The extent of reduction of serum cholesterol achieved by a combination of lipid-lowering measures, is a major determinant of survival in patients with homozygous familial hypercholesterolemia (FH), according to findings from a recent study published in the European Heart Journal.

Researchers divided 133 "previously statin-naïve" FH homozygotes from South Africa and the UK into quartiles based on their on-treatment levels of serum cholesterol. They then compared the occurrence of any death, cardiovascular death, and major adverse cardiovascular events (MACE) between the quartiles during 25 years of follow-up using regression analysis.

Results found that patients in Quartile 4, with an on-treatment serum cholesterol >15.1mmol/L, had a hazard ratio of 11.5 for any death compared with those in Quartile 1, with an on-treatment cholesterol of< 8.1mmol/L. Patients in both Quartiles 2 and 3 combined (on-treatment cholesterol of 8.1–15.1mmol/L) had a hazard ratio of 3.6 compared with those in Quartile 1. According to researchers, these differences were statistically significant (P < 0.001) and remained so after adjustments for confounding factors (P = 0.04). They also observed significant differences between quartiles for cardiovascular deaths and MACE.

"Our findings support proposals that in homozygotes failing to achieve target levels of LDL with high-dose statins and ezetimibe, additional therapy is required," researchers said. In addition to lipoprotein apheresis, current treatment options include evolocumab (except in receptor-negative homozygotes) and either lomitapide or mipomersen. However, researchers also point out that "the choice will be dictated by the availability, affordability, and risks and benefits of the treatment in question."

Keywords: Hyperlipoproteinemia Type II, Hydroxymethylglutaryl-CoA Reductase Inhibitors, omega-Chloroacetophenone, Homozygote, Follow-Up Studies, South Africa, Oligonucleotides, Antibodies, Monoclonal, Cholesterol, Blood Component Removal, Benzimidazoles, Lipoproteins, Regression Analysis, Risk Assessment, Secondary Prevention, Dyslipidemias

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