Medical Oncology Department, Pediatric Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Key Laboratory of Major Disease in Children, Ministry of Education, Beijing, 100045, China.
Clinical Epidemiology and Evidence-based Medicine, National Center for Children's Health, Beijing Children's Hospital, Capital Medical University, Beijing, China.
Ann Hematol. 2024 Mar;103(3):893-903. doi: 10.1007/s00277-023-05568-w. Epub 2023 Dec 13.
The current chemotherapy treatments have led to an improvement in survival rates for pediatric Burkitt's lymphoma (BL). Survival in children with high-grade, mature B-cell non-Hodgkin's lymphoma (B-NHL) has been prolonged by six rituximab doses combined with chemotherapy, whereas the efficacy of four doses has not been reported. This study aimed to explore optimal therapeutic strategies-the number of doses of rituximab based on different risk groups-and also aim to investigate the clinical characteristics of Chinese pediatric BL. This study consecutively enrolled children with BL in Beijing Children's Hospital who received French-American-British mature B-cell lymphoma 96 (FAB/LMB96). The patients were divided into three groups: R0 group (chemotherapy alone), R6 group (chemotherapy combined with six rituximab doses), and R4 group (chemotherapy combined with four rituximab doses). The clinical characteristics and outcomes were evaluated. Univariate and multivariate analyses and prognostic nomogram were used to assess prognostic factors. A nomogram was developed that predicted overall survival based on the Cox proportional hazards model, and the concordance index (C-index) and a calibration curve were used to determine its predictive and discriminatory capacity. We enrolled 385 boys and 71 girls, with a median age of 6 years (1-14 years). Of these, 296 patients (65%) had initial abdominal symptoms, 182 (40%) had bulky disease, 46 (10%) had B symptoms, 77 (16.9%) had BL-ALL (blasts ≥ 25% in bone marrow (BM)), 96 (21%) had central nervous system (CNS) disease, 406 (89%) were in stages III-IV, 378 (83%) were in group C, 170 (37.2%) had lactate dehydrogenase (LDH) levels ≥ 1000 U/L at initial diagnosis, and 137 (30%) had tumor lysis syndrome. The R0, R6, and R4 groups included 79, 144, and 227 patients, respectively. Six patients were excluded due to treatment withdrawal for various reasons. The 3-year overall survival (OS) and event-free survival (EFS) percentages were 92% ± 1.3% and 91.3% ± 1.3%, respectively, in all cohorts, whereas the 3-year EFS percentage was 83.5% ± 4.2%, 93% ± 2.1%, and 92.9% ± 1.8% in the R0, R6, and R4 groups, respectively (P = 0.025). The nomogram included four important variables based on a multivariate analysis of the primary cohort: course of disease ≤ 20 days, presence of bulky disease at the beginning of diagnosis, central nervous system(CNS) invasion, and dosage of rituximab. The calibration curve showed that the nomogram was able to predict 3-year OS accurately. The C-index of the nomogram for OS prediction was 0.79 for both cohorts. In our hospital, pediatric BL was more commonly observed in school-age boys with an abdominal mass and mostly in advanced stages at initial diagnosis. The FAB/LMB96 regimen combined with rituximab significantly increased survival outcomes. We observed no significant differences between four and six doses of rituximab in terms of treatment outcomes. The proposed nomogram provides an individualized risk estimate of OS in patients with BL and may assist treatment decision-making or rituximab dose design.
当前的化疗治疗方案已经提高了儿童伯基特淋巴瘤(BL)的生存率。对于高级别、成熟 B 细胞非霍奇金淋巴瘤(B-NHL)的儿童,通过联合使用 6 个利妥昔单抗剂量和化疗,可以延长生存时间,而 4 个剂量的疗效尚未报道。本研究旨在探索最佳治疗策略——根据不同风险组的利妥昔单抗剂量数,并研究中国儿童 BL 的临床特征。本研究连续纳入在北京儿童医院接受法国-美国-英国成熟 B 细胞淋巴瘤 96(FAB/LMB96)治疗的 BL 患儿。患者被分为三组:R0 组(单独化疗)、R6 组(化疗联合 6 个利妥昔单抗剂量)和 R4 组(化疗联合 4 个利妥昔单抗剂量)。评估了临床特征和结局。采用单因素和多因素分析以及预后列线图评估预后因素。根据 Cox 比例风险模型开发了一个预测总生存期的列线图,并使用一致性指数(C-index)和校准曲线来确定其预测和区分能力。我们共纳入 385 名男孩和 71 名女孩,中位年龄为 6 岁(1-14 岁)。其中,296 名患者(65%)有初始腹部症状,182 名(40%)有肿块性疾病,46 名(10%)有 B 症状,77 名(16.9%)有 BL-ALL(骨髓中blasts≥25%),96 名(21%)有中枢神经系统(CNS)疾病,406 名(89%)处于 III-IV 期,378 名(83%)处于 C 组,170 名(37.2%)在初诊时乳酸脱氢酶(LDH)水平≥1000 U/L,137 名(30%)有肿瘤溶解综合征。R0、R6 和 R4 组分别纳入 79、144 和 227 名患者。由于各种原因,有 6 名患者在治疗过程中退出。所有队列的 3 年总生存(OS)和无事件生存(EFS)百分比分别为 92%±1.3%和 91.3%±1.3%,而 R0、R6 和 R4 组的 3 年 EFS 百分比分别为 83.5%±4.2%、93%±2.1%和 92.9%±1.8%(P=0.025)。列线图基于主要队列的多因素分析纳入了四个重要变量:病程≤20 天、初诊时存在肿块性疾病、中枢神经系统(CNS)侵犯和利妥昔单抗剂量。校准曲线显示,该列线图能够准确预测 3 年 OS。该列线图对 OS 预测的 C-index 在两个队列中均为 0.79。在我院,儿科 BL 更常见于学龄期男孩,表现为腹部肿块,且初诊时多处于晚期。FAB/LMB96 方案联合利妥昔单抗显著提高了生存结果。我们观察到 4 个和 6 个剂量的利妥昔单抗在治疗结果方面没有显著差异。所提出的列线图为 BL 患者的 OS 提供了个体化风险估计,可能有助于治疗决策或利妥昔单抗剂量设计。