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

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。

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)andⅡ~ⅣaGVHD(P=0.001)in MSD+MUD group were significantly different,but the incidences ofⅢ~ⅣaGVHD,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.