Roxadustat treatment for anemia in peritoneal dialysis patients: A randomized controlled trial.

BACKGROUND/PURPOSE: Roxadustat, a first-in-class hypoxia-inducible factor prolyl hydroxylase inhibitor, promotes erythropoiesis and regulates iron metabolism. This study investigated the efficacy and safety of roxadustat in Chinese patients with anemia on peritoneal dialysis (PD). METHODS: One hundred and twenty-nine patients were randomized and treated with roxadustat (n = 86) or erythropoiesis-stimulating agents (ESAs) (n = 43) for 24 weeks. The primary end points were the mean hemoglobin (Hb) level at week 24, the change in average Hb levels from baseline to week 24, and the cumulative response rate throughout the treatment period. The secondary end points included changes in hepcidin and iron indices and serum lipid levels. Subgroup analysis examined the effect of inflammatory status on the efficacy of Hb. Safety was assessed as the occurrence of emergent adverse events after treatment. RESULTS: The mean average Hb levels at week 24 and average change in Hb levels from baseline to week 24 were 11.5 g/dL and 2.5 g/dL in the roxadustat group and 11.2 g/dL and 2.2 g/dL in the ESAs group, respectively. The cumulative response rate was 96% in the roxadustat group and 92% in the ESAs group at week 24. Roxadustat decreased hepcidin levels and increased total iron-binding capacity. The decreases in total cholesterol and low-density lipoprotein cholesterol were greater with roxadustat than with ESAs. Roxadustat-induced Hb increases were independent of baseline C-reactive protein levels. Common adverse events included hyperkalemia, hypertension, and insomnia. CONCLUSION: Roxadustat effectively corrected and maintained target Hb levels in Chinese PD patients. This trial was registered in the Chinese Clinical Trial Register (ChiCTR2000035054).