'New era’ of cystic fibrosis care

A new study by Children’s Health Ireland (CHI) and RCSI has been awarded funding of €5.6 million to work with infants and children in Ireland and the UK over the next five years to build a better understanding of cystic fibrosis.

Children born in the 2020s with cystic fibrosis (CF) have more treatment options and potentially better outcomes than those born with the disease in previous decades. However, there is still much to understand about cystic fibrosis in infants and children.

CF mainly affects the lungs and digestive system. Ireland has the highest incidence of the condition in the world: approximately 1,400 children and adults in Ireland live with the condition and typically more than 30 new cases of CF are diagnosed here each year, usually in the first few weeks of life. 

The ENHANCE study – Establishing Natural History in an Advanced New CF Care Era – will be led by the CF research team at CHI and will be carried out at 13 paediatric CF clinic sites in Ireland and the UK. 

“The ways that we can help children with CF have evolved considerably over the last 15 years, notably with new medicines becoming available, and many children will start these treatments from very early in life,” says Prof Paul McNally, associate professor of paediatrics at RCSI and consultant in respiratory medicine at CHI. 

“This means the outlook for children with CF is considerably better than it was 15 years ago, but there is much about this that we don’t yet understand.”

The ENHANCE study will monitor how CF complications develop in small children and how different groups of children with CF develop features of the disease. The research will also compare children of a similar age with and without CF.

“There is so much to learn about the changes that are happening in children with CF, said Prof McNally. “We spent a long time working with parents of children with CF in Ireland and the UK to understand how best to design the study as we wanted to ensure we are concentrating on the things that are most relevant to children with CF and their parents in this new era.”

Prof McNally will lead ENHANCE with Prof Jane Davies, consultant in paediatric respiratory medicine at Royal Brompton Hospital, part of Guy’s & St Thomas’ NHS Foundation Trust and a professor at the National Heart and Lung Institute, Imperial College London.