Role of Allogeneic Transplantation In Chronic Myelomonocytic Leukemia: An International Collaborative Analysis

• Published in: Blood Vol. 140; no. 12; pp. 1408 - 1418
• Main Authors: Robin, Marie, de Wreede, Liesbeth C., Padron, Eric, Bakunina, Katerina, Fenaux, Pierre, Koster, Linda, Nazha, Aziz, Beelen, Dietrich W., Rampal, Raajit K., Sockel, Katja, Komrokji, Rami S., Gagelmann, Nico, Eikema, Dirk-Jan, Radujkovic, Aleksandar, Finke, Jürgen, Potter, Victoria, Killick, Sally B., Legrand, Faezeh, Solary, Eric, Broom, Angus, Garcia-Manero, Guillermo, Rizzoli, Vittorio, Hayden, Patrick, Patnaik, Mrinal M., Onida, Francesco, Yakoub-Agha, Ibrahim, Itzykson, Raphael
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 22.09.2022; American Society of Hematology
• Subjects: Adolescent; Adult; Aged; Hematopoietic Stem Cell Transplantation - adverse effects; Humans; Leukemia, Myelomonocytic, Chronic - diagnosis; Leukemia, Myelomonocytic, Chronic - therapy; Leukemia, Myelomonocytic, Juvenile; Life Sciences; Male; Middle Aged; Retrospective Studies; Transplantation, Homologous; Young Adult
• ISSN: 0006-4971; 1528-0020; 1528-0020
• DOI: 10.1182/blood.2021015173

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 aged 18-70 years diagnosed between 2000 and 2014 from an International CMML Dataset (ICD, n=730) and from the EBMT registry (n=384). The prognostic impact of allo-HCT was analyzed through univariable and multivariable time-dependent models and with a multi-state model, accounting for age, sex, CMML prognostic scoring system (CPSS low and intermediate-1: lower-risk, intermediate-2 and high: higher-risk) at diagnosis, and AML transformation. In univariable analysis, lower-risk CMMLs had a 5-year OS of 20% (95%CI 12-33%) with allo-HCT versus 42% (95%CI 35-49%) without allo-HCT (P<0.001). In higher-risk patients, 5-year OS was 27% (95%CI 21-34%) with allo-HCT versus 15% (95%CI 11-22%) without allo-HCT (P=0.13). With multi-state models, performing allo-HCT before AML transformation reduced overall survival in patients with lower risk CMML while 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 two years of transplantation (HR=3.19, 95%CI 2.30-4.42, P<0.001), with no significant change in long-term survival beyond this time point (HR=0.98, 95%CI 0.58-1.64, P=0.92). In higher risk patients, allo-HCT significantly increased the risk of death in the first two years after transplant (HR=1.46, 95%CI 1.09-1.96, P=0.01), but not beyond (HR=0.60, 95%CI 0.34-1.08, P=0.09). Performing allo-HCT before AML transformation decreases life expectancy in lower risk patients but may be considered in higher risk patients.