BONE-MARROW TRANSPLANTATION FOR THALASSEMIA - EXPERIENCE IN PESARO, ITALY

Purpose: We reviewed the results of transplanting allegeneic marrow fr om HLA-identical donors in patients with beta-thaiassemia. Among the 4 84 consecutive patients who have received transplants since 1981, surv ival and disease-free survival rates leveled off at similar to 1 year after transplantation, at 82 and 75%, respectively. Patients and Metho ds: Clinical characteristics of patients before transplant have been s tudied to determine their impact on survival, disease-free survival, a nd graft rejection. By multivariate analysis, portal fibrosis, hepatom egaly, and a history of inadequate chelation therapy were identified a s risk factors. The patients were then divided into three classes of r isk. Results: The rate of prolonged disease-free survival was 98% and 87% for class 1 and class 2 patients. This rate of disease-free surviv al is 70% with the use of our last conditioning protocol for class 3 p atients. Older patients (17-32 years) have a 79% probability of prolon ged disease-free survival. Conclusions: We conclude that for patients with thalassemia major, transplantation of bone marrow from a human le ukocyte antigen-identical donor offers a high probability of disease-f ree survival, particularly for those patients in early stages of their disease.