Clinical Research Division, Fred Hutchinson Cancer Research Center, and the University of Washington School of Medicine, Seattle, Washington, USA.
Curr Opin Hematol. 2013 Nov;20(6):494-500. doi: 10.1097/MOH.0b013e328364f547.
The only current treatment capable of curing patients with myelodysplastic syndromes (MDS) is allogeneic haematopoietic stem cell transplantation (HCT). However, many MDS patients are older, often with substantial comorbid 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 nontransplanted 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 a 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 to transplant 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 eighth 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.
目前唯一能够治愈骨髓增生异常综合征（MDS）患者的方法是异基因造血干细胞移植（HCT）。然而，许多 MDS 患者年龄较大，常伴有严重的合并症，且疾病存在异质性。因此，HCT 的结果差异很大，MDS 的 HCT 实践也在不断发展。
新发表的改良国际预后评分系统（IPSS-R），专为未接受移植的患者开发，也与 HCT 后的结果相关，患者的核型影响最大。单体核型和各种遗传及分子标志物的存在也具有预后价值。在 HCT 之前或之后使用低甲基化剂，可能会降低 HCT 后复发的风险或延迟复发。低强度和减轻强度的预处理方案使越来越多的年龄较大的 MDS 患者能够接受移植，新型方案的开发可能会导致即使在细胞遗传学高危患者中也能提高无复发生存率。最佳的干细胞来源可能因患者人群和疾病风险类别而异。
近年来，MDS 的移植结果有所改善。一些甚至八十多岁的患者也成功接受了移植。正在进行的研究旨在进一步降低移植相关毒性、移植物抗宿主病和 HCT 后复发的风险。
