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.

Anti-PCSK9 antibodies for the treatment of heterozygous familial hypercholesterolemia : patient selection and perspectives / A.L. Catapano, A. Pirillo, G.D. Norata. - In: VASCULAR HEALTH AND RISK MANAGEMENT. - ISSN 1176-6344. - 13(2017 Sep), pp. 343-351. [10.2147/VHRM.S130338]

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

A.L. Catapano
Primo
;
G.D. Norata
Ultimo
2017

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.
alirocumab; cholesterol; dyslipidemia; evolocumab; HeFH; endocrinology, diabetes and metabolism; hematology; public health, environmental and occupational health; cardiology and cardiovascular medicine; pharmacology (medical)
Settore BIO/14 - Farmacologia
set-2017
Article (author)
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/527143
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