非血缘脐血干细胞移植治疗高危/难治儿童急性髓系白血病160例临床分析  

Unrelated cord blood stem cell transplantation for high-risk/refractory childhood acute myeloid leukemia:a clinical analysis of 160 cases

作　　者：陈二玲 刘会兰 耿良权 汤宝林 朱小玉 姚雯 宋闿迪 皖湘 孙光宇 强萍 范倩 周紫薇 郑昌成 张磊[1] 张旭晗 童娟 孙自敏 Chen Erling;Liu Huilan;Geng Liangquan;Tang Baolin;Zhu Xiaoyu;Yao Wen;Song Kaidi;Wan Xiang;Sun Guangyu;Qiang Ping;Fan Qian;Zhou Ziwei;Zheng Changcheng;Zhang Lei;Zhang Xuhan;Tong Juan;Sun Zimin(Anhui Provincial Hospital Affiliated to University of Science and Technology of China Institute of Hematology,Hefei 230001,China)

机构地区：[1]中国科学技术大学附属第一医院(安徽省立医院)血液科,合肥230001

出　　处：《中华血液学杂志》2021年第7期549-554,共6页Chinese Journal of Hematology

基　　金：国家自然科学基金(81470350);中央高校基本科研业务费专项资金;“科大新医学”联合基金创新团队项目(WK9110000001)

摘　　要：目的探讨单份非血缘脐血干细胞移植(UCBT)治疗高危/难治儿童急性髓系白血病(AML)的疗效。方法对2008年6月至2018年12月期间接受UCBT的160例高危/难治AML(不含急性早幼粒细胞白血病)患儿进行回顾性分析。全部病例均采用清髓性预处理方案,应用环孢素A(CsA)联合短程霉酚酸酯(MMF)预防移植物抗宿主病(GVHD)。结果160例AML患儿中男103例、女57例,中位年龄7(1~14)岁,中位体重23(8~77)kg。移植后42 d中性粒细胞累积植入率为95.0%(95%CI 90.0%~97.5%),移植后120 d血小板累积植入率为85.5%(95%CI 83.3%~93.4%),中性粒细胞、血小板植入中位时间分别为移植后16(11~38)d、35(13~158)d。Ⅱ~Ⅳ、Ⅲ~Ⅳ度急性GVHD发生率分别为37.3%(95%CI 29.3%~45.2%)、27.3%(95%CI 20.0%~35.0%),慢性GVHD发生率为22.4%(95%CI 15.5%~28.7%)。可评估的移植后360 d移植相关死亡率(TRM)为13.1%(95%CI 8.4%~18.9%)。移植后5年累积复发率为13.8%(95%CI 8.5%~20.3%),无病生存(DFS)率为71.7%(95%CI 62.7%~77.8%)、总生存(OS)率为72.2%(95%CI 64.1%~78.7%),无GVHD无复发生存(GRFS)率为56.1%(95%CI 46.1%~64.9%)。移植前处于第1次完全缓解(CR1)期(95例)、第2次完全缓解(CR2)期(28例)、未缓解患者(37例)的移植后5年累积复发率分别为5.3%(95%CI 1.9%~11.1%)、19.9%(95%CI 6.9%~37.7%)、30.9%(95%CI 14.3%~49.2%)(P=0.001),移植后5年OS率分别为79.9%(95%CI 70.3%~86.7%)、71.1%(95%CI 50.4%~84.4%)、52.9%(95%CI 33.0%~69.3%)(χ^(2)=7.552,P=0.020)。结论UCBT是治愈高危/难治儿童AML安全、有效的治疗方法。第1次完全缓解期进行UCBT有利于改善预后。Objective To retrospectively analyze the clinical outcomes of single unrelated cord blood transplantation(UCBT)in children with high risk and refractory acute myeloid leukemia(AML).Methods Between June 2008 and December 2018,a total of 160 consecutive pediatric patients with AML received single UCBT(excluding acute promyelocytic leukemia).Myeloablative conditioning(MAC)regimen were applied.All patients received a combination of cyclosporine A(CsA)and mycophenolate mofetil(MMF)for the prophylaxis of graft-versus-host disease(GVHD).Results The cumulative incidence of neutrophil cells engraftment at day+42 and platelet recovery at day+120 was 95.0%(95%CI 90.0%-97.5%)at a median of 16 days after transplantation(range,11-38 days)and 85.5%(95%CI 83.3%-93.4%)with a median time to recovery of 35 days(range,13-158),respectively.Incidence of gradesⅡ-ⅣandⅢ-Ⅳacute GVHD and chronic GVHD were 37.3%(95% CI 29.3%-45.2%),27.3%(95% CI 20.0%-35.0%)and 22.4%(95%CI 15.5%-28.7%),respectively.The transplant-related mortality(TRM)at 360 day was 13.1%(95% CI 8.4%-18.9%).The 5-year cumulative incidence of relapse was 13.8%(95% CI 8.5%-20.3%).The 5-year disease-free survival(DFS)and overall survival(OS)were 71.7%(95% CI 62.7%-77.8%)and 72.2%(95% CI 64.1%-78.7%),respectively.The 5-year GVHD and relapse free survival(GRFS)was 56.1%(95% CI 46.1%-64.9%).The 5-year cumulative recurrence rates of CR1,CR2,and NR groups were 5.3%,19.9%,and 30.9%(P=0.001),and the 5-year OS rates were 79.9%(95% CI 70.3%-86.7%),71.1%(95% CI 50.4%-84.4%)and 52.9%(95% CI 33.0%-69.3%)(χ^(2)=7.552,P=0.020),respectively.Conclusions For pediatric patients with high risk and refractory AML,UCBT is a safe and effective treatment option,and it is favorable to improve the survival rate in CR1 stage.

关 键 词：脐血干细胞移植 儿童 急性髓系白血病 

分 类 号：R733.71[医药卫生—肿瘤]