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采用挽救性单倍体相合干细胞进行二次移植的病例系列研究。

Case Series Using Salvage Haplo-Identical Stem Cells for Secondary Transplantation.

机构信息

Fundeni Clinical Institute, 022328 Bucharest, Romania.

Faculty of General Medicine, University of Medicine and Pharmacy "Carol Davila", 020021 Bucharest, Romania.

出版信息

Medicina (Kaunas). 2023 Jun 2;59(6):1077. doi: 10.3390/medicina59061077.

Abstract

In order to expand the donor pool and accessibility of the transplant procedure, it was necessary to introduce haplo-identical stem cell transplants in the Fundeni Clinical Institute from 2015. Even if the Romanian population is an ethnically compact white population, many of the patients referred for bone marrow transplant lack a suitable donor. Hematopoietic stem cell transplant from a haplo-identical donor is an alternative option for those patients without an HLA (Human Leucocyte Antigen)-matched donor (sibling or matched unrelated). This procedure was used also as a salvage option for those who experienced engraftment failure or the rejection of the first stem cell graft. In this case series, we present three such cases, with a haplo-transplant used as a salvage protocol (after an engraftment failure or rejection of the first transplanted cells). The patients we present were diagnosed with AML (acute myeloid leukemia) with MDS (myelodysplastic syndrome), MDS-RAEB 2 (myelodysplastic syndrome-refractory anemia with excess blasts 2), and SAA (severe aplastic anemia). In two of the three cases, the engraftment failure may have been due to the conditioning Fludarabine/Busulfan/Cyclophosphamide (Flu/Bu/CFA) used, combined with marrow grafts. In all three cases, the second transplant was of haplo-identical peripheral blood stem cells using Melphalan/Fludarabine (Mel/Flu) conditioning, the cells engrafted properly and the patients experienced complete chimerism, and two of them are alive with an excellent quality of life.

摘要

为了扩大供者群体并使移植手术更易获得,2015 年有必要在 Fundeni 临床研究所开展半相合干细胞移植。即使罗马尼亚人口是一个种族单一的白种人群体,许多接受骨髓移植的患者也缺乏合适的供者。对于那些没有 HLA(人类白细胞抗原)匹配供者(兄弟姐妹或匹配无关供者)的患者,来自半相合供者的造血干细胞移植是一种替代选择。对于那些经历植入失败或首次干细胞移植物排斥的患者,也可以将此程序用作挽救方案。在本病例系列中,我们介绍了三个这样的病例,使用半相合移植作为挽救方案(在植入失败或首次移植细胞排斥后)。我们介绍的患者被诊断为 AML(急性髓系白血病)伴 MDS(骨髓增生异常综合征)、MDS-RAEB 2(难治性贫血伴原始细胞增多 2 型骨髓增生异常综合征)和 SAA(重型再生障碍性贫血)。在这三个病例中的两个中,植入失败可能是由于使用的条件 Fludarabine/Busulfan/Cyclophosphamide(Flu/Bu/CFA)与骨髓移植物结合所致。在所有三个病例中,第二个移植均为使用 Melphalan/Fludarabine(Mel/Flu)条件的半相合外周血造血干细胞,细胞成功植入,患者经历了完全嵌合体,其中两人生活质量良好且仍然存活。

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Alternative donor transplants for severe aplastic anemia.异体供者移植治疗重型再生障碍性贫血。
Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):467-473. doi: 10.1182/asheducation-2018.1.467.

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