title: Congenital sideroblastic anemia successfully treated by allogeneic bone marrow transplantation.
abstract: Allogeneic bone marrow transplantation (BMT) was carried out on a 34-month-old boy with congenital sideroblastic anemia. The patient had been red blood cell transfusion dependent since the age of 7 weeks. He did not respond to therapy with pyridoxine and developed secondary progressive hemosiderosis. The preparatory regimen consisted of busulfan (3.5 mg/kg for 4 days) and cyclophosphamide (50 mg/kg for 4 days). Full engraftment of donor bone marrow was achieved and effective hemopoiesis is still maintained 3 years after BMT.
authors.affiliation: Department of Pediatrics, University of Graz, Austria.
authors.forename: C
authors.initials: C
authors.lastname: Urban
authors.forename: B
authors.initials: B
authors.lastname: Binder
authors.forename: C
authors.initials: C
authors.lastname: Hauer
authors.forename: G
authors.initials: G
authors.lastname: Lanzer
mesh_terms: D000756:Anemia, Sideroblastic / Q000151:congenital / Q000188:drug therapy / Q000601:surgery*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000008:administration & dosage; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D006801:Humans; D008297:Male; D014184:Transplantation, Homologous
chemical_list: D003520:Cyclophosphamide; D002066:Busulfan