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中国循证儿科杂志

中国循证儿科杂志 ›› 2020, Vol. 15 ›› Issue (2): 96-102.

• 论著 • 上一篇    下一篇

BCH-AML05方案治疗儿童急性髓细胞性白血病的疗效评估

吴颖1，崔蕾2，李静1，李伟京2，郑胡镛1，张瑞东1，刘怡1，高超1，吴敏媛1   

  1. 首都医科大学附属北京儿童医院北京，100045, 1 血液肿瘤中心,2 北京市儿科研究所血液疾病研究室
  • 收稿日期:2019-09-29 修回日期:2020-03-14 出版日期:2020-04-25 发布日期:2020-04-25
  • 通讯作者: 吴敏媛

Results of BCH-AML05 protocol in the treatment of childhood acute myeloid leukemia

WU Ying1, CUI Lei2, LI Jing1, LI Wei-jing2, ZHENG Hu-yong1, ZHANG Rui-dong1, LIU Yi1, GAO Chao1,  WU Min-yuan1   

  1. Beijing Children's Hospital, Capital Medical University, Beijing 100045, China; 1. Hematology Oncology Center; 2. Laboratory of Hematologic Diseases, Beijing Pediatric Research Institute
  • Received:2019-09-29 Revised:2020-03-14 Online:2020-04-25 Published:2020-04-25
  • Contact: WU Min-yuan

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摘要： 目的 总结BCH-AML05方案分层治疗儿童急性髓细胞性白血病（AML）的临床疗效，探讨如何进一步提高儿童AML无事件生存（EFS）率。方法 回顾性分析首都医科大学附属北京儿童医院2005年1月至2014年6月收治的初治AML患儿的临床特点及治疗疗效。采用Kaplan-Meier方法评估患儿的总体生存（OS）率及EFS率。结果 185例初治AML（除M3外）患儿纳入分析，均接受了BCH-AML05方案治疗，其中标危47例、中危90例、高危48例。中位随访时间24（0.5~129）个月。①第一疗程达完全缓解(CR)者106例(57.3%)，第二疗程达CR者45例（24.3%），总CR率为81.6%。②8年OS率和EFS率分别为(66.2±4.2)%和(54.1±5.5)%，标危组分别为(65.7±10.4)%和(57.2±8.5)%，中危组分别为(70.3±5.2)%和(60.2±7.4)%，高危组分别为(51.6±10.1)%和(31.3±14.5)%。③34例（18.4%）接受了造血干细胞移植，8年OS率及EFS率分别为(73.5±9.5)%和(67.7±9.9)%。④复发36例(19.5%)，中位复发时间12(3.5~ 53)月。死亡52例（28.1%），其中化疗相关死亡20例（10.8%）；诱导失败死亡11例（5.9%），复发相关死亡18例（9.7%）；早期死亡率为5.4%。⑤154例行Flt3基因突变检测，Flt3-ITD阳性16例（10.4%），其中6例（37.5%）诱导化疗后达CR，11例（68.8%）死亡。结论 BCH-AML05方案为儿童AML有效的化疗方案，精准的危险度分层更能提高AML患儿的长期生存率。高危患儿应尽早接受造血干细胞移植。

Abstract: Objective To summarize the therapeutic effects of BCH-AML05 protocol on childhood acute myeloid leukemia (AML) and explore how to improve the event-free survival (EFS) rate. Methods The clinical characteristics and therapeutic effects of the patients who were newly diagnosed as AML and treated with BCH-AML05 protocol at Beijing Children's Hospitol from January 2005 to June 2014 were retrespectively studied. Survival rates were evaluated by Kaplan-Meier method with SPSS 19. Results A total of 185 patients were included in the study and divided into groups of LR (low risk, 47 cases), MR (moderate risk, 90 cases) and HR (high risk, 48 cases). The median follow-up time was 24 (0.5-129) months. Among the 185 patients, 106 (57.3%) achieved complete remission (CR) after one course treatment and 45 achieved CR after the second course treatment. The total CR rate was 81.6%. The 8-year overall survival (OS) rate was (66.2±4.2)% and the EFS rate was (54.1±5.5)%. The 8-year OS and EFS rates for LR, MR and HR patients were (65.7±10.4)% and (57.2±8.5)%, (70.3±5.2)% and (60.2±7.4)%, and (51.6±10.1)% and (31.3±14.5)%, respectively. The 8-year OS and EFS rates of 34 patients receiving hematopoietic stem cell transplantation (HSCT) were (73.5±9.5)% and (67.7±9.9)%, respectively. Relapse occurred in 36 patients (19.5%) with a median time of 10 months( 3.5-53 months). Fifty-two （28.1%) patients died, including 20 cases (10.8%) of chemotherapy related death, 11 cases (5.9%) of induced failure related death and 18 cases (9.7%) of relapse related death. The early mortality rate was 5.4%. Among them, 154 patients were tested for Flt3 gene mutation including 16 (10.4%) positive cases with 6 (37.5%) of CR after induced chemotherapy and 11 (68.8%) of death. Conclusion BCH-AML05 protocol was effective for childern with AML. Accurate stratification of risk level could increase the long-term survival rate. Children with high-risk AML should receive HSCT as soon as possible.