Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Congenital Dyserythropoietic Anemia Combined with Thalassemia: A Report of Two Cases
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Congenital dyserythropoietic anemia (CDA) comprises a heterogeneous group of rare hereditary disorders characterized by ineffective erythropoiesis and often presents with clinical features that overlap with thalassemia. Hematopoietic stem cell transplantation (HSCT) remains the only definitive curative intervention for CDA; however, experience with haploidentical HSCT in this population is limited, and the procedure is associated with considerable challenges. We report two pediatric cases of CDA coexisting with thalassemia who underwent haploidentical related donor HSCT using a novel conditioning regimen comprising three alkylating agents. This was combined with graft-versus-host disease prophylaxis utilizing posttransplant cyclophosphamide and anti-thymocyte globulin. Both patients achieved sustained engraftment, transfusion independence, and remained free of severe transplant-related complications. These cases demonstrate the feasibility and therapeutic potential of haploidentical HSCT for patients with CDA, even in the context of concomitant thalassemia.
