ISSN 1662-4009 (online)

ESPE Yearbook of Paediatric Endocrinology (2018) 15 12.13 | DOI: 10.1530/ey.15.12.13

ESPEYB15 12 Type 2 Diabetes, Metabolic Syndrome and Lipid Metabolism New treatments (1 abstracts)

12.13 Effect of Rosuvastatin on Carotid Intima-Media Thickness in Children with Heterozygous Familial Hypercholesterolemia: The CHARON Study (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label)

Braamskamp M , Langslet G , McCrindle BW , Cassiman D , Francis GA , Gagne C , Gaudet D , Morrison KM , Wiegman A , Turner T , Miller E , Kusters DM , Raichlen JS , Martin PD , Stein EA , Kastelein JJP & Hutten BA


Department of Vascular Medicine, Department of Pediatrics, and Department of Clinical Epidemiology, Biostatistics and Bioinformatics, Academic Medical Center, Amsterdam, the Netherlands


To read the full abstract: Circulation 2017;136:359-366

The National Heart, Lung and Blood Institute Panel on integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents recommended a universal screening approach that would include one-time testing of all children aged 9-11 years for dyslipidemia. This recommendation has raised controversy regarding the identification and treatment of lipid abnormalities in children. Despite these recommendations, universal screening has not become routine: 68% of pediatricians never/rarely/sometimes screened healthy 9- to 11-year-olds.12 One of the reasons against universal screening in children has been the lack of long-term studies evaluating the effectiveness of screening for dyslipidemia in childhood in delaying or reducing the incidence of cardiovascular-related events.

Several important findings arise from this study. First, a significant difference in carotid intima-media thickness was detected between children with HeFH and unaffected age-matched siblings. This baseline difference was already observed from a younger age; this supports the initiation of treatment even earlier than currently recommended, before the process of atherosclerosis is detectable. Second, treatment with rosuvastatin lowered LDL cholesterol by up to 45%; the therapy was safe and did not influence either growth or puberty. Most importantly, two years of rosuvastatin treatment eliminated the difference in carotid intima media thickness between children with HeFH and their healthy siblings. These findings support the value of early initiation of aggressive LDL-C reduction in children with heterozygous HeFH to reduce cardiovascular risk.13

12. de Ferranti SD, Rodday AM, Parsons SK, Cull WL, O'Connor KG, Daniels SR et al. Cholesterol Screening and Treatment Practices and Preferences: A Survey of United States Pediatricians. The Journal of Pediatrics 2017; 185: 99-105.e2.

13. Vuorio A, Kuoppala J, Kovanen PT, Humphries SE, Tonstad S, Wiegman A et al. Statins for children with familial hypercholesterolemia. Cochrane Database of Systematic Reviews 2017; (7).

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