Treatment of Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder that increases the level of low-density lipoprotein cholesterol (LDL-C) and causes premature coronary artery disease. HoFH is usually undiagnosed until the cardiac events occur. Most patients with asymptomatic HoFH are also not treated adequately. Proprotein convertase subtilisin/kexin 9 (PCSK9) inhibitor is a powerful LDL-C-lowering agent. In this review, we described the treatment experiences of eight patients with HoFH in our hospital. We also reviewed the clinical trials of PCSK9 inhibitor therapy for HoFH and discussed the current challenges of HoFH treatment in Taiwan.