Document resource
Background and Aim In randomised trials, bempedoic acid (BA), the first-in-class adenosine triphosphate-citrate lyase inhibitor, reduced low-density lipoprotein cholesterol (LDL-C), and risk of cardiovascular (CV) events. We aim to report real-world data on BA use.Methods MILOS ( NCT04579367) is an ongoing prospective, observational study evaluating the effectiveness and safety of BA or BA+ezetimibe fixed-dose combination (BA+EZE-FDC) in adults with hypercholesterolaemia or mixed dyslipidaemia. We present 1-year follow-up data from the United Kingdom (UK) cohort enrolled between March 2023 and February 2024.Results Of 239 patients enrolled, 178 completed the 1-year follow-up and 99 had LDL-C data available before BA initiation and at 1-year follow-up. Mean±SD age was 65.6±10.3 years, 52.3% were female, 23.0% had diabetes and 14.6% had heterozygous familial hypercholesterolemia. Most (69.0%) patients were primary prevention, with 38.9% and 20.1% classified as high and very-high CV risk, respectively ( tables 1 and 2). At 1-year, 78.1% patients were receiving BA in combination with other lipid-lowering therapies (LLTs). Mean±SD LDL-C reduction from BA or BA+EZE-FDC initiation to 8-weeks (n=66) was 32.5±29.3%. Over the 1-year follow-up (mean±SD treatment duration: 495.8±243.9 days), mean±SD LDL-C decreased from 3.6±1.3 mmol/L (138.2±50.6 mg/dL) before BA/BA+EZE-FDC initiation to 2.3±1.1 mmol/L (89.1±41.6 mg/dL) at 1-year, corresponding to a mean±SD relative LDL-C reduction of 32.5±27.9%. At 1-year, LDL-C decreased across all CV risk categories (mean relative reductions: very-high, 41.5%; high, 31.5%; low/moderate, 27.7%; figure 1). Overall, ESC/EAS LDL-C goal attainment increased from 10.3% before BA/BA+EZE-FDC initiation to 42.4% at 1-year. In total, 34 patients experienced 63 adverse drug reactions (ADRs). Most frequently reported ADRs were myalgia (4.2%, n=10) and muscle spasm (1.7%, n=4), with no serious adverse events or any new safety signals reported.Conclusions In UK clinical practice, BA or BA+EZE-FDC, with or without other LLTs, was associated with early and sustained LDL-C reductions along with a marked increase in the proportion of patients achieving their ESC/EAS LDL-C goals. The safety profile of BA/BA+EZE-FDC was consistent with the Phase III clinical trials.Abstract 159 Figure 1Mean relative LDL-C reductions from before BA/BA+EZE-FDC initiation to year-1 in the overall population and across cardiovascular risk categories as classified by investigator at baselineReduction above each bar represents mean relative LDL-C reduction in corresponding populations. Before BA/BA+EZE-FDC initiation LDL-C value refers to the most recent LDL-C value available ≤1 year before starting BA. The horizontal line in the box depicts median, the marker within the box depicts mean, and the whiskers represent the 1.5×IQR. The dots outside the box represent outliers.BA, bempedoic acid; EZE, ezetimibe; FDC, fixed dose combination; IQR, inter-quartile range LDL, low density lipoproteiAbstract 159 Table 1Baseline characteristics of patients from the UK cohortCharacteristicsAll Patients (N=239)Age, years, mean±SD65.6±10.3Female, n (%)120 (52.3%)Body mass index, kg/m2, mean±SD 28.7±4.6LDL-C, mean±SDammol/L,3.6±1.3 Hypertension, n (%)117 (49.0%)HeFH, n (%)b35 (14.6%)Diabetes mellitus, n (%)c55 (23.0%)aLDL-C values before start of BA / BA+EZE-FDC, presented for patients who also had LDL-C values at 1-year (n=99). Before BA initiation LDL-C value refers to the most recent LDL-C value available ≤1 year before starting BA. bData missing for 51 patients. cData missing for 14 patients.BA, bempedoic acid; BA+EZE-FDC. Bempedoic acid/ezetimibe fixed-dose combination; HeFH, heterozygous familial hypercholesterolaemia; LDL-C, low-density lipoprotein cholesterol; SD, standard deviation.Abstract 159 Table 2Baseline prevention category and risk classification from the UK cohortCharacteristicsAll Patients (N=239)Type of prevention, n (%) Primary prevention165 (69.0%)Secondary prevention74 (31.0%)CV risk classification by investigator, n (%)a Low17 (7.1%)Moderate71 (29.7%)High93 (38.9%)Very high48 (20.1%)aData missing for 10 patients.CV, cardovascular