对加速期和胚期骨髓增生性肿瘤踩刹车:当前和新出现的概念。

IF 2.9 3区 教育学 Q1 EDUCATION, SCIENTIFIC DISCIPLINES
Jan Philipp Bewersdorf, Raajit K Rampal
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引用次数: 1

摘要

bcr - abl阴性的骨髓增生性肿瘤(MPN)发展为加速期或细胞期MPN (MPN- ap /MPN- bp)的风险不同,定义为外周血或骨髓中分别存在10%至19%和超过或等于20%的髓细胞母细胞。随着表观遗传修饰因子(如ASXL1, EZH2, TET2), TP53, Ras通路或剪接因子(如SRSF2, U2AF1)的额外突变的获得,MPN-AP/MPN-BP进展的分子过程越来越被理解,这些突变被描述为这一进化过程中的重要步骤。至少部分是由于这些高危分子特征的富集,MPN-BP患者的预后仍然不如其他急性髓性白血病患者,中位总生存期为3至6个月。同种异体造血细胞移植仍然是唯一可能治愈的治疗方式,但只有少数患者符合条件。在没有治愈意图的情况下,可以考虑使用低甲基化药物作为单一疗法或与ruxolitinib或venetoclax联合使用的治疗策略或姑息性治疗。一些新型药物正处于临床开发的不同阶段,但目前还不能用于常规使用,这突出表明需要进行持续研究,并在可行的情况下优先考虑临床试验。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Hitting the brakes on accelerated and blast-phase myeloproliferative neoplasms: current and emerging concepts.

The BCR-ABL-negative myeloproliferative neoplasms (MPNs) have a variable risk of progressing to accelerated- or blast-phase MPN (MPN-AP/MPN-BP), defined by the presence of 10% to 19% and more than or equal to 20% myeloid blasts in the peripheral blood or bone marrow, respectively. The molecular processes underlying the progression to MPN-AP/MPN-BP are becoming increasingly understood with the acquisition of additional mutations in epigenetic modifiers (eg, ASXL1, EZH2, TET2), TP53, the Ras pathway, or splicing factors (eg, SRSF2, U2AF1), having been described as important steps in this evolutionary process. At least partially driven by the enrichment of these high-risk molecular features, the prognosis of patients with MPN-BP remains inferior to other patients with acute myeloid leukemia, with a median overall survival of 3 to 6 months. Allogeneic hematopoietic cell transplantation remains the only potentially curative therapeutic modality, but only a minority of patients are eligible. In the absence of curative intent, therapeutic strategies or palliative treatment with hypomethylating agents as monotherapy or in combination with ruxolitinib or venetoclax can be considered. Several novel agents are in various stages of clinical development but are not available for routine use at this point, highlighting the need for ongoing research and the prioritization of clinical trial enrollment when feasible.

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来源期刊
Hematology. American Society of Hematology. Education Program
Hematology. American Society of Hematology. Education Program EDUCATION, SCIENTIFIC DISCIPLINES-HEMATOLOGY
CiteScore
4.70
自引率
3.30%
发文量
0
期刊介绍: Hematology, the ASH Education Program, is published annually by the American Society of Hematology (ASH) in one volume per year.
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