ABSTRACT
The recommended therapy of hypercholesterolemia in children consists of dietary modification
and bile acid-binding resins. Unfortunately, the lipid-lowering efficacy of bile acid-binding
resins is modest, and moreover, long-term compliance is poor because of side effects.
In contrast, hydroxymethylglutaryl coenzyme A reductase inhibitors (statins) are widely
used in adults and are considered to be the first choice in the treatment of hypercholesterolemia
in that age category. In the last few years, several randomized trials have been conducted
to evaluate the efficacy, safety, and tolerability of statin therapy in both children
and adolescents. In this article, we review statin therapy in hypercholesterolemic
children in terms of efficacy, safety, pharmacokinetics, and psychosocial functioning.
Statins are not only effective in reducing low-density lipoprotein cholesterol levels
in children with familial hypercholesterolemia but also improve endothelial function
and reduce the progressive thickening of the intima media complex of the carotid arteries.
Statins seem safe at the longer term in children in terms of plasma levels of liver
enzymes and liver function, creatine kinase levels, and muscle function, as well as
growth and sexual development. Long-term follow-up studies are needed to assess whether
statin treatment started early in children with familial hypercholesterolemia can
prevent future cardiovascular events.
KEYWORDS
Familial hypercholesterolemia - children - statins - HMG-CoA reductase inhibitors
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Prof.
Dr.
J.J. P Kastelein
Department of Vascular Medicine, Academic Medical Centre
Meibergdreef 9 Room F4-159.2
1105 AZ Amsterdam, The Netherlands