{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T14:43:13Z","timestamp":1787236993304,"version":"build-2736575974"},"reference-count":11,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T00:00:00Z","timestamp":1787184000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T00:00:00Z","timestamp":1787184000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"publisher","award":["Project-ID 431984000 \u2013 CRC 1453 Nephrogenetics"],"award-info":[{"award-number":["Project-ID 431984000 \u2013 CRC 1453 Nephrogenetics"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002428","name":"Austrian Science Fund","doi-asserted-by":"publisher","award":["F4704-B20 and P32470-B"],"award-info":[{"award-number":["F4704-B20 and P32470-B"]}],"id":[{"id":"10.13039\/501100002428","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Exp Hematol Oncol"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    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                    <jats:italic>n<\/jats:italic>\n                    = 419). In this cohort, allo-HCT showed no overall survival (OS) benefit (hazard ratio [HR] 1.21, 95% CI 0.80\u20131.84;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.37), whereas TKI response emerged as a strong independent predictor of survival (HR 0.42;\n                    <jats:italic>p<\/jats:italic>\n                    &lt;0.001). Subtype-stratified analysis revealed an allo-HCT benefit exclusively in patients with systemic mastocytosis associated with acute myeloid leukemia (\n                    <jats:italic>n<\/jats:italic>\n                    = 30; HR 0.20;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.020). Among the 69 transplanted patients, median OS was 49.6\u00a0months 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;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.026) and progression-free survival (HR 1.99;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.003), 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.\n                  <\/jats:p>","DOI":"10.1186\/s40164-026-00819-8","type":"journal-article","created":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T10:03:08Z","timestamp":1787220188000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Subtype-specific outcome after allogeneic hematopoietic cell transplantation in advanced systemic mastocytosis: a registry study of the European competence network on mastocytosis"],"prefix":"10.1186","volume":"15","author":[{"given":"Stefan","family":"Haug","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang 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Br J Haematol. 2025. https:\/\/doi.org\/10.1111\/bjh.70154.","journal-title":"Br J Haematol"},{"issue":"1","key":"819_CR8","doi-asserted-by":"publisher","first-page":"81","DOI":"10.1016\/j.jaip.2018.09.024","volume":"7","author":"P Valent","year":"2019","unstructured":"Valent P, Oude Elberink JNG, Gorska A, Lange M, Zanotti R, van Anrooij B, et al. The Data Registry of the European Competence Network on Mastocytosis (ECNM): set up, projects, and perspectives. J Allergy Clin Immunol Pract. 2019;7(1):81\u20137. https:\/\/doi.org\/10.1016\/j.jaip.2018.09.024.","journal-title":"J Allergy Clin Immunol Pract"},{"issue":"12","key":"819_CR9","doi-asserted-by":"publisher","first-page":"e638","DOI":"10.1016\/S2352-3026(19)30166-8","volume":"6","author":"WR Sperr","year":"2019","unstructured":"Sperr WR, Kundi M, Alvarez-Twose I, van Anrooij B, Oude Elberink JNG, Gorska A, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. 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Blood. 1990;75(3):555\u201362.","journal-title":"Blood"}],"container-title":["Experimental Hematology &amp; Oncology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s40164-026-00819-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s40164-026-00819-8","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s40164-026-00819-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T10:03:09Z","timestamp":1787220189000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1186\/s40164-026-00819-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8,20]]},"references-count":11,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2026,12]]}},"alternative-id":["819"],"URL":"https:\/\/doi.org\/10.1186\/s40164-026-00819-8","relation":{},"ISSN":["2162-3619"],"issn-type":[{"value":"2162-3619","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,8,20]]},"assertion":[{"value":"21 June 2026","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"9 August 2026","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"20 August 2026","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"This study was conducted in accordance with the Declaration of Helsinki. The ECNM registry and this retrospective analysis were approved by the institutional Ethics Committees of all participating centers. All patients provided written informed consent for inclusion of their clinical data in the ECNM registry and for its use in research analyses.","order":1,"name":"Ethics","label":"Ethics approval and consent to participate","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Not applicable.","order":2,"name":"Ethics","label":"Consent for publication","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Stefan Haug: none. Wolfgang R. Sperr: Honoraria, scientific grants or travel support from AbbVie, Blueprint Medicines, BMS-Celgene, Daiichi Sankyo, Jazz, Novartis, Otsuka, Pfizer, Servier, StemLine, Thermo Fisher. Claudia Wehr: Travel support, honoraria, or research funding from Jazz Pharmaceuticals, Mundipharma, Grifols, Medac, and Takeda. Federico Bonofiglio: none. Saskia K. Klein: none. Hanneke C. Kluin-Nelemans: non-paid independent monitoring committee member for avapritinib study. Andr\u00e9 B. Mulder: none. Olivier Hermine: none. Julien Rossignol: Advisory Board, and research grants from Blueprint. Felipe Suarez: none. Ambroise Marcais: none. William Shomali: none. Cecelia Perkins: none. Deborah Christen: Advisory Board and Honoraria: Blueprint Medicines, Novartis, Astra Zeneca, Pfizer, BeiGene. Werner Rabitsch: Travel support and honoraria: Merck, Mallinckrodt, Jazz Pharmaceuticals. Emir Hadzijusufovic: none. Jaroslava Voglov\u00e1: none. Madlen Jentzsch: Advisory board and honoraria: Novartis. Juliana Schwaab: Advisory board and honoraria: Novartis, Blueprint Medicines. Johannes L\u00fcbke: Honoraria from Blueprint Medicines, Novartis, and Incyte. Travel support by Blueprint Medicines, and AOP. Mattias Mattsson: none. Hans Hagglund: none. Ingunn Dybedal: Advisory board fee from Blueprint Medicines. Daniel Baffoe: none. Patrizia Bonnadonna: none. Massimiliano Bonifacio: none. Ilaria Tanasi: none. Mariarita Scium\u00e8: none. Marek Niedoszytko: none. Aleksandar Gorska: none. Andrzej Mital: none. Massimo Triggiani: Advisory Board and Honoraria: Blueprint Medicines, Novartis. Roberta Parente: none. Akif Selim Yavuz: none. Chiara Elena: AB for Cogent and Blueprint. Luca Malcovati: none. Jacqueline Ferrari: none. Friederike Wortmann: Honoraria: Blueprint Medicines. Knut Brockow: Honoraria: Blueprint, Novartis, Thermo Fisher, Advarra, Astra Zeneca, BioMarin Pharmaceutical, UpToDate, ALK, HAL, Bencard. Tobias Michael Franz: none. Michael Makris: none. Sotirios G Papageorgiou: Amgen, Astellas, Genesis Pharma, Sanofi, AbbVie, BMS, Novartis, Sandoz, Pfizer, Janssen, Roche, Takeda, Gilead Sciences, Innovis, Winmedica. Christine Breynaert: none. Toon Ieven: none. Irena Angelova-Fischer: none. Alex Stefan: Honoraria from AOP Orphan, Amgen, Blueprint Medicines, Johnson&Johnson, Novartis, and Sanofi. Judit V\u00e1rkonyi: none. Vito Sabato: Advisory Board: Blueprint Medicines, Novartis, Cogent, Thermofisher. Tanja Daniela Schug: none. Paul LA van Daele: none. Anna Belloni Fortina: none. Francesca Caroppo: none. Theo Gulen: none. Marc Heizmann: Advisory Board and Consulting Honoraria: Novartis Pharma Schweiz AG. Karin Hartmann: Advisory board, honoraria: ALK, Allergopharma, Almirall, BioCryst, Blueprint, Cogent, Galderma, KalVista, Leo, Menarini, Novartis, Otsuka, Pfizer, Sanofi, Takeda, Thermo Fisher. Research grant: Leo. Axel R\u00fcfer: none. Michael Doubek: Consultation fees from AbbVie, AstraZeneca, Novartis, Johnson and Johnson. Jens Panse: Advisory Board and Honoraria: Blueprint Medicines, Novartis, Deciphera. Jason Gotlib: Research Grant (funds for administration of clinical trials): Novartis, Blueprint Medicines, Deciphera, Cogent Biosciences; Advisory Board and Honoraria: Blueprint Medicines, Novartis, Deciphera, Cogent Biosciences; Reimbursement of travel expenses: Novartis, Blueprint Medicines. Michel Arock: Research Grants: Blueprint Medicines; Consulting or Advisory Role: AB Science, Blueprint Medicines, Thermo Fisher. Hanneke N.G. Oude Elberink: none. Andreas Reiter: Honoraria: Novartis, Blueprint Medicines, Incyte, Celgene\/Bristol Myers Squibb, AOP Orphan Pharmaceuticals, GlaxoSmithKline, AbbVie; Consulting or Advisory Role: Novartis, Blueprint Medicines, Incyte, Celgene\/Bristol Myers Squibb, AOP Orphan Pharmaceuticals, AbbVie. Peter Valent: Advisory board and honoraria: Blueprint, BMS, Incyte, Novartis, AOP Orphan, Pfizer, Stemline, Servier, Cogent. Khalid Shoumariyeh: Advisory Board and Honoraria: Blueprint Medicines, Novartis.","order":3,"name":"Ethics","label":"Competing interests","group":{"name":"EthicsHeading","label":"Declarations"}}],"article-number":"79"}}