Wang Zi-Qi, Meng Yan, Dou Ying, Guan Xian-Min, Zhang Lu-Ying, Yu Jie
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.
Zhongguo Dang Dai Er Ke Za Zhi. 2022 Jun 15;24(6):635-642. doi: 10.7499/j.issn.1008-8830.2112098.
To evaluate the clinical effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with hyper-IgM syndrome (HIGM).
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.
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 (=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 (<0.05). Compared with the children with fungal infection after allo-HSCT, the children without fungal infection had significantly higher EFS rate (=0.02) and DFS rate (=0.04).
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.
评估异基因造血干细胞移植（allo-HSCT）治疗儿童高IgM综合征（HIGM）的临床效果。
对17例接受allo-HSCT的HIGM患儿的医疗数据进行回顾性分析。采用Kaplan Meier法对allo-HSCT后的HIGM患儿进行生存分析。
allo-HSCT后，16例患儿被诊断为败血症；14例在allo-HSCT后100天内病毒检测呈阳性，其中11例EB病毒阳性，7例巨细胞病毒阳性，2例JC病毒阳性；9例患儿发生侵袭性真菌病。6例患儿发生急性移植物抗宿主病，3例患儿发生慢性移植物抗宿主病。中位随访时间约2年，3例患儿在allo-HSCT后早期死亡。患儿总体生存率（OS）为82.35%，无事件生存率（EFS）为70.59%，无病生存率（DFS）为76.47%。单因素分析显示，接受HLA匹配的allo-HSCT患儿的EFS率显著高于接受HLA不匹配的allo-HSCT患儿（P = 0.019），接受HLA匹配的非血缘allo-HSCT患儿的OS、EFS和DFS率显著高于接受HLA不匹配的非血缘allo-HSCT患儿（P < 0.05）。与allo-HSCT后发生真菌感染的患儿相比，未发生真菌感染的患儿EFS率（P = 0.02）和DFS率（P = 0.04）显著更高。
allo-HSCT是治疗儿童HIGM的有效方法。HLA匹配的allo-HSCT以及积极预防和治疗真菌感染和机会性感染可能有助于改善此类患儿的预后。
