摘要
Background:Human leukocyte antigen-identical sibling donor(ISD)-hematopoietic stem cell transplantation(SCT)is a potentially curative treatment for high-risk pediatric acute myeloid leukemia(AML).A haploidentical donor(HID)is readily available to almost all children.Previous studies have demonstrated that patients with HID-SCT had similar outcomes compared to ISD-SCT for pediatric and adult AML.However,the role of HID-SCT in high-risk pediatric AML is unclear.Methods:To compare the overall survival of high-risk AML children who underwent either HID-SCT or ISD-SCT,we analyzed 179 cases of high-risk AML patients under 18 years of age treated with either ISD-SCT(n=23)or HID-SCT(n=156).Granulocyte colony-stimulating factor plus anti-thymocyte globulin-based regimens were used for HID-SCT.We also analyzed the subgroup data of AML patients at first complete remission(CR1)before SCT with known cytogenetic risk.Results:The numbers of adverse cytogenetic risk recipients were 8(34.8%)and 13(18.8%)in the ISD-SCT group and the HID-SCT group,and the number of patients with disease status beyond CR1 were 6(26.1%)and 14(20.3%)in the two groups.The cumulative rates of grades II-IV acute graft-versus-host disease(GVHD)were 13.0%in the ISD-SCT group and 34.8%in the HID-SCT group(P=0.062),with a three-year cumulative rates of chronic GVHD at 14.1%and 34.9%,respectively(P=0.091).The relapse rate in the ISD-SCT group was significantly higher than that in the HID-SCT group(39.1%vs.16.4%,P=0.027);with non-relapse mortality at 0.0%and 10.6%(P=0.113),respectively.The three-year overall survival rates were 73.0%for the ISD-SCT group and 74.6%for the HID-SCT group(P=0.689).In subgroup analysis,the three-year relapse rate in the ISD-SCT group was higher than that in the HID-SCT group(50.0%vs.9.2%,P=0.001)and the three-year DFS in the ISD-SCT group(50.0%)was lower than that in the HID-SCT group(81.2%)(P=0.021).Conclusions:Unmanipulated HID-SCT achieved DFS and OS outcomes comparable to those of ISD-SCT for high-risk pediatric AML patients with potentially higher rate but manageable GVHD.
基金
This work was partly supported by grants from the National Key Research and Development Program of China(Grant No.:2017YFA0104500)from the Ministry of Science and Technology
National Natural Science Foundation of China(Grant No.:81770189,81621001,and 81530046)
the Science and Technology Project of Guangdong Province of China(Grant No.:2016B030230003)
Peking University Clinical Scientist Program(Grant No.:BMU2019LCKXJ003)
the Fundamental Research Funds for the Central Universities
the project of health collaborative innovation of Guangzhou city(Grant No.:201704020214).
