Why carry out this study?

In cystic fibrosis (CF), a life-shortening, rare genetic disease, researchers have the unique opportunity to evaluate data from the majority of patients in their respective regions collected by national CF registries, with the US Cystic Fibrosis Foundation Patient Registry (US CFFPR) and the UK Cystic Fibrosis Registry (UK CFR) being the largest.

To evaluate disease progression and clinical outcomes in patients treated with ivacaftor (the first approved CF transmembrane conductance regulator [CFTR] modulator targeting the underlying cause of CF), we conducted a long-term observational study comparing outcomes in patients in the US and UK registries who were treated with ivacaftor versus matched untreated comparator patients.

This study asked, “Among patients with CF treated with ivacaftor in real-world clinical practice, what are the patterns in key clinical outcomes?”

What was learned from the study?

Analyses of up to 5 years of experience with ivacaftor in real-world clinical practice across countries and years identified no new safety concerns and demonstrated consistently favorable outcomes in ivacaftor-treated patients.

These data support the conclusion that highly effective CFTR modulation with ivacaftor leads to disease modification, consistent with the multisystem benefits observed in clinical trials.