Evidence map›Paper›PMID 36986509›Full record

ReviewPharmaceuticals (Basel, Switzerland)2023

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis.

Mafalda Bacalhau, Mariana Camargo, Grace A V Magalhães-Ghiotto, Sybelle Drumond, Carlos Henrique M Castelletti, Miquéias Lopes-Pacheco

Open access · goldAbstract readReview
In one paragraph

Review in Pharmaceuticals (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
60citing papers in PubMed, 1 pooled it
24.8field-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

60 citing papers in PubMed, 1 synthesis or guideline pooled it, 96 citations in OpenAlex.

  1. Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2024
    Pooled it
  2. Article
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  13. Observational
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  15. Tobacco smoke exposure is associated with diminished longitudinal benefit of elexacaftor/tezacaftor/ivacaftor in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025
    Article
  16. Article
  17. Review
  18. Article
  19. Effects of CFTR Modulators onInfectious disease reports · 2025
    Review
  20. Review
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

6 authors at 4 institutions in 2 countries.

Mafalda BacalhauBiosystems & Integrative Sciences Institute (BioISI), Faculty of Sciences, University of Lisbon, 1749-016 Lisbon, Portugal.
Mariana CamargoDepartment of Surgery, Division of Urology, Sao Paulo Federal University, Sao Paulo 04039-060, SP, Brazil.ORCID 0000-0003-2861-8550
Grace A V Magalhães-GhiottoDepartment of Biotechnology, Genetics, and Cell Biology, Biological Sciences Center, State University of Maringa, Maringa 87020-900, PR, Brazil.ORCID 0000-0002-9750-2853
Sybelle DrumondCenter for Research in Bioethics and Social Health, School of Magistracy of the State of Rio de Janeiro, Rio de Janeiro 20010-090, RJ, Brazil.
Carlos Henrique M CastellettiMolecular Prospecting and Bioinformatics Group, Keizo Asami Institute, Federal University of Pernambuco, Recife 50670-901, PE, Brazil.ORCID 0000-0002-9434-9988
Miquéias Lopes-PachecoBiosystems & Integrative Sciences Institute (BioISI), Faculty of Sciences, University of Lisbon, 1749-016 Lisbon, Portugal.ORCID 0000-0002-7444-9359
University of Lisbon · PTUniversidade Estadual de Maringá · BRUniversidade Federal de Pernambuco · BRUniversidade Federal de São Paulo · BR

Funding

Cystic Fibrosis Foundation LOPES21I0Gilead Sciences (United States) 2018 Research Scholars
6 · The paper itself

Abstract

Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly modified the lives of many people with CF (PwCF) by targeting the fundamental cause of the disease. These drugs consist of the potentiator ivacaftor (VX-770) and the correctors lumacaftor (VX-809), tezacaftor (VX-661), and elexacaftor (VX-445). In particular, the triple combination of CFTR modulators composed of elexacaftor, tezacaftor, and ivacaftor (ETI) represents a life-changing therapy for the majority of PwCF worldwide. A growing number of clinical studies have demonstrated the safety and efficacy of ETI therapy in both short- and long-term (up to two years of follow-up to date) and its ability to significantly reduce pulmonary and gastrointestinal manifestations, sweat chloride concentration, exocrine pancreatic dysfunction, and infertility/subfertility, among other disease signs and symptoms. Nevertheless, ETI therapy-related adverse effects have also been reported, and close monitoring by a multidisciplinary healthcare team remains vital. This review aims to address and discuss the major therapeutic benefits and adverse effects reported by the clinical use of ETI therapy for PwCF.

Indexed as

adverse effectscase reportclinical efficacyclinical trialKaftrioobservational studyprecision medicinereal-life studysafetyTrikafta

Identifiers

PMID36986509
PMCPMC10053019
OpenAlexW4323565408

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.