Is a Cure at Hand for Cystic Fibrosis in Some Children?

Oral ivacaftor is associated with early and sustained improvement in pulmonary function in CF patients with the G551D-CFTR mutation.

About 4% to 5% of patients with cystic fibrosis (CF) have a defect in the CF transmembrane conductance regulator (CFTR) protein. In an industry-sponsored, double-blind, randomized, placebo-controlled trial, investigators evaluated the efficacy of oral ivacaftor — a CFTR potentiator — in 161 CF patients older than 12 years with at least one G551D-CFTR mutation.

After 24 weeks of daily treatment (150 mg every 12 hours), the increase in forced expiratory volume at 1 second (FEV₁) from baseline was significantly greater in the ivacaftor group (mean increase, 0.367 liters) than in the placebo group (17.2% vs. 0.1%). A significant treatment effect was noted by day 15 and continued throughout 48 weeks of treatment. At 48 weeks, 67% of ivacaftor-tre…

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)