Impact of Wilms’ tumor 1 gene (WT1) mutation on outcome of allogeneic hematopoietic-cell transplantation for acute myeloid leukemia: a retrospective multicenter cohort study from the ALWP/EBMT registry

Nagler, Arnon;Esteve, Jordi;Galimard, Jacques-Emmanuel;Sanz, Jaime;Mohty, Mohamad;et.al.
(2025) Bone Marrow Transplantation — Vol. 61, n° 1, p. 44-50 (2025)

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  • Nagler, Arnonorcid-logo
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  • Esteve, Jordiorcid-logo
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  • Galimard, Jacques-Emmanuelorcid-logo
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  • Sanz, Jaimeorcid-logo
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  • Poire, XavierUCLouvain
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  • Mohty, Mohamadorcid-logo
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Abstract
(en) We compared transplantation outcomes of AML patients with WT1 mutation (mWT1), identified by next-generation sequencing, to those of patients with wild-type WT1 AML (wtWT1). 703 patients were included, 50 with mWT1 and 653 with wtWT1. Patients with mWT1 were younger (median age: 45.6 vs. 56.4 years, p < 0.001), with a higher proportion of females (66% vs. 47.6%, p = 0.01), higher frequency of mutations in FLT3-ITD (38.3% vs. 21.7%, p = 0.01) and CEBPA (15.8% vs. 5.7%, p = 0.03). Donors were matched siblings in 30.6%, unrelated in 45.6%, and haploidentical in 22.1%. A higher percentage of mWT1 vs. wtWT1 patients received in vivo T-cell depletion (66% vs. 51%, p = 0.03) and 58% vs. 47.1% received myeloablative conditioning. 49 patients with mWT1 were matched to 127 wtWT1 patients in matched-pairs analysis. Outcomes (mWT1 vs. wtWT1) were not significantly different: relapse (2 y: 28.8% vs. 30.4%, HR: 1.14, p = 0.64), NRM (2 y: 15.5% vs. 9.9%, HR: 1.41, p = 0.49), LFS (2 y: 55.7% vs. 59.6%, HR: 1.21, p = 0.39), OS (2 y: 65.4% vs. 73.3%, p = 0.66), and chronic GVHD (2 y:24.3% vs. 25.4%, p = 0.95). In conclusion, WT1 mutation did not influence transplantation outcomes of AML patients in CR1. Bone Marrow Transplantation (2026) 61:44-50; https://doi.
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Nagler, A., Esteve, J., Galimard, J.-E., Sanz, J., Poire, X., Collin, M., Maertens, J., Huang, H., Itäla-Remes, M., Halahleh, K., Pascual Cascon, M., Chiusolo, P., Griskevicius, L., Kaare, A., De Becker, A., Jindra, P., Pérez-Simón, J., Bazarbachi, A., Brissot, E., et al. (2025). Impact of Wilms’ tumor 1 gene (WT1) mutation on outcome of allogeneic hematopoietic-cell transplantation for acute myeloid leukemia: a retrospective multicenter cohort study from the ALWP/EBMT registry. Bone Marrow Transplantation, 61(1), 44-50. https://doi.org/10.1038/s41409-025-02727-7 (Original work published 2025)