The role of microsomal triglyceride transfer protein inhibitors in the treatment of patients with familial hypercholesterolemia: risks, benefits, and management.

Statins fail to adequately reduce low-density lipoprotein-cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia, requiring these patients to undergo weekly or bi-weekly sessions of LDL apheresis. Although efficacious, LDL apheresis is an invasive procedure with high cost and low availability, and additional options, such as inhibitors of microsomal transfer protein (MTP), may have benefit. Inhibition of MTP reduces levels of circulating cholesterol and triglycerides by preventing the formation of very-low-density lipoprotein and chylomicrons. LDL-C levels decrease by as much as 50%. Unfortunately, adverse effects-the most common of which are gastrointestinal-related and hepatic lipid accumulation-limit broader use of the drug. Furthermore, the cardiovascular benefit of MTP inhibition remains unclear. However, MTP inhibition offers a viable additional lipid-lowering option for patients with homozygous familial hypercholesterolemia.