医学
家族性高胆固醇血症
PCSK9
内科学
阿利罗库单抗
Evolocumab公司
胆固醇
不利影响
以兹提米比
耐受性
载脂蛋白B
心脏病学
可欣
脂蛋白
临床试验
作者
Krzysztof Chlebus,Barbara Cybulska,Piotr Dobrowolski,Marzena Romanowska-Kocejko,Marta Żarczyńska-Buchowiecka,Natasza Gilis-Malinowska,Aneta Stróżyk,Justyna Borowiec-Wolna,Marcin Pajkowski,Beata Bobrowska,Renata Rajtar-Salwa,Aleksandra Kwapiszewska,Małgorzata Waluś-Miarka,Magdalena Chmara,Rafał Gałąska,Maciej Małecki,Tomasz Zdrojewski,Marcin Gruchała
标识
DOI:10.5603/cj.a2022.0003
摘要
In Poland, treatment with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors has become available free of charge in a therapeutic program. Assessed herein, is the efficacy and safety of alirocumab and evolocumab in patients with heterozygous familial hypercholesterolemia (FH).Data of 55 adult FH patients who participated in the program were analyzed upon meeting the criteria established by the Ministry of Health (low density lipoprotein cholesterol [LDL-C] above 160 mg/dL on max. tolerated statin dose and ezetimib). The efficacy of PCSK9 inhibitors in reducing LDL-C with drug administration every 2 weeks was assessed after 3 months and 1 year of therapy. A safety profile evaluation was performed at each visit. 48 patients completed the 3-month and 21 for the 1-year observation periods (34 patients treated with alirokumab and 14 with evolocumab).The mean concentration of direct-measured LDL-C decreased from the initial level of 215.1 ± 74.5 mg/dL to 75.3 ± 64.1 mg/dL, i.e., by 65 ± 14% following 3 months of treatment. This effect was stable in 1-year observation (77.7 ± 72.8 mg/dL). Adverse effects were flu-like symptoms (13.0%), injection site reactions (11.1%), fatigue (5.6%) and musculoskeletal symptoms (5.6%). Seven patients failed to complete the 3-month treatment period due to side effects or non-compliance, and one patient failed to complete the 1-year treatment due to myalgia.This study confirmed high effectiveness of PCSK9 inhibitors in reducing LDL-C levels in patients with FH. Due to restrictive inclusion criteria with LDL-C threshold level > 160 mg/dL (> 4.1 mmol/L) required for participation in the therapeutic program, a relatively small number of FH patients were eligible for treatment.
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