Paediatric FH Register Study
Research type
Research Study
Full title
Estimating appropriate lipid lowering management in the United Kingdom Paediatric Familial Hypercholesterolaemia Register
IRAS ID
324246
Contact name
Angela Shone
Contact email
Sponsor organisation
University of Nottingham
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
People with familial hypercholesterolaemia (FH) have raised cholesterol levels in
their blood from birth. Cholesterol is a type of fat our bodies need but having raised levels over a long time causes heart disease, such as heart attacks. To prevent future heart disease, guidelines recommend starting cholesterol-reducing medication, usually statins, by age 10. However, there remain unanswered questions about what is the right age and cholesterol level at which to start statins to prevent later life heart disease. Considerations are: avoiding side effects, giving best value for money for the health service, and ensuring that treatment is acceptable for children/young people and their families.We will answer these questions by:
1. Measuring health benefits and risks of starting cholesterol-reducing medication in childhood and adolescence (“Clinical effectiveness”). Using a national register of children and young people diagnosed with FH, we will follow their medical care over time, to confirm how well medications work in reducing cholesterol and measure any side effects.2. Exploring the “Acceptability” of this medication. Using questionnaires repeated over time, we will ask how regularly they take their medication. We will also arrange interviews to ask the views of children/young people with FH and their families about how acceptable it is to start medication early, and information families need to support ongoing treatment.
3. Assessing value for money, through calculating the costs and benefits of starting medications at various ages and the savings produced by preventing early heart disease (“Cost-effectiveness”). We will find out the costs of starting cholesterol -reducing medication and how often children/young people are seen by doctors and nurses. Using the study findings and information on how cholesterol affects later risk of heart disease, we will calculate the value for money of starting treatment at different ages and cholesterol levels.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
23/NE/0035
Date of REC Opinion
2 Mar 2023
REC opinion
Favourable Opinion