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2024-11-22T15:20:56.000Z

Prevention and treatment of accelerated/blast phase myeloproliferative neoplasms

Nov 22, 2024
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Learning objective: After reading this article, learners will be able to cite a new clinical development in myeloproliferative neoplasms.

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Ph− MPN are clonal hematopoietic stem cell disorders with a risk of progression to MPN-AP/BP, influenced by clinical, pathologic, cytogenetic, and molecular variables.1 A recent review in Haematalogica by Patel and Rampal discusses risk factors for progression, treatment strategies, response criteria, and considerations for future clinical trial design.1

Key learnings
The acquisition of high-risk mutations in the chronic phase of MPN plays a critical role in the progression to MPN-AP/BP, with mutations such as TP53ASXL1IDH1/2EZH2SRSF2, and SF3B1 having a prognostic impact.  
Allo-HSCT remains the only curative approach for disease progression, with optimal timing ideally being during the chronic phase or MPN-AP. 
There is heterogeneity in the treatment response assessment for patients with MPN-AP/BP, highlighting a need for harmonized response criteria. The authors propose OS, CMR, CCyR, CBR, PBR, and HI as clinical trial endpoints.  
As treatment data for patients with MPN-AP/BP is limited, the authors suggest the inclusion of MPN-AP/BP cohorts in early phase studies with patients with chronic phase MPN to identify novel therapies with promising clinical activity.  

Abbreviations: Allo-HSCT, allogenic hematopoietic stem cell transplantation; AP, accelerated phase; BP, blast phase; CBR, complete blast response; CCyR, complete cytogenetic response; CMR, complete molecular response; HI, hematological improvement MPN, myeloproliferative neoplasms; OS, overall survival; PBR, partial blast response; Ph−, Philadelphia chromosome-negative. 

  1. Patel AA, Rampal RK. Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia. 2024. Online ahead of print. DOI: 10.3324/haematol.2023.283950

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