Ivacaftor: five‐year outcomes in the West of Scotland cystic fibrosis population

Yasmin Al‐Din, Carol Dryden, Gordon MacGregor, David Young, Cristina Coelho

Research output: Contribution to journalArticlepeer-review

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Abstract

Introduction: Ivacaftor has shown to be effective in patients with cystic fibrosis (CF) with a G551D mutation. Objectives: This work aims to evaluate ivacaftor's effectiveness and safety in the real world, over 5 years, in the West of Scotland CF population. Methods: We evaluated ivacaftor's effect on pulmonary function, body mass index (BMI), hospital bed occupancy, and adverse effects in patients ≥6 years with at least one G551D mutation. Results: Statistically significant increases from baseline were observed in mean per cent predicted forced expiratory volume in 1 s (FEV1) at year 1 (which was maintained at years 2 and 5) and BMI over 5 years in our adolescent/adult cohort. Improvements were observed in per cent predicted FEV1 within the paediatric cohort with a suggestion of a plateau effect. The increase in paediatric BMI z‐score was nonstatistically significant. There was a reduction in the number of pulmonary exacerbations requiring intravenous antibiotics and hospital bed occupancy. Ivacaftor was well tolerated. Conclusion: Ivacaftor was effective in our population.
Original languageEnglish
Pages (from-to)473-477
Number of pages5
JournalThe Clinical Respiratory Journal
Volume17
Issue number5
Early online date20 Mar 2023
DOIs
Publication statusPublished - 31 May 2023

Keywords

  • cystic fibrosis
  • cystic fibrosis transmembrane conductance regulator
  • G551D mutation
  • ivacaftor
  • pulmonary
  • real world

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