多学科综合治疗47例儿童肝母细胞瘤的疗效评估

Assessment of efficacy of 47 children with hepatoblastoma under multidisciplinary treatment

目的分析应用《儿童肝母细胞瘤多学科诊疗规范》（武汉方案）治疗肝母细胞瘤（HB）的临床疗效，评估该方案的可行性和有效性，为进一步优化HB治疗方案提供依据。方法回顾性分析2008年1月至2013年12月上海交通大学医学院附属新华医院收治并纳入武汉方案治疗的47例HB患儿的临床、影像、病理、化疗及手术记录，分析上述因素对预后的影响。结果47例患儿（男33例，女14例），男女比例为2．4：1．0，中位诊断年龄为13．3（1．3—126．0）个月。5年总体生存率为（83．5±5．9）％，5年无事件生存率为（76．2±6．3）％，高危组和标危组5年总体生存率分别为（59．2±16．9）％和（91．2±4．9）％。单因素分析提示发病年龄≤3岁（P=0．001）、手术完整切除（P：0．000）、确诊时无肝外转移（P：0．002）、新辅助化疗后延期手术（P=0．000）的患儿预后较好。多因素分析提示手术完整切除[北美儿童肿瘤协作组（COG）I期，r=0．064，95％C10．007～0．565，P=0．013]可作为判断预后的独立因素。本组仅1例（2．1％）患儿在治疗过程中因并发严重感染死亡。结论化疗可有效提高手术切除的完整率，应用武汉方案治疗儿童HB的不良反应较小，总体生存率及无病生存率接近国外文献报道的水平，说明该方案对治疗我国HB患儿具有一定的有效性和可行性，但方案仍需要对手术切除指征、按照危险度分层化疗等方面进一步优化。

Objective To retrospectively analyze the clinical efficacy of the patients with hepatoblastoma （HB） treated following Wuhan Protocol in Xinhua Hospital, so as to assess the feasibility and effectiveness of this pro- tocol in order to provide the basis for further optimization of treatment. Methods The medical records of all enrolled 47 HB patients in Xinhua Hospital Affiliated to Shanghai Jiaotong University during January 2008 to December 2013 were retrospectively reviewed and collected, including medical history, CT/MRI images, pathological types, chemothera- py regimens and surgery. All the factors contributing to prognosis were drawn into analysis. Results Forty - seven pa- tients （ boy 33 cases ,girl 14 cases） with HB were enrolled in the cohort. The ratio of male to female was 2.4 ： 1.0 ,the median age at diagnosis was 13.3 （ 1.3 - 126.0） months. The 5 - year overall survival （OS） rate and event - free sur- vival （EFS） rate were （ 83.5 ＋ 5.9 ） % and （76.2 ＋ 6.3 ） %, respectively, the 5 - year OS of the high risk and the standard risk group were （59.2 ＋ 16.9）% and （91.2 ＋4.9）%. The univariate analysis revealed that patients less than 3 years old （P =0.001 ） ,complete surgical resection （P =0. 000） ,no metastases at diagnosis （P =0. 002） and delayed surgery following neoadjuvant chemotherapy （ P = 0. 000 ） produced better prognosis. In multivariate analysis, complete surgical resection （r =0.064,95% CIO. 007 -0.565 ,P =0.013） was regarded as an independent good prog- nostic factor. There was only 1 case died of severe infection during treatment. Conclusions Neoadjuvant chemotherapy could effectively improve the complete surgical resection. The preliminary results of Wuhan Protocol showed less side - effects and similar OS or EFS rates compared to the developed countries, suggesting that the protocol is feasible and ef- fective in China. However, the protocol remains to be further optimized in surgery indications and risk -based chemo- therapy.