title: LOWER, a registry of lomitapide-treated patients with homozygous familial hypercholesterolemia: Rationale and design.
abstract: BACKGROUND
  Lomitapide is an orally active selective inhibitor of microsomal triglyceride transfer protein approved as adjunctive therapy for homozygous familial hypercholesterolemia (HoFH). The Lomitapide Observational Worldwide Evaluation Registry (LOWER) is a global, long-term, prospective, observational treatment registry established as a regulatory requirement.
  
  
  OBJECTIVE
  LOWER will evaluate the long-term safety and effectiveness of lomitapide in clinical practice. The objectives include evaluation of the occurrence of events of special interest and assessment of the long-term effectiveness of lomitapide in maintaining reduced serum lipid levels.
  
  
  METHODS
  LOWER is a noninterventional study open to eligible lomitapide-treated patients. At least 300 patients will be enrolled and followed for at least 10 years. Data will be collected in conjunction with usual care visits and analyzed annually. LOWER includes a cardiovascular imaging substudy; an independent pregnancy exposure registry is also open.
  
  
  RESULTS
  Events of special interest include hepatic abnormalities, gastrointestinal events, certain gastrointestinal tumors, major adverse cardiovascular events, and events associated with coagulopathy. Data will be collected on demographics, diagnosis, patient history, lomitapide dosing, concomitant treatment, lipid profile, and other laboratory results.
  
  
  CONCLUSIONS
  LOWER will assess the long-term safety, efficacy, and patterns of use of lomitapide, increase understanding of the benefit-to-risk profile, and add to knowledge of HoFH.
authors.affiliation: Division of Lipidology, Department of Medicine, University of Cape Town, Cape Town, South Africa. Electronic address: dirk.blom@uct.ac.za.
authors.forename: Dirk J
authors.initials: DJ
authors.lastname: Blom
authors.affiliation: Hess Center for Science and Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
authors.forename: Zahi A
authors.initials: ZA
authors.lastname: Fayad
authors.affiliation: Department of Vascular Medicine, Academic Medical Centre, Amsterdam, The Netherlands.
authors.forename: John J P
authors.initials: JJ
authors.lastname: Kastelein
authors.affiliation: Montpellier School of Medicine, IRB-INSERM1040, Montpellier, France.
authors.forename: Dominique
authors.initials: D
authors.lastname: Larrey
authors.affiliation: Stathmi, Inc., New Hope, PA, USA.
authors.forename: Lukas
authors.initials: L
authors.lastname: Makris
authors.affiliation: Pharmacovigilance, Inc., San Francisco, CA, USA.
authors.forename: Charles
authors.initials: C
authors.lastname: Schwamlein
authors.affiliation: Aegerion Pharmaceuticals, Cambridge, MA, USA.
authors.forename: LeAnne
authors.initials: L
authors.lastname: Bloeden
authors.affiliation: Department of Medicine, New York University Langone Medical Center, New York, NY, USA.
authors.forename: James
authors.initials: J
authors.lastname: Underberg
mesh_terms: D000328:Adult; D000924:Anticholesteremic Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D001562:Benzimidazoles / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D005260:Female; D006720:Homozygote; D006801:Humans; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000188:drug therapy* / Q000453:epidemiology / Q000235:genetics*; D018579:Patient Selection; D058226:Plaque, Atherosclerotic / Q000453:epidemiology; D011247:Pregnancy; D012042:Registries; D012449:Safety; D013997:Time Factors
chemical_list: D000924:Anticholesteremic Agents; C473731:BMS201038; D001562:Benzimidazoles
keywords: Cardiovascular imaging; Effectiveness; Homozygous familial hypercholesterolemia; Lomitapide; Pregnancy; Registry; Safety