异基因造血干细胞移植治疗儿童重型再生障碍性贫血:单中心回顾性研究

Allogeneic hematopoietic stem cell transplantation for children with severe aplastic anemia:a retrospective analysis from a single center

目的探讨异基因造血干细胞移植(allo-HSCT)治疗儿童重型再生障碍性贫血(SAA)的临床疗效。方法回顾性分析我院2020年1月至2022年12月接受allo-HSCT的27例SAA患儿的临床资料。结果(1)27例SAA患儿中,男18例、女9例,中位年龄8(2~15)岁;SAA-Ⅰ型20例,SAA-Ⅱ型7例,3例接受同胞全相合造血干细胞移植,24例接受亲缘单倍体造血干细胞移植;(2)27例患儿均获得造血重建,中性粒细胞中位重建时间为10(9~20)d,血小板中位重建时间为12(7~26)d。急性移植物抗宿主病(GVHD)发生率为66.67%(18/27),其中Ⅰ~Ⅱ度发生率为55.56%(15/27),Ⅲ~Ⅳ度发生率为11.11%(3/27),慢性GVHD发生率为7.41%(2/27)。移植相关血栓性微血管病(TA-TMA)发生率7.41%(2/27)。巨细胞病毒血症、EB病毒血症发生率为62.96%(17/27)、33.33%(9/27),14.81%(4/27)患儿并发移植后淋巴细胞增殖性疾病(PTLD);(3)中位随访时间为12(2~28)个月,总生存率为96.29%,26例存活,1例因重度急性GVHD、TA-TMA、巨细胞病毒感染、PTLT、继发性癫痫等多种并发症死亡。结论allo-HSCT是治疗儿童SAA的有效方法,本研究有效率为96.29%;急性GVHD仍是治疗成败的关键,发生率为66.67%,供受者血型不合可能影响GVHD的发生率;同胞全相合造血干细胞移植可降低预防GVHD强度,以减少病毒复燃、PTLD等并发症的发生。

ObjectiveTo evaluate the clinical efficacy of allogeneic hematopoietic stem cell transplantation(allo-HSCT)in children with severe aplastic anemia(SAA).MethodsTwenty-seven cases with SAA who had been treated with allo-HSCT from January 2020 to December 2022 were retrospectively analyzed and reviewed.Results(1)A total of 27 SAA patients were enrolled,including 18 males and 9 females,with a median age of 8(2-15)years.There were 20 cases of SAA-Ⅰtype,7 cases of SAA-Ⅱtype.Based upon donor sources,three cases of matched sibling donors hematopoietic stem cell transplantation,and 24 cases of haploidentical hematopoietic stem cell transplantation were adopted.(2)Hematopoietic reconstruction was achieved in all 27 cases.The median implantation time of neutrophils and platelets was 10(9-20)days and 12(7-26)days respectively.The cumulative incidence of acute graft-versus-host disease(GVHD)was 66.67%(18/27).The incidence of gradeⅠ-Ⅱwas 55.56%(15/27)and that of gradeⅢ-Ⅳwas 11.11%(3/27).The incidence of chronic GVHD was 7.41%(2/27).Transplant-associated thrombotic microangiopathy(TA-TMA)occurred in 7.41%(2/27)patients,cytomegalovirus viremia in 62.96%(17/27)patients,epstein-barr virus infection in 33.33%(9/27)patients,and 14.81%(4/27)patients progressed to post-transplant lymphoproliferative disorder(PTLD).(3)The median follow-up time was 12(2-28)months.The overall survival rate was 96.29%.Twenty-six patients survived,and one patient died due to multiple complications of severe acute GVHD,TA-TMA,cytomegalovirus infection,PTLD and secondary epilepsy.ConclusionAllo-HSCT is an effective therapy for SAA in children.The effective rate of this research is 96.29%.Acute GVHD is still the key to therapy.The incidence rate of acute GVHD is 66.67%in this study.The blood incompatibility of donor and recipient may affect the incidence of GVHD.The intensity of GVHD prevention should be reduced after HLA-matched sibling donor-hematopoietic stem cell transplantation so as to avoid the complications of virus recurrence and PTLD.