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Prevention and treatment of accelerated/blast phase 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
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| 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 TP53, ASXL1, IDH1/2, EZH2, SRSF2, 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.
- 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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