The best donor option for acute myeloid leukemia (AML) patients lacking an HLA-matched donor has remained intensively debated. We herein report the results of a large retrospective registry study comparing hematopoietic cell transplantation (HCT) outcomes between double-unit umbilical cord blood transplantation (dCBT, n = 209) versus 9/10 HLA-matched unrelated donor (UD) with posttransplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis (UD 9/10, n = 270) in patients with AML in first complete remission (CR1). Inclusion criteria consisted of adult patient, AML in CR1 at transplantation, either peripheral blood stem cells (PBSC) from UD 9/10 with PTCy as GVHD prophylaxis or dCBT without PTCy, transplantation between 2013 and 2021, and no in vivo T-cell depletion. The 180-day cumulative incidence of grade II-IV acute GVHD was 29% in UD 9/10 versus 44% in dCBT recipients (p = .001). After adjustment for covariates, dCBT recipients had a higher non-relapse mortality (HR = 2.35, 95% CI: 1.23-4.48; p = .01), comparable relapse incidence (HR = 1.12, 95% CI: 0.67-1.86; p = .66), lower leukemia-free survival (HR = 1.5, 95% CI: 1.01-2.23; p = .047), and lower overall survival (HR = 1.66, 95% CI: 1.08-2.55; p = .02) compared with patients receiving UD 9/10 HCT. In summary, our results suggest that transplantation outcomes are better with UD 9/10 with PTCy-based GVHD prophylaxis than with dCBT for AML patients in CR1. These data might support the use of UD 9/10 with PTCy-based GVHD prophylaxis over dCBT in AML patients lacking an HLA-matched donor.

CHU Grenoble Alpes Université Grenoble Alpes Grenoble France

CHU Nantes Nantes France

Clinical Hematology and Cellular Therapy Department Saint Antoine Hospital Paris France

Department of Hematology Erasmus MC Cancer Institute University Medical Center Rotterdam Rotterdam Netherlands

EBMT Paris Study Unit Paris France

Faculty of Medicine and Health Sciences University of Nottingham Nottingham UK

Hematology Department University Hospital La Fe Valencia Spain

Hospital Clinic Barcelona Spain

INSERM UMRs 938 Paris France

Institute of Hematology and Blood Transfusion Prague Czech Republic

IRCCS Ospedale San Raffaele s r l Haematology and BMT Milan Italy

Laboratory of Hematology GIGA I3 University of Liege and CHU of Liège Liege Belgium

Leiden University Medical Center Leiden The Netherlands

Programme de transplantation et d'immunothérapie cellulaire département d'hématologie Institut Paoli Calmettes Laboratoire management sport cancer Aix Marseille université Marseille France

RM Gorbacheva Research Institute Pavlov University Saint Petersburg Russian Federation

Royal Marsden Hospital London UK

Service d'Hématologie Clinique et Thérapie Cellulaire CHU Bordeaux Bordeaux France

Sorbonne University Paris France

University Hospitals Bristol and Weston NHS Foundation Trust Bristol UK

Vita Salute San Raffaele University Milano Italy

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