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目的探讨脐血移植对儿童骨髓增生异常综合征的治疗效果。方法 1例12岁患儿行人白细胞抗原相合的同胞脐血移植,预处理采用白消安/环磷酰胺方案:白消安1mg/kg,每6 h一次,用2 d,共8次;环磷酰胺50mg/(kg·d),用4 d;抗胸腺球蛋白100 mg/d,用4 d。输入脐血有核细胞2.57×1 07/kg,CD34+细胞1.18×105,kg。移植物抗宿主疾病的预防采用环胞菌素A+骁悉+甲泼尼龙。移植后应用粒细胞集落刺激因子、白细胞介素-11及促红细胞生成素以加速造血重建。结果 +2l天粒系植入,+48天血小板植入,+28天患者骨髓DNA指纹图示完全嵌合状态。随访11个月,患者各项检查正常,未发生急、慢性移植物抗宿主疾病。结论本例为国内大陆首例成功脐血移植治疗骨髓增生异常综合征,为今后儿童骨髓增生异常综合征的治疗提供了一种新的方法。
Objective To investigate the therapeutic effect of cord blood transplantation on children with myelodysplastic syndrome. Methods 1 case of 12-year-old children with leukocyte antigen matched siblings umbilical cord blood transplantation, pretreatment with busulfan / cyclophosphamide program: busulfan 1mg / kg, once every 6 h, with 2 d, a total of 8 times; ring Phosphoramide 50mg / (kg · d), with 4 d; anti-thymosin 100 mg / d, with 4 d. Enter umbilical cord blood cells 2.57 × 107 / kg, CD34 + cells 1.18 × 105, kg. Prevention of graft-versus-host disease with cyclosporin A + sxp + methylprednisolone. Application of granulocyte colony-stimulating factor, interleukin-11 and erythropoietin after transplantation to accelerate hematopoietic reconstitution. Results + 2l grafts were implanted, +48 days platelet implants, and bone marrow DNA fingerprints on day 28 showed complete chimerism. All cases were followed up for 11 months. All patients were normal and did not have acute and chronic graft-versus-host disease. Conclusion This case is the first successful treatment of myelodysplastic syndrome with cord blood transplantation in mainland China, which provides a new method for the treatment of myelodysplastic syndrome in children in the future.