异基因造血干细胞移植治疗儿童高IgM综合征的临床分析  

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

作　　者：王子奇 孟岩[1] 窦颖[1] 管贤敏[1] 张璐颖[1] 于洁[1] 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)

机构地区：[1]重庆医科大学附属儿童医院血液肿瘤科/国家儿童健康与疾病临床医学研究中心/儿童发育疾病研究教育部重点实验室/儿科学重庆市重点实验室,重庆400014

出　　处：《中国当代儿科杂志》2022年第6期635-642,共8页Chinese Journal of Contemporary Pediatrics

摘　　要：目的评估异基因造血干细胞移植(allogeneic hematopoietic stem cell transplantation,allo-HSCT)治疗儿童高IgM综合征(hyper-IgM syndrome,HIG M)的疗效。方法回顾性收集行allo-HSCT的17例HIGM患儿的临床资料并进行分析,采用Kaplan-Meier法对移植后HIGM患儿进行生存分析。结果移植后,16例患儿临床诊断败血症;14例患儿在移植后100d内病毒检测阳性,包括EB病毒11例、巨细胞病毒7例、JC病毒2例等;9例患儿存在侵袭性真菌病。急/慢性移植物抗宿主病分别有6例和3例。中位随访时间约为2年,3例患儿移植后早期死亡。患儿总生存(overall survival,OS)率、无事件生存(event-free survival,EFS)率和无病生存(diseasefree survival,DFS)率分别为82.35%、70.59%和76.47%。log-rank检验结果显示,全相合移植患儿的EFS率高于不全相合移植患儿(P=0.019),全相合无关供者移植患儿的OS率、EFS率和DFS率均显著优于不全相合无关供者移植患儿(P<0.05),移植后无真菌感染患儿的EFS率与DFS率优于合并真菌感染患儿(P<0.05)。结论alloHSCT能有效治疗HIGM。接受全相合移植并积极防治真菌及机会性感染有助于改善患儿预后。Objective To evaluate the clinical effect of allogeneic hematopoietic stem cell transplantation(alloHSCT)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 HLAmatched 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.

关 键 词：高IGM综合征 异基因造血干细胞移植 原发性免疫缺陷病 儿童 

分 类 号：R725.9[医药卫生—儿科]