How is FH treated?

To reduce the risk of cardiovascular disease in people with FH, it is important that treatment with cholesterol-lowering drugs is started as early as possible – preferably already during childhood. Treatment is usually started with drugs called statins. Statins reduce cholesterol production and increase the ability of the liver to clear cholesterol from the blood. They are among the most widely used drugs worldwide to prevent cardiovascular disease (not only in FH). Many patients with FH will still have elevated levels of cholesterol while receiving statins. The next step is usually to add a drug called ezetimibe, which reduces the uptake of cholesterol from the intestine. If people with FH still have elevated blood cholesterol despite receiving statins and ezetimibe, or if people are unable to take statins because they experience side effects, then they are often treated with a new type of drugs called PCSK9 inhibitors. These drugs provide larger reductions in LDL-cholesterol than statins or ezetimibe, but have to be administered by injections in the skin every two weeks (or sometimes every four weeks).