随机化技术对前后设计模型性能的影响。

IF 3.9 3区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Xinlin Lu, Yahui Zhang, Samuel S Wu, Guogen Shan
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引用次数: 0

摘要

前后设计被广泛应用于临床试验和实验研究中,以评估治疗的有效性。分析前后数据的常用统计方法包括使用后处理或基线变化的方差分析(ANOVA),均匀或非均匀斜率的协方差分析(ANCOVA)以及线性混合模型(LMM)。虽然有许多研究比较了这些方法,但有限的研究调查了在不同随机化方法下调整有影响的基线协变量的影响。在这项研究中,我们进行了一系列全面的模拟研究,以调查在几种随机化方法下调整基线协变量的影响:简单随机化、分层块随机化和使用Pocock和Simon的最小化方法的协变量自适应随机化。结果表明,当随机化方法中不考虑协变量时,两种ANCOVA方法都具有良好的性能。调整相关的基线协变量导致了大量的功率增益,这些增益的程度取决于协变量效应的大小和所采用的随机化方法。在调整协变量后,分层块随机化和协变量自适应随机化在功率增益方面始终优于简单随机化,随着协变量数量的增加,协变量自适应随机化变得更加优越。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The impact of randomization techniques on the performance of pre-post design models.

Pre-post designs are widely used in clinical trials and experimental studies to assess the effectiveness of treatments. Common statistical methods for analyzing pre-post data include analysis of variance (ANOVA) using post-treatment or the change from baseline, analysis of covariance (ANCOVA) with homogeneous or heterogeneous slopes, and linear mixed models (LMM). While numerous studies have compared these methods, limited studies have investigated the impact of adjusting for influential baseline covariates under different randomization approaches. In this study, we conducted a series of comprehensive simulation studies to investigate the impact of adjusting baseline covariates under several randomization approaches: simple randomization, stratified block randomization, and covariate adaptive randomization using the minimization method by Pocock and Simon. Results demonstrated that when no covariates were considered in the randomization approach, the two ANCOVA methods always have good performance. Adjusting for relevant baseline covariates led to substantial power gains, with the extent of these gains depending on the size of the covariate effects and the randomization approach employed. Stratified block randomization and covariate adaptive randomization consistently outperformed simple randomization in terms of power gains after adjusting for covariates, with covariate adaptive randomization becoming more superior as the number of covariates increased.

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来源期刊
BMC Medical Research Methodology
BMC Medical Research Methodology 医学-卫生保健
CiteScore
6.50
自引率
2.50%
发文量
298
审稿时长
3-8 weeks
期刊介绍: BMC Medical Research Methodology is an open access journal publishing original peer-reviewed research articles in methodological approaches to healthcare research. Articles on the methodology of epidemiological research, clinical trials and meta-analysis/systematic review are particularly encouraged, as are empirical studies of the associations between choice of methodology and study outcomes. BMC Medical Research Methodology does not aim to publish articles describing scientific methods or techniques: these should be directed to the BMC journal covering the relevant biomedical subject area.
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