Rom J Intern Med. 2026 Oct 26. doi: 10.2478/rjim-2026-0025. Online ahead of print.
ABSTRACT
INTRODUCTION: Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors emerged as an effective treatment for hypercholesterolemia in clinical trials, however real-world evidence shows considerable heterogeneity. We aimed to assess the effectiveness and safety of injectable PCSK9 inhibitors in real-world patients.
METHODS: Our retrospective analysis included 45 consecutive adults, 18 (40%) men, mean age 57.4 years, inadequately controlled on, or intolerant to, statins and/or ezetimibe, and newly initiated on alirocumab 75 mg or 150 mg, evolocumab 140 mg, or inclisiran 284 mg. Biochemical parameters and adverse events were recorded at baseline and when LDL-C reached the risk-specific absolute threshold or the lowest LDL-C closest to it after starting PCSK9 inhibitors (<55 mg/dL very high, <70 mg/dL high cardiovascular risk, per European Society of Cardiology). LDL-C was also assessed at standardized time points (3, 6, 12, 18, 24 months).
RESULTS: At 3 and 6 months, LDL-C decreased by a median of 54.4% and 59.2% (both p < 0.001), with 52.4% and 50.0% of patients below threshold. The best LDL-C reduction was 66.9% (from 145 mg/dL to 50 mg/dL; p < 0.001) at a median time to first documented threshold attainment of 5 months. In an exploratory analysis, no significant association was detected between LDL-C reduction and sex, diabetes, statin intensity or ezetimibe use. No clinically relevant hepatic or muscular toxicity was attributed to PCSK9 inhibitors.
CONCLUSION: In real-world patients aiming to achieve the absolute LDL-C threshold on PCSK9 inhibitors, treatment was associated with marked LDL-C reduction after a median of 5 months.
PMID:42928644 | DOI:10.2478/rjim-2026-0025