Lai Mark H C, Kwok Oi-Man
a University of Cincinnati.
b Texas A&M University.
Multivariate Behav Res. 2016 Nov-Dec;51(6):740-756. doi: 10.1080/00273171.2016.1231606. Epub 2016 Nov 1.
Although previous research has discussed an effect size estimator for partially nested cluster randomized designs, the existing estimator (a) is not efficient when used with primary data, (b) can be biased when the homogeneity of variance assumption is violated, and (c) has not yet been empirically evaluated for its finite sample properties. The present paper addresses these limitations by proposing an alternative maximum likelihood estimator for obtaining standardized mean difference effect size and the corresponding sampling variance for partially nested data, as well as the variants that do not make an assumption of homogeneity of variance. The typical estimator, denoted as d (d with pooled SD and d with control arm SD), requires input of summary statistics such as observed means, variances, and the intraclass correlation, and is useful for meta-analyses and secondary data analyses; the newly proposed estimator [Formula: see text] ([Formula: see text] and [Formula: see text]) takes parameter estimates from a correctly specified multilevel model as input and is mainly of interest to researchers doing primary research. The simulation results showed that the two methods (d and [Formula: see text]) produced unbiased point and variance estimates for effect size. As expected, in general, [Formula: see text] was more efficient than d with unequal cluster sizes, especially with large average cluster size and large intraclass correlation. Furthermore, under heterogeneous variances, [Formula: see text] demonstrated a greater relative efficiency with small sample size for the unclustered control arm. Real data examples, one from a youth preventive program and one from an eating disorder intervention, were used to demonstrate the methods presented. In addition, we extend the discussion to a scenario with a three-level treatment arm and an unclustered control arm, and illustrate the procedures for effect size estimation using a hypothetical example of multiple therapy groups of clients clustered within therapists.
尽管先前的研究已经讨论了部分嵌套聚类随机设计的效应量估计器,但现有的估计器(a)在用于原始数据时效率不高,(b)在方差齐性假设被违反时可能会有偏差,并且(c)尚未对其有限样本性质进行实证评估。本文通过提出一种替代的最大似然估计器来解决这些局限性,该估计器用于获取部分嵌套数据的标准化均数差异效应量和相应的抽样方差,以及不做方差齐性假设的变体。典型的估计器,记为d(合并标准差的d和对照组标准差的d),需要输入诸如观察到的均值、方差和组内相关等汇总统计量,并且对荟萃分析和二次数据分析很有用;新提出的估计器[公式:见原文]([公式:见原文]和[公式:见原文])将来自正确指定的多水平模型的参数估计作为输入,主要是进行原始研究的研究人员感兴趣。模拟结果表明,两种方法(d和[公式:见原文])对效应量产生了无偏的点估计和方差估计。正如预期的那样,一般来说,[公式:见原文]在聚类大小不相等时比d更有效,特别是在平均聚类大小大且组内相关大的情况下。此外,在方差不齐的情况下,[公式:见原文]在无聚类对照组样本量较小时表现出更高的相对效率。使用了一个来自青少年预防项目和一个来自饮食失调干预的实际数据例子来演示所提出的方法。此外,我们将讨论扩展到一个具有三级治疗组和无聚类对照组的场景,并使用一个假设的例子说明效应量估计的程序,该例子是在治疗师内聚类的多个客户治疗组。