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一名重症再生障碍性贫血患儿在两次移植失败后成功进行了第三次单倍体相合造血干细胞移植:一项五年随访的病例报告

Successful third haploidentical hematopoietic stem cell transplantation after two graft failures in a pediatric patient with severe aplastic anemia: a case report with five-year follow-up.

作者信息

Li Xuewei, Zhang Wenhui, Li Saisai, Ma Xiaolin, Shi Xue, Wang Wei, Sun Lingjie, Qiu Kuan, Zhao Yanxia, Zhao Chunting, Liu Xiaodan

机构信息

Department of Hematology, The Affiliated Hospital of Qingdao University, Qingdao University, Qingdao, Shandong, China.

Department of Bone Marrow Transplantation, The Affiliated Hospital of Qingdao University, Qingdao University, Qingdao, Shandong, China.

出版信息

Front Immunol. 2025 Jul 9;16:1607926. doi: 10.3389/fimmu.2025.1607926. eCollection 2025.

DOI:10.3389/fimmu.2025.1607926
PMID:40703529
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12284796/
Abstract

BACKGROUND

Hematopoietic stem cell transplantation (HSCT) offers a potentially curative option for severe aplastic anemia (SAA). However, graft failure (GF) remains a life-threatening complication following HSCT. Haploidentical HSCT may serve as an effective salvage therapy for the treatment of GF.

CASE PRESENTATION

This report describes a 3-year-old girl with acquired SAA who experienced GF twice following matched unrelated donor (MUD) transplantations. Successful engraftment was ultimately achieved through a third haploidentical donor HSCT. This work was conducted in accordance with the Declaration of Helsinki and the Declaration of Istanbul.

CONCLUSIONS

Based on our experience with this case, we conclude that a third HSCT with a haploidentical donor represents a viable approach to extending survival.

摘要

背景

造血干细胞移植(HSCT)为重型再生障碍性贫血(SAA)提供了一种潜在的治愈选择。然而,移植失败(GF)仍然是HSCT后一种危及生命的并发症。单倍体相合HSCT可作为治疗GF的一种有效挽救疗法。

病例报告

本报告描述了一名3岁获得性SAA女童,在接受匹配无关供体(MUD)移植后发生了两次GF。最终通过第三次单倍体相合供体HSCT成功实现了植入。本研究是根据《赫尔辛基宣言》和《伊斯坦布尔宣言》进行的。

结论

基于我们对该病例的经验,我们得出结论,第三次单倍体相合供体HSCT是延长生存期的一种可行方法。

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本文引用的文献

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Allogeneic Hematopoietic Cell Transplantation for the Treatment of Severe Aplastic Anemia: Evidence-Based Guidelines From the American Society for Transplantation and Cellular Therapy.异基因造血细胞移植治疗重型再生障碍性贫血:美国移植与细胞治疗学会基于证据的指南
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Modified Delphi panel consensus recommendations for management of severe aplastic anemia.改良 Delphi 法-panel 共识推荐的再生障碍性贫血严重程度的管理建议。
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再生障碍性贫血的外周血干细胞移植与骨髓移植:一项系统评价和荟萃分析
Front Med (Lausanne). 2023 Nov 22;10:1289180. doi: 10.3389/fmed.2023.1289180. eCollection 2023.
4
Long-term outcome of second allogeneic hematopoietic stem cell transplantation (HSCT2) for primary graft failure in patients with acute leukemia in remission: A study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.缓解期急性白血病患者原发移植物失败后行第二次异基因造血干细胞移植(HSCT2)的长期疗效:一项代表欧洲血液和骨髓移植学会急性白血病工作组的研究。
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Long-term follow-up of haploidentical transplantation in relapsed/refractory severe aplastic anemia: a multicenter prospective study.单倍型相合移植治疗复发/难治性重型再生障碍性贫血的长期随访:一项多中心前瞻性研究
Sci Bull (Beijing). 2022 May 15;67(9):963-970. doi: 10.1016/j.scib.2022.01.024. Epub 2022 Jan 31.
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Effect of Donor Lymphocyte Infusion From Two Types of Donors on Mixed Chimerism With Secondary Graft Failure After Allogeneic Hematopoietic Stem Cell Transplantation.两种供者来源的供者淋巴细胞输注对异基因造血干细胞移植后混合嵌合体伴二次移植物失败的影响。
Transplant Cell Ther. 2022 Mar;28(3):152.e1-152.e7. doi: 10.1016/j.jtct.2021.12.017. Epub 2021 Dec 29.
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Transplant Cell Ther. 2021 Aug;27(8):642-649. doi: 10.1016/j.jtct.2021.04.007.
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Secondary haploidentical hematopoietic stem cell transplantation in patients with relapse or graft failure after initial hematopoietic stem cell transplantation.初次造血干细胞移植后复发或移植物失败患者的二次单倍型相合造血干细胞移植。
Ann Hematol. 2019 Dec;98(12):2833-2836. doi: 10.1007/s00277-019-03840-6. Epub 2019 Nov 18.
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Aplastic Anemia Preconditioned with Fludarabine, Cyclophosphamide, and Anti-Thymocyte Globulin.氟达拉滨、环磷酰胺和抗胸腺细胞球蛋白预处理的再生障碍性贫血
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Role of interferon-γ in immune-mediated graft failure after allogeneic hematopoietic stem cell transplantation.干扰素-γ 在异基因造血干细胞移植后免疫介导的移植物失败中的作用。
Haematologica. 2019 Nov;104(11):2314-2323. doi: 10.3324/haematol.2019.216101. Epub 2019 Feb 21.