MDS 的翻译和临床研究现状：国际 MDS 协会（icMDS）第一次 MDS 国际研讨会（iwMDS）的会议记录。
Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS).
发表日期：2023 Mar 11
作者：
Jan Philipp Bewersdorf, Zhuoer Xie, Rafael Bejar, Uma Borate, Jacqueline Boultwood, Andrew M Brunner, Rena Buckstein, Hetty E Carraway, Jane E Churpek, Naval G Daver, Matteo Giovanni Della Porta, Amy E DeZern, Pierre Fenaux, Maria E Figueroa, Steven D Gore, Elizabeth A Griffiths, Stephanie Halene, Robert P Hasserjian, Christopher S Hourigan, Tae Kon Kim, Rami Komrokji, Vijay K Kuchroo, Alan F List, Sanam Loghavi, Ravindra Majeti, Olatoyosi Odenike, Mrinal M Patnaik, Uwe Platzbecker, Gail J Roboz, David A Sallman, Valeria Santini, Guillermo Sanz, Mikkael A Sekeres, Maximilian Stahl, Daniel T Starczynowski, David P Steensma, Justin Taylor, Omar Abdel-Wahab, Mina L Xu, Michael R Savona, Andrew H Wei, Amer M Zeidan
来源：
BIOMEDICINE & PHARMACOTHERAPY
摘要：
为了阐明造血干细胞异质性 (MDS) 发病机制中的生物事件，研究人员正在日益认识其中更多细节，并将这些发现转化为合理的治疗策略。本文介绍了来自国际造血干细胞异质性国际联盟 (icMDS) 的首届 MDS 国际研讨会 (iwMDS) 的更新进展，详细讲解了最近对于 MDS 遗传谱系的理解、遗传易感、表观遗传和免疫失调、而且，对于克隆性造血和MDS之间的复杂关系以及疾病新的动物模型有了更深入的认识。这一进展与研制针对特定分子改变、先天免疫系统和免疫检查点抑制剂的新疗法密切相关。虽然其中一些药物已经进入临床试验（例如剪接修饰剂、IRAK1/4抑制剂、抗CD47和抗TIM3抗体以及细胞疗法），但尚未获得 MDS 的批准。我们需要进行额外的临床和前线工作，以开发一种真正个性化的对于 MDS 患者的护理方法。 版权所有 ©2023 Elsevier Ltd.
Biological events that contribute to the pathogenesis of myelodysplastic syndromes/neoplasms (MDS) are becoming increasingly characterized and are being translated into rationally designed therapeutic strategies. Herein, we provide updates from the first International Workshop on MDS (iwMDS) of the International Consortium for MDS (icMDS) detailing recent advances in understanding the genetic landscape of MDS, including germline predisposition, epigenetic and immune dysregulation, the complexities of clonal hematopoiesis progression to MDS, as well as novel animal models of the disease. Connected to this progress is the development of novel therapies targeting specific molecular alterations, the innate immune system, and immune checkpoint inhibitors. While some of these agents have entered clinical trials (e.g., splicing modulators, IRAK1/4 inhibitors, anti-CD47 and anti-TIM3 antibodies, and cellular therapies), none have been approved for MDS. Additional preclinical and clinical work is needed to develop a truly individualized approach to the care of MDS patients.Copyright © 2023 Elsevier Ltd. All rights reserved.