Managing PERT in children with cystic fibrosis.
Research type
Research Study
Full title
A qualitative study to explore the impact of modulator drugs on experiences and attitudes towards dietary therapy including management of pancreatic enzyme replacement therapy (PERT) in children with cystic fibrosis.
IRAS ID
339449
Contact name
Iram Haq
Contact email
Sponsor organisation
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Duration of Study in the UK
2 years, 1 months, 1 days
Research summary
Cystic fibrosis (CF) is an inherited, life-limiting disorder that has no cure and causes severe damage to many organs in the body. People with CF have thick and sticky secretions which block tubes in vital organs such as the lungs, causing lung infection and inflammation.
The majority of children, approximately 85%, also have problems with their pancreas - this is called pancreatic insufficiency (PI). When this happens the pancreas doesn’t produce important chemicals (called enzymes) that are normally needed to digest food. Specifically, fat-containing food cannot be absorbed and digested and children suffer with stomach pain, oily stools, poor nutrition and growth - all of which mpacts on health and quality of life. PI is managed with enzyme medicines called pancreatic enzyme replacement therapy (PERT) which must be taken before every fat-containing meal, drink or snack. For some children, this means taking up to 40 capsules per day. Children can become very poorly if they do not take enough PERT. This leads to malnutrition, hospital admission and missing out on education and activities they enjoy.
In 2020, a new CF treatment was licensed for children in the UK. This ‘modulator’ drug contains a combination of 3 medicines - elexacaftor/tezacaftor/ivacaftor (ETI). ETI has been associated with major improvements in lung health and is often referred to as a ‘miracle drug’.
At present, there is limited information about how it affects PI and PERT management in children with CF. This study will explore this impact in children looked after by the Newcastle paediatric regional CF service which is based at the Great North Children’s Hospital. It will use qualitative interviews and group workshops to investigate opinions with the ultimate aim of developing a tool to support children with CF, their carers and healthcare professionals in managing PERT alongside modulator treatments.
REC name
London - Surrey Research Ethics Committee
REC reference
24/PR/0692
Date of REC Opinion
12 Jul 2024
REC opinion
Further Information Favourable Opinion