Stauder Reinhard, Wimazal Friedrich, Nösslinger Thomas, Krieger Otto, Sperr Wolfgang R, Sill Heinz, Pfeilstöcker Michael, Valent Peter
Division of Hematology and Oncology, Department of Internal Medicine, Innsbruck Medical University, Innsbruck, Austria.
Wien Klin Wochenschr. 2008;120(17-18):523-37. doi: 10.1007/s00508-008-1058-6.
Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis and a tendency to transition to acute myeloid leukemia. Due to the increasing number of older patients in Austria and the high frequency of therapy-associated MDS following successful chemo- and/or radiotherapy of a primary tumor, the frequency and relevance of MDS are continuously increasing. While therapeutic options were until recently limited to best supportive care, AML-like induction chemotherapy and hematopoietic stem cell transplantation (HSCT) in younger patients, in recent years new therapeutic options have become available. Supportive care was improved through the introduction of effective iron chelation and the availability of hematopoietic growth factors like erythropoiesis-stimulating factors (ESF) and granulocyte colony-stimulating factors (G-CSF). In addition, immune-modulating drugs (IMiDs) like lenalidomide or epigenetically effective agents like the cytosine analogues or histone deacetylase (HDAC) inhibitors have become available and are highly effective in distinct subgroups of MDS patients. The development of state-of-the art recommendations is one of the major aims of the MDS Platform of the Austrian Society of Hematology and Oncology. This manuscript reviews recent developments in clinical scoring and targeted and individualized MDS therapy and discusses their relevance in and potential applicability to daily practice.
骨髓增生异常综合征(MDS)的特征是造血无效以及有向急性髓系白血病转变的倾向。由于奥地利老年患者数量不断增加,且原发性肿瘤成功进行化疗和/或放疗后治疗相关MDS的发生率较高,MDS的发病率和相关性在持续上升。虽然直到最近治疗选择仍局限于最佳支持治疗、针对年轻患者的类似急性髓系白血病的诱导化疗和造血干细胞移植(HSCT),但近年来已有了新的治疗选择。通过引入有效的铁螯合疗法以及可获得造血生长因子如促红细胞生成因子(ESF)和粒细胞集落刺激因子(G-CSF),支持治疗得到了改善。此外,来那度胺等免疫调节药物(IMiD)或胞嘧啶类似物或组蛋白脱乙酰酶(HDAC)抑制剂等表观遗传学有效药物已可获得,并且在MDS患者的不同亚组中具有高效性。制定最新的推荐是奥地利血液学和肿瘤学会MDS平台的主要目标之一。本文综述了临床评分以及靶向和个体化MDS治疗的最新进展,并讨论了它们在日常实践中的相关性和潜在适用性。