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

Matthieu Cornet,Geneviève Robin,Fabiana Ciciriello, Tiphaine Bihouee,Christophe Marguet, Valérie Roy,Muriel Lebourgeois,Frédérique Chedevergne, Anne Sophie Bonnel,Mairead Kelly,Philippe Reix,Vincenzina Lucidi,Véronique Stoven,Isabelle Sermet-Gaudelus

Pediatric Pulmonology（2022）

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摘要

Change in ppFEV , 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.

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关键词

children,lumacaftor-ivacaftor,principal component analysis

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