Clinical effect of allogeneic hematopoietic stem cell transplantation in children with hyper-IgM syndrome.
10.7499/j.issn.1008-8830.2112098
- Author:
Zi-Qi WANG
1
;
Yan MENG
1
;
Ying DOU
1
;
Xian-Min GUAN
1
;
Lu-Ying ZHANG
1
;
Jie YU
1
Author Information
1. Department of Hematology and Oncology, Children's Hospital of Chongqing Medical University/National Clinical Research Center for Child Health and Disorders/Ministry of Education Key Laboratory of Child Development and Disorders/Chongqing Key Laboratory of Pediatrics, Chongqing 400014, China.
- Publication Type:Journal Article
- Keywords:
Allogeneic hematopoietic stem cell transplantation;
Child;
Hyper-IgM syndrome;
Primary immunodeficiency disease
- MeSH:
Child;
Epstein-Barr Virus Infections;
Graft vs Host Disease/prevention & control*;
Hematopoietic Stem Cell Transplantation/methods*;
Herpesvirus 4, Human;
Humans;
Hyper-IgM Immunodeficiency Syndrome;
Retrospective Studies
- From:
Chinese Journal of Contemporary Pediatrics
2022;24(6):635-642
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVES:To evaluate the clinical effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with hyper-IgM syndrome (HIGM).
METHODS:A retrospective analysis was performed on the medical data of 17 children with HIGM who received allo-HSCT. The Kaplan Meier method was used for the survival analysis of the children with HIGM after allo-HSCT.
RESULTS:After allo-HSCT, 16 children were diagnosed with sepsis; 14 tested positive for virus within 100 days after allo-HSCT, among whom 11 were positive for Epstein-Barr virus, 7 were positive for cytomegalovirus, and 2 were positive for JC virus; 9 children were found to have invasive fungal disease. There were 6 children with acute graft-versus-host disease and 3 children with chronic graft-versus-host disease. The median follow-up time was about 2 years, and 3 children died in the early stage after allo-HSCT. The children had an overall survival (OS) rate of 82.35%, an event-free survival (EFS) rate of 70.59%, and a disease-free survival (DFS) rate of 76.47%. The univariate analysis showed that the children receiving HLA-matched allo-HSCT had a significantly higher EFS rate than those receiving HLA-mismatched allo-HSCT (P=0.019) and that the children receiving HLA-matched unrelated allo-HSCT had significantly higher OS, EFS, and DFS rates than those receiving HLA-mismatched unrelated allo-HSCT (P<0.05). Compared with the children with fungal infection after allo-HSCT, the children without fungal infection had significantly higher EFS rate (P=0.02) and DFS rate (P=0.04).
CONCLUSIONS:Allo-HSCT is an effective treatment method for children with HIGM. HLA-matched allo-HSCT and active prevention and treatment of fungal infection and opportunistic infection may help to improve the prognosis of such children.
