Yang Jiapeng, Zhu Xiaohua, Zhang Honghong, Fu Yang, Li Zifeng, Xing Ziping, Yu Yi, Cao Ping, Le Jun, Jiang Junye, Li Jun, Wang Hongsheng, Zhai Xiaowen
Department of Hematology and Oncology, National Children's Medical Center, Children's Hospital of Fudan University, Shanghai 201102, China.
Children (Basel). 2024 May 17;11(5):605. doi: 10.3390/children11050605.
This retrospective study aimed to analyze the treatment effect and prognostic factors of pediatric acute myeloid leukemia (AML) patients with t(8;21). A total of 268 newly diagnosed pediatric AML (pAML) enrolled from 1 January 2005 to 31 December 2022 were retrospectively reviewed, and 50 (18.7%) patients harbored t(8;21) translocation. CR rate, OS, EFS, and RFS were assessed by multivariate Logistic and Cox regression models in these patients. Of the 50 patients, 2 patients abandoned treatment during the first induction course. Of the remaining 48 patients who received double-induction therapy and were included in the final analyses, CR1 and CR2 were 75.0% (36/48) and 95.8% (46/48), respectively. The overall three-year OS, EFS, and RFS were 68.4% (95% CI, 55.0-85.1), 64.2% (95% CI, 50.7-81.4), and 65.5% (95% CI, 51.9-82.8), respectively. The presence of loss of sex chromosome (LOS) at diagnosis (n = 21) was associated with a better 3-year OS [87.5% (95% CI, 72.7-100) vs. 52.7% (95% CI, 35.1-79.3), = 0.0089], 3-year EFS [81.6% (95% CI, 64.7-100) vs. 49.7% (95% CI, 32.4-76.4), = 0.023], and 3-year RFS [81.6% (95% CI, 64.7-100) vs. 51.7% (95% CI, 33.9-78.9), = 0.036] than those without LOS (n = 27), and it was also an independent good prognostic factor of OS (HR, 0.08 [95% CI, 0.01-0.48], = 0.005), EFS (HR, 0.22 [95% CI, 0.05-0.85], = 0.029), and RFS (HR, 0.21 [95% CI, 0.05-0.90], = 0.035). However, extramedullary leukemia (EML) featured the independent risk factors of inferior OS (HR, 10.99 [95% CI, 2.08-58.12], = 0.005), EFS (HR, 4.75 [95% CI, 1.10-20.61], = 0.037), and RFS (HR, 6.55 [95% CI, 1.40-30.63], = 0.017) in pediatric individuals with t(8;21) AML. Further analysis of combining LOS with EML indicated that the EML+LOS- subgroup had significantly inferior OS (92.9%, [95% CI, 80.3-100]), EFS (86.2%, [95% CI, 70.0-100]), and RFS (86.2%, [95% CI, 80.3-100]) compared to the other three subgroups (all < 0.001). LOS and EML are independent prognostic factors of OS, EFS, and RFS with t(8;21) pAML patients. LOS combined with EML may help improve risk stratification.
本回顾性研究旨在分析t(8;21)的小儿急性髓系白血病（AML）患者的治疗效果及预后因素。回顾性分析了2005年1月1日至2022年12月31日期间登记的268例新诊断的小儿AML（pAML）患者，其中50例（18.7%）患者存在t(8;21)易位。采用多因素Logistic和Cox回归模型评估这些患者的完全缓解（CR）率、总生存期（OS）、无事件生存期（EFS）和无复发生存期（RFS）。50例患者中，2例在首次诱导疗程中放弃治疗。其余48例接受双诱导治疗并纳入最终分析的患者中，CR1和CR2分别为75.0%（36/48）和95.8%（46/48）。总体三年OS、EFS和RFS分别为68.4%（95%CI，55.0 - 85.1）、64.2%（95%CI，50.7 - 81.4）和65.5%（95%CI，51.9 - 82.）。诊断时存在性染色体缺失（LOS）（n = 21）的患者与无LOS（n = 27）的患者相比，3年OS[87.5%（95%CI，72.7 - 100）vs.52.7%（95%CI，35.1 - 79.3），P = 0.0089]、3年EFS[81.6%（95%CI，64.7 - 100）vs.49.7%（95%CI，32.4 - 76.4），P = 0.0]和3年RFS[81.6%（95%CI，64.7 - 100）vs.51.7%（95%CI，33.9 - 78.9），P = 0.036]更好，并且它也是OS（HR，0.08[95%CI，0.01 - 0.48]，P = 0.005）、EFS（HR，0.22[95%CI，0.05 - 0.85]，P = 0.029）和RFS（HR，0.21[95%CI，0.05 - 0.90]，P = 0.035）的独立良好预后因素。然而，髓外白血病（EML）是t(8;21) AML小儿患者OS（HR，10.99[95%CI，2.08 - 58.12]，P = 0.005）、EFS（HR，4.75[95%CI，1.10 - 20.61]，P = 0.037）和RFS（HR，6.55[95%CI，1.40 - 30.63]，P = 0.017）较差的独立危险因素。将LOS与EML联合进一步分析表明，与其他三个亚组相比，EML + LOS - 亚组的OS（92.9%，[95%CI，80.3 - 100]）、EFS（86.2%，[95%CI，70.0 - 100]）和RFS（86.2%，[95%CI，80.3 - 100]）显著较差（均P < 0.001）。LOS和EML是t(8;21) pAML患者OS、EFS和RFS的独立预后因素。LOS与EML联合可能有助于改善风险分层。
