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Treatment target achievement in patients with familial hypercholesterolemia: A real-world descriptive study
Simon Faubert1, Martine Paquette1, Alexis Baass2
1Lipids, Nutrition, and Cardiovascular Prevention Clinic of the Montreal Clinical Research Institute, Montreal, Québec, Canada.
Insights
Familial hypercholesterolemia (FH) patients on conventional therapy have low LDL-C target achievement. High-intensity statins improve outcomes, but most FH patients still require more intensive treatment for cardiovascular prevention.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Familial hypercholesterolemia (FH) is a genetic disorder causing high cardiovascular risk.
- Statin and ezetimibe are first-line treatments for FH, but real-world target achievement is understudied in Quebec.
Purpose of the Study:
- To assess the proportion of FH patients in primary prevention achieving LDL-C targets without PCSK9 inhibitors.
- To evaluate the effectiveness of conventional lipid-lowering therapies in FH.
Main Methods:
- Retrospective study at the Montreal Clinical Research Institute's Lipid Clinic.
- Included 225 patients with molecularly defined or clinically diagnosed FH.
- Analyzed LDL-C target attainment rates based on treatment intensity.
Main Results:
- Only one-third of FH patients reached the LDL-C target of <2.5 mmol/L.
- Patients on high-intensity statins were twice as likely to achieve this target.
- No significant difference in target achievement between men and women.
Conclusions:
- Conventional lipid-lowering treatments show unacceptably low target achievement rates in FH patients.
- Current therapies may be insufficient for adequate cardiovascular prevention in most FH cases.
- Further research into intensified treatment strategies for FH is warranted.
Background:
Statin and ezetimibe represent the first line of lipid-lowering therapy in patients with familial hypercholesterolemia (FH), a disease associated with a strong cardiovascular risk. The current low-density lipoprotein cholesterol (LDL-C) target achievement rate in a real-world context using these conventional treatments has never been investigated in the Province of Quebec (Canada).
Objective:
The primary objective of this study was to evaluate the proportion of FH patients in primary cardiovascular prevention who attained their recommended LDL-C threshold without being treated with a PCSK9 inhibitor.
Methods:
Patients included in this retrospective study were followed at the Lipid Clinic of the Montreal Clinical Research Institute. All patients were molecularly defined (97%) or had a definite clinical diagnosis of FH.
Results:
A total of 225 patients were included in this study, of which 73% were on high-intensity statin therapy. While two-thirds of the cohort achieved the LDL-C treatment target of ≥ 50% reduction from baseline, only one third attained the target of < 2.5 mmol/L (<97 mg/dL). However, patients on high-intensity statin therapy were two times more likely to achieve the < 2.5 mmol/L targets as compared to those treated with low or moderate statin intensity (p = 0.01). There was no significant difference in treatment target achievement between men and women.
Conclusion:
Target achievement rate was unacceptably low in our FH patients. Conventional lipid-lowering treatments alone may not be sufficient in most FH patients to ensure adequate cardiovascular prevention.
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