Analyzing clustered continuous response variables with ordinal regression models. 2023

Yuqi Tian, and Bryan E Shepherd, and Chun Li, and Donglin Zeng, and Jonathan S Schildcrout
Department of Biostatistics, Vanderbilt University, Nashville, Tennessee, USA.

Continuous response data are regularly transformed to meet regression modeling assumptions. However, approaches taken to identify the appropriate transformation can be ad hoc and can increase model uncertainty. Further, the resulting transformations often vary across studies leading to difficulties with synthesizing and interpreting results. When a continuous response variable is measured repeatedly within individuals or when continuous responses arise from clusters, analyses have the additional challenge caused by within-individual or within-cluster correlations. We extend a widely used ordinal regression model, the cumulative probability model (CPM), to fit clustered, continuous response data using generalized estimating equations for ordinal responses. With the proposed approach, estimates of marginal model parameters, cumulative distribution functions , expectations, and quantiles conditional on covariates can be obtained without pretransformation of the response data. While computational challenges arise with large numbers of distinct values of the continuous response variable, we propose feasible and computationally efficient approaches to fit CPMs under commonly used working correlation structures. We study finite sample operating characteristics of the estimators via simulation and illustrate their implementation with two data examples. One studies predictors of CD4:CD8 ratios in a cohort living with HIV, and the other investigates the association of a single nucleotide polymorphism and lung function decline in a cohort with early chronic obstructive pulmonary disease.

UI MeSH Term Description Entries
D011336 Probability The study of chance processes or the relative frequency characterizing a chance process. Probabilities
D003198 Computer Simulation Computer-based representation of physical systems and phenomena such as chemical processes. Computational Modeling,Computational Modelling,Computer Models,In silico Modeling,In silico Models,In silico Simulation,Models, Computer,Computerized Models,Computer Model,Computer Simulations,Computerized Model,In silico Model,Model, Computer,Model, Computerized,Model, In silico,Modeling, Computational,Modeling, In silico,Modelling, Computational,Simulation, Computer,Simulation, In silico,Simulations, Computer
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D015233 Models, Statistical Statistical formulations or analyses which, when applied to data and found to fit the data, are then used to verify the assumptions and parameters used in the analysis. Examples of statistical models are the linear model, binomial model, polynomial model, two-parameter model, etc. Probabilistic Models,Statistical Models,Two-Parameter Models,Model, Statistical,Models, Binomial,Models, Polynomial,Statistical Model,Binomial Model,Binomial Models,Model, Binomial,Model, Polynomial,Model, Probabilistic,Model, Two-Parameter,Models, Probabilistic,Models, Two-Parameter,Polynomial Model,Polynomial Models,Probabilistic Model,Two Parameter Models,Two-Parameter Model
D035501 Uncertainty The condition in which reasonable knowledge regarding risks, benefits, or the future is not available.

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