Dynamics of Disease Progression and Gastrostomy Tube Placement in Children and Adolescents with Cystic Fibrosis: Application of Joint Models for Longitudinal and Time-to-Event Data

摘要

BackgroundPrevious small-sample studies have examined the effect of gastrostomy (g-) tube placement on weight, height, and lung function in adolescent patients with cystic fibrosis (CF), but there are no RCTs to date reporting efficacy. The goal of this study was to implement a dynamic prediction model to 1) understand the role of rapid lung function decline in g-tube placement in real-world clinical settings; 2) provide a prognostic tool with the potential to aid clinicians in optimizing the timing of g-tube placement, in relation to rate of lung function decline and current nutrition status.