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.

Conclusions: 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.