Plasma and cellular ivacaftor concentrations in patients with cystic fibrosis.

ABSTRACT: Access to cystic fibrosis transmembrane conductance regulator (CFTR) modulators has been gradually increasing for people with cystic fibrosis, the first of which was ivacaftor, a CFTR potentiator that is part of all clinically available modulator treatments. In this study, we hypothesized that the steady-state concentrations in blood and tissue are highly variable in patients taking ivacaftor in a real-world context, which may have an impact on the treatment approach. We collected nasal epithelial cells to estimate target site concentrations and blood samples to estimate pharmacokinetic parameters at a steady state. We found that patients on ivacaftor monotherapy have variable concentrations well above the maximal effective concentration and may maintain concentrations necessary for the cl

SUBMITTER: Guimbellot JS 

PROVIDER: S-EPMC9588676 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature