ABSTRACT
Therapy for patients with congenital sideroblastic anemia has been limited to blood transfusions and chelation. Five patients with Congenital Sideroblastic anemia [CSA] who were blood transfusion dependent underwent stem cell transplantation [SCT] from matched sibling donors. Their ages at SCT were 1,2,3,5 and 8 years. Conditioning consisted of cyclophasphamide 50 mg/kg/day for 4 days, busulfan 4 mg/kg/day for days and antithymocyte globulin [ATG] 30 mg/kg/for 4 doses pretransplant. For Graft vs. Host disease [GVHD] prophylaxis, cyclosporin A and methotrexate were used. All patient engrafted, and are alive with sustained engraftment and are transfusion independent. Two patients developed acute GVHD, and none of the patients developed chronic GVHD. In conclusion SCT can be curative for patients with CSA