Liu Anthony Pak-Yin, Zhen Zijun, Yang Qunying, Yuan Xiaojun, Ma Xiaoli, Chen Jianliang, Wang Jingsheng, Yang Lihua, Guo Haixia, Jiang Lian, Weng Wenjun, Huang Libin, Liu Wei, Wang Jian, Wu Shaoxiong, Zeng Jing, Jiang Mawei, He Kejun, Ge Ming, Cheng Kevin King-Fai, Ho Wilson Wai-Shing, Li Chunyu, Yu Lihua, Zhu Shuai, Ng Ho-Keung, Chan Godfrey Chi-Fung, Sun Xiaofei
Department of Paediatrics and Adolescent Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
Department of Paediatrics and Adolescent Medicine, Hong Kong Children's Hospital, Hong Kong SAR, China.
Neurooncol Adv. 2021 Sep 17;3(1):vdab134. doi: 10.1093/noajnl/vdab134. eCollection 2021 Jan-Dec.
Medulloblastoma (MB) is the most common malignant central nervous system tumor of childhood. Management requires interdisciplinary care and is associated with unique challenges in developing regions. Here, we report the characteristics, clinical outcome and treatment barriers for Chinese children with MB based on a multi-institutional cohort from the Chinese Children's Cancer Group (CCCG).
Retrospective cohort study among 12 Chinese pediatric oncology units from the CCCG Brain Tumor Workgroup on patients aged <18 years diagnosed with MB from 2016 to 2019.
221 patients (male:female = 138:83) were included, 175 (79%) were ≥3 years of age, and 46 (21%) <3 years. 177 patients (80%) were completely staged, among which 50 (28%) had metastasis and 70 (40%) were considered to have high-risk (HR) disease. Gross/near-total resection was achieved in 203 patients (92%). In patients where molecular grouping could be assigned, 19 (16%), 35 (29%), and 65 (54%), respectively had WNT-activated, SHH-activated, and Group 3/4 MB. The median duration between resection and initiation of adjuvant therapy was 36 days. Respective 2-year PFS and OS rates were 76.0 ± 3.0% and 88.0 ± 2.3%. PFS was significantly associated with age, metastatic status and clinical risk grouping. Chemotherapy use during CSI or alkylator choice were not significant predictors for patient outcome.
We reported the clinical profiles and outcome from the largest cohort of Chinese children with MB after multi-modal therapy. Strengths and limitations on the local provision of neuro-oncology service are identified.
髓母细胞瘤（MB）是儿童期最常见的恶性中枢神经系统肿瘤。其治疗需要多学科护理，在发展中地区存在独特挑战。在此，我们基于中国儿童癌症协作组（CCCG）的多机构队列研究，报告中国儿童MB的特征、临床结局及治疗障碍。
对CCCG脑肿瘤工作组中12个中国儿科肿瘤科室在2016年至2019年诊断为MB的18岁以下患者进行回顾性队列研究。
纳入221例患者（男∶女 = 138∶83），175例（79%）年龄≥3岁，46例（21%）<3岁。177例患者（80%）进行了完整分期，其中50例（28%）有转移，70例（40%）被认为患有高危（HR）疾病。203例患者（92%）实现了大体/近全切。在可进行分子分型的患者中，分别有19例（16%）、35例（29%）和65例（54%）为WNT激活型、SHH激活型和3/4组MB。手术切除至辅助治疗开始的中位间隔时间为36天。2年无进展生存率（PFS）和总生存率（OS）分别为76.0±3.0%和88.0±2.3%。PFS与年龄、转移状态和临床风险分组显著相关。全脑脊髓照射（CSI）期间的化疗使用或烷化剂选择并非患者结局的显著预测因素。
我们报告了多模式治疗后中国最大队列儿童MB的临床特征和结局。确定了当地神经肿瘤服务的优势和局限性。
