摘要目的 评估亲缘单倍体造血干细胞移植(haplo-HSCT)治疗儿童血液病的安全性和疗效.方法 回顾性分析航天中心医院2012年7月至2016年6月收治的59例14岁以下血液病患儿,通过分析植入成功率、移植物抗宿主病(GVHD)发生率、感染发生率及移植相关病死率(TRM)、累积复发率、总体生存率(OS)、无病生存率(DFS)进行疗效评估.结果 59例患儿行haplo-HSCT,移植后均顺利植活,白细胞植活的中位时间为18(8~23)d,血小板植活的中位时间为21(11~68)d.移植后28 d评估骨髓,59例患儿均达完全缓解(CR),经DNA检测证实均为完全供者嵌合状态.中位随访时间为19(5~56)个月.23例Ⅰ、Ⅱ度急性GVHD累积发生率为(38.3 ± 6.3)%,10例Ⅲ、Ⅳ度急性GVHD累积发生率为(16.7 ± 4.8)%,30例慢性GVHD的累积发生率为(65.6 ± 7.5)%,27例巨细胞病毒(CMV)血症累积发生率为(45.1 ± 6.5)%,6例EB病毒(EBV)血症累积发生率为(10.0 ± 3.9)%,12例病毒型膀胱炎累积发生率为(20.0 ± 5.2)%,移植相关病死率为(12.8 ± 6.0)%,2年累积复发率CR组为(8.0 ± 5.4)%,未缓解(NR)组为(64.1 ± 11.9)%.CR组2年OS为(78.9 ± 7.5)%,NR组2年OS为(32.5 ± 12.9)%,CR组2年 DFS为(79.5 ± 9.8)%,NR组2年 DFS为(27.4 ± 9.9)%.结论 haplo-HSCT治疗儿童血液病安全有效,尤其是缓解期进行移植的患儿生存率高,复发率低.对于难治复发的患儿预后较差,需要进一步探索预防复发的措施.

abstractsObjective To evaluate the safety and efficacy of haploidentical hematopoietic stem cell transplan-tation(haplo-HSCT)treatment in children with hematological diseases.Methods Fifty-nine cases of less than 14 years old children with hematonosis were analyzed retrospectively,who were enrolled in the Aerospace Central Hospital from July 2012 to June 2016.And the evaluation was carried out by analyzing the success rate of implantation,occu-rrence rate of graft versus host disease(GVHD),infection rate and transplant related mortality(TRM),cumulative re-currence rate,overall survival rate(OS)and disease-free survival rate(DFS).Results In total of 59 cases,the 59 engraftments were successfully transplanted,the median time of leukocyte engraftment was 18(8-23)days,the median time of platelet engraftment was 21(11-68)days,the bone marrow was assessed 28 days after transplanta-tion,which showed that 59 patients achieved complete remission(CR)and DNA test confirmed complete donor chime-rism.With a median of follow-up time of 19(5-56)months,the cumulative recurrence rates ofⅠ,Ⅱgrade andⅢ,Ⅳ grade acute GVHD were(38.3 ± 6.3)%(23 cases)and(16.7 ± 4.8)%(10 cases),respectively,the chronic GVHD cumulative recurrence rate was(65.6 ± 7.5)%(30 cases),the cytomegalovirus(CMV)viremia cumulative recurrence rate was(45.1 ± 6.5)%(27 cases),the Epstein-Barr virus(EBV)viremia cumulative recurrence rate was(10.0 ± 3.9)%(6 cases),the viral cystitis cumulative recurrence rate was(20.0 ± 5.5)%(12 cases),the transplant related mortality was(12.8 ± 6.0)%,the 2-year cumulative recurrence rate of CR group was(8.0 ± 5.4)%,and that of non-remission(NR)group was(64.1 ± 11.9)%.The 2-year OS of CR group was(78.9 ± 7.5)%,the 2-year OS of NR group was(32.5 ± 12.9)%,the 2-year DFS of CR group was(79.5 ± 9.8)%,the 2 years DFS of NR group was(27.4 ± 7.9)%.Conclusions Haplo-HSCT is safe and effective in treating children with hematonosis,and haplo-HSCT has high survival rate and low recurrent,especially when transplantation is per-formed in the remission stage.But the prognosis of haplo-HSCT is poor in the refractory and relapsed patients,and to explore the preventing recurrence measures are very urgent.