Roxadustat in the Treatment of Pure Red Cell Aplasia Associated With Anti-EPO Antibodies

Erythropoiesis-stimulating agents (ESAs) are essential in managing anemia associated with chronic kidney disease (CKD). Although iron deficiency and inflammation are the most frequent causes of ESA hyporesponsiveness, a rare but serious complication is pure red cell aplasia (PRCA) caused by anti-EPO antibodies. This condition results in severe anemia and requires discontinuation of ESA therapy. Roxadustat, a hypoxia-inducible factor prolyl hydroxylase inhibitor, has recently emerged as a promising therapeutic alternative. We report the case of a 72-year-old woman with stage G3bA1 CKD who initially responded to epoetin alfa but developed progressive anemia 11 months later, reaching a hemoglobin level of 5.6 g/dL despite dose escalation. Bone marrow biopsy showed absence of erythroid precursors, and anti-EPO antibodies were detected, confirming PRCA. ESA therapy was stopped, and roxadustat was initiated. Within 7 weeks, the patient achieved transfusion independence with stable hemoglobin levels. Follow-up marrow biopsy demonstrated erythroid recovery, and anti-EPO antibodies became undetectable. ESA-induced PRCA has been linked to subcutaneous administration, formulation changes, and genetic predisposition. Standard treatment includes ESA withdrawal and immunosuppression, although outcomes vary. Roxadustat may restore erythropoiesis without additional immunosuppression, providing both hematologic and immunologic benefit. Further studies are needed to validate its role in this context.