Department of Epidemiology and Biostatistics, Graduate School of Public Health and Health Policy, Institute for Implementation Science in Population Health, City University of New York, New York, New York, United States.
School of Medicine, University of Cuenca, Cuenca, Ecuador.
Stat Med. 2020 Oct 15;39(23):3195-3206. doi: 10.1002/sim.8663. Epub 2020 Jun 25.
We propose a multistate joint model to analyze interval-censored event-history data subject to within-unit clustering and nonignorable missing data. The model is motivated by a study of the neurocysticercosis (NC) cyst evolution at the cyst-level, taking into account the multiple cysts phases with intermittent missing data and loss to follow-up, as well as the intra-brain clustering of observations made on a predefined data collection schedule. Of particular interest in this study is the description of the process leading to cyst resolution, and whether this process varies by antiparasitic treatment. The model uses shared random effects to account for within-brain correlation and to explain the hidden heterogeneity governing the missing data mechanism. We developed a likelihood-based method using a Monte Carlo EM algorithm for the inference. The practical utility of the methods is illustrated using data from a randomized controlled trial on the effect of antiparasitic treatment with albendazole on NC cysts among patients from six hospitals in Ecuador. Simulation results demonstrate that the proposed methods perform well in the finite sample and misspecified models that ignore the data complexities could lead to substantial biases.
我们提出了一个多状态联合模型,用于分析存在单位内聚类和不可忽略缺失数据的区间删失事件历史数据。该模型的动机是研究神经囊尾蚴病(NC)囊泡在囊泡水平上的演变,考虑到具有间歇性缺失数据和随访丢失的多个囊泡阶段,以及在预先定义的数据收集计划上进行的脑内观察的内在聚类。在这项研究中,特别感兴趣的是描述导致囊泡消退的过程,以及这个过程是否因抗寄生虫治疗而有所不同。该模型使用共享随机效应来解释脑内相关性,并解释缺失数据机制的潜在异质性。我们使用基于似然的方法和蒙特卡罗 EM 算法进行推断。使用来自厄瓜多尔六家医院的患者的抗寄生虫治疗阿苯达唑对 NC 囊泡影响的随机对照试验的数据说明了该方法的实际应用。模拟结果表明,所提出的方法在有限样本中表现良好,而忽略数据复杂性的指定模型可能会导致严重的偏差。