The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation: a review

Scottish Paediatric Cystic Fibrosis Managed Clinical Network (SPCFMCN)

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Abstract

We reviewed the impact of ivacaftor on Scottish paediatric cystic fibrosis (CF)
patients ≥ 6 years of age after 12 months of treatment. Statistically significant
improvements in FEV1 and BMI 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
Original languageEnglish
Number of pages8
JournalArchives of Disease in Childhood
Early online date10 Jun 2016
DOIs
Publication statusE-pub ahead of print - 10 Jun 2016

Keywords

  • cystic fibrosis
  • ivacaftor
  • G551D
  • p.Gly551Asp

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