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Published on: September 15, 2018
Attainment of Recommended Lipid Targets in Patients With Familial Hypercholesterolemia: Real-World Experience With
Omar Razek1, Lubormira Cermakova2, Hamidreza Armani2
1Department of Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
Insights
PCSK9 inhibitors significantly improved lipid control in Familial Hypercholesterolemia (FH) patients. More patients achieved LDL-C goals with PCSK9 inhibitors compared to those without, demonstrating real-world effectiveness.
Area of Science:
- Cardiovascular Medicine
- Genetics
- Pharmacology
Background:
- Familial Hypercholesterolemia (FH) is a common genetic disorder causing high LDL-C and increased cardiovascular risk.
- Current lipid-lowering therapies often fail to meet treatment targets for FH patients.
- Achieving lipid goals is crucial for preventing atherosclerotic cardiovascular disease in FH.
Purpose of the Study:
- To evaluate the impact of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors on LDL-C levels in FH patients.
- To compare LDL-C reduction and goal attainment in FH patients with and without PCSK9 inhibitor use.
- To assess the real-world effectiveness of PCSK9 inhibitors in managing FH.
Main Methods:
- Retrospective analysis of FH patients from the British Columbia FH Registry (2015-2017).
- Comparison of LDL-C levels before and after PCSK9 inhibitor availability.
- Stratification of patients based on PCSK9 inhibitor use to analyze treatment outcomes.
Main Results:
- Overall LDL-C decreased in the FH cohort from 2015 to 2017.
- Patients using PCSK9 inhibitors showed a significantly greater LDL-C reduction compared to non-users.
- 85.4% of PCSK9 inhibitor users achieved ≥ 50% LDL-C reduction or LDL-C < 2 mmol/L, versus 50.2% of non-users (P < 0.001).
Conclusions:
- PCSK9 inhibitors have improved lipid level control in FH patients.
- Access to PCSK9 inhibitors facilitates achievement of guideline-directed lipid goals.
- Real-world data confirm the effectiveness of PCSK9 inhibitor therapy for FH management.
Background:
Familial hypercholesterolemia (FH) is the most common inherited dyslipidemia and is characterized by elevated low-density lipoprotein cholesterol (LDL-C) and markedly increased risk for atherosclerotic cardiovascular disease. Lipid-lowering therapy is the mainstay of treatment, but few patients with FH are able to achieve commonly recommended lipid targets.
Methods:
We examined changes in LDL-C levels in patients in the British Columbia FH Registry from 2015 to 2017, corresponding to the period immediately before, and the first 2 years after, availability of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors in Canada.
Results:
Among 275 patients with a clinical diagnosis of FH in whom a lipid profile was available between January 1, 2016 and December 31, 2017, 48 had started using a PCSK9 inhibitor. LDL-C decreased in the cohort overall from 2015 to 2017. When patients were stratified according to PCSK9 inhibitor use, the reduction in LDL-C was significantly greater in patients receiving a PCSK9 inhibitor compared with those who did not receive one. Among patients receiving a PCSK9 inhibitor, 85.4% achieved a ≥ 50% reduction in LDL-C or LDL-C < 2 mmol/L, compared with 50.2% of patients not receiving a PCSK9 inhibitor (P < 0.001).
Conclusions:
Our results suggest that control of lipid levels in patients with FH has improved and that the achievement of guideline-directed goals has been facilitated by access to PCSK9 inhibitors. These observations provide insight into the real-world effectiveness of PCSK9 inhibitor therapy in patients with FH.
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