ArticleBMJ open respiratory research2021
Burden of cystic fibrosis in children <12 years of age prior to the introduction of CFTR modulator therapies.
Article in BMJ open respiratory research, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 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
5 citing papers in PubMed.
- Patient-reported outcome measures developed for non-cystic fibrosis bronchiectasis may be applied to cystic fibrosis bronchiectasis.Health and quality of life outcomes · 2026Article
- Observational
- Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.Thorax · 2024Article
- Article
- Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ⩾6 Years with Cystic Fibrosis and at Least OneAmerican journal of respiratory and critical care medicine · 2023Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundCystic fibrosis (CF) is a genetic, multisystemic, progressive and life-shortening disease caused by mutations in the CF transmembrane conductance regulator (
methodsData from the US Cystic Fibrosis Foundation Patient Registry from 2011 were used to summarise key patient and disease characteristics using descriptive statistics, overall and grouped by age (0 to <2 years, 2 to <6 years and 6 to <12 years) and genotype (
resultsThe analysis included 9185 children. Among 6-year-olds to <12-year-olds, mean (SD) per cent predicted FEV
conclusionsPrior to the approval of the first CFTR modulator therapies in children <12 years of age, CF was associated with substantial BOI from an early age-including respiratory infections, hospitalisations/pulmonary exacerbations, need for supplemental nutrition and pharmacological treatments-irrespective of genotype.
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