Implementation of Comprehensive Surgical Guidelines for Hepatoblastoma: Analysis of the Children's Oncology Group AHEP0731 Phase III Trial.

Abstract

OBJECTIVE To evaluate the impact of surgical guidelines on the management of children with hepatoblastoma. BACKGROUND The Children's Oncology Group study AHEP0731 was the first pediatric cooperative group trial to propose guidelines for the surgical management of children with hepatoblastoma. METHODS AHEP0731 enrolled children with hepatoblastoma between 2009 and 2018. Surgical guidelines were based upon radiographic extent of disease (PRETEXT, POST-TEXT). Planned non-real-time central review of imaging was completed at diagnosis and after two, four, and six cycles of chemotherapy to determine guideline adherence. Resection rate, event-free survival (EFS), overall survival (OS), and complications were compared between guideline adherent and nonadherent groups. RESULTS Of 226 enrolled patients, 221 were evaluable of whom 205 (93%) underwent definitive surgical resection. Guideline adherence for protocol recommended timing of resection was 65% (133/205). The nonadherent group included 44/205 (21%) patients who underwent early resection and 28/205 (14%) patients who underwent late resection or late transplantation. Eighty-five patients met criteria for early referral to a liver transplant center, of whom 55 (65%) were referred within the recommended timeframe. Twelve patients received liver transplants outside of surgical guidelines. There were no significant differences in EFS, OS, or complication rates between adherent and nonadherent groups. CONCLUSIONS Implementation of surgical guidelines, in combination with effective chemotherapy and radiologic staging, produced a higher rate of definitive tumor resection compared to historical trials. Nonadherence with surgical guidelines resulted in liver transplantation in some patients for whom partial hepatectomy might have been an option.