Evolocumab in Children with Heterozygous Familial Hypercholesterolemia

Heterozygous familial hypercholesterolemia — in contrast to the more rare and more severe homozygous familial hypercholesterolemia — is a common genetic cholesterol disorder affecting approximately 1 in 250 persons and resulting in an increased risk of premature atherosclerotic cardiovascular disease.1 Autopsies and subclinical vascular studies indicate that childhood hypercholesterolemia leads to atherosclerosis. Two randomized, controlled trials have shown that reducing low-density lipoprotein (LDL) cholesterol levels with statins in childhood benefits subclinical vascular measures, delaying or even regressing carotid intima–media thickness,1 and late follow-up of one trial showed fewer events of atherosclerotic cardiovascular disease in treated children than in their affected . . .