Abstract 12117: Global Real-World Data on the Use of Lomitapide in Treating Homozygous Familial Hypercholesterolemia: The Lomitapide Observational Worldwide Evaluation Registry (LOWER), Two-Year Data

作者：James A Underberg, C. Cannon, D. Larrey, L. Makris, Charles Schwamlein, H. Phillips, L. Bloeden, D. Blom · 发表于：Circulation · 年份：2016 · DOI：10.1161/circ.134.suppl_1.12117 · 被引用次数：2 · 研究领域：Medicine

Introduction: Lomitapide is a microsomal triglyceride transfer protein inhibitor indicated as adjunctive therapy for adults with homozygous familial hypercholesterolemia (HoFH). LOWER is a global observational registry to prospectively assess long-term, safety and effectiveness of lomitapide in clinical practice. Adult HoFH patients treated with lomitapide in clinical practice are eligible. Results: As of March 1, 2016, 143 patients had been enrolled in the USA, Canada, EU and Taiwan (Table); 139 patients had lomitapide exposure data (median 17.7 months, range 0.3-35.9 months). Globally, median lomitapide dose was 10 mg QD (range 5 mg QOD-40 mg QD, 6-33 months). A ≥ 50% reduction in LDL-C at any time post-baseline was measured in 58% of patients; 62% of patients achieved LDL-C < 100 mg/dL and 37% achieved LDL-C < 70 mg/dL. AEs were experienced by 73% of patients; GI disorders were the most common (45%). Serious AEs occurred in 21 (15%) patients. Thirty-three (24%) patients discontinued lomitapide because of an AE. Events of special interest comprised: major adverse cardiovascular events (11 patients, resulting in 2 deaths); hepatic events (19 patients, 6 discontinuations); GI events (20 patients, 16 discontinuations); oncologic events (2 patients: 1 pancreatic mass [diagnosis unconfirmed], 1 mediastinal B-cell lymphoma); 1 pregnancy; no coagulopathy events. Liver function tests were always performed per the label in 48% of patients. Peak ALT and AST was normal in 35% o...