Observational studyPediatric pulmonology2020
Long-term effects of ivacaftor on nonpulmonary outcomes in individuals with cystic fibrosis, heterozygous for a S1251N mutation.
Observational study in Pediatric pulmonology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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
4 citing papers in PubMed, 9 citations in OpenAlex.
- Development of a Cystic Fibrosis Weight Management Programme: protocol for a co-design study.NIHR open research · 2026Article
- Longitudinal changes in habitual physical activity in adult people with cystic fibrosis in the presence or absence of treatment with elexacaftor/tezacaftor/ivacaftor.Frontiers in sports and active living · 2024Article
- Nutritional Care in Children with Cystic Fibrosis.Nutrients · 2023Review
- Long-term effects of ivacaftor on nonpulmonary outcomes in individuals with cystic fibrosis, heterozygous for a S1251N mutation.Pediatric pulmonology · 2020Observational
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesTo describe the long-term effects of ivacaftor (Kalydeco®) in individuals with cystic fibrosis (CF) on body mass index (BMI), body composition (BC), pulmonary function (PF), resting energy expenditure (REE), and exercise capacity (EC) after ≥12 months of treatment. WORKING HYPOTHESIS: BMI, lean and fat mass, PF, and EC will increase and REE will decrease after treatment. STUDY
designObservational study. METHODOLOGY: Seven individuals with CF (mean age 15.4 ± 5.8 years) heterozygous for S1251N mutation, starting with ivacaftor, were included. Paired t tests were performed to assess the effects of ivacaftor. Height and weight were used to calculate BMI and BMI Z-scores. Dual-energy X-ray absorptiometry was used to assess BC. Spirometry and body plethysmography were used to assess PF. Indirect calorimetry was used to measure REE and cardiopulmonary exercise testing (CPET) was used to measure oxygen uptake (VO
resultsAfter a median of 15 (interquartile range: 13-16) months of treatment, BMI increased significantly (P = .03), but not BMI Z-score (P = .23) or BC. Significant improvements were found for several PF variables, especially measures of hyperinflation (P = .02). Absolute VO
conclusionsThe results showed that long-term treatment of ivacaftor is associated with improvement of BMI and PF, but not of BC and REE. Oxygen uptake reduced after treatment, which may be due to a decrease in work of breathing.
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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.