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Heterozygous familial hypercholesterolaemia in childhood: Cardiovascular risk prevention

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  • Published:
Journal of Inherited Metabolic Disease

Summary

Children with familial hypercholesterolaemia (FH) have severely increased low-density lipoprotein cholesterol (LDL-C) levels that strongly predispose to premature cardiovascular disease (CVD) later in life. Early identification makes it possible to start lipid-lowering therapy at young age to prevent CVD. The atherosclerotic process can be inhibited by potent lipid-lowering therapy. The cornerstone of lipid-lowering therapy is a healthy lifestyle, but most of the time this is insufficient to reach adequate LDL-C goals. Subsequently, pharmacological therapy is initiated with increasing frequency. In the past decade numerous studies have assessed the efficacy and safety of statins in children with FH. Those studies demonstrate that statins are well tolerated, safe and effective. Therefore, these agents have a pivotal role in the treatment of children with FH.

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Abbreviations

AAP:

American Academy of Pediatrics

apoB:

apolipoprotein B100

cIMT:

carotid intima media thickness

CVD:

cardiovascular disease

FDB:

familial defective apolipoprotein B

FH:

familial hypercholesterolaemia

FMD:

flow-mediated dilatation

HDL-C:

high-density lipoprotein cholesterol

LDL-C:

low-density lipoprotein cholesterol

LDL-R:

low-density lipoprotein receptor

PCSK9:

proprotein convertase subtilisin/kexin 9

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Authors and Affiliations

  1. Department of Vascular Medicine, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands

    A. van der Graaf & J. J. P. Kastelein

  2. Department of Paediatrics, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands

    A. Wiegman

  3. Department of Paediatrics, Academic Medical Centre, Meibergdreef 9, 1105 AZ Amsterdam, Amsterdam, The Netherlands

    A. Wiegman

Authors
  1. A. van der Graaf
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  2. J. J. P. Kastelein
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  3. A. Wiegman
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Correspondence to A. Wiegman.

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Communicating editor: Robert Steiner

Competing Interests: None declared

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van der Graaf, A., Kastelein, J.J.P. & Wiegman, A. Heterozygous familial hypercholesterolaemia in childhood: Cardiovascular risk prevention. J Inherit Metab Dis 32, 699–705 (2009). https://doi.org/10.1007/s10545-009-1165-1

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  • DOI: https://doi.org/10.1007/s10545-009-1165-1

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