Advances in Lipid-Lowering Therapy for Homozygous Familial Hypercholesterolemia

Homozygous familial hypercholesterolemia (HoFH) is an extremely rare and severe hereditary lipid metabolism disorder, characterized by markedly elevated levels of plasma low-density lipoprotein cholesterol (LDL-C), significantly increasing the risk of atherosclerotic cardiovascular diseases. Among traditional lipid-lowering therapies, the combination of statins and ezetimibe is the basic treatment approach, but its efficacy is limited. In recent years, notable progress has been made in lipid-lowering therapy for HoFH. New drugs such as proprotein convertase subtilisin/kexin type 9 inhibitors and angiopoietin-like protein 3 inhibitors have demonstrated favorable LDL-C-lowering effects and play an active role in treatment. Lipoprotein apheresis can rapidly reduce LDL-C levels and has become an important adjuvant treatment modality. Although approaches like gene therapy and liver transplantation face many challenges, they offer hope for radical cure. Further research is still needed to optimize treatment strategies and regimens for more effective blood lipid management in patients with HoFH.