Universitätsklinikum Carl-Gustav-Carus, Medizinische Klinik I, Dresden, Germany.
Semin Hematol. 2012 Oct;49(4):342-9. doi: 10.1053/j.seminhematol.2012.07.003.
Despite new developments in innovative and potentially targeted drugs like hypomethylating agents (HMA), allogeneic hematopoietic stem cell transplantation (HCT) is still the only potentially curative treatment option for patients with myelodysplastic syndromes (MDS). Improvements in donor selection and supportive care, as well as the introduction of reduced-intensity conditioning, have improved the feasibility of this approach by reducing early mortality. As a result, the number of allogeneic HCTs performed in MDS patients mainly above the age of 60 years has significantly increased over the last decade. However, the main drawbacks of this procedure, including graft-versus-host disease (GVHD) and hematologic relapse, remain a challenge in daily patient care. Thus, optimization of the pretransplant remission status and post-transplant outcome, eg, by new GVHD prophylaxis regimens or pharmacological maintenance or pre-emptive therapy of minimal residual disease to prevent relapse, is an important goal of current clinical research. There is an urgent need for prospective randomized trials to identify the subgroup of MDS patients who could achieve a better long-term disease control by allogeneic HCT compared to treatment with current standards of care, including HMA.
尽管新型创新药物和潜在靶向药物（如低甲基化药物）有了新的发展，但异基因造血干细胞移植（HCT）仍然是骨髓增生异常综合征（MDS）患者唯一潜在的治愈性治疗选择。通过减少早期死亡率，提高了供者选择和支持治疗的改进，以及引入减强度预处理，使这种方法的可行性得到了提高。因此，过去十年中，在年龄超过 60 岁的 MDS 患者中进行的异基因 HCT 数量显著增加。然而，该手术的主要缺点，包括移植物抗宿主病（GVHD）和血液学复发，仍然是日常患者护理中的一个挑战。因此，优化移植前缓解状态和移植后结果，例如通过新的 GVHD 预防方案或药物维持或最小残留疾病的抢先治疗以预防复发，是当前临床研究的一个重要目标。迫切需要前瞻性随机试验来确定 MDS 患者亚组，与当前的护理标准（包括 HMA）相比，通过异基因 HCT 可以获得更好的长期疾病控制。
