EFFECT OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR ON ANNUAL RATE OF LUNG FUNCTION DECLINE IN PEOPLE WITH CYSTIC FIBROSIS

• DOI: 10.1016/j.jcf.2022.12.009
• Publication Date: 2022-12-27T12:24:36Z
• Authors: Tim Lee; Gregory S. Sawicki; Josje Altenburg; Stefanie J. Millar; Jessica Morlando ...; Mark T. Jennings; Yiyue Lou; Lisa J. McGarry; Kate Van Brunt; Rachel W. Linnemann
• Keywords: Ivacaftor

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) ≥ 1 F508del-CFTR allele Phase 3 clinical trials. ELX/TEZ/IVA treatment led improved lung function, increases percent predicted forced expiratory volume second (ppFEV1) Cystic Fibrosis Questionnaire-Revised respiratory domain score. Here, we evaluated the impact of on rate function decline over time by comparing changes ppFEV1 participants from trials a matched group CF US Foundation Patient Registry not eligible for transmembrane conductance regulator (CFTR) modulator therapy. Participants treated had average no loss pulmonary 2-year period (mean annualized change ppFEV1, +0.39 percentage points [95% CI, −0.06 0.85]) compared 1.92 point annual (95% −2.16 −1.69) untreated controls. is first CFTR therapy halt an extended period.