Objective: To observe the efficacy of matched unrelated donor hematopoietic stem cell transplant (HSCT) with transfusion of multipotent mesenchymal cells (MSC) in pediatric severe aplastic anemia (SAA).
Methods: 19 children with SAA received matched unrelated donor HSCT with MSC, and the hematopoietic recovery and transplant-associated complications of these children were monitored.
Results: All patients achieved rapid hematopoietic reconstruction after HSCT, and the median durations to neutrophil and platelet recovery were 12 (9-21) days and 14 (8-24) days respectively, but delayed rejection occurred in one case four months after HSCT. 9 cases developed grade Ⅰ acute graft-versus-host (aGVHD), and one case grade Ⅲ aGVHD and diffuse chronic graft-versus-host. Cytomegalovirus viremias were observed in 15 patients. 2 cases developed hemorrhagic cystitis, 10 children experienced infections. All the children were alive during a median following-up time of 27(8-70) months, one of them developed LPD and received rituximab and chemotherapy, delayed rejection occurred in this patient four months after HSCT, Haplo-identical HSCT from his father as the donor was performed and achieved successful engraftment.
Conclusion: The matched unrelated donor HSCT with MSC in pediatric SAA was safe and effective.
目的: 探讨HLA相合无关供者造血干细胞移植（MUD-HSCT）联合脐带间充质干细胞（MSC）输注治疗儿童重型再生障碍性贫血（SAA）的疗效及安全性。
方法: 回顾性分析19例行MUDHSCT联合MSC治疗的儿童SAA患者临床资料，观察移植后造血重建及移植相关并发症。
结果: 19例患儿移植后均获得迅速造血重建，粒细胞中位植入时间为12(9~21) d，血小板中位植入时间为14(8~24) d, 1例患儿于移植后4个月出现继发植入失败。9例患儿发生Ⅰ度急性移植物抗宿主病（aGVHD），1例发生Ⅲ度aGVHD并于移植后6个月发生广泛型慢性移植物抗宿主病。CMV阳性15例，出血性膀胱炎2例。10例患儿移植后出现不同程度、不同部位的感染。中位随访时间为27(8~70）个月，19例患儿截至随访终点均无病存活，其中1例患儿于移植后4个月发生淋巴细胞增殖性疾病，接受利妥昔单抗联合化疗治疗后发生继发性植入失败，后接受以父亲为供者的单倍体造血干细胞移植成功植入。
结论: MUD-HSCT联合MSC输注治疗儿童SAA是安全有效的。