Evinacumab Treatment of Homozygous Familial Hypercholesterolemia Patients in a Real-World Setting: An Overview After the First Year of Therapy.

BACKGROUND AND

OBJECTIVES

Homozygous familial hypercholesterolemia (HoFH) is a genetic disorder characterized by lifelong low-density lipoprotein cholesterol (LDL-C) elevation, which results in early onset of atherosclerotic cardiovascular disease. Recently, evinacumab, an angiopoietin-like 3 protein inhibitor, became available to treat patients affected by HoFH. We aimed to investigate safety and efficacy data in HoFH in clinical practice.

METHODS

Our HoFH cohort (n = 7) included 4 patients with 2 LDL receptor (LDLR)-related or LDLRAP1-related copies of the identical variant and 3 patients with 1 copy each of 2 different LDLR-related variants. All subjects were receiving maximally tolerated lipid-lowering therapy, 4 patients had additionally lipoprotein apheresis. The average LDL-C at baseline was 222 mg/dl. Each patient was treated with evinacumab at a dose of 15 mg/kg intravenously every 4 weeks, except for one patient, who received it every 6 to 8 weeks. In one patient, therapy was discontinued after 3 doses, due to a suspected allergic reaction. Clinical and lipid parameters were regularly evaluated over 52-weeks.

RESULTS

An average decrease from baseline in LDL-C of -68.9 ± 14.0% (P < 0,001) was observed throughout the treatment period of 52 weeks. In one patient, reductions of 56% and 52% were achieved after therapy intervals of 6 and 8 weeks, respectively. Five patients reached their LDL-C target, lipoprotein apheresis could be interrupted in 2 patients due to high efficacy of evinacumab. No side effects were observed, except for a suspected allergic reaction.

CONCLUSION

Evinacumab on top of the best standard of care treatment is an effective treatment to further reduce LDL-C levels in HoFH patients.