Profiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.

Matthieu CornetGeneviève RobinFabiana CicirielloTiphaine BihoueeChristophe MarguetValérie RoyMuriel LebourgeoisFrédérique ChedevergneAnne Sophie BonnelMairead KellyPhilippe ReixVincenzina LucidiVéronique StovenIsabelle Sermet-Gaudelus

Published in: Pediatric pulmonology (2022)

, BMI, and ATB course are the most relevant outcomes to discriminate clinical response profiles in children treated with lumacaftor-ivacaftor. Prepubertal and pubertal children display different response profiles.

Keyphrases

cystic fibrosis
young adults
metabolic syndrome
adipose tissue
skeletal muscle
physical activity
insulin resistance
weight gain
newly diagnosed
weight loss
liver fibrosis