我如何管理不符合移植条件的骨髓增生异常肿瘤患者?

Clinical Hematology International Pub Date : 2023-03-01 Epub Date: 2022-12-27 DOI:10.1007/s44228-022-00024-4
Carmelo Gurnari, Zhuoer Xie, Amer M Zeidan
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引用次数: 0

摘要

骨髓增生异常肿瘤,以前被称为骨髓增生异常综合征(MDS),是一组克隆性疾病,其特点是临床和分子高度异质性,并始终倾向于发展为急性髓系白血病。MDS 通常发生在老年人身上,其特征是不同程度的细胞减少和骨髓发育不良。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过程中获益。目前使用的预后方案,如修订版国际预后评分系统(R-IPSS)和最新的分子IPSS(IPSS-M),根据3.5分的临界值将MDS分为两大类:低风险和高风险病例,以此指导临床治疗。低风险组的主要临床问题是处理细胞减少症,而后者的目标是预防继发性白血病的发展。在此,我们将讨论 MDS 的非移植治疗,重点是当前的实践和可用的治疗方案,同时还将介绍可能在不久的将来进入 MDS 治疗领域的新研究药物。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms.

How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms.

How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms.

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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