Evidence map›Paper›PMID 41482647›Full record

Observational studyTherapeutic advances in respiratory disease

Assessment of efficacy and tolerability of elexacaftor-tezacaftor-ivacaftor in an observational cohort study of "aged" people with cystic fibrosis.

Sydney Blankenship, Benjamin V Flueckiger, Godsgift Caleb, Julianna Bailey, Elizabeth Baker, Bryan Garcia, Stefanie Krick

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

Observational study in Therapeutic advances in respiratory disease. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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0cells of the map it votes in
0citing papers in PubMed
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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

7 authors.

Sydney BlankenshipDivision of Internal Medicine, The University of Alabama at Birmingham, Birmingham, AL, USA.
Benjamin V FlueckigerDivision of Internal Medicine, The University of Alabama at Birmingham, Birmingham, AL, USA.
Godsgift CalebDivision of Pulmonary, Allergy, Critical Care, and Sleep Medicine, The University of Alabama at Birmingham, Birmingham, AL, USA.
Julianna BaileyDivision of Pulmonary, Allergy, Critical Care, and Sleep Medicine, The University of Alabama at Birmingham, Birmingham, AL, USA.ORCID 0000-0003-1181-4782
Elizabeth BakerDepartment of Anesthesiology, The University of Alabama at Birmingham, Birmingham, AL, USA.
Bryan GarciaDivision of Pulmonary, Allergy, Critical Care, and Sleep Medicine, The University of Alabama at Birmingham, Birmingham, AL, USA.
Stefanie KrickDivision of Pulmonary, Allergy, Critical Care, and Sleep Medicine, The University of Alabama at Birmingham, THT422, 1900 University Boulevard, Birmingham, AL 35294, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundTherapeutic advancements utilizing modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) have revolutionized the treatment of people with cystic fibrosis (pwCF). Elexacaftor-Tezacaftor-Ivacaftor (ETI) is a highly effective modulator therapy and has been shown to improve health outcomes in people with CF (PwCF). Due to these therapeutic advancements, many pwCF are getting older, but little is known regarding the safety and efficacy of ETI in pwCF at a more advanced age.

objectivesWe aimed to determine the effect of ETI on clinical outcomes in older pwCF.

designThis study was a single-center, retrospective analysis of pwCF who received open-label ETI following FDA approval and were over the age of 40 at the time of ETI initiation.

methodsData were obtained from the electronic medical record from a large CF center in the United States of America between November 2019 and January 2021, including body mass index (BMI), lung function as % predicted FEV

resultsForty-two patients met the inclusion criteria. Mean age at time of ETI initiation was 47.9, 23 patients (54.8%) were male, and 11 (26.2%) were homozygous for the F508del mutation. Linear mixed effects models suggest a monthly increase of 0.24 (95% CI 0.08-0.41,

conclusionTreatment with ETI in this unique cohort of pwCF was safe. Whereas ETI affected BMI in a subtle way, initiation of ETI was associated with stabilization of lung disease with a significant but moderate increase in lung function and a decline in the number of exacerbations in the follow-up period. Longer and larger studies will be needed to analyze the effect of ETI on an aging CF population.

Indexed as

AminophenolsChloride Channel AgonistsCystic FibrosisHeterocyclic CompoundsAdultAge FactorsBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsFemaleHumansIndolesMaleMiddle AgedPyrazolesPyridinesAminophenolsBenzodioxolesCFTR protein, humanChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationselexacaftor, ivacaftor, tezacaftor drug combinationHeterocyclic CompoundsIndolesPyrazolesPyridinesQuinolinesagingCFTR modulator therapycystic fibrosis

Identifiers

PMID41482647
PMCPMC12759134

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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.