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A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
Therapeutic Persistence in the Management of Familial Hypercholesterolemia: A Case of Acquired PCSK9 Inhibitor
1Northwestern University, Feinberg School of Medicine, Chicago, Illinois, USA.
Insights
Familial hypercholesterolemia (FH) management can be challenging, especially with statin intolerance. A novel approach using inclisiran successfully lowered low-density lipoprotein cholesterol in a patient with FH and cardiovascular disease.
Area of Science:
- Cardiology
- Genetics
- Metabolic Disorders
Background:
- Familial hypercholesterolemia (FH) is an inherited lipid metabolism disorder causing high LDL cholesterol and premature cardiovascular disease.
- Managing FH is complex, often requiring persistent treatment adjustments to reach lipid targets, particularly in patients with existing atherosclerotic cardiovascular disease.
Abstract:
Familial hypercholesterolemia (FH) is an inherited disorder of lipid metabolism that causes marked elevations in low-density lipoprotein cholesterol and is associated with premature atherosclerotic cardiovascular disease. A 71-year-old woman with FH, established atherosclerotic cardiovascular disease, and statin intolerance presents to the cardiology clinic to discuss lipid management. Despite having failed combinations of statins, ezetimibe, and 2 proprotein convertase subtilisin/kexin type 9 inhibitors that use monoclonal antibodies, she was able to achieve low-risk low-density lipoprotein cholesterol levels using a novel way to lower proprotein convertase subtilisin/kexin type 9 levels with inclisiran. Therapeutic persistence is a key trait in the management of FH where multiple changes in treatment are often needed to achieve optimal therapeutic targets.
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