Background: Bempedoic acid (BA) is a first-in-class ATP citrate lyase inhibitor that has been shown in the phase III CLEAR clinical trial programme to lower low-density lipoprotein cholesterol (LDL-C) levels. However, real-world data with BA in clinical practice are limited.

Methods: MILOS is a European, prospective, observational, non-interventional study (NCT04579367) evaluating the real-world use of BA and/or BA + ezetimibe (EZE) fixed-dose combination (FDC) in adult patients. The main study objectives were to characterise the LDL-C lowering effectiveness and safety of BA and/or BA+EZE FDC in real-world clinical settings in adult patients with primary hypercholesterolaemia or mixed dyslipidaemia. After baseline measurements, patients will be followed up for a minimum of 1 year and maximum of 2 years. In this interim analysis, based on a snapshot taken on 11 May 2022, we summarise the baseline characteristics of the German cohort of the study. The number of sites involved was 126 and patients were enrolled between January 2021 and May 2022.

Results: Of the 1004 patients enrolled, 731 patients with complete data were included in this analysis; 95% were recruited by specialists (and 5% by general practitioners). Physicians were predominantly office-based (84.3%) vs 15.7% from hospital settings. A similar proportion of patients were initiated with BA (53.2%) or BA+EZE FDC (46.8%) treatment. Patients treated with BA+EZE FDC had lower mean LDL-C concentrations than those treated with BA (3.28 mmol/L vs 3.06 mmol/L, respectively; Table 1). Apart from this, there were no other notable differences in baseline characteristics between the two groups of patients (Table 1). The overall mean (standard deviation) age was 64.7 (10.1) years, with more male patients than female (60.9% vs 39.1%, respectively). The majority of patients treated with BA or BA+EZE FDC were in secondary prevention (80% vs 20% primary prevention; Table 1). 

Overall, 24.2% of BA was given as monotherapy with no other lipid-lowering therapies (LLTs), whereas 75.8% of BA was prescribed in combination with other LLTs (Table 2). 24.9% of patients had BA in dual combination therapy with EZE, given either as separate pills or as a FDC. 27.1% of patients were on triple therapy with BA+EZE+statin, also given either as separate pills or as a FDC. Additionally, BA and/or BA+EZE FDC were used in combination with proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) in 5.3% of patients. 

Conclusion: This baseline analysis of the MILOS study in a cohort of patients in Germany shows that BA is being used in routine practice mainly in combination with other oral LLTs including EZE and statins in very high-risk patients who require further reductions in their LDL-C levels.