Bayesian multivariate growth curve latent class models for mixed outcomes

Abstract

In many clinical studies, the disease of interest is multifaceted, and multiple outcomes are needed to adequately capture information about the characteristics of the disease or its severity. In the analysis of such diseases, it is often difficult to determine what constitutes improvement because of the multivariate nature of the outcome. Furthermore, when the disease of interest has an unknown etiology and/or is primarily a symptom-defined syndrome, there is potential for the disease population to have distinct subgroups. Identification of population subgroups is of interest as it may assist clinicians in providing appropriate treatment or in developing accurate prognoses. We propose multivariate growth curve latent class models that group subjects on the basis of multiple symptoms measured repeatedly over time. These groups or latent classes are defined by distinctive longitudinal profiles of a latent variable, which is used to summarize the multivariate outcomes at each point. The mean growth curve for the latent variable in each class defines the features of the class. We develop this model for any combination of continuous, binary, ordinal, or count outcomes within a Bayesian hierarchical framework. We use simulation studies to validate the estimation procedures. We apply our model to data from a randomized clinical trial evaluating the efficacy of Bacillus Calmette-Guerin in treating symptoms of interstitial cystitis where we are able to identify a class of subjects for whom treatment is effective.

Keywords: latent class; latent variable; longitudinal; randomized trials.