Treat Heterozygous Familial Hypercholesterolemia

If you've been diagnosed with heterozygous familial hypercholesterolemia (HeFH), your doctor will work with you to figure out the best way to treat your disease. Whatever you decide, the goal is to lower your levels of LDL "bad" cholesterol. Often, it can take a combo of drugs -- along with diet and exercise -- to get your cholesterol numbers down.

High-Dose Statins

The first type of drug that your doctor may prescribe is called a "statin." It blocks one of the compounds your body needs to make cholesterol and helps it absorb cholesterol that's in your blood.

Because HeFH can send your cholesterol levels way up, your doctor may suggest high doses of statins. Studies show that taking the maximum amounts of rosuvastatin or atorvastatin -- the strongest statin drugs -- can lower your LDL levels by more than 50%.

"Non-Statin" Cholesterol-Lowering Drugs

High-dose statins might not lower your LDL levels to those your doctor is aiming for. It's not uncommon for people with HeFH to take two, three, or even four drugs to lower cholesterol. Some of the other drugs that might be recommended for you are:

PCSK9 inhibitors. Alirocumab (Praluent) and evolocumab (Repatha) are drugs in this group. They make it easier for your liver to remove LDL cholesterol from your blood. Your doctor may recommend these medications if you take the maximum dose of statins but still have high LDL levels. Evolocumab has actually been approved for treatment in preventing heart attack or stroke in people with cardiovascular disease.

Ezetimibe (Zetia). It's a drug that stops your body from absorbing all the cholesterol you eat. It often works well when combined with statins. Your doctor may suggest Liptruzet, which contains atorvastatin and azetimibe or Vytorin, a medication that's a combo of ezetimibe and a statin called simvastatin.


If medication and lifestyle changes aren't enough to lower your LDL levels, your doctor may suggest other methods to help prevent heart disease.

LDL apheresis is a safe and effective way to remove all the LDL from your blood by filtering it. You'll be asked to relax in a hospital bed while your blood is slowly drawn through a machine and back into your body.

Some people feel light-headed, flushed, or nauseous while it's going on. To have the most benefit, you'll likely have to repeat the procedure every few weeks to continue removing LDL from your blood.

You may hear people talk about liver transplants as a possible treatment, but they're rarely used for HeFH. They're more common to treat a ,ore serious form of the disease called homozygous familial hypercholesterolemia (HoFH).

WebMD Medical Reference Reviewed by Melinda Ratini, DO, MS on January 10, 2018



Medscape: "Familial Hypercholesterolemia Medication," "Familial Hypercholesterolemia Treatment & Management."

Robinson J.G. Journal of Managed Care Pharmacy, 2013.

Mayo Clinic: "Statins: Are these cholesterol-lowering drugs right for you?"

UC San Diego: "LDL Apheresis Patient Handout."

News release, FDA.

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