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中国循证儿科杂志

中国循证儿科杂志 ›› 2022, Vol. 17 ›› Issue (2): 98-103.

• 论著 • 上一篇    下一篇

儿童急性T淋巴细胞白血病生物学特征及其预后的回顾性队列研究

胡海蕊1,袁媛1,李君2,薛天琳2,王营2,刘曙光2,高超2,郑胡镛1   

  1. 1 国家儿童医学中心，首都医科大学附属北京儿童医院，血液病中心，儿童血液病与肿瘤分子分型北京市重点实验室，儿科学国家重点学科，儿科重大疾病研究教育部重点实验室北京，100045；2 国家儿童医学中心，首都医科大学附属北京儿童医院，北京市儿科研究所，血液疾病研究室，儿童血液病与肿瘤分子分型北京市重点实验室，儿科学国家重点学科，儿科重大疾病研究教育部重点实验室北京,100045
  • 收稿日期:2021-03-31 修回日期:2022-04-07 出版日期:2022-04-25 发布日期:2022-04-25
  • 通讯作者: 高超，郑胡镛

Clinical biological characteristics and prognostic significance of pediatric T cell acute lymphoblastic leukemia: A retrospective cohort study

HU Hairui1, YUAN Yuan1, LI Jun2, XUE Tianlin2, WANG Ying2, LIU Shuguang2, GAO Chao2, ZHENG Huyong1    

  1. 1 Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics, Capital Medical University; Key Laboratory of Major Diseases in Children, Ministry of Education; Hematology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing 100045, China; 2 Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics, Capital Medical University; Key Laboratory of Major Diseases in Children, Ministry of Education; Laboratory of Hematologic Diseases, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing 100045, China
  • Received:2021-03-31 Revised:2022-04-07 Online:2022-04-25 Published:2022-04-25
  • Contact: GAO Chao, email: gaochaoconnie@live.cn; ZHENG Huyong, email: zhenghuyong@vip.sina.com

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摘要： 背景儿童急性T淋巴细胞白血病（T-ALL）在现有危险度分层治疗方案中，预后存在差异。目的探究儿童急性T-ALL的生物学特征及其与临床表现、早期治疗反应及长期预后的相关性。设计回顾性队列研究。方法回顾性分析2008年3月至2018年9月首都医科大学附属北京儿童医院收治、使用CCLG-ALL-2008方案治疗的初诊为T-ALL患儿白血病免疫标志和融合基因的特征，及其与患儿临床表现、早期治疗反应和长期预后的相关性。主要结局指标无事件生存（EFS）和总生存（OS）。结果101例T-ALL患儿进入本文分析，其中男76例（75.2%）。SIL-TAL1是最常见的融合基因（21.8%，22/101），SIL-TAL1阳性患儿纳入高危的比例显著高于阴性者。干细胞标志CD34的表达率为45.5%（46/101）。表达免疫标志CD2者与初诊外周血高WBC计数相关；表达CD34者与强的松反应不良及第15 d高微小残留病（MRD）相关；表达CD33者与诱导结束时形态学不缓解及高MRD相关；表达CD10者强的松治疗反应良好者多。单因素生存分析显示，SIL-TAL1阳性、强的松治疗反应不良、治疗第15 d骨髓形态学不缓解、第33 d高MRD及高危危险度与不良的EFS和OS相关（P均<0.05），其中第33 d MRD≥1%和SIL-TAL1融合基因阳性亦是EFS和OS的独立预后影响因素，危险比分别为第33 d MRD≥1%对EFS为1.96（95%CI：1.114~3.452，P=0.020），对OS为2.062（95%CI：1.138~3.734，P=0.017）；SIL-TAL1阳性对EFS为2.536（95%CI：1.053~6.104，P=0.038），对OS为2.921（95%CI：1.144~7.457，P=0.025）。结论T-ALL是一组异质性疾病，在以MRD为分层依据的治疗方案中，SIL-TAL1和治疗早期MRD对预测患儿长期预后有一定价值。

关键词: 急性T淋巴细胞白血病, 儿童, 预后, SIL-TAL1

Abstract: Background：Currently, there are differences in the outcomes of risk-stratified treatment in pediatric acute T lymphocytic leukemia (T-ALL). Objective：To explore the association between biological characteristics and clinical features, early treatment responses and longterm outcomes of pediatric T-ALL. Design：Retrospective cohort study. Methods：The immunophenotypic markers and fusion genes at diagnosis and its association with clinical features, early treatment response and long-term outcomes of T-ALL patients treated with CCLG-ALL-2008 protocol in Beijing Children's Hospital affiliated to Capital Medical University from March 2008 to September 2018 were retrospectively analyzed. Main outcome measures：Event free survival (EFS) and overall survival (OS). Results：A total of 101 children with T-ALL were enrolled, including 76 males (75.25%). SIL-TAL1 was the most common fusion gene (21.8%, 22/101) and the proportion of patients classified into high-risk of SIL-TAL1+ was significantly higher than that of SIL-TAL1-. The expression rate of stem cell marker CD34 was 45.5% (46/101). The expression of CD2 was associated with high peripheral white blood cell; the expression of CD34 was associated with poor prednisone response and high minimal residual disease (MRD) at day 15; the expression of CD33 was associated with morphological non-remission and high MRD at the end of induction; while the expression of CD10 was associated with good prednisone response. By K-M analysis, positive SIL-TAL1, poor response to prednisone treatment, non-remission of bone marrow morphology on day 15, high MRD at day 33 and classification of high-risk were associated with adverse EFS and OS (P＜0.05 respectively) . Furthermore, MRD≥1% at day 33 and SIL-TAL1 positive were also validated as independent prognostic factors for EFS and OS. The hazard ratios of MRD ≥1% at day 33 for EFS was 1.96 (95%CI: 1.114-3.452, P=0.020) and for OS was 2.062 (95%CI: 1.138-3.734, P=0.017); SIL-TAL1+ for EFS was 2.536 (95%CI: 1.053-6.104, P=0.038) and for OS was 2.921 (95%CI: 1.144-7.457, P=0.025). Conclusion：T-ALL is a group of heterogeneous diseases. SIL-TAL1 and early MRD can predict the long-term prognosis on the MRD based treatment protocol.

Key words: T-cell acute lymphoblastic leukemia, Children, Prognosis, SIL-TAL1

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