The effectiveness of non-myeloablative stem cell transplantation and DLI as a selective treatment in class III ß-thalassemic patients

At present, the only curative treatment for β-thalassemia major is allogenic bone marrow transplantation accompanied with considerable mortality and morbidity in class III β-thalassemia. Regarding few case reports on successful non-myeloablative stem cell transplantation in class III β-thalassemic cases, we evaluated the effectiveness of this type of allogenic stem cell transplantation, considering less toxic non-myeloablative conditioning regimen. In this prospective study in Shariati Hospital bone marrow transplantation center during 2001-3, 13 class III β-thalassemia patients (on the basis of history and physical examination and liver biopsy) were transplanted with peripheral blood and bone marrow stem cells from their HLA-identical siblings. Non-myeloablative conditioning regimen included fludarabine; busulan; antithymocyte globulin. Graft versus host disease (GVHD) prophylactic regimen was cyclosporin and metothrexate. In the case of the declining chimerism, the patients were treated with donor lymphocyte infusions (DLI). The conditioning regimen was tolerated well without any considerable toxicity in hematologic, gastrointestinal and pulmonary systems. Five (38.5%) patients had acute and 2(15.4%) had chronic GVHD. Two patients died after transplantation. While two cases had a thalassemia-free survival. Although associated with high graft failure and the recurrence of disease, non-myeloablative stem cell transplantation may be used as a curative, less toxic post-transplantation treatment for class III ß-thalassemia.