The role of microsomal triglyceride transfer protein inhibitors in the treatment of patients with familial hypercholesterolemia: Risks, benefits, and management

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Abstract

Statins fail to adequately reduce low-density lipoprotein-cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia, requiring these patients to undergo weekly or bi-weekly sessions of LDL apheresis. Although efficacious, LDL apheresis is an invasive procedure with high cost and low availability, and additional options, such as inhibitors of microsomal transfer protein (MTP), may have benefit. Inhibition of MTP reduces levels of circulating cholesterol and triglycerides by preventing the formation of very-low-density lipoprotein and chylomicrons. LDL-C levels decrease by as much as 50 %. Unfortunately, adverse effects—the most common of which are gastrointestinal-related and hepatic lipid accumulation—limit broader use of the drug. Furthermore, the cardiovascular benefit of MTP inhibition remains unclear. However, MTP inhibition offers a viable additional lipid-lowering option for patients with homozygous familial hypercholesterolemia.

Original languageEnglish (US)
Article number469
JournalCurrent Atherosclerosis Reports
Volume17
Issue number1
DOIs
StatePublished - Jan 18 2015

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Hyperlipoproteinemia Type II
Risk Management
Blood Component Removal
LDL Cholesterol
Proteins
Lipids
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Chylomicrons
VLDL Lipoproteins
Therapeutics
Triglycerides
Cholesterol
Costs and Cost Analysis
microsomal triglyceride transfer protein
Liver
Pharmaceutical Preparations
oxidized low density lipoprotein

Keywords

  • Abetalipoproteinemia
  • Familial hypercholesterolemia
  • Homozygous familial hypercholesterolemia
  • LDL apheresis
  • LDL-C
  • LDL-Receptor
  • Lomitapide
  • MTP

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine
  • Medicine(all)

Cite this

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title = "The role of microsomal triglyceride transfer protein inhibitors in the treatment of patients with familial hypercholesterolemia: Risks, benefits, and management",
abstract = "Statins fail to adequately reduce low-density lipoprotein-cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia, requiring these patients to undergo weekly or bi-weekly sessions of LDL apheresis. Although efficacious, LDL apheresis is an invasive procedure with high cost and low availability, and additional options, such as inhibitors of microsomal transfer protein (MTP), may have benefit. Inhibition of MTP reduces levels of circulating cholesterol and triglycerides by preventing the formation of very-low-density lipoprotein and chylomicrons. LDL-C levels decrease by as much as 50 {\%}. Unfortunately, adverse effects—the most common of which are gastrointestinal-related and hepatic lipid accumulation—limit broader use of the drug. Furthermore, the cardiovascular benefit of MTP inhibition remains unclear. However, MTP inhibition offers a viable additional lipid-lowering option for patients with homozygous familial hypercholesterolemia.",
keywords = "Abetalipoproteinemia, Familial hypercholesterolemia, Homozygous familial hypercholesterolemia, LDL apheresis, LDL-C, LDL-Receptor, Lomitapide, MTP",
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N2 - Statins fail to adequately reduce low-density lipoprotein-cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia, requiring these patients to undergo weekly or bi-weekly sessions of LDL apheresis. Although efficacious, LDL apheresis is an invasive procedure with high cost and low availability, and additional options, such as inhibitors of microsomal transfer protein (MTP), may have benefit. Inhibition of MTP reduces levels of circulating cholesterol and triglycerides by preventing the formation of very-low-density lipoprotein and chylomicrons. LDL-C levels decrease by as much as 50 %. Unfortunately, adverse effects—the most common of which are gastrointestinal-related and hepatic lipid accumulation—limit broader use of the drug. Furthermore, the cardiovascular benefit of MTP inhibition remains unclear. However, MTP inhibition offers a viable additional lipid-lowering option for patients with homozygous familial hypercholesterolemia.

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