Ivacaftor: Five-year outcomes in the West of Scotland cystic fibrosis population |
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| Authors: | Yasmin Al-Din Carol Dryden Gordon MacGregor David Young Cristina Coelho |
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| Affiliation: | 1. Department of Pharmacy, Queen Elizabeth University Hospital, NHS Greater Glasgow and Clyde, Glasgow, UK;2. Department of Paediatrics, University Hospital Wishaw, NHS Lanarkshire, Wishaw, UK;3. Department of Respiratory Medicine, Queen Elizabeth University Hospital, NHS Greater Glasgow and Clyde, Glasgow, UK;4. Department of Mathematics and Statistics, University of Strathclyde, Glasgow, UK;5. Department of Pharmacy, Glasgow Royal Infirmary, NHS Greater Glasgow and Clyde, Glasgow, UK |
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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. |
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| Keywords: | cystic fibrosis cystic fibrosis transmembrane conductance regulator G551D mutation ivacaftor pulmonary real world |
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