A major new study led by RCSI University of Medicine and Health Sciences and Children's Health Ireland (CHI) has been awarded funding of €5.6M to work with babies 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 often have more treatment options and potentially better outcomes than those born with the disease in previous decades. But there is still much we need to understand about cystic fibrosis in babies and children in this new era of care.
Cystic fibrosis (CF) is an inherited disease that 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 Children's Health Ireland and will be carried out at 13 paediatric (children's) 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 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, we are really excited about this new study," said RCSI's Paul McNally, who emphasised the importance of working closely with the CF community.
"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," he said. "Parents of children with CF and other representatives from the CF community will be part of the study group and help us to ensure that we stay focused on our goals".
Paul McNally will lead ENHANCE with Jane Davies, a 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.
"ENHANCE will be a really important study for the whole CF community," said Professor Davies. "By including children from underrepresented groups and those who cannot access some CF treatments we will get a broad and comprehensive understanding of this new era of CF in childhood. While we have made much progress as a community, more remains to be done, and it is through studies like ENHANCE that we can understand and focus on the next challenges faced by children with CF and their families."
Funding for the study was secured through an internationally reviewed competitive grant application process from the Cystic Fibrosis Foundation in the US ($5.9M), with a separate grant for study equipment funded by Cystic Fibrosis Trust in the UK (£100,000).
Dr Lucy Allen, Director of Research and Healthcare Data at Cystic Fibrosis Trust, said: "Cystic fibrosis is a complex condition affecting many parts of the body. Although significant progress has been made in the treatment of cystic fibrosis in recent decades, we know that understanding the long term effects of CFTR modulator medicines is a top research priority for the CF community. Through our funding of the ENHANCE project we are driving significant research that will address this priority and ultimately support people with cystic fibrosis to live longer, healthier lives."
Cystic Fibrosis Ireland strongly supports the ENHANCE Study led by RCSI and CHI, including its focus on children with CF. This study is particularly important in the new era of innovative CF drug therapies and improved care. With many unknowns and uncertainties remaining, this research is both very welcome and timely from a patient perspective"
Philip Watt, CEO, Cystic Fibrosis Ireland
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