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Prospective evaluation of alternative donor from unrelated donor and cord blood in adult acute leukemia and myelodysplastic syndrome

Bone Marrow Transplantation volume 55, pages 1399–1409 (2020)Cite this article

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Abstract

A prospectively registered observational study was conducted to assess the significance of allogeneic hematopoietic stem cell transplantation from highly HLA-matched unrelated donors (UD) and cord blood (CB) on outcomes in adult acute leukemia (AL) and myelodysplastic syndrome (MDS). Between 2007 and 2015, 231 transplant-eligible patients were registered for a phase 2 study of alternative donor transplantation. After registration, a sufficient time period was given to find appropriate UD. Patients received CB transplantation (CBT) if an appropriate UD was unavailable. In total, 119 patients received CBT (106 AL and 13 MDS) and 91 patients received UD transplantation (UDT) (86 AL and 5 MDS). The median age was 39 years in both groups. The primary objective was overall survival (OS); secondary objectives included cumulative incidences of non-relapse mortality (NRM) and relapse, and disease-free survival. Diagnosis, disease status at transplantation, refined disease risk index, and hematopoietic cell transplant-specific comorbidity index did not differ between UDT and CBT. In multivariate analyses, graft source was not a significant risk factor for all objectives. In adjusted analyses, UDT and CBT showed similar OS, NRM, and relapse in this prospective study. CB can be a comparable alternative stem cell source to UD by achieving a timely transplant.

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Fig. 1: Treatment allocation according to CONSORT guidelines.
Fig. 2: Overall survival (OS) depicted with Kaplan–Meier estimates of cord blood transplantation (CBT) and unrelated donor transplantation (UDT) recipients.
Fig. 3: Disease-free survival (DFS) depicted with Kaplan–Meier estimates of cord blood transplantation (CBT) and unrelated donor transplantation (UDT) recipients.
Fig. 4: Overall survival (OS) stratified by refined disease risk index (rDRI).
Fig. 5: Cumulative incidence curves of non-relapse mortality (NRM) and relapse after stem cell transplantation according to graft source.

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Acknowledgements

The authors thank all colleagues and patients who took part in this study. The authors are grateful to Ms Noriko Mizutani for her excellent help with data management. This work was supported by a Health Labor Science Research Grant (H24-Immunology-008-01, Study ID. 12945283) and the Nagoya Blood and Marrow Transplantation Group.

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Authors and Affiliations

  1. Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan

    Seitaro Terakura, Tetsuya Nishida & Makoto Murata

  2. Department of Hematology and Oncology, Anjo Kosei Hospital, Anjo, Japan

    Masashi Sawa, Tomonori Kato & Kotaro Miyao

  3. Department of Hematology, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan

    Yukiyasu Ozawa, Tatsunori Goto & Koichi Miyamura

  4. Department of Hematology and Oncology, JA Aichi Konan Kosei Hospital, Konan, Japan

    Akio Kohno & Kazutaka Ozeki

  5. Department of Hematology and Rheumatology, Tohoku University Hospital, Sendai, Japan

    Yasushi Onishi & Noriko Fukuhara

  6. Department of Hematology and Oncology, Okayama University Graduate School of Medicine, Okayama, Japan

    Nobuharu Fujii

  7. Department of Hematology, National Hospital Organization Sendai Medical Center, Sendai, Japan

    Hisayuki Yokoyama

  8. Department of Hematology and Oncology, Japanese Red Cross Nagoya Daini Hospital, Nagoya, Japan

    Masanobu Kasai

  9. Department of Hematology, Meitetsu Hospital, Nagoya, Japan

    Hiroatsu Iida

  10. Department of Hematology, National Hospital Organization Nagoya Medical Center, Nagoya, Japan

    Hiroatsu Iida

  11. Department of Hematology, Gifu University Graduate School of Medicine, Gifu, Japan

    Nobuhiro Kanemura

  12. Department of Hematology, Hokkaido University Hospital, Sapporo, Japan

    Tomoyuki Endo

  13. Department of Hematology and Oncology, Shimane Prefectural Central Hospital, Izumo, Japan

    Hiroatsu Ago

  14. Department of Hematology and Oncology, Tokai University Hospital, Isehara, Japan

    Makoto Onizuka

  15. Department of Hematology, Sapporo Medical University Hospital, Sapporo, Japan

    Satoshi Iyama

  16. Department of Hematology, Ehime Prefectural Central Hospital, Matsuyama, Japan

    Yuichiro Nawa

  17. Department of Hematology, Osaka City University Graduate School of Medicine, Osaka, Japan

    Mika Nakamae

  18. Department of Hematology, Hamamatsu University School of Medicine, Hamamatsu, Japan

    Yasuyuki Nagata

  19. Division of Hematology and Oncology, Toyohashi Municipal Hospital, Toyohashi, Japan

    Shingo Kurahashi

  20. Department of Hematology, Miyagi Cancer Center, Natori, Japan

    Yasuo Tomiya

  21. Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan

    Atsumi Yanagisawa, Yachiyo Kuwatsuka & Yoshiko Atsuta

  22. Department of Oncology and Hematology, Shimane University Hospital, Izumo, Japan

    Ritsuro Suzuki

  23. Department of Advanced Medicine, Nagoya University Hospital, Nagoya, Japan

    Yachiyo Kuwatsuka

Authors
  1. Seitaro Terakura
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  31. Makoto Murata
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on behalf of the Nagoya Blood and Marrow Transplantation Group

Contributions

ST and TN designed and conducted the study, interpreted data, and wrote the paper; MS, TK, Kotaro M, Yukiyasu O, TG, AK, KO, Yasushi O, Noriko F, Nobuharu F, HY, MK, HI, NK, TE, HA, MO, SI, YN, MN, YN, SK, and YT recruited patients and collected clinical data; AY, RS, YK, and YA collected and analyzed data; Koichi M and MM supervised the study. All authors read and approved the final paper.

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Correspondence to Seitaro Terakura.

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Terakura, S., Nishida, T., Sawa, M. et al. Prospective evaluation of alternative donor from unrelated donor and cord blood in adult acute leukemia and myelodysplastic syndrome. Bone Marrow Transplant 55, 1399–1409 (2020). https://doi.org/10.1038/s41409-020-0859-8

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