title: Severe hypertriglyceridemia with pancreatitis: thirteen years' treatment with lomitapide.
abstract: OBJECTIVE
  Recurrent pancreatitis is a potentially fatal complication of severe hypertriglyceridemia. Genetic defects and lifestyle risk factors may render this condition unresponsive to current treatments.
  
  
  METHODS
  We report this first case of long-term management of intractable near-fatal recurrent pancreatitis secondary to severe hypertriglyceridemia by a novel use of lomitapide, an inhibitor of microsomal triglyceride transfer protein, recently approved for treatment of familial homozygous hypercholesterolemia. The patient had been hospitalized many times for pancreatitis since age 15 years. Her serum triglyceride level averaged 3900 mg/dL while she received therapy with approved lipid drugs. She is homozygous for a coding mutation (P234L) in lipoprotein lipase, leaving her unable to metabolize triglycerides in chylomicrons and very low density lipoproteins (VLDL). Lomitapide reduces the secretion of chylomicrons and VLDL. Lomitapide, which was started when she was 44 years old after near-fatal pancreatitis, lowered her fasting triglyceride level from greater than 3000 mg/dL to a mean (SD) of 903 (870) mg/dL while she received 30 mg/d and to 524 (265) mg/dL while she received 40 mg/d; eliminated chronic abdominal pain; and prevented pancreatitis. However, fatty liver, present before treatment, progressed to steatohepatitis and fibrosis after 12 to 13 years.
  
  
  CONCLUSIONS
  Lomitapide prevented pancreatitis in severe intractable hypertriglyceridemia but at a potential long-term cost of hepatotoxicity.
authors.affiliation: Department of Nutrition, Harvard School of Public Health, Boston, Massachusetts2Channing Division of Network Medicine, Brigham and Women's Hospital, Boston, Massachusetts3Harvard Medical School, Boston, Massachusetts.
authors.forename: Frank M
authors.initials: FM
authors.lastname: Sacks
authors.affiliation: Harvard Vanguard Medical Associates, Boston, Massachusetts.
authors.forename: Maxine
authors.initials: M
authors.lastname: Stanesa
authors.affiliation: Robarts Research Institute, London, Ontario, Canada.
authors.forename: Robert A
authors.initials: RA
authors.lastname: Hegele
mesh_terms: D000328:Adult; D000924:Anticholesteremic Agents / Q000627:therapeutic use*; D001562:Benzimidazoles / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D015228:Hypertriglyceridemia / Q000150:complications* / Q000188:drug therapy*; D050500:Pancreatitis, Chronic / Q000188:drug therapy* / Q000209:etiology; D011247:Pregnancy; D012008:Recurrence; D016896:Treatment Outcome
chemical_list: D000924:Anticholesteremic Agents; C473731:BMS201038; D001562:Benzimidazoles