Gurnari Carmelo, Xie Zhuoer, Zeidan Amer M
Translational Hematology and Oncology Research Department, Taussig Cancer Center, Cleveland Clinic, Cleveland, OH, USA.
Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy.
Clin Hematol Int. 2023 Mar;5(1):8-20. doi: 10.1007/s44228-022-00024-4. Epub 2022 Dec 27.
Myelodysplastic neoplasms, formerly known as myelodysplastic syndromes (MDS), represent a group of clonal disorders characterized by a high degree of clinical and molecular heterogeneity, and an invariable tendency to progress to acute myeloid leukemia. MDS typically present in the elderly with cytopenias of different degrees and bone marrow dysplasia, the hallmarks of the disease. Allogeneic hematopoietic stem cell transplant is the sole curative approach to date. Nonetheless, given the disease's demographics, only a minority of patients can benefit from this procedure. Currently used prognostic schemes such as the Revised International Prognostic Scoring System (R-IPSS), and most recently the molecular IPSS (IPSS-M), guide clinical management by dividing MDS into two big categories: lower- and higher-risk cases, based on a cut-off score of 3.5. The main clinical problem of the lower-risk group is represented by the management of cytopenias, whereas the prevention of secondary leukemia progression is the goal for the latter. Herein, we discuss the non-transplant treatment of MDS, focusing on current practice and available therapeutic options, while also presenting new investigational agents potentially entering the MDS therapeutic arsenal in the near future.
骨髓增生异常肿瘤,以前称为骨髓增生异常综合征(MDS),是一组克隆性疾病,其特征是具有高度的临床和分子异质性,以及不可避免地发展为急性髓系白血病的倾向。MDS通常发生于老年人,伴有不同程度的血细胞减少和骨髓发育异常,这些是该疾病的标志。同种异体造血干细胞移植是迄今为止唯一的治愈方法。然而,鉴于该疾病的人口统计学特征,只有少数患者能从这一治疗中获益。目前使用的预后方案,如修订后的国际预后评分系统(R-IPSS),以及最近的分子IPSS(IPSS-M),通过将MDS分为低危和高危两大类(基于3.5的临界值)来指导临床管理。低危组的主要临床问题是血细胞减少的管理,而预防继发性白血病进展则是高危组的目标。在此,我们讨论MDS的非移植治疗,重点关注当前的实践和可用的治疗选择,同时也介绍可能在不久的将来进入MDS治疗武器库的新型研究药物。