[Interim results of a multicenter cohort study for Chinese Children Leukemia Group-acute lymphoblastic leukemia 2018 regimen].

Objective: To summarize the therapeutic effects of Chinese Children Leukemia Group-acute lymphoblastic leukemia (CCLG-ALL) 2018 regimen in children with T cell acute lymphoblastic leukemia (T-ALL) and to find out risk indicators for prognosis. Methods: This study was a prospective multicenter cohort study involving 299 newly diagnosed T-ALL children in 21 Grade A tertiary hospitals nationwide. All patients received CCLG-ALL 2018 regimen and clinical data for treatment efficacy evaluating was collected. Variables associated with event free survival (EFS) rate, overall survival (OS) rate and cumulative recurrence rate were evaluated by Lasso regression analysis (including variables selection, model construction and hazard ratio calculating). Results: A total of 299 newly diagnosed T-ALL children were included, accounting for 9.9% (299/3 026) of all ALL patients. Among these patients, there were 224 males and 75 females, and the age of onset was 7.0 (4.7, 10.6) years. All patients received CCLG-ALL 2018 regimen treatment. After 31.1 (17.3, 43.8) months follow-up, 3-year EFS, 3-year OS and cumulative recurrence rate of them were (83.2±2.7)%, (91.3±1.8)%, and (7.9±1.7)%, respectively. Minimal residual disease (MRD) greater than 10.00% on day 15 of induction therapy was a risk factor for EFS (HR=1.89, 95%CI 1.04-3.44), OS (HR=2.82, 95%CI 1.35-5.92), and cumulative recurrence rate (HR=3.05, 95%CI 1.46-6.34). Compared with the medium-risk group, the high-risk group had higher induction failure rate (5.2% (7/134) vs. 0 (0/145), P=0.016) and lower complete remission rate (88.8% (119/134) vs.97.9% (142/145),P=0.004). Most complications happened during induction therapy (95 cases), and the most common complication was serious infection (158 cases). Conclusions: CCLG-ALL 2018 regimen shows good prognosis. MRD greater than 10.00% on day 15 of induction therapy is a strong risk factor, which can indicate the prognosis in the early stage of the disease and guide the appropriate treatment.目的： 总结中国儿童白血病协作组2018（CCLG-ALL 2018）方案对急性T淋巴细胞白血病（T-ALL）的治疗效果，并探讨影响预后的相关因素。 方法： 前瞻性多中心队列研究。纳入国内21家三甲医院的新诊断T-ALL患儿共299例患儿为研究对象，接受CCLG-ALL 2018方案的治疗，收集临床资料，评估治疗效果，通过Lasso回归进行变量筛选，利用筛选的变量进行模型构建，评估与无事件生存率（EFS）、总生存率（OS）及累积复发率相关的预后指标。 结果： 纳入299例新诊断T-ALL患儿，占全部急性淋巴细胞白血病患儿的9.9%（299/3 026），其中男224例、女75例，发病年龄7.0（4.7，10.6）岁。经过规范CCLG-ALL 2018方案治疗，随访31.1（17.3，43.8）个月，患儿3年OS为（91.3±1.8）%，3年EFS为（83.2±2.7）%，累积复发率为（7.9±1.7）%。诱导治疗第15天微小残留病（MRD）>10.00%为EFS（HR=1.89，95%CI 1.04~3.44）、OS（HR=2.82，95%CI 1.35~5.92）及复发（HR=3.05，95%CI 1.46~6.34）的危险因素。高危组患儿的诱导失败率高于中危组［5.2%（7/134）比0（0/145），P=0.016］，完全缓解率低于中危组［88.8%（119/134）比97.9%（142/145），P=0.004］。化疗过程中诱导治疗期间发生并发症最多（95例），所有阶段并发症以严重感染最常见（158例）。 结论： CCLG-ALL 2018方案疗效较好，第15天MRD>10.00%为T-ALL预后不良因素，可在病程早期提示预后并指导治疗。.