Bayesian estimation of two‐part joint models for a longitudinal semicontinuous biomarker and a terminal event with INLA: Interests for cancer clinical trial evaluation

DOI10.1002/BIMJ.202100322MaRDI QIDQ6143322zbMATH OpenOpenAlexFDO

Authors Denis Rustand, Janet van Niekerk, Håvard Rue, Virginie Rondeau, Laurent Briollais

Publication date 4 January 2024

Published in Biometrical Journal (Search for Journal in Brave)

Copyright license Creative Commons Attribution 4.0 International

Full work available at URL https://arxiv.org/abs/2010.13704

zbMATH Keywords

Bayesian estimationcomputational efficiencyINLAsolid tumors cancertwo-part joint model

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10)

Abstract: Two-part joint models for a longitudinal semicontinuous biomarker and a terminal event have been recently introduced based on frequentist estimation. The biomarker distribution is decomposed into a probability of positive value and the expected value among positive values. Shared random effects can represent the association structure between the biomarker and the terminal event. The computational burden increases compared to standard joint models with a single regression model for the biomarker. In this context, the frequentist estimation implemented in the R package frailtypack can be challenging for complex models (i.e., large number of parameters and dimension of the random effects). As an alternative, we propose a Bayesian estimation of two-part joint models based on the Integrated Nested Laplace Approximation (INLA) algorithm to alleviate the computational burden and fit more complex models. Our simulation studies confirm that INLA provides accurate approximation of posterior estimates and to reduced computation time and variability of estimates compared to frailtypack in the situations considered. We contrast the Bayesian and frequentist approaches in the analysis of two randomized cancer clinical trials (GERCOR and PRIME studies), where INLA has a reduced variability for the association between the biomarker and the risk of event. Moreover, the Bayesian approach was able to characterize subgroups of patients associated with different responses to treatment in the PRIME study. Our study suggests that the Bayesian approach using INLA algorithm enables to fit complex joint models that might be of interest in a wide range of clinical applications.

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Cited in

(6)

• Bayesian semi-parametric joint modeling of biomarker data with a latent changepoint: assessing the temporal performance of enzyme-linked immunosorbent assay (ELISA) testing for paratuberculosis
• Assessing importance of biomarkers: A Bayesian joint modelling approach of longitudinal and survival data with semi-competing risks
• Bayesian survival analysis with INLA
• Trivariate joint modeling for family data with longitudinal counts, recurrent events and a terminal event with application to lynch syndrome
• A graphical framework for interpretable correlation matrix models for multivariate regression
• A unified joint modelling of zero-inflated longitudinal measurements and time-to-event outcomes with applications to HIV and colorectal cancer data

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