Allogeneic stem cell transplantation from unrelated donor for class 3 beta-thalassemia major using reduced-intensity conditioning regimen.

摘要

beta-thalassemia major is a genetic disorder resulting in absent or reduced beta globin chain synthesis producing hemolytic anemia. Currently, the only cure for thalassemia is allogeneic stem cell transplantation, which corrects the genetic defect in the hematopoietic system by the use of allogeneic stem cells. According to Pesaro classification, patients with thalassemia major can be categorized into three classes of risk for marrow transplantation, and patients with class 3 who had evidence of organ damage from iron-overload had poor outcomes, and were more likely to experience transplant-related mortality or disease recurrence. One approach to reduce transplant-related mortality is to use reduced-intensity stem cell transplantation (RIST) and here we report the successful transplantation from 6/6 matched unrelated donors using reduced-intensity conditioning for two children with class 3 thalassemia major who had evidence of organ damage.