Treatment target achievement in patients with familial hypercholesterolemia: A real-world descriptive study.

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.