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

Alberico Luigi Catapano, Angela Pirillo, Giuseppe Danilo Norata

Research output: Contribution to journalReview article

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.

Original languageEnglish
Pages (from-to)343-351
Number of pages9
JournalVascular Health and Risk Management
Volume13
DOIs
Publication statusPublished - Sep 4 2017
Externally publishedYes

Fingerprint

Hyperlipoproteinemia Type II
Proprotein Convertases
Patient Selection
Subtilisin
Anti-Idiotypic Antibodies
LDL Cholesterol
Phase III Clinical Trials
Clinical Trials, Phase I
Inborn Genetic Diseases
HDL Cholesterol
Lipoproteins
Therapeutics
Arteries
Cholesterol
Parturition
Population

Keywords

  • Alirocumab
  • Cholesterol
  • Dyslipidemia
  • Evolocumab
  • HeFH

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Hematology
  • Public Health, Environmental and Occupational Health
  • Cardiology and Cardiovascular Medicine
  • Pharmacology (medical)

Cite this

Anti-PCSK9 antibodies for the treatment of heterozygous familial hypercholesterolemia : Patient selection and perspectives. / Catapano, Alberico Luigi; Pirillo, Angela; Norata, Giuseppe Danilo.

In: Vascular Health and Risk Management, Vol. 13, 04.09.2017, p. 343-351.

Research output: Contribution to journalReview article

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