Institut für Epidemiologie, Biostatistik und Prävention, Departement Biostatistik, Universität Zürich, Hirschengraben 84, CH-8001 Zürich, Switzerland.
Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, 171 77 Stockholm, Sweden.
Biostatistics. 2022 Oct 14;23(4):1083-1098. doi: 10.1093/biostatistics/kxab045.
One-stage meta-analysis of individual participant data (IPD) poses several statistical and computational challenges. For time-to-event outcomes, the approach requires the estimation of complicated nonlinear mixed-effects models that are flexible enough to realistically capture the most important characteristics of the IPD. We present a model class that incorporates general normally distributed random effects into linear transformation models. We discuss extensions to model between-study heterogeneity in baseline risks and covariate effects and also relax the assumption of proportional hazards. Within the proposed framework, data with arbitrary random censoring patterns can be handled. The accompanying $\textsf{R}$ package tramME utilizes the Laplace approximation and automatic differentiation to perform efficient maximum likelihood estimation and inference in mixed-effects transformation models. We compare several variants of our model to predict the survival of patients with chronic obstructive pulmonary disease using a large data set of prognostic studies. Finally, a simulation study is presented that verifies the correctness of the implementation and highlights its efficiency compared to an alternative approach.
一项针对个体参与者数据(IPD)的单阶段荟萃分析提出了一些统计和计算方面的挑战。对于生存时间结局,该方法需要估计复杂的非线性混合效应模型,该模型需要足够灵活,以真实地捕捉 IPD 的最重要特征。我们提出了一个模型类别,将一般正态分布的随机效应纳入线性变换模型。我们讨论了扩展模型以模拟基线风险和协变量效应之间的异质性,并放宽了比例风险假设。在所提出的框架内,可以处理具有任意随机删失模式的数据。随附的 tramME $\textsf{R}$ 包利用拉普拉斯近似和自动微分来执行混合效应变换模型中的高效最大似然估计和推断。我们将我们的模型的几个变体进行比较,以使用大型预后研究数据集预测慢性阻塞性肺疾病患者的生存情况。最后,进行了一项模拟研究,以验证实现的正确性,并强调与替代方法相比的效率。