Observational studyThe European respiratory journal2026
Impact of CFTR modulator concentrations on clinical response in cystic fibrosis.
Observational study in The European respiratory journal, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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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.
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.
Who cites it
1 citing paper in PubMed.
- Evolving Cystic Fibrosis Therapy: The Good, the Sad, and the Hopeful.Children (Basel, Switzerland) · 2026Review
Corrections and comments
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Authors and funding
11 authors.
Funding
Abstract
rationaleCystic fibrosis (CF) is caused by variants in the CF transmembrane conductance regulator (
objectivesThis study aimed to evaluate modulator concentrations among people with CF and the potential impact of cytochrome P450 (CYP)3A5 genotypes on sweat chloride.
methodsThis multicentre study enrolled 97 children and adult participants established on elexacaftor/tezacaftor/ivacaftor (ETI) therapy. ETI drug concentrations were quantified and CYP3A5 genotypes were determined. Relationship between drug concentrations, genotype, and sweat chloride response were investigated using correlation and multivariable regression models to examine associations between drug levels and sweat chloride. MEASUREMENTS AND MAIN
resultsPlasma concentrations of elexacaftor, tezacaftor and ivacaftor were highly variable. Analyses revealed that CYP3A5 genotype status had no significant effect on drug concentrations. Association analysis demonstrated an association of sweat chloride with drug concentrations including after adjusting for pre-modulator sweat chloride, age, race, body mass index and sex, showing that lower drug concentrations are associated with worse outcomes in sweat chloride.
conclusionThis study provides evidence that lower drug concentration are associated with worse sweat chloride levels and may be a potential indicator of therapeutic effectiveness, especially for people with high sweat chloride despite treatment. The influence of drug variability underscores the need for personalised dosing strategies to improve CF treatment outcome, although well-known CYP3A5 genotypes are unlikely to be helpful.
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Registered trials
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