Aplastic anemia is a rare disease that manifests as bone marrow failure. The current treatment options include immunoablative therapy or allogeneic hematopoietic stem cell transplantation. We report a successful immunoablative regimen with autologous umbilical cord blood (auto-UCB) transplant in a 3-year-old boy with severe aplastic anemia.
The immunoablation procedure consisted of 5 × 3.75 mg/kg antithymocyte globulin (Thymoglobulin) (total 18.75 mg/kg), methylprednisolone for 4 days, and cyclosporine A. The patient received auto-UCB containing 0.3 × 10 CD34 cells per kilogram of body weight. Recovery of leukocyte count above 1000/μL was reached on post-transplant day +39, and recovery of granulocytes above 500/μL was reached on day +40. The final regular transfusions of packed red blood cells and platelet concentrate were performed on day +55. The complications that occurred in the post-transplant period were nausea, diarrhea, septic fever, and hepatic abscess formation. Post-transplant immunosuppression with cyclosporine A was discontinued 17.5 months after auto-UCB, and the patient remained in complete remission with normal blood counts and bone marrow morphology.
Auto-UCB transplantation without chemotherapy conditioning can be considered a therapeutic option for children with stored cord blood cells.
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Successful Bone Marrow Recovery After an Immunoablative Regimen With Autologous Cord Blood Transplant in a Child With Idiopathic Severe Aplastic Anemia: A Case Report.
