The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation: a review.
We reviewed the impact of ivacaftor on Scottish paediatric patients with cystic fibrosis ≥6 years of age after 12 months of treatment. Statistically significant improvements in FEV1and body mass index and a reduction in sweat chloride, all comparable with previously published data were observed. The findings also suggested reduced use of intravenous antibiotics and oral antibiotics. No significant adverse effects were observed but a possible association with cataract formation could not be excluded. This review suggests that, in the short term at least, ivacaftor is effective and safe in paediatric patients ≥6 years of age with G551D.
Department of Paediatrics, Wishaw General Hospital, Wishaw, UK.
Carol
C
Dryden
CF Unit, Royal Hospital for Children, Glasgow, UK.
Jane
J
Wilkinson
Department of Mathematics and Statistics, University of Strathclyde, Glasgow, UK.
David
D
Young
Department of Medical Paediatrics, Royal Aberdeen Children's Hospital, Aberdeen, UK.
Richard John
RJ
Brooker
D000627:Aminophenols / Q000627:therapeutic use*; D002648:Child; D065101:Chloride Channel Agonists / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D006801:Humans; D009154:Mutation; D015363:Quinolones / Q000627:therapeutic use*; D012606:Scotland; D016896:Treatment Outcome
D000627:Aminophenols; D065101:Chloride Channel Agonists; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor
Cystic Fibrosis; G551D; Ivacaftor; p.Gly551Asp