Attainment of Recommended Lipid Targets in Patients With Familial Hypercholesterolemia: Real-World Experience With PCSK9 Inhibitors.

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.