Cuchel Marina, Blom Dirk J, Averna Maurizio R
Institute for Translational Medicine and Therapeutics, and Department of Medicine, University of Pennsylvania, 8039 Maloney Building, 3600 Spruce Street, Philadelphia, PA 19104, USA.
University of Cape Town, Cape Town, South Africa; Medical Research Council of South Africa, Cape Heart Group, Cape Town, South Africa.
Atheroscler Suppl. 2014 Sep;15(2):33-45. doi: 10.1016/j.atherosclerosissup.2014.07.005.
The microsomal triglyceride transfer protein (MTP) inhibitor lomitapide is a licenced adjunct to a low-fat diet and other lipid-lowering medication, with or without low-density lipoprotein apheresis, for the treatment of adults with homozygous familial hypercholesterolaemia (HoFH). In a recently published phase 3 study, patients with HoFH received lomitapide in addition to maximally tolerated lipid-lowering therapy. Treatment with lomitapide resulted in a mean approximate 50% reduction in LDL-C levels after 26 weeks compared with baseline levels (p < 0.0001). This decrease in LDL-C was maintained at Weeks 56 and 78 (44% [p < 0.0001] and 38% [p = 0.0001], respectively). This paper offers clinical perspectives based on selected case histories of patients participating in the phase 3 lomitapide study. These cases provide illustrative examples of the efficacy of lomitapide, with or without apheresis, and show that the effective management of adverse effects can enable patients to remain on effective treatment.
微粒体甘油三酯转运蛋白（MTP）抑制剂洛美他派是一种经许可的辅助药物，可与低脂饮食及其他降脂药物联合使用，无论是否进行低密度脂蛋白去除法，用于治疗纯合子家族性高胆固醇血症（HoFH）的成人患者。在最近发表的一项3期研究中，HoFH患者在接受最大耐受降脂治疗的基础上加用了洛美他派。与基线水平相比，接受洛美他派治疗26周后，低密度脂蛋白胆固醇（LDL-C）水平平均降低了约50%（p < 0.0001）。在第56周和第78周时，LDL-C的降低幅度分别维持在44%（p < 0.0001）和38%（p = 0.0001）。本文基于参与洛美他派3期研究的患者的选定病例史提供临床观点。这些病例提供了洛美他派无论是否联合去除法的疗效的说明性示例，并表明对不良反应的有效管理可使患者继续接受有效治疗。
