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Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants

  • Researchers

    Nguyen TT, Thia LP, Hoo AF, Bush A, Aurora P, Wade A, Chudleigh J, Lum S, Stocks J; on behalf of the London Cystic Fibrosis Collaboration (LCFC).

  • Place of research

    University College London Institute of Child Health, London, UK.

  • Publication

    Thorax / September 2013

  • Subjects

    , ,

  • We have previously reported that, even by 3 months of age, lung function was already reduced in many babies diagnosed with Cystic Fibrosis (CF) by newborn screening (NBS). In this follow-up study we asked ‘Does lung function change in NBS infants with CF during the first year of life after they have commenced on a standardised treatment regime?’

  • Why is this important?

    Newborn screening for CF was introduced across the UK in 2007, providing a much needed opportunity for early interventions to minimise progression of lung disease. However until we know how lung disease develops in NBS CF infants treated with the best routine treatment, it is difficult to design suitable clinical trials to see if new treatments are more effective.

  • What did you do?

    We invited all families who had taken part in the London CF Collaboration research study when their baby was approximately 3-months old, to return for follow-up at around one year of age so that we could repeat their lung function measurements at the UCL Institute of Child Health/Great Ormond St Hospital.

  • What did you find?

    72 NBS infants with CF and 44 healthy ‘control’ infants had paired measurements of lung function at 3 months and 1 year of age. After adjusting for body size, lung function was unchanged in healthy infants and either remained stable or improved in infants with CF. Consequently, by one year of age any deficits in lung function were much smaller in the NBS CF infants than we have previously observed in infants diagnosed with CF as a result of symptoms (i.e. prior to the introduction of NBS). The majority of CF infants with normal lung function on the first test occasion continued to have normal function at one year, whereas abnormal lung function during early life was predictive of poorer function at one year of age. There were no differences in body size at one year between those with CF and healthy controls.

  • What does this mean and reasons for caution?

    The results from this study suggest that early identification of CF through NBS and rapid implementation of standard therapy is beneficial both for growth and lung development. The findings of such mild changes at one year of age in the majority of NBS CF infants does, however, mean that very large numbers of infants would need to be recruited to any intervention study in order to demonstrate benefits of any innovative treatments on lung function at this age. This study is the largest of its kind and the only one to compare results directly with those from healthy babies tested with identical methods and over the same time period, which allows results to be interpreted with confidence.

  • What's next?

    This cohort of NBS CF infants and healthy controls is currently being followed up to assess the pattern of lung development during the first five years of life. This will help us to know the best time to commence any studies based on new treatments and how many infants and young children would need to be recruited to any clinical intervention trials.