Department of Statistical Data Science, The Institute of Statistical Mathematics, Tachikawa, Tokyo, Japan.
Clinical Science and Strategy Department, Chugai Pharmaceutical Co., Ltd., Chuo-ku, Tokyo, Japan.
Biom J. 2020 Mar;62(2):350-360. doi: 10.1002/bimj.201800389. Epub 2019 Aug 8.
For continuous variables of randomized controlled trials, recently, longitudinal analysis of pre- and posttreatment measurements as bivariate responses is one of analytical methods to compare two treatment groups. Under random allocation, means and variances of pretreatment measurements are expected to be equal between groups, but covariances and posttreatment variances are not. Under random allocation with unequal covariances and posttreatment variances, we compared asymptotic variances of the treatment effect estimators in three longitudinal models. The data-generating model has equal baseline means and variances, and unequal covariances and posttreatment variances. The model with equal baseline means and unequal variance-covariance matrices has a redundant parameter. In large sample sizes, these two models keep a nominal type I error rate and have high efficiency. The model with equal baseline means and equal variance-covariance matrices wrongly assumes equal covariances and posttreatment variances. Only under equal sample sizes, this model keeps a nominal type I error rate. This model has the same high efficiency with the data-generating model under equal sample sizes. In conclusion, longitudinal analysis with equal baseline means performed well in large sample sizes. We also compared asymptotic properties of longitudinal models with those of the analysis of covariance (ANCOVA) and t-test.
对于随机对照试验的连续变量,最近,作为双变量反应的预处理和后处理测量的纵向分析是比较两组治疗方法的分析方法之一。在随机分配下,各组之间的预处理测量的平均值和方差预计是相等的,但协方差和后处理方差则不然。在协方差和后处理方差不等的随机分配下,我们比较了三种纵向模型中治疗效果估计值的渐近方差。数据生成模型具有相等的基线均值和方差,以及不相等的协方差和后处理方差。具有相等基线均值和不相等方差协方差矩阵的模型具有冗余参数。在大样本量下,这两个模型保持名义第一类错误率并具有高效率。具有相等基线均值和相等方差协方差矩阵的模型错误地假设了相等的协方差和后处理方差。只有在相等的样本量下,该模型才保持名义第一类错误率。在相等的样本量下,该模型与数据生成模型具有相同的高效率。总之,具有相等基线均值的纵向分析在大样本量下表现良好。我们还比较了纵向模型与协方差分析(ANCOVA)和 t 检验的渐近性质。