Annals of Hematology
Variables associated with outcome in patients with Philadelphia chromosome-negative accelerated- or blast-phase myeloproliferative neoplasms
By Jing Liu, Sen Yang, Lu Yu and 13 others.
Abstract
Background
Poor outcomes have been reported when patients with myeloproliferative neoplasm (MPN) progress to the accelerated phase (AP) or blast phase (BP).
Methods
Data on consecutive patients with Ph − MPN-AP/BP diagnosed and treated at three hospitals between 1 January 2015 and 18 July 2025 were reviewed. Efficacy and a Cox regression risk model was used to identify clinical variables associated with the outcome.
Results
Data on 88 consecutive patients with Ph− MPN-AP/BP (32 AP and 56 BP) were reviewed. The median follow-up was 16 months. In MPN-AP, treatment with hypomethylating agents (HMA) + ruxolitinib or HMA + venetoclax achieved comparable response rates (returning to CP, 63% vs. 75%, p = 0.551). In MPN-BP, treatment with HMA + venetoclax or intensive chemotherapy achieved comparable response rates. The median survival was significantly longer in patients with MPN-AP than in those with MPN-BP (32 months [95%CI 6–58 months] versus 12 months [95%CI 9–15 months, p = 0.039]). In patients with MPN-AP, older age was significantly associated with poorer survival. In patients with MPN-BP, multivariate analysis revealed that WBC count ≥ 55 × 109/L, prior myelofibrosis history, and adverse karyotype were significantly associated with poorer survival; whereas HMA + venetoclax or intensive chemotherapy were associated with longer survival. In the 70 AP or BP patients with next-generation sequencing data TP53 , EZH2 , and SF3B1 mutations were associated with poorer survival.