title: Joint modeling of multiple longitudinal patient-reported outcomes and survival.
abstract: Researchers often include patient-reported outcomes (PROs) in Phase III clinical trials to demonstrate the value of treatment from the patient's perspective. These data are collected as longitudinal repeated measures and are often censored by occurrence of a clinical event that defines a survival time. Hierarchical Bayesian models having latent individual-level trajectories provide a flexible approach to modeling such multiple outcome types simultaneously. We consider the case of many zeros in the longitudinal data motivating a mixture model, and demonstrate several approaches to modeling multiple longitudinal PROs with survival in a cancer clinical trial. These joint models may enhance Phase III analyses and better inform health care decision makers.
authors.affiliation: Division of Biostatistics, University of Minnesota, Minneapolis, Minnesota, USA.
authors.forename: Laura A
authors.initials: LA
authors.lastname: Hatfield
authors.forename: Mark E
authors.initials: ME
authors.lastname: Boye
authors.forename: Bradley P
authors.initials: BP
authors.lastname: Carlin
mesh_terms: D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use; D001499:Bayes Theorem; D017326:Clinical Trials, Phase III as Topic / Q000379:methods / Q000706:statistics & numerical data*; D018450:Disease Progression; D018572:Disease-Free Survival; D005971:Glutamates / Q000627:therapeutic use; D006147:Guanine / Q000031:analogs & derivatives / Q000627:therapeutic use; D006801:Humans; D008137:Longitudinal Studies; D008654:Mesothelioma / Q000188:drug therapy / Q000453:epidemiology; D015233:Models, Statistical; D009369:Neoplasms / Q000188:drug therapy; D000068437:Pemetrexed; D012107:Research Design; D058028:Research Report; D057566:Self Report; D016019:Survival Analysis; D013997:Time Factors; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D000964:Antimetabolites, Antineoplastic; D005971:Glutamates; D000068437:Pemetrexed; D006147:Guanine