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Insights Into Patient Variability During Ivacaftor-Lumacaftor Therapy in Cystic Fibrosis.


ABSTRACT: Background: The advent of cystic fibrosis transmembrane conductance regulator protein (CFTR) modulators like ivacaftor have revolutionised the treatment of cystic fibrosis (CF). However, due to the plethora of variances in disease manifestations in CF, there are inherent challenges in unified responses under CFTR modulator treatment arising from variability in patient outcomes. The pharmacokinetic (PK) data available for ivacaftor-lumacaftor cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator drug combination is limited. Methods: Secondary objectives were to identify (1) patient characteristics and (2) the interactions between ivacaftor-lumacaftor responsible for interindividual variability (IIV). Results: Peak plasma concentrations (Cmax

SUBMITTER: Hanafin PO 

PROVIDER: S-EPMC8365608 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature