Allogeneic stem cell transplantation in de novo core-binding factor acute myeloid leukemia in active disease: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Research output: Contribution to journalResearch articleContributedpeer-review

Contributors

  • Sara Tarantino - , Sorbonne Université (Author)
  • Myriam Labopin - , EBMT Paris Study Unit (Author)
  • Robert Zeiser - , University Medical Center Freiburg (Author)
  • Matthias Stelljes - , University of Münster (Author)
  • Thomas Schroeder - , University Hospital Essen (Author)
  • Nicolaus Kröger - , University Hospital Hamburg Eppendorf (Author)
  • Wolfgang Bethge - , University Hospital Tübingen (Author)
  • Jakob Passweg - , University Hospital Basel (Author)
  • Martin Bornhäuser - , Department of Internal Medicine I, University Hospital Carl Gustav Carus Dresden (Author)
  • Christoph Schmid - , University Hospital Augsburg (Author)
  • Johanna Tischer - , Hospital of the Ludwig-Maximilians-University (LMU) Munich (Author)
  • Matthias Eder - , Hannover Medical School (MHH) (Author)
  • Eolia Brissot - , EBMT Paris Study Unit (Author)
  • Jordi Esteve - , Hospital Clinic of Barcelona (Author)
  • Arnon Nagler - , Sheba Medical Center at Tel Hashomer (Author)
  • Mohamad Mohty - , EBMT Paris Study Unit (Author)
  • Fabio Ciceri - , IRCCS Hospital San Raffaele - Milano (Author)

Abstract

Core-binding factor acute myeloid leukemia (CBF-AML) generally has a favorable prognosis, with allogeneic hematopoietic stem cell transplantation (allo-SCT) recommended for relapsed/ refractory (R/R) cases achieving second complete remission (CR). However, clinical outcomes remain suboptimal for patients who relapse or fail to achieve CR following induction chemotherapy. Allo-SCT in non-CR is a potential strategy for such patients, though supporting evidence in CBF-AML is limited. To assess outcomes and prognostic factors of allo-SCT in R/R CBF-AML with active disease, we conducted a retrospective analysis of 610 patients with CBF-AML in non-CR undergoing allo-SCT from 2010 to 2021 across 174 centers within the European Society for Blood and Marrow Transplantation. Graft sources included matched sibling (MSD, n = 151), unrelated (UD, n = 368), and haploidentical donors (Haplo, n = 91). Among patients, 124 had inv(16), and 486 had t(8;21). Two-year overall survival (OS) and leukemia-free survival (LFS) were 53.6% and 42.7%, respectively. Haplo-SCT showed inferior OS compared to MSD (HR 1.79, p = 0.003) and UD (HR 1.64, p = 0.004) and reduced chronic graft-versus-host disease. Patients with t(8;21) exhibited higher relapse incidence (HR 2.04, p = 0.002) and poorer survival outcomes than those with inv(16). These findings confirm the therapeutic role of allo-SCT in R/R CBF-AML in non-CR, supporting its favorable risk profile.

Details

Original languageEnglish
Pages (from-to)1027-1035
Number of pages9
JournalBone marrow transplantation
Volume60
Issue number7
Publication statusPublished - Jul 2025
Peer-reviewedYes

External IDs

Scopus 105003215545

Keywords

Keywords

  • Humans, Leukemia, Myeloid, Acute/therapy, Male, Female, Adult, Middle Aged, Hematopoietic Stem Cell Transplantation/methods, Retrospective Studies, Adolescent, Transplantation, Homologous/methods, Young Adult, Aged, Core Binding Factors, Transplantation Conditioning/methods