Update on Cystic Fibrosis in Pediatric Patients.

Abstract

Cystic fibrosis is an inherited, multisystem disease that affects the gastrointestinal system in numerous ways. This article reviews the nutritional, gastrointestinal, and hepatobiliary manifestations of cystic fibrosis with an emphasis on the effects of CFTR modulator therapy. The life expectancy of individuals with cystic fibrosis has increased substantially in recent years. CFTR modulator therapy improves pulmonary function and results in weight gain. An individualized approach to nutrition is encouraged. Pancreatic exocrine function may improve with intervention early in life. The use of non-invasive methods to screen for hepatobiliary involvement is recommended. Highly effective CFTR modulators lead to increased survival and improved quality of life for many individuals. Their effects on gastrointestinal symptoms and hepatobiliary disease are not fully understood. Patient-reported outcome measures and biomarkers are important clinical endpoints for studying the effects of modulators.