Rationale: The markedly improved life expectancy of children with cystic fibrosis (CF) has created a new challenge, as traditional markers of lung disease are frequently normal in young children. This prevents identification of individuals who may benefit from more aggressive therapy and also obliges large study numbers and prolonged duration for intervention studies. There is an urgent need for alternative surrogates that detect early lung disease and track through early childhood.
Objectives: This study aimed to determine whether multiple-breath washout (MBW) results at preschool age can predict subsequent abnormal lung function.
Methods: Preschool children (3-5 yr) with CF and healthy control subjects underwent spirometry and MBW with testing repeated during early school age (6-10 yr). Primary outcomes were FEV1 from spirometry and lung clearance index (LCI) from MBW.
Measurements And Main Results: Forty-eight children with CF and 45 healthy children completed testing. Thirty-five (73%) children with CF had abnormal LCI at preschool age, whereas only five had abnormal FEV1. The positive predictive value of preschool LCI for predicting any abnormal school-age result was 94%, with a negative predictive value of 62%. Only one child with abnormal FEV1 at school age had had a normal preschool LCI. In contrast, for preschool FEV1 the positive predictive value was 100%, but negative predictive value was only 25%.
Conclusions: This study demonstrates that an abnormal preschool LCI predicts subsequent lung function abnormalities, whereas a normal preschool LCI usually remains normal. MBW has potential as a clinical and research outcome in young children with CF.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1164/rccm.200911-1646OC | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Evaluation of safety and effectiveness of the LISTENT LCI-20PI cochlear implant in prelingually deafened children.
Int J Pediatr Otorhinolaryngol
November 2024
ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, 200031, China; NHC Key Laboratory of Hearing Medicine (Fudan University), Shanghai, 200031, China; Shanghai Hearing Medical Center, Shanghai, China. Electronic address:
Objectives: This prospective multicenter clinical trial was to evaluate the safety and effectiveness of a novel cochlear implant (CI) system, the LISTENT LCI-20PI device in prelingually deafened children (<6 years old).
Design: The LCI-20PI CI system was implanted in 70 prelingually deafened children (<6 years old). The median age (interquartile range) at implantation was 3 years old (2-4 years old).
Global Lung Function Initiative reference values for multiple breath washout indices.
Eur Respir J
December 2024
Division of Infection, Immunity and Respiratory Medicine, University of Manchester, Manchester, UK
Background: Multiple breath washout is a lung function test based on tidal breathing that assesses lung volume and ventilation distribution. The aim of this analysis was to use the Global Lung Function Initiative methodology to develop all-age reference equations for the multiple breath washout indices lung clearance index (LCI) and functional residual capacity (FRC).
Methods: Multiple breath washout data from healthy individuals were collated from sites.
Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for : A Phase 2, Open-Label Clinical Trial.
Ann Am Thorac Soc
November 2024
Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and.
Clinical trials show that lumacaftor/ivacaftor (LUM/IVA) treatment has the potential to modify early cystic fibrosis (CF) disease progression in children as young as 2 years of age. To assess the long-term impact of LUM/IVA treatment on CF disease progression in children aged 2-5 years. This phase 2 trial had two parts: part 1, a 48-week, randomized, double-blind, placebo-controlled study of LUM/IVA in children aged 2-5 years (previously reported) was followed by a 48-week open-label treatment period in which all children received LUM/IVA (part 2; reported here).
View Article and Find Full Text PDFComparative sensitivity of early cystic fibrosis lung disease detection tools in school aged children.
J Cyst Fibros
September 2024
The Children's Hospital at Westmead, Westmead, New South Wales, Australia; Woolcock Institute of Medical Research, Sydney, New South Wales, Australia; The University of Sydney, Sydney, New South Wales, Australia; Children's Health and Environment Program, Child Health Research Centre, University of Queensland, South Brisbane, Australia. Electronic address:
Background: Effective detection of early lung disease in cystic fibrosis (CF) is critical to understanding early pathogenesis and evaluating early intervention strategies. We aimed to compare ability of several proposed sensitive functional tools to detect early CF lung disease as defined by CT structural disease in school aged children.
Methods: 50 CF subjects (mean±SD 11.
Lung structural and functional impairments in young children with cystic fibrosis diagnosed following newborn screening - A nationwide observational study.
J Cyst Fibros
September 2024
Pediatric Respiratory Medicine, Department of Pediatrics, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland; Wal-yan Respiratory Research Centre, Telethon Kids Institute, University of Western Australia, Perth WA Australia. Electronic address:
Background: Non-invasive and sensitive clinical endpoints are needed to monitor onset and progression of early lung disease in children with cystic fibrosis (CF). We compared lung clearance index (LCI), FEV, functional and structural lung magnetic resonance imaging (MRI) outcomes in Swiss children with CF diagnosed following newborn screening.
Methods: Lung function (LCI, FEV) and unsedated functional and structural lung MRI was performed in 79 clinically stable children with CF (3 - 8 years) and 75 age-matched healthy controls.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!