Department of Cardiology, Onassis Cardiac Surgery Center, Greece.
Molecular Immunology Laboratory, Onassis Cardiac Surgery Center, Greece.
Eur J Prev Cardiol. 2020 Jan;27(2):157-165. doi: 10.1177/2047487319870007. Epub 2019 Aug 12.
The aim of this study was to evaluate the effect of microsomal triglyceride transfer protein inhibitor (lomitapide) in patients with homozygous familial hypercholesterolaemia.
In 12 homozygous familial hypercholesterolaemia patients treated with lipid-lowering drugs ± biweekly lipoprotein apheresis sessions (nine patients), daily lomitapide was added. The lipid profile (total cholesterol, low-density lipoprotein cholesterol, triglycerides, high-density lipoprotein cholesterol) before and after lomitapide treatment was evaluated. The follow-up period with lomitapide treatment was 3-24 months (13.8 ± 7.9). The median baseline low-density lipoprotein cholesterol level was 900 mg/dl (348-1070), after lipid-lowering drugs therapy was 383.5 mg/dl (214-866) and after lipid-lowering drugs + time-averaged level was 288 mg/dl (183.7-716.6). The addition of lomitapide lowered low-density lipoprotein cholesterol levels further by 56.8% compared to lipid-lowering drugs alone (mean reduction 262, 95% confidence interval (105.5-418.7), = 0.005) and by 54% (mean reduction 182.9, 95% confidence interval (-342 - -23), = 0.031) comparing to lipid-lowering drugs + lipoprotein apheresis (time-averaged level). The time-averaged level of low-density lipoprotein cholesterol in lipid-lowering drugs + lipoprotein apheresis patients compared with lipid-lowering drugs + lomitapide was 54% in favour of lomitapide ( = 0.031).
Treatment with lomitapide in homozygous familial hypercholesterolaemia patients has a beneficial effect with a constant decrease of low-density lipoprotein cholesterol by 57% compared with classical lipid-lowering therapy and by 54% compared with classical lipid-lowering therapy and time-averaged level of low-density lipoprotein cholesterol.
本研究旨在评估微粒体甘油三酯转移蛋白抑制剂（洛美他派）在家族性高胆固醇血症纯合子患者中的疗效。
在 12 名接受降脂药物治疗的家族性高胆固醇血症纯合子患者（9 名患者）中，每周两次进行脂蛋白吸附治疗，在此基础上每日添加洛美他派。评估洛美他派治疗前后的血脂谱（总胆固醇、低密度脂蛋白胆固醇、甘油三酯、高密度脂蛋白胆固醇）。洛美他派治疗的随访时间为 3-24 个月（13.8±7.9）。中位基线低密度脂蛋白胆固醇水平为 900mg/dl（348-1070），降脂药物治疗后为 383.5mg/dl（214-866），降脂药物+平均时间水平为 288mg/dl（183.7-716.6）。与单独使用降脂药物相比，添加洛美他派可使低密度脂蛋白胆固醇水平进一步降低 56.8%（平均降低 262mg/dl，95%置信区间（105.5-418.7）， = 0.005），与降脂药物+脂蛋白吸附治疗相比降低 54%（平均降低 182.9mg/dl，95%置信区间（-342- -23）， = 0.031）。与降脂药物+脂蛋白吸附治疗相比，降脂药物+洛美他派患者的平均时间水平的低密度脂蛋白胆固醇水平有利于洛美他派 54%（ = 0.031）。
与经典降脂治疗相比，洛美他派在家族性高胆固醇血症纯合子患者中的治疗效果有益，可使低密度脂蛋白胆固醇持续降低 57%，与经典降脂治疗和平均时间水平的低密度脂蛋白胆固醇相比，降低 54%。
