Affiliation:
1. University of the Witwatersrand
Abstract
Patients with severe hypercholesterolaemia have a high lifetime risk for developing cardiovascular disease. These patients are traditionally treated with high-intensity statins and ezetimibe. Some patients are refractory to treatment and cannot achieve a desirable reduction in low-density lipoprotein cholesterol (LDL-C). LDL apheresis or lipoprotein apheresis (LA) is a radical treatment which involves the intermittent extracorporeal removal of atherogenic apolipoprotein B-100-containing lipoproteins from the systemic circulation. The procedure requires the use of highly specialised equipment and is carried out under medical supervision for patients with severe hypercholesterolaemia, refractory to treatment with high-intensity statins and ezetimibe. The advent of targeted therapies, including monoclonal antibodies and gene silencing therapies, offer treatment options that could replace LA for these patients. Large scale clinical trials for the PCSK inhibitors evolocumab and alirocumab show favourable outcomes in terms of lipid lowering, with a 50% to 60% improvement in baseline LDL-C levels. This suggests that these therapies could reduce the need for LA in patients with hypercholesterolaemia. This review describes the main clinical trials for the PCSK inhibitors and discusses the place of these therapies in the management of severe hypercholesterolaemia. While these new therapies show promise as an effective option for lowering LDL-C levels in patients refractory to conventional treatment and have added benefits of ease of administration and compliance to treatment, long-term safety data is still needed. Favourable safety data could relegate the use of LA for a select few patients who may not tolerate the new therapies.