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Abstract
Single-drug therapy is often not sufficient to lower total and low-density lipoprotein
(LDL) cholesterol levels in patients with familial hypercholesterolemia to desirable
or target levels. Therefore, combination drug therapy is often necessary.The most
potent therapy to achieve this goal is a combination of a 3-hydroxy-3-methylglutaryl
coenzyme A (HMG CoA) reductase inhibitor, which reduces cholesterol synthesis, and
a bile acid sequestrant, which indirectly depletes the intrahepatic cholesterol pool.
LDL cholesterol reductions reportedly vary between 52 and 54% in short-term trials.
Combining a bile acid sequestrant with nicotinic acid reduces LDL cholesterol 34-55%
and with a fibrate, 12-42%. The triple-drug regimen of bile acid sequestrant, an HMG
CoA reductase inhibitor, and nicotinic acid is even more effective, achieving reductions
of 59-67%. All these regimens elevate high-density lipoprotein cholesterol levels
concomitantly by 2-37%.