Evidence map›Paper›PMID 39227072›Full record

ArticleThe European respiratory journal2024

Impact of the expanded label for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with no F508del variant in the USA.

Elizabeth A Cromwell, Josh S Ostrenga, Don B Sanders, Wayne Morgan, Carlo Castellani, Rhonda Szczesniak, Pierre-Regis Burgel

Abstract read
In one paragraph

Article in The European respiratory journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed, 1 pooled it
–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

11 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
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  8. Review
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors.

Elizabeth A CromwellCystic Fibrosis Foundation, Bethesda, MD, USA ecromwell@cff.org.
Josh S OstrengaCystic Fibrosis Foundation, Bethesda, MD, USA.
Don B SandersDepartment of Pediatrics, Indiana University School of Medicine and Riley Hospital for Children, Indianapolis, IN, USA.
Wayne MorganDepartment of Pediatrics, University of Arizona College of Medicine, Tucson, AZ, USA.
Carlo CastellaniCystic Fibrosis Center, IRCCS Instituto Giannina Gaslini, Genoa, Italy.
Rhonda SzczesniakCincinnati Children's Hospital Medical Center and University of Cincinnati, Cincinnati, OH, USA.
Pierre-Regis BurgelRespiratory Medicine and French National Reference CF Center, Hôpital Cochin, AP-HP, Paris, France.ORCID https://orcid.org/0000-0003-0903-9828

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundElexacaftor/tezacaftor/ivacaftor (ETI), which is approved for people with cystic fibrosis (pwCF) with a F508del variant, was further approved based on

methodsPwCF, aged ≥6 years, carrying no F508del variant but with at least one of these 177 rare variants, were identified within the US Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2020 and 2022. The evolution of forced expiratory volume in 1 s (FEV

resultsA total of 1791 individuals aged ≥6 years with rare

conclusionsExtension of the ETI label to rare

Indexed as

AminophenolsBenzodioxolesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsIndolesPyrazolesQuinolonesAdolescentAdultChildChloride Channel AgonistsDisease ProgressionFemaleForced Expiratory VolumeHumansAminophenolsBenzodioxolesCFTR protein, humanChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftorelexacaftor, ivacaftor, tezacaftor drug combinationIndolesivacaftorPyrazolesPyridinesPyrrolidinesQuinolinesQuinolonestezacaftor

Identifiers

PMID39227072
PMCPMC11561404

What OpenQuestion holds

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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.