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Review
. 2017 Sep 4:13:343-351.
doi: 10.2147/VHRM.S130338. eCollection 2017.

Anti-PCSK9 antibodies for the treatment of heterozygous familial hypercholesterolemia: patient selection and perspectives

Alberico Luigi Catapano  1   2 , Angela Pirillo  1   2 , Giuseppe Danilo Norata  1   3   4
Affiliations
Review

Anti-PCSK9 antibodies for the treatment of heterozygous familial hypercholesterolemia: patient selection and perspectives

Alberico Luigi Catapano et al. Vasc Health Risk Manag. .

Abstract

Heterozygous familial hypercholesterolemia (FH) is a genetic disorder characterized by high low-density lipoprotein cholesterol levels from birth, which exposes the arteries to high levels of atherogenic lipoproteins lifelong and results in a significantly increased risk of premature cardiovascular events. The diagnosis of FH, followed by an appropriate and early treatment is critical to reduce the cardiovascular burden in this population. Phase I-III clinical trials showed the benefit of proprotein convertase subtilisin kexin 9 inhibitors, both alirocumab and evolocumab, in these patients with an average low-density lipoprotein cholesterol reduction ranging from -40% to -60%. The aim of this review is to address the unmet needs in cholesterol management, elucidate the biology and the clinical benefit of proprotein convertase subtilisin kexin 9 inhibition and finally discuss the open gaps and future directions in the treatment of patients with heterozygous FH.

Keywords: HeFH; alirocumab; cholesterol; dyslipidemia; evolocumab.

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Conflict of interest statement

Disclosure The authors have received research funding, and/or honoraria for advisory boards, consultancy or speaker bureau from Aegerion (ALC, GDN), Amgen (ALC, GDN), AstraZeneca (ALC), Eli Lilly (ALC), Genzyme (ALC), Mediolanum (ALC), Merck or MSD (ALC), Pfizer (ALC, GDN), Recordati (ALC, GDN), Rottapharm (ALC), Sanofi-Regeneron (ALC, GDN), Sigma-Tau (ALC). AP reports no conflicts of interest in this work.

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