246: Allogeneic blood and marrow transplantation in thalassemia major class 3: An experience of Iran

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Objective: Our aim for this study was to describe the outcome of blood and marrow transplantation in patients with class 3 Thalassemia major. Methods: Since December 1992 till september 2006, fifty-two patients with Thalassemia class 3 received blood and marrow transplantation from their Human Leukocyte Antigen(HLA)-identical siblings. Thirty-two patients received bone marrow and twenty patients received peripheral blood stem cell transplantation. Conditioning regimen in 47 patients was Cyclophosphamide 40 mg/kg/day (from day–5 to -2) and Busulfan 3.5 mg/kg/day (from day –9 to -6) and in these patients Graft Versus Host Disease (GVHD) prophylaxis regimen was Cyclosporine.A (CY.A) 1.5mg/kg /day/IV (day–3), then 3mg/kg/day IV (days +7, +11), then 12.5 mg/kg/day/PO and Methotrexate 10mg/m2 (day +1), 6mg/m2(days +3, +6). Conditioning regimen in 5 patients was Fludarabin 40 mg/m2 (from –6 to –2) and Busulfan 4 mg/kg (from –5 to –2) and in this patients GVHD prophylaxis regimen was (CY.A) 3 mg/kg/day IV (days –3 and +7), then 12.5 mg/kg/day PO. Results: Median age at time of transplantation was 8.5 years (age range: 1-26), Male/Female: 21/31.Median time of absolute neutrophil count ≥ 0.5×109/L was +14 and Median time of platelet recovery ≥20×109/L was +25. At present 42 out of 52 are alive and 10 patients died due to acute GVHD, chronic GVHD, rejection, veno-occlusive disease, infection and the others. Thirty-four patients (64%) developed acute GVHD (grade I = 13, grade II = 7, grade III= 10, grade IV= 4). Three patients (5.8%) developed chronic GVHD (limited= 2, extensive= 1). 8-year disease free survival were 73.17%. 8-year overall survival were 79.85%. Conclusion: According to this study, for an acceptable outcome in Thalassemia class 3We need better conditioning and GVHD prophylaxis regimens to decrease cardiopulmonary and liver complications. The results of blood and marrow transplantation showed that it is better than supportive therapy such as transfusion and desferal therapy.