Current and Future Treatment of Severe Hypercholesterolemia

• Therapy for severe FH:

• Pharmacotherapy – lipid-modifying drugs

• Extracorporeal removal of LDL-apheresis

• Surgical therapy – portacaval shunt, partial bypass

• Methods to restore LDL receptor activity – liver transplantation, gene therapy.

• Despite the absence of RCTs in subjects with FH, since their introduction in the late 1980s, statins have become the mainstay of therapy. While statins and ezetimibe constitute first-line therapy, they provide less than optimal LDL-C reduction in severe FH.

• Homozygous FH mainly requires combination therapy: high-dose statin, ezetimibe, with or without apheresis.