双份脐带血移植治疗黏多糖贮积症临床疗效分析  

Clinical analysis of double umbilical cord blood transplantation in the treatment of mucopolysaccharidosis

作　　者：乔广明 王志敏 张雪欣 时亚娟 齐晓玉 贠美玲 岳燕 师晓东[2] QIAO Guangming;WANG Zhimin;ZHANG Xuexin;SHI Yajuan;QI Xiaoyu;YUN Meiling;YUE Yan;SHI Xiaodong(Hematology Oncology Center,Hebei children′s Hospital of Integrated Traditional Chinese and Western Medicine,Shijiazhuang 050000,China;Department of Hematology,Capital Institute of Pediatrics,Beijing 10020,China)

机构地区：[1]河北中西医结合儿童医院血液肿瘤中心,石家庄050000 [2]首都儿科研究所血液科,北京100020

出　　处：《中国小儿血液与肿瘤杂志》2021年第4期223-226,共4页Journal of China Pediatric Blood and Cancer

基　　金：河北省卫健委医学科学研究重点课题(20201208)。

摘　　要：目的了解黏多糖贮积症患儿非血缘异基因双份脐带血干细胞移植的疗效及特点。方法回顾性分析13例黏多糖贮积症患儿接受双份非血缘异基因脐带血干细胞移植的临床资料,包括受者情况、供者选取情况、移植后粒细胞及血小板植入时间、酶学指标、移植嵌合度及移植并发症情况。结果13例患儿脐带血干细胞均顺利植活,其中单份植入10例,双份植入3例,此3例监测半年到一年,最终转为单份优势脐带血完全植入。移植后酶学检查均恢复正常,所有患儿均存活,发生Ⅰ度GVHD 2例,无慢性GVHD,4例发生巨细胞病毒血症,2例出现了BK病毒性膀胱炎,1例患儿发生了肾病综合征。结论双份脐带血干细胞移植是治疗大体重或单份脐血干细胞数量不足黏多糖贮积症患儿的有效治疗方法。Objective To analyze the effect and characteristics of unrelated allogeneic double umbilical cord blood stem cell transplantation in children with mucopolysaccharidosis.Methods Clinical data including the recipient situation,donor selection,time of granulocyte and platelet implantation after transplantation,enzyme indexes,graft chimerism and transplant complications of 13 children with mucopolysaccharidosis receiving unrelated allogeneic double umbilical cord blood stem cells were analyzed retrospectively.Results All 13 patients were successfully transplanted umbilical cord blood stem cells,including 10 cases of single implantation and 3 cases of double implantation.The monitoring lasted from half a year to one year,and finally the single dominant umbilical cord blood was completely implanted.After transplantation,the enzyme examination returned to normal.All patients survived.Grade I graft versus host reaction(GVHD)occurred in 2 cases,no chronic GVHD occurred,cytomegalovirus disease occurred in 4 cases,BK virus cystitis occurred in 2 cases,and nephrotic syndrome occurred in 1 case.Conclusions Double umbilical cord blood stem cell transplantation is an effective treatment for pediatric mucopolysaccharidosis with large body weight or insufficient number of single umbilical cord blood stem cells.

关 键 词：黏多糖贮积症 非血缘异基因造血干细胞移植 双份脐带血干细胞移植 

分 类 号：R72[医药卫生—儿科]