FH Treatments

Homozygous familial hypercholesterolemia (HoFH) is the rare and most severe form of FH, characterized by LDL cholesterol levels over 400 mg/dL and a family history of high cholesterol and/or early heart disease, usually on both sides of the family. Treatment for HoFH should begin as soon as possible, regardless of age. Anyone with HoFH, or suspecting HoFH, should be seen by a specialist.

A great deal of progress in HoFH treatment has been made in recent years. There are multiple effective treatments for HoFH available today. A combination of treatments is usually required to lower LDL cholesterol to recommended levels.

 

 

 

FH Treatment
Family with HoFH

Tools and Resources

The Family Heart Foundation has created these resources for understanding treatment options for homozygous 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.

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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.

Clinical Trials

Additional therapies are in clinical trials and development. If you are interested in learning about a clinical trial in your area, visit our clinical trials page.

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