Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data

Body composition changes and clinical outcomes in pediatric cystic fibrosis during 24 months of lumacaftor ivacaftor therapy based on real-world data

Abstract Clinical trials demonstrate the short-term efficacy of dual CFTR modulators, but long-term real-world data is limited. We aimed to investigate the effects of 24-month lumacaftor/ivacaftor (LUM/IVA) therapy in pediatric CF patients (pwCF). This observational study included pwCF homozygous fo...

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Main Authors: Marcell Imrei, Adrienn F. Kéri, Éva Gács, Ildikó Gönczi, Melinda Meláth, Éva Kosaras, Botond Demeter, Csaba Péterfia, Klára Vass, Gyöngyi Székely, Klementina Ocskay, Andrea Párniczky
Format: Article
Language:English
Published: Nature Portfolio 2025-01-01
Series:Scientific Reports
Subjects:
Normal-weight obesity
Fecal elastase
Pancreatic function
Body composition
CFTR modulator therapy
LUM/IVA
Online Access:https://doi.org/10.1038/s41598-025-86010-1
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