51. Ivacaftor: Five-year outcomes in the West of Scotland cystic fibrosis population.
- Author
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Al-Din Y, Dryden C, MacGregor G, Young D, and Coelho C
- Subjects
- Adult, Adolescent, Humans, Child, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Cystic Fibrosis Transmembrane Conductance Regulator therapeutic use, Aminophenols adverse effects, Forced Expiratory Volume, Mutation, Cystic Fibrosis drug therapy, Cystic Fibrosis genetics, Quinolones therapeutic use
- 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 (FEV
1 ) 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., (© 2023 The Authors. The Clinical Respiratory Journal published by John Wiley & Sons Ltd.)- Published
- 2023
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