Why carry out this study?

Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator developed to treat the underlying cause of CF. Treatment with ivacaftor improves clinical outcomes in people with CF (pwCF) with certain gating and non-gating CFTR mutations.

Since CF is a lifelong disease, it is important to understand the long-term impact of ivacaftor treatment in a real-world setting.

This observational study aimed to assess the real-world effectiveness and healthcare resource utilization after ivacaftor initiation in pwCF in France.

What was learned from the study?

At 35 CF centers in France, 129 pwCF were enrolled; 58.9% were < 18 years of age. Improvements in lung function, rate of pulmonary exacerbations, nutritional status, and growth metrics were observed with ivacaftor use and were sustained over 36 months of follow-up. Additionally, ivacaftor usage was associated with a reduction in healthcare resource utilization.

The results from this real-world study of ivacaftor use in France were consistent with prior clinical trial outcomes and confirm the clinical effectiveness of ivacaftor.