无关供者与同胞相合供者异基因造血干细胞移植治疗儿童和青少年重型再生障碍性贫血疗效比较  

A comparative study of unrelated donor and matched-sibling donor allogeneic hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia

作　　者：周健[1] 符粤文[1] 梁利杰[1] 王倩[1] 韩利杰[1] 祖璎玲[1] 张龚莉 朱兴虎[1] 喻凤宽[1] 房佰俊[1] 魏旭东[1] 宋永平[1] 

机构地区：[1]郑州大学附属肿瘤医院(河南省肿瘤医院)血液科,450003

出　　处：《中华内科杂志》2016年第12期927-931,共5页Chinese Journal of Internal Medicine

摘　　要：目的评估无关供者异基因造血干细胞移植（allo—HSCT）治疗儿童和青少年重型再生障碍性贫血（SAA）的疗效。方法回顾性分析河南省肿瘤医院2001年10月—2015年10月接受allo—HSCT治疗的34例儿童和青少年SAA患者临床资料，根据供者来源不同，将患者分为无关供者组（URD组，15例）和同胞相合供者组（MSD组，19例），通过与同期MSD组疗效的比较，评估无关供者allo．HSCT在儿童和青少年SAA治疗中的地位。结果URD组和MSD组在移植后造血重建率、粒系和血小板植入时间、干细胞植活率和移植排斥率方面的差异均无统计学意义（P值均〉0．05）。URD组的急性移植物抗宿主病（GVHD）发生率高于MSD组[42．9％（6／14）比10．5％（2／19），P=0．047]；URD组Ⅱ～Ⅳ度急性GVHD和慢性GVHD的发生率虽然高于MSD组，但差异均尚无统计学意义[21．4％（3／14）比5．3％（1／19），P=0．288；35．7％（5／14）比5．3％（1／19），P=0．062]；两组移植后肺部感染、巨细胞病毒血症、EB病毒血症和出血性膀胱炎发生率的差异均无统计学意义（P值均〉0．05）。URD组和MSD组的5年总生存率和无病生存率的差异均无统计学意义[（84．4±6．6）％比（89．4±7．1）％，（82．5±5．4）％比（82．1±4．3）％，P值均〉0．05]。结论无关供者allo-HSCT治疗儿童和青少年SAA的疗效与同胞相合供者allo—HSCT相当，可作为无HLA匹配同胞的儿童和青少年SAA患者的一线治疗选择。Objective To evaluate the efficacy of unrelated donor allogeneic hematopoietic stem cell transplantation（ URD allo-HSCT） for children and adolescents with severe aplastic anemia （SAA）. Methods Clinical data of 34 SAA children and adolescents undergoing allo-HSCT were retrospectively analyzed from October 2001 to October 2015. According to the source of donor, the patients were divided into matched sibling donor allo-HSCT group （MSD group） and unrelated donor group （URD group）. The clinical outcome of SAA children and adolescents receiving URD a11o-HSCT was assessed, and patients in MSD allo-HSCT group were enrolled as control at the same period. Results The rate of hematopoietic reconstitution, the time of neutrophil and platelet engraftment, incidence of chimerism and graft rejection between two groups were not statistically different. The incidence of acute graft-versus-host disease （GVHD） in URD group was significantly higher than that in MSD group [42. 9% （6/14） vs 10. 5% （2/19） ,P = 0. 0471- The incidence of grade Ⅱ - Ⅳ acute GVHD and chronic GVHD in URD were higher than those in MSD group [21.4% （3/14） vs 5.3% （ 1/19）, P =0. 288 ; 35.7% （5/14） vs 5.3% （ 1/19）, P = 0. 062,respectively], yet without significant difference between two groups. Other transplant-related complications including pulmonary complications, hemorrhagic cystitis, incidence of EBV and CMV reactivation and venous occlusive disease were comparable with two regimens. Estimated 5-years overall survival （OS） rate and disease free survival （DFS） rate were not statistically significant between URD group and MSD group [（84.4±6.6）% vs （89.4±7.1）%, （82.5±5.4）% vs （82.1±4.3）%; P=0.766, P=0.884, respectively]. Conclusions By multivariate analysis, the outcome of URD allo-HSCT in SAA children and adolescent is similar to MSD allo-HSCT. It could be an alternative option as the first-line treatment for SAA children and adolescents without HLA matched sibling donors.

关 键 词：贫血 再生障碍性 重型 儿童 青少年 造血干细胞移植 无关供者 同胞相合供者 

分 类 号：R725.5[医药卫生—儿科] R457.7[医药卫生—临床医学]