Pagliuca Simona, Gurnari Carmelo, Visconte Valeria
Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH 44195, USA.
Hematology, Oncogenesis and Biotherapies, University of Paris, 75013 Paris, France.
Cancers (Basel). 2021 Feb 13;13(4):784. doi: 10.3390/cancers13040784.
Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic disorders characterized by ineffective hematopoiesis, progressive cytopenias and increased risk of transformation to acute myeloid leukemia. The improved understanding of the underlying biology and genetics of MDS has led to better disease and risk classification, paving the way for novel therapeutic opportunities. Indeed, we now have a vast pipeline of targeted agents under pre-clinical and clinical development, potentially able to modify the natural history of the diverse disease spectrum of MDS. Here, we review the latest therapeutic approaches (investigational and approved agents) for MDS treatment. A deep insight will be given to molecularly targeted therapies by reviewing new agents for individualized precision medicine.
骨髓增生异常综合征（MDS）是一组异质性的克隆性造血疾病，其特征为造血无效、进行性血细胞减少以及转化为急性髓系白血病的风险增加。对MDS潜在生物学和遗传学的深入了解已带来更好的疾病和风险分类，为新的治疗机会铺平了道路。事实上，我们目前有大量处于临床前和临床开发阶段的靶向药物，有可能改变MDS多样疾病谱的自然病程。在此，我们综述MDS治疗的最新治疗方法（研究性药物和已批准药物）。通过回顾用于个体化精准医学的新药，将深入探讨分子靶向治疗。
