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ACUTE MYELOID LEUKEMIA

Relationship between donor source, pre-transplant measurable residual disease, and outcome after allografting for adults with acute myeloid leukemia

Leukemia volume 39, pages 381–390 (2025)Cite this article

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Abstract

Lack of HLA-matched related/unrelated donor remains a barrier to allogeneic hematopoietic cell transplantation (HCT) for adult acute myeloid leukemia (AML), with ongoing uncertainty about optimal donor type if more than one alternative donor is available. To assess the relationship between donor type, pre-HCT measurable residual disease (MRD), and post-HCT outcomes, we retrospectively analyzed 1265 myelodysplastic neoplasm (MDS)/AML and AML patients allografted in first or second remission with an HLA-matched sibling (MSD) or unrelated donor (MUD), HLA-mismatched unrelated donor (MMD), an HLA-haploidentical donor, or umbilical cord blood (UCB) at a single institution. Relapse risk was non-significantly higher after HLA-haploidentical and lower after UCB HCT. Non-relapse mortality (NRM) was significantly higher in patients undergoing MMD HCT, HLA-haploidentical HCT, and UCB, translating into significantly lower relapse-free survival (RFS) and overall survival for MMD and HLA-haploidentical HCT. There was a significant interaction between conditioning intensity and post-HCT outcomes for UCB HCT with better RFS for UCB HCT after MAC but higher NRM after non-MAC. In patients with pre-HCT MRD receiving MAC, relapse risk was significantly lower and RFS higher in those who underwent UCB HCT in comparison to MSD/MUD. Together, UCB HCT is a valuable alternative for MAC HCT, particularly in patients with pre-HCT MRD.

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Fig. 1: Post-HCT outcomes for the whole cohort.
Fig. 2: Post-HCT outcomes according to conditioning intensity.
Fig. 3: Relapse-free survival according to pre-HCT MRD status and conditioning intensity.

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Data availability

The dataset analyzed in this study is available from the corresponding author on reasonable request.

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Acknowledgements

Research reported in this publication was supported by grants P01-CA078902, P01-CA018029, and P30-CA015704 from the National Cancer Institute/National Institutes of Health (NCI/NIH), Bethesda, MD, USA. The authors acknowledge the excellent care provided by the physicians and nurses of the HCT teams, the staff in the Long-Term Follow-up office at the Fred Hutchinson Cancer Center, the Hematopathology Laboratory at the University of Washington, and the patients for participating in our research protocols. R.B.W. acknowledges support from the José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research.

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Authors and Affiliations

  1. Translational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA, USA

    Corentin Orvain, Filippo Milano, Eduardo Rodríguez-Arbolí, Effie W. Petersdorf, Brenda M. Sandmaier & Roland B. Walter

  2. Maladies du Sang, CHU d’Angers, Angers, France

    Corentin Orvain

  3. Fédération Hospitalo-Universitaire Grand-Ouest Acute Leukemia, FHU-GOAL, Angers, France

    Corentin Orvain

  4. Université d’Angers, Inserm UMR 1307, CNRS UMR 6075, Nantes Université, CRCI2NA, Angers, France

    Corentin Orvain

  5. Department of Medicine, Division of Hematology and Oncology, University of Washington, Seattle, WA, USA

    Filippo Milano, Effie W. Petersdorf, Brenda M. Sandmaier, Frederick R. Appelbaum & Roland B. Walter

  6. Department of Hematology, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBIS/CSIC), University of Seville, Seville, Spain

    Eduardo Rodríguez-Arbolí

  7. Public Health Sciences Division, Fred Hutchinson Cancer Center, Seattle, WA, USA

    Megan Othus

  8. Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA, USA

    Frederick R. Appelbaum

  9. Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA, USA

    Roland B. Walter

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Contributions

Conception and Design: RBW. Collection and Assembly of Data: CO, FM, ERA, EWP, BMS, FRA, RBW. Data Analysis and Interpretation: CO, FM, MO, RBW. Manuscript Writing: All authors. Final Approval of the Manuscript: All authors.

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Correspondence to Roland B. Walter.

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Employment or Leadership Position: none; Consultant or Advisory Role: none: Stock Ownership: none; Honoraria: none; Research Funding: none; Expert Testimony: none; Patents: none; Other Remuneration: none.

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All patients were treated on Institutional Review Board (IRB)-approved research protocols (all registered with ClinicalTrials.gov) or standard treatment protocols. All analyses conducted herein were performed under a research protocol approved by the Fred Hutchinson Cancer Center’s IRB (#FH2562).

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Orvain, C., Milano, F., Rodríguez-Arbolí, E. et al. Relationship between donor source, pre-transplant measurable residual disease, and outcome after allografting for adults with acute myeloid leukemia. Leukemia 39, 381–390 (2025). https://doi.org/10.1038/s41375-024-02497-z

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