Objective: To explore the predictive effect of European treatment and outcome study long term survival (ELTS) score on survival outcomes in chronic myeloid leukemia of chronic phase (CML-CP) children. Methods: A single-center retrospective cohort study was conducted. Clinical data of 216 children with CML-CP in Peking University People's Hospital from January 2010 to December 2023 were analyzed. Children were divided into low, intermediate and high-risk groups according to ELTS score. The survival outcomes and prognostic factors were analyzed. Kaplan-Meier method and Log-Rank test were used for survival analysis.Cox regression model was applied for analysis of prognostic factors. Results: Among the 216 children with CML-CP, there were 122 males and 94 females, with the diagnosis age of 11.0 (8.0, 14.7) years. The follow-up time was 77 (57, 99) months. According to ELTS score, 145, 52, and 19 children were classified as low, intermediate and high-risk group. For the low-risk and intermediate/high-risk groups, the 6-year failure-free survival (FFS) rates were (83.0±3.1)% and (64.6±5.7)%, the 6-year progression-free survival (PFS) rates were (91.4±2.3)% and (78.7±4.8)%, and the 6-year event-free survival (EFS) rates were (80.8±3.3)% and (64.2±5.7)%, with statistically significant difference (χ2=9.45, 7.16, 7.40, P=0.002, 0.007, 0.007), respectively.The 6-year overall survival (OS) rates were (98.5±1.0)% and (95.6±2.4)%, without statistically significant difference (χ2=0.35, P=0.550). Multivariate analysis showed that ELTS score was an independent prognostic factor or tendency for FFS (HR=1.97, 95%CI 1.11-3.49), PFS (HR=2.95, 95%CI 1.18-7.39), and no independent prognostic factor for EFS and OS were found. Conclusions: ELTS score at diagnosis can help stratify the risk of children with CML-CP. The children in intermediate/high-risk group are more likely to have treatment failure, disease progression than those in low-risk group, but the predictive ability of ELTS score for OS is limited.
目的： 探讨欧洲治疗与预后研究长期生存（ELTS）评分对慢性髓系白血病慢性期（CML-CP）患儿生存结局的预测作用。 方法： 回顾性队列研究。分析2010年1月至2023年12月北京大学人民医院收治的216例CML-CP患儿的临床资料。根据ELTS评分分为低危组、中危组、高危组。分析患儿生存结局情况及其影响因素。采用Kaplan-Meier法绘制生存曲线并用Log-Rank检验进行比较，Cox回归模型进行预后相关因素分析。 结果： 216例CML-CP患儿中男122例、女94例，诊断年龄11.0（8.0，14.7）岁。随访时间77（57，99）个月。按照ELTS评分，低危组145例、中危组52例、高危组19例。低危组、中高危组患儿的6年无失败生存率（FFS）分别为（83.0±3.1）%和（64.6±5.7）%、6年无进展生存率（PFS）分别为（91.4±2.3）%和（78.7±4.8）%、6年无事件生存率（EFS）分别为（80.8±3.3）%和（64.2±5.7）%，组间比较差异均有统计学意义（χ²=9.45、7.16、7.40，P=0.002、0.007、0.007），6年总生存率（OS）分别为（98.5±1.0）%和（95.6±2.4）%，组间比较差异无统计学意义（χ²=0.35，P=0.550）。多因素分析显示，ELTS评分是FFS（HR=1.97，95%CI 1.11~3.49）、PFS（HR=2.95，95%CI 1.18~7.39）的独立影响因素，未发现EFS、OS的独立影响因素。 结论： ELTS评分有助于儿童CML-CP的疾病预后风险分层，ELTS评分中高危组较低危组更易出现治疗失败、疾病进展，但ELTS评分对OS的预测作用较小。.