Role of allogeneic transplantation in chronic myelomonocytic leukemia: an international collaborative analysis
Marie Robin, Liesbeth C. de Wreede, Eric Padron, Katerina Bakunina, Pierre Fenaux, Linda Köster, Aziz Nazha, Dietrich W. Beelen, Raajit K. Rampal, Katja Sockel, Rami S. Komrokji, Nico Gagelmann, Dirk‐Jan Eikema, Aleksandar Radujkovic, Jürgen Finke, Victoria Potter, Sally Killick, Faézeh Legrand, Éric Solary, Angus Broom, Guillermo Garcia‐Manero, Vittorio Rizzoli, Patrick Hayden, Mrinal M. Patnaik, Francesco Onida, Ibrahim Yakoub‐Agha, Raphaël Itzykson
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.