Survival, surgical resectability, and late effects in the hepatoblastoma patients treated by cisplatin plus pirarubicin (CITA) chemotherapy.

Abstract

10038 Background: The Japanese Study Group for Pediatric Liver Tumor(JPLT) is running cooperative treatment studies on hepatoblastoma (HB) since 1991. The main aim in JPLT-2 study was to evaluate the efficacy of cisplatin/pirarubicin. JPLT-2 protocol was launched in 1999 to evaluate the cure rate of risk-stratified HB: standard risk HB (a tumor involving three or fewer sectors of the liver), intermediate risk HB (a tumor involving all sectors of the liver or invasion into portal or hepatic vein) and high risk HB (a tumor involving all sectors of the liver or with metastasis). Methods: Until 2011, 313 children with hepatic tumors who were younger than 15 years of age were eligible for inclusion in the JPLT2 study. The cisplatin/pirarubicin regimen (CITA) is kept as the first line in 254 cases. In this study, we examined the outcome and late effects of the HB patients by the risk-stratified three groups (standard, intermediate and high risk groups). Results: Until 2011, 254 cases underwent the CITA protocol including 78 cases (18%) with metastatic tumors. The 3-year OS of the cases with standard risk HB were 94%, while that of the cases with Intermediate and high risk HB was 64% and 34%, respectively. Except for23 cases who underwent primary resection, complete resection of primary after CITA was performed 86% of standard risk, 66% of intermediate risk and 56% of high risk patients. And the late phase complications were 4 cases with maldevelopment, 15 with cardiac complications, 24 with ototoxicity and 5 with second malignancies. Conclusions: As compared with other regimens, CITA regimens achieved similar or superior rates of survival and resectability in standard risk patients. More promising strategies including adequate liver transplantation and new targeting drugs should be developed for intermediate and high risk HBs. Clinical trial information: UMIN000001116.