Allogeneic hematopoietic stem cell transplantation (HSCT) can be used to treat children with refractory acute myeloid leukemia (AML). This retrospective analysis aimed to describe the outcomes and risk factors in such children. Data were collected through the nation-wide registry program in Japan. A total of 417 AML (median age: 13 years) patients 20 years or younger at HSCT, between January 2001 and December 2015, were included. A total of 314 patients died, and the median follow-up duration of the survivors was 1052 days. The most frequent cause of death was leukemia progression (58%). The 3-year overall survival (OS) rate was 23% (95% confidence interval [CI]: 19–28%). Chronic GVHD was associated with improved 3-year OS (47%, 95% CI, 36–57%, hazard ratio: 0.603, p = 0.001). Low performance status, presence of more than 25% of marrow blasts, presence of blasts in the blood at transplantation, FAB (other than M1 or M2), male donors, and number of transplantations ≥ 2 were adverse pre-HSCT variables. Patients with 0, 1–2, 3–4, 5, and 6–7 pre-HSCT variables had 3-year OS rates of 52%, 32%, 19%, 8, and 0%, respectively. Our findings may help experts decide if HSCT should be performed in such cases.

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We are grateful to all the clinicians, hospital, and health-center administrators who provided precise data via the registry of the Japan Society 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) from the Japan Agency for Medical Research and Development, AMED under Grant Number 18ek0510023h0002. We acknowledge the efforts of Dr. Chihaya Koriyama, Professor of Epidemiology and Preventive Medicine, Graduate School of Medical and Dental Sciences, Kagoshima University regarding statistical analysis.

Author contributions

YO designed the study, reviewed and analyzed data, and wrote the paper; HI, KKudo, KT, DT, and TT contributed to the critique of the manuscript; HG, HY, YN, KKoh, KI, NY, NU, KW, and YK identified patients for this study; and MI, KKato, and YA contributed to data collection and the critique of the manuscript.

Author information


  1. Department of Pediatrics, Kagoshima University Graduate School of Medical and Dental Sciences, 8-35-1 Sakuragaoka, Kagoshima, 890-8544, Japan

    • Yasuhiro Okamoto
  2. Department of Pediatrics, Fujita Health University Hospital, 1-98 Dengakugakubo Kutsukake-cho, Toyoake-city, Aichi, 470-1192, Japan

    • Kazuko Kudo
  3. Department of Pediatrics, Tokyo Metropolitan Cancer and Infectious Disease Center Komagome Hospital, 3-18-22 Honkomagome, Bunkyo-ku, Tokyo, 113-8677, Japan

    • Ken Tabuchi
  4. Children’s Cancer Center, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo, 157-8535, Japan

    • Daisuke Tomizawa
  5. Department of Pediatrics, Shiga University of Medical Science Hospital, Seta Tsukinowa-cho, Otsu, Shiga, 520-2192, Japan

    • Takashi Taga
  6. Division of Hematology/Oncology, Kanagawa Children’s Medical Center, 2-138-4 Mutsukawa, Minami-ku, Yokohama, Kanagawa, 232-8555, Japan

    • Hiroaki Goto
  7. Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, 4-1-1 Kitakaname, Hiratsuka, Kanagawa, 259-1292, Japan

    • Hiromasa Yabe
  8. Department of Pediatrics, Shinshu University School of Medicine, 3-1-1 Asahi, Matsumoto, Nagano, 390-8621, Japan

    • Yozo Nakazawa
  9. Department of Hematology/Oncology, Saitama Children’s Medical Center, 1-2 Shintoshin, Chuo-ku, Saitama, 330-8777, Japan

    • Katsuyoshi Koh
  10. Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan

    • Kazuhiro Ikegame
  11. Department of Hematology and Oncology, Children’s Medical Center, Japanese Red Cross Nagoya First Hospital, 3-35 Michishita-cho, Namamura-ku, Nagoya, Aichi, 453-0046, Japan

    • Nao Yoshida
    •  & Koji Kato
  12. Department of Hematology, Federation of National Public Service Personnel Mutual Aid Associations Toranomon Hospital, 2-2-2 Toranomon, Minato-ku, Tokyo, 105-8470, Japan

    • Naoyuki Uchida
  13. Department of Hematology and Oncology, Shizuoka Children’s Hospital, 860 Urushiyama, Aoi-ku, Shizuoka, 420-8660, Japan

    • Kenichiro Watanabe
  14. Department of Pediatrics, Kyushu University Hospital, 3-1-1, Maidashi, Higashi-ku, Fukuoka, 812-8582, Japan

    • Yuuki Koga
  15. Department of Hematology/Oncology, Osaka Women’s and Children’s Hospital, 840 Murodo-cho, Izumi, Osaka, 594-1101, Japan

    • Masami Inoue
  16. Department of Healthcare Administration, Nagoya University Graduate School of Medicine, 65 Tsurumai-cho, Showa-ku, Nagoya, 466-8550, Japan

    • Yoshiko Atsuta
  17. Japanese Data Center for Hematopoietic Cell Transplantation, 1-1-20 Daikominami, Higashi-ku, Nagoya, Aichi, 461-0047, Japan

    • Yoshiko Atsuta
  18. Department of Pediatrics, Kyoto City Hospital, 1-2 Mibuhigashitakadacho, Nakagyo Ward, Kyoto, 604-8845, Japan

    • Hiroyuki Ishida


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The authors declare that they have no conflict of interest.

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Correspondence to Yasuhiro Okamoto.

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