Inference on the marginal distribution of clustered data with informative cluster size

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Publication:2442679



Abstract: In spite of recent contributions to the literature, informative cluster size settings are not well known and understood. In this paper, we give a formal definition of the problem and describe it from different viewpoints. Data generating mechanisms, parametric and nonparametric models are considered in light of examples. Our emphasis is on nonparametric and robust approaches to the inference on the marginal distribution. Descriptive statistics and parameters of interest are defined as functionals and they are accompanied with a generally applicable testing procedure. The theory is illustrated with an example on patients with incomplete spinal cord injuries.


The authors consider models for clustered data where the cluster size could carry information about the marginal distribution of the outcome. In the first sections the concept of informative cluster size is developed--formal definitions are given, examples illustrate the defined models. Based on a nonparametric approach quantities of interest are defined as functionals, and the properties of their sample counterparts are discussed. The following quantities are considered: Mean and variance functionals, sign and rank based functionals accompanied with testing procedures, and regression functionals. The theory is illustrated with an example on patients with incomplete spinal cord injuries.




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