Chen Chyong-Mei, Shen Pao-Sheng, Lin Chih-Ching, Wu Chih-Cheng
Institute of Public Health, School of Medicine, National Yang-Ming University, Taipei, Taiwan, R.O.C.
Department of Statistics, Tugman University, Taichung, Taiwan, R.O.C.
Stat Med. 2020 Nov 30;39(27):4086-4099. doi: 10.1002/sim.8711. Epub 2020 Aug 13.
The article is motivated by a nephrology study in Taiwan, which enrolled hemodialysis patients who suffered from vascular access thrombosis. After treatment, some patients were cured of thrombosis, while some may experience recurrence of either type (acute or nonacute) of vascular access thrombosis. Our major interest is to estimate the cumulative incidence probability of time to the first recurrence of acute thrombosis after therapy. Since the occurrence of one type of vascular access thrombosis precludes occurrence of the other type, patients are subject to competing risks. To account for the presence of competing risks and cured patients, we develop a mixture model approach to the regression analysis of competing-risks data with a cure fraction. We make inference about the effects of factors on both the cure rate and cumulative incidence function (CIF) for a failure of interest, which are separately specified in the logistic regression model and semiparametric regression model with time-varying and time-invariant effects. Based on two-stage method, we develop novel estimation equations using the inverse probability censoring weight techniques. The asymptotic properties of the estimators are rigorously studied and the plug-in variance estimators can be obtained for constructing interval estimators. We also propose a lack-of-fit test for assessing the adequacy of the proposed model and several tests for time-varying effects. The simulation studies and vascular access thrombosis data analysis are conducted to illustrate the proposed method.
本文受台湾一项肾脏病学研究的启发，该研究招募了患有血管通路血栓形成的血液透析患者。治疗后，一些患者的血栓形成得到治愈，而一些患者可能会经历血管通路血栓形成（急性或非急性）的复发。我们主要关注的是估计治疗后急性血栓形成首次复发时间的累积发生率概率。由于一种类型的血管通路血栓形成的发生排除了另一种类型的发生，患者面临竞争风险。为了考虑竞争风险和治愈患者的存在，我们开发了一种混合模型方法，用于对具有治愈比例的竞争风险数据进行回归分析。我们推断因素对治愈率和感兴趣的失败的累积发生率函数（CIF）的影响，这些影响在逻辑回归模型和具有时变和时不变效应的半参数回归模型中分别指定。基于两阶段方法，我们使用逆概率删失权重技术开发了新的估计方程。对估计量的渐近性质进行了严格研究，并可以获得插件方差估计量来构建区间估计量。我们还提出了一个拟合优度检验来评估所提出模型的充分性，以及几个用于时变效应的检验。进行了模拟研究和血管通路血栓形成数据分析以说明所提出的方法。
