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Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5 years of age
journal contribution
posted on 2013-07-01, 00:00 authored by C Byrnes, S Vidmar, J Cheney, J Carlin, D Armstrong, P Cooper, K Grimwood, Marj MoodieMarj Moodie, C Robertson, M Rosenfeld, H Tiddens, C WairwrightBackground Newborn screening allows novel treatments for cystic fibrosis (CF) to be trialled in early childhood before irreversible lung injury occurs. As respiratory exacerbations are a potential trial outcome variable, we determined their rate, duration and clinical features in preschool children with CF; and whether they were associated with growth, lung structure and function at age 5 years. Methods: Respiratory exacerbations were recorded prospectively in Australasian CF Bronchoalveolar Lavage trial subjects from enrolment after newborn screening to age 5 years, when all participants underwent clinical assessment, chest CT scans and spirometry. Results 168 children (88 boys) experienced 2080 exacerbations, at an average rate of 3.66 exacerbations per person-year; 80.1% were community managed and 19.9% required hospital admission. There was an average increase in exacerbation rate of 9% (95% CI 4% to 14%; p<0.001) per year of age. Exacerbation rate differed by site (p<0.001) and was 26% lower (95% CI 12% to 38%) in children receiving 12 months of prophylactic antibiotics. The rate of exacerbations in the first 2 years was associated with reduced forced expiratory volume in 1 s z scores. Ever having a hospitalmanaged exacerbation was associated with bronchiectasis (OR 2.67, 95% CI 1.13 to 6.31) in chest CT scans, and lower weight z scores at 5 years of age (coefficient -0.39, 95% CI -0.74 to -0.05). Conclusions Respiratory exacerbations in young children are markers for progressive CF lung disease and are potential trial outcome measures for novel treatments in this age group.
History
Journal
ThoraxVolume
68Issue
7Pagination
643 - 651Publisher
BMJ GroupLocation
London, EnglandISSN
0040-6376Language
engPublication classification
C1 Refereed article in a scholarly journalUsage metrics
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cystic fibrosisnewborn screeningrespiratory exacerbationsScience & TechnologyLife Sciences & BiomedicineRespiratory SystemINHALED HYPERTONIC SALINEPULMONARY EXACERBATIONSPSEUDOMONAS-AERUGINOSACONTROLLED-TRIALCLINICAL-TRIALSSCORING SYSTEMTRACT ILLNESSINFECTIONSBIRTHEFFICACYBronchiectasisRespiratory InfectionACFBAL Study Investigators
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