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利用目标治疗估计值消除模糊的治疗效果。

Eliminating Ambiguous Treatment Effects Using Estimands.

出版信息

Am J Epidemiol. 2023 Jun 2;192(6):987-994. doi: 10.1093/aje/kwad036.


DOI:10.1093/aje/kwad036
PMID:36790803
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10236519/
Abstract

Most reported treatment effects in medical research studies are ambiguously defined, which can lead to misinterpretation of study results. This is because most authors do not attempt to describe what the treatment effect represents, and instead require readers to deduce this based on the reported statistical methods. However, this approach is challenging, because many methods provide counterintuitive results. For example, some methods include data from all patients, yet the resulting treatment effect applies only to a subset of patients, whereas other methods will exclude certain patients while results will apply to everyone. Additionally, some analyses provide estimates pertaining to hypothetical settings in which patients never die or discontinue treatment. Herein we introduce estimands as a solution to the aforementioned problem. An estimand is a clear description of what the treatment effect represents, thus saving readers the necessity of trying to infer this from study methods and potentially getting it wrong. We provide examples of how estimands can remove ambiguity from reported treatment effects and describe their current use in practice. The crux of our argument is that readers should not have to infer what investigators are estimating; they should be told explicitly.

摘要

大多数医学研究报告中的治疗效果定义不明确,这可能导致对研究结果的误解。这是因为大多数作者并没有试图描述治疗效果代表什么,而是要求读者根据报告的统计方法来推断。然而,这种方法具有挑战性,因为许多方法提供了违反直觉的结果。例如,一些方法包括所有患者的数据,但得出的治疗效果仅适用于患者的一部分子集,而其他方法则会排除某些患者,而结果将适用于所有人。此外,一些分析提供了与患者永不死亡或停止治疗的假设情况相关的估计。在这里,我们引入估计目标作为解决上述问题的一种方法。估计目标是对治疗效果代表什么的清晰描述,从而避免读者试图从研究方法中推断出这一点,并且可能会推断错误。我们提供了一些示例,说明估计目标如何消除报告的治疗效果中的模糊性,并描述了它们在实践中的当前使用情况。我们论点的关键是,读者不应该推断研究人员正在估计什么;他们应该被明确告知。

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本文引用的文献

[1]
Using modified intention-to-treat as a principal stratum estimator for failure to initiate treatment.

Clin Trials. 2023-6

[2]
Single-Dose Psilocybin for a Treatment-Resistant Episode of Major Depression.

N Engl J Med. 2022-11-3

[3]
Evaluating how clear the questions being investigated in randomised trials are: systematic review of estimands.

BMJ. 2022-8-23

[4]
Estimands in cluster-randomized trials: choosing analyses that answer the right question.

Int J Epidemiol. 2023-2-8

[5]
Estimation of treatment effects in short-term depression studies. An evaluation based on the ICH E9(R1) estimands framework.

Pharm Stat. 2022-9

[6]
Re-randomisation trials in multi-episode settings: Estimands and independence estimators.

Stat Methods Med Res. 2022-7

[7]
Early phase clinical trials extension to guidelines for the content of statistical analysis plans.

BMJ. 2022-2-7

[8]
Estimands in observational studies: Some considerations beyond ICH E9 (R1).

Pharm Stat. 2022-9

[9]
Estimands: bringing clarity and focus to research questions in clinical trials.

BMJ Open. 2022-1-3

[10]
Independence estimators for re-randomisation trials in multi-episode settings: a simulation study.

BMC Med Res Methodol. 2021-10-30

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