Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan
Hematopoietic cell transplantation (HCT) is established as a curative treatment for severe chronic granulomatous disease (CGD). However, outcomes of HCT for CGD in Japan had not been precisely reported. We evaluated the outcome of HCT for CGD in Japan by means of a nationwide survey. A total of 91 p...
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Frontiers Media S.A.
2020-07-01
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Series: | Frontiers in Immunology |
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Online Access: | https://www.frontiersin.org/article/10.3389/fimmu.2020.01617/full |
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Article |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Masakatsu Yanagimachi Masakatsu Yanagimachi Koji Kato Koji Kato Akihiro Iguchi Koji Sasaki Chikako Kiyotani Katsuyoshi Koh Takashi Koike Hideki Sano Tomonari Shigemura Hideki Muramatsu Keiko Okada Masami Inoue Ken Tabuchi Toyoki Nishimura Tomoyuki Mizukami Tomoyuki Mizukami Hiroyuki Nunoi Kohsuke Imai Masao Kobayashi Tomohiro Morio |
spellingShingle |
Masakatsu Yanagimachi Masakatsu Yanagimachi Koji Kato Koji Kato Akihiro Iguchi Koji Sasaki Chikako Kiyotani Katsuyoshi Koh Takashi Koike Hideki Sano Tomonari Shigemura Hideki Muramatsu Keiko Okada Masami Inoue Ken Tabuchi Toyoki Nishimura Tomoyuki Mizukami Tomoyuki Mizukami Hiroyuki Nunoi Kohsuke Imai Masao Kobayashi Tomohiro Morio Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan Frontiers in Immunology hematopoietic cell transplantation chronic granulomatous disease CYBB adult cord blood transplantation low-dose irradiation |
author_facet |
Masakatsu Yanagimachi Masakatsu Yanagimachi Koji Kato Koji Kato Akihiro Iguchi Koji Sasaki Chikako Kiyotani Katsuyoshi Koh Takashi Koike Hideki Sano Tomonari Shigemura Hideki Muramatsu Keiko Okada Masami Inoue Ken Tabuchi Toyoki Nishimura Tomoyuki Mizukami Tomoyuki Mizukami Hiroyuki Nunoi Kohsuke Imai Masao Kobayashi Tomohiro Morio |
author_sort |
Masakatsu Yanagimachi |
title |
Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan |
title_short |
Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan |
title_full |
Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan |
title_fullStr |
Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan |
title_full_unstemmed |
Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan |
title_sort |
hematopoietic cell transplantation for chronic granulomatous disease in japan |
publisher |
Frontiers Media S.A. |
series |
Frontiers in Immunology |
issn |
1664-3224 |
publishDate |
2020-07-01 |
description |
Hematopoietic cell transplantation (HCT) is established as a curative treatment for severe chronic granulomatous disease (CGD). However, outcomes of HCT for CGD in Japan had not been precisely reported. We evaluated the outcome of HCT for CGD in Japan by means of a nationwide survey. A total of 91 patients (86 males and 5 females) with CGD who received HCT between 1992 and 2013 was investigated. Their median age at HCT was 11 years (0–39). Sixty-four patients had X-linked CGD caused by CYBB gene mutations, 13 had autosomal recessive CGD (7 CYBA and 6 NCF2), and 14 were genetically undetermined. Seventy patients are still alive at a median follow-up of 38.9 (3.7–230) months. Three-year OS and EFS was 73.7 and 67.6%, respectively. Twenty-one patients died mainly from transplant-related mortality. The cumulative incidence of grade II to IV acute GVHD and extensive chronic GVHD was 27.2 and 17.9%, respectively. Risk factors for EFS after HCT for CGD were age >30 years (P < 0.01), non-CYBB gene mutations (P < 0.01) and CBT (P < 0.01). Regarding the reduced intensity conditioning (RIC) regimen, risk factors for EFS included anti-thymocyte globulin (P = 0.048) and not using low-dose irradiation therapy (P < 0.01), in addition to the preceding risk factors. We report outcomes of HCT for CGD in Japan. Future studies are needed to improve such outcomes, especially for patients harboring non-CYBB gene mutations and suffering from adult CGD. A RIC regimen including low-dose irradiation may be a good option to explore further. |
topic |
hematopoietic cell transplantation chronic granulomatous disease CYBB adult cord blood transplantation low-dose irradiation |
url |
https://www.frontiersin.org/article/10.3389/fimmu.2020.01617/full |
work_keys_str_mv |
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doaj-5ee250a523cb45debaca13a5242daa5e2020-11-25T03:27:57ZengFrontiers Media S.A.Frontiers in Immunology1664-32242020-07-011110.3389/fimmu.2020.01617560924Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in JapanMasakatsu Yanagimachi0Masakatsu Yanagimachi1Koji Kato2Koji Kato3Akihiro Iguchi4Koji Sasaki5Chikako Kiyotani6Katsuyoshi Koh7Takashi Koike8Hideki Sano9Tomonari Shigemura10Hideki Muramatsu11Keiko Okada12Masami Inoue13Ken Tabuchi14Toyoki Nishimura15Tomoyuki Mizukami16Tomoyuki Mizukami17Hiroyuki Nunoi18Kohsuke Imai19Masao Kobayashi20Tomohiro Morio21Department of Pediatrics, Tokyo Medical and Dental University, Tokyo, JapanDepartment of Hematology/Oncology, Kanagawa Children's Medical Center, Yokohama, JapanDepartment of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanCentral Japan Cord Blood Bank, Seto, JapanDepartment of Pediatrics, Hokkaido University Hospital, Sapporo, JapanDepartment of Pediatrics, Yokohama City University, Yokohama, JapanChildren's Cancer Center, National Center for Child Health and Development, Tokyo, JapanDepartment of Hematology/Oncology, Saitama Children's Medical Center, Saitama, JapanDepartment of Pediatrics, Tokai University School of Medicine, Isehara, Japan0Department of Pediatric Oncology, Fukushima Medical University Hospital, Fukushima, Japan1Department of Pediatrics, Shinshu University School of Medicine, Nagano, Japan2Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan3Department of Pediatric Hematology/Oncology, Osaka City General Hospital, Osaka, Japan4Department of Pediatric Hematology/Oncology, Osaka Women's and Children's Hospital, Osaka, Japan5Division of Pediatrics, Tokyo Metropolitan Cancer and Infectious Disease Center Komagome Hospital, Tokyo, Japan6Division of Pediatrics, Developmental and Urological-Reproductive Medicine Faculty of Medicine, University of Miyazaki, Miyazaki, Japan6Division of Pediatrics, Developmental and Urological-Reproductive Medicine Faculty of Medicine, University of Miyazaki, Miyazaki, Japan7Department of Pediatrics, NHO Kumamoto Medical Center, Kumamoto, Japan6Division of Pediatrics, Developmental and Urological-Reproductive Medicine Faculty of Medicine, University of Miyazaki, Miyazaki, JapanDepartment of Pediatrics, Tokyo Medical and Dental University, Tokyo, Japan8Department of Pediatrics, Hiroshima University Graduate School of Biomedical & Health Sciences, Hiroshima, JapanDepartment of Pediatrics, Tokyo Medical and Dental University, Tokyo, JapanHematopoietic cell transplantation (HCT) is established as a curative treatment for severe chronic granulomatous disease (CGD). However, outcomes of HCT for CGD in Japan had not been precisely reported. We evaluated the outcome of HCT for CGD in Japan by means of a nationwide survey. A total of 91 patients (86 males and 5 females) with CGD who received HCT between 1992 and 2013 was investigated. Their median age at HCT was 11 years (0–39). Sixty-four patients had X-linked CGD caused by CYBB gene mutations, 13 had autosomal recessive CGD (7 CYBA and 6 NCF2), and 14 were genetically undetermined. Seventy patients are still alive at a median follow-up of 38.9 (3.7–230) months. Three-year OS and EFS was 73.7 and 67.6%, respectively. Twenty-one patients died mainly from transplant-related mortality. The cumulative incidence of grade II to IV acute GVHD and extensive chronic GVHD was 27.2 and 17.9%, respectively. Risk factors for EFS after HCT for CGD were age >30 years (P < 0.01), non-CYBB gene mutations (P < 0.01) and CBT (P < 0.01). Regarding the reduced intensity conditioning (RIC) regimen, risk factors for EFS included anti-thymocyte globulin (P = 0.048) and not using low-dose irradiation therapy (P < 0.01), in addition to the preceding risk factors. We report outcomes of HCT for CGD in Japan. Future studies are needed to improve such outcomes, especially for patients harboring non-CYBB gene mutations and suffering from adult CGD. A RIC regimen including low-dose irradiation may be a good option to explore further.https://www.frontiersin.org/article/10.3389/fimmu.2020.01617/fullhematopoietic cell transplantationchronic granulomatous diseaseCYBBadultcord blood transplantationlow-dose irradiation |