BONE-MARROW TRANSPLANTATION FOR THALASSEMIA IN THAILAND

We report our experience of bone marrow transplantation for thalassemi a in Thailand. From July 1988 to September 1992, 10 thalassemic patien ts 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 co nditioning regimen comprised busulfan 14 mg/kg and cyclophosphamide 20 0 mg/kg. For GVHD prophylaxis, either cyclosporine alone or in combina tion with short methotrexate was given. Five patients were alive and w ell 104-1534 days after transplantation. Three patients with severe ma nifestations at the time of transplant had partial engraftment, and lo st their graft within 3 months. They survived with thalassemia 1041-13 57 days after transplantation. One patient who received one antigen mi smatched marrow from her brother had no engraftment and was alive with thalassemia 1429 days posttrans plant. One patient died early on day 9 from CNS complication. No GVHD was observed in this series. These re sults indicate that bone marrow transplantation can cure thalassemia b ut there is still high autologous recovery rate in those with severe m anifestations. In Southeast Asia, both thalassemias and hemoglobinopat hies are prevalent(1,2). Those abnormal genes in various combinations lead to more than 60 different thalassemic syndromes. Of these, homozy gous beta-thalassemia and double heterozygosity between beta-thalassem ia and hemoglobin(Hb)E are the most severe forms in which patients can be born alive. It has been estimated that 600 new cases of homozygous beta-thalassemia and 3000 new cases of beta-thalassemia /Hb E disease are born annually, beta-thalassemia/Hb E disease is more common than homozygous beta-thalassemia in Southeast Asia because of much higher f requencies of Hb E(1,2). At present bone marrow transplantation is the only mode of curative therapy.for thalassemia(3-7). Up to now more th an 500 patients have been successfully transplanted(8). We report here in our experience of bone marrow transplantation for the treatment of thalassemia in Thailand.