Familial Hypercholesterolemia.

Heterozygous familial hypercholesterolemia is a common, severe form of elevated plasma, low-density lipoprotein (LDL) cholesterol. Familial hypercholesterolemia is under-recognized and under-treated despite well-known benefits from available lipid-lowering medications. To help address these problems, the humanitarian MEDPED (Make Early Diagnoses, Prevent Early Deaths) organization was developed as a means to find and help persons with familial hypercholesterolemia all over the world. Although some patients with familial hypercholesterolemia can be treated adequately with a statin alone, many require aggressive treatment with a combination of drugs, including a statin, bile acid sequestrant, and niacin to reach LDL goals. Such treatment has resulted in regression of angiographically monitored coronary lesions in patients with familial hypercholesterolemia.