Author Interviews, Lancet, Lipids / 02.10.2014
Evolocumab For Resistant Familial Hypercholesterolemia
MedicalResearch.com Interview with:
Dr. Raul Santos
Unidade Clínica de Lipides InCor-HCFMUSP
Sao Paulo, Brazil.
Medical Research: What are the main findings of the study?
Dr. Santos: Evolocumab 420 mg injected subcutaneously every 4 weeks reduced LDL-C by 31% on average, in relation to placebo, in Homozygous familial hypercholesterolemia patients that were using maximally tolerated lipid lowering therapy but not on lipid apheresis regimen. Patients were separated according to the type of LDL receptor mutation, those with at least one allele codifyng a defective mutation on the LDL receptor (residual receptor activity 2-25%) had on average a 41% reduction on LDL-cholesterol. The 2 patients homozygotes with alleles that codify a null mutation )receptor activity < 2%), did not respond to treatment. This was expected since PCSK9 inhibitors need a functional LDL receptor do work. Basically they increase the expression of the receptor that facilitates the clearance from plasma of circulating LDL particles. In those patients with defective LDL receptor mutations there was 24% reduction of lipoprotein(a) concentrations, an extra risk factor for cardiovascular disease in familial hypercholesterolemia patients.
















