Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Article
  • Published:

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

Bone Marrow Transplantation (2025)Cite this article

Subjects

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.

Access through your institution
Buy or subscribe

This is a preview of subscription content, access via your institution

Access options

Access through your institution

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Fig. 1: Transplant outcomes for patients with CBF-AML allografted during the active disease phase according to CBF subtype.
Fig. 2: Transplant outcomes for patients with CBF-AML allografted during the active disease phase according to donor type.

Similar content being viewed by others

Data availability

Data will be available upon request to the Acute Leukemia Working Party (ALWP) office of the European Society for Blood and Marrow Transplantation (EBMT).

References

  1. Darwish C, Farina K, Tremblay D. The core concepts of core binding factor acute myeloid leukemia: Current considerations for prognosis and treatment. Blood Rev. 2023;62:101117. https://doi.org/10.1016/j.blre.2023.101117.

    Article  PubMed  Google Scholar 

  2. Ye Y, Labopin M, Gérard S, Yakoub-Agha I, Blau IW, Aljurf M, et al. Lower relapse incidence with haploidentical versus matched sibling or unrelated donor hematopoietic cell transplantation for core-binding factor AML patients in CR2: A study from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Am J Hematol. 2024. https://doi.org/10.1002/ajh.27342.

  3. Halaburda K, Labopin M, Mailhol A, Socié G, Craddock C, Aljurf M, et al. Allogeneic stem cell transplantation in second complete remission for core binding factor acute myeloid leukemia: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Haematologica. 2020;105:1723–30. https://doi.org/10.3324/haematol.2019.222810.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  4. Solh M, Yohe S, Weidorf D, Ustun C. Core-binding factor acute myeloid leukemia: heterogeneity, monitoring and therapy. Am J Hematol. 2014;89:1121–31.

    Article  PubMed  Google Scholar 

  5. Sinha C, Cunningham LC, Liu PP. Core binding factor acute myeloid leukemia: new prognostic categories and therapeutic opportunities. Semin Hematol. 2015;52:215–22.

  6. Al-Harbi S, Aljurf M, Mohty M, Almohareb F, Ahmed SOA. An update on the molecular pathogenesis and potential therapeutic targeting of AML with t(8;21)(q22;q22.1);RUNX1-RUNX1T1. Blood Adv. 2020;4:229–38.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  7. Döhner H, Estey E, Grimwade D. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129:424–47. https://doi.org/10.1182/blood-2016-08-733196.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  8. Appelbaum FR, Kopecky KJ, Tallman MS. The clinical spectrum of adult acute myeloid leukaemia associated with core binding factor translocations. Br J Haeamtol. 2006;135:165–73.

    Article  Google Scholar 

  9. Brissot E, Labopin M, Ehninger G, Stelljes M, Brecht A, Ganser A, et al. Haploidentical versus unrelated allogeneic stem cell transplantation for relapsed/refractory acute myeloid leukemia: a report on 1578 patients from the Acute Leukemia Working Party of the EBMT. Haematologica. 2019;104:524–32. https://doi.org/10.3324/haematol.2017.187450.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  10. Maffini E, Labopin M, Kröger N, Finke J, Stelljes M, Schroeder T et al. Allogeneic hematopoietic cell transplantation for older patients with AML with active disease. A study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2024. https://doi.org/10.1038/s41409-024-02275-6.

  11. Duval M, Klein JP, He W. Hematopoietic stem-cell transplantation for acute leukemia in relapse or primary induction failure. J Clin Oncol. 2010;28:3730–8. https://doi.org/10.1200/JCO.2010.28.8852.

    Article  PubMed  PubMed Central  Google Scholar 

  12. Nagler A, Savani BN, Labopin M. Outcomes after use of two standard ablative regimens in patients with refractory acute myeloid leukaemia: a retrospective, multicentre, registry analysis. Lancet Haematol. 2015;2:e384–392.

    Article  PubMed  Google Scholar 

  13. Gyurkocza B, Lazarus HM, Giralt S. Allogeneic hematopoietic cell transplantation in patients with AML not achieving remission: potentially curative therapy. Bone Marrow Transplant. 2017;52:1083–90.

    Article  CAS  PubMed  Google Scholar 

  14. Jabbour E, Daver N, Champlin R, Mathisen M, Oran B, Ciurea S, et al. Allogeneic stem cell transplantation as initial salvage for patients with acute myeloid leukemia refractory to high-dose cytarabine-based induction chemotherapy. Am J Hematol. 2014;89:395–8.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  15. Brissot E, Labopin M, Stelljes M. Comparison of matched sibling donors versus unrelated donors in allogeneic stem cell transplantation for primary refractory acute myeloid leukemia: a study on behalf of the Acute Leukemia Working Party of the EBMT. J Hematol Oncol. 2017;10:130.

    Article  PubMed  PubMed Central  Google Scholar 

  16. Craddock C, Labopin M, Pillai S. Factors predicting outcome after unrelated donor stem cell transplantation in primary refractory acute myeloid leukaemia. Leukemia. 2011;25:808–13. https://doi.org/10.1038/leu.2011.13.

    Article  CAS  PubMed  Google Scholar 

  17. Bacigalupo A, Ballen K, Rizzo D, Giralt S, Lazarus H, Ho V, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009;15:1628–33.

    Article  PubMed  PubMed Central  Google Scholar 

  18. Harris AC, Young R, Devine S, Hogan WJ, Ayuk F, Bunworasate U, et al. International, multicenter standardization of acute graft-versus-host disease clinical data collection: a report from the Mount Sinai acute GVHD international consortium. Biol Blood Marrow Transplant. 2016;22:4–10.

    Article  PubMed  Google Scholar 

  19. Jagasia MH, Greinix HT, Arora M, Williams KM, Wolff D, Cowen EW, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. The 2014 diagnosis and staging working group report. Biol Blood Marrow Transplant. 2015;21:389–401.e381.

    Article  PubMed  Google Scholar 

  20. Ruggeri A, Labopin M, Ciceri F, Mohty M, Nagler A. Definition of GvHD-free, relapse-free survival for registry-based studies: an ALWP-EBMT analysis on patients with AML in remission. Bone Marrow Transplant. 2016;51:610–1.

    Article  CAS  PubMed  Google Scholar 

  21. Döhner H, Wei AH, Appelbaum FR, Craddock C, DiNardo CD, Dombret H, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN. Blood. 2022;140:1345–77. https://doi.org/10.1182/blood.2022016867.

    Article  CAS  PubMed  Google Scholar 

  22. Brunner A, Blonquist T, Sadrzadeh H, Perry A, Attar E, Amrein P, et al. Population-based disparities in survival among patients with Core-binding factor acute myeloid leukemia: a SEER database analysis. Leuk Res. 2014;38:773–80.

    Article  PubMed  PubMed Central  Google Scholar 

  23. Tazi Y, Arango-Ossa JE, Zhou Y, Bernard E, Thomas I, Gilkes A, et al. Unified classification and risk-stratification in acute myeloid leukemia. Nat Commun. 2022;13:4622.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  24. Wang Y, Wu DP, Liu QF, Qin YZ, Wang JB, Xu LP, et al. In adults with t(8;21)AML, posttransplant RUNX1/RUNX1T1-based MRD monitoring, rather than c-KIT mutations, allows further risk stratification. Blood. 2014;124:1880–6.

    Article  CAS  PubMed  Google Scholar 

  25. Hospital MA, Prebet T, Bertoli S, Thomas X, Tavernier E, Braun T, et al. Core binding factor acute myeloid leukemia in first relapse: a retrospective study from the French AML intergroup. Blood. 2014;124:1312–1319.17.

    Article  CAS  PubMed  Google Scholar 

  26. Burnett AK, Goldstone A, Hills RK, Milligan D, Prentice A, Yin J, et al. Curability of patients with acute myeloid leukemia who did not undergo transplanta tion in first remission. J Clin Oncol. 2013;31:1293–301.

    Article  PubMed  Google Scholar 

  27. Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory and early relapsed acute myeloid leukemia. Blood. 2015;126:319–27.

    Article  CAS  PubMed  Google Scholar 

  28. Yanada M, Yamasaki S, Kondo T, Kawata T, Harada K, Uchida N, et al. Allogeneic hematopoietic cell transplantation for patients with acute myeloid leukemia not in remission. Leukemia. 2024;38:513–20.

    Article  CAS  PubMed  Google Scholar 

  29. Cairoli R, Beghini A, Grillo G, Nadali G, Elice F, Ripamonti CB, et al. Prognostic impact of c-KIT mutations in core binding factor leukemias: an Italian retrospective study. Blood. 2006;107:3463–8.

    Article  CAS  PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Department of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy

    Sara Tarantino

  2. Department of Hematology and Cell therapy, Saint Antoine Hospital, Paris, France

    Sara Tarantino, Eolia Brissot & Mohamad Mohty

  3. Hematology Division and Bone Marrow Unit, IRCCS San Gerardo, Monza, Italy

    Sara Tarantino

  4. Sorbonne University, Paris, France

    Sara Tarantino, Eolia Brissot & Mohamad Mohty

  5. EBMT Paris study office / CEREST-TC, Paris, France

    Myriam Labopin, Eolia Brissot & Mohamad Mohty

  6. Leiter der Abteilung für Tumorimmunologie und Immunregulation Klinik für Innere Medizin, Schwerpunkt Hämatologie, Onkologie und Stammzelltransplantation, Universitätsklinikum Freiburg, Hugstetter Str, 55 79106, Freiburg, Germany

    Robert Zeiser

  7. University of Muenster, Muenster, Germany

    Matthias Stelljes

  8. University Hospital Essen, Essen, Germany

    Thomas Schroeder

  9. Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

    Nicolaus Kröger

  10. Universitaet Tuebingen, Tuebingen, Germany

    Wolfgang Bethge

  11. University Hospital, Hematology – Basel, Basel, Switzerland

    Jakob Passweg

  12. Universitaetsklinikum Dresden, Dresden, Germany

    Martin Bornhäuser

  13. Klinikum Augsburg- University of Augsburg, Augsburg, Germany

    Christoph Schmid

  14. Klinikum Grosshadern- University of Munich, Munich, Germany

    Johanna Tischer

  15. Hannover Medical School, Hannover, Germany

    Matthias Eder

  16. Hospital Clinic Institut d’investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain

    Jordi Esteve

  17. Hematology Division, Chaim Sheba Medical Center and Tel Aviv University, Tel-Hashomer, Ramat-Gan, Israel

    Arnon Nagler

  18. Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Hospital, Vita-Salute San Raffaele University, Milano, Italy

    Fabio Ciceri

Authors
  1. Sara Tarantino
    View author publications

    Search author on:PubMed Google Scholar

  2. Myriam Labopin
    View author publications

    Search author on:PubMed Google Scholar

  3. Robert Zeiser
    View author publications

    Search author on:PubMed Google Scholar

  4. Matthias Stelljes
    View author publications

    Search author on:PubMed Google Scholar

  5. Thomas Schroeder
    View author publications

    Search author on:PubMed Google Scholar

  6. Nicolaus Kröger
    View author publications

    Search author on:PubMed Google Scholar

  7. Wolfgang Bethge
    View author publications

    Search author on:PubMed Google Scholar

  8. Jakob Passweg
    View author publications

    Search author on:PubMed Google Scholar

  9. Martin Bornhäuser
    View author publications

    Search author on:PubMed Google Scholar

  10. Christoph Schmid
    View author publications

    Search author on:PubMed Google Scholar

  11. Johanna Tischer
    View author publications

    Search author on:PubMed Google Scholar

  12. Matthias Eder
    View author publications

    Search author on:PubMed Google Scholar

  13. Eolia Brissot
    View author publications

    Search author on:PubMed Google Scholar

  14. Jordi Esteve
    View author publications

    Search author on:PubMed Google Scholar

  15. Arnon Nagler
    View author publications

    Search author on:PubMed Google Scholar

  16. Mohamad Mohty
    View author publications

    Search author on:PubMed Google Scholar

  17. Fabio Ciceri
    View author publications

    Search author on:PubMed Google Scholar

Contributions

ST, EB and MM designed the study. ML performed the statistical analysis. ST wrote the manuscript. EB and MM revised the manuscript. RZ, MS, TS, NK, WB, JP, MB, CS, JT, ME, JE, AN, MM, and FC were the principal investigators at the centers recruiting the highest number of patients for the study and contributed key clinical data. All authors were involved in manuscript preparation and approved the final version.

Corresponding author

Correspondence to Sara Tarantino.

Ethics declarations

Competing interests

The authors declare no competing interests.

Ethics approval and consent to participate

The study was approved by the Institutional Review Board of the ALWP of the EBMT and complied with country-specific regulatory requirements. All methods involving human participants were carried out in accordance with the ethical standards of the institutional and national research committee and with the 1964 Helsinki Declaration and its later amendments. Patients provide informed consent authorizing the use of their personal information for research purposes.

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Supplementary information

Rights and permissions

Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Tarantino, S., Labopin, M., Zeiser, R. et al. 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. Bone Marrow Transplant (2025). https://doi.org/10.1038/s41409-025-02596-0

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • DOI: https://doi.org/10.1038/s41409-025-02596-0

Search

Advanced search

Quick links