违规和数据缺失而变得复杂。在单侧不遵医嘱的情况下,我们关注的是遵医嘱者和非遵医嘱者的平均因果效应,我们处理的是潜在随机缺失类型的结果缺失(LMAR,也称为潜在无知)。也就是说,在协变量和治疗分配的条件下,遗漏率可能取决于遵从类型。在处理不遵从的工具变量(IV)方法中,已经提出了处理 LMAR 结果的方法,这些方法额外援引了关于缺失的排除限制型假设,但还没有提出使用非 IV 方法时的解决方案。本文重点讨论存在 LMAR 结果时的效应识别,以期灵活地适应不同的主要识别方法。我们的研究表明,仅在处理分配无知和 LMAR 的情况下,效应不可识别性可归结为涉及未识别的特定分层反应概率和结果均值的两个相连混合方程组。这就说明(除特殊情况外)效应识别一般需要两个额外的假设:特定的遗漏机制假设和主要识别假设。这为根据这些假设的不同选择来识别效应提供了一个模板。我们考虑了一系列特定的缺失假设,包括文献中出现过的假设和一些新的假设。顺便提一下,我们发现了现有假设中的一个问题,并提出了修改假设以避免该问题的建议。我们使用巴尔的摩体验团试验的数据说明了不同假设下的结果。
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Identification of complier and noncomplier average causal effects in the presence of latent missing-at-random (LMAR) outcomes: a unifying view and choices of assumptions.

The study of treatment effects is often complicated by noncompliance and missing data. In the one-sided noncompliance setting where of interest are the complier and noncomplier average causal effects, we address outcome missingness of the latent missing at random type (LMAR, also known as latent ignorability). That is, conditional on covariates and treatment assigned, the missingness may depend on compliance type. Within the instrumental variable (IV) approach to noncompliance, methods have been proposed for handling LMAR outcome that additionally invoke an exclusion restriction-type assumption on missingness, but no solution has been proposed for when a non-IV approach is used. This article focuses on effect identification in the presence of LMAR outcomes, with a view to flexibly accommodate different principal identification approaches. We show that under treatment assignment ignorability and LMAR only, effect nonidentifiability boils down to a set of two connected mixture equations involving unidentified stratum-specific response probabilities and outcome means. This clarifies that (except for a special case) effect identification generally requires two additional assumptions: a specific missingness mechanism assumption and a principal identification assumption. This provides a template for identifying effects based on separate choices of these assumptions. We consider a range of specific missingness assumptions, including those that have appeared in the literature and some new ones. Incidentally, we find an issue in the existing assumptions, and propose a modification of the assumptions to avoid the issue. Results under different assumptions are illustrated using data from the Baltimore Experience Corps Trial.

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来源期刊
Biostatistics
Biostatistics 生物-数学与计算生物学
CiteScore
5.10
自引率
4.80%
发文量
45
审稿时长
6-12 weeks
期刊介绍: Among the important scientific developments of the 20th century is the explosive growth in statistical reasoning and methods for application to studies of human health. Examples include developments in likelihood methods for inference, epidemiologic statistics, clinical trials, survival analysis, and statistical genetics. Substantive problems in public health and biomedical research have fueled the development of statistical methods, which in turn have improved our ability to draw valid inferences from data. The objective of Biostatistics is to advance statistical science and its application to problems of human health and disease, with the ultimate goal of advancing the public''s health.
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