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; London Cystic Fibrosis Collaboration (LCFC)

doi:10.1183/13993003.00326-2017

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

Eur Respir J. 2017 Nov 9;50(5):1700326. doi: 10.1183/13993003.00326-2017. Print 2017 Nov.

Authors

Gwyneth Davies^{1

2}, Janet Stocks³, Lena P Thia³, Ah-Fong Hoo^{3

2}, Andrew Bush^{4

5}, Paul Aurora^{3

2}, Lucy Brennan³, Simon Lee³, Sooky Lum³, Philippa Cottam³, Joanne Miles², Jane Chudleigh^{6

7}, Jane Kirkby^{3

2}, Ian M Balfour-Lynn^{4

5}, Siobhán B Carr^{4

8}, Colin Wallis², Hilary Wyatt⁶, Angie Wade⁹; London Cystic Fibrosis Collaboration (LCFC)

Affiliations

¹ Respiratory, Anaesthesia and Critical Care Section, Infection, Immunity, Inflammation Programme, UCL Great Ormond Street Institute of Child Health, London, UK gwyneth.davies@ucl.ac.uk.
² Respiratory Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
³ Respiratory, Anaesthesia and Critical Care Section, Infection, Immunity, Inflammation Programme, UCL Great Ormond Street Institute of Child Health, London, UK.
⁴ Dept of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK.
⁵ National Heart and Lung Institute, Imperial College London, London, UK.
⁶ Dept of Child Health, King's College London, London, UK.
⁷ Dept of Child Health, City, University of London, London, UK.
⁸ Dept of Paediatric Respiratory Medicine, Royal London Hospital, London, UK.
⁹ Clinical Epidemiology, Nutrition and Biostatistics, Population, Policy and Practice Programme, UCL Great Ormond Street Institute of Child Health, London, UK.

PMID: 29122914
DOI: 10.1183/13993003.00326-2017

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_0.5), 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_0.5 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_0.5 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.

Publication types

Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Case-Control Studies
Cystic Fibrosis / diagnosis*
Cystic Fibrosis / physiopathology*
Female
Forced Expiratory Volume
Functional Residual Capacity
Humans
Infant
Infant, Newborn
Lung / physiopathology*
Male
Neonatal Screening*
Regression Analysis
United Kingdom