Department of Hematologic Oncology and Blood Disorders, Cleveland Clinic Taussig Cancer Institute, Cleveland, OH 44195, USA.
Hematology Am Soc Hematol Educ Program. 2009:656-63. doi: 10.1182/asheducation-2009.1.656.
As opposed to the treatment landscape for myelodysplastic syndromes (MDS) two decades ago, potential therapies now abound for the treatment of lower-risk and higher-risk populations. In lower-risk patients, decision tools can be used to determine the likelihood of response to erythropoiesis stimulating agents (ESAs), which have demonstrated survival advantages in retrospective studies in patients with MDS, and whether these patients should be treated initially with ESAs or non-growth factor ("active") therapies. Lenalidomide has shown good activity in transfusion-dependent patients with the del(5q) cytogenetic abnormality and modest activity in other lower-risk patients. In higher-risk patients, the DNA methyltransferase inhibitors produce complete and partial responses in 20% to 30% of patients, and for the first time, the MDS drug azacitidine has demonstrated a survival advantage when compared with conventional therapies. Newer therapies stimulate platelet production and target novel pathways, while a panoply of combination studies are underway or recently completed and that likely represent the next frontier in MDS therapy.
与二十年前骨髓增生异常综合征 (MDS) 的治疗现状相比，现在有很多潜在的治疗方法可以用于治疗低危和高危人群。在低危患者中，可以使用决策工具来确定对红细胞生成刺激剂 (ESA) 反应的可能性，在 MDS 患者的回顾性研究中，ESA 已显示出生存优势，并且这些患者是否应该最初用 ESA 或非生长因子（“活性”）治疗。来那度胺在依赖输血的 del(5q) 细胞遗传学异常患者中显示出良好的活性，在其他低危患者中也有适度的活性。在高危患者中，DNA 甲基转移酶抑制剂在 20%至 30%的患者中产生完全和部分缓解，并且 MDS 药物阿扎胞苷首次与常规治疗相比显示出生存优势。新型疗法刺激血小板生成并针对新的途径，同时正在进行或最近完成了一系列联合研究，这可能代表 MDS 治疗的下一个前沿领域。
