Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation.

ABSTRACT:

Background

The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study describes the long-term clinical effectiveness of ivacaftor in this population.

Methods

We conducted a multicenter, prospective, longitudinal, observational study of people with CF ages ≥6 years with at least one copy of the G551D CFTR mutation. Measurements of lung function, growth, quality of life, and sweat chloride were performed after ivacaftor initiation (baseline, 1 month, 3 months, 6 months, and annually thereafter until 5.5 years).

Results

Ninety-six participants were enrolled, with 81% completing all study measures through 5.5 years. This cohort experienced significant improvements in

SUBMITTER: Guimbellot JS 

PROVIDER: S-EPMC8183611 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature