|
|
|
|
| LEADER |
01000caa a22002652 4500 |
| 001 |
NLM367989255 |
| 003 |
DE-627 |
| 005 |
20240411232308.0 |
| 007 |
cr uuu---uuuuu |
| 008 |
240204s2024 xx |||||o 00| ||eng c |
| 024 |
7 |
|
|a 10.1016/j.clim.2024.109919
|2 doi
|
| 028 |
5 |
2 |
|a pubmed24n1372.xml
|
| 035 |
|
|
|a (DE-627)NLM367989255
|
| 035 |
|
|
|a (NLM)38309448
|
| 035 |
|
|
|a (PII)S1521-6616(24)00030-5
|
| 040 |
|
|
|a DE-627
|b ger
|c DE-627
|e rakwb
|
| 041 |
|
|
|a eng
|
| 100 |
1 |
|
|a Si, Yingjian
|e verfasserin
|4 aut
|
| 245 |
1 |
0 |
|a Effect of allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease in children
|b A multicentre, retrospective cohort study in China
|
| 264 |
|
1 |
|c 2024
|
| 336 |
|
|
|a Text
|b txt
|2 rdacontent
|
| 337 |
|
|
|a ƒaComputermedien
|b c
|2 rdamedia
|
| 338 |
|
|
|a ƒa Online-Ressource
|b cr
|2 rdacarrier
|
| 500 |
|
|
|a Date Completed 19.02.2024
|
| 500 |
|
|
|a Date Revised 10.04.2024
|
| 500 |
|
|
|a published: Print-Electronic
|
| 500 |
|
|
|a Citation Status MEDLINE
|
| 520 |
|
|
|a Copyright © 2024. Published by Elsevier Inc.
|
| 520 |
|
|
|a Chronic granulomatous disease (CGD) in children is a rare primary immunodeficiency disorder that can lead to life-threatening infections and inflammatory complications. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is increasingly being used to treat severe CGD in children. We conducted a multicenter retrospective analysis of children with CGD who were treated with allo-HSCT at four pediatric hematopoietic stem cell transplant centers in China from September 2005 to December 2019. The study included a total of 171 patients (169 males and 2 females). The median age at the time of transplantation was 6.1 (0-16.4) years. Among them, 154 patients had X-linked recessive inheritance caused by CYBB gene mutations, 12 patients were autosomal recessive, 1 patient had DNAH11 and HYDIN gene mutations, and 4 patients had no gene mutations. The median follow-up period was 36.3 (1.9-79) months. All participating patients were applied to myeloablative conditioning (MAC) regimens. The rates of OS, EFS, and GEFS within three years were 87.5%, 85.3%, and 75.2%, respectively. The total graft failure and the total mortality rate were 5.3% and 11.1%. The cumulative incidence of acute GVHD was 53.8% and the incidence of chronic GVHD was 12.9%, The incidence of chronic GVHD was higher for patients who received unrelated donor cord blood stem cell transplantation (UD-CB) (P = 0.001). Chronic GVHD and coinfections are the risk factors for OS and EFS in patients with CGD after receiving allo-HSCT. UD-CB is a risk factor for EFS and the presence of pneumonia before transplantation is a risk factor for OS. In conclusion, through this study, we have demonstrated that allo-HSCT has excellent efficacy in the treatment of CGD in children, especially, RD-haplo is associated with a lower rate of graft failure incidence and mortality than the treatment modalities of other donor type. Therefore, allo-HSCT is strongly recommended when a well-matched donor is available. If a well-matched donor is not available, the HLA-mismatched donor should be carefully evaluated, and the conditioning regimen modified accordingly
|
| 650 |
|
4 |
|a Multicenter Study
|
| 650 |
|
4 |
|a Journal Article
|
| 650 |
|
4 |
|a Research Support, Non-U.S. Gov't
|
| 650 |
|
4 |
|a Allogeneic hematopoietic stem cell transplantation
|
| 650 |
|
4 |
|a Chronic granulomatous disease
|
| 650 |
|
4 |
|a GVHD
|
| 650 |
|
4 |
|a Graft failure
|
| 650 |
|
4 |
|a Infections
|
| 700 |
1 |
|
|a Dou, Ying
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Zhai, Xiaowen
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Zhou, Chen
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Lu, Wei
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Meng, Yan
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Qian, Xiaowen
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Chen, Jing
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Wang, Ping
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Luo, Changying
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Yu, Jie
|e verfasserin
|4 aut
|
| 700 |
1 |
|
|a Tang, Xiangfeng
|e verfasserin
|4 aut
|
| 773 |
0 |
8 |
|i Enthalten in
|t Clinical immunology (Orlando, Fla.)
|d 1999
|g 260(2024) vom: 02. März, Seite 109919
|w (DE-627)NLM098196855
|x 1521-7035
|7 nnns
|
| 773 |
1 |
8 |
|g volume:260
|g year:2024
|g day:02
|g month:03
|g pages:109919
|
| 856 |
4 |
0 |
|u http://dx.doi.org/10.1016/j.clim.2024.109919
|3 Volltext
|
| 912 |
|
|
|a GBV_USEFLAG_A
|
| 912 |
|
|
|a SYSFLAG_A
|
| 912 |
|
|
|a GBV_NLM
|
| 912 |
|
|
|a GBV_ILN_11
|
| 912 |
|
|
|a GBV_ILN_24
|
| 912 |
|
|
|a GBV_ILN_350
|
| 951 |
|
|
|a AR
|
| 952 |
|
|
|d 260
|j 2024
|b 02
|c 03
|h 109919
|