Evidence map›Paper›PMID 37945033›Full record

Trial reportThe European respiratory journal2023

Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one

Cori L Daines, Elizabeth Tullis, Stefano Costa, Rachel W Linnemann, Marcus A Mall, Edward F McKone, Deepika Polineni, Bradley S Quon, Felix C Ringshausen, Steven M Rowe and 14 more

Open access · greenAbstract readClinical Trial, Phase III
In one paragraph

Trial report in The European respiratory journal, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed, 4 pooled it
20.9field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

47 citing papers in PubMed, 4 syntheses or guidelines pooled it, 81 citations in OpenAlex.

  1. Guideline
  2. Pooled it
  3. Pooled it
  4. Pooled it
  5. Trial
  6. Trial
  7. Efficacy and safety of LAU-7b in a Phase 2 trial in adults with cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025
    Trial
  8. Trial
  9. Review
  10. The dipeptidyl peptidase 1 inhibition landscape in bronchiectasis and its future direction.European respiratory review : an official journal of the European Respiratory Society · 2026
    Review
  11. Article
  12. Pulmonary exacerbations in primary ciliary dyskinesia.European journal of pediatrics · 2026
    Review
  13. Article
  14. Article
  15. Article
  16. mSphere · 2026
    Article
  17. Observational
  18. Article
  19. Article
  20. The impact of elexacaftor-tezacaftor-ivacaftor on cardiometabolic risk factors: a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2026
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

24 authors at 15 institutions in 8 countries.

Cori L DainesUniversity of Arizona, Banner University Medical Center, Tucson, AZ, USA cdaines@arizona.edu.
Elizabeth TullisSt Michael's Hospital, Toronto, ON, Canada.
Stefano CostaUniversity of Messina, Messina, Italy.
Rachel W LinnemannEmory University, Atlanta, GA, USA.ORCID 0000-0002-6839-0936
Marcus A MallCharité - Universitätsmedizin Berlin, Berlin, Germany.ORCID 0000-0002-4057-2199
Edward F McKoneSt Vincent's University Hospital, Dublin, Ireland.
Deepika PolineniWashington University in St Louis, St Louis, MO, USA.
Bradley S QuonUBC and St Paul's Hospital, Vancouver, BC, Canada.
Felix C RingshausenHannover Medical School and German Center for Lung Research (DZL), Hannover, Germany.ORCID 0000-0001-9084-4968
Steven M RoweUniversity of Alabama at Birmingham, Birmingham, AL, USA.
Hiran SelvaduraiThe Children's Hospital at Westmead, Westmead, Australia.
Jennifer L Taylor-CousarNational Jewish Health, Denver, CO, USA.ORCID 0000-0002-5436-9722
Nicholas J WithersRoyal Devon and Exeter NHS Foundation Trust, Exeter, UK.
Neil AhluwaliaVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Samuel M MoskowitzVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Valentin Prieto-CenturionVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Yaoyuan Vincent TanVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Simon TianVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Tanya WeinstockVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Fengjuan XuanVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Yaohua ZhangVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Bonnie RamseySeattle Children's Hospital, Seattle, WA, USA.
Matthias GrieseLudwig Maximilian University and German Center for Lung Research (DZL), Munich, Germany.ORCID 0000-0003-0113-912X
VX17-445-105 Study Group
Vertex Pharmaceuticals (United States) · USZero to Three · USChildren's Hospital at Westmead · AUEmory University · USGerman Center for Lung Research · DEMedizinische Hochschule Hannover · DENational Jewish Health · USRoyal Devon & Exeter NHS Foundation Trust · GBSeattle Children's Hospital · USSt. Michael's Hospital · CASt. Paul's Hospital · CASt. Vincent's University Hospital · IEUniversity of Alabama at Birmingham · USUniversity of Messina · ITWashington University in St. Louis · US

Funding

UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Brian R. Davis · 2007 to 2026
$23.0M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss · 2010 to 2026
$21.4M
NIDDK NIH HHS P30 DK072482NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

backgroundIn two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in patients with cystic fibrosis (CF) ≥12 years of age who have at least one

methodsIn this phase 3, open-label, single-arm extension study, participants with

resultsAt the Week 144 interim analysis, mean duration of exposure to ELX/TEZ/IVA in the extension study was 151.1 weeks. Exposure-adjusted rates of adverse events (AEs) (586.6 events per 100 participant-years) and serious AEs (22.4 events per 100 participant-years) were lower than in the ELX/TEZ/IVA treatment group in the 24-week parent study (1096.0 and 36.9 events per 100 participant-years, respectively); most participants had AEs classified as mild (16.4% of participants) or moderate (60.3% of participants) in severity. 14 participants (2.8%) had AEs that led to treatment discontinuation. Following initiation of ELX/TEZ/IVA, participants had increases in forced expiratory volume in 1 s (FEV

conclusionsELX/TEZ/IVA was generally safe and well tolerated, with a safety profile consistent with the 24-week parent study. Participants had sustained improvements in lung function, respiratory symptoms, CF transmembrane conductance regulator function, pulmonary exacerbation rates and nutritional status. These results support the favourable safety profile and durable, disease-modifying clinical benefits of ELX/TEZ/IVA.

Indexed as

Cystic FibrosisAllelesAminophenolsBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorHumansIndolesMutationPyrazolesPyridinesPyrrolidinesQuinolonesAminophenolsBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorelexacaftorIndolesivacaftorPyrazolesPyridinesPyrrolidinesQuinolonestezacaftor

Identifiers

PMID37945033
PMCPMC10701091
OpenAlexW4388524266

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.