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- Title
Anti-PCSK9 antibodies for the treatment of heterozygous familial hypercholesterolemia: patient selection and perspectives.
- Authors
Catapano, Alberico Luigi; Pirillo, Angela; Norata, Giuseppe Danilo
- 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.
- Subjects
HYPERCHOLESTEREMIA treatment; GENETIC disorders; DYSLIPIDEMIA; CHOLESTEROL; LIPOPROTEINS; THERAPEUTIC use of monoclonal antibodies; THERAPEUTIC use of protease inhibitors; ANIMAL experimentation; ANTILIPEMIC agents; CLINICAL trials; DISEASE susceptibility; LOW density lipoproteins; MONOCLONAL antibodies; PHENOTYPES; EVIDENCE-based medicine; PROTEASE inhibitors; TREATMENT effectiveness; GENETIC carriers; FAMILIAL hypercholesterolemia
- Publication
Vascular Health & Risk Management, 2017, Vol 13, p343
- ISSN
1176-6344
- Publication type
journal article
- DOI
10.2147/VHRM.S130338