Hematopoietic stem-cell transplantation for advanced systemic mastocytosis Journal Article

Authors: Ustun, C.; Reiter, A.; Scott, B. L.; Nakamura, R.; Damaj, G.; Kreil, S.; Shanley, R.; Hogan, W. J.; Perales, M. A.; Shore, T.; Baurmann, H.; Stuart, R.; Gruhn, B.; Doubek, M.; Hsu, J. W.; Tholouli, E.; Gromke, T.; Godley, L. A.; Pagano, L.; Gilman, A.; Wagner, E. M.; Shwayder, T.; Bornhauser, M.; Papadopoulos, E. B.; Bohm, A.; Vercellotti, G.; Van Lint, M. T.; Schmid, C.; Rabitsch, W.; Pullarkat, V.; Legrand, F.; Yakoub-agha, I.; Saber, W.; Barrett, J.; Hermine, O.; Hagglund, H.; Sperr, W. R.; Popat, U.; Alyea, E. P.; Devine, S.; Deeg, H. J.; Weisdorf, D.; Akin, C.; Valent, P.
Article Title: Hematopoietic stem-cell transplantation for advanced systemic mastocytosis
Abstract: 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. (C) 2014 by American Society of Clinical Oncology
Keywords: tyrosine kinase inhibitors; minimal residual disease; remission; bone-marrow-transplantation; acute myeloid-leukemia; neoplastic; prognostic impact; mast-cells; 1st complete; c-kit mutations; chromosome-negative acute; lineage disease
Journal Title: Journal of Clinical Oncology
Volume: 32
Issue: 29
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 2014-10-10
Start Page: 3264
End Page: 3274
Language: English
ACCESSION: WOS:000342988000011
DOI: 10.1200/jco.2014.55.2018
PUBMED: 25154823
PMCID: PMC4876356
Notes: Article -- Source: Wos
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MSK Authors
  1. Miguel-Angel Perales
    382 Perales