Department of Medicine, Section of Hematology-Oncology, University of Chicago, Chicago, IL.
Division of Hematologic Malignancies, Department of Medicine, Beth Israel Deaconess Medical Center, Boston, MA.
Blood Adv. 2024 Jul 9;8(13):3468-3477. doi: 10.1182/bloodadvances.2024012880.
Progression of myeloproliferative neoplasms (MPNs) to accelerated or blast phase is associated with poor survival outcomes. Since 2017 there have been several therapies approved for use in acute myeloid leukemia (AML); these therapies have been incorporated into the management of accelerated/blast-phase MPNs (MPN-AP/BP). We performed a multicenter analysis to investigate outcomes of patients diagnosed with MPN-AP/BP in 2017 or later. In total, 202 patients were identified; median overall survival (OS) was 0.86 years. We also analyzed patients based on first-line treatment; the 3 most common approaches were intensive chemotherapy (n = 65), DNA methyltransferase inhibitor (DNMTi)-based regimens (n = 65), and DNMTi + venetoclax-based regimens (n = 54). Median OS was not significantly different by treatment type. In addition, we evaluated response by 2017 European LeukemiaNet AML criteria and 2012 MPN-BP criteria in an effort to understand the association of response with survival outcomes. We also analyzed outcomes in 65 patients that received allogeneic hematopoietic stem cell transplant (allo-HSCT); median OS was 2.30 years from time of allo-HSCT. Our study demonstrates that survival among patients with MPN-AP/BP is limited in the absence of allo-HSCT even in the current era of therapeutics and underscores the urgent need for new agents and approaches.
骨髓增殖性肿瘤(MPN)进展为加速期或急变期与不良生存结局相关。自 2017 年以来,已有几种疗法被批准用于治疗急性髓系白血病(AML);这些疗法已被纳入加速/急变期 MPN(MPN-AP/BP)的治疗管理中。我们进行了一项多中心分析,以研究 2017 年或之后诊断为 MPN-AP/BP 的患者的结局。共有 202 例患者被确定;中位总生存期(OS)为 0.86 年。我们还根据一线治疗对患者进行了分析;最常见的三种方法是强化化疗(n=65)、DNA 甲基转移酶抑制剂(DNMTi)为基础的方案(n=65)和 DNMTi+venetoclax 为基础的方案(n=54)。治疗类型之间的中位 OS 无显著差异。此外,我们根据 2017 年欧洲白血病网 AML 标准和 2012 年 MPN-BP 标准评估了反应,以了解反应与生存结局的关系。我们还分析了接受异基因造血干细胞移植(allo-HSCT)的 65 例患者的结局;allo-HSCT 后中位 OS 为 2.30 年。我们的研究表明,在当前治疗时代,即使接受了治疗,MPN-AP/BP 患者的生存仍然有限,迫切需要新的药物和方法。