Familial Hypercholesterolemia Treatment Guide

Options For Medications

Nearly all patients with familial hypercholesterolemia will need to take cholesterol-lowering medication. Children with this condition may need to start medication as early as ten years old. Options for appropriate medications include statins, bile acid-binding resins, and cholesterol absorption inhibitors. Some patients may be able to take a combined pill that contains a statin and a cholesterol absorption inhibitor. In addition to these medicines, alirocumab and evolocumab are injectable medications specifically designed to treat familial hypercholesterolemia. Both drugs enable the liver to absorb a higher amount of low-density lipoprotein cholesterol. Patients can self-administer these injections, and they are typically taken two or three times per month. For patients who have high triglycerides and high low-density lipoprotein cholesterol, doctors may recommend using some of the aforementioned medications with omega-3 supplements, niacin, and fibrates. While taking these medications, patients may need to have routine blood tests to monitor their cholesterol and liver function. Patients should report any muscle pain, weakness, or other side effects to their medical team. If possible, an alternate medication can be prescribed to reduce side effects.

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