JCL Roundtable: Drug treatment of severe forms of familial hypercholesterolemia

► Two experts on the treatment of severe hypercholesterolemia answer the Editor's questions in this Roundtable discussion. Questions are about the management of patients whose LDL cholesterol concentration is too high or unresponsive to currently used treatments such as statins, alone or in combination. The experts share information about mipomersen and lomitapide, new drugs recently released by the Food and drug Administration for use in patients with homozygous familial hypercholesterolemia.

Clinical lipidologists are often asked to manage patients with severely elevated low-density lipoprotein cholesterol (LDL-C) and other apolipoprotein B-containing lipoproteins. Statins at maximum doses and in combination with other drugs may not achieve adequate reductions in LDL-C in such patients. The most dramatic elevations are usually in patients with genetic abnormalities in the LDL receptor gene on both chromosome pairs. LDL-C values well in excess of 400 mg/dL are not fully responsive to current treatments. In the past few months, the Food and Drug Administration has approved 2 new drugs for special use in such patients; these are mipomersen and lomitapide. During the National Lipid Association’s Scientific Sessions, 2 highly experienced clinician scientists who have completed research studies with these agents agreed to answer questions pertinent to the prescription use of these agents. These scientists are Dr Anne Goldberg from Washington University in St. Louis and Dr Daniel Rader from the University of Pennsylvania.