Key result
Novel therapies including PCSK9 inhibitors, inclisiran, and evinacumab provide substantial LDL-C reductions of 35% to 60% in patients with familial hypercholesterolemia.
Why the study?
Despite treatment with traditional lipid-lowering therapies, most patients with familial hypercholesterolemia are unable to achieve target LDL-C.
Do novel lipid-lowering therapies (PCSK9 inhibitors, inclisiran, evinacumab) reduce LDL-C in patients with familial hypercholesterolemia?
Do novel lipid-lowering therapies (PCSK9 inhibitors, inclisiran, evinacumab) reduce LDL-C in patients with familial hypercholesterolemia?
Answers are grounded in this paper and cite their sources.
Novel lipid-lowering therapies such as PCSK9 inhibitors, inclisiran, and evinacumab provide significant LDL-C reductions in patients with familial hypercholesterolemia who cannot achieve targets with standard therapy.
PURPOSE OF REVIEW: Familial hypercholesterolemia is a genetic disorder of defective clearance and subsequent increase in serum LDL cholesterol (LDL-C) with a resultant increased risk of premature atherosclerotic cardiovascular disease. Despite treatment with traditional lipid-lowering therapies (LLT), most patients with familial hypercholesterolemia are unable to achieve target LDL-C. We review current and future novel therapeutic options available for familial hypercholesterolemia. RECENT FINDINGS: The use of proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors are effective in lowering LDL-C in patients with familial hypercholesterolemia, with a reduction in LDL-C of 60% in heterozygous familial hypercholesterolemia (HeFH) and up to 35% in homozygous familial hypercholesterolemia (HoFH). Inclisiran, another novel agent, is a small-interfering ribonucleic acid that reduces hepatic production of PCSK9 to provide a prolonged and sustained reduction in LDL-C of nearly 50% in HeFH. However, both agents require LDL receptor (LDLR) activity. Evinacumab, a novel monoclonal antibody against angiopoetin-like 3 (ANGPTL3), reduces LDL-C by 50% independent of LDLR activity. SUMMARY: Achieving a target LDL-C in familial hypercholesterolemia can be challenging with standard LLT; however, novel therapeutic modalities show remarkable reductions in LDL-C allowing nearly all patients with HeFH and a significant proportion of patients with HoFH to achieve acceptable LDL-C levels.
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Mohamed et al. (2020) conducted a review in Familial hypercholesterolemia. Novel lipid-lowering therapies (PCSK9 inhibitors, inclisiran, evinacumab) was evaluated on LDL cholesterol reduction. Novel therapies including PCSK9 inhibitors, inclisiran, and evinacumab provide substantial LDL-C reductions of 35% to 60% in patients with familial hypercholesterolemia.
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