Cystic fibrosis: Which Babies are at Risk | Action Research

Cystic fibrosis: which babies are at highest risk of the most severe disease?

First published on 26 October 2012

Updated on 24 October 2017

What did the project achieve?

Dr Paul Aurora and his team at University College London Institute of Child Health have moved closer to their goal of finding ways to identify babies and young children with cystic fibrosis (CF) who are at highest risk of the most severe disease. Identifying these children while they’re very young would mean they could be given the most intensive, early treatment.

Around one baby in every 2,500 is born with CF.1 The condition is progressive, mainly affecting the lungs and digestive system, and making it hard to breathe and digest food properly. A key aim of early treatment is to protect the lungs from long-term damage.

In this project, Dr Aurora and his team gathered detailed lung function measurements from a group of pre-school children with and without cystic fibrosis when they were, on average, just under five years old. The team had already studied lung function in the same children when they were babies and at two years of age.

“Our goal was to find out if improvements in lung function seen when the children were two years of age would persist as they got older,” explains Dr Aurora. “Unfortunately, our results showed that many children experienced deteriorations in lung function by pre-school age, despite early diagnosis and treatment.”

However, the researchers found that, by pre-school age, the children’s lung function deteriorated less in the current study than in a similar study 10 years ago. This suggests that overall, the outlook for children with CF is improving.

“Our results also suggest that two different measurements of lung function – one performed soon after birth, and another at two years of age – might help us to predict which children are at particular risk of CF complications,” says Dr Aurora. “If this is so, it would have major implications for our understanding of CF, allowing treatment and monitoring to be tailored to children’s needs.”

References

1. Cystic Fibrosis Trust. Frequently asked questions about cystic fibrosis. How common is cystic fibrosis? https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/faqs

Website accessed 11 August 2017.

This research was completed on 30 November 2016

Over 9,000 people in the UK have cystic fibrosis (CF) – one of the most common, life-threatening inherited diseases.1 When babies are born with CF, it’s hard to predict how severe their illness will be. Dr Paul Aurora, at the University College London Institute of Child Health, is looking for ways to identify which children are at highest risk of developing the most severe disease, so they can be treated more intensively to try and delay the onset of serious damage to their lungs.

What is the problem and who does it affect?

Each week in the UK, five babies are born with CF.1 “CF can make it hard for children to breathe and for their bodies to digest food,” explains Dr Aurora. Symptoms include a persistent cough, recurrent chest infections and poor weight gain.”

Most babies with CF are now diagnosed soon after birth thanks to a nationwide screening programme, which has made early treatment possible for more babies.

“Life expectancy has been increasing, with most children with CF now living through into adulthood,” explains Dr Aurora. Treatment, though increasingly effective, can be time consuming and complex, involving a combination of exercise, medication, a special diet and daily physiotherapy sessions to clear mucus from the lungs.

“CF is progressive, mostly affecting the digestive tract and lungs, which become clogged with thick, sticky mucus, leading to tissue damage,” explains Dr Aurora. “A key aim of early treatment is to protect the lungs from this damage. However, it is difficult to predict how bad a child’s illness will be and how quickly damage will occur within the lungs. This uncertainty makes it hard to tell which children do, and which children do not, need the most intensive early treatment.”

What is the project trying to achieve?

“We aim to find a way to identify which babies and young children with CF are in danger of doing badly in the future,” explains Dr Aurora. “Those who are at high risk of developing severe disease could then be given more intensive treatment to try and delay the onset of serious lung damage. At the same time, identifying children who are likely to do well with minimal care, could spare them from having unnecessary treatments.”

“We are also testing the reliability of some new equipment, which has recently become available commercially. The equipment has the potential to allow sophisticated tests of children’s lung function, which are currently available only in state-of-the-art research centres, to become widely available in CF clinics around the world.”

Dr Aurora is studying 60 preschool children with CF, who were all diagnosed via the UK’s nationwide newborn screening programme, as well as 30 healthy children.

What are the researchers’ credentials?

Dr Aurora and the project team are members of the London Cystic Fibrosis Collaboration, which has been researching the progress of young children with CF since 1999. They are widely accepted as being world leaders in developing and validating ways to monitor the lung function of babies and children.

Project LeaderDr Paul Aurora MBBS BSc MSc MRCP PhD
Project team
  • Dr Jane Chudleigh PhD MSc PGDip BSc RN
  • Professor Andy Bush MD FRCP FRCPCH
  • Dr A Wade PhD MSc BSc
  • Professor Janet Stocks PhD BSc SRN
LocationPortex Respiratory Unit, Institute of Child Health, University College London
Other locations
  • Department of Respiratory Medicine, Royal Brompton Hospital, London
  • Department of Statistics and Epidemiology, Institute of Child Health, University College London
Grant awarded26 July 2012
Provisional start date1 May 2013
Provisional end date30 November 2016
Grant amount£135,693.00
Grant codeGN2062
AcknowledgementsThis project is supported by a generous grant from The Henry Smith Charity

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References

  1. Cystic Fibrosis Trust, What is Cystic Fibrosis? http://www.cftrust.org.uk/aboutcf/whatiscf/ Website accessed 24 September 2012.
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