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

Authors Catapano AL, Pirillo A, Norata GD

Received 8 June 2017

Accepted for publication 6 July 2017

Published 4 September 2017 Volume 2017:13 Pages 343—351


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Dr Naga Venkata Amarnath Kommuri

Alberico Luigi Catapano,1,2 Angela Pirillo,1,2 Giuseppe Danilo Norata1,3,4

1Department of Pharmacological and Biomolecular Sciences, Università degli Studi di Milano, 2IRCCS Multimedica Hospital, Sesto San Giovanni, 3Center for the Study of Atherosclerosis, E. Bassini Hospital, Cinisello Balsamo, Milan, Italy; 4School of Biomedical Sciences, Curtin Health Innovation Research Institute, Curtin University, Perth, WA, Australia

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, dyslipidemia, cholesterol, alirocumab, evolocumab

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