Hematopoietic Stem Cell Transplantation For Major Thalassemia: Nineteen Years Experience In Iran
Biology of Blood and Marrow Transplantation
Objective: Hematopoietic Stem Cell Transplantation (HSCT) still remains the only cure available for Major Thalassemia. Below there are the results of 19 years using HSCT as treatment in our centre. Patients and Methods: 425 Major Thalassemia Patients, (242 male and 183 female) with a median age of 7 (Range: 2-28 years), have received HSCT from July 1991 through October, 2009. From 425 patients, 113 patients were in class I, 146 in class II, 142 in class III and 24 in Thalassemia Intermediate.
... mia Intermediate. They received allogeneic transplantation from 407 HLA full matched siblings, nine HLA mismatched siblings or other relatives, nine HLA full matched other relatives. The sources of HSCT were 158 bone marrow, 194 peripheral blood, 9 cord blood and one bone marrow combined with peripheral blood. Since three years ago 58 patients with class III thalassemia were randomized for double blind study with two groups; one with co-transplantation (mesenchymal cells) and another without mesenchymal cells. Results: The median time to Absolute Neutrophil Count $0.5 Â 10 9 / L and platelet count $20 Â 10 9 /L were +15 and +22, respectively. Acute GvHD occurred in 281 (66%) and chronic GvHD in 91 (21.4%) patients. 356 (83.8%) patients are still living and 69 (16.2%) are deceased. The most common cause of death was GvHD. 5-year overall survival (OS) and disease-free survival (DFS) were 79.8% and 70.2%, respectively. The 5-year OS of patients with peripheral blood and bone marrow stem cell were 75.5% and 84.2%, respectively (p 5 .141). The 5-year DFS of peripheral blood and bone marrow recipients were 69.3% and 73.1%, respectively (p 5 .773). Up to now the results of HSCT for both groups in double blind randomized study is same and we have to wait for future analysis. Conclusion: HSCT, based on our experience and other documented studies, is an acceptable treatment for Major Thalassemia with better results in younger patients.