Japan Breast Cancer Research Group

2009 4 2009

9577 Background: The Japanese Study Group for Pediatric Liver Tumor is running cooperative treatment studies on hepatoblastoma (HBL) since 1991. The main aim in JPLT-1 study was to evaluate the efficacy of cisplatin/pirarubicin (CITA). A total of 145 cases were registered and their 5-year overall survival (OS) and event-free survival (EFS) were 73.4% and 66%, respectively (J Pediatr Surg 37: 851-6, 2002). Then, JPLT-2 protocol, in which CITA is kept as the first-line treatment, and ifomide, etoposide, pirarubicin, and carboplatin (ITEC) is the second-line regimen for CITA-resistant cases, was launched to evaluate the cure rate of risk-stratified HBL: standard risk HBL (a tumor involving three or fewer sectors of the liver) and high risk HBL (a tumor involving all sectors of the liver or with metastasis). Methods: In JPLT-2, 281 HBL cases were registered in JPLT-2 between 1999 and 2010, and 69 cases underwent ITEC protocol due to poor response to the CITA regimen. To evaluate the efficacy of the ITEC regimen, surgical resectability and outcome of these 69 patients who underwent this treatment. Results: These 69 cases were divided into 53 high-risk (metastatic, involvement of all sectors of the liver, vascular invasion and/or extra-hepatic intra-abdominal disease) and 16 standard-risk HBL (all others). All cases were initially treated with the CITA regimen and then underwent the ITEC regimen due to poor response to CITA. Complete resection of the liver tumor could be achieved in 48 patients (69.6%) consisting of 13 (81%) standard, 35 (66%) high- risk cases. The 5-year OS and EFS of the cases with standard risk HB were 96% and 76%, while that of the cases with high risk HB was 54% and 34%. The late phase complications in 281 cases were 3 cases with maldevelopment, 13 with cardiac complications, 20 with ototoxicity and 5 with second malignancies. Conclusions: As compared with the CITA-sensitive cases, ITEC regimen achieved similar rates of survival for CITA-resistant cases both in standard and high-risk HBL cases, indicating ITEC is effective as a second-line chemotherapeutic regimen for HBL.