Preisser John S, Young Mary L, Zaccaro Daniel J, Wolfson Mark
Department of Biostatistics, University of North Carolina School of Public Health, Chapel Hill, North Carolina 27599-7420, USA.
Stat Med. 2003 Apr 30;22(8):1235-54. doi: 10.1002/sim.1379.
While the mixed model approach to cluster randomization trials is relatively well developed, there has been less attention given to the design and analysis of population-averaged models for randomized and non-randomized cluster trials. We provide novel implementations of familiar methods to meet these needs. A design strategy that selects matching control communities based upon propensity scores, a statistical analysis plan for dichotomous outcomes based upon generalized estimating equations (GEE) with a design-based working correlation matrix, and new sample size formulae are applied to a large non-randomized study to reduce underage drinking. The statistical power calculations, based upon Wald tests for summary statistics, are special cases of a general power method for GEE.
虽然用于整群随机试验的混合模型方法相对较为成熟，但对于随机和非随机整群试验的总体平均模型的设计和分析关注较少。我们提供了一些常用方法的新颖实现方式来满足这些需求。一种基于倾向得分选择匹配对照社区的设计策略、一种基于具有基于设计的工作相关矩阵的广义估计方程（GEE）的二分结局统计分析计划以及新的样本量公式被应用于一项大型非随机研究以减少未成年人饮酒。基于Wald检验汇总统计量的统计功效计算是GEE一般功效方法的特殊情况。
