Czauderna Piotr
On behalf of SIOPEL group - from Department of Surgery and Urology for Children and Adolescents, Medical University of Gdansk, Poland.
Front Biosci (Elite Ed). 2012 Jan 1;4(1):470-9. doi: 10.2741/e392.
International Childhood Liver Tumors Strategy Group (SIOPEL) introduced the concept of preoperative chemotherapy in hepatoblastoma, most common malignant liver tumor in children. This required introduction of the preoperative tumor staging system called PRETEXT. SIOPEL 1 study proved the value of preoperative chemotherapy consisting of cisplatin and doxorubicin (PLADO) in hepatoblastoma leading to 5-year overall survival of 75 % and event-free-survival of 66 %. Both presence of metastases and PRETEXT were significant prognostic factors which led to development of two risk categories: standard (SR) and high risk (HR) hepatoblastomas. In SIOPEL 2 study two different strategies were developed for SR and HR tumors with corresponding 3-year overall and progression-free survival of 91 % and 89 %, and 53 % and 48 % respectively. In the next SIOPEL 3 SR arm study monotherapy regimen based on CDDP alone was non-inferior to PLADO for SR hepatoblastoma and less toxic. Cisplatin-based chemotherapy in combination with delayed definitive surgery / liver transplantation improved the survival of children with hepatoblastoma. However certain patients, especially those with metastatic disease and low alphafetoprotein still have inferior prognosis.
国际儿童肝肿瘤战略小组（SIOPEL）引入了儿童最常见的恶性肝肿瘤——肝母细胞瘤术前化疗的概念。这就需要引入名为PRETEXT的术前肿瘤分期系统。SIOPEL 1研究证明了由顺铂和阿霉素（PLADO）组成的术前化疗在肝母细胞瘤中的价值，5年总生存率达75%，无事件生存率达66%。转移灶的存在和PRETEXT都是重要的预后因素，由此产生了两个风险类别：标准风险（SR）和高风险（HR）肝母细胞瘤。在SIOPEL 2研究中，针对SR和HR肿瘤制定了两种不同策略，相应的3年总生存率和无进展生存率分别为91%和89%，以及53%和48%。在下一项SIOPEL 3 SR组研究中，对于SR肝母细胞瘤，仅基于顺铂的单药治疗方案不劣于PLADO且毒性更小。基于顺铂的化疗联合延迟确定性手术/肝移植提高了肝母细胞瘤患儿的生存率。然而，某些患者，尤其是那些有转移疾病且甲胎蛋白水平低的患者，预后仍然较差。
