Bone marrow transplantation for thalassemia in Thailand

We report our experience of bone marrow transplantation for thalassemia in Thailand. From July 1988 to September 1992, 10 thalassemic patients underwent allogeneic bone marrow transplantation. Two of them were homozygous beta-thalassemia and 8 were beta-thalassemia/Hb E disease. Seven were male and 3 female. The age ranged from 1.8-14 years. The conditioning regimen comprised busulfan 14 mg/kg and cyclophosphamide 200 mg/kg. For GVHD prophylaxis, either cyclosporine alone or in combination with short methotrexate was given. Five patients were alive and well 104-1534 days after transplantation. Three patients with severe manifestations at the time of transplant had partial engraftment, and lost their graft within 3 months. They survived with thalassemia 1041-1357 days after transplantation. One patient who received one antigen mismatched marrow from her brother had no engraftment and was alive with thalassemia 1429 days posttransplant. One patient died early on day 9 from CNS complication. No GVHD was observed in this series. These results indicate that bone marrow transplantation can cure thalassemia but there is still high autologous recovery rate in those with severe manifestations.