Real-World Safety and Effectiveness of Elexacaftor, Tezacaftor, and Ivacaftor in People with Cystic Fibrosis and Advanced Lung Disease: A Two-Year Multicenter Cohort Study.

ABSTRACT: Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in people with CF (pwCF) carrying the F508del (F) variant, both in homozygosity and heterozygosity with a minimal function (MF) variant. Limited data exist on the effects of ETI in pwCF with advanced lung disease. Our aim was to investigate ETI safety and effectiveness in this patient group in a real-life setting over 2 years. A multicenter observational cohort study was designed to gather real-world information on the effect of ETI treatment on CF patients (aged >12 years, genotype: F/MF mutation) with advanced lung disease as defined by a FEV1 < 40% predicted. Retrospective demographic and clinical data were recorded for the two years preceding and the

SUBMITTER: Volpi S 

PROVIDER: S-EPMC12609796 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature