Long-Term Efficacy and Safety of the Microsomal Triglyceride Transfer Protein Inhibitor Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

著者: Dirk Blom, Maurizio Averna, Emma A. Meagher, Hendrik du Toit Theron, Cesare R. Sirtori, Robert A. Hegele, Prediman K. Shah, Daniel Gaudet, Claudia Stefanutti, Giovanni Battista Vigna, Dominique Larrey, LeAnne T. Bloedon, Pamela Foulds, Daniel J. Rader, Marina Cuchel - Circulation 2017 被引用: 137

omozygous familial hypercholesterolemia is a genetic disorder characterized by low-density lipoprotein (LDL)-receptor dysfunction, markedly elevated levels of LDL-cholesterol (LDL-C) and premature atherosclerosis. Patients are often poorly responsive to conventional lipid-lowering therapies that upregulate LDL-receptor expression. In a pivotal 78-week openlabel trial, lomitapide, titrated to the maximal tolerable dose, decreased LDL-C by 50% at the end of the efficacy phase (week 26) in patients with homozygous familial hypercholesterolemia.