造血干细胞移植治疗儿童急性重型再生障碍性贫血的效果  

Stem cell transplantation for children with acquired severe aplastic anaemia

作　　者：王彬[1] 吴润晖[1] 庞一琳[2] 朱光华[1] 周翾[1] 杨骏[1] 李斯丹[1] 秦茂权[1] 

机构地区：[1]首都医科大学附属北京儿童医院血液肿瘤中心血液病与肿瘤分子分型北京市重点实验室儿科学国家重点学科,北京100045 [2]首都医科大学附属北京儿童医院急诊科,北京100045

出　　处：《中国医药导报》2017年第20期97-100,共4页China Medical Herald

基　　金：国家自然科学基金资助项目(81641006);北京市医院管理局重点医学发展专项经费资助项目(ZY201404);北京市高层次创新创业人才支持计划青年拔尖项目(2016000021223ZK16)

摘　　要：目的探讨造血干细胞移植(HSCT)治疗儿童急性重型再生障碍性贫血的效果,评价其安全性及预后。方法回顾性分析首都医科大学附属北京儿童医院血液科2006年1月~2015年12月采用HSCT治疗51例急性重型再生障碍性贫血患儿的临床资料,评价其植入情况、并发症及移植疗效。结果 98%患者获得造血重建。中性粒细胞≥0.5×109/L的中位时间为11 d;血小板≥20×109/L的中位时间为15 d。10例合并巨细胞病毒血症,2例合并出血性膀胱炎,7例发生Ⅰ~Ⅱ度急性移植物抗宿主病,2例发生Ⅲ度急性移植物抗宿主病。46例患者存活,3年存活率Allo-HSCT、Haplo-HSCT、MUD-HSCT分别是100%、89%、75%,无感染<5岁的极重型再生障碍性贫血任何移植成功率均达100%。结论 HSCT治疗儿童急性重型再生障碍性贫血的方法有效,并发症少,生存率高。Objective To explore the effect of hematopoietic stem cell transplantation（HSCT） in the treatment of severe aplastic anemia, and evaluate its safety and prognosis. Methods Clinical data of 51 children with severe aplastic anaemia received HSCT in Hematology Department of Beijing Children′s Hospital Affiliated to Capital Medical University from January 2006 to December 2015 were retrospectively analyzed. The engraftment, complications and curative effect were evaluated. Results 98 percent of patients had rapid engraftment. The median time of neutrophil exceeded0.5 ×109/L and platelets recovery exceeded 20 ×109/L were 11 and 15 days for all transplantation patients. In this research, 10 patients were diagnosed with CMV viremia. 2 patients developed hemorrhagic cystitis. 7 cases had grade Ⅰ-Ⅱa GVHD and 2 cases had grade Ⅲ a GVHD. 46 patients were still alive. The estimated 3-year OS of the Allo-HSCT, HaploHSCT and MUD-HSCT group was 100%, 89% and 75% respectively. All children less than 5 years of age（without infection） were transplanted successfully. Conclusion HSCT is effective in the treatment of acute severe aplastic anemia in children, with fewer complications and high survival rates

关 键 词：重型再生障碍性贫血 造血干细胞移植 儿童 

分 类 号：R556[医药卫生—血液循环系统疾病]