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The UK Paediatric Familial Hypercholesterolaemia Register: Statin-related safety and 1-year growth data

Steve E Humphries1, Jackie Cooper1, Peter Dale2

  • 1Centre for Cardiovascular Genetics, Institute for Cardiovascular Science, University College London, London, UK.

Journal of Clinical Lipidology
|December 7, 2017
PubMed

Insights

UK guidelines recommend statin therapy for children with familial hypercholesterolemia (FH) by age 10. This study found statins are safe and effective in FH children, with no impact on growth, though many over age 10 are untreated.

Area of Science:

  • Pediatric Cardiology
  • Clinical Genetics
  • Pharmacology

Background:

  • Familial hypercholesterolemia (FH) is a genetic condition leading to high LDL cholesterol.
  • UK guidelines suggest statin therapy for FH children by age 10.
  • Dietary and lifestyle advice is also recommended for weight management.

Purpose of the Study:

  • To assess liver toxicity and muscle damage markers in statin-treated FH children.
  • To compare obesity prevalence in FH children versus the general UK population.
  • To evaluate growth rate differences between statin-treated and untreated FH children.

Main Methods:

  • Utilized the UK Paediatric Familial Hypercholesterolemia Register.
  • Compared registration and 1-year characteristics using Mann-Whitney U tests.
  • Assessed BMI percentiles against UK growth charts.

Main Results:

  • Statin treatment significantly reduced LDL cholesterol by 31% without safety concerns.
  • No children exhibited elevated liver or muscle damage markers.
  • Obesity rates were lower in FH children (11.1%) compared to UK non-FH children (21.2%).
  • No significant differences in annual growth rates were observed between statin-treated and untreated groups.

Conclusions:

  • Statin therapy in children with FH shows no evidence of safety or growth issues.
  • A significant proportion of FH children over 10 years of age remain untreated.
  • Children with FH in the UK have a lower prevalence of obesity compared to their non-FH peers.
Abstract

Keywords:
Familial hypercholesterolemiaLDL-C levelsObesityOverweightPediatric

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