Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.

ABSTRACT:

Background

Ivacaftor (IVA) has been shown to improve lung function and other clinical outcomes in people with cystic fibrosis (CF). A decade of real-world IVA availability has enabled the examination of long-term outcomes with this treatment. This retrospective, longitudinal cohort study investigated the impact of IVA on mortality rate and health outcomes among people with CF in the US.

Methods

Data from the US CF Foundation Patient Registry from January 2010 to December 2019 were analysed. The IVA-treated cohort included people with a CF transmembrane conductance regulator (CFTR) gating mutation (excluding R117H); age-matched comparator cohort included people with a F508del and a minimal function CFTR mutation who had no prior CFTR modulator trea

SUBMITTER: Merlo CA 

PROVIDER: S-EPMC11503052 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature