Originally published by 2 Minute Medicine® (view original article). Reused on AccessMedicine with permission.

Chronic kidney disease (CKD)-related anemia is associated with significant morbidity, and remains undertreated worldwide. Adequate treatment of CKD-related anemia is associated with reduced transfusion rates and improved clinical outcomes. Roxadustat is a hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitor that stabilizes HIF-alpha subunits, resulting in increased transcriptional activity and functional activation of target genes encoding erythropoietin, erythropoein receptor, heme biosynthesis enzymes, iron absorption and transport proteins. In this phase III trial of Chinese patients with CKD not undergoing dialysis, 154 patients were randomized to receive roxadustat three times a week for 8 weeks or placebo to evaluate the efficacy of roxadustat in the treatment of anemia in this patient population. This was followed by an 18-week open-label phase where all patients received roxadustat, and parenteral iron was held. The primary outcome of interest was mean change from baseline in hemoglobin level, averaged between weeks 7 and 9. Researchers found that the mean change from baseline hemoglobin level was 1.9 g/dL (SD 1.22 g/dL) with roxadustat versus 0.4 g/dL (SD 0.8 g/dL) in the control group (difference 2.2 g/dL, 95% CI 1.9 g/dL to 2.6 g/dL, p<0.001). A hemoglobin response of at least 1.0 g/dL increase from baseline by week 9 was observed in 85 (84%) patients receiving roxadustat and 0 (0%) patients in the control group (difference 84%, 95% CI 75% to 91%). The roxadustat group also demonstrated significantly greater mean reductions from baseline in hepcidin and total cholesterol levels, and more frequent episodes of hyperkalemia and metabolic acidosis. The efficacy of roxadustat was maintained during the 18-week open-label period in terms of improvement and maintenance of hemoglobin. The findings of this study therefore show that roxadustat may be effective in CKD patients not on dialysis in terms of correcting and maintaining hemoglobin levels in the setting of anemia.

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