Long-Term Evolocumab in Patients With Familial Hypercholesterolemia.

Autor:	Santos RD; Lipid Clinic Heart Institute (InCor), University of São Paulo Medical School Hospital and Hospital Israelita Albert Einstein, São Paulo, Brazil. Electronic address: raul.santos@incor.usp.br., Stein EA; Department of Medicine, Cardiology, University of Chicago Pritzker School of Medicine, Chicago, Illinois., Hovingh GK; Department of Vascular Medicine, Amsterdam UMC, Location AMC, Amsterdam, the Netherlands., Blom DJ; Division of Lipidology and Hatter Institute for Cardiovascular Research in Africa, Department of Medicine, University of Cape Town, Cape Town, South Africa., Soran H; Cardiovascular Trials Unit, Manchester University Hospital NHS Foundation Trust, Manchester, United Kingdom., Watts GF; School of Medicine, Faculty of Health and Medical Sciences, University of Western Australia, Perth, Western Australia, Australia; Lipid Disorders Clinic, Department of Cardiology, Royal Perth Hospital, Perth, Western Australia, Australia., López JAG; Amgen, Thousand Oaks, California., Bray S; Amgen, Thousand Oaks, California., Kurtz CE; Amgen, Thousand Oaks, California., Hamer AW; Amgen, Thousand Oaks, California., Raal FJ; Carbohydrate and Lipid Metabolism Research Unit, Faculty of Health Sciences, University of Witwatersrand, Johannesburg, South Africa.
Jazyk:	angličtina
Zdroj:	Journal of the American College of Cardiology [J Am Coll Cardiol] 2020 Feb 18; Vol. 75 (6), pp. 565-574.
DOI:	10.1016/j.jacc.2019.12.020
Abstrakt:	Background: Proprotein convertase subtilisin/kexin type 9 inhibitor therapy is a treatment option for patients with familial hypercholesterolemia (FH) who are unable to reach low-density lipoprotein cholesterol (LDL-C) goals. Objectives: The aim of this study was to provide long-term safety and efficacy data for evolocumab in patients with homozygous FH (HoFH) and severe heterozygous FH (HeFH). Methods: In this open-label, single-arm study, patients with HoFH or severe HeFH ≥12 years of age and on stable lipid-lowering therapy began subcutaneous evolocumab 420 mg monthly or 420 mg every 2 weeks if on lipoprotein apheresis. After 12 weeks, those not on apheresis could be up-titrated to 420 mg every 2 weeks. The primary endpoint was the incidence of treatment-emergent adverse events; secondary endpoints were changes in LDL-C and other lipids. Results: In total, 300 patients (106 with HoFH, including 14 <18 years of age at enrollment) received evolocumab for a median of 4.1 years. Adverse events occurred in 89.3% of patients, the most common of which were nasopharyngitis, influenza, upper respiratory tract infection, and headache. Mean change in LDL-C from baseline to week 12 was -21.2% (-59.8 mg/dl) in patients with HoFH and -54.9% (-104.4 mg/dl) in those with severe HeFH and was sustained over time. Of 48 patients with HoFH who were up-titrated, mean change in LDL-C improved from -19.6% at week 12 to -29.7% after 12 weeks of 420 mg every 2 weeks. The adjudicated cardiovascular event rate was 2.7% per year. Of 61 patients receiving apheresis at enrollment, 16 discontinued apheresis. Conclusions: Evolocumab was well tolerated and effectively reduced plasma LDL-C levels in patients with HoFH and severe HeFH over a median of 4.1 years. (Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.)
Databáze:	MEDLINE
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