6566 When should statins be started in children with familial hypercholesterolaemia? developing evidence from a united kingdom paediatric fh register

Objectives Affecting about 1 in every 270 people, heterozygous Familial Hypercholesterolaemia (FH) is one of the commonest inherited disorders, causing raised low density lipoprotein cholesterol (LDL-C) from birth. Whilst recent NICE FH guideline recommendations are to initiate lipid lowering treatment (LLT) by 10 years of age, they recognise this is currently based on limited evidence and does not consider baseline LDL-C.¹ There is no clear guidance paediatric doses of LLT, nor are there clear treatment targets. Parents and specialists are concerned about LLT safety, particularly statin safety, which may affect delayed initiation or medication adherence. Whilst the United Kingdom Paediatric FH Register has provided initial evidence of statin safety,² the objective of this study is to determine the effectiveness of commencing LLT treatment at difference ages and LDL-C thresholds. Methods The Paediatric FH Register, dormant since 2019, was recommenced in 2023 as a 5-year NIHR HTA award. Patients are being recruited using a new electronic consent and data entry system from clinical sites seeing paediatric FH patients. Participants are also invited to a longitudinal follow-up questionnaire study and interviews to assess medication adherence and side-effects, and information needs for families and patients. To answer the main objective, we will use linear mixed-effects models with inverse probability of treatment weighting to balance groups according to confounders. Effectiveness will be measured as absolute, relative and cumulative changes in LDL-C, the latter providing the most optimal measure of cholesterol burden across childhood. Input from a Parent and Carer Advisory Group and a Children and Young Person's Advisory group has been critical to co-designing the study, its ongoing modifications and communication. Results The Paediatric FH Register is now live in 4 hospital trusts. Recruitment is open to any trust across England, Wales and Northern Ireland, with a focus on trusts with larger case numbers, socioeconomically and ethnically diverse populations. Parental consent is running smoothly; obtaining assent from children and young people is challenging. Retrospective and prospective longitudinal data is being collected with a target of 1,300 patients by 2027. Conclusion Through close cooperation with families and specialists, we can reinstate this important disease register. After completion of the current objective, this register will be an important audit tool for paediatric metabolic specialists to monitor the health and wellbeing of children and young people with FH. Within 6 years, we anticipate directly informing recommendations on treatment of paediatric FH in the next NICE FH guidelines. References NICE Clinical guideline 71 (CG71): Familial hypercholesterolaemia: identification and management. NICE 2019. https://www.nice.org.uk/guidance/cg71 Humphries SE, Cooper J, Dale P, Ramaswami U. The UK Paediatric Familial. Hypercholesterolaemia Register: Statin-related safety and 1-year growth data. Journal of Clinical Lipidology. 2018;12(1):25–32. doi:10.1016/j.jacl.2017.11.005