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Reviews in Cardiovascular Medicine  2017, Vol. 18 Issue (1): 21-28     DOI: 10.3909/ricm0834
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Balancing Low-density Lipoprotein Cholesterol Reduction and Hepatotoxicity With Lomitapide Mesylate and Mipomersen in Patients With Homozygous Familial Hypercholesterolemia
Jane I. Won, Jun Zhang, Kristen M. Tecson , Peter A. McCullough
Baylor University Medical Center, Baylor Jack and Jane Hamilton Heart and Vascular Hospital, Baylor Heart and Vascular Institute, Dallas, TX; The Heart Hospital, Plano, TX
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Abstract:
Homozygous familial hypercholesterolemia (HoFH) is an autosomal codominant disorder manifested by high concentrations of total cholesterol and low-density lipoprotein (LDL) cholesterol, and premature cardiovascular disease. Despite conventional lipid-lowering therapy, LDL cholesterol levels remain elevated in patients with HoFH; these patients are considered to be at high risk for cardiovascular events. In 2012-2013, two drugs with novel mechanisms of action were approved by the US Food and Drug Administration for the treatment of HoFH: lomitapide mesylate and mipomersen. Both of these treatments reduce total cholesterol, LDL cholesterol, non–high-density lipoprotein cholesterol, apolipoprotein B, lipoprotein a, and triglyceride levels. This review describes the clinical tradeoffs in efficacy and hepatotoxicity of these drugs in two cases of HoFH.
Key words:  Apolipoprotein B synthesis inhibitor      Familial hypercholesterolemia      Hepatotoxicity      Lomitapide mesylate      Mipomersen      Microsomal triglyceride transfer protein inhibitor     
Published:  30 March 2017     

Cite this article: 

Jane I. Won, Jun Zhang, Kristen M. Tecson , Peter A. McCullough. Balancing Low-density Lipoprotein Cholesterol Reduction and Hepatotoxicity With Lomitapide Mesylate and Mipomersen in Patients With Homozygous Familial Hypercholesterolemia. Reviews in Cardiovascular Medicine, 2017, 18(1): 21-28.

URL: 

https://rcm.imrpress.com/EN/10.3909/ricm0834     OR     https://rcm.imrpress.com/EN/Y2017/V18/I1/21

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