IMR Press / RCM / Volume 18 / Issue 1 / DOI: 10.3909/ricm0834

Reviews in Cardiovascular Medicine (RCM) is published by IMR Press from Volume 19 Issue 1 (2018). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by IMR Press on as a courtesy and upon agreement with MedReviews, LLC.

Open Access Case Review
Balancing Low-density Lipoprotein Cholesterol Reduction and Hepatotoxicity With Lomitapide Mesylate and Mipomersen in Patients With Homozygous Familial Hypercholesterolemia
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1 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
Rev. Cardiovasc. Med. 2017, 18(1), 21–28;
Published: 30 March 2017
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.
Apolipoprotein B synthesis inhibitor
Familial hypercholesterolemia
Lomitapide mesylate
Microsomal triglyceride transfer protein inhibitor
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