Objectives: To investigate the effectiveness of high-dose chemotherapy combined with autologous hematopoietic stem cell transplantation (ASCT) in the treatment of children with high-risk neuroblastoma (NB).
Methods: A retrospective analysis was performed on 29 children with high-risk NB who were admitted to Shanghai Children's Hospital and were treated with high-dose chemotherapy combined with ASCT from January 2013 to December 2021, and their clinical features and prognosis were analyzed.
Results: Among the 29 children treated by high-dose chemotherapy combined with ASCT, there were 18 boys (62%) and 11 girls (38%), with a median age of onset of 36 (27, 59) months. According to the International Neuroblastoma Staging System, 6 children (21%) had stage III NB and 23 children (79%) had stage IV NB, and the common metastatic sites at initial diagnosis were bone in 22 children (76%), bone marrow in 21 children (72%), and intracalvarium in 4 children (14%). All 29 children achieved reconstruction of hematopoietic function after ASCT. After being followed up for a median time of 25 (17, 45) months, 21 children (72%) had continuous complete remission and 8 (28%) experienced recurrence. The 3-year overall survival rate and event-free survival rate were 68.9%±16.1% and 61.4%±14.4%, respectively. Presence of bone marrow metastasis, neuron-specific enolase ≥370 ng/mL and positive bone marrow immunophenotyping might reduce the 3-year event-free survival rate (P<0.05).
Conclusions: Children with high-risk NB who have bone marrow metastasis at initial diagnosis tend to have a poor prognosis. ASCT combined with high-dose chemotherapy can effectively improve the prognosis of children with NB with a favorable safety profile.
目的: 探讨大剂量化学药物治疗（简称化疗）联合自体造血干细胞移植（autologous hematopoietic stem cell transplantation，ASCT）治疗高危神经母细胞瘤（neuroblastoma，NB）患儿的疗效。方法: 回顾性选择2013年1月—2021年12月期间上海市儿童医院收治的予以大剂量化疗联合ASCT的29例高危NB患儿为研究对象，分析患儿的临床特点及预后。结果: 29例大剂量化疗联合ASCT的患儿中，男18例（62%），女11例（38%），中位发病年龄为36（27，59）个月。根据国际神经母细胞瘤分期系统标准分期，Ⅲ期6例（21%），Ⅳ期23例（79%），初诊时常见转移部位为骨22例（76%）、骨髓21例（72%）、颅内4例（14%）。29例患儿ASCT后造血功能均获得满意重建，中位随访时间为25（17，45）个月，移植后持续完全缓解21例（72%），复发8例（28%），3年总生存率及无事件生存率分别为68.9%±16.1%、61.4%±14.4%。存在骨髓转移、神经元特异性烯醇化酶≥370 ng/mL、骨髓免疫分型阳性可降低患儿的3年无事件生存率（P<0.05）。结论: 初诊时存在骨髓转移的高危NB患儿预后差。ASCT联合大剂量化疗可有效改善NB患儿预后，且安全性高。.
Keywords: Autologous hematopoietic stem cell transplantation; Child; Conditioning regimen; Neuroblastoma; Prognosis.