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1
Alternative donor transplants for severe aplastic anemia.异体供者移植治疗重型再生障碍性贫血。
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2
Beneficial Role of Low-Dose Antithymocyte Globulin in Unrelated Stem Cell Transplantation for Adult Patients with Acquired Severe Aplastic Anemia: Reduction of Graft-versus-Host Disease and Improvement of Graft-versus-Host Disease-Free, Failure-Free Survival Rate.低剂量抗胸腺细胞球蛋白在成人获得性重型再生障碍性贫血无关供者干细胞移植中的有益作用:降低移植物抗宿主病并提高无移植物抗宿主病、无失败生存率
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3
Alternative donor transplants for severe aplastic anemia: current experience.重型再生障碍性贫血的替代供体移植:当前经验
Semin Hematol. 2016 Apr;53(2):115-9. doi: 10.1053/j.seminhematol.2016.01.002. Epub 2016 Jan 15.
4
Allogeneic hematopoietic stem cell transplantation in aplastic anemia: current indications and transplant strategies.再生障碍性贫血的异基因造血干细胞移植:当前的适应证及移植策略
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Effect of Antithymocyte Globulin Source on Outcomes of HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Severe Aplastic Anemia.抗胸腺细胞球蛋白来源对重型再生障碍性贫血患者 HLA 匹配同胞异基因造血干细胞移植结局的影响。
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Impact of T-cell depletion strategies on outcomes following hematopoietic stem cell transplantation for idiopathic aplastic anemia: A study on behalf of the European blood and marrow transplant severe aplastic anemia working party.T 细胞耗竭策略对特发性再生障碍性贫血造血干细胞移植后结局的影响:代表欧洲血液和骨髓移植严重再生障碍性贫血工作组的一项研究。
Am J Hematol. 2019 Jan;94(1):80-86. doi: 10.1002/ajh.25314. Epub 2018 Nov 25.
7
Reduced-Intensity Conditioning with Busulfan, Fludarabine, and Antithymocyte Globulin for Hematopoietic Cell Transplantation from Unrelated or Haploidentical Family Donors in Patients with Acute Myeloid Leukemia in Remission.使用白消安、氟达拉滨和抗胸腺细胞球蛋白进行减低强度预处理用于缓解期急性髓系白血病患者接受无关或单倍体相合家庭供者造血细胞移植
Biol Blood Marrow Transplant. 2017 Sep;23(9):1555-1566. doi: 10.1016/j.bbmt.2017.05.025. Epub 2017 May 25.
8
Treatment of adults with severe aplastic anemia: primary therapy with antithymocyte globulin (ATG) and rescue of ATG failures with bone marrow transplantation.成人重型再生障碍性贫血的治疗:抗胸腺细胞球蛋白(ATG)作为初始治疗及骨髓移植挽救ATG治疗失败的患者
Am J Med. 1992 Jun;92(6):596-602. doi: 10.1016/0002-9343(92)90776-8.
9
Effect of antithymocyte globulin source on outcomes of bone marrow transplantation for severe aplastic anemia.抗胸腺细胞球蛋白来源对重型再生障碍性贫血骨髓移植结局的影响。
Haematologica. 2017 Jul;102(7):1291-1298. doi: 10.3324/haematol.2017.164459. Epub 2017 Mar 24.
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Allogeneic hematopoietic stem cell transplantation in children with aplastic anemia.再生障碍性贫血患儿的异基因造血干细胞移植
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2
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Case Series Using Salvage Haplo-Identical Stem Cells for Secondary Transplantation.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。
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The efficiency of human umbilical cord mesenchymal stem cells as a salvage treatment for steroid-refractory acute graft-versus-host disease.人脐带间充质干细胞作为挽救治疗类固醇耐药性急性移植物抗宿主病的疗效。
Clin Exp Med. 2023 Oct;23(6):2561-2570. doi: 10.1007/s10238-022-00983-1. Epub 2023 Jan 4.
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A multicentre trial of intensive immunosuppressive therapy combined with umbilical cord blood for the treatment of severe aplastic anaemia.一项多中心试验,研究强化免疫抑制疗法联合脐带血治疗严重再生障碍性贫血。
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本文引用的文献

1
Transplant outcome for patients with acquired aplastic anemia over the age of 40: has the outcome improved?40岁以上获得性再生障碍性贫血患者的移植结局:结局是否有所改善?
Blood. 2018 Apr 26;131(17):1989-1992. doi: 10.1182/blood-2017-09-807859. Epub 2018 Mar 16.
2
Is the use of unrelated donor transplantation leveling off in Europe? The 2016 European Society for Blood and Marrow Transplant activity survey report.在欧洲,无关供者移植的应用是否达到平稳状态?2016 年欧洲血液和骨髓移植学会活动调查报道。
Bone Marrow Transplant. 2018 Sep;53(9):1139-1148. doi: 10.1038/s41409-018-0153-1. Epub 2018 Mar 14.
3
Improved outcome of haploidentical transplantation in severe aplastic anemia using reduced-intensity fludarabine-based conditioning.使用基于氟达拉滨的低强度预处理改善重型再生障碍性贫血单倍体移植的结局。
Oncotarget. 2017 Jul 31;8(48):83817-83830. doi: 10.18632/oncotarget.19745. eCollection 2017 Oct 13.
4
Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.艾曲泊帕添加至标准免疫抑制方案用于治疗再生障碍性贫血
N Engl J Med. 2017 Apr 20;376(16):1540-1550. doi: 10.1056/NEJMoa1613878.
5
Effect of antithymocyte globulin source on outcomes of bone marrow transplantation for severe aplastic anemia.抗胸腺细胞球蛋白来源对重型再生障碍性贫血骨髓移植结局的影响。
Haematologica. 2017 Jul;102(7):1291-1298. doi: 10.3324/haematol.2017.164459. Epub 2017 Mar 24.
6
Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant.前瞻性单倍体相合移植治疗获得性重型再生障碍性贫血:与匹配相关移植的基于注册的比较。
J Hematol Oncol. 2017 Jan 21;10(1):25. doi: 10.1186/s13045-017-0398-y.
7
How I treat acquired aplastic anemia.我如何治疗获得性再生障碍性贫血。
Blood. 2017 Mar 16;129(11):1428-1436. doi: 10.1182/blood-2016-08-693481. Epub 2017 Jan 17.
8
Alternative Donor Transplantation with High-Dose Post-Transplantation Cyclophosphamide for Refractory Severe Aplastic Anemia.采用大剂量移植后环磷酰胺的替代供体移植治疗难治性重型再生障碍性贫血
Biol Blood Marrow Transplant. 2017 Mar;23(3):498-504. doi: 10.1016/j.bbmt.2016.12.628. Epub 2016 Dec 21.
9
Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study.接受非亲缘骨髓移植的重型再生障碍性贫血患者的环磷酰胺预处理:一项1-2期剂量递减研究
Lancet Haematol. 2015 Sep;2(9):e367-75. doi: 10.1016/S2352-3026(15)00147-7. Epub 2015 Sep 2.
10
Guidelines for the diagnosis and management of adult aplastic anaemia.成人再生障碍性贫血诊断与治疗指南。
Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16.

异体供者移植治疗重型再生障碍性贫血。

Alternative donor transplants for severe aplastic anemia.

机构信息

Department of Hematology, Fondazione Policlinico Universitario Gemelli IRCCS, Universita' Cattolica del Sacro Cuore, Rome, Italy.

出版信息

Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):467-473. doi: 10.1182/asheducation-2018.1.467.

DOI:10.1182/asheducation-2018.1.467
PMID:30504347
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6245994/
Abstract

Allogeneic hematopoietic stem-cell transplantation remains the only curative treatment for patients with acquired severe aplastic anemia (SAA). When a matched sibling is not available, one can search for a matched unrelated donor or a cord blood unit (CB) in the international registries or, more recently, for an HLA haploidentical (HAPLO) family member. International guidelines call for a course of antithymocyte globulin (ATG) and cyclosporine before a patient with SAA receives a transplant from a donor other than an HLA identical sibling, but whether this is necessary for patients age <20 years is less clear. Here I will examine the rapid increase in HAPLO transplantations for SAA, showing encouraging early results both in children and young adults. Graft-versus-host disease prophylaxis remains of primary importance in patients with SAA, and in vivo T-cell depletion with either ATG or alemtuzumab offers a significant survival advantage. Finally, I will discuss the strong age effect, which is particularly evident at >40 and 50 years of age for reasons not entirely clear and which should be taken into account when designing a treatment strategy for a given patient.

摘要

同种异体造血干细胞移植仍然是获得性严重再生障碍性贫血(SAA)患者的唯一治愈性治疗方法。当没有匹配的兄弟姐妹时,可以在国际登记处寻找匹配的无关供体或脐带血单位(CB),或者最近,可以寻找 HLA 单倍体(HAPLO)家庭成员。国际指南要求在接受非 HLA 同型兄弟姐妹供体移植的 SAA 患者之前,先使用抗胸腺细胞球蛋白(ATG)和环孢菌素进行治疗,但对于年龄<20 岁的患者是否有必要,目前还不太清楚。在这里,我将探讨 HAPLO 移植治疗 SAA 的快速增加,并展示在儿童和年轻成人中令人鼓舞的早期结果。移植物抗宿主病预防仍然是 SAA 患者的首要任务,用 ATG 或阿仑单抗进行体内 T 细胞耗竭可显著提高生存率。最后,我将讨论强烈的年龄效应,对于>40 岁和>50 岁的患者,这种效应尤其明显,但原因尚不完全清楚,在为特定患者设计治疗策略时应考虑到这一点。