Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two <i>CFTR</i> Class I variants: real-world data from the French compassionate programme.

ABSTRACT:

Background

The European Medicines Agency has recently expanded the label of elexacaftor-tezacaftor-ivacaftor (ETI) to all people with cystic fibrosis (pwCF) aged 2 years and older who have at least one non-Class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. No large-scale data exist on the use of ETI in pwCF harbouring two Class I CFTR variants.

Methods

PwCF with two Class I variants who received ETI for an individual 4-6 week trial were identified within the real-world French compassionate programme and their response to ETI, as assessed by a centralized adjudication committee, was evaluated based on evolution of clinical data, lung function and sweat chloride. The lists of Class I CFTR variants were established usin

SUBMITTER: Burgel PR 

PROVIDER: S-EPMC12418867 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature