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.
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%。