[Bempedoic acid in real-world clinical practice: baseline and 8-week data from the Italian prospective, non-interventional MILOS study].

Abstract

Bempedoic acid (BA), an adenosine triphosphate-citrate lyase inhibitor, effectively reduces LDL cholesterol (LDL-C) and cardiovascular (CV) risk, as demonstrated in randomized clinical trials. However, real-world data on its use in clinical practice remain limited. MILOS (NCT04579367) is a European prospective observational study evaluating the effectiveness and safety of BA, either as monotherapy or in fixed-dose combination with ezetimibe (BA+EZE FDC), in adult patients with primary hypercholesterolemia or mixed dyslipidemia. This article presents interim 8-week follow-up data for Italian patients enrolled between March and December 2023. Of the 1310 patients enrolled, 855 (BA, n = 445; BA+EZE FDC, n = 410) had available LDL-C data at both pre-treatment and 8-week follow-up. The overall mean age was 66.0 ± 10.7 years; heterozygous familial hypercholesterolemia was present in 11.1%, and 15.1% had diabetes. A larger proportion of patients (57.7%) were in secondary prevention, and 82.4% were at high or very high CV risk. Before starting BA or BA+EZE FDC, 33.1% of patients were not receiving lipid-lowering therapy. After an average of 59.3 ± 19.3 days of treatment, a mean LDL-C reduction of 22.6 ± 32.3% was observed, with a median reduction of 25.4% (interquartile range 8.1-43.2), from 2.9 ± 1.2 mmol/l (110.7 ± 44.8 mg/dl) to 2.1 ± 0.8 mmol/l (79.2 ± 32.2 mg/dl), following BA/BA+EZE FDC treatment, with or without background lipid-lowering therapy. The overall proportion of patients achieving LDL-C targets increased from 7.1% (61/855) before treatment to 37.2% (318/855) at 8-week follow-up with a 29.4% increase among patients at very high CV risk. During follow-up, 56 of 1310 patients (4.3%) reported at least one adverse drug reaction, with no unexpected adverse drug reactions observed; this rate is consistent with data reported in the literature. In the Italian cohort of the MILOS study, BA, alone or in BA+EZE FDC, proved to be an effective therapeutic option for managing patients at CV risk, supporting its role in achieving guideline-recommended lipid-lowering goals in real-world clinical practice.