Transplantation for myelodysplastic syndromes 2013.
Transplantation for myelodysplastic syndromes 2013.
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DOI：
10.1097/moh.0b013e328364f547
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发表时间：
2013-11
影响因子：
3.2
通讯作者：
Deeg HJ
中科院分区：
文献类型：
--
作者：
Vaughn JE;Scott BL;Deeg HJ
目前唯一能够治愈骨髓增生异常综合征（MDS）患者的治疗方法是异体造血干细胞移植（HCT）。然而，许多MDS患者年龄较大，通常伴有大量合并症，并且该疾病具有异质性。因此，HCT的结果差异很大，MDS的HCT实践也在不断发展。新发布的针对非移植患者的改良国际预后评分系统（IPSS-R）也与hct后的预后相关，其中患者的核型影响最大。单染色体核型和各种遗传和分子标记的存在也被证明具有预后价值。在HCT之前或之后使用低甲基化药物可降低HCT后复发风险或延迟复发。低强度和低强度的调节方案已经允许越来越多的老年MDS患者进行移植，新方案的发展可能会提高无复发生存率，即使是高危细胞遗传学患者。对于不同的患者群体和不同的疾病风险类别，最佳的干细胞来源可能有所不同。近年来，MDS的移植效果有所改善。有些患者甚至在80岁时移植成功。正在进行的研究旨在进一步降低移植相关的毒性、移植物抗宿主病和hct后的复发。
The only current treatment capable of curing patients with myelodysplastic syndromes (MDS) is allogeneic hematopoietic stem cell transplant (HCT). However, many MDS patients are older, often with substantial co-morbid conditions, and the disease is heterogeneous. As a consequence, results of HCT vary considerably, and the practices of HCT for MDS are evolving. The newly published modified International Prognostic Scoring System (IPSS-R), developed for non-transplanted patients, also correlates with post-HCT outcome, with the patient’s karyotype having the strongest impact. The presence of monosomal karyotype and various genetic and molecular markers have also been shown to have prognostic value. The use of hypomethylating agents, before or after HCT, may reduce the post-HCT relapse risk or delay relapse. Low and reduced-intensity conditioning regimens have allowed transplant for growing numbers of older patients with MDS, and the development of novel regimens may lead to improved relapse-free survival even in patients with high-risk cytogenetics. The optimal stem cell source may differ for different patient populations and different disease risk categories. Transplant results for MDS have improved in recent years. Some patients even in the 8th decade of life have been transplanted successfully. Ongoing studies are aimed at further reducing transplant-related toxicity, graft-versus-host disease and post-HCT relapse.