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Hematopoietic Cell Transplantation for Inborn Errors of Immunity Other than Severe Combined Immunodeficiency in Japan: Retrospective Analysis for 1985–2016

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Journal of Clinical Immunology Aims and scope Submit manuscript

Abstract

Purpose

Hematopoietic cell transplantation (HCT) is a curative therapy for most patients with inborn errors of immunity (IEI). We conducted a nationwide study on HCT for patients with IEI other than severe combined immunodeficiency (non-SCID) in Japan.

Methods

Data from the Japanese national database (Transplant Registry Unified Management Program, TRUMP) for 566 patients with non-SCID IEI, who underwent their first HCT between 1985 and 2016, were retrospectively analyzed.

Results

The 10-year overall survival (OS) and event-free survival (EFS) were 74% and 64%, respectively. The 10-year OS for HCT from unrelated bone marrow (URBM), accounting for 39% of HCTs, was comparable to that for HCT from matched sibling donor (MSD), 79% and 81%, respectively. HCT from unrelated cord blood (URCB), accounting for 28% of HCTs, was also common, with a 10-year OS of 69% but less robust engraftment. The intensity of conditioning was not associated with OS or neutrophil recovery; however, myeloablative conditioning was more frequently associated with infection-related death. Patients who received myeloablative irradiation showed poor OS. Multivariate analyses revealed that HCT in 1985–1995 (hazard ratio [HR], 2.0; P = 0.03), URCB (HR, 2.0; P = 0.01), and related donor other than MSD (ORD) (HR, 2.9; P < 0.001) were associated with poor OS, and URCB (HR, 3.6; P < 0.001) and ORD (HR, 2.7; P = 0.02) showed a higher incidence of retransplantation.

Conclusions

We present the 1985–2016 status of HCT for non-SCID IEI in Japan with sufficient statistical power, highlighting the potential of URBM as an alternative donor and the feasibility of reduced intensity conditioning.

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Data Availability

The datasets presented in this article are not readily available because they belong to the JSTCT and the Japanese Data Center for Hematopoietic Cell Transplantation (JDCHCT). Requests to access the datasets should be directed to http://www.jdchct.or.jp/.

Code Availability

All statistical analyses were performed using the Stata software v16.1 and EZR 1.42.

Abbreviations

aGVHD:

Acute graft-versus-host disease

BM:

Bone marrow

CGD:

Chronic granulomatous disease

cGVHD:

Chronic graft-versus-host disease

CI:

Confidence interval

CID:

Combined immunodeficiency

EFS:

Event-free survival

FHL:

Familial hemophagocytic lymphohistiocytosis

GVHD:

Graft-versus-host disease

HCT:

Hematopoietic cell transplantation

HLH:

Hemophagocytic lymphohistiocytosis

HR:

Hazard ratio

IEI:

Inborn errors of immunity

IUIS:

International Union of Immunological Societies

JSTCT:

Japanese Society for Transplantation and Cellular Therapy

MAC:

Myeloablative conditioning

MSD:

Matched sibling donor

ORD:

Other related donor

OS:

Overall survival

PB:

Peripheral blood

PGF:

Primary graft failure

PIRD:

Primary immune regulatory disorder

RIC:

Reduced intensity conditioning

SCID:

Severe combined immunodeficiency

SCN:

Severe congenital neutropenia

SGF:

Secondary graft failure

TBI:

Total body irradiation

TRUMP:

Transplant Registry Unified Management Program

URBM:

Unrelated bone marrow

URCB:

Unrelated cord blood

URCBT:

Unrelated cord blood transplantation

WAS:

Wiskott–Aldrich syndrome

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Acknowledgements

We thank the Japan Marrow Donor Program, the cord blood banks in Japan, and the staff at the participating hospitals who attended to the patients and provided information for the TRUMP registry. We also thank Soichi Adachi, Shunichi Kato, Yasuo Horikoshi, Miharu Yabe, Nao Yoshida, Hiromitsu Takakura, Sae Ishimaru, Shinya Osone, Hidetoshi Takada, Nozomu Kawashima, Shinobu Tamura, Ayako Yamamori, Koji Kawaguchi, Akira Nishimura, Risa Matsumura, and Takako Miyamura, who supported this study as members of the Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy. We would also like to thank Kay Tanita for her support in preparing the figures.

Funding

This work was supported by the Japanese Ministry of Health, Labor, and Welfare [grant number 20FC1053], and Japan Agency for Medical Research and Development [grant numbers JP19lk0201100 and JP19gk0110041].

Author information

Authors and Affiliations

  1. Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan

    Satoshi Miyamoto & Tomohiro Morio

  2. Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko Minami, Higashi-ku, Nagoya, Aichi, Japan

    Satoshi Miyamoto, Katsutsugu Umeda, Masakatsu Yanagimachi, Akihiro Iguchi, Yoji Sasahara, Hiromasa Yabe, Koji Kato, Kohsuke Imai & Tomohiro Morio

  3. Department of Pediatrics, Graduate School of Medicine, Kyoto University, 54 Kawaharacho, Shogoin, Sakyo-ku, Kyoto, Japan

    Katsutsugu Umeda

  4. Japanese Data Center for Hematopoietic Cell Transplantation, 1-1-20 Daiko Minami, Higashi-ku, Nagoya, Aichi, Japan

    Mio Kurata & Yoshiko Atsuta

  5. Division of Hematology/Oncology, Kanagawa Children’s Medical Center, 2-138-4 Mutsukawa, Minami-ku, Yokohama, Kanagawa, Japan

    Masakatsu Yanagimachi

  6. Department of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo, Hokkaido, Japan

    Akihiro Iguchi

  7. Department of Pediatrics, Tohoku University Graduate School of Medicine, 1-1 Seiryo-machi, Aoba-ku, Sendai, Miyagi, Japan

    Yoji Sasahara

  8. Department of Pediatric Hematology/Oncology, Osaka City General Hospital, 2-13-22 Miyakojima-hondori, Miyakojima-ku, Osaka, Japan

    Keiko Okada

  9. Department of Pediatrics, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, Japan

    Takashi Koike

  10. Department of Pediatrics, Yokohama City University Hospital, 3-9 Fukuura, Kanazawa-ku, Yokohama, Kanagawa, Japan

    Reo Tanoshima

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

    Masataka Ishimura

  12. Department of Pediatrics, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, North 15 West 7, Kita-ku, Sapporo, Hokkaido, Japan

    Masafumi Yamada

  13. Department of Hematology/Oncology, Osaka Women’s and Children’s Hospital, 840 Murodocho, Izumi, Osaka, Japan

    Maho Sato & Masami Inoue

  14. Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumaicho, Showa-ku, Nagoya, Aichi, Japan

    Yoshiyuki Takahashi

  15. Center for Transfusion Medicine and Cell Therapy, Medical Hospital, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan

    Michiko Kajiwara

  16. Department of Pediatrics, Hiroshima University Graduate School of Biomedical & Health Sciences, Kasumi 1-2-3 Minami-ku, Hiroshima, Japan

    Hiroshi Kawaguchi

  17. Department of Cancer Immunotherapy, Osaka University Graduate School of Medicine, Yamadaoka, Suita, Osaka, 2-15, Japan

    Yoshiko Hashii

  18. Department of Innovative Medical Science, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, Japan

    Hiromasa Yabe

  19. Central Japan Cord Blood Bank, 539-3 Minami-Yamaguchi-cho, Aichi Red Cross Blood Center 4F, Seto, Aichi, Japan

    Koji Kato

  20. Department of Healthcare Administration, Nagoya University Graduate School of Medicine, 65 Tsurumaicho, Showa-ku, Nagoya, Aichi, Japan

    Yoshiko Atsuta

  21. Department of Community Pediatrics, Perinatal, and Maternal Medicine, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, 113-8519, Japan

    Kohsuke Imai

Authors
  1. Satoshi Miyamoto
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  2. Katsutsugu Umeda
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  3. Mio Kurata
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  4. Masakatsu Yanagimachi
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  5. Akihiro Iguchi
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  6. Yoji Sasahara
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  7. Keiko Okada
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  8. Takashi Koike
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  9. Reo Tanoshima
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  10. Masataka Ishimura
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  11. Masafumi Yamada
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  12. Maho Sato
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  13. Yoshiyuki Takahashi
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  14. Michiko Kajiwara
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  15. Hiroshi Kawaguchi
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  16. Masami Inoue
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  17. Yoshiko Hashii
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  18. Hiromasa Yabe
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  19. Koji Kato
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  20. Yoshiko Atsuta
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  21. Kohsuke Imai
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  22. Tomohiro Morio
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Contributions

SM designed the research, analyzed the data, and wrote the manuscript. KU, MYan, AI, YS, HY, and KK revised the manuscript. MKu and YA verified the analytical method and analyzed the data. KO, TK, RT, MI, MYam, MS, YT, MKa, and HK recruited the patients and collected the data. MI, YH, and KK contributed to transplantation data management as members of the Japanese Data Center for hematopoietic cell transplantation. KI and TM designed the research and revised the manuscript. All authors contributed to the article and approved the submitted version.

Corresponding author

Correspondence to Kohsuke Imai.

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Miyamoto, S., Umeda, K., Kurata, M. et al. Hematopoietic Cell Transplantation for Inborn Errors of Immunity Other than Severe Combined Immunodeficiency in Japan: Retrospective Analysis for 1985–2016. J Clin Immunol 42, 529–545 (2022). https://doi.org/10.1007/s10875-021-01199-w

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