ArticlePediatric pulmonology2022
Plasma and cellular ivacaftor concentrations in patients with cystic fibrosis.
Article in Pediatric pulmonology, 2022. 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.
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
11 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.
- Pharmacokinetics of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor in Special Cystic Fibrosis Populations: A Systematic Review.Clinical pharmacokinetics · 2025Pooled it
- Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial.American journal of respiratory and critical care medicine · 2025Trial
- Physiologically Based Pharmacokinetic Modeling of Elexacaftor-Tezacaftor-Ivacaftor in Adults and Pediatric People with Cystic Fibrosis: Exploring Brain Exposure and Neuropsychiatric Adverse Events.Clinical pharmacokinetics · 2026Article
- Impact of CFTR modulator concentrations on clinical response in cystic fibrosis.The European respiratory journal · 2026Observational
- Predicting Maternal and Fetal Exposures of Elexacaftor-Tezacaftor-Ivacaftor during Pregnancy through Physiologically Based Pharmacokinetic Models.Clinical pharmacology and therapeutics · 2025Article
- VX-445 (elexacaftor) inhibits chloride secretion across human bronchial epithelial cells by directly blocking KCa3.1 channels.PNAS nexus · 2025Article
- Evaluating the effects of ivacaftor exposure onJAC-antimicrobial resistance · 2024Article
- Article
- Pharmacokinetic variability of CFTR modulators from standard and alternative regimens.Pulmonary pharmacology & therapeutics · 2024Article
- Differential distribution of ivacaftor and its metabolites in plasma and human airway epithelia.Pulmonary pharmacology & therapeutics · 2024Article
- Drug metabolism of ciprofloxacin, ivacaftor, and raloxifene by Pseudomonas aeruginosa cytochrome P450 CYP107S1.The Journal of biological chemistry · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 2 institutions in 1 country.
Funding
Abstract
Access to cystic fibrosis transmembrane conductance regulator (CFTR) modulators has been gradually increasing for people with cystic fibrosis, the first of which was ivacaftor, a CFTR potentiator that is part of all clinically available modulator treatments. In this study, we hypothesized that the steady-state concentrations in blood and tissue are highly variable in patients taking ivacaftor in a real-world context, which may have an impact on the treatment approach. We collected nasal epithelial cells to estimate target site concentrations and blood samples to estimate pharmacokinetic parameters at a steady state. We found that patients on ivacaftor monotherapy have variable concentrations well above the maximal effective concentration and may maintain concentrations necessary for the clinical benefit even if dosing is reduced. We also are the first to provide detailed target site concentration data over time, which shows that tissue concentrations do not fluctuate significantly and do not correlate with plasma concentrations. These findings show that some patients may have higher-than-expected concentrations and may benefit from tailored dosing to balance clinical response with side effects or adherence needs.
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Registered trials
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.