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

Abstract

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 The members of the ''on behalf of the BRIO Study Group'' was processed under acknowledgements section.

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

“…These results were confirmed in the BRIO study, a prospective observational study conducted in 35 centers in the French CF network, which included 129 pwCF with gating mutations (40.1% children <12 years) and was designed to assess the effectiveness of IVA in pwCF in terms of clinical outcomes and healthcare resource utilization within 2 years after IVA initiation [ 29 ]. The main results included substantial improvements in ppFEV 1 (least-squares mean of 8.49 percentage points), growth metrics, and nutritional status within the first 6–12 months and up to 36 months after IVA initiation [ 29 ]. The rate of pulmonary exacerbations (PEx) decreased during the 12 months post-ivacaftor in comparison to the 12 months prior, with an estimated risk ratio (RR) of 0.57 (95% Confidence Interval (95%CI) 0.43–0.75) for PEx events and 0.25 (0.13–0.48) for PEx requiring hospitalization [ 29 ].…”

“…The main results included substantial improvements in ppFEV 1 (least-squares mean of 8.49 percentage points), growth metrics, and nutritional status within the first 6–12 months and up to 36 months after IVA initiation [ 29 ]. The rate of pulmonary exacerbations (PEx) decreased during the 12 months post-ivacaftor in comparison to the 12 months prior, with an estimated risk ratio (RR) of 0.57 (95% Confidence Interval (95%CI) 0.43–0.75) for PEx events and 0.25 (0.13–0.48) for PEx requiring hospitalization [ 29 ]. Hubert et al also observed a decrease in the proportion of pwCF who had a positive sputum culture over 12 months and lower methicillin-sensitive S. aureus and P. aeruginosa colonization rates [ 29 ].…”

“…The rate of pulmonary exacerbations (PEx) decreased during the 12 months post-ivacaftor in comparison to the 12 months prior, with an estimated risk ratio (RR) of 0.57 (95% Confidence Interval (95%CI) 0.43–0.75) for PEx events and 0.25 (0.13–0.48) for PEx requiring hospitalization [ 29 ]. Hubert et al also observed a decrease in the proportion of pwCF who had a positive sputum culture over 12 months and lower methicillin-sensitive S. aureus and P. aeruginosa colonization rates [ 29 ]. Little to no difference was observed between the pre-and post-ivacaftor periods in terms of classic CF comorbidities (diabetes, exocrine pancreatic insufficiency) or in medication requirements.…”

“…Little to no difference was observed between the pre-and post-ivacaftor periods in terms of classic CF comorbidities (diabetes, exocrine pancreatic insufficiency) or in medication requirements. IVA was generally well-tolerated, and no deaths occurred [ 29 ].…”

CFTR Modulators: Current Status and Evolving Knowledge

“…Observational and registry-based studies show that survival is improved, while exacerbation frequency, need for lung transplantation (LTx), and prevalence of Pseudomonas aeruginosa and Staphylococcus aureus are reduced. 27 28 29 30 31 Longitudinal data also indicate that starting IVA early was likely to obtain a greater long-term impact. 32…”