Hematopoietic Cell Transplantation for Severe Combined Immunodeficiency Patients: a Japanese Retrospective Study.

Adolescent Child Child, Preschool Cord Blood Stem Cell Transplantation Female Graft vs Host Disease / etiology Hematopoietic Stem Cell Transplantation / adverse effects Humans Infant Japan Kaplan-Meier Estimate Male Retrospective Studies Severe Combined Immunodeficiency / mortality
Cord blood transplantation Hematopoietic cell transplantation Japan Newborn screening Retrospective study Severe combined immunodeficiency

Journal

Journal of clinical immunology
ISSN: 1573-2592
Titre abrégé: J Clin Immunol
Pays: Netherlands
ID NLM: 8102137

Informations de publication

Date de publication:
11 2021
Historique:
received: 24 11 2020
accepted: 25 07 2021
pubmed: 28 8 2021
medline: 19 2 2022
entrez: 27 8 2021
Statut: ppublish

Résumé

Hematopoietic cell transplantation (HCT) is a curative therapy for patients with severe combined immunodeficiency (SCID). Here, we conducted a nationwide study to assess the outcome of SCID patients after HCT in Japan. A cohort of 181 SCID patients undergoing their first allogeneic HCT in 1974-2016 was studied by using the Japanese national database (Transplant Registry Unified Management Program, TRUMP). The 10-year overall survival (OS) of the patients who received HCT in 2006-2016 was 67%. Umbilical cord blood (UCB) transplantation was performed in 81 patients (45%). The outcomes of HCT from HLA-matched UCB (n = 21) and matched sibling donors (n = 22) were comparable, including 10-year OS (91% vs. 91%), neutrophil recovery (cumulative incidence at 30 days, 89% vs. 100%), and platelet recovery (cumulative incidence at 60 days, 89% vs. 100%). Multivariate analysis of the patients who received HCT in 2006-2016 demonstrated that the following factors were associated with poor OS: bacterial or fungal infection at HCT (hazard ratio (HR): 3.8, P = 0.006), cytomegalovirus infection prior to HCT (HR: 9.4, P = 0.03), ≥ 4 months of age at HCT (HR: 25.5, P = 0.009), and mismatched UCB (HR: 19.8, P = 0.01). We showed the potential of HLA-matched UCB as a donor source with higher priority for SCID patients. We also demonstrated that early age at HCT without active infection is critical for a better prognosis, highlighting the importance of newborn screening for SCID.

Identifiants

DOI: 10.1007/s10875-021-01112-5 PMID: 34448087 PMC: PMC8390179
pubmed: 34448087
doi: 10.1007/s10875-021-01112-5
pii: 10.1007/s10875-021-01112-5
pmc: PMC8390179
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1865-1877

Informations de copyright

© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

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Auteurs

Satoshi Miyamoto (S)

Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan.
Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.

Katsutsugu Umeda (K)

Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Department of Pediatrics, Graduate School of Medicine, Kyoto University, 54 Kawaharacho, Shogoin, Sakyo-ku, Kyoto, Japan.

Mio Kurata (M)

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

Akira Nishimura (A)

Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan.
Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.

Masakatsu Yanagimachi (M)

Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Division of Hematology/Oncology, Kanagawa Children's Medical Center, 2-138-4 Mutsukawa, Minami-ku, Yokohama, Kanagawa, Japan.

Masataka Ishimura (M)

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

Maho Sato (M)

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

Tomonari Shigemura (T)

Department of Pediatrics, Shinshu University School of Medicine, 3-1-1 Asahi, Matsumoto, Nagano, Japan.

Motohiro Kato (M)

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

Yoji Sasahara (Y)

Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Department of Pediatrics, Tohoku University Graduate School of Medicine, 1-1 Seiryo-machi, Aoba-ku, Sendai, Miyagi, Japan.

Akihiro Iguchi (A)

Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Department of Pediatrics, Hokkaido University Hospital, Kita14, Nishi5, Kita-Ku, Sapporo, Hokkaido, Japan.

Takashi Koike (T)

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

Yoshiyuki Takahashi (Y)

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

Michiko Kajiwara (M)

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

Masami Inoue (M)

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

Yoshiko Hashii (Y)

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

Hiromasa Yabe (H)

Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Department of Innovative Medical Science, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, Japan.

Koji Kato (K)

Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Central Japan Cord Blood Bank, 539-3 Minami-Yamaguchi-cho, Aichi Red Cross Blood Center 4F, Seto, Aichi, Japan.

Yoshiko Atsuta (Y)

Japanese Data Center for Hematopoietic Cell Transplantation, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.
Department of Registry Science for Transplant and Cellular Therapy, Aichi Medical University School of Medicine, 1-1 Yazakokarimata, Nagakute, Aichi, Japan.

Kohsuke Imai (K)

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.
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.
ORCID: 0000-0003-2132-8403

Tomohiro Morio (T)

Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, Japan.
Hereditary Disorder Working Group of the Japanese Society for Transplantation and Cellular Therapy, 1-1-20 Daiko-Minami, Higashi-ku, Nagoya, Aichi, Japan.

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Enfant d'âge préscolaire › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches de sang du cordon › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Maladies du système immunitaire › Maladie du greffon contre l'hôte › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Personnes › Tranches d'âge › Nourrisson › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Régions géographiques › Iles › Japon › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Statistiques comme sujet › Analyse de survie › Estimation de Kaplan-Meier › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études rétrospectives › Hematopoietic Cell Transplantation for Severe...
questionsmedicales.fr › Maladies du système immunitaire › Déficits immunitaires › Maladies d'immunodéficience primaire › Immunodéficience combinée grave › Hematopoietic Cell Transplantation for Severe...