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Treatment of Familial Hypercholesterolemia: Is There a Need Beyond Statin Therapy?

  • Statin Drugs (M Clearfield, Section Editor)
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Abstract

Familial hypercholesterolemia (FH) is a genetic lipid disorder that is characterized by severely elevated cholesterol levels and premature cardiovascular disease. Both the heterozygous and homozygous forms of FH require aggressive cholesterol-lowering therapy. Statins alone frequently do not lower these patients’ cholesterol to therapeutic levels, and some patients are intolerant to statins. Combination or monotherapy with other current pharmacotherapies are options, but even with these some FH patients do not meet their low-density lipoprotein (LDL) cholesterol goals. In the cases of statin intolerance, LDL apheresis may be another treatment option. There are currently several novel therapies in development for LDL lowering that target either production or catabolism of LDL, plaque regression, and potentially gene transfer. We conclude that there is a need beyond statins for patients with FH, especially in cases of statin intolerance, and when even the highest doses of statin do not get patients to goal cholesterol levels.

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Disclosure

A. Raper: none; D.M. Kolansky: none; M. Cuchel has received research funding from the US Food and Drug Administration Office of Orphan Product Development (FD-R-003098) and Aegerion Pharmaceuticals.

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Authors and Affiliations

  1. Institute of Translational Medicine and Therapeutics, University of Pennsylvania School of Medicine, 3600 Spruce Street, Philadelphia, PA, 19104, USA

    Anna Raper & Marina Cuchel

  2. Cardiovascular Medicine Division, University of Pennsylvania School of Medicine, Philadelphia, PA, USA

    Daniel M. Kolansky

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  1. Anna Raper
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Correspondence to Marina Cuchel.

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Raper, A., Kolansky, D.M. & Cuchel, M. Treatment of Familial Hypercholesterolemia: Is There a Need Beyond Statin Therapy?. Curr Atheroscler Rep 14, 11–16 (2012). https://doi.org/10.1007/s11883-011-0215-y

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