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

• DOI: 10.1007/s41030-021-00158-5
• Publication Date: 2021-06-08T13:05:14Z
• Authors: Dominique Hubert; Christophe Marguet; Jacques Benichou; Cynthia DeSouza; Catherine Payen-C...; Nils Kinnman; Keval Chandarana; Anne Munck; Isabelle Fajac; ; Sophie Ramel; Philippe Vigneron; Veronique Storni; Natacha Remus; Laurence Bassinet...; Muriel Laurans; Cinthia Rames Fra...; Marie-Laure Dalph...; Isabelle Pin; Boubou Camara Ann...; Jeanne Languepin; Isabelle Danner-B...; Marc Albertini Ju...; Nathalie Wizla; Laure Cosson; Eric Deneuville M...; Philippe Reix; Nadine Dufeu; Valérie David; Thierry Langin St...; Raphael Chiron; Dominique Grenet; Frédérique Chedev...; Chantal Belleguic; Laurent Mely
• Keywords: Ivacaftor

Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has demonstrated clinical benefits in phase 3 trials. We report results from real-world study (BRIO) to assess the effectiveness of ivacaftor people with (pwCF) France. BRIO was an observational conducted at 35 centers Both pwCF initiating treatment and those already taking were included prospectively followed for 24 months. The primary objective evaluate effect on percent predicted forced expiratory volume 1 s (ppFEV1); secondary objectives evaluating effectiveness, healthcare resource utilization (HCRU), safety. A total 129 enrolled; 58.9% aged < 18 years; 64.3% had G551D-CFTR allele. Mean age initiation 19.1 years (range, 2–64 years); ppFEV1 increased by least squares mean 8.49 percentage points first 6 months sustained through 36 use. Growth metrics during 12 post-ivacaftor remained stable. rate pulmonary exacerbations (PEx) decreased compared pre-ivacaftor; estimated ratios (95% CI) 0.57 (0.43–0.75) PEx events 0.25 (0.13–0.48) requiring hospitalization. No new safety concerns identified; no deaths occurred. this usage France consistent prior trial outcomes, confirming ivacaftor, as well associated reduction HCRU.