Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia.

IF 8.2 1区医学 Q1 HEMATOLOGY

Haematologica Pub Date : 2024-10-24 DOI:10.3324/haematol.2023.283950

Anand A Patel,Raajit K Rampal

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引用次数: 0

Abstract

Philadelphia-chromosome negative (Ph-neg) myeloproliferative neoplasms (MPNs) are hematopoietic stem disorders with a risk of progression to the accelerated-phase (AP) or blastphase (BP) that is influenced by clinical, pathologic, cytogenetic, and molecular variables. Overall survival is limited in MPN-AP/BP with current treatment approaches, particularly in those patients that cannot receive an allogeneic hematopoietic stem cell transplant (allo-HCT). In addition, long-term survival with allo-HCT is predominantly seen in chronic-phase MPNs which suggests that the ideal time for intervention may be before MPNs evolve to AP/BP. Over the course of this review we will focus on the risk factors for progression to MPN-AP/BP, identification of high-risk chronic-phase MPNs, potential early-intervention strategies, and considerations around the timing of allo-HCT. We will also summarize current survival outcomes in MPN-AP/BP, discuss the uncertainty around how to best gauge response to therapy, and outline clinical trial considerations for this patient population. Lastly, we will highlight future directions in the management of high-risk MPNs.

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骨髓增生性肿瘤向急性髓性白血病转化的预防和治疗。

费城染色体阴性（Ph-neg）骨髓增殖性肿瘤（MPNs）是一种造血干细胞疾病，有进展到加速期（AP）或爆炸期（BP）的风险，受临床、病理、细胞遗传学和分子变量的影响。采用目前的治疗方法，MPN-AP/BP的总生存率有限，尤其是那些不能接受异基因造血干细胞移植（allo-HCT）的患者。此外，异体造血干细胞移植的长期存活率主要见于慢性期多发性骨髓瘤，这表明理想的干预时机可能是在多发性骨髓瘤演变为 AP/BP 之前。在本综述中，我们将重点讨论进展为 MPN-AP/BP 的风险因素、高风险慢性期 MPN 的识别、潜在的早期干预策略以及围绕异体肝移植时机的考虑因素。我们还将总结 MPN-AP/BP 目前的生存结果，讨论如何最好地衡量治疗反应的不确定性，并概述针对这一患者群体的临床试验注意事项。最后，我们将强调高风险 MPN 管理的未来方向。

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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来源期刊

Haematologica 医学-血液学

CiteScore

14.10

自引率

2.00%

发文量

349

审稿时长

3-6 weeks

期刊介绍： Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research. Scope: The scope of the journal includes reporting novel research results that: Have a significant impact on understanding normal hematology or the development of hematological diseases. Are likely to bring important changes to the diagnosis or treatment of hematological diseases.