Improving clinical care for young people with FH
The Paediatric FH Register collects information on the clinical care of children and young people with familial hypercholesterolaemia (FH) from all over the United Kingdom.
It uses medical records from early childhood through adolescence to look at safety and efficacy of cholesterol-reducing treatments, health, and growth of young people with FH. Medical records of consented paediatric patients with FH are held in a secure centralised database at the University of Nottingham. Participants’ identity is fully protected.
Aims of the Register
The Paediatric FH Register has NIHR funding from 2023 to 2028 to strengthen current evidence on when to start cholesterol-reducing medications in young people with FH.
Key questions are:
- At what age and cholesterol concentration should treatment be started?
- Can current NICE guidance to start by 10 years of age be more specific?
- Are there potential side effects and how can they be avoided?
- What information and support do young people and families need for starting and continuing treatment?
- What is the value for money for the health service based on the costs and benefits of starting medications at various ages and the savings produced by preventing early heart disease?
Although FH is a relatively common genetic condition where people typically have raised cholesterol from birth, it is currently widely underdiagnosed. With the expansion of genetic testing for FH, more children and young people should be diagnosed, further strengthening the need for clear treatment guidelines.
Joining the Register
The Paediatric FH Register can be joined through participating clinical centres with lipid clinics for paediatric patients with FH. For children with FH younger than 16 years, a parent or guardian must give their permission to take part (also called giving consent) alongside the child’s agreement (also called giving assent) if they are between age 5-15 years. From age 16 years onwards, young people can directly consent to join the Register.
After consenting, clinical teams enrol participants on the Register and enter information from medical notes. To look at clinical care and health over time, they enter information following each FH clinic visit. There is nothing that children need to do to take part beyond their usual clinical care. There are no changes to care or treatment and no additional blood tests or procedures because of taking part.
Details are available below (contact us for information in other languages using our translation service):
|
Participant information sheets Links below provide information for: |
Consent and assent forms Links below are for consent (giving permission) and assent (giving agreement) forms |
| Parents and guardians of children with FH | Parents and guardians of children with FH (consent form) |
| Young people with FH - age 16 years and older | Young people with FH - age 16 years and older (consent form) |
| Young people with FH - age 11-15 years | Young people with FH - age 11-15 years (assent form) |
| Young people with FH - age 5-10 years (asset form) |
Who organises and funds the Register?
From 2023 the Register is organised and hosted by the University of Nottingham with 5 years of funding from the National Institute for Health and Care Research Health Technology Assessment (NIHR HTA). The Register is led by Co-Leads Professor Nadeem Qureshi and Professor Laila Tata and Co-Clinical Leads Uma Ramaswami and Lorraine Priestly-Barnham. They work with a wider of team of health professionals, academics, and patient representatives on a research project to improve clinical care for young people with FH. The Register is supported and guided by our Children and Young Persons Advisory Group (CYPAG) and Parent and Carer Advisory Group (PCAG).
The Paediatric FH Register was set up in 2012 by Professor Steve Humphries and Dr Uma Ramaswami and has been hosted previously by the Royal College of Physicians and HEART UK.
The Paediatric FH Steering Group:
- oversees and provides expert direction for the Register
- ensures it is useful for clinicians registering and entering clinical information
- ensures it is valuable for patients and families
- identifies directions for sustainability and development of the Register
- considers approval of applications for use of the anonymised data by researchers where there is clear benefit to patients and society
Members:
| Name | Organisation | Position |
| Steve Humphries (Current Chair of Steering Group) |
University College London | Emeritus British Heart Foundation Professor of Cardiovascular Genetics |
| Nadeem Qureshi | University of Nottingham | Professor of Primary Care |
| Laila Tata | University of Nottingham | Professor of Epidemiology |
| Uma Ramaswami | Royal Free Hospital | Consultant Metabolic Paediatrician British Inherited Metabolic Disease Group Representative |
| Jules Payne | HEART UK | Chief Executive |
| Lorraine Priestley-Barnham | Royal Brompton Hospital Harefield | Paediatric FH Nurse |
| Andrew Neil | University of Oxford | Emeritus Professor of Clinical Epidemiology Adult Lipidologist |
| Kate Haralambos | Wales FH Service | FH Network Manager PASS database Project Co-Ordinator |
| Peter Dale | Aneurin Bevan University Health Board NHS Wales for Gwent |
Paediatrician, Royal College of Paediatrics and Child Health representative |
| Mark Fisher | Independent | Patient Representative |
| Andrew Morris | Manchester University NHS Trust | Paediatrician, Royal College of Paediatrics and Child Health representative |
Involving young people with FH and their families
Input from patient and family representatives has shaped the development of the Paediatric FH Register since its foundation. This has included co-design of Register recruitment materials such as participant information sheets, and identifying the top priority questions young people and their families want answering, such as the safety of FH treatments in childhood and into adulthood.
Starting in 2023, patients and families are working with the research team to develop the information and support young people and families need for starting and continuing treatment, addressing any concerns they may have. Their involvement in is accordance with NHS England’s guidance on patient and public involvement in research through 2 advisory groups:
Children and Young Persons Advisory Group (CYPAG)
The Paediatric FH CYPAG is a group of young people (mostly aged 12 to 18) who have a diagnosis of FH and those without FH who are interested in becoming involved in shaping research. They meet periodically to review and feedback on documents and design communication outputs from the Register research.
We are currently recruiting for the CYPAG - If you or a young person you know would be interested in shaping FH care for the future, you can find out more information about joining the CYPAG here.
Parent and Carer Advisory Group (PCAG)
The Paediatric FH PCAG is chaired by Mark Fisher who has long-standing involvement in the Register. He is a father, son and grandson of FH patients, and was one of the first users of statins, being diagnosed at the age of 18. Members of the PCAG have children who have been diagnosed with FH, generally still in paediatric care. The group meet periodically to review and feedback on documents and design communication outputs from the Register research.
Contact Information
Please email us at paedsfh@nottingham.ac.uk for further information
You can also request further information using this form or using the QR code below.
