Long-Term Efficacy and Tolerability of PCSK9 Targeted Therapy: A Review of the Literature

Long-Term Efficacy and Tolerability of PCSK9 Targeted Therapy: A Review of the Literature

25 January 2024 | Willemijn A. M. Schonck¹, Erik S. G. Stroes¹, G. Kees Hovingh¹², Laurens F. Reeskamp¹
PCSK9-targeted therapy effectively lowers LDL-C levels and reduces the risk of atherosclerotic cardiovascular disease (ASCVD). It is well tolerated with a low incidence of side effects in the short term. Long-term studies show continued efficacy and safety, with PCSK9 inhibitors like evolocumab, alirocumab, and inclisiran demonstrating significant LDL-C reduction and positive effects on atherosclerotic plaque burden. PCSK9-targeted therapy is recommended for high-risk patients with familial hypercholesterolemia or those requiring additional LDL-C lowering. It has been shown to reduce cardiovascular events, including myocardial infarction, stroke, and revascularization. While some studies suggest potential adverse effects such as myalgia, diabetes, and neurocognitive events, the overall safety profile is favorable. PCSK9-targeted therapy does not appear to significantly affect fat-soluble vitamin levels or steroid hormone synthesis. Long-term adherence to PCSK9-targeted therapy is high, with most patients continuing treatment after 1-2 years. Future advancements in formulations, such as oral pills or gene therapy, may further improve accessibility and effectiveness of PCSK9-targeted therapy in the fight against atherosclerotic cardiovascular disease.PCSK9-targeted therapy effectively lowers LDL-C levels and reduces the risk of atherosclerotic cardiovascular disease (ASCVD). It is well tolerated with a low incidence of side effects in the short term. Long-term studies show continued efficacy and safety, with PCSK9 inhibitors like evolocumab, alirocumab, and inclisiran demonstrating significant LDL-C reduction and positive effects on atherosclerotic plaque burden. PCSK9-targeted therapy is recommended for high-risk patients with familial hypercholesterolemia or those requiring additional LDL-C lowering. It has been shown to reduce cardiovascular events, including myocardial infarction, stroke, and revascularization. While some studies suggest potential adverse effects such as myalgia, diabetes, and neurocognitive events, the overall safety profile is favorable. PCSK9-targeted therapy does not appear to significantly affect fat-soluble vitamin levels or steroid hormone synthesis. Long-term adherence to PCSK9-targeted therapy is high, with most patients continuing treatment after 1-2 years. Future advancements in formulations, such as oral pills or gene therapy, may further improve accessibility and effectiveness of PCSK9-targeted therapy in the fight against atherosclerotic cardiovascular disease.
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[slides and audio] Long-Term Efficacy and Tolerability of PCSK9 Targeted Therapy%3A A Review of the Literature