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https://observatorio.fm.usp.br/handle/OPI/35412
Title: | Long-Term Evolocumab in Patients With Familial Hypercholesterolemia |
Authors: | SANTOS, Raul D.; STEIN, Evan A.; HOVINGH, G. Kees; BLOM, Dirk J.; SORAN, Handrean; WATTS, Gerald F.; LOPEZ, J. Antonio G.; BRAY, Sarah; KURTZ, Christopher E.; HAMER, Andrew W.; RAAL, Frederick J. |
Citation: | JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, v.75, n.6, p.565-574, 2020 |
Abstract: | 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. (C) 2020 The Authors. |
Appears in Collections: | Artigos e Materiais de Revistas Científicas - FM/MCP Artigos e Materiais de Revistas Científicas - HC/InCor Artigos e Materiais de Revistas Científicas - ODS/03 |
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art_SANTOS_LongTerm_Evolocumab_in_Patients_With_Familial_Hypercholesterolemia_2020.PDF | publishedVersion (English) | 871.28 kB | Adobe PDF | View/Open |
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