Abstract | INTRODUCTION: 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:
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Authors | Yasmin Al-Din, Carol Dryden, Gordon MacGregor, David Young, Cristina Coelho |
Journal | The clinical respiratory journal
(Clin Respir J)
Vol. 17
Issue 5
Pg. 473-477
(May 2023)
ISSN: 1752-699X [Electronic] England |
PMID | 36938952
(Publication Type: Journal Article)
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Copyright | © 2023 The Authors. The Clinical Respiratory Journal published by John Wiley & Sons Ltd. |
Chemical References |
- ivacaftor
- Cystic Fibrosis Transmembrane Conductance Regulator
- Aminophenols
- Quinolones
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Topics |
- Adult
- Adolescent
- Humans
- Child
- Cystic Fibrosis
(drug therapy, genetics)
- Cystic Fibrosis Transmembrane Conductance Regulator
(genetics, therapeutic use)
- Aminophenols
(adverse effects)
- Quinolones
(therapeutic use)
- Forced Expiratory Volume
- Mutation
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