title: Lomitapide for the treatment of hypertriglyceridemia.
abstract: Severe genetic forms of hypertriglyceridemia carry a risk of life-threatening pancreatitis and lack available effective treatments. Lomitapide is a microsomal triglyceride transfer protein inhibitor currently approved for treatment of homozygous familial hypercholesterolemia that may be useful in the management of severe hypertriglyceridemia. Areas covered: Published trials of lomitapide that reported plasma triglyceride response were reviewed, as was a case report of a patient with hypertriglyceridemia who was treated for 13 years with lomitapide. ClinicalTrials.gov was also reviewed for any unpublished results and ongoing trials. Expert opinion: Lomitapide demonstrates effective triglyceride lowering and may be a useful treatment for patients with genetic hypertriglyceridemia and recurrent acute pancreatitis who are refractory to traditional treatment. However, long term hepatic safety may be a concern and direct clinical trial-level data are lacking for this indication.
authors: a Department of Medicine and Robarts Research Institute, Schulich School of Medicine and Dentistry , Western University , London , Ontario , Canada.
authors: Amanda J
authors: AJ
authors: Brahm
authors: a Department of Medicine and Robarts Research Institute, Schulich School of Medicine and Dentistry , Western University , London , Ontario , Canada.
authors: Robert A
authors: RA
authors: Hegele
mesh_terms: D000818:Animals; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D001562:Benzimidazoles / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D015228:Hypertriglyceridemia / Q000188:drug therapy* / Q000503:physiopathology; D010195:Pancreatitis / Q000188:drug therapy; D016896:Treatment Outcome
chemical_list: D000924:Anticholesteremic Agents; C473731:BMS201038; D001562:Benzimidazoles
keywords: Familial chylomicronemia syndrome; hypertriglyceridemia; lipoprotein disorders; microsomal triglyceride transfer protein (MTP); pancreatitis