Congenital sideroblastic anaemia successfully treated using allogeneic stem cell transplantation

Therapy for patients with congenital sideroblastic anaemia has been limited to blood transfusions and chelation. Three children with congenital sidero blastic anaemia (SA) who were blood transfusion dependent underwent stem ce ll transplantation (SCT) from matched sibling donors. Conditioning consiste d of cyclophosphamide 50 mg/kg/d for 4 d, busulphan 4 mg/kg/d for 4 d and a ntithymocyte globulin (ATG) 30 mg/kg for four doses pretransplant. Graft-ve rsus-host disease (GVHD) prophylaxis was with cyclosporin A and methotrexat e. All patients engrafted, and are alive and transfusion independent. SCT c an be curative for patients with SA.