非血缘脐血或单倍体来源的造血干细胞移植治疗儿童原发性免疫缺陷病的疗效观察  

Observation of efficacy of allogeneic hematopoietic cell transplantation using unrelated cord blood or haploidentical donors in children with primary immunodeficiency diseases

作　　者：唐湘凤 卢伟 郗晓芹 井远方 员涵子 Tang Xiangfeng;Lu Wei;Xi Xiaoqin;Jing Yuanfang;Yuan Hanzi(Department of Pediatrics,Chinese People′s Liberation Army General Hospital-Sixth Medical Center,Beijing 100048,China)

机构地区：[1]中国人民解放军总医院第六医学中心儿科,北京100048

出　　处：《中华实用儿科临床杂志》2022年第1期32-36,共5页Chinese Journal of Applied Clinical Pediatrics

基　　金：首都临床特色应用研究(Z18110000718032)。

摘　　要：目的评估非血缘脐血或单倍体供者来源的异基因造血干细胞移植(allo-HSCT)治疗儿童原发性免疫缺陷病(PID)的临床疗效。方法回顾性分析2014年4月至2019年10月中国人民解放军总医院第六医学中心收治的60例PID患儿的临床资料,包括56例慢性肉芽肿病,2例严重联合免疫缺陷性疾病,1例高IgM血症和1例严重先天性中性粒细胞减少症,均接受allo-HSCT,其中12例非血缘脐血(非血缘脐血组),48例单倍体供者(单倍体组)。男59例,女1例,中位年龄3.4岁。预处理选用白消安为基础的清髓方案。预防急性移植物抗宿主病(aGVHD)以环孢素为基础方案。非血缘脐血组单个核细胞和CD_(34)^(+)细胞的中位数分别为0.67×10^(8)/kg和0.51×10^(6)/kg;单倍体组于01 d和02 d分别回输骨髓和外周干细胞,回输骨髓前4 h输注第三方脐血,回输的单倍体骨髓和外周干细胞的单个核细胞和CD_(34)^(+)细胞的中位数分别为9.97×10^(8)/kg和5.12×10^(6)/kg。采用Kaplan-Meier方法进行总生存率分析。结果粒系和巨核系植入中位天数分别为+13.0 d和+23.5 d,+30.0 d为供者型完全嵌合,未发生原发性植入失败,1例发生继发性植入失败。Ⅰ-Ⅱ度和Ⅲ-Ⅳ度aGVHD发生率分别为43.3%和15.5%,慢性移植物抗宿主病的局限型和全身型发生率为6.7%和1.1%。中位随访时间818 d,死亡6例,其中5例死于感染,1例死于心功能衰竭,总移植相关死亡率为11.9%。53例无病存活,5年的无失败生存率和整体生存率分别为83.9%和88.1%。结论非血缘脐血和单倍体来源的allo-HSCT治疗PID的疗效明确。Objective To evaluate the efficacy of allogeneic hematopoietic cell transplantation(allo-HSCT)using unrelated cord blood or haploidentical donors in the treatment of children with primary immunodeficiency diseases(PID).Methods The clinical data of 60 children with PID admitted to Chinese People′s Liberation Army General Hospital-Sixth Medical Center from April 2014 to October 2019 were retrospectively analyzed,including 56 cases of chronic granulomatous disease,2 cases of severe combined immunodeficiency disease,1 case of high-IgM syndrome and 1 case of severe congenital neutropenia.All patients underwent allo-HSCT,including 12 cases receiving the transplantation from unrelated cord blood(UCB group)and 48 cases from haploidentical donors combined with a third party unrelated cord blood(haploid group).Among these patients,there were 59 males and 1 female,with a median age of 3.4 years.All patients received a myeloablative conditioning regimen based on Busulfan.The prophylaxis of acute graft versus host disease(aGVHD)was performed based on Cyclosporine.In the UCB group,the median dose of mononuclear cells and CD_(34)^(+)cells was 0.67×10^(8)/kg and 0.51×10^(6)/kg recipient body weight,respectively;In the haploid group,bone marrow and peripheral stem cells from haploid donors were infused on day 01 and day 02,respectively.The third party cord blood was infused 4 hours before bone marrow infusion.The median dose of mononuclear cells and CD_(34)^(+)cells of bone marrow and peripheral stem cells from haploid donors was 9.97×10^(8)/kg and 5.12×10^(6)/kg recipient body weight,respectively.Kaplan-Meier method was used to analyze the overall survival rate.Results The median day to neutrophil and platelet engraftment was 13.0 days and 23.5 days,respectively.The rate of complete donor chime-rism was shown 30.0 days after transplantation.There was no case with primary engraftment failure,and 1 case with secondary engraftment failure.The incidence of gradeⅠ-Ⅱand gradeⅢ-ⅣaGVHD was 43.3%and 15.5%,respectively.The i

关 键 词：异基因造血干细胞移植 单倍体 非血缘脐血 原发性免疫缺陷病 儿童 

分 类 号：R725.9[医药卫生—儿科]