Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function.

Abstract Background Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA) in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and percent predicted forced expiratory volume in 1 s (ppFEV1) between 40 and 90. Marketing authorizations have been granted for patients at all levels of ppFEV1. Methods To evaluate the safety and effectiveness of LUMA-IVA over the first year of treatment in patients with ppFEV1 Results 827 patients were classified into 3 subgroups according to ppFEV1 at treatment initiation (ppFEV1 Conclusion Phe508del homozygous CF patients benefit from LUMA-IVA at all levels of baseline lung function, but the characteristics and magnitude of the response vary depending on ppFEV1 at baseline.