Abstract:

Objective　To summarize the efficacy and safety of arsenic trioxide combined with chemotherapy for children with recurrent/refractory neuroblastoma. Methods　Retrospective analysis was made in seven cases of neuroblastoma treated with traditional chemotherapy combined with venous arsenic trioxide after diagnosis with poor prognosis or the condition deteriorated. Results　Among the seven patients treated with chemotherapy combined with venous arsenic trioxide for 3 to 7 courses，4 were effective and 3 were ineffective. Patient 3 was treated with arsenic trioxide combined with following regimen after tumor was not resected totally，and patient 5，6 and 7 were given arsenic trioxide combined with preoperative chemotherapy. Patient 3 and patient 5 achieved complete remission after combination chemotherapy，and patient 6 and patient 7 showed decreased tumor volume and tumor marker. Patient 1，2 and 4 were given arsenic trioxide after failure of conventional chemotherapy. Metastasis in patient 1 disappeared at first，but the disease relapsed after 11 months. The tumor responded poorly and increased in size and number in patient 2 and patient 4. Patient 1 the use of arsenic trioxide was stopped in patient 1 because of prolonged QT interval（0.51s），who returned normal after symptomatic treatment. Five patients are still alive and they have lived for 3.5 y，3.0 y，1.2 y，1.0 y and 0.7 y，respectively. Conclusion　Venous arsenic trioxide combined with chemotherapy can be a choice of treatment for recurrent/refractory neuroblastoma，whose effect and safety requires further investigation.

Key words: arsenic trioxide, refractory, neuroblastoma, chemotherapy

摘要：

目的    探讨静脉三氧化二砷（As2O3）联合传统化疗药物对复发、难治性神经母细胞瘤（NB）的疗效及安全性。方法　回顾性分析2009年9月至2016年6月中山大学孙逸仙纪念医院收治的7例复发、难治性NB患儿，因在化疗过程中病情出现进展或初发时存在预后差的指标而在化疗中联合使用静脉As2O3。结果　患儿在化疗中联合应用As2O3共3～7个疗程，4例有效，3例无效。例3因手术无法完全切除原发灶，在术后方案中联合As2O3；例5、6、7在术前化疗方案中即联合As2O3。例3和例5联合化疗后完全缓解，例6和例7联合化疗后原发灶缩小，肿瘤标志物下降。例1、2、4因常规方案化疗无效，在后续的方案中联合As2O3。例1联合化疗后复查转移灶消失，但随访11个月后复发；例2和例4联合化疗后转移灶数量增多，最终放弃治疗。本组病例中例1使用5个疗程As2O3，后因心电图出现治疗相关性QT间期延长（0.51 s）而停药，经对症处理后恢复正常，其余与化疗联合应用As2O3共3～7个疗程，均耐受良好。目前7例患儿除2例死亡外，另5例均存活，起病至今生存时间分别为3.5、3.0、1.2、1.0及0.7年。结论　静脉As2O3联合化疗可安全用于复发、难治性NB的治疗，其疗效及安全性有待进一步多中心临床研究的验证。

关键词: 三氧化二砷, 难治性, 神经母细胞瘤, 化疗