Early Detection of Rapid Cystic Fibrosis Disease Progression Tailored to Point of Care: A Proof-of-Principle Study

Abstract

Slowing cystic fibrosis (CF) lung disease progression is crucial to survival, but point-of-care technologies aimed at early detection—and possibly prevention—of rapid lung function decline are limited. This proof-of-principle study leverages a rich national patient registry and follow-up data on a local CF cohort to build an algorithm and prototype prognostic tool aimed at early detection of rapid lung function decline. The algorithm was developed using a novel longitudinal analysis of lung function (measured as forced expiratory volume in 1 s of % predicted, FEV1). Covariates included clinical and demographic characteristics selected from the registry based on information criterion. Preliminary assessment of algorithm performance suggested excellent predictive accuracy and earlier detection of rapid decline than standard of care being applied at a local center. Graphical displays were presented and evaluated for clinical utility. Predictions from the algorithms and chosen graphical displays were translated into a prototype web application using RShiny and underwent iterative development based on clinician feedback. This paper suggests that the algorithm and its translation could offer a means for earlier detection and treatment of rapid decline, providing clinicians with a viable point-of-care technology to intervene prior to irreversible lung damage.