Treatment of children with familial hypercholesterolemia.

Abstract

Heterozygous familial hypercholesterolemia affects one in every 500 persons and is the most common cause of markedly elevated cholesterol levels in children. Some male patients experience their first coronary event before the age of 30 years. Although dietary measures prevent atherosclerosis in adult populations, the effect of diet on children's lipid levels is limited and compliance is difficult. Trials lasting up to 2 years have shown that statins effectively lower low density lipoprotein (LDL)-cholesterol levels and in one study, restored endothelial dysfunction in children with no clinical adverse effects. To fully assess the effect of drugs on growth and development, especially in prepubertal children, longer trials are required. Gender, family history and LDL-cholesterol level can be used to stratify risk of coronary heart disease. Children that carry very high risk may benefit from starting statins after puberty.