单倍体造血干细胞移植与强化免疫抑制治疗儿童重型再生障碍性贫血的临床观察

Clinical observation of haploid hematopoietic stem cell transplantation and intensive immunosuppressive therapy for the treatment of severe aplastic anemia in pediatric patients

目的对比儿童重型再生障碍性贫血(SAA)行单倍体造血干细胞移植(haplo-HSCT)和强化免疫抑制治疗(IST)的疗效。方法回顾性分析2013年1月至2018年6月郑州大学第一附属医院新诊断为SAA的患儿病历资料,其中33例接受haplo-HSCT;24例接受抗胸腺细胞球蛋白(ATG)联合环孢素(CsA)的IST治疗。比较haplo-HSCT组和IST组患儿的有效率、总生存(OS)率、无失败生存(FFS)率等情况。结果中位随访25个月(9~60个月),haplo-HSCT组和IST组早期死亡例数分别为5例和4例,差异无统计学意义(P=0.822);除早期死亡患儿,haplo-HSCT组治疗3个月时有效率[100%(28/28例)]高于IST组[30%(6/20例)],差异有统计学意义(χ2=27.671,P<0.01)。haplo-HSCT组治疗6个月时有效率[92.9%(26/28例)]高于IST组[65.0%(13/20例)],差异有统计学意义(χ2=5.943,P=0.015)。haplo-HSCT组治疗12个月时有效率[89.3%(25/28例)]高于IST组[70.0%(14/20例)],差异无统计学意义(P>0.05)。haplo-HSCT组和IST组患儿3年预期OS率分别为75.0%和70.3%,差异无统计学意义(χ2=0.133,P=0.716)。haplo-HSCT组3年预期FFS率74.2%高于48.7%,差异有统计学意义(χ2=4.036,P=0.045)。结论对于儿童SAA的治疗,如无同胞全合造血干细胞移植供者,haplo-HSCT也不失为一种有效的治疗方案。

Objective To compare the efficacy of haploid hematopoietic stem cell transplantation(haplo-HSCT)and intensive immunosuppressive therapy(IST)in children with severe aplastic anemia(SAA).Methods The medical records of children newly diagnosed as SAA in the First Affiliated Hospital of Zhengzhou University from January 2013 to June 2018 were retrospectively analyzed.Among them,33 patients received haplo-HSCT and 24 patients received IST that combined anti-thymocyte globulin(ATG)with Cyclosporine(CsA).The effective rate,overall survival(OS)rate,and failure free survival(FFS)rate of children in the haplo-HSCT group and the IST group were compared.Results The median follow-up period was 25 months(9-60 months).There were 5 cases of early death in the haplo-HSCT group and 4 cases in the IST group,and the differences were not statistically significant(P=0.822).Leaving out the early death cases,the effective rate in the haplo-HSCT group[100%(28/28 cases)]was higher than that in the IST group[30%(6/20 cases)]after 3 months of treatment,the difference was statistically significant(χ2=27.671,P<0.01).After 6 months of treatment,the effective rate in the haplo-HSCT group[92.9%(26/28 cases)]was higher than that in the IST group[65.0%(13/20 cases)],and the difference was statistically significant(χ2=5.943,P=0.015).After 12 months of treatment,the effective rate in the haplo-HSCT group[89.3%(25/28 cases)]was higher than that in the IST group[70.0%(14/20 cases)],but the difference was not statistically significant(P>0.05).The 3-year expected OS rate of children in the haplo-HSCT group and the IST group were 75.0%and 70.3%,respectively,with no statistically significant difference(χ2=0.133,P=0.716).The 3-year expected FFS rate of children in the haplo-HSCT group(74.2%)was significantly higher than that in the IST group(48.7%),and the difference was statistically significant(χ2=4.036,P=0.045).Conclusion For children with SAA,haplo-HSCT is also an effective treatment if there is no sibling donor of hematopoietic stem cell transplantation.