Prakt. Lékáren. 2018; 14(1e) [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 hypercholesterolaemiais caused by mutations in genes encoding key proteins involved in low density lipoprotein cholesterol (LDL-C) metabolism,which leads to reduced cellular uptake of LDL cholesterol, increased plasma LDL cholesterol concentrations, and premature development ofcardiovascular disease. Despite the availability of reliable diagnostic criteria (high LDL-C levels, family history or premature CHD and hypercholesterolemia,cerebral/peripheral vascular disease, and the presence of 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 preventearly-onset heart disease in individuals with FH, because of the high baseline levels of LDL-C, the achievement of target LDL-C levels remainsa challenge. In recent years, a number of novel therapies to lower LDL-C levels in FH have been developed, including the monoclonal antibodiesagainst serine protease proprotein convertase subtilisin/kexin type 9 (PCSK9), alirocumab and evolocumab, which have the potentialto reduce LDL-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: March 24, 2018  Show citation