儿童急性淋巴细胞白血病睾丸复发的临床特征及预后因素分析  

Clinical features and prognostic analysis of testicular relapse in pediatric acute lymphoblastic leukemia

作　　者：王凝 高洋洋 戚本泉 阮敏 吕惠 张小燕 张然然 刘天峰 陈玉梅 邹尧 郭晔 杨文钰 张丽 竺晓凡 陈晓娟 Wang Ning;Gao Yangyang;Qi Benquan;Ruan Min;Lyu Hui;Zhang Xiaoyan;Zhang Ranran;Liu Tianfeng;Chen Yumei;Zou Yao;Guo Ye;Yang Wenyu;Zhang Li;Zhu Xiaofan;Chen Xiaojuan(Pediatric Blood Diseases Center,Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences,State Key Laboratory of Experimental Hematology,National Clinical Research Center for Blood Diseases,Haihe Laboratory of Cell Ecosystem,Tianjin Institutes of Health Science,Tianjin 300020,China)

机构地区：[1]中国医学科学院血液病医院(中国医学科学院血液学研究所)儿童血液病诊疗中心、实验血液学国家重点实验室、国家血液系统疾病临床医学研究中心、细胞生态海河实验室、天津医学健康研究院,天津300020

出　　处：《中华儿科杂志》2024年第3期262-267,共6页Chinese Journal of Pediatrics

基　　金：国家重点研发计划(2019YFA0110803);天津市卫生健康科技重点项目(TJWJ2022ZD001)。

摘　　要：目的探讨急性淋巴细胞白血病(ALL)睾丸复发患儿的临床特征及预后影响因素。方法回顾性分析2011年11月至2022年12月中国医学科学院血液病医院收治的37例ALL睾丸复发患儿的年龄、复发时间、复发部位、复发后治疗等临床资料,按照不同临床资料对患儿进行分组,采用Kaplan-Meier进行单因素分析评估各组患儿总生存率(OS)及无事件生存率(EFS),采用Cox比例风险回归模型进行多因素分析,评估OS及EFS的独立影响因素。结果37例ALL睾丸复发患儿初诊年龄为(5±3)岁,复发时间为(37±15)个月,随访时间为43(22,56)个月,其中23例(62%)为孤立睾丸复发。37例ALL睾丸复发患儿5年OS和5年EFS分别为(60±9)%和(50±9)%。单因素分析显示睾丸复发时间>28个月组2年EFS优于复发时间≤28个月组[(69±10)%比(11±11)%],孤立睾丸复发组2年EFS优于联合复发组[(66±11)%比(20±13)%],复发后含嵌合抗原受体T细胞(CAR-T)治疗组2年EFS优于不含CAR-T治疗的患儿[(78±10)%比(15±10)%],差异均有统计学意义(均P<0.05)。ETV6-RUNX1是最常见的分子生物学异常(38%,14/37),4年OS和4年EFS分别为(80±13)%、(64±15)%。多因素分析显示复发时间≤28个月(HR=3.09,95%CI 1.10~8.72)、联合复发(HR=4.26,95%CI 1.34~13.53)、复发后治疗含CAR-T(HR=0.15,95%CI 0.05~0.51)均是患儿2年EFS的独立影响因素(均P<0.05)。结论儿童ALL睾丸复发时间较晚,通常发生在初诊ALL 3年后,生存率低,ETV6-RUNX1是最常见的分子生物学异常,预后相对较好,早期复发、联合复发者预后差,CAR-T治疗可以显著提高儿童ALL睾丸复发者的生存率。Objective To investigate the clinical features and prognosis of testicular relapse in pediatric acute lymphoblastic leukemia(ALL).Methods Clinical data including the age,time from initial diagnosis to recurrence,relapse site,and therapeutic effect of 37 pediatric ALL with testicular relapse and treated in Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences between November 2011 and December 2022 were analyzed retrospectively.Patients were grouped according to different clinical data.Kaplan-Meier analysis was used to evaluate the overall survival(OS)rate and event free survival(EFS)rate for univariate analysis,and Cox proportional-hazards regression model was used to evaluate the influencing factors of OS rate and EFS rate for multivariate analysis.Results The age at initial diagnosis of 37 pediatric testicular relapse patients was(5±3)years and the time from initial diagnosis to testicular recurrence was(37±15)months.The follow-up time was 43(22,56)months.Twenty-three patients(62%)were isolated testis relapse.The 5-year OS rate and EFS rate of the 37 relapsed children were(60±9)%and(50±9)%respectively.Univariate analysis showed that the 2-year EFS rate in the group of patients with time from initial diagnosis to testicular recurrence>28 months was significantly higher than those≤28 months((69±10)%vs.(11±11)%,P<0.05),2-year EFS rate of the isolated testicular relapse group was significantly higher than combined relapse group((66±11)%vs.(20±13)%,P<0.05),2-year EFS rate of chimeric antigen receptor T(CAR-T)cell treatment after relapse group was significantly higher than without CAR-T cell treatment after relapse group((78±10)%vs.(15±10)%,P<0.05).ETV6-RUNX1 was the most common genetic aberration in testicular relapsed ALL(38%,14/37).The 4-year OS and EFS rate of patients with ETV6-RUNX1 positive were(80±13)%and(64±15)%,respectively.Multivariate analysis identified relapse occurred≤28 months after first diagnosis(HR=3.09,95%CI 1.10-8.72),combined relapse(HR=4.26,95%CI 1.

关 键 词：白血病 复发 睾丸 儿童 

分 类 号：R733.71[医药卫生—肿瘤]