Objective: To investigate the efficacy and the prognostic factors in pediatric hepatoblastoma according to the standard diagnostic and therapeutic regimen. Method: Eighty-four consecutive patients were enrolled in this study between June 2000 and June 2015. Diagnosis and staging was decided by the multi-disciplinary team including oncologists, surgeons, pathologists and sub-specialized radiologists refering to protocol of Children's Oncology Group(COG) and International Society of Pediatric Oncology Liver Tumor Study Group (SIOPEL) in a case observational study. Univariate analysis was tested by the log-rank and multivariate analysis by COX regression. All consecutive cases were divided into low risk group and high risk group according to grouping criteria. Complete remission was defined as both imaging negative and α fetoprotein (AFP) normalization. Retrospective analysis was performed in clinical features, long-term outcomes and prognostic factors. Result: Ten patients were excluded because of giving up after less than or equal to three cycles of treatment. A total of 74 cases were included in this study; 45 males and 29 females. The median age at diagnosis was 1.7 years(range 0.2-14.8 years). Untill August 30, 2016, the median follow-up time was 24.2 months (range 4.1-135.3 months); 59 cases achieved complete remission.The estimated five years overall survival (OS) and event free survival(EFS) were 90%(68/74)and 72%(58/74). AFP could be normalized after 5 circles of treatment or 2 circles of postoperation.In univariate analysis , the five years OS and EFS in low risk group were both 100%(18/18), and those in high risk group were 88%(50/56)and 68%(40/56), respectively. The five years OS rates were 75%(15/19) and 95%(53/55) in patients with or without distant metastasis (P=0.016). After 3 cycles of chemotherapy post tumor resection, we divided these patients into 2 groups according to AFP recover or not, the five years OS were 100%(43/43)and 81%(22/26), respectively (P=0.011). Conclusion: The result of this protocol is reasonable when comparing with other worldwide research. Except for staging, metastasis, pathological subtypes, postoperative AFP recover or not is a prognostic factor after 3 cycles of chemotherapy.
目的： 总结在规范化诊断与治疗方案下儿童肝母细胞瘤(HB)的疗效，分析预后相关因素。 方法： 病例观察性研究，选自2000年6月到2015年6月上海交通大学附属上海儿童医学中心收治的84例HB患儿，借鉴国际方案制定和采用外科手术、内科化疗的儿童HB诊断与治疗方案，符合入组标准的连续病例进入统计分析。单因素分析采用Log－rank检验，多因素预后分析采用COX回归模型。回顾性分析其临床特征、远期预后和预后相关因素，并与国外美国儿童肿瘤协会和国际儿童肿瘤协会肝肿瘤研究组HB治疗方案进行比较。分为低危、高危组，低危组先手术、后化疗；高危组先活检、确诊后化疗、肿瘤缩小后再手术、再化疗。手术完全切除影像学提示无残留病灶且甲胎蛋白(AFP)恢复正常定义为完全缓解。 结果： 剔除≤3个疗程后好转但家长放弃治疗10例，共74例纳入统计分析。男45例，女29例，中位年龄1.7岁(0.2～14.8岁)。截至2016年8月30日，中位随访24.2 (4.1～135.3)个月。末次随访完全缓解59例(80%)。5年总生存率(OS)和无事件生存率(EFS)分别为90%(68/74)和72%(58/74)。AFP在中位5个疗程、术后中位2个疗程后恢复正常。单因素预后分析，低危组5年OS和EFS均为100%(18/18)，高危组分别为88%(50/56)和68%(40/56)。全组伴或不伴肝外转移者5年OS分别为75%(15/19)和95%(53/55)(P＝0.016)。肿瘤完整切除术后3个疗程后AFP恢复正常与否，其5年OS分别为100% (43/43)和81%(22/26)(P＝0.011)。 结论： 诊断治疗方案及预后与国外HB方案有可比性，具有临床应用合理性。分期、远程转移、病理分型是预后相关因素外，肿瘤切除术后3个疗程后AFP恢复正常与否对预后有影响。.
Keywords: Child; Follow-up studies; Hepatoblastoma; Surgical procedures, operative.