Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis

Source:
Cochrane Database of Systematic Reviews
Publisher:
Cochrane Database of Systematic Reviews
Publication date:
28 February 2019

Abstract

Review of 5 RCTs (n=447) found no evidence supporting use of ivacaftor in people with F508del mutation. Both phase 3 trials of people with G551D mutation demonstrated a clinically relevant impact of ivacaftor on outcomes at 24 and 48 weeks in adults and children (age >6 years).