Zhang Dongdong, Kaweme Natasha Mupeta, Duan Peng, Dong Youhong, Yuan Xiaojun
Department of Pediatric Hematology/Oncology, Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Department of Oncology, Xiangyang No. 1 People's Hospital, Hubei University of Medicine, Xiangyang, China.
Front Oncol. 2021 Nov 15;11:745794. doi: 10.3389/fonc.2021.745794. eCollection 2021.
The Chinese Children's Cancer Group developed the CCCG-NB-2014 study to formulate optimal treatment strategies for high-risk (HR) neuroblastoma (NB). The safety and efficacy of this protocol were evaluated.
Patients with newly diagnosed neuroblastoma and defined as HR according to the Children's Oncology Group study were included. They were treated with a combination of chemotherapy, surgery, and radiotherapy. The treatment-related toxicities, response rate, 3-year progression-free survival (PFS), and overall survival (OS) were analyzed.
Of 159 patients enrolled between 2014 and 2018, 80 were eligible, including 19 girls and 61 boys, with a median age of 3.9 years (range 0.9-11). After a median follow-up of 24 months (range 3-40), the median OS was 31.8 months, and 3-year OS was 83.8%. In multivariate analyses, the OS was affected by N-MYC amplification (hazard ratio 0.212, 95% confidence interval (CI) 0.049-0.910; = 0.037) and giant tumor mass (hazard ratio 0.197, 95% CI 0.071-0.552; = 0.002). The median 3-year PFS was 25.8 months, and 3-year PFS was 57.5%. The univariate analysis showed that only the giant tumor mass was associated with the outcome. Of the 13 deaths, 11 died from the rapid progression of the disease and two from treatment-related toxicities. The most common adverse reaction was chemotherapy-induced hematological toxicity.
The PFS and OS reported in our study were similar to Western countries. The CCCG-NB-2014 protocol proved to be an efficient regimen with tolerable side-effect for the treatment of pediatric HR-NB.
中国儿童癌症协作组开展了CCCG-NB-2014研究，以制定高危（HR）神经母细胞瘤（NB）的最佳治疗策略。对该方案的安全性和有效性进行了评估。
纳入新诊断的神经母细胞瘤患者，根据儿童肿瘤协作组研究定义为HR。他们接受了化疗、手术和放疗的联合治疗。分析了治疗相关毒性、缓解率、3年无进展生存期（PFS）和总生存期（OS）。
2014年至2018年纳入的159例患者中，80例符合条件，包括19例女孩和61例男孩，中位年龄为3.9岁（范围0.9 - 11岁）。中位随访24个月（范围3 - 40个月）后，中位OS为31.8个月，3年OS为83.8%。多因素分析中，OS受N-MYC扩增影响（风险比0.212，95%置信区间（CI）0.049 - 0.910；P = 0.037）和巨大肿瘤肿块影响（风险比0.197，95%CI 0.071 - 0.552；P = 0.002）。中位3年PFS为25.8个月，3年PFS为57.5%。单因素分析显示，只有巨大肿瘤肿块与预后相关。13例死亡患者中，11例死于疾病快速进展，2例死于治疗相关毒性。最常见的不良反应是化疗引起的血液学毒性。
我们研究中报告的PFS和OS与西方国家相似。CCCG-NB-2014方案被证明是一种治疗儿童HR-NB有效的方案，且副作用可耐受。
