Cited by 56Cystic Fibrosis Research AdvancesInhalation and Respiratory Drug DeliveryGenetic and Kidney Cyst Diseases

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

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Lee, Tim, Sawicki, Gregory S., Altenburg, Josje, Millar, Stefanie J., Geiger, Jessica Morlando, Jennings, Mark T., Lou, Yiyue, McGarry, Lisa J. +2 more·2023·Journal of Cystic Fibrosis

Abstract

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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Crossref · 10.1016/j.jcf.2022.12.009

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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.
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
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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