Subtype-specific outcome after allogeneic hematopoietic cell transplantation in advanced systemic mastocytosis: a registry study of the European competence network on mastocytosis.
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Haug Stefan, Sperr Wolfgang R, Wehr Claudia, Bonofiglio Federico, Klein Saskia K, Kluin-Nelemans Hanneke C, Mulder André B, Hermine Olivier, Rossignol Julien, Suarez Felipe, Marcais Ambroise, Shomali William, Perkins Cecelia, Christen Deborah, Rabitsch Werner, Hadzijusufovic Emir, Voglová Jaroslava, Jentzsch Madlen, Schwaab Juliana, Lübke Johannes, Mattsson Mattias, Hagglund Hans, Dybedal Ingunn, Baffoe Daniel, Bonnadonna Patrizia, Bonifacio Massimiliano, Tanasi Ilaria, Sciumè Mariarita, Niedoszytko Marek, Gorska Aleksandar, Mital Andrzej, Triggiani Massimo, Parente Roberta, Yavuz Akif Selim, Elena Chiara, Malcovati Luca, Ferrari Jacqueline, Wortmann Friederike, Brockow Knut, Franz Tobias Michael, Makris Michael, Papageorgiou Sotirios G, Breynaert Christine, Ieven Toon, Angelova-Fischer Irena, Stefan Alex, Várkonyi Judit, Sabato Vito, Schug Tanja Daniela, van Daele Paul L A, Fortina Anna Belloni, Caroppo Francesca, Gulen Theo, Heizmann Marc, Hartmann Karin, Rüfer Axel, Doubek Michael, Panse Jens, Gotlib Jason, Arock Michel, Oude Elberink Hanneke N G, Reiter Andreas, Valent Peter, Shoumariyeh Khalid
Аннотация
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.