Dugourd Charlotte, Abichou-Klich Amna, Ecochard René, Subtil Fabien
Service de Biostatistique, Hospices Civils de Lyon, Lyon, France.
Université de Lyon, Lyon, France.
Stat Med. 2023 Nov 10;42(25):4570-4581. doi: 10.1002/sim.9876. Epub 2023 Aug 14.
Classifying patient biomarker trajectories into groups has become frequent in clinical research. Mixed effects classification models can be used to model the heterogeneity of longitudinal data. The estimated parameters of typical trajectories and the partition can be provided by the classification version of the expectation maximization algorithm, named CEM. However, the variance of the parameter estimates obtained underestimates the true variance because classification uncertainties are not taken into account. This article takes into account these uncertainties by using the stochastic EM algorithm (SEM), a stochastic version of the CEM algorithm, after convergence of the CEM algorithm. The simulations showed correct coverage probabilities of the 95% confidence intervals (close to 95% except for scenarios with high bias in typical trajectories). The method was applied on a trial, called low-cyclo, that compared the effects of low vs standard cyclosporine A doses on creatinine levels after cardiac transplantation. It identified groups of patients for whom low-dose cyclosporine may be relevant, but with high uncertainty on the dose-effect estimate.
在临床研究中,将患者生物标志物轨迹分类成组已变得很常见。混合效应分类模型可用于对纵向数据的异质性进行建模。典型轨迹的估计参数和划分可由期望最大化算法的分类版本(称为CEM)提供。然而,由于未考虑分类不确定性,所获得的参数估计值的方差低估了真实方差。本文在CEM算法收敛后,通过使用随机期望最大化算法(SEM)(CEM算法的随机版本)来考虑这些不确定性。模拟显示95%置信区间的正确覆盖概率(除典型轨迹存在高偏差的情况外,接近95%)。该方法应用于一项名为低环孢素的试验,该试验比较了低剂量与标准剂量环孢素A对心脏移植后肌酐水平的影响。它识别出了低剂量环孢素可能适用的患者组,但剂量效应估计存在高度不确定性。