Abstract IA29: Present and future of precision oncology in hepatoblastoma

Abstract Pediatric liver cancer is rare (~1 case/1 million) with increasing incidence. The commonest tumor seen usually in young children and infants without underlying liver disease is hepatoblastoma (HB). Despite significant advances during last decades thanks to improvements in chemotherapeutic regimens and efficient surgical approaches, treatment options remain limited for ~20% of patients with aggressive resistant tumours and more than 40% survivors have long-term effects derived from treatment toxicity. Because of the rarity of the disease, global international efforts on clinical, pathological and biological fields are crucial to improve the current clinical management of HB. In this talk, current clinical patient stratification (Children's Hepatic tumors International Collaboration Hepatoblastoma Stratification, CHIC-HS) and treatment of patients with HB (Pediatric Hepatic International Tumor Trial, PHITT) will be exposed. The main clinical, pathological and biological prognostic factors used at present will be also explained. Since it is well-known that therapy response in oncology is intimately associated with cancer biology, the need to improve the current CHIC stratification by incorporating biological data will be emerged as a way to significant move forward towards precision medicine in pediatric liver cancer patients. Moreover, a summary of the latest advances in genomic, transcriptomic and methylation studies and the diagnostic/prognostic biomarkers as well as newest therapeutic targets/drugs will be exposed. As a rare disease, the unmet need for banking human samples, patient-derived cell lines and animal models to enable and boost translational research of childhood liver tumors will be stressed. Finally, Dr. Armengol will explain the latest advances on biological and pathological studies associated to PHITT in Europe, ranging from sample collection to identification and validation of diagnostic/prognostic biomarkers. The ongoing PHITT patient cohort and its associated biorepositories will be exposed as an exceptional opportunity to validate a panel of diagnostic and prognostic biomarkers and novel therapeutic strategies that could be incorporated in the upcoming clinical trial (PHITT-2). Nowadays, ongoing international efforts at clinical, pathological and biological level from the three main childhood liver study groups of Europe (SIOPEL), United States (COG) and Japan (JCCG) are being conducted and will move forward precision medicine in HB, impacting on the quality of life and survival of patients with HB in a near future. Citation Format: Carolina Armengol. Present and future of precision oncology in hepatoblastoma [abstract]. In: Proceedings of the AACR Special Conference: Advances in the Pathogenesis and Molecular Therapies of Liver Cancer; 2022 May 5-8; Boston, MA. Philadelphia (PA): AACR; Clin Cancer Res 2022;28(17_Suppl):Abstract nr IA29.