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Homozygous Familial Hypercholesterolemia (HoFH)

Real-world study assesses use of evinacumab plus lipid-lowering therapy for HoFH

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Evinacumab as an adjunct to lipid-lowering therapy resulted in sustained low-density lipoprotein cholesterol (LDL-C) lowering and improved cardiovascular event-free survival in patients with homozygous familial hypercholesterolemia (HoFH).

The real-world OLE ELIPSE HoFH study enrolled 12 newly diagnosed HoFH patients and those completing the ELIPSE HoFH trial from 3 centers in France. Participants (7 males; age range, 12-57 years) were on stable lipid-lowering therapy, including lipoprotein apheresis but excluding lomitapide, and received evinacumab 15 mg/kg intravenously every 4 weeks for 6 months.

Patients were compared with an age-, sex-, and lipoprotein apheresis treatment-matched control cohort that did not receive evinacumab or lomitapide.

The addition of evinacumab resulted in a significant mean LDL-C reduction of 3.7 mmol/L (56%), from 6.5 mmol/L at baseline to 2.8 mmol/L at 6 months (P<.0001), which was sustained over a median 3.5 years of follow-up. No cardiovascular events were reported in patients receiving evinacumab compared with a 24% incidence (n=5/21; 13 events) in the control cohort (likelihood P=.0267).

Reference

Béliard S, Saheb S, Litzler-Renault S, et al. Evinacumab and cardiovascular outcome in patients with homozygous familial hypercholesterolemia. Arterioscler Thromb Vasc Biol. 2024. doi:10.1161/ATVBAHA.123.320609

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