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Hematopoietic Stem-Cell Transplantation for Advanced Systemic Mastocytosis

2014; Lippincott Williams & Wilkins; Volume: 32; Issue: 29 Linguagem: Inglês

10.1200/jco.2014.55.2018

1527-7755

Celalettin Üstün, Andreas Reiter, Bart L. Scott, Ryotaro Nakamura, Gandhi Damaj, Sebastian Kreil, Ryan Shanley, William J. Hogan, Miguel‐Angel Perales, Tsiporah B. Shore, H. Baurmann, Robert K. Stuart, Bernd Gruhn, Michael Doubek, Jack W. Hsu, Eleni Tholouli, Tanja Gromke, Lucy A. Godley, Livio Pagano, Andrew L. Gilman, Eva Wagner, Tor Shwayder, Martin Bornhäuser, Esperanza B. Papadopoulos, Alexandra Böhm, Gregory M. Vercellotti, Maria Teresa Van Lint, Christoph Schmid, Werner Rabitsch, Vinod Pullarkat, Faézeh Legrand, Ibrahim Yakoub‐Agha, Wael Saber, J. Carl Barrett, Olivier Hermine, Hans Hägglund, Wolfgang R. Sperr, Uday Popat, Edwin P. Alyea, Steven M. Devine, H. Joachim Deeg, Daniel J. Weisdorf, Cem Akin, Peter Valent,

Asthma and respiratory diseases

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.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).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.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.