Treatment of Childhood Myelodysplastic Syndrome by Sibling Cord Blood Transplantation: the First Case Report in China

Abstract:

OBJECTIVE: To explore the efficacy of cord blood stem cell transplantation (CBSCT) for treatment of myelodysplastic syndrome (MDS) in children. METHODS: A 12 year old child with MDS received the treatment of HLA matched sibling CBSCT. The pre treatment regimen was “BU/CY+ATG” [busulfan (BU) with the dosage of 1 mg/kg, once every 6 hrs, and 8 times in all; cyclophosphamide (CY) with the dosage of 50 mg/kg daily for 4 days and antihuman thymocyte globulin (ATG) with the dosage of 100 mg daily for 4 days]. After pre treatment, 2.57 ×10 7/kg of cord blood nucleated cells and 1.18 ×10 5/kg of CD 34 positive cells were transplanted into the child with MDS. The combination of cyclosporine A, mycophenolate mofetil and ET methylprednisolone was administrated for prevention of graft versus host disease (GVHD). After transplantation the patient was given a combination of granulocyte colony stimulating factor, interleukin 11 and erythropoietin to promote reconstitution of hematopoiesis. RESULTS: The reconstitution time of granulocyte cell and platelet were respectively 21 days and 48 days. Microsatellite DNA fingerprinting showed a full donor chimerism on day 28 after transplantation. In an 11 month follow up, the patient did not develop GVHD and other transplantation related complications. CONCLUSIONS: It is the first case report in China on the successful treatment of MDS by CBSCT, which can provide guidelines for the future treatment of childhood MDS.