Bacigalupo Andrea, Sica Simona
Department of Hematology, Universita' Cattolica del Sacro Cuore, Fondazione Policlinico Universitario Gemelli Roma, Largo Agostino Gemelli 1, Rome, Italy.
Department of Hematology, Universita' Cattolica del Sacro Cuore, Fondazione Policlinico Universitario Gemelli Roma, Largo Agostino Gemelli 1, Rome, Italy.
Semin Hematol. 2016 Apr;53(2):115-9. doi: 10.1053/j.seminhematol.2016.01.002. Epub 2016 Jan 15.
Patients with acquired severe aplastic anemia (SAA), who lack a human leukocyte antigen (HLA) identical sibling donor (SIB), have two therapeutic options: immunosuppressive therapy with anti-thymocyte globulin (ATG) and cyclosporine (CsA), or a transplant from an alternative donor. In these patients, the current guidelines of the European Group for Blood and Marrow Transplantation (EBMT) call for a course of ATG + CsA first and transplantation in case of no response. The alternative donor source can be an unrelated donor (UD), a cord blood (CB) unit, or a family mismatched member, in most instances genetically HLA haplo-mismatched (HAPLO). In the present review, we will discuss recent results of transplants from matched UD and SIB donors, with significantly improved outcome, especially with UD in the past decade. We will also be looking at CB transplants, and the problems of limited stem cell dose. Finally HAPLO grafts have been explored in patients lacking or having rejected an unrelated or CB graft: early results seem encouraging, though the procedure should still be considered experimental.
获得性重型再生障碍性贫血(SAA)患者若缺乏人类白细胞抗原(HLA)相合同胞供者(SIB),有两种治疗选择:使用抗胸腺细胞球蛋白(ATG)和环孢素(CsA)进行免疫抑制治疗,或接受来自替代供者的移植。对于这些患者,欧洲血液和骨髓移植组(EBMT)的现行指南要求先进行一个疗程的ATG + CsA治疗,若无效则进行移植。替代供者来源可以是无关供者(UD)、脐血(CB)单位或家族配型不合成员,在大多数情况下为基因HLA单倍型不合(HAPLO)。在本综述中,我们将讨论来自匹配的UD和SIB供者移植的近期结果,其结局显著改善,尤其是在过去十年中UD移植。我们还将审视CB移植以及干细胞剂量有限的问题。最后,已对缺乏或排斥无关或CB移植物的患者进行了HAPLO移植探索:早期结果似乎令人鼓舞,尽管该方法仍应被视为实验性的。