Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization

Introduction Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has demonstrated clinical benefits in phase 3 trials. We report results from a real-world study (BRIO) to assess the effectiveness of ivacaftor in people with cystic fibrosis (pwCF) in France. Met...

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Bibliographic Details
Published in:Pulmonary therapy 2021-12, Vol.7 (2), p.455-468
Main Authors: Hubert, Dominique, Marguet, Christophe, Benichou, Jacques, DeSouza, Cynthia, Payen-Champenois, Catherine, Kinnman, Nils, Chandarana, Keval, Munck, Anne, Fajac, Isabelle
Format: Article
Language:English
Subjects:
Family Medicine
General Practice
Internal Medicine
Medicine
Medicine & Public Health
Original Research
Pharmacoeconomics and Health Outcomes
Pharmacotherapy
Pneumology/Respiratory System
Quality of Life Research
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Summary:Introduction Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has demonstrated clinical benefits in phase 3 trials. We report results from a real-world study (BRIO) to assess the effectiveness of ivacaftor in people with cystic fibrosis (pwCF) in France. Methods BRIO was an observational study conducted at 35 centers in France. Both pwCF initiating ivacaftor treatment and those already taking ivacaftor were included and prospectively followed for 24 months. The primary objective was to evaluate the effect of ivacaftor on percent predicted forced expiratory volume in 1 s (ppFEV 1 ); secondary objectives were evaluating the effect of ivacaftor on clinical effectiveness, healthcare resource utilization (HCRU), and safety. Results A total of 129 pwCF were enrolled; 58.9% were aged 
ISSN:2364-1754
2364-1746
DOI:10.1007/s41030-021-00158-5