根据相对效果测量的可迁移性假设,了解新目标人群的治疗效果。

IF 7.7 1区 医学 Q1 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH European Journal of Epidemiology Pub Date : 2024-09-01 Epub Date: 2024-05-10 DOI:10.1007/s10654-023-01067-4
Issa J Dahabreh, Sarah E Robertson, Jon A Steingrimsson
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引用次数: 0

摘要

研究人员通常认为,以风险比和均值比等协变因素为条件的相对效应量可以跨人群 "传递"。在此,我们假设条件相对效应量可以从试验转移到目标人群,并以此为基础研究目标人群中因果效应的识别。我们表明,相对效应测量的可迁移性与差异效应测量的可迁移性在很大程度上是不相容的,除非治疗对平均水平没有影响,或者人们愿意做出更强的可迁移性假设,这意味着相对效应测量和差异效应测量都具有可迁移性。然后,我们描述了当我们对一种新的试验性治疗方法在目标人群中的有效性感兴趣时,如何在相对效应测量的可迁移性假设下确定目标人群中的边际(人群平均)因果估计值,而在目标人群中使用的唯一治疗方法就是试验中评估的对照治疗方法。我们将这些结果扩展到考虑试验中评估的对照治疗只是目标人群中使用的治疗方法之一的情况,并额外假设目标人群中存在部分可交换性(即假设目标人群中没有与试验中对照治疗下的潜在结果相关的未测量混杂因素)。我们还开发了识别结果,允许相对效应测量可迁移性所需的协变量只是控制目标人群混杂所需协变量的一小部分。最后,我们提出了可以在标准统计软件中轻松实现的估计方法,并使用一项关于稳定型缺血性心脏病的综合队列研究数据对其使用进行了说明。
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Learning about treatment effects in a new target population under transportability assumptions for relative effect measures.

Investigators often believe that relative effect measures conditional on covariates, such as risk ratios and mean ratios, are "transportable" across populations. Here, we examine the identification of causal effects in a target population using an assumption that conditional relative effect measures are transportable from a trial to the target population. We show that transportability for relative effect measures is largely incompatible with transportability for difference effect measures, unless the treatment has no effect on average or one is willing to make even stronger transportability assumptions that imply the transportability of both relative and difference effect measures. We then describe how marginal (population-averaged) causal estimands in a target population can be identified under the assumption of transportability of relative effect measures, when we are interested in the effectiveness of a new experimental treatment in a target population where the only treatment in use is the control treatment evaluated in the trial. We extend these results to consider cases where the control treatment evaluated in the trial is only one of the treatments in use in the target population, under an additional partial exchangeability assumption in the target population (i.e., an assumption of no unmeasured confounding in the target population with respect to potential outcomes under the control treatment in the trial). We also develop identification results that allow for the covariates needed for transportability of relative effect measures to be only a small subset of the covariates needed to control confounding in the target population. Last, we propose estimators that can be easily implemented in standard statistical software and illustrate their use using data from a comprehensive cohort study of stable ischemic heart disease.

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来源期刊
European Journal of Epidemiology
European Journal of Epidemiology 医学-公共卫生、环境卫生与职业卫生
CiteScore
21.40
自引率
1.50%
发文量
109
审稿时长
6-12 weeks
期刊介绍: The European Journal of Epidemiology, established in 1985, is a peer-reviewed publication that provides a platform for discussions on epidemiology in its broadest sense. It covers various aspects of epidemiologic research and statistical methods. The journal facilitates communication between researchers, educators, and practitioners in epidemiology, including those in clinical and community medicine. Contributions from diverse fields such as public health, preventive medicine, clinical medicine, health economics, and computational biology and data science, in relation to health and disease, are encouraged. While accepting submissions from all over the world, the journal particularly emphasizes European topics relevant to epidemiology. The published articles consist of empirical research findings, developments in methodology, and opinion pieces.
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