Clinical effect of allogeneic hematopoietic stem cell transplantation in children with hyper-IgM syndrome

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

doi:10.7499/j.issn.1008-8830.2112098

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Chinese Journal of Contemporary Pediatrics ›› 2022, Vol. 24 ›› Issue (6) : 635-642. DOI: 10.7499/j.issn.1008-8830.2112098

CLINICAL RESEARCH

Clinical effect of allogeneic hematopoietic stem cell transplantation in children with hyper-IgM syndrome

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

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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.

Key words

Hyper-IgM syndrome / Allogeneic hematopoietic stem cell transplantation / Primary immunodeficiency disease / Child

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WANG Zi-Qi, MENG Yan, DOU Ying, GUAN Xian-Min, ZHANG Lu-Ying, YU Jie. Clinical effect of allogeneic hematopoietic stem cell transplantation in children with hyper-IgM syndrome[J]. Chinese Journal of Contemporary Pediatrics. 2022, 24(6): 635-642 https://doi.org/10.7499/j.issn.1008-8830.2112098

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