Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis.

ABSTRACT:

Rationale

Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator recently approved for patients with CF age 6 and older with the G551D mutation.

Objectives

To evaluate ivacaftor in a postapproval setting and determine mechanism of action and response of clinically relevant markers.

Methods

We conducted a longitudinal cohort study in 2012-2013 in G551D CF patients age 6 and older with no prior exposure to ivacaftor. Study assessments were performed at baseline, 1, 3, and 6 months after ivacaftor initiation. Substudies evaluated mucociliary clearance, β-adrenergic sweat secretion rate, gastrointestinal pH, and sputum inflammation and microbiology Measurements and Main Results: A total of 151 of 153 subjects were prescribed ivacaftor and 88

SUBMITTER: Rowe SM 

PROVIDER: S-EPMC4226057 | biostudies-literature | 2014 Jul

REPOSITORIES: biostudies-literature