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arXiv 2104.09090stat.ME
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失效原因缺失条件下竞争风险数据的半参数边际回归

Semiparametric Marginal Regression for Clustered Competing Risks Data with Missing Cause of Failure

Wenxian Zhou, Giorgos Bakoyannis, Ying Zhang, Constantin T. Yiannoutsos

摘要:

在多中心研究中,经常会遇到不可靠的竞争风险数据。这类数据的分析往往是复杂的,由于信息集群的大小,在这种情况下,所研究的结果与集群的大小。此外,在实际环境中,故障原因经常无法完全观察到。据我们所知,没有方法对聚类竞争风险数据进行群体平均分析,其中聚类大小为信息量,失效原因缺失。为了解决这个问题,我们考虑了半参数边际比例原因特定风险模型,并提出了一个最大的部分伪概率估计下的随机缺失的假设。为了使后一个假设在实践中更加合理,我们允许可能与失踪概率相关的辅助变量。所提出的方法不施加假设集群内的依赖性,并允许信息集群大小。所提出的估计的渐近性质的回归系数和无穷维参数,如边际累积发生率函数,严格建立。仿真结果表明,该方法性能良好,忽略类内依赖性和信息簇大小的方法会导致无效的推理。所提出的方法适用于竞争风险的数据,从一个大的多中心艾滋病毒研究在撒哈拉以南非洲的失败原因的一个重要部分是失踪。

英文摘要:

Clustered competing risks data are commonly encountered in multicenter studies. The analysis of such data is often complicated due to informative cluster size, a situation where the outcomes under study are associated with the size of the cluster. In addition, cause of failure is frequently incompletely observed in real-world settings. To the best of our knowledge, there is no methodology for population-averaged analysis with clustered competing risks data with informative cluster size and missing causes of failure. To address this problem, we consider the semiparametric marginal proportional cause-specific hazards model and propose a maximum partial pseudolikelihood estimator under a missing at random assumption. To make the latter assumption more plausible in practice, we allow for auxiliary variables that may be related to the probability of missingness. The proposed method does not impose assumptions regarding the within-cluster dependence and allows for informative cluster size. The asymptotic properties of the proposed estimators for both regression coefficients and infinite-dimensional parameters, such as the marginal cumulative incidence functions, are rigorously established. Simulation studies show that the proposed method performs well and that methods that ignore the within-cluster dependence and the informative cluster size lead to invalid inferences. The proposed method is applied to competing risks data from a large multicenter HIV study in sub-Saharan Africa where a significant portion of causes of failure is missing.

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