Low-Density Lipoprotein Apheresis in Patients with Severe Familial Hypercholesterolemia Refractory to, or Intolerant of, Lipid-Lowering Drug Therapy: Preventing the Onset or Progression of Cardiovascular Disease

Background: Familial hypercholesterolemia (FH) is a genetic disorder of lipoprotein metabolism characterized by extremely high plasma concentrations of low-density lipoprotein cholesterol (LDLc), tendon xanthomas, and increased risk of premature coronary heart disease (CHD). Worldwide, over 10 million people are currently afflicted with FH, the majority being heterozygous. If appropriate preventive efforts are not employed before the age of 65, approximately 85% of males and 50% of females will suffer a coronary event (Civeira, 2003). LDL apheresis is an efficacious method of decreasing LDLc concentrations in patients with FH who are either refractory to, or intolerant of, pharmacologic therapy. Method: An extensive literature search was performed in October through November of 2010 using the databases of PubMed, Endotext, Science Direct, and the journals of Cardiology in Review and The Journal of Clinical Lipidology. The key words searched included “low-density lipoprotein apheresis” and “familial hypercholesterolemia” individually and in various combinations. Inclusion criteria consisted of full text articles in the English language pertinent to LDL apheresis and its effect on cardiovascular disease risk factors or events. Exclusion criteria consisted of articles describing the use of LDL apheresis in non-cardiovascular disease processes, articles older than 15 years, and study designs other than randomized, controlled trials, cohort and case control studies. Results: Altogether, four studies of randomized, controlled design were selected for review. In all four trials, angiographic evidence of atherosclerotic plaque regression was analyzed. The clinical effects of LDL apheresis include arrest of progression of coronary stenosis, reduction of calcified plaque volume and plaque area, expansion of coronary reserve, and reduction of morbidity and mortality secondary to coronary heart disease. LDL apheresis is an adjunctive therapy to lipid-lowering drugs, commanding a vital role in the prevention of cardiac events through restructuring and stabilization of coronary plaque. Conclusion: Low-density lipoprotein apheresis is a safe and effective procedure in patients with severe familial hypercholesterolemia refractory, or intolerant of, lipid-lowering drug therapy as a means to prevent the onset or progression of cardiovascular events in this high risk population