Authors
Stefan Haug, Wolfgang R Sperr, Claudia Wehr, Federico Bonofiglio, Saskia K Klein, Hanneke C Kluin-Nelemans, André B Mulder, Olivier Hermine, Julien Rossignol, Felipe Suarez, Ambroise Marcais, William Shomali, Cecelia Perkins, Deborah Christen, Werner Rabitsch, Emir Hadzijusufovic, Jaroslava Voglová, Madlen Jentzsch, Juliana Schwaab, Johannes Lübke, Mattias Mattsson, Hans Hagglund, Ingunn Dybedal, Daniel Baffoe, Patrizia Bonnadonna, Massimiliano Bonifacio, Ilaria Tanasi, Mariarita Sciumè, Marek Niedoszytko, Aleksandar Gorska, Andrzej Mital, Massimo Triggiani, Roberta Parente, Akif Selim Yavuz, Chiara Elena, Luca Malcovati, Jacqueline Ferrari, Friederike Wortmann, Knut Brockow, Tobias Michael Franz, Michael Makris, Sotirios G Papageorgiou, Christine Breynaert, Toon Ieven, Irena Angelova-Fischer, Alex Stefan, Judit Várkonyi, Vito Sabato, Tanja Daniela Schug, Paul L A van Daele, Anna Belloni Fortina, Francesca Caroppo, Theo Gulen, Marc Heizmann, Karin Hartmann, Axel Rüfer, Michael Doubek, Jens Panse, Jason Gotlib, Michel Arock, Hanneke N G Oude Elberink, Andreas Reiter, Peter Valent, Khalid Shoumariyeh
Published in
Experimental hematology & oncology. Volume 15. Issue 1. Aug 20, 2026. Epub Aug 20, 2026.
Abstract
Advanced systemic mastocytosis (AdvSM), comprising aggressive systemic mastocytosis, mast cell leukemia, and systemic mastocytosis with an associated hematological neoplasm, is a heterogeneous myeloid neoplasm with poor prognosis. Allogeneic hematopoietic cell transplantation (allo-HCT) remains the only potentially curative treatment, yet its benefit across AdvSM subtypes in the era of KIT-targeted tyrosine kinase inhibitors (TKIs) such as midostaurin and avapritinib remains unclear. To identify patient subgroups benefiting from allo-HCT and to define prognostic factors for post-transplant survival, we analyzed 631 AdvSM patients from the European Competence Network on Mastocytosis registry, including 69 who underwent allo-HCT. Treatment effects were assessed using time-dependent Cox regression in a transplant-eligible complete-case cohort (n = 419). In this cohort, allo-HCT showed no overall survival (OS) benefit (hazard ratio [HR] 1.21, 95% CI 0.80-1.84; p = 0.37), whereas TKI response emerged as a strong independent predictor of survival (HR 0.42; p<0.001). Subtype-stratified analysis revealed an allo-HCT benefit exclusively in patients with systemic mastocytosis associated with acute myeloid leukemia (n = 30; HR 0.20; p = 0.020). Among the 69 transplanted patients, median OS was 49.6 months with 2-year and 5-year survival of 63% and 46%, respectively. The International Prognostic Scoring System for Mastocytosis (IPSM) at transplantation independently predicted both OS (HR per category 1.68; p = 0.026) and progression-free survival (HR 1.99; p = 0.003), whereas the Mutation-Adjusted Risk Score and diagnostic subtype did not reach statistical significance. Competing risk analysis demonstrated that higher IPSM captured both relapse-related and transplant-related mortality. These findings suggest that the survival benefit of allo-HCT in AdvSM is driven primarily by control of the associated myeloid neoplasm. Accordingly, allo-HCT should be prioritized in patients with SM-AML, whereas TKI-directed strategies may be preferred in other subtypes, with IPSM at transplantation potentially guiding transplant selection and timing.
PMID:
42625226
Bibliographic data and abstract were imported from PubMed on 21 Aug 2026.
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