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

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.

• 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)
• Copyright: © 2023 The Authors. The Clinical Respiratory Journal published by John Wiley & Sons Ltd.

Chemical References

• ivacaftor
• Cystic Fibrosis Transmembrane Conductance Regulator
• Aminophenols
• Quinolones

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