Efficacy and safety of low-dose atorvastatin plus ezetimibe for primary hypercholesterolemia: A randomized, double-blind, multicenter phase 3 trial

Abstract

Studies have suggested that low-dose statin monotherapy may be insufficient for target LDL-C levels. In this randomized, double-blind, multicenter phase 3 trial, we evaluated the efficacy of combined ezetimibe and low-dose atorvastatin in 222 Korean patients with primary hypercholesterolemia. Participants received either 10-mg ezetimibe/5-mg atorvastatin (EZE10/ATV5), 10-mg ezetimibe (EZE10), 5-mg atorvastatin (ATV5), or 10-mg atorvastatin (ATV10). At 8 weeks, EZE10/ATV5 achieved the greatest LDL-C reduction (-44.8%) compared with EZE10 (-12.7%, p < 0.0001), ATV5 (-27.3%, p < 0.0001), and ATV10 (-32.0%, p = 0.0012). The combination therapy showed the highest LDL-C goal achievement rate (41.1% vs. EZE10 8.9%, p < 0.0001; ATV5 10.9%, p < 0.0001; ATV10 27.3%, p = 0.0342), particularly in moderate to high-risk patients. Additionally, EZE10/ATV5 had the lowest adverse events among all groups (6.9% vs. 15.0%, 12.3%, and 27.6%, p = 0.017), with most events being mild. These findings suggest that the combination of ezetimibe and low-dose atorvastatin provides superior lipid-lowering efficacy with an improved safety profile, offering an effective treatment for primary hypercholesterolemia in Korean patients.

Keywords: LDL; atorvastatin; clinical trial; ezetimibe; hypercholesterolemia; lipoprotein; phase III.