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

Knapper, Steven ORCID: https://orcid.org/0000-0002-6405-4441 2026. 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. British Journal of Haematology 209 (1) , pp. 160-171. 10.1111/bjh.70511

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Abstract

Summary: 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.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Schools > Medicine
Additional Information:	License information from Publisher: LICENSE 1: URL: http://creativecommons.org/licenses/by/4.0/
Publisher:	Wiley
ISSN:	0007-1048
Date of First Compliant Deposit:	6 May 2026
Date of Acceptance:	10 April 2026
Last Modified:	04 Aug 2026 13:26
URI:	https://orca.cardiff.ac.uk/id/eprint/186812

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