In this work, we propose nonparametric two-sample tests for population-averaged transition and state occupation probabilities for continuous-time and finite state space processes with clustered, right-censored, and/or left-truncated data. We consider settings where the two groups under comparison are independent or dependent, with or without complete cluster structure. The proposed tests do not impose assumptions regarding the structure of the within-cluster dependence and are applicable to settings with informative cluster size and/or non-Markov processes. The asymptotic properties of the tests are rigorously established using empirical process theory. Simulation studies show that the proposed tests work well even with a small number of clusters, and that they can be substantially more powerful compared to the only, to the best of our knowledge, previously proposed nonparametric test for this problem. The tests are illustrated using data from a multicenter randomized controlled trial on metastatic squamous-cell carcinoma of the head and neck.

在这项工作中，我们提出了对具有聚类、右删失和/或左截断数据的连续时间和有限状态空间过程的总体平均转移和状态占用概率的非参数双样本检验。我们考虑比较的两组是独立的还是依赖的，有或没有完整的聚类结构的设置。所提出的测试不会强加关于簇内依赖性结构的假设，并且适用于具有信息性簇大小和/或非马尔可夫过程的设置。检验的渐近性质是使用经验过程理论严格建立的。模拟研究表明，即使对于少量集群，所提出的测试也能很好地工作，并且据我们所知，与之前针对该问题提出的唯一非参数测试相比，它们的功能要强大得多。这些测试使用来自头颈部转移性鳞状细胞癌的多中心随机对照试验的数据进行说明。