Long-term real-world effectiveness and tolerability of alirocumab in high-risk patients with uncontrolled ldl-cholesterol

Background/Introduction Despite maximally tolerated statin therapy, many high-risk patients fail to achieve LDL-C targets. Real-world data on long-term effectiveness of PCSK9 inhibition with alirocumab remain limited. Purpose To evaluate long-term LDL-C reduction, lipid target attainment, tolerability, and cardiovascular outcomes in patients treated with alirocumab in routine clinical practice. Methods A retrospective observational study was performed in patients who initiated alirocumab for either statin intolerance or inadequately controlled LDL-C despite statin ± ezetimibe. Clinical characteristics, lipid trends, dosing patterns, adherence, tolerability, and cardiovascular events were analysed. LDL-C percentage change from baseline was the primary endpoint. Results Seventy-eight patients were included; 33% had statin intolerance and 67% had uncontrolled LDL-C. Alirocumab was administered at 75 mg in 46% and 150 mg in 54%. Median follow-up was 45 months. Overall LDL-C reduction was 63.9%. Reduction was 56.8% in the statin-intolerant group and 69.2% in patients with uncontrolled LDL-C. LDL-C reductions remained stable throughout prolonged follow-up. Treatment was well tolerated, with high adherence and no significant adverse events reported. Nine patients (11.5%) presented cardiovascular events: 2 heart failure admissions, 1 myocardial infarction, and 6 cases of unstable angina requiring revascularisation. Conclusions Alirocumab demonstrated sustained LDL-C reduction over long-term follow-up in real-world patients with statin intolerance or insufficient LDL-C control. Its favourable tolerability and adherence profile, along with event rates consistent with very-high-risk populations, support its role as an effective strategy for lipid optimisation in clinical practice.