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Reviews in Cardiovascular Medicine  2014, Vol. 15 Issue (1): 1-10     DOI: 10.3909/ricm0702
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Use of Microsomal Triglyceride Transfer Protein Inhibitors in Patients With Homozygous Familial Hypercholesterolemia: Translating Clinical Trial Experience Into Clinical Practice
Peter P. Toth1, Prediman K. Shah2, Michael J. Wilkinson3, Michael H. Davidson3, Peter A. McCullough4
1 CGH Medical Center, Sterling, IL, and University of Illinois College of Medicine, Peoria, IL
2 Division of Cardiology and Oppenheimer Atherosclerosis Research Center, Cedars Sinai Heart Institute, Los Angeles, CA
3 Department of Medicine, University of Chicago, Chicago, IL
4 St. John Providence Health System, Warren, MI, and Providence Hospitals and Medical Centers, Southfield and Novi, MI
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Abstract:
Homozygous familial hypercholesterolemia (HoFH) is associated with severe hypercholesterolemia and premature cardiovascular morbidity and mortality. The most frequent cause of HoFH is loss of function mutations in the gene for the low-density lipoprotein receptor, resulting in reduced clearance of low-density lipoprotein (LDL) cholesterol from the circulation. Patients with HoFH have attenuated responsiveness to lipid-lowering therapies such as statins, cholesterol absorption inhibition, and bile acid-binding resins because of impaired LDL receptor expression. Lomitapide is a novel microsomal triglyceride transfer protein inhibitor that does not depend on the ability to upregulate LDL receptors on the surface of hepatocytes. Lomitapide reduces production of apolipoprotein B-containing lipoproteins, significantly reduces serum levels of LDL cholesterol, and is approved for use in patients with HoFH in the United States and the European Union.
Key words:  Atherosclerosis      Homozygous familial hypercholesterolemia      Lomitapide      Low-density lipoprotein cholesterol      Microsomal triglyceride transfer protein      Very low-density lipoprotein cholesterol     
Published:  30 March 2014     

Cite this article: 

Peter P. Toth, Prediman K. Shah, Michael J. Wilkinson, Michael H. Davidson, Peter A. McCullough. Use of Microsomal Triglyceride Transfer Protein Inhibitors in Patients With Homozygous Familial Hypercholesterolemia: Translating Clinical Trial Experience Into Clinical Practice. Reviews in Cardiovascular Medicine, 2014, 15(1): 1-10.

URL: 

https://rcm.imrpress.com/EN/10.3909/ricm0702     OR     https://rcm.imrpress.com/EN/Y2014/V15/I1/1

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