Su Yan, Qin Hong, Chen Chenghao, Wang Shengcai, Zhang Shihan, Zhang Dawei, Jin Mei, Peng Yun, He Lejian, Wang Xiaoman, Qian Suyun, Qin Maoquan, Ge Ming, Zhang Fuquan, Zeng Qi, Wang Huanmin, Ma Xiaoli, Ni Xin
Beijing Key Laboratory of Pediatric Hematology Oncology National Key Discipline of Pediatrics Ministry of Education Key Laboratory of Major Diseases in Children Hematology Oncology Center Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Department of Surgical Oncology Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Pediatr Investig. 2020 Sep 27;4(3):157-167. doi: 10.1002/ped4.12214. eCollection 2020 Sep.
Neuroblastoma is the most common extracranial malignant solid tumor in children. Multidisciplinary care is critical to improving the survival of pediatric patients with neuroblastoma.
To systematically summarize the clinical characteristics of children with neuroblastoma and evaluate their prognosis with multidisciplinary care provided in a single center.
This retrospective study analyzed the clinical data of 1041 patients with neuroblastoma who were diagnosed, treated, and followed-up in the Hematology-Oncology Center of Beijing Children's Hospital from 2007 to 2019.
The median age at diagnosis was 34 months; 80.8% of the patients were younger than 5 years of age. Notably, 243 patients (23.3%) were classified as low-risk, 249 patients (23.9%) were classified as intermediate-risk, and 549 (52.7%) were classified as high-risk. Furthermore, 956 patients underwent surgical resections; 986 (94.7%) patients received chemotherapy; and 176 patients with high-risk neuroblastoma received hematopoietic stem cell transplantation. The 5-year event-free survival (EFS) rate was 91.3% and 5-year overall survival (OS) rate was 97.5% in low-risk group; in the intermediate-risk group, these rates were 85.1% and 96.7%, respectively, while they were 37.7% and 48.9% in the high-risk group ( 0.001 for both). The 5-year EFS and OS rates were significantly higher in patients diagnosed between 2015 and 2019 than in patients diagnosed between 2007 and 2014 ( 0.001). In total, 278 patients (26.7%) exhibited tumor relapse or progression; the median interval until relapse or progression was 14 months. Of the 233 patients who died, 83% died of relapse or progression of neuroblastoma and 4.3% died of therapy-related complications.
The 5-year OS rate was low in high-risk patients, compared with low-and intermediate-risk patients. Multidisciplinary care is critical for improvement of survival in pediatric patients with neuroblastoma. Additional treatment strategies should be sought to improve the prognosis of patients with high-risk neuroblastoma.
神经母细胞瘤是儿童最常见的颅外恶性实体瘤。多学科护理对于提高神经母细胞瘤患儿的生存率至关重要。
系统总结神经母细胞瘤患儿的临床特征，并评估在单一中心提供的多学科护理对其预后的影响。
这项回顾性研究分析了2007年至2019年在北京儿童医院血液肿瘤中心诊断、治疗和随访的1041例神经母细胞瘤患者的临床资料。
诊断时的中位年龄为34个月；80.8%的患者年龄小于5岁。值得注意的是，243例（23.3%）患者被归类为低风险，249例（23.9%）为中风险，549例（52.7%）为高风险。此外，956例患者接受了手术切除；986例（94.7%）患者接受了化疗；176例高风险神经母细胞瘤患者接受了造血干细胞移植。低风险组的5年无事件生存率（EFS）为91.3%，5年总生存率（OS）为97.5%；中风险组的这些比率分别为85.1%和96.7%，而高风险组分别为37.7%和48.9%（两者均P<0.001）。2015年至2019年诊断的患者的5年EFS和OS率显著高于2007年至2014年诊断的患者（P<0.001）。共有278例患者（26.7%）出现肿瘤复发或进展；复发或进展的中位间隔时间为14个月。在233例死亡患者中，83%死于神经母细胞瘤的复发或进展，4.3%死于治疗相关并发症。
与低风险和中风险患者相比，高风险患者的5年OS率较低。多学科护理对于提高神经母细胞瘤患儿的生存率至关重要。应寻求额外的治疗策略以改善高风险神经母细胞瘤患者的预后。
