Evidence map›Paper›PMID 36440690›Full record

Observational studyPharmacology research & perspectives2022

Real-life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients.

Renate Kos, Anne H Neerincx, Dominic W Fenn, Paul Brinkman, Rianne Lub, Steffie E M Vonk, Jolt Roukema, Monique H Reijers, Suzanne W J Terheggen-Lagro, Josje Altenburg and 5 more

Open access · goldAbstract readObservational StudyMulticenter Study
In one paragraph

Observational study in Pharmacology research & perspectives, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed, 1 pooled it
4.3field-weighted citation impact, top 4% 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

19 citing papers in PubMed, 1 synthesis or guideline pooled it, 28 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Observational
  4. Article
  5. State-of-the-Art Review: Transformative Changes in the Care of People With Cystic Fibrosis: Implications for Infectious Diseases Specialists.Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2025
    Review
  6. Review
  7. Article
  8. Article
  9. Observational
  10. Article
  11. Article
  12. Article
  13. Article
  14. [Cystic fibrosis in childhood and adulthood].Innere Medizin (Heidelberg, Germany) · 2024
    Review
  15. A longitudinal analysis of respiratory symptoms in people with cystic fibrosis with advanced lung disease on and off ETI.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024
    Article
  16. Review
  17. Article
  18. Review
  19. Observational
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors at 3 institutions in 1 country.

Renate KosDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.ORCID 0000-0002-9895-9636
Anne H NeerincxDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Dominic W FennDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Paul BrinkmanDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Rianne LubDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Steffie E M VonkDepartment of Hospital Pharmacy, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Jolt RoukemaDepartment of Paediatric Pulmonology, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, The Netherlands.
Monique H ReijersDepartment of Pulmonology, Radboud University Medical Center, Nijmegen, The Netherlands.
Suzanne W J Terheggen-LagroDepartment Paediatric Respiratory Medicine and Allergy, Emma Children's Hospital, Amsterdam University Medical Centres, Amsterdam, The Netherlands.
Josje AltenburgDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Christof J MajoorDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Lieuwe D BosDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Eric G HaarmanDepartment Paediatric Respiratory Medicine and Allergy, Emma Children's Hospital, Amsterdam University Medical Centres, Amsterdam, The Netherlands.
Anke H Maitland-van der ZeeDepartment of Respiratory Medicine, Amsterdam University Medical Centres - loc. AMC, Amsterdam, The Netherlands.
Amsterdam Mucociliary Clearance Disease (AMCD) Research Group
Amsterdam University Medical Centers · NLEmma Kinderziekenhuis · NLRadboud University Nijmegen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator, which has shown efficacy in CF patients (≥6 years) with ≥1 Phe508del mutation and a minimal function mutation. In October 2019, ETI became available on compassionate use basis for Dutch CF patients with severe lung disease. Our objective was to investigate safety and efficacy of ETI in this patient group in a real-life setting. A multicenter longitudinal observational study was conducted to examine changes in FEV

Indexed as

Cystic FibrosisAminophenolsBenzodioxolesHumansIndolesPyrazolesPyridinesPyrrolidinesQuinolonesAminophenolsBenzodioxoleselexacaftorIndolesivacaftorPyrazolesPyridinesPyrrolidinesQuinolonestezacaftorcystic fibrosiselexacaftor/tezacaftor/ivacaftormodulatorssputum

Identifiers

PMID36440690
PMCPMC9703582
OpenAlexW4310197911

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.