[ effectiveness of non-myeloablative stem cell transplantation and DLI as a selective treatment in class III beta-thalassemic patients]

ABSTRACT

At present, the only curative treatment for beta-thalassemia major is allogenic bone marrow transplantation accompanied with considerable mortality and morbidity in class III beta-thalassemia. Regarding few case reports on successful non-myeloablative stem cell transplantation in class III beta-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 beta-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, nonmyeloablative stem cell transplantation may be used as a curative, less toxic posttransplantation treatment for class III beta-thalassemia