我如何处理不适合移植的骨髓增生异常肿瘤患者。

Carmelo Gurnari, Zhuoer Xie, Amer M Zeidan
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引用次数: 2

摘要

骨髓增生异常肿瘤,以前称为骨髓增生异常综合征(MDS),代表了一组克隆性疾病,其特征是高度的临床和分子异质性,并且有发展为急性骨髓性白血病的不变趋势。MDS通常出现在不同程度的细胞减少和骨髓发育不良的老年人中,这是该疾病的标志。同种异体造血干细胞移植是迄今为止唯一的治疗方法。然而,考虑到这种疾病的人口特征,只有少数患者能从这种手术中受益。目前使用的预后方案,如修订的国际预后评分系统(R-IPSS)和最近的分子IPSS (IPSS- m),根据3.5分的临界值将MDS分为两大类:低风险病例和高风险病例,指导临床管理。低风险组的主要临床问题是细胞减少症的管理,而后者的目标是预防继发性白血病的进展。在此,我们讨论了MDS的非移植治疗,重点关注当前的实践和可用的治疗方案,同时也介绍了在不久的将来可能进入MDS治疗库的新研究药物。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms.

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.

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