Peripheral lung effect of elexacaftor/tezacaftor/ivacaftor in adult cystic fibrosis

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) significantly reduced symptoms and improved forced expiratory volume in one second (FEV1) in randomized controlled trials (RCTs) in both Phe508del homozygous (F/F) [1] and Phe508del heterozygous patients with a minimal function mutation (F/MF) [2]. Patients with an FEV1 below 40%predicted were deliberately excluded from these RCTs. A mono-centric Irish study in 14 adult CF patients with FEV1 below 40%predicted demonstrated an increase of 10.6%predicted in FEV1 at four weeks along with a reduction in pulmonary exacerbation rate during a 5 month follow-up [3].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Short communication Source Type: research