Satoshi Miyamoto1,2, Katsutsugu Umeda2,3, Mio Kurata4, Masakatsu Yanagimachi2,5, Akihiro Iguchi2,6, Yoji Sasahara2,7, Keiko Okada8, Takashi Koike9, Reo Tanoshima10, Masataka Ishimura11, Masafumi Yamada12, Maho Sato13, Yoshiyuki Takahashi14, Michiko Kajiwara15, Hiroshi Kawaguchi16, Masami Inoue13, Yoshiko Hashii17, Hiromasa Yabe2,18, Koji Kato2,19, Yoshiko Atsuta4,20, Kohsuke Imai21,22, Tomohiro Morio1,2. 1. Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan. 2. Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko Minami, Higashi-ku, Nagoya, Aichi, Japan. 3. Department of Pediatrics, Graduate School of Medicine, Kyoto University, 54 Kawaharacho, Shogoin, Sakyo-ku, Kyoto, Japan. 4. Japanese Data Center for Hematopoietic Cell Transplantation, 1-1-20 Daiko Minami, Higashi-ku, Nagoya, Aichi, Japan. 5. Division of Hematology/Oncology, Kanagawa Children's Medical Center, 2-138-4 Mutsukawa, Minami-ku, Yokohama, Kanagawa, Japan. 6. Department of Pediatrics, Hokkaido University Hospital, North 14, West 5, Kita-Ku, Sapporo, Hokkaido, Japan. 7. Department of Pediatrics, Tohoku University Graduate School of Medicine, 1-1 Seiryo-machi, Aoba-ku, Sendai, Miyagi, Japan. 8. Department of Pediatric Hematology/Oncology, Osaka City General Hospital, 2-13-22 Miyakojima-hondori, Miyakojima-ku, Osaka, Japan. 9. Department of Pediatrics, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, Japan. 10. Department of Pediatrics, Yokohama City University Hospital, 3-9 Fukuura, Kanazawa-ku, Yokohama, Kanagawa, Japan. 11. Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-ku, Fukuoka, Japan. 12. Department of Pediatrics, Faculty of Medicine and Graduate School of Medicine, Hokkaido University, North 15 West 7, Kita-ku, Sapporo, Hokkaido, Japan. 13. Department of Hematology/Oncology, Osaka Women's and Children's Hospital, 840 Murodocho, Izumi, Osaka, Japan. 14. Department of Pediatrics, Nagoya University Graduate School of Medicine, 65 Tsurumaicho, Showa-ku, Nagoya, Aichi, Japan. 15. Center for Transfusion Medicine and Cell Therapy, Medical Hospital, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan. 16. Department of Pediatrics, Hiroshima University Graduate School of Biomedical & Health Sciences, Kasumi 1-2-3 Minami-ku, Hiroshima, Japan. 17. Department of Cancer Immunotherapy, Osaka University Graduate School of Medicine, Yamadaoka, Suita, Osaka, 2-15, Japan. 18. Department of Innovative Medical Science, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, Japan. 19. Central Japan Cord Blood Bank, 539-3 Minami-Yamaguchi-cho, Aichi Red Cross Blood Center 4F, Seto, Aichi, Japan. 20. Department of Healthcare Administration, Nagoya University Graduate School of Medicine, 65 Tsurumaicho, Showa-ku, Nagoya, Aichi, Japan. 21. Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko Minami, Higashi-ku, Nagoya, Aichi, Japan. kimai.ped@tmd.ac.jp. 22. Department of Community Pediatrics, Perinatal, and Maternal Medicine, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, 113-8519, Japan. kimai.ped@tmd.ac.jp.
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.
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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