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 (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 FEV ₁ 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.

Abstract

Ivacaftor's effectiveness and safety was evaluated in the real world, over 5 years, in patients with at least one G551D mutation within the West of Scotland. Findings include the following: Improvement in mean per cent predicted forced expiratory volume in 1 s (FEV ₁), sustained increase in body mass index, decreased intravenous antibiotic use from baseline, reduced hospital bed occupancy from baseline, and good safety profile.