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Outcome of peripheral blood stem cell transplantation from HLA-identical sibling donors for adult patients with aplastic anemia

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Abstract

Although bone marrow transplantation is the recommended form of allogeneic hematopoietic stem cell transplantation for aplastic anemia, some patients undergo peripheral blood stem cell transplantation (PBSCT). Therefore, there is critical demand to identify factors affecting transplantation outcomes. Using the Japanese registry database, we retrospectively analyzed outcomes of 94 adult patients with aplastic anemia who underwent PBSCT from HLA-identical sibling donors. The cumulative incidence of neutrophil engraftment was 94% (95% confidence interval [CI] 86–97%), and was significantly higher in patients who received anti-thymocyte globulin (ATG) in conditioning. The cumulative incidence rate was 26% (95% CI 17–35%) in grades II–IV acute graft-versus-host disease (GVHD) and 20% (95% CI 13–29%) in extensive chronic GVHD, and tended to be lower in patients with chronic GVHD who received ATG-based conditioning. The 5-year overall survival (OS) rate was 70% (95% CI 59–78%). In multivariate analysis, patient age < 40 years, shorter period from diagnosis to transplantation, better performance status, and ATG-based conditioning were significantly correlated with favorable OS. In conclusion, PBSCT from HLA-identical sibling donors for aplastic anemia would result in acceptable outcomes. Several risk factors identified in our study should be considered when selecting a stem cell source.

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Data availability statement

The data that support the findings of this study are available from the corresponding author upon reasonable request.

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Acknowledgements

The authors would like to thank all the physicians and data managers at each transplantation center and the staff members at the Japanese Data Center for Hematopoietic Cell Transplantation.

Funding

Takehiko Mori received research funding from Mochida Pharmaceutical; MSD K.K.; Novartis Pharma K.K.; Eisai Pharmaceuticals; Medical & Biological Laboratories; Otsuka Pharmaceutical; Kyowa Kirin Co. Ltd.; Shionogi & Co.; Chugai Pharmaceutical; Takeda Pharmaceutical; Asahi Kasei Corporation; Bristol Myers Squibb; AbbVie GK; CSL Behring; Teijin Pharma; and KM Biologics, outside the submitted work. The remaining authors did not receive support from any organization for the submitted work.

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Authors and Affiliations

  1. Third Department of Internal Medicine, Yamaguchi University Hospital, 1-1-1 Minamikogushi, Ube, Yamaguchi, 755-8505, Japan

    Yukinori Nakamura

  2. Department of Hematology, Tokyo Medical and Dental University, Tokyo, Japan

    Takehiko Mori

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

    Shinichi Kako & Yoshinobu Kanda

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

    Hirohito Yamazaki

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

    Naoyuki Uchida

  6. Department of Hematology, Kanagawa Cancer Center, Yokohama, Japan

    Masatsugu Tanaka

  7. Division of Hematology, Ehime Prefectural Central Hospital, Matsuyama, Japan

    Yuichiro Nawa

  8. Hematopoietic Stem Cell Transplantation Division, National Cancer Center Hospital, Tokyo, Japan

    Takahiro Fukuda

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

    Tatsuo Ichinohe

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

    Yoshiko Atsuta

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

    Yoshiko Atsuta

  12. Department of Hematology, Tohoku University Hospital, Sendai, Japan

    Yasushi Onishi

Authors
  1. Yukinori Nakamura
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  2. Takehiko Mori
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  3. Shinichi Kako
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  4. Hirohito Yamazaki
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  5. Yoshinobu Kanda
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  7. Masatsugu Tanaka
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  8. Yuichiro Nawa
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  9. Takahiro Fukuda
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  10. Tatsuo Ichinohe
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  11. Yoshiko Atsuta
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  12. Yasushi Onishi
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Contributions

YN, TM, SK, HY and YO: designed the study. YN: analyzed the data and wrote the manuscript. All the authors have discussed the results and approved the final manuscript.

Corresponding author

Correspondence to Yukinori Nakamura.

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Conflict of interest

Takehiko Mori has received personal fees from Pfizer Inc.; MSD K.K.; Janssen Pharma; Sumitomo Pharma; Novartis Pharma K.K.; Kyowa Kirin Co. Ltd.; Chugai Pharmaceutical; Asahi Kasei Corporation; Japan Blood Products Organization; Takeda Pharmaceutical; Ono Pharmaceutical; Eisai Pharmaceuticals; Astellas Pharma; AbbVie GK; Meiji Seika Pharma; CSL Behring; SymBio Pharmaceuticals; AstraZeneca; Daiichi Sankyo; Mochida Pharmaceutical; Sanofi, Otsuka Pharmaceutical; and Mitsubishi Tanabe Pharma, outside the submitted work. Hirohito Yamazaki has received personal fees from Novartis Pharma K.K. and Kyowa Kirin Co. Ltd., outside the submitted work. The remaining authors have no conflicts of interest to declare that are relevant to the content of this article.

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Nakamura, Y., Mori, T., Kako, S. et al. Outcome of peripheral blood stem cell transplantation from HLA-identical sibling donors for adult patients with aplastic anemia. Int J Hematol 117, 356–365 (2023). https://doi.org/10.1007/s12185-022-03487-6

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  • DOI: https://doi.org/10.1007/s12185-022-03487-6

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