异基因造血干细胞移植治疗儿童骨髓衰竭性疾病69例临床分析

崔云平; 王叨; 刘健; 刘玉峰

doi:10.13201/j.issn.1004-2806.2020.11.012

异基因造血干细胞移植治疗儿童骨髓衰竭性疾病69例临床分析

- 郑州大学第一附属医院小儿内科(郑州, 450000)

详细信息

通讯作者: 刘玉峰,E-mail:lyf6012@163.com

中图分类号: R457.7

收稿日期: 2020-06-14

Clinical analysis of allogeneic hematopoietic stem cell transplantation in the treatment of 69 children with bone marrow failure

- Department of Pediatrics, the First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450000, China

More Information

Corresponding author: LIU Yu-feng, E-mail: lyf6012@163.com

Received Date: 14 June 2020

摘要

摘要: 目的:分析总结我院异基因造血干细胞移植（allo-HSCT）治疗儿童骨髓衰竭性疾病的疗效,为儿童allo-HSCT的临床实践提供依据。方法:对我院2015-12-2018-12进行的69例儿童骨髓衰竭性疾病allo-HSCT的临床资料进行回顾性总结和分析,探讨肝功能对造血重建及移植物抗宿主病（GVHD）发生的影响、患儿的长期生存情况以及影响预后的因素等。结果:植入率为95.65%（66/69）,不同供者类型中性粒细胞及血小板植入时间差异无统计学意义（P＞0.05）。急性GVHD发生率为37.68%,单倍体及脐血细胞移植组Ⅲ～Ⅳ度急性GVHD发生率较高（P=0.018）;慢性GVHD发生率为37.68%。预处理前肝功能异常可致植入时间延长[中性粒细胞植入平均时间（14.00±1.73） d,P＜0.001;血小板植入平均时间（33.36±8.08） d,P=0.001],急性GVHD发生率升高（72.73%,P=0.013）。有1例患者于移植后1个月内发生肝静脉栓塞综合征,5例发生巨细胞病毒或EB病毒感染,7例发生出血性膀胱炎,8例发生肺部感染,8例发生皮肤黏膜损伤。中位随访时间为26（1～48）个月,2年总生存率为92.65%,2年无事件生存率为89.72%。诊断到移植时间是影响患儿长期生存的危险因素（P＜0.05,HR=1.405,95%CI 1.143～1.727）。结论:肝功能异常可能影响患儿的造血重建,使植入时间延长,可能增加急性GVHD的发生率。单倍体造血干细胞移植对于患儿的长期生存未产生明显影响。诊断到移植时间是影响其长期生存的独立危险因素,因此对于重症骨髓衰竭性疾病患儿应尽早接受allo-HSCT。
- 异基因造血干细胞移植 /
- 骨髓衰竭性疾病 /
- 儿童 /
- 生存分析
Abstract: Objective:To analyze and summarize the curative effect of allogeneic hematopoietic stem cell transplantation（allo-HSCT） in children with bone marrow failure and to provide the basis for the clinical practice of children's allo-HSCT.Method:The clinical data of 69 children with allo-HSCT from December 2015 to December 2018 in our hospital were retrospectively summarized.We analyzed the effects of liver function on hematopoietic reconstruction and graft versus host disease（GVHD） occurrence,the long-term survival of children and the factors affecting prognosis.Result:The implantation rate was 95.65%（66/69）,and there was no significant difference in the implantation time of neutrophils and platelets in different donor types（P＞0.05）.The incidence of aGVHD was 37.68%,and the incidences of Ⅲ-Ⅳ degree aGVHD in haploid and UCBT were higher（P=0.018）;the incidence of cGVHD was 37.68%.The abnormal liver function before pretreatment could lead to prolonged implantation time [mean time of neutrophil implantation was（14.00±1.73） days,P＜0.001;mean time of platelet implantation was（33.36±8.08） days,P＜0.001] and increased incidence of aGVHD（72.73%,P=0.013）.One patient developed hepatic veno-occlusive disease within one month after transplantation,5 cases developed cytomegalovirus or EB virus infection,7 cases developed hemorrhagic cystitis,8 cases developed pulmonary infection,and 8 cases developed skin and mucosa injury.The median follow-up time was 26（1-48） months.The 2-year overall survival rate was 92.65%,and the 2-year event-free survival rate was 89.72%.The time from diagnosis to transplantation was a risk factor for long-term survival（P＜0.05,HR=1.405,95%CI 1.143-1.727）.Conclusion:Abnormal liver function may affect the hemopoietic reconstruction of children,prolong the implantation time,and increase the incidence of aGVHD.Amploid hematopoietic stem cell transplantation has no obvious effect on the long-term survival of children.Diagnosis of transplantation time is an independent risk factor for their long-term survival,so children with severe bone marrow failure should receive allo-HSCT as soon as possible.
- allogeneic hematopoietic stem cell transplantation /
- bone marrow failure disease /
- children /
- survival analysis

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