OBJECTIVES : The efficacy of ivacaftor in cystic fibrosis (CF) patients with non-G551D gating mutations was demonstrated in a Phase 3 randomized-controlled trial (KONNECTION). An observational study (VOCAL) is ongoing to evaluate real world outcomes of ivacaftor in the non-G551D gating population; this interim analysis describes healthcare resource utilization (HCRU) over 12 months from the VOCAL study.

METHODS : VOCAL is a Phase 4, multi-center, multinational observational study of patients with CF on ivacaftor in UK, Italy and Netherlands, aged ≥6 years, with one non-G551D gating mutation. VOCAL has 4 years of planned prospective data collection from time of enrollment. In the current interim analysis, HCRU including hospitalizations and antibiotic use were evaluated as exploratory endpoints for 12 months before and 12 months after ivacaftor initiation using a negative binomial model.

RESULTS : CONCLUSIONS : This study demonstrates the significant impact of ivacaftor on HCRU including hospitalizations and antibiotic use in CF patients with non-G551D gating mutations, following initiation of ivacaftor treatment in a real-world setting.