Effectiveness, safety, and treatment persistence of bempedoic acid in a Spanish real-world cohort

BACKGROUND Bempedoic acid (BA) is an oral adenosine triphosphate-citrate lyase inhibitor that reduces low-density lipoprotein cholesterol (LDL-C) and is particularly relevant in patients with statin intolerance. However, real-world evidence on its effectiveness and safety remains limited. OBJECTIVE To assess the effectiveness, safety, and treatment persistence of BA in a multicenter Spanish real-world cohort. METHODS This retrospective observational study included 814 patients treated with BA across 21 vascular risk units in Spain. Lipid and laboratory parameters were evaluated at baseline and after 3 months. Generalized mixed models were used to estimate adjusted marginal means and treatment effects. RESULTS BA significantly reduced total cholesterol (207.4-167.8 mg/dL; -19.1%) and LDL-C (121.3-86.5 mg/dL; -28.8%) (both P < .001). Smaller reductions were observed for high-density lipoprotein cholesterol (-3.9%) and triglycerides (-2.4%), while high-sensitivity C-reactive protein decreased nonsignificantly (-13.7%; P = .102). Uric acid increased (+11.4%; P < .001). LDL-C reduction was consistent across subgroups, with greater absolute reductions in patients with statin intolerance (-43.2 vs -26.7 mg/dL; P for interaction = .046). Concomitant ezetimibe use was associated with numerically greater LDL-C reduction without significant interaction. Patients receiving proprotein convertase subtilisin/kexin type 9 inhibitors or inclisiran achieved lower LDL-C levels and greater reductions. The discontinuation rate was 4.18%, mainly because of gastrointestinal symptoms and myalgias. CONCLUSION In routine clinical practice, BA is an effective and generally well-tolerated lipid-lowering therapy, particularly in patients with statin intolerance or requiring combination treatment. These findings support its use as an effective lipid-lowering option in patients with statin intolerance or requiring combination therapy.