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Comparison of single-unit umbilical cord blood transplantation and haploidentical transplantation using posttransplant cyclophosphamide during first complete remission of acute myeloid leukemia

Bone Marrow Transplantation (2025)Cite this article

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Abstract

Choosing an optimal alternative donor is an important clinical concern in allogeneic hematopoietic cell transplantation (HCT). In Japan, single-unit umbilical cord blood transplantation (UCBT) has been widely used in the last two decades, whereas HCT from HLA-haploidentical related donors (haplo-HCT) has been increasingly used following the advent of posttransplant cyclophosphamide (PTCY) for graft-versus-host disease (GVHD) prophylaxis. This registry-based study aimed to compare outcomes between single-unit UCBT (n = 848) and PTCY-based haplo-HCT (n = 241) performed during first complete remission in patients with acute myeloid leukemia. UCBT was associated with a lower likelihood of engraftment (P < 0.001), a higher risk of grade 2–4 and grade 3–4 acute GVHD (P = 0.003 each), and a lower risk of extensive chronic GVHD (P = 0.048). The UCBT and haplo-HCT groups did not significantly differ in 3-year probabilities of overall survival (68% versus 69%, P = 0.686), GVHD/relapse-free survival (55% versus 54%, P = 0.866), relapse (14% versus 16%, P = 0.463), and non-relapse mortality (21% versus 19%, P = 0.403), respectively, which were confirmed with multivariate analysis. These results indicate that both procedures should be considered viable options for patients lacking a matched donor.

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Fig. 1: Engraftment according to the donor source.
Fig. 2: Outcomes according to the donor source.
Fig. 3: GVHD according to the donor source.

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Data availability

The data of this study are not publicly available due to ethical restrictions that it exceeds the scope of the recipient/donor’s consent for research use in the registry. However, data may be available from the corresponding author upon reasonable request and with permission of the JSTCT/JDCHCT.

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Acknowledgements

This work was supported in part by the Grants-in-Aid for Scientific Research from JSPS KAKENHI (grant number: JP24K11566) (MY), and the Practical Research Project for Allergic Diseases and Immunology (Research Technology of Medical Transplantation) from the Japan Agency for Medical Research and Development (grant number: 19ek0510023h0003) (YA).

Author information

Authors and Affiliations

  1. Nagoya City University East Medical Center, Nagoya, Japan

    Masamitsu Yanada

  2. St. Mary’s Hospital, Kurume, Japan

    Satoshi Yamasaki

  3. Aichi Medical University, Nagakute, Japan

    Shohei Mizuno & Yoshiko Atsuta

  4. Sapporo Hokuyu Hospital, Sapporo, Japan

    Junichi Sugita & Shuichi Ota

  5. Kyoto Prefectural University of Medicine, Kyoto, Japan

    Takahiro Fujino

  6. Jichi Medical University Saitama Medical Center, Saitama, Japan

    Yukiko Misaki & Hideki Nakasone

  7. Kanagawa Cancer Center, Yokohama, Japan

    Masatsugu Tanaka

  8. Toranomon Hospital, Tokyo, Japan

    Naoyuki Uchida

  9. Tokai University School of Medicine, Isehara, Japan

    Makoto Onizuka

  10. Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan

    Noriko Doki

  11. Anjo Kosei Hospital, Anjo, Japan

    Masashi Sawa

  12. National Hospital Organization Kumamoto Medical Center, Kumamoto, Japan

    Toshiro Kawakita

  13. Hokkaido University Hospital, Sapporo, Japan

    Yuta Hasegawa

  14. Osaka Metropolitan University Hospital, Osaka, Japan

    Hirohisa Nakamae

  15. Toranomon Hospital Kajigaya, Kawasaki, Japan

    Kazuya Ishiwata

  16. Kobe City Medical Center General Hospital, Kobe, Japan

    Nobuhiro Hiramoto

  17. Japanese Red Cross Kanto-Koshinetsu Block Blood Center, Tokyo, Japan

    Fumihiko Ishimaru

  18. Kyoto University Hospital, Kyoto, Japan

    Junya Kanda

  19. Japanese Data Center for Hematopoietic Cell Transplantation, Nagakute, Japan

    Marie Ohbiki & Yoshiko Atsuta

  20. Jichi Medical University, Shimotsuke, Japan

    Hideki Nakasone

  21. The Institute of Medical Science, The University of Tokyo, Tokyo, Japan

    Takaaki Konuma

Authors
  1. Masamitsu Yanada
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  2. Satoshi Yamasaki
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  3. Shohei Mizuno
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Contributions

MY designed the study, analyzed data, interpreted the results, and drafted the paper; SY, SM, JS, TF, YM, MT, NU, M Onizuka, ND, SO, MS, T Kawakita, YH, H Nakamae, KI, NH, H Nakasone, and T Konuma interpreted the results and edited the paper; and FI, JK, M Ohbiki, and YA contributed to data management, interpreted the results, and edited the paper. All authors approved the final version of the manuscript.

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Correspondence to Masamitsu Yanada.

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The authors declare no competing interests.

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This study was approved by the Nagoya City University Institutional Review Board. All methods were performed in accordance with the Declaration of Helsinki, and all patients provided informed consent for the utilization of their clinical data for research.

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Yanada, M., Yamasaki, S., Mizuno, S. et al. Comparison of single-unit umbilical cord blood transplantation and haploidentical transplantation using posttransplant cyclophosphamide during first complete remission of acute myeloid leukemia. Bone Marrow Transplant (2025). https://doi.org/10.1038/s41409-025-02729-5

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