Guo B Y, Wang Y, Li J, Li C F, Feng X Q, Zheng M C, Liu S X, Yang L H, Jiang H, Xu H G, He X L, Wen H
Department of Pediatrics, the First Affiliated Hospital of Xiamen University, Xiamen 361003, China.
Department of Pediatrics, Fujian Medical University Union Hospital, Fuzhou 350001, China.
Zhonghua Er Ke Za Zhi. 2023 Oct 2;61(10):881-888. doi: 10.3760/cma.j.cn112140-20230224-00126.
To analyze the clinical features, efficacy and prognosis factors of core binding factor (CBF) acute myeloid leukemia (AML) children in South China. This was a retrospective cohort study. Clinical data of 584 AML patients from 9 hospitals between January 2015 to December 2020 was collected. According to fusion gene results, all patients were divided into two groups: CBF-AML group (189 cases) and non-CBF-AML group (395 cases). CBF-AML group were divided into AML1-ETO subgroup (154 cases) and CBFβ-MYH11 subgroup (35 cases). Patients in CBF-AML group chosen different induction scheme were divided into group A (fludarabine, cytarabine, granulocyte colony stimulating factor and idarubicin (FLAG-IDA) scheme, 134 cases) and group B (daunorubicin, cytarabine and etoposide (DAE) scheme, 55 cases). Age, gender, response rate, recurrence rate, mortality, molecular genetic characteristics and other clinical data were compared between groups. Kaplan-Meier method was used for survival analysis and survival curve was drawn. Cox regression model was used to analyze prognostic factors. A total of 584 AML children were diagnosed, including 346 males and 238 females. And a total of 189 children with CBF-AML were included, including 117 males and 72 females. The age of diagnosis was 7.3 (4.5,10.0)years, and the white blood cell count at initial diagnosis was 21.4 (9.7, 47.7)×10/L.The complete remission rate of the first course (CR1) of induction therapy, relapse rate, and mortality of children with CBF-AML were significantly different from those in the non-CBF-AML group (91.0% (172/189) 78.0% (308/395); 10.1% (19/189) 18.7% (74/395); 13.2% (25/189) 25.6% (101/395), all <0.05). In children with CBF-AML, the CBFβ-MYH11 subgroup had higher initial white blood cells and lower proportion of extramedullary invasion than the AML1-ETO subgroup, with statistical significance (65.7% (23/35) 14.9% (23/154), 2.9% (1/35) 16.9% (26/154), both <0.05). AML1-ETO subgroup had more additional chromosome abnormalities (75/154), especially sex chromosome loss (53/154). Compared with group B, group A had more additional chromosome abnormalities and a higher proportion of tumor reduction regimen, with statistical significance (50.0% (67/134) 29.1% (16/55), 34.3% (46/134) 18.2% (10/55), both 0.05). Significant differences were found in 5-years event free survival (EFS) rate and 5-year overall survival (OS) rate between CBF-AML group and non-CBF-AML group ((77.0±6.4)% (61.9±6.7)%,(83.7±9.0)% (67.3±7.2)%, both <0.05).EFS and OS rates of AML1-ETO subgroup and CBFβ-MYH11 subgroup in children with CBF-AML were not significantly different (both >0.05). Multivariate analysis showed in the AML1-ETO subgroup, CR1 rate and high white blood cell count (≥50×10/L) were independent risk factors for EFS (=0.24, 95% 0.07-0.85,=1.01, 95% 1.00-1.02, both <0.05) and OS (=0.24, 95% 0.06-0.87; =1.01, 95% 1.00-1.02; both <0.05). In CBF-AML, AML1-ETO is more common which has a higher extramedullary involvement and additional chromosome abnormalities, especially sex chromosome loss. The prognosis of AML1-ETO was similar to that of CBFβ-MYH11. The selection of induction regimen group FLAG-IDA for high white blood cell count and additional chromosome abnormality can improve the prognosis.
分析中国南方核心结合因子（CBF）急性髓系白血病（AML）患儿的临床特征、疗效及预后因素。这是一项回顾性队列研究。收集了2015年1月至2020年12月期间9家医院584例AML患者的临床资料。根据融合基因结果，将所有患者分为两组：CBF-AML组（189例）和非CBF-AML组（395例）。CBF-AML组又分为AML1-ETO亚组（154例）和CBFβ-MYH11亚组（35例）。将选择不同诱导方案的CBF-AML组患者分为A组（氟达拉滨、阿糖胞苷、粒细胞集落刺激因子和伊达比星（FLAG-IDA）方案，134例）和B组（柔红霉素、阿糖胞苷和依托泊苷（DAE）方案，55例）。比较两组患者的年龄、性别、缓解率、复发率、死亡率、分子遗传学特征及其他临床资料。采用Kaplan-Meier法进行生存分析并绘制生存曲线。采用Cox回归模型分析预后因素。共诊断出584例AML患儿，其中男性346例，女性238例。共纳入189例CBF-AML患儿，其中男性117例，女性72例。诊断年龄为7.3（4.5，10.0）岁，初诊时白细胞计数为21.4（9.7，47.7）×10⁹/L。CBF-AML患儿诱导治疗第1疗程完全缓解率（CR1）、复发率和死亡率与非CBF-AML组有显著差异（91.0%（172/189）对78.0%（308/395）；10.1%（19/189）对18.7%（74/395）；13.2%（25/189）对25.6%（101/395），均<0.05）。在CBF-AML患儿中，CBFβ-MYH11亚组初诊时白细胞较高，髓外浸润比例低于AML1-ETO亚组，差异有统计学意义（65.7%（23/35）对14.9%（23/154），2.9%（1/35）对16.9%（26/154），均<0.05）。AML1-ETO亚组有更多额外染色体异常（75/154），尤其是性染色体缺失（53/154）。与B组相比，A组有更多额外染色体异常，肿瘤减量方案比例更高，差异有统计学意义（50.0%（67/134）对29.1%（16/55），34.3%（46/134）对18.2%（10/55），均<0.05）。CBF-AML组与非CBF-AML组5年无事件生存率（EFS）和5年总生存率（OS）有显著差异（（77.0±6.4）%对（61.9±6.7）%，（83.7±9.0）%对（67.3±7.2）%，均<0.05）。CBF-AML患儿中AML1-ETO亚组和CBFβ-MYH11亚组的EFS和OS率无显著差异（均>0.05）。多因素分析显示，在AML1-ETO亚组中，CR1率和高白细胞计数（≥50×10⁹/L）是EFS（P=0.24，95%CI 0.07-0.85；P=1.01，95%CI 1.00-1.02，均<0.05）和OS（P=0.24，95%CI 0.06-0.87；P=1.01，95%CI 1.00-1.02；均<0.05）独立危险因素。在CBF-AML中，AML1-ETO更常见，髓外浸润和额外染色体异常更多，尤其是性染色体缺失。AML1-ETO的预后与CBFβ-MYH11相似。对于高白细胞计数和有额外染色体异常的患者选择FLAG-IDA诱导方案可改善预后。
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