中国小儿血液与肿瘤杂志2024,Vol.29Issue(1) :44-49.DOI:10.3969/j.issn.1673-5323.2024.01.009

单倍体供者与HLA相合供者HSCT治疗儿童MDS的疗效和安全性对比

Comparative analysis of the efficacy and safety of haploidentical donor transplantation and HLA matched donor transplantation in the treatment of children with myelodysplastic syndrome

戴银亮 夏子豪 胡轶歆 高莉 高伟 李捷 卢俊 肖佩芳 胡绍燕
中国小儿血液与肿瘤杂志2024,Vol.29Issue(1) :44-49.DOI:10.3969/j.issn.1673-5323.2024.01.009

单倍体供者与HLA相合供者HSCT治疗儿童MDS的疗效和安全性对比

Comparative analysis of the efficacy and safety of haploidentical donor transplantation and HLA matched donor transplantation in the treatment of children with myelodysplastic syndrome

戴银亮 1夏子豪 1胡轶歆 1高莉 1高伟 1李捷 1卢俊 1肖佩芳 1胡绍燕1
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作者信息

  • 1. 215025 苏州,苏州大学附属儿童医院
  • 折叠

摘要

目的 评价单倍体供者移植作为替代方案用于异基因造血干细胞移植(allo-HSCT)治疗儿童骨髓增生异常综合征(MDS)的疗效及安全性.方法 对2013年4月1日—2022年8月30日至苏州大学附属儿童医院接受allo-HSCT的22例儿童进展型MDS进行回顾性分析,其中全相合移植6例,单倍体移植16例.结果 22例患儿均实现中性粒细胞植入,全相合组和单倍体组中性粒细胞植入中位时间分别为移植后11(10~12)天和11(9~17)天,血小板植入中位时间分别为移植后11(8~16)天和12(7~28)天.两组aGVHD的发生率分别为50%和100%(P=0.013),Ⅱ~Ⅳ度aGVHD发生率分别为0%和81%(P=0.001),aGVHD发生时间、Ⅲ~Ⅳ度aGVHD、cGVHD、广泛型cGVHD发生率无统计学意义.单倍体组移植后围植入综合征发生率为81%,显著高于全相合组的17%(P=0.011).全相合组和单倍体组在CMV血症、EBV血症、出血性膀胱炎、肺部感染及神经系统并发症发生率上差异无统计学意义.至随访截止日期,全相合组中位随访时间为20.82(8.00~68.00)个月,单倍体组中位随访时间为21.38(2.77~69.77)个月,两组5年总体生存率分别为100%和(72.7±17.7)%,两组差异无统计学意义.结论 单倍体移植与同胞全相合移植后细胞植入、严重的移植相关并发症发生率、免疫重建及长期存活率无明显差异,因此,对于无全相合供者的儿童MDS患者,单倍体供体移植可作为合适的替代方案.

Abstract

Objective To evaluate efficacy and safety of haploidentical donor transplantation as an alternative to allogeneic hematopoietic stem cell transplantation(allo-HSCT)in children with myelodysplastic syndrome(MDS).Methods Clinical data of 22 children with MDS who received allo-HSCT from April 2013 to August 2022 in Children's Hospital of Suzhou University were collected.The patients were analyzed as follows:HLA matched donor(MSD+MUD)group and HLA-haploidentical related donor(HID)group.Results 22 cases were successfully engrafted with neutrophil and platelet.The median time of neutrophil and platelet engraftment were 11(10~12)and 11(9~17)days after transplantation in HID-HSCT group and MSD/MUD-HSCT group respectively.The median time of platelet engraftment were 11(8~16)and 12(7~28)days after transplantation respectively.Compared with HID group,the incidences of aGVHD(P=0.013)and Ⅱ~Ⅳ aGVHD(P=0.001)in MSD+MUD group were significantly different,but the incidences of Ⅲ~Ⅳ aGVHD,cGVHD,extensive cGVHD and the time to aGVHD were not significantly different.The incidence of preimplantation syndrome was 81.3%in the HID group,which was significantly higher than 17%in the MSD+MUD group(P=0.011).There were no significant difference in the incidence of CMV viremia,EBV viremia,hemorrhagic cystitis,pulmonary infection,and neurological complications between the MSD+MUD group and HID group.At the end of follow-up,the median follow-up time were 20.82(8.00~68.00)months in MSD+MUD group and 21.38(2.77~69.77)months in HID group.The 5-year OS rates were 100%and(72.7±17.7)%respectively.Conclusions There were no significant differences in cell engraftment,serious transplantation-related complications,immune reconstitution and long-term survival between haplo-HSCT and MSD/MUD-HSCT.Therefore,haplo-HSCT may be a suitable alternative for pediatric MDS patients who do not have a matched donor.

关键词

造血干细胞移植/儿童/骨髓增生异常综合征/单倍体供者移植

Key words

Hematopoietic stem cell transplantation/Child/Myelodysplastic syndrome/Haploidentical stem cell transplantation

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基金项目

江苏省科技厅项目(BE2021654)

苏州市科技计划(GSWS2020039)

苏州市科技计划(SZS201615)

国家临床研究中心血液系统疾病项目(2020ZKPB02)

出版年

2024
中国小儿血液与肿瘤杂志
中日友好医院,中国抗癌协会

中国小儿血液与肿瘤杂志

CSTPCD
影响因子:0.515
ISSN:1673-5323
参考文献量21
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