多学科综合治疗儿童神经母细胞瘤的临床疗效评估

Clinical outcomes of children with neuroblastoma under multidisciplinary treatment

目的探讨多学科综合治疗下的儿童神经母细胞瘤（neuroblastoma，NB）的临床疗效评估，为进一步优化治疗方案提供依据。方法回顾性分析2007年1月至2012年12月上海交通大学医学院附属新华医院收治且随访资料完整的95例NB患儿临床资料。95例患儿中，男女比例为1．6：1，中位诊断年龄为31．6个月（1．1～159．8个月），中位随访时间为32个月（13～80个月）。中晚期（INSS分期Ⅲ期和Ⅳ期）病例占66．3％（63／95）。结果按照不同的危险度分组，选择性地给予患儿不同强度的化疗、手术、局部放疗、自体造血干细胞移植、全顺式维甲酸诱导分化治疗。预计5年总体生存率（5y-OS）为（67．2±5．3）％，5年无事件生存率（5y-EFS）为（57．8±5．5）％；其中Ⅰ、Ⅱ期患儿的5y-OS均为100％，5y-EFS为分别为（92．3±7．4）％、100％；Ⅲ期患儿5y-OS为（63．6±14．5）％，5y-EFS为（42．4士19．8）％；Ⅳ期5y-OS为（46．9±8．3）％，5y-EFS为（35．4±7．8）％。低危组、中危组预后明显好于高危组、极高危组（P均〈0．001）。本组共有28例（30．4％）患儿死亡，死亡首要原因是肿瘤进展所致（20／28，71．4％），其次为化疗相关的严重感染（6／28，21．4％）。结论多学科综合治疗可有效改善儿童神经母细胞瘤的预后，应重视化疗后并发严重感染导致的死亡事件。

Objective To explore the effects of multidisciplinary treatment on clinical features and outcomes of children with neuroblastoma and provide rationales for optimized treatment. Methods A total of 95 neuroblastoma patients enrolled into our hospital from January 2007 to December 2012 were retrospectively analyzed. Their median age at diagnosis was 31.6 （1.1-159. 8） months, male to female ratio 1.6：1 and median follow-up time 32 （13-80） months. Advanced neuroblastoma of International Neuroblastoma Staging System stages Ⅲ-Ⅳ accounted for 66. 3% （63/95）. Results According to different risk groups, all patients received intensive chemotherapy, surgery, radiotherapy, autologous hematopoietic stem cell transplantation （AS（T） and 13-cis-retinoid for induced differentiation therapy. Estimated 5-year overall survival （5y-OS） was 67. 2% ± 5.3% and event-free survival （5y-EFS） 57. 8% ± 5.5%. The 5y-OS was 100% for stage Ⅰ , 100% for stage Ⅱ , 63.6% ±14. 5% for stage Ⅲ and 46. 9% ＋ 8. 3% for stage IV. And the 5y-EFS was 92. 3% ＋ 7. 4% for stage Ⅰ , 100% for stage Ⅱ , 42. 4% ±19. 8% for stage Ⅲ and 35.4% ＋ 7. 8% for stage Ⅳ. The prognosis of low-riskers and middle-riskers was much better than that of high-riskers and very-high- riskers （P〈0. 001）. Among 28 mortalities, the leading causes were tumor progression （20/28, 71.4%） and severe chemotherapy-related infection （6/28, 21.4%）. Conclusions Multi-disciplinary comprehensive treatment can effectively improve the prognosis of children with.neuroblastoma. And mortality due to severe chemotherapy-related infection should raise high alerts.