Work

CPX-351 in combination with ruxolitinib for advanced-phase myeloproliferative neoplasms: results of a phase 1 study

Year: 2026

Type: article

Abstract: Accelerated phase myeloproliferative neoplasms (MPN-AP; 10%-19% blasts) and blast phase myeloproliferative neoplasms (MPN-BP; ≥20% blasts) remain among the most challenging myeloid malignancies, with no established standard of care.Median overall survival (OS) in MPN-BP has ranged from 3 to 6 months with intensive chemotherapy.[1][2][3] Less-intensive approaches, including hypomethylating agents (HMAs) with/without venetoclax, offer improved tolerability and modest survival benefit over historical controls but rarely durable remissions.4 Allogeneic stem cell transplantation (allo-SCT) remains the only curative option, although long-term survival is suboptimal (3-year OS of 36%).5 CPX-351, a liposomal formulation of daunorubicin and cytarabine delivered at a fixed 5:1 molar ratio, improved survival compared with conventional 7+3 in AML with myelodysplasia-related changes.6 However, patients with antecedent MPNs were excluded from the pivotal CPX-351 studies. 6,7urrent data for CPX-351 in MPN-BP are limited to a single retrospective series, and its combination with ruxolitinib has not been explored.8 Furthermore, although JAK inhibition has been studied in combination with HMAs, no prospective trials have evaluated the combination of intensive induction with JAK inhibition.[9][10][11]

Source: Blood Neoplasia

DOI: 10.1016/j.bneo.2026.100274