Chest
-
Editorial Comment
Radiation and chest CT scans: are there problems? What should we do?
-
Editorial Comment
Emerging bronchoscopic therapies for stage IV advanced emphysema.
-
Randomized Controlled Trial Multicenter Study
Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation.
Ivacaftor (VX-770) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that was approved in the United States for the treatment of cystic fibrosis (CF) in patients ≥ 6 years of age who have a G551D mutation; however, the most prevalent disease-causing CFTR mutation, F508del, causes a different functional defect. The objectives of this study were to evaluate the safety of ivacaftor in a larger population and for a longer time period than tested previously and to assess the efficacy of ivacaftor in subjects with CF who are homozygous for F508del-CFTR. ⋯ These results expand the safety information for ivacaftor and support its continued evaluation. Lack of a clinical effect suggests that a CFTR potentiator alone is not an effective therapeutic approach for patients who have CF and are homozygous for F508del-CFTR.