The currently preferred treatment is LDL-apheresis available in a few specialised centers.Patient's blood is run through a special instrument (with LDL antibodies) which removes patient's LDL-cholesterol.
The standard of care for HoFH is plasmapheresis or LDL-apheresis, every one to two weeks. Usual oral medications, such as statins, ezetimibe, niacin and bile acid resins, are typically about 5-10% effective in reducing LDL cholesterol, although a few fortunate patients may get a better response than this. Lomitapide (proprietary name “Juxatpid”), an oral medication, was recently approved in Canada and the US for limited use in HoFH and can lower LDL cholesterol by up to 40%. An injectable drug called mipomersen (proprietary name “Kynamro”) lowers LDL cholesterol by a similar amount, and was recently approved in the US but not Canada. There are other even new injectable medications – PCSK9 inhibitors – in development that appear to work very well in heterozygous FH and could work moderately well in certain patients with HoFH.