Citations 2
Role of allogeneic transplantation in chronic myelomonocytic leukemia: an international collaborative analysis
Robin, Marie , Wreede, Liesbeth C. de , Padron, Eric , Bakunina, Katerina , Fenaux, Pierre , Köster, Linda, Nazha, Aziz , Rampal, Raajit K. , Sockel, Katja , Gagelmann, Nico , Eikema, Dirk‐Jan, Potter, Victoria , Killick, Sally , Legrand, Faézeh, Broom, Angus, Garcia‐Manero, Guillermo , Rizzoli, Vittorio, Hayden, Patrick , Patnaik, Mrinal M. , Yakoub‐Agha, Ibrahim , Itzykson, Raphaël , de Wreede, Liesbeth C , Komrokji, Rami S , Gagelmann, Nico , Finke, Jürgen , Solary, Eric , Patnaik, Mrinal , Yakoub-Agha, Ibrahim , Itzykson, Raphael , Robin, Marie , Beelen, Dietrich Wilhelm , Komrokji, Rami S. , Radujkovic, Aleksandar , Solary, Éric , Onida, Francesco
Blood | June 6, 2022 | Volume: 140 | Issue: 12 | Pages: 1408-1418
Abstract To determine the survival benefit of allogeneic hematopoietic cell transplantation (allo-HCT) in chronic myelomonocytic leukemias (CMML), we assembled a retrospective cohort of CMML patients 18-70 years old diagnosed between 2000 and 2014 from an international CMML dataset (n = 730) and the EBMT registry (n = 384). The prognostic impact of allo-HCT was analyzed through univariable and multivariable time-dependent models and with a multistate model, accounting for age, sex, CMML prognostic scoring system (low or intermediate-1 grouped as lower-risk, intermediate-2 or high as higher-risk) at diagnosis, and AML transformation. In univariable analysis, lower-risk CMMLs had a 5-year overall survival (OS) of 20% with allo-HCT vs 42% without allo-HCT (P < .001). In higher-risk patients, 5-year OS was 27% with allo-HCT vs 15% without allo-HCT (P = .13). With multistate models, performing allo-HCT before AML transformation reduced OS in patients with lower-risk CMML, and a survival benefit was predicted for men with higher-risk CMML. In a multivariable analysis of lower-risk patients, performing allo-HCT before transformation to AML significantly increased the risk of death within 2 years of transplantation (hazard ratio [HR], 3.19; P < .001), with no significant change in long-term survival beyond this time point (HR, 0.98; P = .92). In higher-risk patients, allo-HCT significantly increased the risk of death in the first 2 years after transplant (HR 1.46; P = .01) but not beyond (HR, 0.60; P = .09). Performing allo-HCT before AML transformation decreases life expectancy in lower-risk patients but may be considered in higher-risk patients.
Hematopoietic Stem-Cell Transplantation for Advanced Systemic Mastocytosis
Üstün, Celalettin , Reiter, Andreas , Scott, Bart L. , Nakamura, Ryotaro , Damaj, Gandhi , Kreil, Sebastian, Shanley, Ryan , Baurmann, H., Doubek, Michael , Hsu, Jack W., Tholouli, Eleni , Gromke, Tanja , Godley, Lucy A. , Pagano, Livio , Gilman, Andrew L. , Wagner, Eva , Shwayder, Tor , Papadopoulos, Esperanza B. , Böhm, Alexandra, Vercellotti, Gregory M. , Lint, Maria Teresa Van, Rabitsch, Werner , Pullarkat, Vinod , Legrand, Faézeh, Yakoub‐Agha, Ibrahim , Saber, Wael , Barrett, J. Carl, Hermine, Olivier , Hägglund, Hans , Sperr, Wolfgang R. , Alyea, Edwin P. , Deeg, H. Joachim , Weisdorf, Daniel J. , Akin, Cem , Valent, Peter , Shore, Tsiporah, Baurmann, Herrad, Stuart, Robert, Gruhn, Bernd, Gilman, Andrew, Wagner, Eva Maria, Bornhäuser, Martin, Vercellotti, Gregory, Van Lint, Maria Teresa, Schmid, Christoph, Yakoub-Agha, Ibrahim, Barrett, John , Popat, Uday, Devine, Steven, Weisdorf, Daniel, Hogan, William J. , Perales, Miguel‐Angel , Shore, Tsiporah B. , Stuart, Robert K. , Gruhn, Bernd , Bornhäuser, Martin , Schmid, Christoph , Popat, Uday R , Devine, Steven M. , Weisdorf, Dan , Sperr, Wolfgang , Valent, Peter , Yakoub-Agha, Ibrahim , Hermine, Olivier , Akin, Cem , Godley, Lucy A
Journal of Clinical Oncology | August 26, 2014 | Volume: 32 | Issue: 29 | Pages: 3264-3274
PurposeAdvanced 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 MethodsIn 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).ResultsResponses 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.ConclusionAlloHCT 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.