Pulmonary function deficits in newborn screened infants with cystic fibrosis managed with standard UK care are mild and transient.

Gwyneth Davies, Janet Stocks, Lena P Thia, Ah-Fong Hoo, Andrew Bush, Paul Aurora, Lucy Brennan, Simon Lee, Sooky Lum, Philippa Cottam, Joanne Miles, Jane Chudleigh, Jane Kirkby, Ian M Balfour-Lynn, Siobhán B Carr, Colin Wallis, Hilary Wyatt, Angie Wade

Journal: The European respiratory journal 2018;50(5):1700326

PMID: 29122914

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Abstract

With the advent of novel designer molecules for cystic fibrosis (CF) treatment, there is huge need for early-life clinical trial outcomes, such as infant lung function (ILF). We investigated the degree and tracking of ILF abnormality during the first 2 years of life in CF newborn screened infants.Forced expiratory volume in 0.5 s (FEV), lung clearance index (LCI) and plethysmographic functional residual capacity were measured at ∼3 months, 1 year and 2 years in 62 infants with CF and 34 controls.By 2 years there was no significant difference in FEV z-score between CF and controls, whereas mean LCI z-score was 0.81 (95% CI 0.45-1.17) higher in CF. However, there was no significant association between LCI z-score at 2 years with either 3-month or 1-year results. Despite minimal average group changes in any ILF outcome during the second year of life, marked within-subject changes occurred. No child had abnormal LCI or FEV on all test occasions, precluding the ability to identify "high-risk" infants in early life.In conclusion, changes in lung function are mild and transient during the first 2 years of life in newborn screened infants with CF when managed according to a standardised UK treatment protocol. Their potential role in tracking disease to later childhood will be ascertained by ongoing follow-up.

Address: Respiratory, Anaesthesia and Critical Care Section, Infection, Immunity, Inflammation Programme, UCL Great Ormond Street Institute of Child Health, London, UK [email protected].; Respiratory Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.; Respiratory, Anaesthesia and Critical Care Section, Infection, Immunity, Inflammation Programme, UCL Great Ormond Street Institute of Child Health, London, UK.; Respiratory, Anaesthesia and Critical Care Section, Infection, Immunity, Inflammation Programme, UCL Great Ormond Street Institute of Child Health, London, UK.; Respiratory Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.; Dept of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK.; National Heart and Lung Institute, Imperial College London, London, UK.; Respiratory Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.; Dept of Child Health, King's College London, London, UK.; Dept of Child Health, City, University of London, London, UK.; Dept of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK.; Dept of Paediatric Respiratory Medicine, Royal London Hospital, London, UK.; Dept of Child Health, King's College London, London, UK.; Clinical Epidemiology, Nutrition and Biostatistics, Population, Policy and Practice Programme, UCL Great Ormond Street Institute of Child Health, London, UK.

DOI: 10.1183/13993003.00326-2017

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MeSH Terms: Case-Control Studies, Cystic Fibrosis, Female, Forced Expiratory Volume, Functional Residual Capacity, Humans, Infant, Infant, Newborn, Lung, Male, Neonatal Screening, Regression Analysis, United Kingdom