Department of Hematology and Oncology, Shenzhen Children's Hospital, Shenzhen, China.
Department of Pathology, Shenzhen Children's Hospital, Shenzhen, China.
Cancer Control. 2023 Jan-Dec;30:10732748231187837. doi: 10.1177/10732748231187837.
Recent therapeutic advances have greatly enhanced the survival rates of patients with neuroblastoma (NB). However, the outcomes of neuroblastoma patients in China, particularly those with high-risk (HR) NB, remain limited.
We retrospectively analyzed the clinical data and outcomes of NB patients who were treated at a tertiary pediatric cancer facility in China between January 2013 and October 2021.
A total of 117 NB patients were recruited. Patients with very low-risk (VLR), low-risk (LR), intermediate-risk (IR), and HR-NB patients made up 4%, 27%, 15%, and 54% of total patient population, respectively. Patients diagnosed between 2013 and 2018 were treated according to the protocol of Sun Yat-Sen University Cancer Center and those diagnosed between 2019 and 2021 were treated according to the COG ANBL0531 or ANBL0532 protocol with or without autologous stem cell transplantation (ASCT). The 5-year EFS and OS of all risk groups of patients were 67.29% and 77.90%, respectively. EFS and OS were significantly decreased in patients with higher risk classifications (EFS: VLR/LR vs IR vs HR: 97.22% vs 67.28% vs 51.83%; ***P = .001; OS: VLR/LR vs IR vs HR: 97.06% vs 94.12% vs 64.38%; *P = .046). In HR-NB patients treated according to the COG protocol between 2019 and 2021, the 3-year OS of patients who received tandem ASCT was significantly greater than those who did not receive ASCT (93.33% % vs 47.41%; *P = .046; log-rank test). EFS was not significantly different between patients with and without ASCT (72.16% vs 60.32%).
Our findings show that patients with lower risk classification have a positive prognosis for survival. The prognosis of patients with HR-NB remains in need of improvement. ASCT may enhance OS in HR-NB patients; however, protocol adjustment may be necessary to increase EFS in these patients.
神经母细胞瘤（NB）的治疗进展显著提高了患者的生存率。然而，中国 NB 患者的预后，尤其是高危（HR）NB 患者的预后仍受到限制。
我们回顾性分析了 2013 年 1 月至 2021 年 10 月在中国一家三级儿科癌症中心治疗的 NB 患者的临床数据和结局。
共纳入 117 例 NB 患者。极低危（VLR）、低危（LR）、中危（IR）和高危-NB（HR-NB）患者分别占总患者人群的 4%、27%、15%和 54%。2013 年至 2018 年诊断的患者根据中山大学肿瘤中心的方案进行治疗，2019 年至 2021 年诊断的患者根据 COG ANBL0531 或 ANBL0532 方案进行治疗，同时联合或不联合自体干细胞移植（ASCT）。所有风险组患者的 5 年 EFS 和 OS 分别为 67.29%和 77.90%。EFS 和 OS 在风险分级较高的患者中显著降低（EFS：VLR/LR 与 IR 与 HR：97.22% 与 67.28% 与 51.83%；***P =.001；OS：VLR/LR 与 IR 与 HR：97.06% 与 94.12% 与 64.38%；*P =.046）。2019 年至 2021 年根据 COG 方案治疗的 HR-NB 患者中，接受串联 ASCT 的患者 3 年 OS 显著高于未接受 ASCT 的患者（93.33% 与 47.41%；*P =.046；log-rank 检验）。接受 ASCT 与未接受 ASCT 的患者的 EFS 无显著差异（72.16% 与 60.32%）。
我们的研究结果表明，风险分级较低的患者有良好的生存预后。HR-NB 患者的预后仍需改善。ASCT 可能提高 HR-NB 患者的 OS，但可能需要调整方案以提高这些患者的 EFS。
