获得性再生障碍性贫血二次移植疗效分析

Second allogeneic hematopoietic stem cell transplantation,an important rescue option for graft failure in children with acquired aplastic anemia

目的探讨获得性再生障碍性贫血(AAA)患儿异基因造血干细胞移植(allo-HSCT)植入失败后行二次移植的疗效和安全性。方法对2002年7月-2017年12月因allo-HSCT植入失败在上海儿童医学中心行二次移植的AAA患儿的临床资料进行回顾性分析。结果共有283例AAA患儿行allo-HSCT,其中8例因植入失败行二次移植。2例为原发植入失败,6例为继发植入失败。6例在二次移植后14(10~26)天中性粒细胞植活,4例在二次移植后15.5(11~35)天血小板植活。1例在二次移植造血重建前死于感染,1例二次移植发生原发植入失败,5例发生急性移植物抗宿主病,3例发生慢性移植物抗宿主病。中位随访17个月,3例死于感染,5例长期生存(包括2例慢性移植物抗宿主病)。结论二次移植作为AAA患儿植入失败的挽救性治疗,是有效可行的。

Objective To investigate the efficacy and safety of second allogeneic hematopoietic stem cell transplantation(allo-SHCT)for graft failure(GF)in children with acquired aplastic anemia(AAA).Methods The clinical data of AAA children receiving second allo-HSCT for GF was analyzed retrospectively between July 2002 and December 2017 at Shanghai Children′s Medical Center.Results Among 283 AAA children who received allo-HSCT,8 received second allo-HSCT for GF,including 2 primary GF and 6 second GF.6 patients acquired neutrophil engraftment with median time of 14(10~26)days.4 patients acquired platelet engraftment with median time of 15.5(11~35)days.One patient died of infection before engraftment,and 1 patient encountered primary GF.Five patients developed acute graft versus host disease(GVHD),and 3 patients developed chronic GVHD.After a median follow-up of 17 months,3 patients died of infection,and the other 5 patients were alive,including two with chronic GVHD.Conclusions Second allo-HSCT is a feasible and effective rescue option for GF secondary to the first allo-HSCT in AAA children.