骨髓增生性肿瘤（MPNs）是一组包括真性红细胞增多症（PV）、原发性血小板增多症（ET）和骨髓纤维化（MF）在内的克隆性血液恶性肿瘤。MPNs的特征是JAK/STAT通路激活突变和转化为一种侵袭性急性白血病形式（MPN-爆发期，MPN-BP）风险增加。MPN-BP被定义为血液或骨髓中⩾20%的幼稚细胞存在，几乎总是在加速期（MPN-AP）前出现，加速期被定义为血液或骨髓中⩾10-19%的幼稚细胞存在。这些先进的疾病形式与预后差相关，MPN-BP的中位总生存期（mOS）为3-5个月，MPN-AP为13个月。MPN-AP/BP具有独特的分子特征，表现为肿瘤内部复杂性增加。常用于初发性急性髓系白血病（AML）的标准治疗未显示出总生存期的改善。异基因造血干细胞移植（HSCT）仍然是唯一的治愈性疗法，但与明显的发病率和死亡率相关，并且在临床实践中很少使用。因此，这种先进阶段患者群体中迫切需要有效治疗的需求仍然存在。在这里，我们回顾了MPN-AP/BP的目前管理和未来治疗方向。© 作者（们），2023。

Myeloproliferative neoplasms (MPNs) are a group of clonal hematologic malignancies that include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). MPNs are characterized by activating mutations in the JAK/STAT pathway and an increased risk of transformation to an aggressive form of acute leukemia, termed MPN-blast phase (MPN-BP). MPN-BP is characterized by the presence of ⩾20% blasts in the blood or bone marrow and is almost always preceded by an accelerated phase (MPN-AP) defined as ⩾10-19% blasts in the blood or bone marrow. These advanced forms of disease are associated with poor prognosis with a median overall survival (mOS) of 3-5 months in MPN-BP and 13 months in MPN-AP. MPN-AP/BP has a unique molecular landscape characterized by increased intratumoral complexity. Standard therapies used in de novo acute myeloid leukemia (AML) have not demonstrated improvement in OS. Allogeneic hematopoietic stem cell transplant (HSCT) remains the only curative therapy but is associated with significant morbidity and mortality and infrequently utilized in clinical practice. Therefore, an urgent unmet need persists for effective therapies in this advanced phase patient population. Here, we review the current management and future directions of therapy in MPN-AP/BP.© The Author(s), 2023.