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用于具有线性和对数线性增长的纵向干预研究的贝叶斯样本量确定

Bayesian sample size determination for longitudinal intervention studies with linear and log-linear growth.

作者信息

Lösener Ulrich, Moerbeek Mirjam

机构信息

Department of Methodology and Statistics, Utrecht University, Utrecht, Netherlands.

出版信息

Behav Res Methods. 2025 Jul 28;57(9):239. doi: 10.3758/s13428-025-02749-5.

Abstract

A priori sample size determination (SSD) is essential for designing cost-efficient trials and in avoiding underpowered studies. In addition, reporting a solid justification for a certain sample size is required by most ethics committees and many funding agencies. Often, SSD is based on null hypothesis significance testing (NHST), an approach that has received severe criticism in the past decades. As an alternative, Bayesian hypothesis evaluation using Bayes factors has been developed. Bayes factors quantify the relative support in the data for a pair of competing hypotheses without suffering from some of the drawbacks of NHST. SSD for Bayesian hypothesis testing relies on simulations and has only been studied recently. Available software for this is limited to simple models such as ANOVA and the t test, in which observations are assumed to be independent from each other. However, this assumption is rendered untenable in longitudinal experiments where observations are nested within individuals. In that case, a multilevel model should be used. This paper provides researchers with a valuable tool for performing SSD for multilevel models with longitudinal data in a Bayesian framework, along with the necessary theoretical background and concrete empirical examples. The open-source R function that enables researchers to tailor the simulation to their trial at hand can be found on the GitHub page of the first author.

摘要

先验样本量确定(SSD)对于设计具有成本效益的试验以及避免效能不足的研究至关重要。此外,大多数伦理委员会和许多资助机构都要求为特定样本量提供充分的理由。通常,SSD基于零假设显著性检验(NHST),而在过去几十年中,这种方法受到了严厉批评。作为一种替代方法,已经开发了使用贝叶斯因子的贝叶斯假设评估。贝叶斯因子量化了数据中对一对相互竞争假设的相对支持程度,而不会受到NHST的一些缺点的影响。贝叶斯假设检验的SSD依赖于模拟,并且直到最近才得到研究。为此可用的软件仅限于诸如方差分析(ANOVA)和t检验等简单模型,其中假设观测值相互独立。然而在纵向实验中,观测值嵌套在个体内部,这种假设就站不住脚了。在这种情况下,应该使用多层模型。本文为研究人员提供了一个有价值的工具,用于在贝叶斯框架下对具有纵向数据的多层模型进行SSD,并提供了必要的理论背景和具体的实证例子。能够让研究人员根据手头试验调整模拟的开源R函数可在第一作者的GitHub页面上找到。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b5a0/12304047/4593c946d91b/13428_2025_2749_Fig1_HTML.jpg

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