J Clin Oncol 2014 Oct 25;32(29):3264-74. Epub 2014 Aug 25.
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 (SFGM-TC); Ibrahim Yakoub-agha, Centre Hospitalier Régionale Universitaire, Lille; Olivier Hermine, Assistance Publique-Hôpitaux de Paris (AP-HP), 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
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.