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Abstract：
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), p = 0.005) and by 54% (mean reduction 182.9, 95% confidence interval (-342 - -23), p = 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 (p = 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.
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), p = 0.005) and by 54% (mean reduction 182.9, 95% confidence interval (-342 - -23), p = 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 (p = 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.
摘要：
这项研究的目的是评估微粒体甘油三酸酯转移蛋白抑制剂（lomitapide）在纯合子家族性高胆固醇血症患者中的作用。
在12名接受降脂药±双周脂蛋白单采治疗的纯合子家族性高胆固醇血症患者（9名患者）中，每天加入洛米他必特。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）在lomitapide治疗前后进行了评估。lomitapide治疗的随访时间为3-24个月（13.8±7.9）。基线低密度脂蛋白胆固醇水平中位数为900mg/dl（348-1070），降脂药治疗后为383.5mg/dl（214-866），降脂药治疗后时间平均水平为288mg/dl（183.7-716.6）。与单独使用降脂药相比，添加洛米他必特可使低密度脂蛋白胆固醇水平进一步降低56.8％（平均降低262，95％置信区间（105.5-418.7），p=0.005)和54%(平均减少182.9，95%置信区间(-342--23)，p=0.031）与降脂药物+脂蛋白单采术（时间平均水平）相比。降脂药+脂蛋白单采患者低密度脂蛋白胆固醇的时间平均水平与降脂药+lomitapide相比为54％，有利于lomitapide（p=0.031）。
在纯合子家族性高胆固醇血症患者中，洛米他皮特治疗具有有益作用，与经典降脂治疗相比，低密度脂蛋白胆固醇持续降低57％，与经典降脂治疗相比，低密度脂蛋白胆固醇持续降低54％。
在12名接受降脂药±双周脂蛋白单采治疗的纯合子家族性高胆固醇血症患者（9名患者）中，每天加入洛米他必特。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）在lomitapide治疗前后进行了评估。lomitapide治疗的随访时间为3-24个月（13.8±7.9）。基线低密度脂蛋白胆固醇水平中位数为900mg/dl（348-1070），降脂药治疗后为383.5mg/dl（214-866），降脂药治疗后时间平均水平为288mg/dl（183.7-716.6）。与单独使用降脂药相比，添加洛米他必特可使低密度脂蛋白胆固醇水平进一步降低56.8％（平均降低262，95％置信区间（105.5-418.7），p=0.005)和54%(平均减少182.9，95%置信区间(-342--23)，p=0.031）与降脂药物+脂蛋白单采术（时间平均水平）相比。降脂药+脂蛋白单采患者低密度脂蛋白胆固醇的时间平均水平与降脂药+lomitapide相比为54％，有利于lomitapide（p=0.031）。
在纯合子家族性高胆固醇血症患者中，洛米他皮特治疗具有有益作用，与经典降脂治疗相比，低密度脂蛋白胆固醇持续降低57％，与经典降脂治疗相比，低密度脂蛋白胆固醇持续降低54％。
