Roxadustat After PCI Did Not Reduce Infarct Size in STEMI Trial

Preclinical studies suggest that stabilizing hypoxia-inducible factor may attenuate reperfusion injury. The single-center, open-label phase 2 ROXAMI trial randomized 158 adults undergoing primary PCI for STEMI to oral roxadustat or standard care alone. Roxadustat was started within 2 hours after PCI at 100 mg three times weekly for 2 weeks. The primary outcome was imaging-assessed infarct size as a percentage of the left ventricle at 30 days; follow-up continued for 1 year.

Among 148 patients with evaluable imaging, mean infarct size was 23.3% with roxadustat versus 22.0% with control, a difference of 1.3 percentage points (95% CI, −2.7 to 5.3; P = .51). Major adverse cardiovascular events occurred in 3 of 79 patients receiving roxadustat (3.8%) and 7 of 79 controls (8.9%; hazard ratio, 0.42; 95% CI, 0.11–1.63). Prespecified safety endpoint incidence did not differ between groups.

Multiplicity-unadjusted secondary analyses showed a greater improvement in left ventricular ejection fraction at 12 months with roxadustat, with an adjusted between-group difference of 2.9 percentage points (95% CI, 0.4–5.3). The corresponding difference in left ventricular end-diastolic volume index was −4.5 mL/m² (95% CI, −8.9 to −0.1). These exploratory findings do not establish clinical benefit. Interpretation is limited by the small, single-center, open-label design, incomplete primary-outcome imaging, and lack of multiplicity adjustment for secondary analyses.