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Hematopoietic stem-cell transplantation for advanced systemic mastocytosis

Authors :
Ustun, Celalettin
Reiter, Andreas
Scott, Bart L
Nakamura, Ryotaro
Damaj, Gandhi
Kreil, Sebastian
Shanley, Ryan
Hogan, William J
Perales, Miguel-Angel
Shore, Tsiporah
Baurmann, Herrad
Stuart, Robert
Gruhn, Bernd
Doubek, Michael
Hsu, Jack W
Tholouli, Eleni
Gromke, Tanja
Godley, Lucy A
Pagano, Livio
Gilman, Andrew
Wagner, Eva Maria
Shwayder, Tor
Bornhäuser, Martin
Papadopoulos, Esperanza B
Böhm, Alexandra
Vercellotti, Gregory
Van Lint, Maria Teresa
Schmid, Christoph
Rabitsch, Werner
Pullarkat, Vinod
Legrand, Faezeh
Yakoub-Agha, Ibrahim
Saber, Wael
Barrett, John
Hermine, Olivier
Hägglund, Hans
Sperr, Wolfgang R
Popat, Uday
Alyea, Edwin P
Devine, Steven
Deeg, H Joachim
Weisdorf, Daniel
Akin, Cem
Valent, Peter
Ustun, Celalettin
Reiter, Andreas
Scott, Bart L
Nakamura, Ryotaro
Damaj, Gandhi
Kreil, Sebastian
Shanley, Ryan
Hogan, William J
Perales, Miguel-Angel
Shore, Tsiporah
Baurmann, Herrad
Stuart, Robert
Gruhn, Bernd
Doubek, Michael
Hsu, Jack W
Tholouli, Eleni
Gromke, Tanja
Godley, Lucy A
Pagano, Livio
Gilman, Andrew
Wagner, Eva Maria
Shwayder, Tor
Bornhäuser, Martin
Papadopoulos, Esperanza B
Böhm, Alexandra
Vercellotti, Gregory
Van Lint, Maria Teresa
Schmid, Christoph
Rabitsch, Werner
Pullarkat, Vinod
Legrand, Faezeh
Yakoub-Agha, Ibrahim
Saber, Wael
Barrett, John
Hermine, Olivier
Hägglund, Hans
Sperr, Wolfgang R
Popat, Uday
Alyea, Edwin P
Devine, Steven
Deeg, H Joachim
Weisdorf, Daniel
Akin, Cem
Valent, Peter
Publication Year :
2014

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.

Details

Database :
OAIster
Notes :
English
Publication Type :
Electronic Resource
Accession number :
edsoai.on1235010643
Document Type :
Electronic Resource
Full Text :
https://doi.org/10.1200.JCO.2014.55.2018