Barrett A John, Savani Bipin N
Hematology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Semin Hematol. 2008 Jan;45(1):49-59. doi: 10.1053/j.seminhematol.2007.10.005.
Although it is only used to treat a minority of patients with myelodysplastic syndromes, stem cell transplantation (SCT) is the only proven curative treatment for this condition. Because MDS occurs in a population of older adults with significant comorbidities, reduced-intensity conditioning (RIC) regimens have been particularly important in extending safe SCT to the large MDS population over the age of 60 years. Extension of the unrelated donor pool together with the introduction of umbilical cord blood transplants in adults has extended the number of patients with suitable donors. Nevertheless overall mortality from SCT is greater than 50% because of relapse and non-relapse mortality (NRM). New developments to improve outcome include the tailoring of the transplant approach to the individual based on age and comorbidity, and the use of pretransplant chemotherapy to reduce disease bulk prior to transplant, as well as the introduction of post-transplant immunotherapy (pre-emptive donor lymphocyte infusions) and chemotherapy to prevent relapse. Further improvements in transplant outcome await better ways to reconstitute immunity and amplify the graft-versus-leukemia (GVL) effect without causing graft-versus-host disease (GVHD), as well as further extension of the donor pool and exploration of risk-adapted regimens for the population of MDS in their seventh to eighth decade.
尽管干细胞移植（SCT）仅用于治疗少数骨髓增生异常综合征患者，但它是这种疾病唯一经证实的治愈性疗法。由于骨髓增生异常综合征发生于有显著合并症的老年人群中，降低强度预处理（RIC）方案对于将安全的干细胞移植扩展至60岁以上的大量骨髓增生异常综合征患者尤为重要。无关供体库的扩大以及成人脐血移植的引入增加了有合适供体的患者数量。然而，由于复发和非复发死亡率（NRM），干细胞移植的总体死亡率超过50%。改善预后的新进展包括根据年龄和合并症为个体量身定制移植方法，在移植前使用化疗以减少疾病负荷，以及引入移植后免疫疗法（先发制人的供体淋巴细胞输注）和化疗以预防复发。移植预后的进一步改善有待于找到更好的方法来重建免疫并增强移植物抗白血病（GVL）效应而不引发移植物抗宿主病（GVHD），以及进一步扩大供体库并探索针对七、八十岁骨髓增生异常综合征患者群体的风险适应性方案。
