Heterozygous familial hypercholesterolaemia in childhood: cardiovascular risk prevention.

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.