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A High-Throughput Luciferase Assay to Evaluate Proteolysis of the Single-Turnover Protease PCSK9
Published on: August 28, 2018
PCSK9 inhibitors: A new improvement for health
1Servicio de Endocrinología y Nutrición, Hospital Clínico Universitario de Valencia, Valencia, España.
Insights
This case study discusses a patient with heterozygous familial hypercholesterolemia and a history of heart attack. LDL-apheresis was used for 8 years, and PCSK9 inhibitors offer a new treatment option.
Area of Science:
- Cardiology
- Metabolic Disorders
- Pharmacology
Background:
- Heterozygous familial hypercholesterolemia (HeFH) is a genetic disorder characterized by high cholesterol levels.
- Patients with HeFH have an increased risk of premature cardiovascular events, such as acute myocardial infarction.
- Effective lipid-lowering therapy is crucial for managing HeFH and preventing complications.
Observation:
- The patient presented with HeFH and a history of acute myocardial infarction.
- Oral lipid-lowering medications were insufficient to achieve therapeutic goals.
- LDL-apheresis was initiated as a fortnightly treatment for 8 years.
Findings:
- Despite long-term LDL-apheresis, the patient's lipid profile may still require optimization.
- The advent of PCSK9 inhibitors presents a novel therapeutic avenue for managing refractory hypercholesterolemia.
- This case highlights the evolving treatment landscape for complex lipid disorders.
Implications:
- PCSK9 inhibitors offer a potentially more convenient and effective alternative to LDL-apheresis for select patients.
- This case underscores the importance of personalized treatment strategies in managing familial hypercholesterolemia.
- Further research is warranted to evaluate the long-term efficacy and safety of PCSK9 inhibitors in HeFH patients previously treated with apheresis.
Abstract:
It is a patient with heterozygous familial hypercholesterolemia and a personal history of acute myocardial infarction, which is referred to our lipid unit for hypocholesterolemic treatment adjustment. Since he does not reach therapeutic goals with oral medication, he starts a treatment with fortnightly sessions of LDL-apheresis, which he keeps for 8 years. With the introduction and availability of PCSK9 inhibitors, a new treatment option is possible for this patient.
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