Clinical significance of screening donor related genes for allogeneic hematopoietic stem cell transplantation in treatment of inherited bone marrow failure syndrome in adults and analysis on clinical efficacy

Lidan ZHU; Xi ZHANG; Peiyan KONG; Yao LIU; Cheng ZHANG; Li GAO; Yingying MA; Xixi XIANG; Jun LIU; Lei GAO

doi:10.16016/j.1000-5404.202006089

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Clinical significance of screening donor related genes for allogeneic hematopoietic stem cell transplantation in treatment of inherited bone marrow failure syndrome in adults and analysis on clinical efficacy

更新时间：2026-01-09

- Clinical significance of screening donor related genes for allogeneic hematopoietic stem cell transplantation in treatment of inherited bone marrow failure syndrome in adults and analysis on clinical efficacy
- Journal of Third Military Medical University Vol. 42, Issue 17, Pages: 1730-1734(2020)
- 作者机构：
- 作者简介：
- 基金信息：
  
  Clinical Research Talent Training Plan of Army Medical University(2018XLC2012);Key Program of Natural Science Foundation of Chongqing(CSTC2019jcyj-zdxmX0023);Open Project of the Key Laboratory of Tumor Immunopathology of Ministry of Education(2018jsz102)
- DOI：10.16016/j.1000-5404.202006089
  CLC：
- Received：10 June 2020，
  
  Published：15 September 2020
- 稿件说明：
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Lidan ZHU, Xi ZHANG, Peiyan KONG, et al. Clinical significance of screening donor related genes for allogeneic hematopoietic stem cell transplantation in treatment of inherited bone marrow failure syndrome in adults and analysis on clinical efficacy[J]. Journal of Third Military Medical University, 2020, 42(17): 1730-1734.
DOI：

Lidan ZHU, Xi ZHANG, Peiyan KONG, et al. Clinical significance of screening donor related genes for allogeneic hematopoietic stem cell transplantation in treatment of inherited bone marrow failure syndrome in adults and analysis on clinical efficacy[J]. Journal of Third Military Medical University, 2020, 42(17): 1730-1734. DOI： 10.16016/j.1000-5404.202006089.

摘要

Abstract

Objective

To investigate the effect of screening donor related genes for allogeneic hematopoietic stem cell transplantation (allo-HSCT) on the efficacy of the transplantation for adult patients with inherited bone marrow failure syndrome (IBMFS).

Methods

Clinical data of 65 adult BMFS patients undergoing allo-HSCT in our medical center from July 2014 to February 2019 were collected in this study. According to the results of bone marrow failure gene detection

the positive ones were assigned into IBMFS group and the negative into acquired bone marrow failure syndrome (ABMFS). Donor selection for IBMFS patients were performed based on the results of bone marrow failure gene of donor. While

for ABMFS patients

HLA-sibling donors

unrelated donors or HLA-haploid matched donors were selected according to the results of HLA matching. Then the hematopoietic reconstitution

transplantation related complications and total survival was observed.

Results

IBMFS was diagnosed in 5 patients out of the 65 patients (7.7%) due to positivity of bone marrow failure gene detection. Among the 5 IBMFS patients

3 cases underwent unrelated HLA-matched allo-HSCT because hematopoietic failure genes were detected in their related donors

and the other 2 cases received HLA-haploid allo-HSCT from their healthy related donors. There were no significant differences in donor hematopoietic implantation

acute and chronic graft-versus-host diseases

fever and cytomegalovirus infection between the IBMFS group and the ABMFS group. Till September 2019

all the 5 patients from the IBMFS group were survived

so were the 58 patients from the 60 ABMFS patients.

Conclusion

As long as healthy donors are selected according to bone marrow failure gene detection

adult IBMFS patients can achieve the same effect as ABMFS patients after allo-HSCT.

关键词

Keywords

references

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