Josey Kevin P, Ringham Brandy M, Barón Anna E, Schenkman Margaret, Sauder Katherine A, Muller Keith E, Dabelea Dana, Glueck Deborah H
Department of Biostatistics and Informatics, Colorado School of Public Health, University of Colorado Denver, Denver, Colorado, USA.
Lifecourse Epidemiology of Adiposity and Disease (LEAD) Center, Colorado School of Public Health, University of Colorado Denver, Denver, Colorado, USA.
Commun Stat Theory Methods. 2023;52(1):46-64. doi: 10.1080/03610926.2021.1909732. Epub 2021 Apr 5.
When designing repeated measures studies, both the amount and the pattern of missing outcome data can affect power. The chance that an observation is missing may vary across measurements, and missingness may be correlated across measurements. For example, in a physiotherapy study of patients with Parkinson's disease, increasing intermittent dropout over time yielded missing measurements of physical function. In this example, we assume data are missing completely at random, since the chance that a data point was missing appears to be unrelated to either outcomes or covariates. For data missing completely at random, we propose noncentral power approximations for the Wald test for balanced linear mixed models with Gaussian responses. The power approximations are based on moments of missing data summary statistics. The moments were derived assuming a conditional linear missingness process. The approach provides approximate power for both complete-case analyses, which include independent sampling units where all measurements are present, and observed-case analyses, which include all independent sampling units with at least one measurement. Monte Carlo simulations demonstrate the accuracy of the method in small samples. We illustrate the utility of the method by computing power for proposed replications of the Parkinson's study.
在设计重复测量研究时,缺失结局数据的数量和模式都会影响检验效能。某次观测数据缺失的可能性可能因测量而异,并且缺失情况可能在各次测量之间存在相关性。例如,在一项针对帕金森病患者的物理治疗研究中,随着时间推移间歇性失访的增加导致了身体功能测量数据的缺失。在这个例子中,我们假定数据是完全随机缺失的,因为某个数据点缺失的可能性似乎与结局或协变量均无关。对于完全随机缺失的数据,我们针对具有高斯响应的平衡线性混合模型的Wald检验提出了非中心检验效能近似值。检验效能近似值基于缺失数据汇总统计量的矩。这些矩是在假定条件线性缺失过程的情况下推导得出的。该方法为完全病例分析(包括所有测量值均存在的独立抽样单元)和观察病例分析(包括至少有一次测量值的所有独立抽样单元)都提供了近似检验效能。蒙特卡罗模拟证明了该方法在小样本中的准确性。我们通过计算帕金森病研究拟进行的重复研究的检验效能来说明该方法的实用性。