Results of therapy for beta-thalassemia major.

During the last 10 years, 92 transfusion-dependent beta-thalassemia patients have been encountered at the National Taiwan University Hospital and Provincial Taoyuan General Hospital. Seventy-seven of them were followed up regularly. Long-term results of conventional therapy in 63 cases and allogeneic bone marrow transplantation (BMT) in 14 cases are reported. The conventional therapy included regular red cell transfusion and desferrioxamine iron chelation therapy. Preliminary results of conventional therapy showed a mortality of 7/63 (11%). Of those who were alive, the morbidity was 56/56 (100%). There was no disease-free-survival (0/56; 0%). BMT was performed after preparatory regimens of busulfan, cyclophosphamide, and/or total body or lymphoid irradiation. Preliminary results of BMT showed a mortality of 5/14 (36%). For those who were alive, the morbidity was 3/9 (33%), and the disease-free-survival rate was 6/9 (67%) during a follow-up period of three to six years. It is concluded that the only way to cure beta-thalassemia major at present is BMT. However, the risks of BMT and donor non-availability make conventional therapy unavoidable. Further study is needed to decrease the risk of BMT and to improve the efficacy of conventional therapy.