Patients with homozygous familial hypercholesterolemia (FH) have severe hypercholesterolemia from birth and if untreated may experience very early onset of coronary artery disease in childhood or young adulthood with an aggressive course resulting in early death. Early initiation of aggressive low-density lipoprotein cholesterol (LDL-C) lowering is the mainstay of treatment, which requires the use of a multidrug treatment regimen, often in combination with lipoprotein apheresis, but LDL-C goal achievement is frequently unattainable due to the severity of baseline hypercholesterolemia and hyporesponsiveness to many LDL-C-lowering medications. Evinacumab, a monoclonal antibody that sequesters angiopoietin-like 3 protein and lowers LDL-C by an average of 49% in patients with homozygous FH, was approved by the Food and Drug Administration in February 2021 and is a major advance in treatment of these high-risk patients. In this report, we describe the complementary role of evinacumab in combination with lipoprotein apheresis in two patients with homozygous FH.
- angiopoietin-like 3 (ANGPTL3)
- coronary artery disease (CAD)
- homozygous familial hypercholesterolemia (HoFH)
- lipoprotein apheresis
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