Interv Akut Kardiol. 2017;16(4):158-164 [Klin Farmakol Farm. 2017;31(3):28-34]

Evolocumab for the treatment of familial hypercholesterolemia

Vladimír Bláha

III. interní gerontometabolická klinika, Lékařská fakulta UK a Fakultní nemocnice Hradec Králové

Familial hypercholesterolaemia (FH) is the most common dominantly inherited monogenic disorder in human beings worldwide.Familial hypercholesterolaemia is caused by mutations in genes encoding key proteins involved in low density lipoproteincholesterol (LDL-C) metabolism, which leads to reduced cellular uptake of LDL cholesterol, increased plasma LDL cholesterolconcentrations, and premature development of cardiovascular disease. Despite the availability of reliable diagnostic criteria (highLDL-C levels, family history or premature CHD and hypercholesterolemia, cerebral/peripheral vascular disease, and the presenceof tendon xanthomata or presence of arcus cornealis before age of 45), FH is underdiagnosed and undertreated worldwide.Moreover, while there are effective treatments available to decrease LDL-C and prevent early-onset heart disease in individualswith FH, because of the high baseline levels of LDL-C, the achievement of target LDL-C levels remains a challenge. In recent years,a number of novel therapies to lower LDL-C levels in FH have been developed, including the monoclonal antibodies against serineprotease proprotein convertase subtilisin/kexin type 9 (PCSK9), alirocumab and evolocumab, which have the potential to reduceLDL-C by an additional 50–60 % when prescribed in combination with standard lipid-lowering drugs. This review summarizes thechallenges in clinical management of subjects with FH, with a focus on treatment by evolocumab.

Keywords: familial hypercholesterolemia, hypolipidemic treatment, cardiovascular risk, atherosclerosis, evolocumab

Published: December 1, 2017  Show citation