伴MYC基因重排的儿童急性淋巴细胞白血病的临床特征及预后分析

The clinical characteristics and prognosis of pediatric acute lymphoblastic leukemia with MYC rearrangement

目的 分析伴MYC重排的儿童急性淋巴细胞白血病(MYC-r ALL)患者的临床特征和预后。方法 以2013年9月—2022年8月在中国医学科学院血液病医院诊治的19例初诊MYC-r ALL患儿为研究对象,对其临床特征、治疗方案及预后进行分析。结果 19例MYC-rALL患儿,男∶女为13∶6,中位年龄6(1-13)岁,白细胞中位计数为9.28(2.20-806.04)×10~9/L,诱导治疗中15.8%患儿(3/19)发生肿瘤溶解综合征(TLS)。19例MYC-r ALL患儿完全缓解率(CR)为94.7%,3年无事件生存率(EFS)、总生存率(OS)分别为(72.0±10.7)%、(71.4±10.8)%。治疗方案非随机选择ALL方案(简称A方案)或BNHL方案(简称B方案),A方案治疗18例,CR率为94.4%,3年EFS、OS分别为(71.8±10.7)%、(71.4±10.8)%;B方案治疗1例,一疗程诱导治疗后完全缓解至末次随访。MYC-rB-ALL患儿9例,3年EFS、OS分别为(88.9±10.5)%、(87.5±11.7)%;T-ALL患儿10例,3年EFS、OS分别为(58.3±16.1)%、(57.1±16.4)%,P>0.05。结论 儿童MYC-r ALL预后较差,发生肿瘤溶解综合征风险高,免疫表型对预后无显著影响,但B细胞型患儿有更好的预后趋势,临床需扩大样本量,亟待开展多中心研究以寻找更优的治疗方案。

Objective To study the clinical characteristics and prognosis of pediatric acute lymphoblastic leukemia with MYC rearrangement(MYC-r ALL).Methods A total of 19 pediatric acute lymphoblastic leukemia with MYC rearrangement(MYC-r ALL)who were diagnosed and treated at Hematology Hospital,Chinese Academy of Medical Sciences from September 2013 to August 2022 were enrolled in the study.The clinical characteristics,treatment regimens,and prognosis were analyzed.Results Among the 19 cases of MYC-r ALL,there were 13 males and 6 females,with a median age of 6 years(range 1-13 years) and a median white blood cell count of 9.28×10~9/L(range 2.20-806.04×10~9/L),15.8% of patient(3/19) developed tumor lysis syndrome(TLS) during induction therapy.The complete response(CR) rate was 94.7%,the 3-year event-free survival(EFS) rate and the 3-year overall survival(OS) rate were(72.0±10.7)% and(71.40±10.8)%.ALL regimen(referred to as regimen A) or BNHL regimen(referred to as regimen B) were nonrandomly selected,18 cases were treated initially with regimen A,the CR rate was 94.4%;the 3-year EFS rate and the 3-year OS rate were(71.8±10.7)% and(71.4±10.8)%;1 case was treated initially with regimen B,achieved complete response after first induction course and maintained until the last follow-up.There were 9 patients with MYC-r B-ALL,the 3-year EFS rate and 3-year OS rate of MYC-r B-ALL children were(88.9±10.5)% and(87.5±11.7)%,respectively;While there were 10 children with MYC-r T-ALL,the 3-year EFS rate and 3-year OS rate of MYC-r T-ALL children were(58.3±16.1)% and(57.1±16.4)%,(P>0.05).Conclusions The prognosis of MYC-r ALL in children is poor,with a high risk of developing TLS,the immune phenotype has no significant impact on the prognosis,but B-cell immunophenotype children have a better prognosis trend.Clinical trials need to expand the sample size and conduct multi-center studies to find better treatment options.