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多中心临床试验中评估治疗效果的随机化模型方法。

Randomization model methods for evaluating treatment efficacy in multicenter clinical trials.

作者信息

Davis C S, Chung Y

机构信息

Department of Preventive Medicine and Environmental Health, University of Iowa, Iowa City 52242, USA.

出版信息

Biometrics. 1995 Sep;51(3):1163-74.

PMID:7548700
Abstract

This paper studies randomization model methods for analyzing data from a multicenter study comparing the effectiveness of two treatments. The Mantel-Haenszel mean score statistic, which can be used for continuous or ordered categorical response variables, is shown to be a useful nonparametric alternative to standard linear model methods for testing the significance of the average treatment difference. In an extensive simulation study, the mean score test performs nearly as well as the optimal linear model methods when the normal-theory assumptions are satisfied. A related estimator of the average treatment difference is also studied. This estimator, which is a weighted average of the center-specific mean differences, is analogous to the commonly used Mantel-Haenszel estimator of the average odds ratio in stratified 2 x 2 contingency tables. The proposed estimator is equivalent to the fixed-effects analysis of variance estimator from the main effects model, and valid estimation of its variance is feasible under very general assumptions.

摘要

本文研究了用于分析多中心研究数据的随机化模型方法,该研究比较了两种治疗方法的有效性。曼特尔 - 亨泽尔平均得分统计量可用于连续或有序分类响应变量,它被证明是用于检验平均治疗差异显著性的标准线性模型方法的一种有用的非参数替代方法。在一项广泛的模拟研究中,当满足正态理论假设时,平均得分检验的表现几乎与最优线性模型方法一样好。还研究了平均治疗差异的相关估计量。该估计量是中心特定平均差异的加权平均值,类似于分层2×2列联表中常用的平均优势比的曼特尔 - 亨泽尔估计量。所提出的估计量等同于主效应模型的固定效应方差分析估计量,并且在非常一般的假设下对其方差进行有效估计是可行的。

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引用本文的文献

1
MULTI-CENTER CLINICAL TRIALS: RANDOMIZATION AND ANCILLARY STATISTICS.多中心临床试验:随机化与辅助统计
Ann Appl Stat. 2008 Jun;2(2):582-600. doi: 10.1214/07-AOAS151. Epub 2008 Jul 3.