Browsing by Subject "Informative cluster size"

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    Nonparametric tests for multistate processes with clustered data
    (Springer, 2022) Bakoyannis, Giorgos; Bandyopadhyay, Dipankar; Biostatistics and Health Data Science, Richard M. Fairbanks School of Public Health
    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 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.
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    Semiparametric marginal regression for clustered competing risks data with missing cause of failure
    (Oxford University Press, 2023) Zhou, Wenxian; Bakoyannis, Giorgos; Zhang, Ying; Yiannoutsos, Constantin T.; Biostatistics and Health Data Science, School of Medicine
    Clustered competing risks data are commonly encountered in multicenter studies. The analysis of such data is often complicated due to informative cluster size (ICS), a situation where the outcomes under study are associated with the size of the cluster. In addition, the 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 an ICS 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 ICS. 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 ICS 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.