Physicians' Academy for Cardiovascular Education

Longer-term treatment with PCSK9 antibody safely lowers LDL-c in HoFH patients

Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study

Literature - Raal FJ, Hovingh GK, Blom D, et al., - Lancet Diabetes Endocrinol. 2017 Feb 16. doi: 10.1016/S2213-8587(17)30044-X

Main results

LDL-c reductions in those on apheresis at week 12 and 48 did not significantly differ from reductions achieved in those only receiving drug therapy.


This interim analysis represents the largest and longest study of lipid-lowering medication in HoFH patients to date, the results of which support the long-term tolerability and safety of the PCSK9 antibody evolocumab. 95% of patients were still in the study at 48 weeks. Evolocumab substantially reduced LDL-c concentrations in HoFH patients on standard lipid-lowering therapy, with or without apheresis.

The study also describes treatment of adolescent patients, who showed a decreased treatment response, but numbers were low.


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Find this article online at Lancet Diabetes Endocrinol.

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