Real-world use of bempedoic acid in women and men: pooled 1-year follow-up data from the observational, prospective, real-world MILOS study in Austria, Belgium, Germany, Italy, and the United Kingdom

Background/Introduction

Cardiovascular (CV) disease is frequently underdiagnosed and undertreated in women, who remain underrepresented in CV trials. Additional real-world data are needed to better characterise treatment differences between sexes.

Purpose

This pooled analysis of data from the Austrian, Belgian, German, Italian and United Kingdom cohorts of the MILOS study aimed to evaluate sex-based differences in patient characteristics, lipid-lowering therapy (LLT) use, low-density lipoprotein cholesterol (LDL-C) and adverse drug reactions (ADRs) over 1 year of treatment with bempedoic acid (BA) or the BA and ezetimibe fixed-dose combination (FDC).

Methods

MILOS is an ongoing, European, prospective, observational study evaluating the effectiveness and safety of BA/FDC in adults with hypercholesterolaemia or mixed dyslipidaemia.

Results

Among 3138 patients enrolled, 40.7% (n=1276) were women and 59.3% (n=1862) were men. A higher proportion of women than men had heterozygous familial hypercholesterolaemia (HeFH; 15.2% vs 9.3%), were treated for primary prevention (51.3% vs 26.3%) and were classified by the investigator as having low/moderate (21.4% vs 9.3%) or high (34.7% vs 24.7%) CV risk. In contrast, a higher proportion of men than women were classified as having very high CV risk (65.9% vs 43.1%). At BA/FDC initiation, most women (77.6%) and men (84.6%) received BA in combination with other LLTs. BA was initiated with a statin, with or without ezetimibe, in 27.7% of women and 43.9% of men (Table).

Overall, 2071 patients (40.4% [n=837] women, 59.6% [n=1234] men) had LDL-C data available before BA/FDC initiation and at 1 year. Before BA/FDC initiation, mean (standard deviation [SD]) LDL-C was 130.5 (50.9) mg/dL in women and 105.5 (43.5) mg/dL in men; at 1 year, mean (SD) LDL-C was 82.5 (37.3) mg/dL in women and 72.8 (32.4) mg/dL in men. This corresponded to a mean (SD) relative reduction of 31.0% (34.3) in women and 25.3% (34.0) in men (Figure). At 1 year, there was an absolute increase in goal attainment of approximately 31% in both women and men. Overall, 10.3% (131/1276) of women and 8.3% (155/1862) of men reported ADRs considered causally related to BA/FDC by the investigator, most commonly myalgia in both women (3.8%) and men (1.6%).

Conclusions

In this real-world dataset from five European countries, women receiving bempedoic acid were more often diagnosed with HeFH, treated for primary prevention, and considered at lower CV risk than men. Bempedoic acid was predominantly used in combination with other LLTs in both sexes; however, women less often received bempedoic acid with a statin. Bempedoic acid, with or without other LLTs, was associated with a >25% reduction in LDL-C in both sexes. ADRs were similar between women and men, with no new safety signals identified. These findings are consistent with evidence from clinical trials and support the use of bempedoic acid in both sexes.