关键词:

单倍体造血干细胞移植 , 脐带血 , 重型再生障碍性贫血 , 儿童

Abstract:

ObjectiveTo compare the efficacy of combining haploidentical donor blood and cord bloodstem cell transplantation （Haplo-cord HSCT） with haploidentical stem cell transplantation （Haplo-HSCT） inthe treatment of severe aplastic anemia （SAA） in children. MethodsA retrospective analysis was performed on38 cases of SAA who underwent haplo-HSCT in Department of Hematology and Oncology of Shanghai Children’s Hospitalfrom May 2017 to February 2023. A total number of 21 children with SAA received Haplo-Cord HSCT， while 17 childrenreceived Haplo-SCT. The regimen used myeloablative conditioning （fludarabine， busulfan， cyclophosphamide and rabbit anti-thymocyte globulin）. ResultsNo primary graft failure （PGF） occurred in any of the 21 patients in Haplocord cohort， and one patient developed PGF in the Haplo cohort. Two patients in Haplo-cord cohort and one patient inHaplo cohort developed Secondary graft failure，respectively. The median time for engraftment for granulocytes in the Haplo core group and Haplo group were 12（10，19） d and 11（10，16） d, respectively（P ＝ 0.630）. While the median time for platelet implantation in these groups were 16（12，32） and 16（13，24）d, respectively（P ＝ 0.461）. Patients who underwent an Haplo-cord HSCT had a lower cumulative incidence of Grade Ⅱ to Ⅳ aGVHD compared with the Haplo HSCT cohort 〔（23.8±9.3）% vs. 25±10.8）%，P ＝ 0.770）〕， they also had lower cumulative incidence of Grade Ⅲ to Ⅳ aGVHD comparedto the Haplo cohort 〔（9.5±6.4）% vs.（18.7±9.8）%，P ＝ 0.374）〕. The cumulative incidence of chronic GVHD was （14.3±7.6）% in Haplo-cord cohort and was（33±12.2）% in Haplo cohort（P ＝ 0.226）. The 3-year overall， failure-free， and GVHD failure-free （GFFS） survival rates in Haplo-cord cohort and Haplo cohort were 〔（95.2±4.6）% vs.（85.6±9.7）%， P ＝ 0.405）〕，〔（83.5±8.9）% vs.（82.4±9.2）%，P ＝ 0.642）〕 and 〔（81±8.6）% vs.（52.9±12.1）%，P ＝ 0.046）〕，respectively. We found that cord blood with a high degree of HLA matching and high CD34+ cell counts achieved high GFFS. PCR analyses of short tandem repeats （STR） showed persistent and sustained full haplo donor myeloid chimaerism， withoutany evidence of umbilical cord blood engraftment. We found that Haplo-cord HSCT in children with SAA achieved higher GFFS. Conclusion Haplo-HSCT is an effective treatment for children with SAA， with a high engraftment rate. The coinfusion of an UCB may improve survival of Haplo-HSCT in SAA patients. 

Key words:

Haploidentical hematopoietic stem cell transplantation , Cord blood , Severe aplastic anemia, Children