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

Authors
  • Patel, Sanjay1
  • Sinha, Ian P
  • Dwan, Kerry
  • Echevarria, Carlos
  • Schechter, Michael
  • Southern, Kevin W
  • 1 Department of Women's and Children's Health, University of Liverpool, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, Merseyside, UK, L12 2AP.
Type
Published Article
Journal
The Cochrane database of systematic reviews
Publication Date
Mar 26, 2015
Issue
3
Identifiers
DOI: 10.1002/14651858.CD009841.pub2
PMID: 25811419
Source
Medline
License
Unknown

Abstract

Both G551D phase 3 trials (n = 219) demonstrated a clinically relevant impact of the potentiator ivacaftor on outcomes at 24 and 48 weeks, providing evidence for the use of this treatment in adults and children (over six years of age) with cystic fibrosis and the G551D mutation (class III). There is no evidence to support the use of ivacaftor in people with the ΔF508 mutation (class II) (n = 140). Trials on ivacaftor in people with different mutations are ongoing.

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