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Novel therapies in low- and high-risk myelodysplastic syndrome

机译：低风险骨髓增生综合征的新型疗法

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ABSTRACT Introduction: Myelodysplastic syndromes (MDS) comprise a heterogeneous group of myeloid neoplasms with diverse clinical courses. The revised version of the international prognostic scoring system (IPSS-R) provides risk stratification into 5 different groups. Areas covered: For lower-risk patients, red blood cell transfusions and iron chelation are the backbone of supportive care. In addition, erythropoiesis-stimulating agents (ESA) are used to ameliorate anemia. Lenalidomide is approved for the treatment of lower-risk patients with del(5q) who are transfusion-dependent. Patients with higher-risk disease should be offered allogeneic stem cell transplantation whenever possible. If they are unfit for transplantation or an appropriate donor cannot be found, hypomethylating agents may be used. Expert opinion: New therapeutic options for lower-risk patients include thrombopoietin analogues, the TGF-beta family ligand trapping drug Luspatercept, and the telomerase inhibitor Imetelstat. Combinations of hypomethylating agents (HMA) with other compounds, and inhibitors of bcl2, such as venetoclax are being developed for higher-risk patients. Finally, hypomethylating agents in combination with donor lymphocytes may lead to long-term remission following molecular or hematological relapse after allogeneic SCT.KEYWORDS Myelodysplastic syndromes; risk-adapted therapy; hypomethylating agents; erythropoiesis stimulating agents; thrombopoiesis stimulating agents; immunomodulating drugs; TGF-beta ligand trapping; bcl2 inhibition; investigational drugs; MRD monitoring
机译：摘要介绍：髓细胞增强型综合征（MDS）包含具有不同临床课程的异质组骨髓肿瘤。国际预后评分系统（IPSS-R）的修订版将风险分层提供为5种不同的群体。所涵盖的区域：对于较低风险的患者，红细胞输血和铁螯合是支持性护理的骨干。此外，促红细胞生成刺激剂（ESA）用于改善贫血。 Lenalidomide被批准用于治疗患有输血依赖的Del（5Q）的低危患者。每当尽可能提供具有更高风险疾病的患者。如果它们不适用于移植或不能发现适当的供体，则可以使用下甲基化试剂。专家意见：下风险患者的新治疗选择包括血小菌蛋白类似物，TGF-β家族配体捕获药物Luspatercept，以及端粒酶抑制剂咪δtat。对于更高风险的患者，正在开发用于更高风险的患者的背甲基化试剂（HMA）与其他化合物和BCL2的抑制剂。最后，与供体淋巴细胞组合的低甲基化试剂可能导致同种异体SCT的分子或血液复发后的长期缓解.Keywords骨髓增生综合征;风险适应治疗;低甲基化剂;促红细胞刺激剂;血栓刺激剂;免疫调节药物; TGF-Beta配体捕获; BCL2抑制;调查药物; MRD Monitoring.

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