TY - JOUR
T1 - Hematopoietic stem-cell transplantation for advanced systemic mastocytosis
AU - Ustun, Celalettin
AU - Reiter, Andreas
AU - Scott, Bart L.
AU - Nakamura, Ryotaro
AU - Damaj, Gandhi
AU - Kreil, Sebastian
AU - Shanley, Ryan
AU - Hogan, William J.
AU - Perales, Miguel Angel
AU - Shore, Tsiporah
AU - Baurmann, Herrad
AU - Stuart, Robert
AU - Gruhn, Bernd
AU - Doubek, Michael
AU - Hsu, Jack W.
AU - Tholouli, Eleni
AU - Gromke, Tanja
AU - Godley, Lucy A.
AU - Pagano, Livio
AU - Gilman, Andrew
AU - Wagner, Eva Maria
AU - Shwayder, Tor
AU - Bornhäuser, Martin
AU - Papadopoulos, Esperanza B.
AU - Böhm, Alexandra
AU - Vercellotti, Gregory
AU - Van Lint, Maria Teresa
AU - Schmid, Christoph
AU - Rabitsch, Werner
AU - Pullarkat, Vinod
AU - Legrand, Faezeh
AU - Yakoub-Agha, Ibrahim
AU - Saber, Wael
AU - Barrett, John
AU - Hermine, Olivier
AU - Hagglund, Hans
AU - Sperr, Wolfgang R.
AU - Popat, Uday
AU - Alyea, Edwin P.
AU - Devine, Steven
AU - Deeg, H. Joachim
AU - Weisdorf, Daniel
AU - Akin, Cem
AU - Valent, Peter
N1 - Publisher Copyright:
© 2014 by American Society of Clinical Oncology.
PY - 2014/10/10
Y1 - 2014/10/10
N2 - 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.Purpose: Advanced systemic mastocytosis (SM), a fatal hematopoietic malignancy characterized by drug resistance, has no standard therapy. The effectiveness of allogeneic hematopoietic stem-cel 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 conditionng (RIC).
AB - 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.Purpose: Advanced systemic mastocytosis (SM), a fatal hematopoietic malignancy characterized by drug resistance, has no standard therapy. The effectiveness of allogeneic hematopoietic stem-cel 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 conditionng (RIC).
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U2 - 10.1200/JCO.2014.55.2018
DO - 10.1200/JCO.2014.55.2018
M3 - Article
C2 - 25154823
AN - SCOPUS:84905257818
SN - 0732-183X
VL - 32
SP - 3264
EP - 3274
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 29
ER -
