EFFECT OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR ON ANNUAL RATE OF LUNG FUNCTION DECLINE IN PEOPLE WITH CYSTIC FIBROSIS
Open accessAbstract
Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) with ≥ 1 F508del-CFTR allele in Phase 3 clinical trials. ELX/TEZ/IVA treatment led to improved lung function, with increases in percent predicted forced expiratory volume in 1 second (ppFEV 1 ) and Cystic Fibrosis Questionnaire-Revised respiratory domain score. Here, we evaluated the impact of ELX/TEZ/IVA on the rate of lung function decline over time by comparing changes in ppFEV 1 in participants from the Phase 3 trials with a matched group of people with CF from the US Cystic Fibrosis Foundation Patient Registry not eligible for cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy. Participants treated with ELX/TEZ/IVA had on average no loss of pulmonary function over a 2-year period (mean annualized rate of change in ppFEV 1 , +0.39 percentage points [95% CI, −0.06 to 0.85]) compared with a 1.92 percentage point annual decline (95% CI, −2.16 to −1.69) in ppFEV 1 in untreated controls. ELX/TEZ/IVA is the first CFTR modulator therapy shown to halt lung function decline over an extended time period.
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- Authors
- Lee, T., Sawicki, G. S., Altenburg, J., Millar, S. J., Geiger, J. M., Jennings, M. T., Lou, Y., McGarry, L. J., Van Brunt, K., & Linnemann, R. W.
- Affiliations
- Leeds Teaching Hospitals NHS Trust (GB)
- Boston Children's Hospital (US)
- Amsterdam University Medical Centers (NL)
- Imperial Consultants (GB)
- Vertex Pharmaceuticals (United States) (US)
- Emory University (US)
- Corresponding author
- Lee, Tim
- Sawicki, Gregory S.
- Altenburg, Josje
- Millar, Stefanie J.
- Geiger, Jessica Morlando
- Jennings, Mark T.
- Lou, Yiyue
- McGarry, Lisa J.
- Linnemann, Rachel W.
- ORCID
- Venue
- Journal of Cystic Fibrosis
- Publisher
- Elsevier BV
- Published
- 2023-05
- Volume
- 22
- Issue
- 3
- Pages
- 402-406
- ISSN
- 1569-1993 (ISSN-L)
- OpenAlex
- W4313254968
- Cited by
- 56
- References
- 10
- Citations by year
- 2026: 9 · 2025: 11 · 2024: 23 · 2023: 13
- FWCI
- 7.58
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Journal of Cystic Fibrosis (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Amsterdam UMC (VU Amsterdam) - Institutional Repository (publishedVersion, other-oa)
- Pure Amsterdam UMC (submittedVersion, cc-by)
- Data Archiving and Networked Services (DANS) (submittedVersion, other-oa)
- Funders
- Vertex Pharmaceuticals Incorporated
- Cystic Fibrosis Foundation
- Topics
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic and Kidney Cyst Diseases
- Keywords
- elexacaftor/tezacaftor/ivacaftor, CFTR modulator therapy, lung function decline, FEV1, F508del-CFTR, CFTR, lung function preservation
- MeSH terms
- Aminophenols, Cystic Fibrosis, Double-Blind Method, Humans, Indoles, Lung, Mutation, Pyrazoles, Pyridines, Pyrrolidines, Quinolones, Cystic Fibrosis Transmembrane Conductance Regulator, Benzodioxoles, Chloride Channel Agonists
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