Treatment approach and outcomes of patients with accelerated/blast-phase myeloproliferative neoplasms in the current era Journal Article


Authors: Patel, A. A.; Yoon, J. J.; Johnston, H.; Davidson, M. B.; Shallis, R. M.; Chen, E. C.; Burkart, M.; Oh, T. S.; Iyer, S. G.; Madarang, E.; Muthiah, C.; Gross, I.; Dean, R.; Kassner, J.; Viswabandya, A.; Madero-Marroquin, R.; Rampal, R. K.; Murthy, G. S. G.; Bradley, T.; Abaza, Y.; Garcia, J. S.; Gupta, V.; Pettit, K. M.; Cursio, J. F.; Odenike, O.
Article Title: Treatment approach and outcomes of patients with accelerated/blast-phase myeloproliferative neoplasms in the current era
Abstract: Progression of myeloproliferative neoplasms (MPNs) to accelerated or blast phase is associated with poor survival outcomes. Since 2017 there have been several therapies approved for use in acute myeloid leukemia (AML); these therapies have been incorporated into the management of accelerated/blast-phase MPNs (MPN-AP/BP). We performed a multicenter analysis to investigate outcomes of patients diagnosed with MPN-AP/BP in 2017 or later. In total, 202 patients were identified; median overall survival (OS) was 0.86 years. We also analyzed patients based on first-line treatment; the 3 most common approaches were intensive chemotherapy (n = 65), DNA methyltransferase inhibitor (DNMTi)-based regimens (n = 65), and DNMTi + venetoclax-based regimens (n = 54). Median OS was not significantly different by treatment type. In addition, we evaluated response by 2017 European LeukemiaNet AML criteria and 2012 MPN-BP criteria in an effort to understand the association of response with survival outcomes. We also analyzed outcomes in 65 patients that received allogeneic hematopoietic stem cell transplant (allo-HSCT); median OS was 2.30 years from time of allo-HSCT. Our study demonstrates that survival among patients with MPN-AP/BP is limited in the absence of allo-HSCT even in the current era of therapeutics and underscores the urgent need for new agents and approaches. © 2024 by The American Society of Hematology.
Keywords: adult; cancer chemotherapy; aged; unclassified drug; overall survival; janus kinase 2; thrombopoietin receptor; myeloid metaplasia; creatinine; hemoglobin; retrospective study; protein p53; tumor marker; cancer therapy; bilirubin; splenomegaly; remission; allogeneic hematopoietic stem cell transplantation; leukocyte count; disease exacerbation; myeloproliferative neoplasm; polycythemia vera; thrombocythemia; platelet count; transcription factor runx1; genetic parameters; transcription factor ezh2; isocitrate dehydrogenase 1; calreticulin; clinical outcome; multicenter study (topic); dna methyltransferase inhibitor; mutation status; first-line treatment; complete cytogenetic response; partial remission; isocitrate dehydrogenase 2; human; male; female; article; venetoclax; complete molecular response; blast phase myeloproliferative neoplasm; asxl transcriptional regulator 1; serine and arginine rich splicing factor 2; accelerated myeloproliferative neoplasm; acute leukemia response partial; intensive cancer chemotherapy; marrow blast; peripheral blast
Journal Title: Blood Advances
Volume: 8
Issue: 13
ISSN: 2473-9529
Publisher: American Society of Hematology  
Date Published: 2024-07-09
Start Page: 3468
End Page: 3477
Language: English
DOI: 10.1182/bloodadvances.2024012880
PUBMED: 38739724
PROVIDER: scopus
PMCID: PMC11260843
DOI/URL:
Notes: Source: Scopus
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  1. Raajit Kumar Rampal
    338 Rampal