Frederic Baron, MD, PhD1, Myriam Labopin2,3*, Jurjen Versluis, MD, PhD4*, Jan Vydra5*, Peter A. Von Dem Borne, MD, PhD6*, Emma Nicholson, MD, PhD7*, Didier Blaise8*, Stephen Robinson Jr.9*, Aleksandr Kulagin Sr.10*, Claude-Éric Bulabois, MD11*, Montserrat Rovira, MD, PhD12*, Patrice Chevallier, MD13, Édouard Forcade14*, Jenny Louise Byrne, MD, PhD15*, Jaime Sanz Caballer, MD, PhD16*, Annalisa Ruggeri, MD, PhD17*, Mohamad Mohty, MD, PhD18 and Fabio Ciceri19*
1University of Liege, Liege, Belgium
2EBMT Statistical Unit, Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France
3Hôpital Saint-Antoine, Université Sorbonne, INSERM UMRs 938, Paris, France
4Erasmus MC Cancer Institute, University Medical Center Rotterdam, Department of Hematology, Rotterdam, Netherlands
5Institute of Hematology and Blood Transfusion, Prague, Czech Republic
6Hematology, Leiden University Medical Center, Leiden, Netherlands
7Department of Haematology, Royal Marsden Hospital, London, United Kingdom
8Institut Paoli-Calmettes, Aix Marseille Université, Management Sport Cancer Laboratoire (MSC), Marseille, France
9University Hospitals Bristol, Bristol, GBR
10b. First State Pavlov Medical University of St. Petersburg, Raisa Gorbacheva Memorial Research Institute for Paediatric Oncology, Hematology, and Transplantation, St-Petersburg, Russia, St. Petersburg, RUS
11Hématologie Soins Intensifs, CHU Grenoble Alpes, Grenoble, France
12Hospital Clínic de Barcelona, Barcelona, Spain
13Service d'hématologie, CHU de Nantes, Nantes, France
14Service d'Hématologie Clinique et Thérapie Cellulaire, CHU Bordeaux, F-33000, Bordeaux, France
15Nottingham University, Nottingham, ENG, GBR
16Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Valencia, ESP
17Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
18Department of Haematology and EBMT Paris study office / CEREST-TC, Saint Antoine Hospital, INSERM UMR 938 and Université Pierre et Marie Curie, Paris, France
19Unit of Hematology and Stem Cell Transplantation, Ospedale San Raffaele, University Vita-Salute San Raffaele, Milan, Italy
Background: The best donor option for acute myeloid leukemia (AML) patients lacking an HLA-matched donor has remained unknown. The recently reported BNT CTN 1101 trial observed higher non-relapse mortality (NRM) and lower overall survival (OS) in patients randomized to double-unit unrelated umbilical cord blood transplantation (dCBT) in comparison to those randomized to HLA-haploidentical bone marrow transplantation with post-transplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis
1. In addition, recent registry studies observed at least as good transplantation outcomes in AML patients given grafts from 9/10 HLA-matched unrelated donor (UD 9/10) with PTCy-based GVHD prophylaxis as those given grafts from HLA-haploidentical donors
2,3. These observations prompted us to perform a retrospective registry study comparing hematopoietic cell transplantation (HCT) outcomes between UD 9/10 and dCBT.
Methods: 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, transplantation between 2013 and 2021, and no
in vivo T-cell depletion.
Results: A total of 208 dCBT patients and 270 UD 9/10 allo-HCT were included. The 180-day cumulative incidence of grade II-IV acute GVHD was 29% in UD 9/10 versus 44% in dCBT recipients (P=0.001). The 2-year cumulative incidences of relapse and of NRM were 23.5% and 12.5%, respectively, in UD 9/10 recipients versus 27% (P=0.39) and 18% (P=0.07), respectively in dCBT recipients. Two-year OS and LFS were 70% and 64%, respectively, in UD 9/10 recipients versus 60% (P=0.016) and 55% (P=0.028), respectively in dCBT recipients. In multivariate analyses, in comparison with UD 9/10 recipients, dCBT patients had a higher non-relapse mortality (HR=2.35, 95% CI: 1.23-4.48; P=0.01), comparable relapse incidence (HR=1.12, 95% CI: 0.67-1.86; P=0.66), lower leukemia-free survival (HR=1.5, 95% CI: 1.01-2.23; P=0.047), and lower overall survival (HR=1.66, 95% CI: 1.08-2.55; P=0.02).
Conclusions: 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 in AML patients lacking an HLA-matched donor.
References
- Fuchs, E. J. et al. Double unrelated umbilical cord blood vs HLA-haploidentical bone marrow transplantation: the BMT CTN 1101 trial. Blood 137, 420–428 (2021).
- Baron, F. et al. Comparison of HLA-mismatched unrelated donor transplantation with post-transplant cyclophosphamide versus HLA-haploidentical transplantation in patients with active acute myeloid leukemia. Bone Marrow Transplant. 57, 1657–1663 (2022).
- Battipaglia, G. et al. Post-transplant cyclophosphamide in one-antigen mismatched unrelated donor transplantation versus haploidentical transplantation in acute myeloid leukemia: a study from the Acute Leukemia Working Party of the EBMT. Bone Marrow Transplant. 57, 562–571 (2022).
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