Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine , New York, USA.
Department of Medicine, Icahn School of Medicine , New York, USA.
Expert Rev Anticancer Ther. 2020 Jun;20(6):465-482. doi: 10.1080/14737140.2020.1770088. Epub 2020 Jun 1.
Myelodysplastic syndromes (MDS) represent a range of bone marrow disorders, with patients affected by cytopenias and risk of progression to AML. There are limited therapeutic options available for patients, including hypomethylating agents (azacitidine/decitabine), growth factor support, lenalidomide, and allogeneic stem cell transplant.
This review provides an overview of the progress made over the past decade for emerging therapies for lower- and higher-risk MDS (MDS-HR). We also cover advances in prognostication, supportive care, and use of allogeneic SCT in MDS.
While there have been no FDA-approved therapies for MDS in the past decade, we anticipate the approval of luspatercept based on results from the MEDALIST trial for patients with lower-risk MDS (MDS-LR) and ringed sideroblasts who have failed or are ineligible for erythropoiesis stimulating agents (ESAs). With growing knowledge of the biologic and molecular mechanisms underlying MDS, it is anticipated that new therapies will be approved in the coming years.
骨髓增生异常综合征（MDS）代表了一系列骨髓疾病，患者会出现细胞减少症，并有可能进展为急性髓系白血病（AML）。目前，患者的治疗选择有限，包括低甲基化药物（阿扎胞苷/地西他滨）、生长因子支持、来那度胺和异基因造血干细胞移植。
本篇综述概述了过去十年中针对低危和高危 MDS（MDS-HR）的新兴疗法所取得的进展。我们还介绍了 MDS 预后、支持性治疗以及异基因 SCT 应用方面的进展。
虽然过去十年中 FDA 尚未批准用于 MDS 的疗法，但我们预计，基于 MEDALIST 试验结果，luspatercept 将获批用于治疗低危 MDS（MDS-LR）和环形铁幼粒细胞性难治性贫血患者，这些患者对红细胞生成刺激剂（ESA）治疗无效或不适用。随着对 MDS 潜在生物学和分子机制的认识不断深入，预计未来几年将有新的疗法获得批准。
