Evidence map›Paper›PMID 39978537›Full record

Observational studyPulmonary pharmacology & therapeutics2025

Real-life impact of genotype and severity of lung disease on efficacy of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis.

Shahid Sheikh, Melissa Holtzlander, Mariah Eisner, Courtney Gushue, Sabrina Palacios, Kavitha Kotha, Sehyr Imran, Karen S McCoy

Abstract readObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Pulmonary pharmacology & therapeutics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

8 authors.

Shahid SheikhDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA. Electronic address: shahid.sheikh@nationwidechildrens.org.
Melissa HoltzlanderDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA.
Mariah EisnerNationwide Children's Hospital, Columbus, OH, USA; Biostatistics Resource, Columbus, OH, USA.
Courtney GushueDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA.
Sabrina PalaciosDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA.
Kavitha KothaDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA.
Sehyr ImranDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA.
Karen S McCoyDepartments of Pediatrics, Ohio State University College of Medicine, Columbus, OH, USA; Division of Pulmonary Medicine, Columbus, OH, USA; Nationwide Children's Hospital, Columbus, OH, USA.

Funding

The OSU Center for Clinical and Translational Science: Advancing Today's Discoveries to Improve HealthUL1TR002733 · NCATS · OHIO STATE UNIVERSITY · PI RINGEL, MATTHEW D · 2018 to 2022
$28.9M
NCATS NIH HHS UL1 TR002733
6 · The paper itself

Abstract

backgroundElexacaftor-tezacaftor-ivacaftor (ETI) therapy has shown improvement in lung function, BMI and reduction in pulmonary exacerbations but the impact of genotype and severity of lung disease on heterogeneity of ETI efficacy in real life is not known.

methodsThis is a prospective observational study. Clinical data at baseline and at one-year of therapy were compared for the total cohort and for two subgroups; genotype [homozygous vs. heterozygous for F508del], and severity of lung disease at ETI initiation (ppFEV

resultsAmong the total cohort of 115 pwCF, median age of 23 (17, 32) years, 66 (58 %) were homozygous, 76 (66 %) had ppFEV

conclusionETI therapy improved clinical outcomes in pwCF which were impacted by severity of underlying lung disease.

Indexed as

AminophenolsBenzodioxolesCystic FibrosisIndolesPyrazolesPyridinesQuinolonesAdolescentAdultCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleGenotypeHumansMaleProspective StudiesAminophenolsBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftor, ivacaftor, tezacaftor drug combinationIndolesPyrazolesPyridinesPyrrolidinesQuinolinesQuinolonesCFTR modulatorsCystic fibrosisETIGenotypePulmonary function tests

Identifiers

PMID39978537
PMCPMC13309060

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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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.