Giralt Sergio
University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Box 462, Houston, TX 77030, USA.
Curr Hematol Rep. 2005 May;4(3):200-7.
Myelodysplastic syndrome (MDS) comprises a heterogeneous group of hematologic disorders characterized by a clonal abnormality of hematopoietic stem cells that results in a varying degree of cytopenias and risk of transformation into acute leukemia. Only allogeneic transplant has been shown to cure this disease. However, high nonrelapse mortality rates limit the applicability of conventional allografts and, even in young patients, long-term disease control ranging from only 30% to 50% has been reported. Novel transplant regimens, availability of alternative donors, and busulfan targeting promise to increase transplant applicability and reduce nonrelapse mortality rates. However, high relapse rates in patients with high-risk disease limit the success of this procedure to the point that a definite advantage of allografting over standard therapy remains controversial. New agents being developed for MDS may have a potential impact on transplant outcomes. Therefore, design and implementation of clinical trials of transplant for MDS should be encouraged to improve the natural history of this disease.
骨髓增生异常综合征（MDS）是一组异质性血液系统疾病，其特征为造血干细胞的克隆异常，可导致不同程度的血细胞减少以及转化为急性白血病的风险。只有异基因移植已被证明可治愈该疾病。然而，高非复发死亡率限制了传统同种异体移植的适用性，并且即使在年轻患者中，据报道长期疾病控制率也仅为30%至50%。新型移植方案、替代供体的可用性以及白消安靶向治疗有望提高移植适用性并降低非复发死亡率。然而，高危疾病患者的高复发率限制了该手术的成功率，以至于同种异体移植相对于标准治疗的明确优势仍存在争议。正在为MDS开发的新型药物可能会对移植结果产生潜在影响。因此，应鼓励设计和开展MDS移植的临床试验，以改善该疾病的自然病程。
