The Clinical Respiratory Journal (May 2023)

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

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

DOI
https://doi.org/10.1111/crj.13602
Journal volume & issue
Vol. 17, no. 5
pp. 473 – 477

Abstract

Read online

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.

Keywords