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.

中文翻译：

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预防和治疗骨髓增生性肿瘤向急性髓性白血病的转化。


费城染色体阴性 （Ph-neg） 骨髓增生性肿瘤 （MPN） 是造血干细胞疾病，具有进展至加速期 （AP） 或急变期 （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 的总生存期有限，尤其是在那些无法接受同种异体造血干细胞移植 （allo-HCT） 的患者中。此外，allo-HCT 的长期生存率主要见于慢性期 MPN，这表明干预的理想时间可能是 MPN 演变为 AP/BP 之前。在本综述的过程中，我们将重点关注进展为 MPN-AP/BP 的危险因素、高危慢性期 MPN 的识别、潜在的早期干预策略以及有关同种异体 HCT 时间的考虑。我们还将总结 MPN-AP/BP 的当前生存结果，讨论如何最好地衡量对治疗反应的不确定性，并概述该患者群体的临床试验注意事项。最后，我们将重点介绍高风险 MPN 管理的未来方向。