骨髓增生异常综合征。

Wolf-Karsten Hofmann, Michael Lübbert, Dieter Hoelzer, H Phillip Koeffler
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引用次数: 10

摘要

骨髓增生异常综合征(MDS)是一种以造血功能低下为特征的克隆性疾病,可导致致命性细胞减少症或急性髓性白血病(AML)。在过去的15年中,人们对MDS的生物学和预后的认识取得了重要进展。由于MDS患者的中位年龄(60-75岁)较高,以及该疾病的个人病史(细胞减少次数、细胞遗传学改变、输血需求),建立了适应风险的治疗策略。同种异体骨髓移植目前是MDS患者唯一可能治愈的治疗方法,但这种治疗方式不适用于>60岁的“典型”MDS患者。开发针对特定分子靶点且副作用最小的小分子是未来的希望。免疫调节剂的创新使用和细胞毒性治疗的优化将继续有助于MDS的治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Myelodysplastic syndromes.

Myelodysplastic syndrome (MDS) is a clonal disorder characterized by ineffective hematopoiesis, which can lead to either fatal cytopenias or acute myelogenous leukemias (AML). During the last 15 years, important progress has been made in the understanding of the biology and prognosis of MDS. Risk-adapted treatment strategies were established due to the high median age (60-75 years) of MDS patients and the individual history of the disease (number of cytopenias, cytogenetic changes, transfusion requirements). The use of allogeneic bone marrow transplantation for MDS patients currently offers the only potentially curative treatment, but this form of therapy is not available for the 'typical' MDS patient who is >60 years of age. The development of small molecules directed against specific molecular targets with minimal adverse effects is the hope for the future. Innovative uses of immunomodulatory agents and the optimizing of cytotoxic treatment should continue to help in the treatment of MDS.

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