国内首例同胞脐血移植治疗儿童骨髓增生异常综合征

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

目的 探讨脐血移植对儿童骨髓增生异常综合征 (MDS)的治疗效果。方法 一例 1 2岁MDS患儿行HLA相合的同胞脐血移植 ,预处理采用BU/CY +ATG方案 :马利兰 (BU) 1mg/kg ,每 6h一次 ,用 2d ;环磷酰胺 (CY)每日 5 0mg/kg ,用 4d ;抗胸腺球蛋白 (ATG) 1 0 0mg/d ,用 4d。输入脐血有核细胞 2 .5 7× 1 0 7/kg ,CD3 4 +细胞 1 .1 8× 1 0 5/kg。移植物抗宿主病 (GVHD)的预防采用环胞菌素A (CsA) +骁悉 (MMF) +甲基泼尼松龙(MP)。移植后应用惠尔血、白介素 1 1及重组红细胞生成素以加速造血重建。结果 +2 1d粒系植入 ,+48d血小板植入 ,+2 8d患者骨髓DNA指纹图示完全嵌合状态。随访 1 1个月 ,患者各项检查正常 ,未发生急、慢性GVHD。结论 本例为国内大陆首例成功脐血移植治疗MDS 。

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.