title: Evolution of cystic fibrosis lung function in the early years.
abstract: OBJECTIVE
  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).
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: aImperial College bNational Heart and Lung Institute cRoyal Brompton Harefield NHS Foundation Trust London, UK dThe University of Queensland eQueensland Children's Medical Research Institute fDepartment of Children's Health and Environment gWHO Collaborating Centre for Children's Health and Environment, Brisbane, Australia.
authors.forename: Andrew
authors.initials: A
authors.lastname: Bush
authors.forename: Peter D
authors.initials: PD
authors.lastname: Sly
mesh_terms: D003550:Cystic Fibrosis / Q000503:physiopathology*; D006801:Humans; D007231:Infant, Newborn; D008168:Lung / Q000503:physiopathology*; D015997:Neonatal Screening; D016032:Randomized Controlled Trials as Topic; D012129:Respiratory Function Tests