异基因造血干细胞移植治疗儿童高IgM综合征的临床分析

doi:10.7499/j.issn.1008-8830.2112098

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Abstract Objective 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.

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	WANG Zi-Qi
	MENG Yan
	DOU Ying
	GUAN Xian-Min
	ZHANG Lu-Ying
	YU Jie

Key words： Hyper-IgM syndrome Allogeneic hematopoietic stem cell transplantation Primary immunodeficiency disease Child

Received: 21 December 2021

Cite this article:

WANG Zi-Qi,MENG Yan,DOU Ying et al. Clinical effect of allogeneic hematopoietic stem cell transplantation in children with hyper-IgM syndrome[J]. CJCP, 2022, 24(6): 635-642.

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http://www.zgddek.com/EN/10.7499/j.issn.1008-8830.2112098 OR http://www.zgddek.com/EN/Y2022/V24/I6/635

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