单倍体相合造血干细胞移植联合骨髓间充质干细胞输注治疗儿童重型再生障碍性贫血的临床观察  

Clinical Observation of Haploidentical Hematopoietic Stem Cell Transplantation Combined with Bone-marrow Derived Mesenchymal Stem Cells Transfusion for Treatment of Children with Severe Aplastic Anemia

作　　者：危薇[1] 王志东[2] 郑晓丽[2] 丁丽[2] 韩冬梅[2] 闫洪敏[2] 王恒湘[1] 

机构地区：[1]安徽医科大学空军临床学院,北京100142 [2]中国人民解放军空军总医院,北京100142

出　　处：《中国实验血液学杂志》2017年第4期1158-1164,共7页Journal of Experimental Hematology

摘　　要：目的:探讨单倍体相合造血干细胞移植(haploidentical-HSCT,hi-HSCT)联合骨髓间充质干细胞(BMM SC)输注治疗儿童重型再生障碍性贫血(SAA)的疗效。方法:回顾性分析25例行hi-HSCT联合BM-M SC输注治疗儿童SAA的临床资料,观察移植后造血恢复及移植相关并发症。结果:25例患儿中性粒细胞均植入,植入中位时间为12(11-22)d,其中23例血小板植入,中位植入时间为21(11-130)d。16例发生急性移植物抗宿主病(a GVHD),其中11例为Ⅰ度,5例为Ⅱ-Ⅳ度,1例在移植后115 d死于累及皮肤、肠道及肝脏的Ⅳ度a GVHD,5例发生慢性GVHD(c GVHD),均为局限性c GVHD。发生CMV血症的有23例,无1例进展为CMV病。23例患儿发生EBV血症,且其中有3例发生移植后淋巴增殖性疾病,经利妥昔单抗治疗后均治愈。出血性膀胱炎发生9例,仅1例为Ⅲ度。22例发生感染,其中10例为肺部感染,4例为肝脏感染。1例发生格林-巴利综合征。1例出现自身免疫性溶血性贫血。22例患儿存活且脱离输血,中位随访时间为14(3-27)个月。结论:hi-HSCT联合BM-MSC输注治疗儿童SAA是安全、可行的。Objective： To investigate the efficacy and safety of haploidentical hematopoietic stem cell transplantation（ hi-HSCT） combined with bone-marrow derived mesenchymal stem cell（ BM-MSC） tranfusion for treatment of children with severe apastic anemia（ SAA）. Methods： The clinical data of 25 children with SAA undergoing hi-HSCT and BMMSC tranfusion were retrospectively analyzed from August 2014 to July 2016. Results： neutrophil engraftment was achieved in all 25（ 100%） children,with the median time 12（ 11-22） days. The median time of platelet engraftment was 21（ 11-130） days in 23（ 92%） children. Acute graft-versus-host disease（ aGVHD） was observed in 16（ 64%）cases,including 11 case of grade I and 5 cases of aGVHD grade Ⅱ-Ⅳ,and one of them died of grade Ⅳ of skin,gut and liver at day 115; 5 cases of chronic GVHD were found,all of them were limited cGVHD. Cytomegalovirus（ CMV）viremia was observed in 23（ 92%） cases,but no CMV disease was developed after therapy. 3 cases of post-transplant lymphoroliferative disease with 23 EBV viremia positive occurred,all of them were cured after rituximab. Hemorrhagic cystitis appeared in 9 cases with only one case of grade Ⅲ,22 children suffered from infection,involving 10 cases in lung and 4 cases in liver,1 patient was diagnosed as Guillain-Barre syndrome. Autoimmune hemolytic anemia was recorded in 1 patient,22 children survived during a median following-up time of 14（ 3-27） months. Conclusion： The hi-HSCT combined with BM-MSC transfusion for treatment of children with SAA has been confirmed to be safe and feasible.

关 键 词：造血干细胞移植 间充质干细胞 儿童重型再生障碍性贫血 

分 类 号：R457.7[医药卫生—治疗学] R556.5[医药卫生—临床医学]