Urban, C; Binder, B; Hauer, C; Lanzer, G.
Congenital sideroblastic anemia successfully treated by allogeneic bone marrow transplantation.
BONE MARROW TRANSPLANT. 1992; 10(4): 373-375.
(- Case Report)
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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.
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