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真实世界环境中的前蛋白转化酶枯草溶菌素9(PCSK-9)抑制剂以及阿利西尤单抗与依洛尤单抗在杂合子家族性高胆固醇血症(FH)患者中的比较

PCSK-9 Inhibitors in a Real-World Setting and a Comparison Between Alirocumab and Evolocumab in Heterozygous FH Patients.

作者信息

Ceballos-Macías José Juan, Lara-Sánchez Carolina, Flores-Real Jorge, Aguilar-Salinas Carlos Alberto, Ortega-Gutiérrez Guillermo, Vargas-Sánchez Joel, Madriz-Prado Ramón, Derosa Giuseppe, Rodríguez-Benítez Hazel, Baltazar-Romero Ricardo, Lopez-Mezquita Dante José

机构信息

Servicio de Endocrinología, Unidad de Especialidades Médicas de la Secretaría de la Defensa Nacional, Ciudad de México, Mexico.

Servicio Endocrinología del Hospital Central Militar, Ciudad de México, Mexico.

出版信息

J Endocr Soc. 2020 Nov 18;5(1):bvaa180. doi: 10.1210/jendso/bvaa180. eCollection 2021 Jan 1.

Abstract

A real-world setting study of familial hypercholesterolemia (FH) patients who received Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors in a specialized referral center in Mexico City. Ten patients between the ages of 18 and 70 years, with a diagnosis of FH according to Dutch Lipid Clinic Network (DLCN) criteria, with failure to achieve their Low-density lipoprotein Cholesterol (LDL-C) goals, and with standard therapy between 2016 and 2017 enrolled in a simple randomization in which a group of 5 participants received alirocumab (75 mg every 2 weeks) and the remaining 5 patients received evolocumab (140 mg every 2 weeks). Comparative analysis was made, analyzing the means of LDL at baseline at 4, 6, and 12 weeks. The evolocumab group had an average initial LDL-C of 277 mg/dL, which, after 12 weeks of treatment, was significantly reduced to 116 mg/dL;  = 0.04 (95% confidence interval [CI]: 11.5-310.9). The alirocumab group with a mean initial LDL-C of 229 mg/dL showed a reduction of LDL-C levels at 12 weeks of treatment to 80 mg/dL;  = 0.008 (95% CI: 63.8-233.7). In conclusion, PCSK9 inhibitors are an excellent treatment option in patients with FH who do not reach their LDL-C goals with standard therapy or due to intolerance to the standard therapy. There is no difference in the lipid-lowering effect between both PSCK9 inhibitors.

摘要

在墨西哥城一家专业转诊中心对接受前蛋白转化酶枯草溶菌素9型(PCSK9)抑制剂治疗的家族性高胆固醇血症(FH)患者进行的一项真实世界研究。10名年龄在18至70岁之间的患者,根据荷兰脂质诊所网络(DLCN)标准诊断为FH,未达到其低密度脂蛋白胆固醇(LDL-C)目标,且在2016年至2017年接受标准治疗,纳入一项简单随机分组,其中一组5名参与者接受阿利西尤单抗(每2周75毫克),其余5名患者接受依洛尤单抗(每2周140毫克)。进行了比较分析,分析了基线时、4周、6周和12周时LDL的均值。依洛尤单抗组初始LDL-C平均为277毫克/分升,治疗12周后显著降至116毫克/分升;P = 0.04(95%置信区间[CI]:11.5 - 310.9)。阿利西尤单抗组初始LDL-C均值为229毫克/分升,治疗12周时LDL-C水平降至80毫克/分升;P = 0.008(95% CI:63.8 - 233.7)。总之,对于那些通过标准治疗未达到LDL-C目标或因对标准治疗不耐受的FH患者,PCSK9抑制剂是一种出色的治疗选择。两种PCSK9抑制剂之间的降脂效果没有差异。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/83ad/7747364/e212bc6a0864/bvaa180_fig1.jpg

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