PND80 - AN OBSERVATIONAL STUDY OF IVACAFTOR IN PATIENTS WITH CYSTIC FIBROSIS IN FRANCE: FIRST INTERIM ANALYSIS OF HEALTH CARE RESOURCE UTILIZATION FROM THE BRIO STUDY

Abstract

The efficacy of ivacaftor in cystic fibrosis (CF) patients has been demonstrated in Phase 3 randomized-controlled trials. BRIO is a real-world study assessing effectiveness of ivacaftor in patients with CF and ivacaftor-responsive mutations in France. This interim analysis describes the impact of ivacaftor on healthcare resource utilization (HCRU) in patients ≥6 years. BRIO is an ongoing observational, non-interventional multi-centre study. Of 55 CF centers contacted, 33 participated. Ivacaftor-naive and -treated patients were eligible. Patient data are collected prospectively for two years after enrolment, and retrospectively for 12 months preceding initiation of ivacaftor, and up to enrolment in the study. This interim analysis describes HCRU, including all-cause hospitalisation and antibiotics use (oral/IV/inhaled) due to pulmonary exacerbations (PEx), for 12 months pre-ivacaftor initiation and 12 months post-ivacaftor initiation. HCRU analysis used a negative binomial model adjusted for time on study in the 12-month period, with baseline defined as ivacaftor initiation date. Of 107 enrolled patients, 100 received ≥12 months treatment with ivacaftor. At baseline, mean (SD) age was 21.1 (14.3) years, 56% were male. All-cause hospitalisations showed a substantial reduction per patient year (PPY) in 12 months post-ivacaftor initiation vs 12 months pre-ivacaftor initiation (rate ratio [RR] 0.40; 95% CI: 0.26–0.61). Corresponding hospitalisation days PPY were reduced from 5.3 days pre-ivacaftor to 2.5 days post-ivacaftor (RR 0.46; 95% CI: 0.22–0.95). Number of unique antibiotics PPY for treatment of PEx was also reduced 12 months post-ivacaftor vs 12 months pre-ivacaftor (RR 0.47; 95% CI: 0.32–0.68). Corresponding days of antibiotic use PPY declined from 20.9 days pre-ivacaftor to 11.4 days post-ivacaftor (RR: 0.54; 95% CI: 0.40–0.72). Treatment of CF patients with ivacaftor in a real-world setting in France led to substantial reduction in HCRU, as evidenced by decreased all-cause hospitalisations and antibiotics use due to PEx.