FH Treatments

Receiving news that you’ve been diagnosed with familial hypercholesterolemia (FH) can be scary. What does this mean for your overall health? How does it affect your daily life? How will it affect your family?

By seeking help right away, you can live a longer, healthier life. That’s because the earlier you are diagnosed, the earlier you can start cholesterol lowering treatments that can slow down or prevent heart disease, or prevent or delay another event, procedure or surgery if you already have cardiovascular disease.

FH Treatment

Working with an FH specialist, your familial hypercholesterolemia treatment may consist of one or a combination of medications. Using these therapies is important to your long-term health and several treatment resources are available to help you succeed. In addition to treatment, it is important to maintain a heart healthy diet (low in saturated fat and cholesterol), regular exercise, and not smoke tobacco.

Unlike high cholesterol that develops later in life due to age or lifestyle factors, high cholesterol levels in those with FH are present from birth. Treatment of this condition is a lifelong journey, requiring a combination of lifestyle and therapies to reduce high LDL (bad)-cholesterol. High cholesterol can be managed successfully despite the presence of a gene that raises cholesterol.

Family with HoFH

Taking medication regularly remains a crucial component of FH management to reduce the chance of a heart attack, or the need for stents or bypass surgery. If you already have cardiovascular disease, treatment can lower your chances of having another event or needing additional stents or bypass. Statins are first-line treatment. They are effective cholesterol lowering medications and are safe for those with FH including children.  Individuals with FH can be treated with additional medications, depending on the response to statins and medical recommendations, and often take more than one.

Tools and Resources

The Family Heart Foundation has created these resources for understanding treatment options for familial hypercholesterolemia and lowering high cholesterol:

Cholesterol Lowering Medications for Familial Hypercholesterolemia

FH Cholesterol Lowering Medication - Statin

Statins

These medications block the production of cholesterol in the liver and make the liver use cholesterol from your bloodstream, thereby reducing your cholesterol.

Cholesterol Lowering Medication - Ezetimibe

Cholesterol Absorption Inhibitor (Ezetimibe)

These medications work in the small intestine by decreasing cholesterol absorption.

Cholesterol Lowering Medication - Bile Acid Sequestrant

Bile Acid Sequestrants

This medication also works in the intestine by binding bile. Since bile is composed, in large part, of cholesterol, these medications lead to loss of cholesterol in the stool.

Cholesterol Lowering Medication - PCSK9

PCSK9 Inhibitors

These antibodies target PCSK9, a protein that interferes with clearance of blood cholesterol.

Cholesterol Lowering Medication - Bempedoic Acid

Bempedoic Acid

Bempedoic acid is a non-statin treatment to lower LDL-cholesterol in individuals with FH or established heart disease who require additional LDL-C lowering even after maximally tolerated statin treatment.

Lipoprotein Apheresis

Lipoprotein Apheresis

This treatment involves removing bad LDL-cholesterol from the blood. Since the cholesterol will build up again over time, this procedure is done every week to every other week.

Evinacumab (HoFH only)

Evinacumab (HoFH only)

Evinacumab was approved by the Food and Drug Administration (FDA) in 2021 for LDL reduction in people with HoFH aged 12 and over.

Lomitapide

Lomitapide (HoFH only)

Lomitapide is a treatment designed to inhibit the function of microsomal triglyceride transfer protein (MTP), which plays an essential role in the liver and intestines by synthesizing lipoproteins that contain cholesterol.

Style Adjustments

Note: Liver Transplant for HoFH
In very rare cases, a liver transplant is recommended to replace a person’s liver with a new liver that does not have any FH causing genes. It generally is reserved for very young patients with Homozygous Familial Hypercholesterolemia and advanced atherosclerosis for whom other treatment options have not been sufficient.

Lifestyle Changes to Manage Familial Hypercholesterolemia

Lifestyle changes enhance medication efficacy. Heart-healthy decisions benefit you and your entire family if they join you in your efforts. Don’t be afraid to seek advice from a registered dietician who understands FH.

 

Lifestyle management includes:

  • Diets low in saturated and trans fats, low in cholesterol
  • Regular exercise
  • Weight control
  • Not smoking
Lifestyle Changes to Manage Familial Hypercholesterolemia

It is also important to manage all other risk factors for heart disease, including hypertension (high blood pressure), diabetes, and smoking. The Family Heart Foundation can connect you to other individuals with FH to see what works for them.  We can help you maintain your health through our CASCADE FH® Patient Portal, which provides resources to track your heart health, medications, and more.

Veggies

What's a good FH diet?

Eating a low-fat diet can help you manage the high cholesterol associated with FH. Our experts and FH community have put together recommendations for you.

Page reviewed by: Michael Shapiro DO