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July 21, 2004

Targeting High-Risk Young Patients for Statin Therapy

Author Affiliations

Author Affiliation: The Stephen and Suzanne Weiss Dean, Weill Medical College of Cornell University, New York, NY.

JAMA. 2004;292(3):377-378. doi:10.1001/jama.292.3.377

Familial hypercholesterolemia is the genetic model for the role of hypercholesterolemia in the pathology of coronary heart disease (CHD), with a frequency of heterozygous familial hypercholesterolemia of 1 in 500.1 Most heterozygous familial hypercholesterolemia patients have low-density lipoprotein cholesterol (LDL-C) values that are greater than 190 mg/dL (4.91 mmol/L), and premature heart disease is a usual occurrence.2 Therefore, physicians may classify familial hypercholesterolemia patients as being at high coronary risk and may begin preventive efforts as early as childhood, with lifestyle therapy as the foundation of any regimen. A healthful diet, antismoking, and exercise are important messages for all children3; however, because of their genetic predisposition for elevated LDL-C, young persons with familial hypercholesterolemia may require a degree of cholesterol modification that lifestyle measures alone generally cannot provide. In such cases, a more aggressive approach is justified, but the options are limited. The extracorporeal procedure of LDL apheresis is reserved generally for patients with familial hypercholesterolemia and extreme elevation of LDL-C that is unresponsive to drug therapy.4 Thus, pharmacologic intervention likely will be needed in the majority of children with familial hypercholesterolemia.