Objective To evaluate chemotherapy-related toxicity and the short-term efficacy of topotecan and cydophosphamide as maintenance chemotherapy for stage Ⅳ neuroblastoma in complete remission.Methods The clinical data of 16 children with stage Ⅳ neuroblastoma received 3 cycles of maintenance chemotherapy with topotecan (0.75 mg· m2· day-l,infused on days 0-4) and cyclophosphamide 250 mg· m-2· day-1,infused on days 0-4).The two-year event-free survival after complete remission was recorded and the chemotherapy-related toxicities were evaluated according to the Common Terminology Criteria for Adverse Events of the National Cancer Institute.Results The most common chemotherapy-related toxicity was bone marrow suppression and suppressions of neutrophils,hemoglobin and platelets,which occurred in all the patients mostly of grade Ⅲ and Ⅳ.All the patients experienced episodes of infections,which were controlled effectively with antibiotics.Impairment of gastrointestinal and liver functions in these cases was mostly mild (grade Ⅰ and Ⅱ) and recovered after corresponding treatments.None of the patients exhibited damages in the nervous system or the renal or cardiac functions.After complete remission,the two-year event-free survival rate of these patients was 68.75％ (11/16).Conclusion Topotecan plus cyclophosphamide for maintenance chemotherapy can be effective and relative safe for stage Ⅳ neuroblastoma in complete remission,thus giving a chance to those patients who choose not to have stem cell transplantation.%目的 观察拓扑替康与环磷酰胺作为维持化疗方案治疗完全缓解的晚期神经母细胞瘤患儿的相关毒副反应及近期疗效.方法 回顾分析经诱导化疗和手术治疗后完全缓解的16例Ⅳ期神经母细胞瘤患儿临床资料,均予3疗程拓扑替康联合环磷酰胺的维持化疗方案(拓扑替康0.75 mg·m-2·day-1,第0～4天；环磷酰胺:250 mg·m-2·day-1,第0～4天).应用美国国立癌症研究所通用不良反应分级标准对化疗相关毒副反应进行评价,观察完全缓解后2年的无病生存率.结果 16例患儿化疗相关毒副反应主要是对骨髓的抑制,粒系100％达到Ⅳ级,红系、巨核系Ⅲ、Ⅳ级毒性损害累积达100％.所有患儿均出现不同程度的感染,经积极的抗感染治疗均可以控制.消化道、肝脏损害多为Ⅰ、Ⅱ级,经治疗可恢复.未见肾脏、神经、心脏毒副反应病例.该维持化疗方案2年内无病生存率可达68.75％(11/16).结论 扑替康联合环磷酰胺的维持化疗方案可以安全、有效的治疗Ⅳ期完全缓解的神经母细胞瘤患儿,是无经济条件应用造血干细胞移植患儿可以选择的维持化疗方案之一.
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