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. 2021 Dec;7(2):455-468.
doi: 10.1007/s41030-021-00158-5. Epub 2021 Jun 8.

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

Dominique Hubert  1 , Christophe Marguet  2 , Jacques Benichou  3 , Cynthia DeSouza  4 , Catherine Payen-Champenois  5 , Nils Kinnman  6 , Keval Chandarana  7 , Anne Munck  8 , Isabelle Fajac  9   10   11 ; BRIO Study Group
Collaborators, Affiliations

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

Dominique Hubert et al. Pulm Ther. 2021 Dec.

Erratum in

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 (ppFEV1); 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 < 18 years; 64.3% had a G551D-CFTR allele. Mean age at ivacaftor initiation was 19.1 years (range, 2-64 years); ppFEV1 increased by a least squares mean of 8.49 percentage points in the first 6 months and was sustained through 36 months of ivacaftor use. Growth metrics increased during the first 12 months post-ivacaftor and remained stable. The rate of pulmonary exacerbations (PEx) decreased during the 12 months post-ivacaftor compared with the 12 months pre-ivacaftor; estimated rate ratios (95% CI) were 0.57 (0.43-0.75) for PEx events and 0.25 (0.13-0.48) for PEx requiring hospitalization. No new safety concerns were identified; no deaths occurred.

Conclusions: The results from this real-world study of ivacaftor usage in France were consistent with prior clinical trial outcomes, confirming the clinical effectiveness of ivacaftor, as well as an associated reduction in HCRU.

Keywords: Adults; Children; Cystic fibrosis; Exacerbation; Ivacaftor; Lung function; Real-world experience.

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Figures

Fig. 1
Fig. 1
Absolute change from baseline in a ppFEV1, b BMI-for-age z score, and c BMI. BMI body mass index, LS least squares, ppFEV1 percent predicted forced expiratory volume in 1 s
See this image and copyright information in PMC

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