Meletis J, Terpos E
First Department of Internal Medicine, National and Kapodistrian University of Athens, School of Medicine, Laikon General Hospital, Athens, Greece.
J BUON. 2009 Oct-Dec;14(4):551-64.
Allogeneic stem cell transplantation (SCT) is the only therapeutic modality at present that may be delivered with curative intent in patients with myelodysplastic syndromes (MDS). Allogeneic CST replaces recipient dysplastic hemopoiesis with healthy donor haemopoiesis and immune system with an attendant graft-versus-leukemia (GvL) effect. Its applicability, however, is limited by the age of MDS patients, high rates of transplant-related mortality (TRM) and availability of a suitable HLA-matched donor. Results from several large centres indicated 3-year overall survival (OS) rates of 20-45%, which are almost equal with the results obtained by intensive chemotherapy alone. Failure was due primarily to TRM in patients with low-risk MDS and to disease recurrence in patients with high-risk MDS. Allogeneic SCT from matched unrelated donors produce poorer results than matched related siblings' transplantations. In an attempt to reduce TRM and deliver allogeneic SCT in a greater subgroup of MDS patients, many researchers used reduced-intensity allografts (RIC or "mini"-allograft) for MDS. Although differences in patient populations, preparative regimens, and graft-versus-host disease (GvHD) prophylaxis, as well as donor source (related vs. unrelated) have to be considered, OS of up to 40% at 3 years and disease-free survival (DFS) rates of almost 35% at 3 years have been reported in selected centres. However, randomized prospective studies are needed to further address the optimal choice of transplant conditioning intensity in MDS. Autologous SCT has been extremely investigated in MDS. It is limited to patients who have achieved a complete remission (CR), can be harvested, and are candidates for the procedure. Autologous SCT after successful induction chemotherapy may increase the proportion of long-term survivors, thus improving CR duration in some patients with MDS, particularly in younger patients in remission. Results for older patients are unsatisfactory. The relapse rate is up to 75%, with a 2-year probability of DFS of only 25% for patients 40-60 years of age. Therefore, there is very limited enthusiasm for the future of autologous SCT in the management of MDS patients.
异基因干细胞移植（SCT）是目前唯一一种可用于有治愈意向的骨髓增生异常综合征（MDS）患者的治疗方式。异基因SCT用健康供体的造血和免疫系统替代受体发育异常的造血，同时伴有移植物抗白血病（GvL）效应。然而，其适用性受到MDS患者年龄、移植相关死亡率（TRM）高以及合适的HLA匹配供体可用性的限制。几个大型中心的结果表明，3年总生存率（OS）为20% - 45%，这与单独强化化疗的结果几乎相当。失败主要是由于低风险MDS患者的TRM以及高风险MDS患者的疾病复发。来自匹配无关供体的异基因SCT比匹配相关同胞移植的结果更差。为了降低TRM并在更大的MDS患者亚组中进行异基因SCT，许多研究人员对MDS采用了减低强度的同种异体移植（RIC或“微型”移植）。尽管必须考虑患者群体、预处理方案、移植物抗宿主病（GvHD）预防以及供体来源（相关与无关）的差异，但在选定的中心报告的3年OS高达40%，3年无病生存率（DFS）接近35%。然而，需要进行随机前瞻性研究以进一步确定MDS移植预处理强度的最佳选择。自体SCT在MDS中已得到广泛研究。它仅限于已实现完全缓解（CR）、能够采集且适合该手术的患者。成功诱导化疗后的自体SCT可能会增加长期存活者的比例，从而延长一些MDS患者的CR持续时间，特别是在缓解期的年轻患者中。老年患者的结果不理想。复发率高达75%，40 - 60岁患者的2年DFS概率仅为25%。因此，对于自体SCT在MDS患者管理中的未来前景，人们的热情非常有限。
