Treatment of pure red cell aplasia in a chronic kidney disease patient with roxadustat: A case report.

Abstract

Pure red cell aplasia (PRCA) is a rare blood disorder that is characterized by severe hypo-erythroid bone marrow hypoplasia leading to severe anemia that usually does not respond to standard treatment. In patients with chronic kidney disease (CKD), especially in those patients who are erythropoiesis-stimulating agent (ESA) resistant, management can be quite difficult. In this case report, we will present a 67-year-old woman with CKD (not on dialysis) who presented with refractory anemia (hemoglobin 45-65 g/L) and was ultimately diagnosed with PRCA after bone marrow aspirate. This patient had previously received treatment (ESAs, iron supplementation, and multiple blood transfusions) without improvement in her blood counts. After initiation of oral Roxadustat 100 mg t.i.w., her hemoglobin increased gradually and stabilized between 100 and 106 g/L, and she no longer required blood transfusions. This case report highlights the potential role of Roxadustat as a new therapeutic option for PRCA and ESA-resistant CKD. While these data are encouraging, larger controlled studies are going to be required to evaluate measures of efficacy, dosing, and long-term safety in this population.