Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia Review


Authors: Patel, A. A.; Rampal, R. K.
Review Title: Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia
Abstract: Philadelphia-chromosome negative myeloproliferative neoplasms (MPN) are hematopoietic stem disorders with a risk of progression to an accelerated phase (AP) or blast phase (BP) that is influenced by clinical, pathological, cytogenetic, and molecular variables. Overall survival of patients with MPN-AP/BP is limited with current treatment approaches, particularly in those patients who cannot receive an allogeneic hematopoietic stem cell transplant (allo-HCT). In addition, long-term survival with allo-HCT is predominantly seen in chronic-phase MPN, which suggests that the ideal time for intervention may be before the MPN evolves to AP/BP. In this review we focus on the risk factors for progression to MPN-AP/BP, identification of high-risk chronic-phase MPN, potential early-intervention strategies, and considerations around the timing of allo-HCT. We also summarize current survival outcomes of patients with MPN-AP/BP, discuss the uncertainty around how to best gauge response to therapy, and outline clinical trial considerations for this population of patients. Lastly, we highlight future directions in the management of high-risk MPN. ©2024 Ferrata Storti Foundation.
Keywords: myeloproliferative disorders; transplantation, homologous; overall survival; genetics; myeloproliferative disorder; leukemia, myeloid, acute; clinical trial; mortality; review; risk factors; hematopoietic stem cell transplantation; pathology; risk factor; cell transformation, neoplastic; disease progression; diagnosis; surgery; hematopoietic stem cell; disease management; drug therapy; disease exacerbation; therapy; allotransplantation; myeloproliferative neoplasm; prevention; early intervention; acute myeloid leukemia; procedures; long term survival; humans; human; male; neoplastic cell transformation
Journal Title: Haematologica
Volume: 110
Issue: 4
ISSN: 0390-6078
Publisher: Ferrata Storti Foundation  
Date Published: 2025-04-01
Start Page: 828
End Page: 839
Language: English
DOI: 10.3324/haematol.2023.283950
PUBMED: 39445417
PROVIDER: scopus
PMCID: PMC11959254
DOI/URL:
Notes: Review -- MSK corresponding author is Raajit Rampal -- Source: Scopus
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MSK Authors
  1. Raajit Kumar Rampal
    338 Rampal