Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR

Compared with patients with higher lung function, respiratory events were more common in patients with ppFEV1 < 40; aside from these events, the lumacaftor/ivacaftor safety profile was consistent with previous studies. Results suggest that patients with ppFEV1 < 40 may benefit from treatment initiation at a lower dose with augmented monitoring before increasing to the full dose.