Treatment

Familial hypercholesterolemia treatment focuses on reducing the extremely high levels of LDL (bad) cholesterol. This helps lower the risk of heart attack and death.

Medications

Most people with familial hypercholesteremia will need to take more than one medication to control their LDL cholesterol levels. Options include:

• Statins. These drugs block a substance the liver needs to make cholesterol. Examples include atorvastatin (Lipitor), fluvastatin (Lescol XL), lovastatin (Altoprev), pitavastatin (Livalo), pravastatin, rosuvastatin (Crestor) and simvastatin (Zocor).
• Ezetimibe (Zetia). This drug limits the absorption of cholesterol contained in the food you eat. If statins don't reduce cholesterol enough, doctors often suggest adding ezetimibe.
• PCSK9 inhibitors. These newer drugs — alirocumab (Praluent) and evolocumab (Repatha) — help the liver absorb more LDL cholesterol, which lowers the amount of cholesterol circulating in the blood. They're injected under the skin every few weeks and are very expensive.

Other treatments

In severe cases, people with familial hypercholesterolemia may also need to periodically undergo a procedure that filters the excess cholesterol from their blood. Some may need liver transplants.