Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5 years of age

Byrnes, Catherine Ann, Vidmar, Suzanna, Cheney, Joyce L., Carlin, John B., Armstrong, David S., Cooper, Peter J., Grimwood, Keith, Moodie, Marj, Robertson, Colin F., Rosenfeld, Margaret, Tiddens, Harm A. and Wairwright, Claire E. 2013, Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5 years of age, Thorax, vol. 68, no. 7, pp. 643-651.

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Title Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5 years of age
Author(s) Byrnes, Catherine Ann
Vidmar, Suzanna
Cheney, Joyce L.
Carlin, John B.
Armstrong, David S.
Cooper, Peter J.
Grimwood, Keith
Moodie, Marj
Robertson, Colin F.
Rosenfeld, Margaret
Tiddens, Harm A.
Wairwright, Claire E.
Journal name Thorax
Volume number 68
Issue number 7
Start page 643
End page 651
Total pages 9
Publisher BMJ Group
Place of publication London, England
Publication date 2013-07
ISSN 0040-6376
Keyword(s) cystic fibrosis
newborn screening
respiratory exacerbations
Summary Background 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.
Language eng
Field of Research 111403 Paediatrics
Socio Economic Objective 920115 Respiratory System and Diseases (incl. Asthma)
HERDC Research category C1 Refereed article in a scholarly journal
Persistent URL http://hdl.handle.net/10536/DRO/DU:30053692

Document type: Journal Article
Collection: Population Health
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