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Long-term outcome in patients with Fanconi anemia who received hematopoietic stem cell transplantation: a retrospective nationwide analysis

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Abstract

We retrospectively analyzed nationwide records of 163 Fanconi anemia (FA) patients [aplastic anemia (AA), n = 118; myelodysplastic syndrome (MDS), n = 30; acute leukemia, n = 15] who underwent first allogeneic hematopoietic stem cell transplantation (HSCT) between 1987 and 2015 in Japan. An alternative donor was used in 119 (73%) patients, and 160 (98%) patients received a non-T-cell-depleted graft. With an 8.7-year median follow-up, 5-year overall survival (OS) was 81%. The 5-year OS was significantly higher in AA patients than in MDS and acute leukemia patients (89%, 71%, and 44%, respectively). In the MDS/leukemia group, factors associated with poor outcome in univariate analysis were older age at HSCT (≥ 18 years), conditioning regimen without anti-thymocyte or lymphocyte globulin, and grade II–IV acute graft-versus-host disease. After 1 year, of 137 survivors, 15 developed subsequent malignancies, of whom 12 were diagnosed with head and neck (HN)/esophageal cancer. An irradiation regimen and older age were associated with the risk of HN/esophageal cancer. Five of seven deaths were attributed to subsequent malignancies more than 5 years after HSCT. On the basis of the risk factors for HSCT in MDS/leukemia patients and subsequent malignancies, a more effective HSCT approach is required.

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Acknowledgements

We thank all the clinicians and hospital administrators who provided precise data via the registry of the Japan Society for Stem Cell Transplantation.

Author information

Authors and Affiliations

  1. Department of Innovative Medical Science, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, 259-1193, Japan

    Miharu Yabe & Hiromasa Yabe

  2. Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan

    Tomohiro Morio

  3. Department of Pediatrics and Data Center, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan

    Ken Tabuchi

  4. Children’s Cancer Center, National Center for Child Health and Development, Tokyo, Japan

    Daisuke Tomizawa

  5. Department of Hematology and Oncology, Hyogo Prefectural Kobe Children’s Hospital, Kobe, Japan

    Daiichiro Hasegawa

  6. Department of Pediatrics, Kyoto City Hospital, Kyoto, Japan

    Hiroyuki Ishida

  7. Department of Hematology and Oncology, Children’s Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan

    Nao Yoshida

  8. Department of Pediatrics, Tokai University School of Medicine, Isehara, Japan

    Takashi Koike

  9. Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan

    Yoshiyuki Takahashi

  10. Department of Hematology/Oncology, Saitama Children’s Medical Center, Saitama, Japan

    Katsuyoshi Koh

  11. Department of Pediatrics, Kagoshima University Graduate School of Medical and Dental Sciences, Kagoshima, Japan

    Yasuhiro Okamoto

  12. Department of Pediatric Oncology, Fukushima Medical University Hospital, Fukushima, Japan

    Hideki Sano

  13. Division of Pediatric Hematology and Oncology, Ibaraki Children’s Hospital, Mito, Japan

    Keisuke Kato

  14. Division of Cell Therapy/Hematology, Jichi Medical University Hospital, Shimotsuke, Japan

    Yoshinobu Kanda

  15. Division of Hematology/Oncology, Kanagawa Children’s Medical Center, Yokohama, Japan

    Hiroaki Goto

  16. Department of Pediatrics, Kyoto University Hospital, Kyoto, Japan

    Junko Takita

  17. Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan

    Takako Miyamura

  18. Department of Pediatrics, National Hospital Organization Kyushu Cancer Center, Fukuoka, Japan

    Maiko Noguchi

  19. Central Japan Cord Blood Bank, Seto, Japan

    Koji Kato

  20. Department of Cancer Immunotherapy, Osaka University Graduate School of Medicine, Osaka, Japan

    Yoshiko Hashii

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

    Yoshiko Astuta

Authors
  1. Miharu Yabe
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  2. Tomohiro Morio
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  3. Ken Tabuchi
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  4. Daisuke Tomizawa
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  6. Hiroyuki Ishida
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  7. Nao Yoshida
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  8. Takashi Koike
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  9. Yoshiyuki Takahashi
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  10. Katsuyoshi Koh
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  11. Yasuhiro Okamoto
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  12. Hideki Sano
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  13. Keisuke Kato
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  14. Yoshinobu Kanda
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  15. Hiroaki Goto
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  16. Junko Takita
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  17. Takako Miyamura
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  18. Maiko Noguchi
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  19. Koji Kato
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  20. Yoshiko Hashii
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  21. Yoshiko Astuta
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  22. Hiromasa Yabe
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Contributions

MY and HY designed the research, organized project, analyzed the data, and wrote the manuscript; KT and HY supported the statistical analysis; TM, TD, DH, HI, and NY interpreted the data; TT, YT, KK, YO, HS, KK, YK, JK, TM, and MN provided patients data; KK, YH, and YA had responsibility for maintenance of the database; all authors approved the final manuscript.

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Correspondence to Miharu Yabe.

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Yabe, M., Morio, T., Tabuchi, K. et al. Long-term outcome in patients with Fanconi anemia who received hematopoietic stem cell transplantation: a retrospective nationwide analysis. Int J Hematol 113, 134–144 (2021). https://doi.org/10.1007/s12185-020-02991-x

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  • DOI: https://doi.org/10.1007/s12185-020-02991-x

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