title: Treatment of familial hypercholesterolemia: is there a need beyond statin therapy?
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.
authors.affiliation: Institute of Translational Medicine and Therapeutics, University of Pennsylvania School of Medicine, 3600 Spruce Street, Philadelphia, PA 19104, USA.
authors.forename: Anna
authors.initials: A
authors.lastname: Raper
authors.forename: Daniel M
authors.initials: DM
authors.lastname: Kolansky
authors.forename: Marina
authors.initials: M
authors.lastname: Cuchel
mesh_terms: D008078:Cholesterol, LDL / Q000097:blood*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000627:therapeutic use*; D006938:Hyperlipoproteinemia Type II / Q000097:blood / Q000628:therapy*; D016896:Treatment Outcome
chemical_list: D008078:Cholesterol, LDL; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors