Bayesian joint modeling of longitudinal patient-reported outcomes and survival: an application to chronic obstructive pulmonary disease

Abstract

Questionnaire-based patient-reported outcomes (PROs) are discrete, bounded and overdispersed, yet joint models relating them to survival may ignore these features or estimate both processes sequentially. We propose a Bayesian joint model combining a beta-binomial mixed-effects submodel with a Weibull proportional hazards submodel, linked through the subject-specific response probability. Simulations show that simultaneous estimation reduces bias in the longitudinal slope and yields practically unbiased association estimates, unlike two-stage estimation. In a cohort of 543 patients with chronic obstructive pulmonary disease, the model identified associations for all eight SF-36 dimensions and for two of three SGRQ dimensions, including several associations not detected by the two-stage approach, and provided dynamic survival predictions.