Hematopoietic stem cell transplantation for beta-thalassemia major: experience in south of Iran.

Introduction: Allogeneic stem cell transplantation as a curative treatment for thalassemia major was established in Shiraz in 1993. In this article we describe our results of 10 years experience with allogeneic bone marrow transplantation for thalassemia major.
Methods: From June 1993 to January 2003, 112 cases of beta-thalassemia major underwent allogeneic marrow transplantation from HLA-identical or one antigen-mismatched related donors. Conditioning chemotherapy included busulfan (14 to 15 mg/kg), cyclophosphamide (200 mg/kg), and antithymocyte globulin (ATG; 40 mg/kg). Prophylaxis for graft-versus-host disease consisted of cyclosporine, prednisolone, and methotrexate.
Results: One hundred twelve patients with a diagnosis of beta-thalassemia major underwent allogeneic marrow transplantation during this period. The mean age of the patients was 9.5 years with the range of 2 to 20 years. The distribution of cases according to the Lucarelli classification were: 27 cases class I, 38 cases class II, and 47 cases class III. Eighty-seven of 112 patients (77.6%) with diagnosis of beta-thalassemia major are living with full engraftment at a median follow-up of 6 years (range 2 to 119 months).
Conclusion: Allogeneic bone marrow transplantation has changed the outcome of disease dramatically. According to our results stem cell transplantation is the treatment of choice for class I and II (Lucarelli risk groups). Also, we recommend transplantation as a curative method for treatment of class III beta-thalassemic patients.