Outcomes and prognostic factors in patients with systemic mastocytosis and an associated myeloid neoplasm: an international ECNM registry study

@article{hermine2026outcomes,
  title = {{Outcomes and prognostic factors in patients with systemic mastocytosis and an associated myeloid neoplasm: an international ECNM registry study}},
  author = {Olivier Hermine and Orianne Debeaupuis and Gandhi Damaj and Johannes Lübke and Juliana Schwaab and Marc Heizmann and Frank Siebenhaar and Polina Pyatilova and Nicole Nojarov and Marek Niedoszytko and Aleksandra Gorska and Mattias Mattsson and Khalid Shoumariyeh and Stephan de Bra and Friederike Wortmann and Nikolas von Bubnoff and Horia Bumbea and Delia Soare and Chiara Elena and Judit Várkonyi and Anna Belloni Fortina and Simge Erdem and Knut Brockow and Tobias Michael Franz and Jens Panse and Deborah Christen and Christine Breynaert and Akif Selim Yavuz and Luca Malcovati and Michael Doubek and Vito Sabato and Ilaria Tanasi and Massimiliano Bonifacio and Karin Hartmann and Axel Rüfer and Mariarita Sciumè and Valentina Bellani and Irena Angelova-Fischer and Alex Stefan and Massimo Triggiani and Roberta Parente and Saskia K Klein and Michael Makris and Sotirios Papageorgiou and Paul van Daele and Ingunn Dybedal and Daniel Baffoe and Theo Gülen and Madlen Jentzsch and Olivier Lortholary and Mael Heiblig and Cristina Bulai Livideanu and Stéphane Barete and Clément Gourguechon and Sophie Dimicoli-Salazar and Olivier Tournilhac and Laurent Frenzel and Michel Arock and Andrzej Mital and Johannes N Woltsche},
  journal = {Journal of Hematology and Oncology},
  year = {2026},
  month = jun,
  doi = {10.1186/s13045-026-01819-1},
  url = {https://hal.science/hal-05790722v1},
  note = {CC BY-NC-ND 4.0},
}

TY  - JOUR
TI  - Outcomes and prognostic factors in patients with systemic mastocytosis and an associated myeloid neoplasm: an international ECNM registry study
AU  - Olivier Hermine
AU  - Orianne Debeaupuis
AU  - Gandhi Damaj
AU  - Johannes Lübke
AU  - Juliana Schwaab
AU  - Marc Heizmann
AU  - Frank Siebenhaar
AU  - Polina Pyatilova
AU  - Nicole Nojarov
AU  - Marek Niedoszytko
AU  - Aleksandra Gorska
AU  - Mattias Mattsson
AU  - Khalid Shoumariyeh
AU  - Stephan de Bra
AU  - Friederike Wortmann
AU  - Nikolas von Bubnoff
AU  - Horia Bumbea
AU  - Delia Soare
AU  - Chiara Elena
AU  - Judit Várkonyi
AU  - Anna Belloni Fortina
AU  - Simge Erdem
AU  - Knut Brockow
AU  - Tobias Michael Franz
AU  - Jens Panse
AU  - Deborah Christen
AU  - Christine Breynaert
AU  - Akif Selim Yavuz
AU  - Luca Malcovati
AU  - Michael Doubek
AU  - Vito Sabato
AU  - Ilaria Tanasi
AU  - Massimiliano Bonifacio
AU  - Karin Hartmann
AU  - Axel Rüfer
AU  - Mariarita Sciumè
AU  - Valentina Bellani
AU  - Irena Angelova-Fischer
AU  - Alex Stefan
AU  - Massimo Triggiani
AU  - Roberta Parente
AU  - Saskia K Klein
AU  - Michael Makris
AU  - Sotirios Papageorgiou
AU  - Paul van Daele
AU  - Ingunn Dybedal
AU  - Daniel Baffoe
AU  - Theo Gülen
AU  - Madlen Jentzsch
AU  - Olivier Lortholary
AU  - Mael Heiblig
AU  - Cristina Bulai Livideanu
AU  - Stéphane Barete
AU  - Clément Gourguechon
AU  - Sophie Dimicoli-Salazar
AU  - Olivier Tournilhac
AU  - Laurent Frenzel
AU  - Michel Arock
AU  - Andrzej Mital
AU  - Johannes N Woltsche
PY  - 2026
DA  - 2026/06/26
JO  - Journal of Hematology and Oncology
DO  - 10.1186/s13045-026-01819-1
UR  - https://hal.science/hal-05790722v1
AB  - Background Systemic mastocytosis (SM) is a spectrum of hematologic disorders characterized by accumulation of atypical mast cells (MCs) in extracutaneous organs. SM with an associated hematologic neoplasm (SM-AHN), the most frequent subtype of advanced SM, is predominantly associated with myeloid neoplasms, consistent with shared clonal architecture. Because of its rarity and heterogeneity, robust outcome data aligned with contemporary classifications are needed to inform risk stratification.  MethodsWe analyzed the 10th data wave of the European Competence Network on Mastocytosis registry (34 European centers and 1 US center). SM and AHN diagnoses followed the 2022 World Health Organization classification. Baseline characteristics and overall survival (OS) were compared between patients with myeloid SM-AHN and SM without AHN (SM-no-AHN). Within SM-AHN, outcomes were analyzed by SM component (advanced:  aggressive SM [ASM] or MC leukemia [MCL] vs. non-advanced: bone marrow mastocytosis, indolent SM, or smoldering SM) and AHN subtype. Results Among 3,925 patients with SM, 467 (11.9%) had myeloid SM-AHN. Myelodysplastic/myeloproliferative neoplasms (MDS/MPN) were the most frequent AHN category (41.1%), with chronic myelomonocytic leukemia as the most common subtype (29.6%). Compared with SM-no-AHN, SM-AHN patients were older, more often male, and less frequently had skin involvement. The SM component was advanced in 55.0% of SM-AHN versus 5.8% of SM-no-AHN (p < 0.001). OS was shorter in SM-AHN than SM-no-AHN (median 36.1 vs. 340.9 months; p < 0.001) and was reduced across SM subtypes, including ASM (31.0 vs. 81.1 months) and MCL (7.1 vs. 23.2 months). Within SM-AHN, advanced SM-AHN had shorter OS than non-advanced SM-AHN (28.3 vs. 70.9 months; p < 0.001). Median OS differed by AHN subtype (57.0 months in SM-MPN, 35.7 in SM-MDS, 34.2 in SM-MDS/MPN, and 14.7 in SM associated with acute myeloid leukemia; p < 0.001). In multivariable analysis, non-advanced SM (vs. advanced) remained independently associated with improved OS (hazard ratio = 0.44; p < 0.001).  Conclusions SM-AHN is associated with reduced survival compared with SM without AHN across SM subtypes. Outcomes in SM-AHN are driven primarily by the aggressiveness of the mastocytosis component, supporting recognition and classification of SM in patients with concomitant myeloid neoplasms, given approved KIT-targeted tyrosine kinase inhibitors for advanced SM.
SN  - 1756-8722
ER  -