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Real-world treatment patterns and outcomes in accelerated and blast-phase myeloproliferative neoplasms: Insights from a large multi-centre cohort analysis in the United Kingdom

Lookup NU author(s): Dr Andrew McGregor

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).

Abstract

© 2026 The Author(s). British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd. This UK-based retrospective analysis describes real-world treatment patterns and outcomes in 175 patients with accelerated (AP, n = 69) or blast-phase (BP, n = 106) ‘Philadelphia-negative’ myeloproliferative neoplasms (MPN-AP/BP) diagnosed between 2013 and 2025. Median age at transformation was 71 years. With a median follow-up of 45.2 months, median overall survival (OS) was 14.9 months, significantly worse for MPN-BP (6.7 months) versus MPN-AP (25.3 months). Treatment selection was heterogeneous across centres. Intensive chemotherapy (IC) improved outcomes only when followed by allogeneic haematopoietic stem cell transplant (allo-HSCT) (median OS 24.7 months). Ruxolitinib-based regimens, particularly combined with azacitidine, showed acceptable activity in AP (median OS 27.2 months). Venetoclax-based regimens achieved a median OS of 14.9 months across the cohort. Multivariable analysis identified IC and venetoclax-based therapy as independently associated with better outcomes, reflecting patient selection, while TP53 mutations predicted inferior survival. IC carried high rates of febrile neutropenia and sepsis; venetoclax was associated with prolonged cytopenias. This study confirms the poor prognosis of MPN-AP/BP, the absence of a unified UK consensus approach and the need for improved therapies and prospective studies to determine optimal treatment approaches for this challenging cohort.

Publication metadata

Author(s): Rampotas A, Naylor-Layland G, Brown C, Alabdulkarim A, Ryan J, Wadelin F, Alimam S, Tun PWW, Lambert J, O'Sullivan J, Wilson AJ, Tafesh L, McGregor A, Claudiani S, Innes A, Booth E, Leveson J, Knapper S, Garg M, Dubey M, Vatopoulou T, Brierley C, Leung WKN, Greenfield G, McMullin MF, Khan A, Milne K, Brian D, Godfrey A, Harrison CN, Psaila B, Harrington P, Kirkwood A, Somervaille TCP, McLornan DP

Publication type: Article

Publication status: Published

Journal: British Journal of Haematology

Year: 2026

Pages: Epub ahead of print

Online publication date: 03/05/2026

Acceptance date: 10/04/2026

Date deposited: 26/05/2026

ISSN (print): 0007-1048

ISSN (electronic): 1365-2141

Publisher: John Wiley and Sons Ltd

URL: https://doi.org/10.1111/bjh.70511

DOI: 10.1111/bjh.70511

Data Access Statement: The data that support the findings of this study are available on request from the corresponding author. The data are not publicly available due to privacy or ethical restrictions.

PubMed id: 42070782

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