Department of Pediatric Surgery and Organ Transplantation, Children's Memorial Health Institute, 04-730 Warsaw, Poland.
J Pediatr Surg. 2012 Jul;47(7):1331-9. doi: 10.1016/j.jpedsurg.2011.11.073.
BACKGROUND/PURPOSE: The aim of the study was to analyze changing management and survival of children with hepatoblastoma (HBL) treated in one center.
Over the last 20 years, 51 children with HBL were treated. Surgery was performed in 48 children (94.1%), conventional liver resection in 38 (of those, 2 received a rescue liver transplantation [LTx] for relapse), and total hepatectomy and primary LTx in 10 patients. The remaining 3 patients received only palliative treatment. Patient data were analyzed for survival with respect to PRETreatment EXTent of disease (PRETEXT), metastases, histopathology, conventional resection, and LTx.
Survival of children with HBL treated with liver resection is 71% and 80% for primary LTx. Favorable prognostic factors for patient survival was tumor histology as epithelial-fetal subtype and mixed epithelial and mesenchymal type, without teratoid features, and good response to chemotherapy (necrosis, fibrosis). Unfavorable prognostic factors were small cells undifferentiated, transitional liver cell tumor, α-fetoprotein level above 1,000,000 IU/mL and below 100 IU/mL at diagnosis, lung metastases, and local recurrence after initial resection. Survival was related to PRETEXT stage. However, among patients with PRETEXT III and IV, LTx resulted in better survival.
Liver transplantation is a good option for children with advanced HBL. Early referral of children with potentially unresectable tumors to centers where combined treatment (chemotherapy, surgery including LTx) is available is crucial.
背景/目的：本研究旨在分析一个中心治疗的肝母细胞瘤（HBL）患儿治疗方法的变化和生存情况。
在过去的 20 年中，对 51 例 HBL 患儿进行了治疗。48 例患儿（94.1%）接受了手术，其中 38 例接受了常规肝切除术（其中 2 例因复发接受了挽救性肝移植[LTx]），10 例接受了全肝切除术和原发性 LTx。其余 3 例仅接受姑息治疗。对患者的生存数据进行了分析，分析因素包括 PRETEXT 分期、转移、组织病理学、常规切除和 LTx。
接受肝切除术治疗的 HBL 患儿的生存率为 71%，接受原发性 LTx 治疗的生存率为 80%。患者生存的有利预后因素是肿瘤组织学为上皮-胎儿亚型和混合上皮和间充质型，无畸胎瘤特征，对化疗有良好反应（坏死、纤维化）。不利的预后因素是小细胞未分化、过渡性肝母细胞瘤、甲胎蛋白（AFP）水平在诊断时高于 100 万 IU/mL 且低于 100 IU/mL、肺转移和初始切除后局部复发。生存率与 PRETEXT 分期有关。然而，在 PRETEXT III 和 IV 期患儿中，LTx 可获得更好的生存。
肝移植是治疗晚期 HBL 患儿的良好选择。对于那些可能无法切除的肿瘤患儿，应尽早转至能够提供联合治疗（化疗、手术包括 LTx）的中心，这一点至关重要。
