急性淋巴细胞白血病；MLL基因重排；儿童；疗效；预后因素," /> 急性淋巴细胞白血病；MLL基因重排；儿童；疗效；预后因素,"/>  Acute lymphoblastic leukemia,MLL?rearrangement,Children,Curative effect,Prognostic factor,"/> 单中心CCLG-ALL 2018方案治疗MLL基因重排阳性儿童急性淋巴细胞白血病的临床研究

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中国癌症防治杂志

中国癌症防治杂志 ›› 2023, Vol. 15 ›› Issue (6): 637-643.doi: 10.3969/j.issn.1674-5671.2023.06.07

• 临床研究 • 上一篇    下一篇

单中心CCLG-ALL 2018方案治疗MLL基因重排阳性儿童急性淋巴细胞白血病的临床研究

  

  1. 郑州大学附属儿童医院，河南省儿童医院，郑州儿童医院血液肿瘤科；郑州大学附属儿童医院，河南省小儿血液医学重点实验室；首都医科大学附属北京儿童医院血液肿瘤中心
  • 出版日期:2023-12-25 发布日期:2023-12-26
  • 通讯作者: 刘炜 E-mail： liuweixinxiang123@163.com
  • 基金资助:
    河南省科技攻关项目（222102310616）

Clinical study of single-center CCLG-ALL 2018 regimen in the treatment of MLL-rearrangement childhood acute lymphoblastic leukemia

  • Online:2023-12-25 Published:2023-12-26

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摘要： 目的 探讨单中心中国儿童白血病协作组（CCLG⁃ALL 2018）方案治疗MLL基因重排阳性儿童急性淋巴细胞白血病（MLL⁃rearrangement acute lymphoblastic leukemia，MLL⁃r ALL）患儿的疗效及预后相关因素。方法 收集2018年4月至2022年7月于郑州大学附属儿童医院接受CCLG⁃ALL 2018方案诊治的19例初诊MLL⁃r ALL患儿进行回顾性分析。结果 19例MLL⁃r ALL患儿中男12（63.2%）例、女7（36.8%）例，年龄≤1岁的为15（78.9%）例、年龄>1岁的为4（21.1%）例，诊断时的中位年龄为10.2个月（0.5~156.0个月）。18（94.7%）例为BCP⁃ALL，1（5.3%）例为T⁃ALL，融合基因类型以MLL⁃AF4多见（7/19）。中位随访时间为19.0个月（范围：0.1～62.0个月），1例在确诊后5 d死亡，诱导化疗第33天完全缓解率为61.1%。19例MLL⁃r ALL患儿死亡9 例，死亡率为47.4%，其中死于治疗相关并发症3例，1例为肺出血、2例为重症感染；1例死于白血病持续未缓解；死于复发5例，复发率为26.3%（5/19），均为极早期骨髓复发，中位复发时间为6个月（范围：5~9个月）。Kaplan‑Meier分析19例患儿2年总生存率、无事件生存率分别为57.9%和52.6%。单因素分析结果显示，诱导化疗第15天骨髓流式微小残留病（minimal residual disease，MRD）<1×10-3组与≥1×10-3组、第33天MLL重排基因转阴与未转阴组、MLL⁃AF4组与非MLL⁃AF4组患儿的2年OS率比较差异均有统计学意义（均P<0.05）。Cox模型多因素分析显示，重排基因MLL⁃AF4是影响MLL⁃r ALL患儿OS的独立预后不良因素（P=0.032）。结论 CCLG⁃ALL 2018方案可使MLL重排基因阳性的部分患儿达到缓解，但总体预后较差，容易复发且复发时死亡率较高。重排基因MLL⁃AF4是影响MLL⁃r ALL患儿应用CCLG⁃ALL 2018方案治疗效果的不良因素。

关键词: 急性淋巴细胞白血病；MLL基因重排；儿童；疗效；预后因素')">">急性淋巴细胞白血病；MLL基因重排；儿童；疗效；预后因素

Abstract: Objective To investigate the efficacy and prognostic factors of single⁃center CCLG⁃ALL 2018 regimen in the treatment of children with MLL⁃rearrangement acute lymphoblastic leukemia (MLL⁃r ALL). Methods A total of 19 newly diagnosed children with MLL⁃r ALL who received CCLG⁃ALL 2018 regimen in Children's Hospital Affiliated to Zhengzhou University from April 2018 to July 2022 were retrospectively analyzed. Results Among the 19 children with MLL⁃r ALL, 12 (63.2%) were males and 7 (36.8%) were females; 15 (78.9%) aged ≤1 year old, and 4 (21.1%) aged>1 year old. The median age at diagnosis was 10.2 months (range: 0.5-156.0 months). There were 18 (94.7%)  cases of BCP⁃ALL and 1(5.3%) case of T⁃ALL. MLL⁃AF4 was the most common fusion genotype (7/19). The median follow⁃up time was 19.0 months (range: 0.1-62.0 months), and 1 patient died 5 days after diagnosis, with a complete response rate of 61.1% on the 33rd day of induction chemotherapy. In total, 9 of 19 children died (47.4%), including 3 cases of treatment⁃related complications (1 case of pulmonary hemorrhage and 2 cases of severe infection). One case died of leukemia without remission, 5 cases died of recurrence with a recurrence rate of 26.3% (5/19), and all these cases were early bone marrow recurrence, with a median recurrence time of 6 months (range: 5-9 months). Kaplan⁃Meier showed that the 2⁃year overall survival (OS) and event⁃free survival (EFS) rates of 19 children were 57.9 % and 52.6%, respectively. The results of univariable analysis showed that there were significant differences in 2⁃year OS rate between the <1×10-3 group and ≥1×10-3 group on the 15th day of minimal residual disease (MRD), between the MLL rearrangement gene negative and non⁃negative groups on the 33rd day, and between the MLL⁃AF4 group and the non⁃MLL⁃AF4 group (all P<0.05). Multivariable analysis of Cox model showed that MLL⁃AF4 was an independent adverse prognostic factor affecting the OS in children with MLL⁃r ALL (P=0.032). Conclusions CCLG⁃ALL 2018 regimen could achieve remission in some of children with MLL⁃rearrangement, but the overall prognosis is poor, prone to recurrence and high mortality rate at recurrence. The MLL⁃AF4 rearrangement is the adverse factor affecting the treatment effect of CCLG⁃ALL 2018 regimen in children with MLL⁃r ALL.

Key words:  Acute lymphoblastic leukemia')"> Acute lymphoblastic leukemia, MLL?rearrangement, Children, Curative effect, Prognostic factor

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  • 引用本文

    李晨, 刘炜, 王亚峰, 王天有. 单中心CCLG-ALL 2018方案治疗MLL基因重排阳性儿童急性淋巴细胞白血病的临床研究[J]. 中国癌症防治杂志, 2023, 15(6): 637-643.

    LI Chen, LIU Wei, WANG Yafeng, WANG Tianyou. Clinical study of single-center CCLG-ALL 2018 regimen in the treatment of MLL-rearrangement childhood acute lymphoblastic leukemia[J]. Chinese Journal of Oncology Prevention and Treatment, 2023, 15(6): 637-643.