ArticlePediatric pulmonology2025
Introduction of Ivacaftor/Lumacaftor in Children With Cystic Fibrosis Homozygous for F508del in the Netherlands: A Nationwide Real-Life Study.
Article in Pediatric pulmonology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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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
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Who cites it
3 citing papers in PubMed.
- A descriptive case series of hepatotoxicity associated with CFTR modulators and possible relevance of pharmacogenetic polymorphisms in cystic fibrosis patients.British journal of clinical pharmacology · 2026Article
- Real-life effectiveness and safety of lumacaftor/ivacaftor in preschool children with cystic fibrosis: data from an italian multicentre study.Pediatric research · 2026Article
- Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis: A Review of Registry-Based Evidence.Journal of clinical medicine · 2025Review
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Authors and funding
14 authors.
Funding
Abstract
introductionLumacaftor/ivacaftor (lum/iva) was introduced in the Netherlands in 2017. We investigated 1-year efficacy of lum/iva on lung function and small airway and structural lung disease evaluated by multiple breath nitrogen washout and CT scan. Additionally, we investigated effects of lum/iva on exacerbations, anthropometry, sweat chloride and safety in children with CF in the Netherlands.
methodsChildren with CF aged 6-18 years and homozygous for F508del treated in one of the seven Dutch CF centers for at least 12 months were eligible. Data were extracted from the Dutch CF Registry and electronic patient records. Primary outcome was change in percent predicted FEV
resultsNationwide, 247 children with CF were eligible for lum/iva. Eight patients discontinued lum/iva due to side effects. In total, 223/247 children (90.3%) were evaluated. Mean (range) age at baseline was 11.0 (6.0-17.1) years. There was no change in FEV
conclusionLum/iva was generally well tolerated in a real-life, nationwide pediatric cohort. The efficacy of lum/iva was comparable to phase 3 studies in children. LCI and BA analysis as markers of small airway and structural lung disease showed significant improvement which indicates the importance of these parameters to evaluate treatment effects of CF modulators in children.
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