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Haploidentical versus matched unrelated donor hematopoietic cell transplantation for adult patients with refractory/relapsed acute lymphoblastic leukemia: a study from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Bone Marrow Transplantation (2025)Cite this article

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Abstract

The optimal alternative donor for relapsed/refractory acute lymphoblastic leukemia (R/R ALL) patients remains controversial. We retrospectively analyzed adult R/R ALL patients who underwent a first allogeneic hematopoietic cell transplantation (allo-HCT) between 2010 and 2022 from either a haploidentical donor (Haplo-HCT) or a 10/10 matched unrelated donor (MUD-HCT). The analysis comprised 249 patients (80 Haplo-HCT and 169 MUD-HCT). For graft-versus-host disease (GVHD) prophylaxis, all Haplo-HCT recipients received post-transplant cyclophosphamide (PTCy)-based regimen, whereas all MUD-HCT recipients received anti-thymocyte globulin (ATG) only. After propensity score matching, 47 Haplo-HCT and 69 MUD-HCT recipients were enrolled. No significant differences were observed in the incidence of Day 180 grade II–IV aGVHD (34.4% vs. 26.8%; P = 0.27), or 2-year cGVHD (19.2% vs. 13.8%; P = 0.543). However, the incidence of Day 180 grade III–IV aGVHD was significantly higher in the Haplo-HCT group (23.4% vs. 8%; P = 0.048). Two-year overall survival (31.8% vs. 29.2%; P = 0.742), leukemia-free survival (20.4% vs. 24.4%; P = 0.531), relapse incidence (48.1% vs. 53.1%; P = 0.113), or non-relapse mortality (31.5% vs. 22.5%; P = 0.191) were comparable between Haplo-HCT group and MUD-HCT group, respectively. In conclusion, despite a higher incidence of grade III–IV aGVHD, outcomes for R/R ALL patients receiving Haplo-HCT with PTCy were comparable to those receiving 10/10 MUD-HCT with ATG.

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Fig. 1: Incidence of GVHD following Haplo-HCT versus MUD-HCT in R/R ALL patients.
Fig. 2: Outcomes of Haplo-HCT versus MUD-HCT in R/R ALL patients.

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Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author upon reasonable request. The study was presented at the 51st annual meeting of the EBMT, Florence, Italy, April 2025.

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Funding

The study was supported by the National Key R&D Program of China (2022YFC2502700); National Natural Science Foundation of China (82200237, 82370215).

Author information

Author notes

  1. These authors contributed equally: Yuhua Ru, Jia Chen.

Authors and Affiliations

  1. National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, Department of Hematology, The First Affiliated Hospital of Soochow University, Suzhou, China

    Yuhua Ru, Jia Chen & Depei Wu

  2. EBMT Paris Office, Hôpital Saint Antoine APHP, Sorbonne University, Paris, France

    Allain-Thibeault Ferhat, Mohamad Mohty & Norbert-Claude Gorin

  3. Universitaetsklinikum Wuerzburg, Wuerzburg, Germany

    Hermann Einsele

  4. Klinikum Grosshadern, Munich, Germany

    Johanna Tischer

  5. Medizinische Klinik M. S. Hamatologie, Onkologie und Tumorimmunologie, Berlin, Germany

    Igor-Wolfgang Blau

  6. IRCCS Ospedale Policlinico San Martino, Genova, Italy

    Emanuele Angelucci

  7. Universitaet Tuebingen, Tuebingen, Germany

    Wolfgang Bethge

  8. Azienda Ospedaliero Universitaria di Udine, Udine, Italy

    Renato Fanin

  9. Department of Internal Medicine I, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany

    Robert Zeiser

  10. Università Vita-Salute San Raffaele, Milan, Italy

    Fabio Ciceri

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  1. Yuhua Ru
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Contributions

YR, JC, DW, and NCG designed the study. ATF performed the statistical analysis. HE, JT, IWB, EA, WB, RF, RZ, MM, and FC contributed data for the study. YR and JC wrote the manuscript. NCG revised the manuscript. All authors contributed to improving the initial manuscript and approved its final version.

Corresponding authors

Correspondence to Depei Wu or Norbert-Claude Gorin.

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The authors declare no competing interests.

Ethics approval and consent to participate

EBMT centers are committed to obtain informed consent according to the local regulations applicable at the time of transplantation in order to report data to the EBMT. The Global Committee and the ALWP of the EBMT approved this study, in accordance with the guidelines of the Declaration of Helsinki.

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Ru, Y., Chen, J., Ferhat, AT. et al. Haploidentical versus matched unrelated donor hematopoietic cell transplantation for adult patients with refractory/relapsed acute lymphoblastic leukemia: a study from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Bone Marrow Transplant (2025). https://doi.org/10.1038/s41409-025-02708-w

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