ELTS评分对慢性髓系白血病慢性期患儿生存结局的预测作用分析

Analysis of predictive effect of European treatment and outcome study long term survival score on survival outcomes in children with chronic myeloid leukemia of chronic phase

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(χ^(2)=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(χ^(2)=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。

ObjectiveTo 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.MethodsA 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.ResultsAmong 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(χ^(2)=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(χ^(2)=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.ConclusionsELTS 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.