Xue Xiaonan, Xie Xianhong, Strickler Howard D
Department of Epidemiology and Population Health, Albert Einstein College of Medicine, Bronx, NY, USA.
Stat Methods Med Res. 2018 Mar;27(3):955-965. doi: 10.1177/0962280216648724. Epub 2016 May 10.
The commonly used statistical model for studying time to event data, the Cox proportional hazards model, is limited by the assumption of a constant hazard ratio over time (i.e., proportionality), and the fact that it models the hazard rate rather than the survival time directly. The censored quantile regression model, defined on the quantiles of time to event, provides an alternative that is more flexible and interpretable. However, the censored quantile regression model has not been widely adopted in clinical research, due to the complexity involved in interpreting its results properly and consequently the difficulty to appreciate its advantages over the Cox proportional hazards model, as well as the absence of adequate validation procedure. In this paper, we addressed these limitations by (1) using both simulated examples and data from National Wilms' Tumor clinical trials to illustrate proper interpretation of the censored quantile regression model and the differences and the advantages of the model compared to the Cox proportional hazards model; and (2) developing a validation procedure for the predictive censored quantile regression model. The performance of this procedure was examined using simulation studies. Overall, we recommend the use of censored quantile regression model, which permits a more sensitive analysis of time to event data together with the Cox proportional hazards model.
用于研究事件发生时间数据的常用统计模型——Cox比例风险模型,受到随时间风险比例恒定(即比例性)这一假设的限制,且它直接建模的是风险率而非生存时间。基于事件发生时间分位数定义的删失分位数回归模型提供了一种更灵活且更具可解释性的替代方法。然而,删失分位数回归模型在临床研究中尚未得到广泛应用,这是由于正确解释其结果存在复杂性,进而难以认识到它相对于Cox比例风险模型的优势,以及缺乏适当的验证程序。在本文中,我们通过以下方式解决了这些局限性:(1)使用模拟示例和来自国家肾母细胞瘤临床试验的数据来说明对删失分位数回归模型的正确解释,以及该模型与Cox比例风险模型相比的差异和优势;(2)为预测性删失分位数回归模型开发一种验证程序。通过模拟研究检验了该程序的性能。总体而言,我们建议使用删失分位数回归模型,它可以与Cox比例风险模型一起对事件发生时间数据进行更敏感的分析。