脐带血辅助单倍体外周血干细胞移植与无关供者外周血干细胞移植治疗恶性血液病的疗效比较

Haploidentical donor peripheral blood stem cell transplantation using third-party cord blood compared with matched unrelated donor transplantation for patients with hematologic malignancies

目的比较脐带血辅助单倍体外周血干细胞移植(haplo-cord-PBSCT)与无关供者外周血干细胞移植(UD-PBSCT)治疗恶性血液病的疗效。方法对中南大学湘雅医院2016年1月至2021年12月的104例接受haplo-cord-PBSCT和52例接受UD-PBSCT)的恶性血液病患者进行回顾性分析。结果①haplo-cord-PBSCT和UD-PBSCT组中性粒细胞中位植入时间分别为13(9~22)d、13(10~24)d(P=0.834),血小板植入中位时间分别为15(7~103)d、14(8~38)d(P=0.816)。haplo-cord-PBSCT组和UD-PBSCT组移植后30 d中性粒细胞累积植入率均为100.0%(P=0.314),移植后100 d血小板累积植入率分别为95.2%(95%CI 88.3%~98.1%)、100.0%(P=0.927)。移植后30 d两组患者均达到完全供者嵌合状态,未发生脐带血干细胞植入。②haplo-cord-PBSCT组与UD-PBSCT组移植后100 d内Ⅱ~Ⅳ度急性GVHD的累积发生率分别为29.1%(95%CI 20.1%~38.1%)、28.8%(95%CI 17.2%~41.6%(P=0.965),Ⅲ/Ⅳ度急性GVHD的累积发生率分别为7.8%(95%CI 3.6%~14.0%)、9.6%(95%CI 3.5%~19.5%)(P=0.725)。haplo-cord-PBSCT组与UD-PBSCT组2年慢性GVHD的累积发生率分别为45.3%(95%CI 36.1%~56.1%)、35.1%(95%CI 21.6%~44.1%)(P=0.237),移植后2年重度慢性GVHD的累积发生率分别为13.6%(95%CI 7.6%~21.3%)、12.9%(95%CI 5.1%~24.3%)(P=0.840)。③haplo-cord-PBSCT组、UD-PBSCT组移植后2年CIR分别为12.8%(95%CI 7.0%~20.5%)、10.0%(95%CI 3.6%~20.2%)(P=0.341),NRM分别为14.7%(95%CI 8.4%~22.6%)、16.2%(95%CI 7.4%~28.0%)(P=0.681)。④haplo-cord-PBSCT组、UD-PBSCT组移植后2年OS率分别为82.2%(95%CI 74.8%~90.3%)、75.5%(95%CI 64.2%~88.7%)(P=0.276),2年DFS率分别为69.9%(95%CI 61.2%~79.8%)、73.8%(95%CI 62.4%~87.3%)(P=0.551),2年无GVHD无复发生存(GRFS)率分别为55.3%(95%CI 44.8%~64.8%)、64.7%(95%CI 52.8%~79.3%)(P=0.284)。结论haplo-cord-PBSCT与UD-PBSCT治疗恶性血液病具有相似的疗效和安全性,可作为恶性血液病的替代治疗方案。

Objectives To assess the efficacy of cord blood-assisted haploid peripheral blood stem cell transplantation(haplo-cord-PBSCT)versus unrelated donor peripheral blood stem cell transplantation(UD-PBSCT)in the treatment of malignant hematological diseases.Methods A retrospective analysis was performed on one hundred and four patients with malignant hematological diseases who underwent haplo-cord-PBSCT and fifty-two patients who underwent UD-PBSCT at Xiangya Hospital of Central South University between January 2016 and December 2021.Results The median implantation time for neutrophils in the haplo-cord-PBSCT and UD-PBSCT groups was 13(9-22)days and 13(10-24)days,respectively(P=0.834),whereas the median implantation time for platelets was 15(7-103)days and 14(8-38)days,respectively(P=0.816).The cumulative implantation rate of neutrophils at 30 days after transplantation in the haplo-cord-PBSCT group and the UD-PBSCT group was 100%(P=0.314),and the cumulative platelet implantation rate at 100 days after transplantation was 95.2%(95%CI 88.3%-98.1%)and 100%(P=0.927),respectively.30 days after transplantation,both groups of patients achieved complete donor chimerism,and no umbilical cord blood stem cells were implanted.②The cumulative incidence rates of grade Ⅱ-IV acute GVHD within 100 days after transplantation in the haplo-cord-PBSCT group and the UD-PBSCT group were 29.1%(95%CI 20.1%-38.1%)and 28.8%(95%CI 17.2%-41.6%)(P=0.965),respectively.The cumulative incidence rates of grade Ⅲ/IV acute GVHD were 7.8%(95%CI 3.6%-14.0%)and 9.6%(95%CI 3.5%-19.5%)(P=0.725).The cumulative incidence rates of 2-year chronic GVHD in the haplo-cord-PBSCT group and the UD-PBSCT group were 45.3%(95%CI 36.1%-56.1%)and 35.1%(95%CI 21.6%-44.1%),respectively(P=0.237).The cumulative incidence rates of severe chronic GVHD at 2 years after transplantation were 13.6%(95%CI 7.6%-21.3%)and 12.9%(95%CI 5.1%-24.3%),respectively(P=0.840).③The 2-year CIR after transplantation in the haplo-cord-PBSCT group and UD-PBSCT group were 12.8%(95%CI 7.0%-20.5%)and 10.0%(95%CI 3.6%-20.2%),respectively(P=0.341),and the NRM were 14.7%(95%CI 8.4%-22.6%)and 16.2%(95%CI 7.4%-28.0%),respectively(P=0.681).①The 2-year OS rates in the haplo-cord-PBSCT and UD-PBSCT groups after transplantation were 82.2%(95%CI 74.8%-90.3%)and 75.5%(95%CI 64.2%-88.7%),respectively(P=0.276).The 2-year DFS rates were 69.9%(95%CI 61.2%-79.8%)and 73.8%(95%CI 62.4%-87.3%),respectively(P=0.551).The 2-year rates of GVHD-free/recurrence-free survival(GRFS)were 55.3%(95%CI 44.8%-64.8%)and 64.7%(95%CI 52.8%-79.3%),respectively(P=0.284).Conclusion The findings of this study indicate that haplo-cord-PBSCT and UD-PBSCT have comparable efficacy and safety in the treatment of malignant hematological diseases and can be used as an alternative treatment options.