Evidence map›Paper›PMID 40163448›Full record

ReviewTherapeutic advances in respiratory disease

Cystic fibrosis: new challenges and perspectives beyond elexacaftor/tezacaftor/ivacaftor.

Vito Terlizzi, Miquéias Lopes-Pacheco

Abstract readReview
In one paragraph

Review in Therapeutic advances in respiratory disease. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
–field-weighted citation impact
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

17 citing papers in PubMed.

  1. Article
  2. Epigenetic regulation and chromatin organization in cystic fibrosis airways.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026
    Review
  3. Article
  4. Review
  5. Review
  6. A therapeutic revolution: CFTR modulators in cystic fibrosis and their impacts on pregnant women and the fetus.Journal of perinatology : official journal of the California Perinatal Association · 2026
    Review
  7. Review
  8. Article
  9. Prevalence and risk factors for recurrent Staphylococcus aureus small-colony variants in people with cystic fibrosis followed at the Tuscan Regional Reference Center.European journal of clinical microbiology & infectious diseases : official publication of the European Society of Clinical Microbiology · 2026
    Article
  10. Article
  11. Article
  12. Review
  13. Review
  14. Review
  15. Article
  16. Review
  17. MycoKeys · 2025
    Article
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

2 authors.

Vito TerlizziDepartment of Pediatric Medicine, Cystic Fibrosis Regional Reference Center, Meyer Children's Hospital IRCCS, Viale Gaetano Pieraccini 24, Florence, Italy.ORCID 0000-0003-1106-4424
Miquéias Lopes-PachecoDepartment of Pediatrics, Cystic Fibrosis and Airway Disease Research Center, Emory University School of Medicine, Atlanta, GA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Over the past decade, major clinical advances have been made in the healthcare and therapeutic development for cystic fibrosis (CF), a lethal genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. CFTR modulators represent innovative treatments that directly target the primary defects in the mutated CFTR protein and have demonstrated significant clinical benefits for many people with CF (pwCF) who are eligible for these treatments. In particular, the triple combination therapy composed of elexacaftor, tezacaftor, and ivacaftor (ETI) has changed the CF therapeutic landscape by significantly improving lung function, quality of life, and predicted survival rates. Here, we provided a comprehensive summary of the impact of ETI on clinical outcomes and the need for further research on long-term efficacy, side effects, pregnancy, possible drug-drug interactions, and extra-pulmonary manifestations. Moreover, a significant number of pwCF are unresponsive to these drugs or cannot afford their high costs. We, therefore, discussed health inequity issues and alternative therapeutic strategies under development aiming to obtain effective therapies for all pwCF.

Indexed as

AminophenolsBenzodioxolesChloride Channel AgonistsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorIndolesPyrazolesPyridinesPyrrolidinesQuinolonesDrug CombinationsDrug InteractionsHumansMutationQuality of LifeQuinolinesAminophenolsBenzodioxolesCFTR protein, humanChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftorelexacaftor, ivacaftor, tezacaftor drug combinationIndolesivacaftorPyrazolesPyridinesPyrrolidinesQuinolinesQuinolonestezacaftorCFTR modulatorefficacyhealth inequityoutcomessafetytherapy development

Identifiers

PMID40163448
PMCPMC11960163

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.