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Comparable survival outcomes with haploidentical stem cell transplantation and unrelated bone marrow transplantation

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Abstract

We retrospectively compared outcomes of unrelated donor bone marrow transplant (UBMT) and HLA-haploidentical peripheral blood stem cell transplantation using post-transplant cyclophosphamide (PTCy-haploPBSCT) using the Japanese registry data. Recipients of first HCT for acute leukemia and myelodysplastic syndromes between 2012 and 2015 were included. The analyzed subjects comprised UBMT recipients with 8/8 matched HLA alleles (n = 1470), 7/8 matched alleles (n = 859), 6/8 matched alleles (n = 186), and recipients of PTCy-haploPBSCT (n = 133). In multivariate analyses with 8/8 matched UBMT as the reference, PTCy-haploPBSCT showed similar overall mortality, decreased risk of non-relapse mortality (NRM), increased risk of relapse, and decreased risk of grade II–IV acute graft-versus-host disease (GVHD) and chronic GVHD. Adjusted probabilities for 8/8 matched UBMT, PTCy-haploPBSCT, and 7/8 and 6/8 matched UBMT groups at 2 years post-transplant were 61%, 60%, 58%, and 52% for overall survival, 23%, 28%, 21%, and 19% for relapse, and 20%, 7%, 24%, and 33% for NRM. PTCy-haploPBSCT was associated with remarkably low NRM, contributing to survival outcomes that were comparable to 8/8 matched UBMT. The higher relapse rate in the PTCy-haploPBSCT group might be associated with the higher proportion of high-risk patients. PTCy-haploPBSCT may be a viable alternative when HLA-matched related donors are not available.

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Fig. 1: Adjusted probabilities of overall and relapse-free survival.
Fig. 2: Adjusted probabilities of relapse and non-relapse mortality.

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Data available on request due to privacy/ethical restrictions.

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Acknowledgements

The authors are grateful for the assistance and cooperation of all the staff members at the following collaborating institutes: the Japan Society for Hematopoietic Cell Transplantation, the Japan Marrow Donor Program, and the Japanese Data Center for Hematopoietic Cell Transplantation. This work was supported in part by the Practical Research Project for Allergic Diseases and Immunology (Research Technology of Medical Transplantation) of the Japan Agency for Medical Research and Development (AMED) under Grant Numbers 19ek0510023h0002 and 19ek0510023h0002.

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Author notes

  1. These authors contributed equally: Yoshiko Atsuta, Junichi Sugita.

Authors and Affiliations

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

    Yoshiko Atsuta & Mio Kurata

  2. Department of Registry Science for Transplant and Cellular Therapy, Aichi Medical University School of Medicine, Nagakute, Japan

    Yoshiko Atsuta

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

    Junichi Sugita, Souichi Shiratori & Takanori Teshima

  4. Hematology, Osaka City University Hospital, Osaka, Japan

    Hirohisa Nakamae

  5. Department of Hematology, University of Tsukuba Hospital, Tsukuba, Japan

    Yumiko Maruyama

  6. Department of Hematology, Kanazawa University Hospital, Kanazawa, Japan

    Ken Ishiyama

  7. Department of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan

    Takahiro Fukuda

  8. Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan

    Naoki Shingai

  9. Department of Hematology, Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital, Nagoya, Japan

    Yukiyasu Ozawa

  10. Department of Hematopoietic Cell Therapy, Niigata University Medical and Dental Hospital, Niigata, Japan

    Masayoshi Masuko

  11. Division of Hematology and Oncology, Department of Medicine, Kurume University Hospital, Kurume, Japan

    Koji Nagafuji

  12. Leukemia Research Center, Saiseikai Maebashi Hospital, Maebashi, Japan

    Satoru Takada

  13. Division of Hematology, Jichi Medical University Saitama Medical Center, Saitama, Japan

    Shinichi Kako

  14. Division of Hematology, Jichi Medical University, Shimotsuke, Japan

    Yoshinobu Kanda

  15. Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University, Kyoto, Japan

    Junya Kanda

  16. Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, Hiroshima, Japan

    Tatsuo Ichinohe

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  1. Yoshiko Atsuta
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Contributions

YA, JS, and TT designed the study and wrote the paper; MK and YA analyzed the results and created the figures; JK, TI, and TF reviewed and cleaned the clinical data and reviewed the results; HN, YM, KI, SS, TF, NS, YO, MM, KN, ST, SK, and YK submitted and cleaned the clinical data and reviewed the results.

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Correspondence to Takanori Teshima.

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Atsuta, Y., Sugita, J., Nakamae, H. et al. Comparable survival outcomes with haploidentical stem cell transplantation and unrelated bone marrow transplantation. Bone Marrow Transplant 57, 1781–1787 (2022). https://doi.org/10.1038/s41409-022-01822-3

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