Evinacumab Enables Discontinuation of LDL Apheresis in Homozygous Familial Hypercholesterolemia: A Case Report

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) levels and early cardiovascular disease. Despite intensive lipid-lowering therapy, achieving target LDL-C levels remains challenging, and many patients require lifelong LDL apheresis. We report a 33-year-old Mexican–Lebanese woman with confirmed HoFH and multiple comorbidities, including systemic lupus erythematosus. Severe hypercholesterolemia was evident from early childhood, with total cholesterol levels of approximately 800 mg/dL and development of xanthomas at age 3. The patient underwent long-term multimodal therapy, including statins, ezetimibe, PCSK9 inhibitors, and LDL apheresis initiated at age 9 and continued for approximately 20 years, achieving only partial lipid control. Evinacumab, an angiopoietin-like protein 3 (ANGPTL3) inhibitor, was initiated in adulthood as adjunctive therapy. Within two months, significant and sustained LDL-C reduction was achieved, allowing complete discontinuation of LDL apheresis. Follow-up demonstrated stable LDL-C levels between 180–225 mg/dL. This case highlights the clinical effectiveness of Evinacumab in patients with HoFH, demonstrating its potential to achieve sustained lipid reduction and eliminate the need for invasive apheresis. ANGPTL3 inhibition may represent a transformative strategy for improving long-term management and quality of life in this high-risk population, particularly in settings with limited access to apheresis.