Hematopoietic Cell Transplantation for Chronic Granulomatous Disease in Japan.
Adolescent
Adult
Child
Child, Preschool
Disease Susceptibility
Female
Genetic Predisposition to Disease
Graft vs Host Disease
/ etiology
Granulomatous Disease, Chronic
/ diagnosis
Hematopoietic Stem Cell Transplantation
/ adverse effects
Histocompatibility Testing
Humans
Infant
Infant, Newborn
Japan
Kaplan-Meier Estimate
Male
Mutation
NADPH Oxidase 2
/ genetics
Prognosis
Transplantation Conditioning
Young Adult
CYBB
adult
chronic granulomatous disease
cord blood transplantation
hematopoietic cell transplantation
low-dose irradiation
Journal
Frontiers in immunology
ISSN: 1664-3224
Titre abrégé: Front Immunol
Pays: Switzerland
ID NLM: 101560960
Informations de publication
Date de publication:
2020
2020
Historique:
received:
11
05
2020
accepted:
17
06
2020
entrez:
28
8
2020
pubmed:
28
8
2020
medline:
20
4
2021
Statut:
epublish
Résumé
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 (
Identifiants
pubmed: 32849547
doi: 10.3389/fimmu.2020.01617
pmc: PMC7403177
doi:
Substances chimiques
CYBB protein, human
EC 1.6.3.-
NADPH Oxidase 2
EC 1.6.3.-
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
1617Informations de copyright
Copyright © 2020 Yanagimachi, Kato, Iguchi, Sasaki, Kiyotani, Koh, Koike, Sano, Shigemura, Muramatsu, Okada, Inoue, Tabuchi, Nishimura, Mizukami, Nunoi, Imai, Kobayashi and Morio.
Références
N Engl J Med. 2010 Dec 30;363(27):2600-10
pubmed: 21190454
Biol Blood Marrow Transplant. 2005 Dec;11(12):945-56
pubmed: 16338616
Blood. 2002 Dec 15;100(13):4344-50
pubmed: 12393596
Bone Marrow Transplant. 2019 Aug;54(8):1227-1236
pubmed: 30531957
Blood. 2005 Oct 15;106(8):2912-9
pubmed: 15994282
Br J Haematol. 2011 Aug;154(3):363-72
pubmed: 21569009
Scand J Immunol. 2019 Feb;89(2):e12737
pubmed: 30506560
Biol Blood Marrow Transplant. 2009 Dec;15(12):1628-33
pubmed: 19896087
Transplantation. 2005 Jun 15;79(11):1596-606
pubmed: 15940051
J Allergy Clin Immunol. 2012 Jan;129(1):176-83
pubmed: 22078471
J Clin Immunol. 2013 Nov;33(8):1276-84
pubmed: 24078260
Clin Infect Dis. 2017 Mar 15;64(6):767-775
pubmed: 28362954
Biol Blood Marrow Transplant. 2012 Sep;18(9):1368-77
pubmed: 22326631
Hum Genet. 2000 May;106(5):473-81
pubmed: 10914676
N Engl J Med. 2001 Mar 22;344(12):881-8
pubmed: 11259721
Blood. 2016 Jul 21;128(3):440-8
pubmed: 27216217
Medicine (Baltimore). 2000 May;79(3):155-69
pubmed: 10844935
J Allergy Clin Immunol. 2013 Nov;132(5):1150-5
pubmed: 23870668
Bone Marrow Transplant. 1995 Jun;15(6):825-8
pubmed: 7581076
J Allergy Clin Immunol. 2018 Jan;141(1):365-371
pubmed: 28528201
Front Pediatr. 2019 Oct 30;7:445
pubmed: 31737589
J Clin Immunol. 2017 Aug;37(6):548-558
pubmed: 28752258
Curr Opin Hematol. 2011 Jan;18(1):36-41
pubmed: 21076296
Bone Marrow Transplant. 2013 Mar;48(3):452-8
pubmed: 23208313
Lancet. 2014 Feb 1;383(9915):436-48
pubmed: 24161820
Blood. 2018 Feb 22;131(8):917-931
pubmed: 29279357