PURPOSE: Advanced systemic mastocytosis (SM), a fatal hematopoietic malignancy characterized by drug resistance, has no standard therapy. The effectiveness of allogeneic hematopoietic stem-cell transplantation (alloHCT) in SM remains unknown. PATIENTS AND METHODS: In a global effort to define the value of HCT in SM, 57 patients with the following subtypes of SM were evaluated: SM associated with clonal hematologic non-mast cell disorders (SM-AHNMD; n = 38), mast cell leukemia (MCL; n = 12), and aggressive SM (ASM; n = 7). Median age of patients was 46 years (range, 11 to 67 years). Donors were HLA-identical (n = 34), unrelated (n = 17), umbilical cord blood (n = 2), HLA-haploidentical (n = 1), or unknown (n = 3). Thirty-six patients received myeloablative conditioning (MAC), and 21 patients received reduced-intensity conditioning (RIC). RESULTS: Responses in SM were observed in 40 patients (70%), with complete remission in 16 patients (28%). Twelve patients (21%) had stable disease, and five patients (9%) had primary refractory disease. Overall survival (OS) at 3 years was 57% for all patients, 74% for patients with SM-AHNMD, 43% for those with ASM, and 17% for those with MCL. The strongest risk factor for poor OS was MCL. Survival was also lower in patients receiving RIC compared with MAC and in patients having progression compared with patients having stable disease or response. CONCLUSION: AlloHCT was associated with long-term survival in patients with advanced SM. Although alloHCT may be considered as a viable and potentially curative therapeutic option for advanced SM in the meantime, given that this is a retrospective analysis with no control group, the definitive role of alloHCT will need to be determined by a prospective trial.

Celalettin Ustun Ryan Shanley Gregory Vercellotti and Daniel Weisdorf University of Minnesota Minneapolis; William J Hogan Mayo Clinic Rochester MN; Andreas Reiter and Sebastian Kreil Universitätsmedizin Mannheim Mannheim; Herrad Baurmann Stiftung Deutsche Klinik für Diagnostik Wiesbaden; Bernd Gruhn Jena University Hospital Jena; Tanja Gromke University of Essen Essen; Eva Maria Wagner Universitätsmedizin Mainz Mainz; Martin Bornhäuser Universitätsklinikum Dresden Dresden; Christoph Schmid Klinikum Augsburg Ausburg Germany; Bart L Scott and H Joachim Deeg University of Washington Seattle WA; Ryotaro Nakamura City of Hope National Medical Center Duarte; Vinod Pullarkat University of Southern California Los Angeles CA; Gandhi Damaj Université de Caen Faculté de Médecine Caen; Faezeh Legrand Nice University Hospital Nice and Société Française de Greffe de Moelle et de Thérapie Cellulaire Hôpital Necker Enfants Malades Université Paris 5 Faculté de Médecine et AP HP Necker Enfants Malades and Centre de Référence des Mastocytoses Paris France; Miguel Angel Perales and Esperanza B Papadopoulos Memorial Sloan Kettering Cancer Center; Tsiporah Shore New York Presbyterian Hospital Weill Cornell Medical Center New York NY; Robert Stuart Medical University of South Carolina Charleston SC; Michael Doubek Masaryk University Brno Czech Republic; Jack W Hsu University of Florida Gainesville FL; Eleni Tholouli Manchester Royal Infirmary Manchester United Kingdom; Lucy A Godley University of Chicago Chicago IL; Livio Pagano Università Cattolica del Sacro Cuore Milan; Maria Teresa Van Lint Istituto Di Ricovero e Cura a Carattere Scientifico San Martino IST Genova Italy; Andrew Gilman Levine Children's Hospital Charlotte NC; Tor Shwayder Henry F

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Mast cell leukemia: clinical and molecular features and survival outcomes of patients in the ECNM Registry

International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study