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660 Comparative Study for Haplo, MSD and MUD Allo-Hcts for AML Patients with KMT2A Rearrangement: A Study from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Program: Oral and Poster Abstracts
Type: Oral
Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Use of Molecular Techniques for Risk Stratification
Hematology Disease Topics & Pathways:
Research, adult, Clinical Research, Therapies, registries, Human, Study Population
Sunday, December 10, 2023: 5:45 PM

Yishan Ye, MD1*, Myriam Labopin2*, Depei Wu3*, Jia Chen4*, Tobias Gedde-Dahl, MD5*, Didier Blaise, MD, PhD6, Regis Peffault De Latour7*, Édouard Forcade8*, Urpu Salmenniemi9*, Sébastien Maury10*, Jurjen Versluis, MD, PhD11*, Ali Bazarbachi, MD, PhD12, Arnon Nagler, MD13, Eolia Brissot14,15*, Lin Li16*, Yi Luo17*, Jimin Shi17*, Fabio Ciceri18*, He Huang, MD16*, Mohamad Mohty, MD, PhD19,20 and Norbert Claude Gorin Sr.15,21

1Bone Marrow Transplantation Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Paris, France
2EBMT Statistical Unit, Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France
3The First Affilliated Hospital of Soochow University, Suzhou, China
4National Clinical Research Center for Hematologic Diseases, The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, Institute of Blood and Marrow Transplantation, Collaborative Innovation Center of Hematology, Soochow Univers, Suzhou, China
5Oslo University Hospital, Rikshospitalet, Clinic for Cancer Medicine, Hematology Department, Section for Stem Cell Transplantation, Oslo, Norway
6Program of Transplant and cellular immunotherapy, Department of Hematology, Institut Paoli Calmettes, Marseille, France
7Hôpital Saint-Louis,, Paris, France
8Service d'Hématologie Clinique et Thérapie Cellulaire, CHU Bordeaux, Pessac, France
9Dept of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland
10Service d’Hématologie, Hôpital Henri Mondor, Créteil, France
11Erasmus MC Cancer Institute, University Medical Center Rotterdam, Department of Hematology, Rotterdam, Netherlands
12American University of Beirut Dept. of Medicine, Beirut, Lebanon
13Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel Hashomer, Israel
14Sorbonne Université Service d' Hématologie Clinique et Thérapie Cellulaire, Hospital Saint-Antoine, Centre de Recherche Saint-Antoine (CRSA), Paris, France
15EBMT Paris Office, Hôpital Saint Antoine, Paris, France
16The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China
17Bone Marrow Transplantation Center, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China
18Unit of Hematology and Stem Cell Transplantation, Ospedale San Raffaele, University Vita-Salute San Raffaele, Milan, Italy
19Saint-Antoine Hospital, Sorbonne University, Paris, France
20Department of Hematology and Cell Therapy, EBMT Paris study office, Saint Antoine Hospital, Paris, France
21Department of Hematology and Cell therapy, Hospital Saint-Antoine, Sorbonne University, Paris, France


Acute myeloid leukemia (AML) patients with KMT2A rearrangement (KMT2Ar, except for t(9;11)(p21.3;q23.3)/MLLT3::KMT2A) are classified as adverse risk in the 2022 European LeukemiaNet (ELN) classification. There is uncertainty on their outcome following allogeneic cell transplantation (allo-HCT). We compared, in an European Society for Blood and Marrow Transplantation (EBMT) global multi-center registry-based analysis, outcomes following an allogeneic stem cell transplantation (allo-HCT) with either a matched sibling donor (MSD), matched unrelated donor (MUD) or haploidentical donor (HAPLO).


Data from 586 AML patients with KMT2Ar receiving a first allo-HCT in 183 EBMT centers from 2010-2022 were analyzed. All patients achieved first complete remission (CR1) before transplantation. Patients with t(9;11), without details on translocation, receiving mismatched UD (<10/10), umbilical cord blood, or grafts with ex-vivo manipulation were excluded. Univariant analysis and Cox regression models were used.


KMT2A rearranged AML patients received allo-HCT from 201 MSD, 256 10/10 MUD, and 129 HAPLOs. For KMT2Ar type, t(6;11), t(11;19), t(10;11) or other translocations account for 27.8%, 37.5%, 23.9% and 10.8%, respectively. Peripheral blood was the major graft source (83.2%), and myeloablative conditioning accounted for the majority (60.2%) of all allo-HCTs. Engraftment rates were comparable in the three cohorts (MSD 99.5%, MUD 98.4% and HAPLO 96.9% p=0.17). AML relapse was the major cause of death in each donor cohort.

On univariate analysis, HAPLO was associated with a lower 2-year relapse incidence (RI) (MSD 41.1%, MUD 37.2%, HAPLO 20%) but a higher 2-year non-relapse mortality (NRM) (MSD 6.1%, MUD 19.3%, HAPLO 21.3%) compared with MSD and MUD. The 180-day cumulative incidence of grade II-IV acute GVHD (aGVHD) was lower in MSD (18.8%) than in MUD (31.3%) or HAPLO groups (26.5%), respectively. The 2-year leukemia-free survival (LFS), overall survival (OS) and GVHD-free/relapse free survival (GRFS) were comparable among the three cohorts. Lower RI was observed in patients with t(11;19) compared with t(6;11) or t(10;11). Complex karyotype (CK) (at least 3 abnormalities) or monosomal karyotype (MK) were both associated with higher RI compared with those without. In addition, in-vivo T cell depletion was related to lower intensive chronic GVHD (cGVHD) incidence.

On multivariate analysis, HAPLO HCT was associated with lower RI (hazard ratio [HR=0.46], 95% CI 0.28-0.77; p<0.01) but higher incidence of NRM (HR=2.36, 95% CI 1.17-4.78; p<0.01) compared with MSD, while HAPLO had no significant impact on LFS, OS and GRFS. MUD HCT was associated with higher incidence of grade II-IV aGVHD (HR=1.82, 95% CI 1.19-2,78; p<0.01), NRM (HR=2.73, 95% CI 1.51-4.94; p<0.01), and lower LFS (HR=1.37, 95% CI 1.03-1.83; p<0.05) compared with MSD (reference). Concerning translocation type, t(11;19) was associated with a lower RI (HR=0.64, 95% CI 0.43-0.94; p=0.022), a better LFS (HR=0.66, 95% CI 0.48-0.91; p=0.012), OS (HR=0.63, 95% CI 0.45-0.89; p=0.008) and GRFS (HR=0.75, 95% CI 0.57-1; p=0.049) compared with t(6;11)(reference). Translocations other than t(6;11), t(11;19) or t(10;11) were related to a lower RI (HR=0.45, 95% CI 0.24-0.87) and better OS (HR=0.57, 95% CI 0.34-0.95) compared to t(6;11). MK was associated with higher RI (HR=1.59, 95% CI 1.04-2.45; p<0.05) and lower LFS (HR=1.47, 95% CI 1.01-2.14; p<0.05).


For AML patients with KMT2Ar, HAPLO HCT was associated with a lower RI but higher NRM, leading to similar LFS, OS and GRFS compared with MUD and MSD HCTs. In the setting of allo-HCT, t(11;19) seems to be associated with favorable outcomes compared with other KMT2Ar.

Disclosures: Peffault De Latour: Jazz Pharmaceuticals: Honoraria. Forcade: Jazz: Other: Travel support; Astellas: Speakers Bureau; MSD: Other: Travel support; Sanofi: Speakers Bureau; GSK: Speakers Bureau; Novartis: Consultancy, Other: Travel support, Speakers Bureau; Gilead Sciences: Other: Travel support, Speakers Bureau; Alexion: Other: Travel support, Speakers Bureau. Salmenniemi: Astra Zeneca: Membership on an entity's Board of Directors or advisory committees; Immedia Pharma AB: Membership on an entity's Board of Directors or advisory committees; Takeda: Membership on an entity's Board of Directors or advisory committees; Viatris: Consultancy. Versluis: ExCellThera: Consultancy; AbbVie: Honoraria. Ciceri: ExCellThera: Other: Scientific Advisory Board . Mohty: JAZZ PHARMACEUTICALS: Honoraria, Research Funding.

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