S60 Observational study of ivacaftor in people with cystic fibrosis and selected non-G551D gating mutations: final results from VOCAL

Introduction and ObjectivesVOCAL, a Phase 4 observational study (NCT02445053), assessed real-world effectiveness of ivacaftor (IVA) in people with cystic fibrosis (pwCF) with ≥1 non–G551D gating mutation (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D).MethodsPwCF aged ≥6 years in Italy, the Netherlands and the UK who were IVA-naïve or on IVA for ≤18 months at enrolment were eligible. Data were recorded for 12 months pre-IVA and up to 48 months after enrolment. Continuous outcomes (e.g. percent predicted forced expiratory volume in 1 second [ppFEV1], body mass index [BMI]) were assessed from baseline (the last pre-IVA value recorded) in 6-month intervals up to 48 months post-IVA using a mixed model for repeated measures; total pulmonary exacerbations (PEx) and healthcare resource utilisation (HCRU) post- vs pre-IVA were assessed using a negative binomial model.Results65 of 73 (89%) completed the study; mean IVA exposure was 49.5 months (range, 2–64). Mean baseline age was 26.9 years (standard deviation [SD], 13.5). Mean baseline ppFEV1 (64.83 [SD, 23.61]) increased by a least-squares (LS) mean of 10.77 (standard error [SE], 1.28) within 6 months that was sustained up to 48 months (10.27 [SE, 1.45]). Mean baseline BMI (pwCF ≥20 years, n=49; 22.95 kg/m2 [SD, 3.81]) increased by an LS mean of 0.79 (SE, 0.14) within 6 months and 1.30 (SE, 0.24) at 48 months. Mean baseline BMI z score (pwCF 50% in the first 12 months post- vs 12 months pre-IVA, and changes were sustained during treatment. No new safety concerns were identified.ConclusionsIVA showed sustained effectiveness in clinical outcomes and decreased HCRU.Please refer to page A189 for declarations of interest related to this abstract.