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Nationwide retrospective review of hematopoietic stem cell transplantation in children with refractory Langerhans cell histiocytosis

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Abstract

The efficacy of and indications for hematopoietic stem cell transplantation (HSCT) in pediatric Langerhans cell histiocytosis (LCH) remain undetermined. This retrospective study analyzed 30 children with refractory LCH who underwent HSCT in Japan between 1996 and 2014. Eleven patients received a myeloablative conditioning (MAC) regimen, while 19 patients received a reduced-intensity conditioning (RIC) regimen. Among the 26 patients with complete data, 23 patients had risk organ (RO) involvement during clinical course. Disease status at HSCT was no active disease (NAD) (4), active disease-regression (AD-r) (2), active disease-stable (AD-s) (4), and active disease-progressive (AD-p) (16). Seventeen of the 30 patients (57%) were alive with a median follow-up of 433 days (range 9–5307) after HSCT. Death occurred within 3 months after HSCT in eight of 13 patients. RIC and MAC patients were similar in both overall survival (OS) (56.8% vs. 63.6%, respectively, p = 0.789) and failure-free survival (56.8% vs. 54.6%, respectively, p = 0.938). Regarding disease status at HSCT, the six patients with NAD/AD-r experienced better outcomes than the 20 with AD-s/AD-p (5-year OS, 100% vs. 54.5%, respectively, p = 0.040). Disease state at the time of HSCT was the most important prognostic factor.

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Acknowledgements

We thank all participating doctors and patients who were involved in the Japanese Hematopoietic Cell Transplantation Registry. 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 under grant number 18ek0510023h0002.

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Authors and Affiliations

  1. Department of Pediatrics, Fujita Health University School of Medicine, 1-98, Dengakugakubo, Kutsukake-cho, Toyoake, Aichi, 470-1192, Japan

    Kazuko Kudo

  2. Department of Pediatrics, Nippon Medical School, 1-1-5 Sendagi, Bunkyo-ku, Tokyo, 113-8603, Japan

    Miho Maeda

  3. Hokkaido Government, Kita 3-jo, Nishi 7-chome, Chuo-ku, Sapporo, 060-8588, Japan

    Nobuhiro Suzuki

  4. Department of Child Health and Development, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University, Yushima, Bunkyo-ku, Tokyo, 113-8519, Japan

    Hirokazu Kanegane

  5. Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1, Maidashi Higashi-ku, Fukuoka, 812-8582, Japan

    Shouichi Ohga & Yuuki Koga

  6. Department of Pediatrics, Ehime University Graduate School of Medicine, Shizukawa, Toon, 791-0295, Japan

    Eiichi Ishii

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

    Yoko Shioda

  8. Department of Pediatrics, Kyoto Prefectural University of Medicine, Graduate School of Medical Science, 465, Kajii-cho, Kawaramachi-Hirokoji, Kamigyo-ku, Kyoto, 602-8566, Japan

    Toshihiko Imamura

  9. Department of Laboratory Medicine, Uji-Tokushukai Medical Center, 145, Ishibashi, Makishima-cho, Uji, 611-0041, Japan

    Shinsaku Imashuku

  10. Department of Pediatrics, Juntendo University School of Medicine, 3-1-3, Hongo, Bunkyo-ku, Tokyo, 113-8431, Japan

    Yukiko Tsunematsu

  11. Department of Pediatrics, Iwate Medical University, 19-1, Uchimaru, Morioka, 020-8505, Japan

    Mikiya Endo

  12. Department of Pediatric Hematology/Oncology, Okayama University, Graduate School of Medicine, 2-5-1, Shikata-cho, Kita-ku, Okayama, 700-8558, Japan

    Akira Shimada

  13. Department of Pediatrics, Osaka University Graduate School of Medicine, 2-2, Yamada-Oka, Suita, Osaka, 565-0871, Japan

    Yoshiko Hashii

  14. Department of Pediatrics, National Kyushu Cancer Center, 3-1-1, Nodame, Minami-ku, Fukuoka, 811-1395, Japan

    Maiko Noguchi

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

    Masami Inoue

  16. Division of Pediatrics, Tokyo Cancer Registry, Tokyo Metropolitan Cancer and Infectious Disease Center Komagome Hospital, 3-18-22, Honkomagome, Bunkyo-ku, Tokyo, 113-8677, Japan

    Ken Tabuchi

  17. Department of Pediatrics, Jichi Medical University of Medicine, 3311-1, Yakushiji, Shimotsuke, Tochigi, 329-0498, Japan

    Akira Morimoto

Authors
  1. Kazuko Kudo
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  2. Miho Maeda
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  3. Nobuhiro Suzuki
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  4. Hirokazu Kanegane
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  5. Shouichi Ohga
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  6. Eiichi Ishii
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  7. Yoko Shioda
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  8. Toshihiko Imamura
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  9. Shinsaku Imashuku
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  10. Yukiko Tsunematsu
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  11. Mikiya Endo
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  12. Akira Shimada
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  13. Yuuki Koga
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  14. Yoshiko Hashii
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  15. Maiko Noguchi
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  16. Masami Inoue
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  17. Ken Tabuchi
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  18. Akira Morimoto
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Consortia

The Histiocytosis study group of the Japanese Society of Pediatric Hematology/Oncology

Contributions

KK and AM designed the research, analyzed the data, and wrote the manuscript. MM, NS, HK, SO, EI, YS, TI, SI, YT, ME, AS, YK, YH, MN, MI, and KT collected and managed the clinical data and discussed the results.

Corresponding author

Correspondence to Kazuko Kudo.

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We have no conflicts of interest with regard to this manuscript.

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Kudo, K., Maeda, M., Suzuki, N. et al. Nationwide retrospective review of hematopoietic stem cell transplantation in children with refractory Langerhans cell histiocytosis. Int J Hematol 111, 137–148 (2020). https://doi.org/10.1007/s12185-019-02760-5

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  • DOI: https://doi.org/10.1007/s12185-019-02760-5

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