Department of Hematology, Fondazione Policlinico Universitario Gemelli IRCCS, Universita' Cattolica del Sacro Cuore, Rome, Italy.
Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):467-473. doi: 10.1182/asheducation-2018.1.467.
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.
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。