[Clinical features and prognostic analysis of hepatoblastoma in children under six years old].

Objective: To summarize and analyze the clinical features, treatment effects and related factors affecting the prognosis of hepatoblastoma (HB) in children under six years old. Methods: Clinical data of 382 children with HB under six years old who were pathologically diagnosed at the Pediatric Single Center of Beijing Tongren Hospital from May 2005 to May 2019 were analyzed retrospectively. The factors affecting the treatment effect and survival rate of HB were analyzed. The independent risk factors affecting the prognosis of HB were studied by Cox regression model. The χ(2) test was used to compare the enumeration data between groups. Kaplan-Meier method was used for survival analysis. Log-rank test was used to compare the survival rates among subgroups. Results: Children enrolled were with median age of 1.75 (0.08 ~ 5.92) years old and a male to female ratio of 1.5. Alpha-fetoprotein (AFP) median level was 197 406.5 μg/L at initial diagnosis, and the pathological tissue type was mainly epithelial (55.8%). Preoperative PRETEXT stage was mostly stage III (58.6%). 86 cases (22.5%) had portal vein or hepatic vein, and vena cava invasion. 73 cases (19.1%) had extrahepatic adjacent tissues and organs invasion. Twenty-four cases (6.3%) had tumor rupture and bleeding. 171 cases (44.8%) had distant metastases, and 96 cases (25.1%) had multiple intrahepatic lesions. Patients were followed-up to May 2020 (median follow-up time was 56 months). After comprehensive treatment, 218 cases were completely relieved, and 69 cases were partially relieved, and the treatment efficiency was 75.1%. Kaplan-Meier survival analysis showed that the 1, 3, and 5-years overall survival rates (OS) were 93.7%, 84.0%, and 73.9%, respectively, and the event-free survival rates were 90.5%, 79.2%, and 67.5%, respectively. Comparison of the clinical factors of 5-year OS showed that AFP < 100 μg/L (HR = 3.341, P = 0.005), PRETEXT stage IV (HR = 4.026, P = 0.001), vascular invasion (HR = 2.178, P = 0.019) and distant metastasis (HR = 2.634, P = 0.010) were independent risk factors in each subgroup affecting the prognosis of children with HB, and the difference was statistically significant. Conclusion: HB prognosis is related to AFP level, PRETEXT stage, presence or absence of vascular invasion and distant metastasis. Therefore, its survival and prognosis will be different in the presence of different risk factors.目的： 总结6岁以下儿童肝母细胞瘤（HB）的临床特点、治疗效果，分析影响预后的相关因素。 方法： 回顾性分析2005年5月-2019年5月就诊于北京同仁医院儿科单中心的382例经病理确诊、年龄为6岁以下的HB患儿的临床资料，分析影响HB治疗效果及生存率的因素，通过COX回归模型研究影响HB预后的独立危险因素。计数资料组间比较采用χ(2)检验，并采用Kaplan-Meier法进行生存分析，Log-rank检验进行各亚组间生存率的比较。 结果： 入组患儿中位年龄为1.75（0.08~5.92）岁，男/女= 1.5；初诊时甲胎蛋白（AFP）中位水平为197 406.5 μg/L，病理组织分型以上皮型为主（55.8%），PRETEXT术前分期以III期居多（58.6%）；有86例（22.5%）存在门静脉或肝静脉、腔静脉浸润，73例（19.1%）存在肝外邻近组织器官浸润，24例（6.3%）出现肿瘤破裂出血，171例（44.8%）存在远处转移，96例（25.1%）存在肝内多发病灶。随访至2020年5月（中位随访时间56个月），经综合治疗后，完全缓解218例，部分缓解69例，治疗有效率75.1%；Kaplan-Meier生存分析显示1、3、5年的总体生存率（OS）分别为93.7%、84.0%、73.9%，无事件生存率分别为90.5%、79.2%、67.5%。比较各临床因素亚组间的5年OS显示，AFP < 100 μg/L（HR = 3.341，P = 0.005）、PRETEXT IV期（HR = 4.026，P = 0.001）、血管侵犯（HR = 2.178，P = 0.019）及远处转移（HR = 2.634，P = 0.010）是影响HB患儿预后的独立危险因素，差异有统计学意义。 结论： HB的预后与AFP水平、PRETEXT分期以及有无血管侵犯、远处转移相关，在不同的危险因素存在下其生存与预后会有差异。.