Evolution of cystic fibrosis lung function in the early years

Bush, Andrew and Sly, Peter D. (2015) Evolution of cystic fibrosis lung function in the early years. Current Opinion in Pulmonary Medicine, 21 6: 602-608. doi:10.1097/MCP.0000000000000209

Author Bush, Andrew
Sly, Peter D.
Title Evolution of cystic fibrosis lung function in the early years
Journal name Current Opinion in Pulmonary Medicine   Check publisher's open access policy
ISSN 1070-5287
Publication date 2015-09-25
Year available 2015
Sub-type Critical review of research, literature review, critical commentary
DOI 10.1097/MCP.0000000000000209
Open Access Status Not Open Access
Volume 21
Issue 6
Start page 602
End page 608
Total pages 7
Place of publication London, United Kingdom
Publisher Lippincott Williams and Wilkins
Collection year 2016
Language eng
Formatted abstract
Purpose of review: Most treatment of newborn screening-diagnosed cystic fibrosis is not evidence-based; there are very few randomized controlled trials (RCTs). Furthermore, the advent of novel molecular therapies, which could be started at diagnosis, mandates performing RCTs in very young children. However, unless the natural history of early cystic fibrosis lung disease is known, RCTs are impossible. Here, we review the results of two large prospective cohorts of these infants – London Cystic Fibrosis Collaboration (LCFC) (London, UK) and Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST-CF) (Australia).

Recent findings: Nutritional status remained excellent in both the cohorts. Both cohorts reported abnormal lung function aged at 3 months. AREST-CF, which previously reported rapidly declining preschool lung function, now report good conventional school-age spirometry. LCFC reported improvement between 3 months and 1 year, and stability in the second year. AREST-CF also reported a high prevalence of high resolution computed tomographic abnormalities related to free neutrophil elastase in bronchoalveolar lavage; LCFC reported high resolution computed tomographic changes at 1 year, which were too mild to be scored reproducibly.

Summary: At least in the first 2 years of life, lung function is not a good end-point for RCTs; routine bronchoalveolar lavage and HRCT cannot be justified. Newborn screening has greatly improved outcomes, but we need better point-of-care biomarkers
Keyword Bronchiectasis
HRCT scan
Newborn screening
Pulmonary function tests
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collections: Official 2016 Collection
School of Medicine Publications
Child Health Research Centre Publications
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