Clinical analysis of combining haploidentical donor blood and cord blood stem cell transplantation in the treatment of severe aplastic anemia in children
Clinical analysis of combining haploidentical donor blood and cord blood stem cell transplantation in the treatment of severe aplastic anemia in children
Objective To compare the efficacy of combining haploidentical donor blood and cord blood stem cell transplantation(Haplo-cord HSCT)with haploidentical stem cell transplantation(Haplo-HSCT)in the treatment of severe aplastic anemia(SAA)in children.Methods A retrospective analysis was performed on 38 cases of SAA who underwent haplo-HSCT in Department of Hematology and Oncology of Shanghai Children's Hospital from May 2017 to February 2023.A total number of 21 children with SAA received Haplo-Cord HSCT,while 17 children received Haplo-SCT.The regimen used myeloablative conditioning(fludarabine,busulfan,cyclophosphamide and rabbit anti-thymocyte globulin).Results No primary graft failure(PGF)occurred in any of the 21 patients in Haplo-cord cohort,and one patient developed PGF in the Haplo cohort.Two patients in Haplo-cord cohort and one patient in Haplo 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 compared to 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,without any 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 co-infusion of an UCB may improve survival of Haplo-HSCT in SAA patients.
维普
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