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 Wainwright, Claire E. (2013) Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5 years of age. Thorax, 68 7: 643-651. doi:10.1136/thoraxjnl-2012-202342


Author 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.
Wainwright, Claire E.
Title Prospective evaluation of respiratory exacerbations in children with cystic fibrosis from newborn screening to 5 years of age
Journal name Thorax   Check publisher's open access policy
ISSN 0040-6376
1468-3296
Publication date 2013-07
Sub-type Article (original research)
DOI 10.1136/thoraxjnl-2012-202342
Volume 68
Issue 7
Start page 643
End page 651
Total pages 9
Place of publication United Kingdom
Publisher B M J Group
Collection year 2014
Language eng
Formatted abstract
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 hospital-managed 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.
Keyword Randomized controlled-trial
Inhaled hypertonic saline
Pulmonary exacerbations
Pseudomonas-aeruginosa
Clinical-trials
Scoring system
Tract illness
Birth cohort
Infections
Life
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

 
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