BACKGROUND: The aim of this study was to compare two immunosuppressive strategies, based on the use of either rabbit antithymocyte globulin (ATG) or posttransplant cyclophosphamide (PTCY), as a prophylaxis of graft-versus-host disease (GVHD) for patients with acute lymphoblastic leukemia (ALL) in first complete remission who underwent hematopoietic cells transplantation from matched unrelated donors. METHODS: Overall, 117 and 779 adult patients who received PTCY and ATG, respectively, between the years 2015 and 2020 were included in this retrospective study. The median patient age was 40 and 43 years in the PTCY and ATG groups, respectively, and 37% and 35% of patients, respectively, had Philadelphia chromosome-positive ALL. RESULTS: In univariate analysis, the cumulative incidence of acute and chronic GVHD did not differ significantly between the study groups. The cumulative incidence of relapse at 2 years was reduced in the PTCY group (18% vs. 25%; p = .046) without a significant impact on nonrelapse mortality (11% vs. 16% in the ATG group; p = .29). The rates of leukemia-free survival (LFS) and overall survival were 71% versus 59%, respectively (p = .01), and 82% versus 74%, respectively (p = .08). In multivariate analysis, the receipt of ATG compared with PTCY was associated with a reduced risk of extensive chronic GVHD (hazard ratio, 0.54; 95% confidence interval, 0.3-0.98; p = .04) and an increased risk of low LFS (hazard ratio, 1.57; 95% confidence interval, 1.01-2.45; p = .045). CONCLUSIONS: The receipt of ATG compared with PTCY, despite the reduced risk of extensive chronic GVHD, is associated with inferior LFS in adults with ALL who undergo hematopoietic cell transplantation from 10/10 human leukocyte antigen-matched unrelated donors. These findings warrant verification in prospective trials.

Bone Marrow Transplant Unit Department of Hematology La Fe University and Polytechnic Hospital Valencia Spain

Bone Marrow Transplantation Center Eppendorf University Hospital Hamburg Germany

Department of Hematology and Cellular Therapy National Institute of Health and Medical Research Unit UMR S 938 Sorbonne University and St Anthony Scientific Research Center Public Assistance Hospital of Paris St Anthony Hospital Paris France

Department of Hematology Hospital Papa Giovanni XXIII Bergamo Italy

European Society for Blood and Marrow Transplantation Paris Study Office CEREST TC Paris France

Hematology Division Chaim Sheba Medical Center Ramat Gan Israel

Hematology National Institute of Health and Medical Research Unit U976 Public Assistance Hospital of Paris St Louis Hospital University of Paris Paris France

Hematopoietic Cell Transplantation Unit Hematology Department G Papanikolaou Hospital Thessaloniki Greece

Institute of Hematology and Blood Transfusion Prague Czech Republic

Institute of Hematology L e A Seràgnoli IRCCS University Hospital Bologna Italy

IRCCS San Raffaele Scientific Institute Milan Italy

Maria Sklodowska Curie National Research Institute of Oncology Gliwice Poland

New Civil Hospital Strasbourg France

RM Gorbacheva Research Institute Pavlov University St Petersburg Russia

Stem Cell Transplantation Unit Helsinki University Hospital Comprehensive Cancer Center Helsinki Finland

Transplantation and Cellular Therapy Program Cancer Research Center of Marseille Paoli Calmettes Institute Marseille France

University Hospital of Patras Patras Greece

University of Zagreb School of Medicine Zagreb Croatia

Vanderbilt University Medical Center Nashville Tennessee USA

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Unrelated donor transplantation with posttransplant cyclophosphamide vs ATG for myelodysplastic neoplasms