Real-world use of bempedoic acid for hypercholesterolaemia: a German multicentre, retrospective, cohort study

Abstract Background Dyslipidaemia remains a leading cause of death in Germany despite an improving therapeutic landscape, with many patients not achieving guideline-recommended low-density lipoprotein cholesterol (LDL-C) goals. Bempedoic acid, an adenosine triphosphate citrate lyase inhibitor, is a recent addition to the therapeutic landscape with proven efficacy in randomised controlled trials; however, its use varies between centres. We aimed to assess the short-term, real-world effectiveness of bempedoic acid in lowering LDL-C levels in patients in Germany. Methods This retrospective, observational study was conducted using data from patients with primary hypercholesterolaemia or mixed dyslipidaemia, collected before and approximately 3 months after initiating bempedoic acid in 7 specialised lipid outpatient clinics in Germany. Results Data were analysed from a total of 641 patients, with a mean age (standard deviation [SD]) of 63.2 (11.3) years. The addition of bempedoic acid significantly reduced LDL-C levels from a mean of 133.6 mg/dL at baseline to 101.8 mg/dL after 3 months (p < 0.001). The mean within-patient LDL-C reduction was 31.8 mg/dL and the mean patient-level percentage change was -17.5%. Mean patient-level changes were -4.9% for HDL-C and -12.0% for total cholesterol; triglycerides did not change significantly. At 3 months, patients not receiving any lipid-lowering therapy (LLT) other than bempedoic acid had a mean patient-level LDL-C reduction of 27.1%; corresponding mean reductions were 23.7% with ezetimibe, 20.3% with PCSK9 inhibitor therapy, 9.0% with statin therapy and 6.1% with statin plus ezetimibe. Suspected adverse events were documented in 218/641 patients (34.0%), and 171/641 (26.7%) had discontinued bempedoic acid by follow-up. Myalgia was the most commonly reported adverse event in patients discontinuing treatment (75 cases). Conclusions Findings from this real-world, multicentre study conducted in Germany show that bempedoic acid may present an effective treatment option for patients with hypercholesterolaemia who are already receiving LLT, in addition to being an effective option for patients not receiving/tolerating other LLTs. The magnitude of LDL-C reduction differed across background treatment strategies, while suspected adverse events and treatment discontinuation were common in this specialist-care cohort.