Children with familial hypercholesterolemia have very high total cholesterol and LDL-cholesterol levels in blood which may result in endothelial dysfunction and increase in carotid intima-media thickness. When untreated in childhood, familial hypercholesterolemia is associated with a premature atherosclerotic cardiovascular disease in adulthood. According to the results of clinical studies in children with familial hypercholesterolemia conducted in the last two decades, as well as statements of American Heart Association (AHA), American Academy of Pediatrics (AAP) and Polish Statement called Stanowisko Ekspertów Lipidowych, the recommendations of treatment were published. In children with familial hypercholesterolemia aged 8 years and more statins are one of the first-line medications, thanks to their hypolipemic and pleiotropic activities and well established position in treatment of adult patients with hypercholesterolemia and cardiovascular disease prevention. This paper provides data on pharmacodynamic and pharmacokinetic properties of statins, as well as overview of clinical studies in children with heterozygous familial hypercholesterolemia, regarding efficacy and safety of statins. The studies have revealed significant lowering of LDL cholesterol level (20-50%) and total cholesterol level (20-30%) by statins used in the lowest recommended dose (compared to placebo) in children aged 8 years and more, in a period from 8 weeks to 24 months. In addition to the fact that statin treatment is efficacious, the safety was also confirmed by the meta-analyses of randomized controlled trials in children. The results showed that statin therapy did not impair growth and sexual development in children. The adverse effects were generally mild and did not differ as compared to placebo. However, it should be emphasized that efficacy and safety assessment of statins is limited to 24 months only. Large long-term clinical studies are needed to establish the long-term safety issues of statins in children.
Keywords: adverse effects; children; clinical trials; familial hypercholesterolemia; statins.