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Cystic fibrosis
Original article
Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
  1. The Thanh-Diem Nguyen1,2,
  2. Lena P Thia1,
  3. Ah-Fong Hoo1,3,
  4. Andrew Bush4,
  5. Paul Aurora1,3,
  6. Angie Wade5,
  7. Jane Chudleigh1,3,
  8. Sooky Lum1,
  9. Janet Stocks1
  10. on behalf of the London Cystic Fibrosis Collaboration (LCFC)
  1. 1Portex Unit: Respiratory Physiology and Medicine, UCL Institute of Child Health, London, UK
  2. 2Department of Respiratory Medicine, Centre Hospitalier Universitaire Sainte-Justine, Montreal, Quebec, Canada
  3. 3Respiratory Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK
  4. 4Department of Paediatric Respiratory Medicine, Imperial College & Royal Brompton & Harefield Hospital NHS Foundation Trust, London, UK
  5. 5Centre for Paediatric Epidemiology and Biostatistics, UCL Institute of Child Health, MRC Centre for Epidemiology of Child Health, London, UK
  1. Correspondence to Dr The Thanh Diem Nguyen, Portex Respiratory Unit, UCL Institute of Child Health, 30 Guilford Street, LondonWC1N 1EH, UK; t.nguyen{at}ucl.ac.uk

Abstract

Rationale Newborn screening (NBS) for cystic fibrosis (CF) allows early intervention. Design of randomised controlled trials (RCT) is currently impeded by uncertainty regarding evolution of lung function, an important trial end point in such infants.

Objective To assess changes in pulmonary function during the first year of life in CF NBS infants.

Methods Observational longitudinal study. CF NBS infants and healthy controls were recruited between 2009 and 2011. Lung Clearance Index (LCI), plethysmographic lung volume (plethysmographic functional residual capacity (FRCpleth)) and forced expired volume (FEV0.5) were measured at 3 months and 1 year of age.

Main results Paired measurements were obtained from 72 CF infants and 44 controls. At 3 months, CF infants had significantly worse lung function for all tests. FEV0.5 improved significantly (0.59 (95% CI 0.18 to 0.99) z-scores; p<0.01) in CF infants between 3 months and 1 year, and by 1 year, FEV0.5 was only 0.52 (0.89 to 0.15) z-scores less than in controls. LCI and FRCpleth remained stable throughout the first year of life, being on average 0.8 z-scores higher in infants with CF. Pulmonary function at 1 year was predicted by that at 3 months. Among the 45 CF infants with entirely normal LCI and FEV0.5 at 3 months, 80% remained so at 1 year, while 74% of those with early abnormalities remained abnormal at 1 year.

Conclusions This is the first study reporting improvements in FEV0.5 over time in stable NBS CF infants treated with standard therapy. Milder changes in lung function occurred by 1 year than previously reported. Lung function at 3 months predicts a high-risk group, who should be considered for intensification of treatment and enrolment into RCTs.

  • Cystic Fibrosis
  • Lung Physiology
  • Paediatric Lung Disaese

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 3.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/3.0/

https://doi.org/10.1136/thoraxjnl-2013-204023

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