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The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation: a review
  1. Carol Dryden1,
  2. Jane Wilkinson2,
  3. David Young3,
  4. Richard John Brooker4
  5. on behalf of the Scottish Paediatric Cystic Fibrosis Managed Clinical Network (SPCFMCN)
  1. 1Department of Paediatrics, Wishaw General Hospital, Wishaw, UK
  2. 2CF Unit, Royal Hospital for Children, Glasgow, UK
  3. 3Department of Mathematics and Statistics, University of Strathclyde, Glasgow, UK
  4. 4Department of Medical Paediatrics, Royal Aberdeen Children's Hospital, Aberdeen, UK
  1. Correspondence to Dr Carol Dryden, Department of Paediatrics, Wishaw General Hospital, Netherton Street, Wishaw ML2 0DP, UK; carol.dryden{at}lanarkshire.scot.nhs.uk

Abstract

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 FEV1 and 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.

  • Cystic Fibrosis
  • G551D
  • p.Gly551Asp
  • Ivacaftor

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