Observational studyThorax2014
Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants.
Observational study in Thorax, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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
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
26 citing papers in PubMed.
- A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous forAmerican journal of respiratory and critical care medicine · 2022Trial
- Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2016Trial
- Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study.The Lancet. Respiratory medicine · 2016Trial
- Persistence of lung hyperinflation and small airway dysfunction in school-aged children with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: results from the real-world MODUL-CF study.ERJ open research · 2026Article
- Early structural lung changes in young children with cystic fibrosis: a call to action.Respiratory research & clinical practice · 2026Article
- Pilot Evaluation of Intestinal Current Measurement in Cystic Fibrosis and CRMS/CFSPID Patients in Poland.Journal of clinical medicine · 2025Article
- Infant Lung Function in Cystic Fibrosis: A Real-World Study.Pediatric pulmonology · 2025Article
- Levelling the playing field through the London Network of the UK clinical trials accelerator platform.Contemporary clinical trials communications · 2024Article
- Nationwide lung function monitoring from infancy in newborn-screened children with cystic fibrosis.ERJ open research · 2023Article
- Pulmonary Function Tests in the Evaluation of Early Lung Disease in Cystic Fibrosis.Journal of clinical medicine · 2023Article
- Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children.Children (Basel, Switzerland) · 2022Review
- Hyperinflation is associated with increased respiratory rate and is a more sensitive measure of cystic fibrosis lung disease during infancy compared to forced expiratory measures.Pediatric pulmonology · 2021Article
- Quantification of Phenotypic Variability of Lung Disease in Children with Cystic Fibrosis.Genes · 2021Review
- Toward the Establishment of New Clinical Endpoints for Cystic Fibrosis: The Role of Lung Clearance Index and Cardiopulmonary Exercise Testing.Frontiers in pediatrics · 2021Review
- Pulmonary function testing in children's interstitial lung disease.European respiratory review : an official journal of the European Respiratory Society · 2020Review
- The future of cystic fibrosis care: a global perspective.The Lancet. Respiratory medicine · 2020Review
- AGTR2 absence or antagonism prevents cystic fibrosis pulmonary manifestations.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2019Article
- Article
- Best practices in the treatment of early cystic fibrosis lung disease.Therapeutic advances in respiratory disease · 2017Review
- Early pulmonary disease manifestations in cystic fibrosis mice.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2016Article
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
rationaleNewborn screening (NBS) for cystic fibrosis (CF) allows early intervention. Design of randomised controlled trials (RCT) is currently impeded by uncertainty regarding evolution of lung function, an important trial end point in such infants.
objectiveTo assess changes in pulmonary function during the first year of life in CF NBS infants.
methodsObservational longitudinal study. CF NBS infants and healthy controls were recruited between 2009 and 2011. Lung Clearance Index (LCI), plethysmographic lung volume (plethysmographic functional residual capacity (FRCpleth)) and forced expired volume (FEV₀.₅) were measured at 3 months and 1 year of age. MAIN
resultsPaired measurements were obtained from 72 CF infants and 44 controls. At 3 months, CF infants had significantly worse lung function for all tests. FEV₀.₅ improved significantly (0.59 (95% CI 0.18 to 0.99) z-scores; p<0.01) in CF infants between 3 months and 1 year, and by 1 year, FEV₀.₅ was only 0.52 (0.89 to 0.15) z-scores less than in controls. LCI and FRCpleth remained stable throughout the first year of life, being on average 0.8 z-scores higher in infants with CF. Pulmonary function at 1 year was predicted by that at 3 months. Among the 45 CF infants with entirely normal LCI and FEV₀.₅ at 3 months, 80% remained so at 1 year, while 74% of those with early abnormalities remained abnormal at 1 year.
conclusionsThis is the first study reporting improvements in FEV₀.₅ over time in stable NBS CF infants treated with standard therapy. Milder changes in lung function occurred by 1 year than previously reported. Lung function at 3 months predicts a high-risk group, who should be considered for intensification of treatment and enrolment into RCTs.
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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.