A comparison of change point models with application to longitudinal lung function measurements in children with cystic fibrosis

• Published in: Statistics in medicine Vol. 35; no. 12; pp. 2058 - 2073
• Main Authors: Moss, Angela, Juarez-Colunga, E., Nathoo, Farouk, Wagner, Brandie, Sagel, Scott
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 30.05.2016; Wiley Subscription Services, Inc
• Subjects: Adolescent; Age Factors; broken stick models; Child; Children & youth; Comparative analysis; Cystic fibrosis; Cystic Fibrosis - physiopathology; Data Interpretation, Statistical; Disease Progression; Female; Forced Expiratory Volume; Humans; joinpoint models; Longitudinal Studies; Lung - physiopathology; Lungs; Male; marginal likelihood; mixture models; Models, Statistical; piecewise models; Probability; Respiratory Function Tests - statistics & numerical data; Simulation; Young Adult; marginal likelihood; mixture models; joinpoint models; broken stick models; piecewise models
• ISSN: 0277-6715; 1097-0258
• DOI: 10.1002/sim.6845

Cystic fibrosis (CF) is a hereditary lung disease characterized by loss of lung function over time. Lung function in CF is believed to decline at a higher rate during the adolescence period. It has been also hypothesized that there is a subgroup of individuals for whom lung disease remains relatively stable with only a slight decline over their lifetime. Using data from the University of Colorado CF Children's Registry, we investigate four change point models to model the decline of lung function in children and adolescents: (i) a two‐component mixture random change point model, (ii) a two‐component mixture‐fixed change point model, (iii) a random change point model, and (iv) a fixed change point model. The models are investigated through posterior predictive simulation at the individual and population levels, and a simulation study examining the effects of model misspecification. The data support the mixed random change point model as the preferred model, with roughly 30% of adolescents experiencing a steady decline of 0.5 %FEV1 per year and 70% experiencing an increase in decline of 4.4 %FEV1 per year beginning on average at 14.6 years of age. Copyright © 2016 John Wiley & Sons, Ltd.