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Clinical trials of cholesterol lowering for primary prevention of coronary heart disease

Robert S Rosenson, MD
Section Editor
Mason W Freeman, MD
Deputy Editor
Gordon M Saperia, MD, FACC


Treating hypercholesterolemia in patients who do not have clinical evidence of coronary heart disease (CHD) is called primary prevention. The rationale for this approach is based upon epidemiologic data documenting a continuous, graded relationship between the total plasma cholesterol concentration and CHD events and mortality (figure 1A-B) [1-3]. (See "Overview of the risk equivalents and established risk factors for cardiovascular disease".)

The causal role of cholesterol in this relationship is suggested by clinical trials that have demonstrated that targeted lowering of low-density lipoprotein cholesterol (LDL-C) in patients with hypercholesterolemia reduces CHD morbidity. Multiple mechanisms that may be involved are discussed elsewhere. (See "Clinical trials of cholesterol lowering in patients with cardiovascular disease or diabetes".)


Effect of early trials on mortality — Three early trials each showed some benefits in the treated group:

World Health Organization (WHO) Cooperative Trial, which used clofibrate – 10,577 patients [4,5].

Lipid Research Clinics Coronary Primary Prevention Trial, which used cholestyramine – 3806 patients [6].


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Literature review current through: Sep 2016. | This topic last updated: Apr 6, 2016.
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