ArticlePediatric research2026
Real-life effectiveness and safety of lumacaftor/ivacaftor in preschool children with cystic fibrosis: data from an italian multicentre study.
Article in Pediatric research, 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.
- Early CFTR modulation and clinical outcomes in preschool cystic fibrosis.Pediatric research · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundLumacaftor-ivacaftor (LUM-IVA) is a CFTR modulator approved for children aged >1 year who are homozygous for the F508del variant, targeting the underlying molecular defect. Despite evidence from clinical trials, real-world data in preschool-aged children remain limited. This study evaluated the effectiveness, safety, and tolerability of LUM-IVA in this population over 12 months of follow-up.
methodsWe conducted a multicenter, prospective study in Italy including 69 children with CF aged 1-6 years treated with LUM-IVA. Anthropometric, laboratory, microbiological, and functional parameters were collected at baseline, and after 3, 6 and 12 months.
resultsThe median age was 3.82 years. LUM-IVA treatment was associated with significant reductions in sweat chloride concentration and pulmonary exacerbation frequency between baseline and 3/6 months. Weight improved significantly between T0 and T3 and between T1 and T2. Lung Clearance Index (LCI) also showed significant improvement between baseline and 6-12 months. Adverse events were mild and manageable; temporary treatment interruptions occurred, with no permanent discontinuations.
conclusionIn conclusion, in this real-world cohort of preschool children with CF homozygous for F508del, LUM-IVA therapy was associated with improvements in sweat chloride, pulmonary exacerbation frequency, and weight over 12 months, with an acceptable safety profile. These observational findings support the feasibility of early CFTR modulator therapy. IMPACT: LUM-IVA therapy in preschool children with CF shows significant real-world improvements in sweat chloride, exacerbations, weight, and LCI over 12 months, with an acceptable safety profile. It provides crucial real-world, observational data on LUM-IVA effectiveness and safety. The findings support the early use of LUM-IVA in young children with CF and potentially mitigate early progressive CF damage.
Identifiers
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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.