不进行体外T细胞去除的单倍体相合造血干细胞移植治疗儿童血液系统恶性肿瘤

Haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for treatment of hematologic malignancies in children.

作者信息

Huang Xiaojun, Liu Daihong, Liu Kaiyan, Xu Lanping, Chen Huan, Han Wei, Chen Yuhong, Wang Yu, Zhang Xiaohui

机构信息

Peking University Institute of Hematology, People's Hospital, 11 South Street of Xizhimen, Xicheng District, Beijing, 100044, People's Republic of China.

出版信息

Biol Blood Marrow Transplant. 2009 Jan;15(1 Suppl):91-4. doi: 10.1016/j.bbmt.2008.10.019.

DOI:10.1016/j.bbmt.2008.10.019

PMID:19147085

Abstract

OBJECTIVE

To evaluate the efficacy and safety of haploidentical (from family member donors) hematopoietic stem cell transplantation (HSCT) for children.

PATIENTS AND METHODS

Fifty-eight children under fourteen years old with hematological malignancies underwent haploidentical HSCT. Outcomes were analyzed.

RESULTS

Of Fifty-eight patient/donor pairs, seven (12.1%) were mismatched in two HLA loci, twenty (34.5%) in three loci, and thirty-one (53.4%) in four loci. Follow-ups were performed for a median of 915 (227-1898) days after transplantation. All patients achieved stable engraftments. The cumulative incidence of acute graft-versus-host disease (GVHD) of grade 2-4 was 54.8%7.6%, and that of grade 3-4 was 11.4%4.8%. The cumulative incidence of chronic GVHD was 45.6%7.8% for total and 19.6%6.5% for extensive. Fourty patients survived with a 3-year probability of leukemia-free survival (LFS) 44.7%13.9%. Eighteen patients died, five from infection, eight from relapse of leukemia, two from heart failure, two from GVHD, and one from lymphoproliferative disorders.

CONCLUSION

The results encourage extending haploidentical HSCT without T-cell depletion treatments to children with an indication for transplantation.

摘要

目的

评估单倍体相合（来自家庭成员供者）造血干细胞移植（HSCT）治疗儿童的疗效和安全性。

患者与方法

58例14岁以下血液系统恶性肿瘤患儿接受了单倍体相合HSCT。对结果进行分析。

结果

58例患者/供者对中，7例（12.1%）在两个HLA位点不相合，20例（34.5%）在三个位点不相合，31例（53.4%）在四个位点不相合。移植后中位随访915（227 - 1898）天。所有患者均实现稳定植入。2 - 4级急性移植物抗宿主病（GVHD）的累积发生率为54.8%±7.6%，3 - 4级为11.4%±4.8%。慢性GVHD的累积发生率总体为45.6%±7.8%，广泛型为19.6%±6.5%。40例患者存活，3年无白血病生存（LFS）概率为44.7%±13.9%。18例患者死亡，5例死于感染，8例死于白血病复发，2例死于心力衰竭，2例死于GVHD，1例死于淋巴增殖性疾病。