Department of Medicine, Section of Hematology, Yale University, 333 Cedar Street, PO Box 208028, New Haven, CT, 06520-8028, USA.
Ann Hematol. 2020 Apr;99(4):677-692. doi: 10.1007/s00277-020-03963-1. Epub 2020 Feb 20.
The development in the therapeutic landscape of myelodysplastic syndromes (MDS) has substantially lagged behind other hematologic malignancies with no new drug approvals for MDS for 13 years since the approval of decitabine in the United States in 2006. While therapeutic concepts for MDS patients continue to be primarily defined by clinical-pathologic risk stratification tools such as the International Prognostic Scoring System (IPSS) and its revised version IPSS-R, our understanding of the genetic landscape and the molecular pathogenesis of MDS has greatly evolved over the last decade. It is expected that the therapeutic approach to MDS patients will become increasingly individualized based on prognostic and predictive genetic features and other biomarkers. Herein, we review the current treatment of lower-risk MDS patients and discuss promising agents in advanced clinical testing for the treatment of symptomatic anemia in lower-risk MDS patients such as luspatercept and imetelstat. Lastly, we review the clinical development of new agents and the implications of the wider availability of mutational analysis for the management of individual MDS patients.
骨髓增生异常综合征（MDS）的治疗领域发展明显滞后于其他血液恶性肿瘤，自 2006 年美国批准地西他滨以来，13 年来MDS 领域没有新的药物批准。虽然 MDS 患者的治疗理念主要仍由国际预后评分系统（IPSS）及其修订版 IPSS-R 等临床病理危险分层工具定义，但过去十年中，我们对 MDS 的遗传特征和分子发病机制的理解已经有了很大的发展。预计 MDS 患者的治疗方法将根据预后和预测性遗传特征和其他生物标志物变得越来越个体化。本文回顾了低危 MDS 患者的当前治疗方法，并讨论了在临床试验中进展迅速的药物，如 luspatercept 和 imetelstat，用于治疗低危 MDS 患者的症状性贫血。最后，我们回顾了新药物的临床开发以及更广泛地获得突变分析对个体 MDS 患者管理的影响。
