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Total body irradiation-containing conditioning regimens without antithymocyte globulin in adults with aplastic anemia undergoing umbilical cord blood transplantation

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Abstract

Thus far, there have been no large cohort studies on total body irradiation (TBI)-containing conditioning regimens without antithymocyte globulin (ATG) in adults with aplastic anemia (AA) undergoing umbilical cord blood (UCB) transplantation (UCBT). We retrospectively analyzed 115 adults with idiopathic AA undergoing UCBT using TBI-containing reduced-intensity conditioning (RIC) regimens without ATG between 2000 and 2018 on behalf of the Adult Aplastic Anemia Working Group of the Japanese Society for Hematopoietic Cell Transplantation. We then compared transplantation outcomes between a fludarabine (Flu)- and melphalan (Mel)-based regimen (FM) and a Flu- and cyclophosphamide (Cy)-based regimen (FC). The median patient age at UCBT was 41 years. The median total nucleated cell and total CD34+ cell doses in a UCB unit at cryopreservation were 2.5 × 107/kg and 0.7 × 105/kg, respectively. The median follow-up period for survivors was 47 months. The cumulative incidence rate of neutrophil engraftment was 76.5%, and the 4-year overall survival (OS) rate was 64.3%. In multivariate analysis, the covariates that were significantly associated with a higher neutrophil engraftment were total CD34+ cell dose in an UCB unit (≥ 0.7 × 105/kg; hazard ratio, 0.57, P = 0.01) and total dose of TBI (4 Gy of TBI; hazard ratio, 0.32, P = 0.01). There was no significant difference in the cumulative incidence of neutrophil engraftment and the 4-year OS between the FM and FC groups. In conclusion, TBI-containing RIC regimens without ATG are suitable for adults with AA undergoing UCBT. There were no significant differences in transplantation outcomes between the FM and FC groups.

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Acknowledgements

We thank all physicians and data managers at the centers who contributed to gathering transplantation data from the TRUMP. We also appreciate the contribution of the members of the Data Management Committees of the Japan Society for Hematopoietic Cell Transplantation.

On behalf of the Adult Aplastic Anemia Working Group of the Japanese Society for Hematopoietic Cell Transplantation

Funding

This study was supported in part by the Practical Research Project for Allergic Disease and Immunology (Research Technology of Medical Transplantation), Japan Agency for Medical Research and Development (AMED).

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Authors and Affiliations

  1. Department of Hematology, Kobe City Medical Center General Hospital, 2-2-1, Minatojima-Minamimachi, Chuo-ku, Kobe, 650-0047, Japan

    Nobuhiro Hiramoto & Satoshi Yoshioka

  2. Division of Transfusion Medicine, Kanazawa University Hospital, Kanazawa, Japan

    Hirohito Yamazaki

  3. Third Department of Internal Medicine, Yamaguchi University School of Medicine, Ube, Japan

    Yukinori Nakamura

  4. Department of Hematology, Federation of National Public Service Personnel Mutual Aid Associations Toranomon Hospital, Tokyo, Japan

    Naoyuki Uchida

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

    Makoto Murata

  6. Department of Hematology and Oncology, Kyoto University Hospital, Kyoto, Japan

    Tadakazu Kondo

  7. Department of Hematology, Hamanomachi Hospital, Fukuoka, Japan

    Tetsuya Eto

  8. Department of Hematology/Oncology, Wakayama Medical University, Wakayama, Japan

    Akinori Nishikawa

  9. Japanese Red Cross Kinki Block Blood Center, Osaka, Japan

    Takafumi Kimura

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

    Tatsuo Ichinohe

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

    Yoshiko Atsuta

  12. Department of Hematology and Rheumatology, Tohoku University Hospital, Miyagi, Japan

    Yasushi Onishi

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

    Ritsuro Suzuki

  14. Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan

    Takehiko Mori

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  1. Nobuhiro Hiramoto
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  2. Hirohito Yamazaki
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  10. Takafumi Kimura
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  15. Takehiko Mori
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on behalf of the Adult Aplastic Anemia Working Group of the Japanese Society for Hematopoietic Cell Transplantation

Contributions

NH, HY, RS, YO, and TM designed the study. NH performed statistical analyses and wrote the manuscript. NU, MM, TK, SY, TE, AN, and TK provided patient data. TI and YA collected patient data. All authors interpreted the data, and reviewed and approved the final manuscript.

Corresponding author

Correspondence to Nobuhiro Hiramoto.

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All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and national research committee and with the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.

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Informed consent was obtained from all participants included in the study.

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The authors declare no competing interests.

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Hiramoto, N., Yamazaki, H., Nakamura, Y. et al. Total body irradiation-containing conditioning regimens without antithymocyte globulin in adults with aplastic anemia undergoing umbilical cord blood transplantation. Ann Hematol 101, 165–175 (2022). https://doi.org/10.1007/s00277-021-04664-z

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