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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.

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。

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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。
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