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Abstract IA28: Advances in the pathogenesis and classification of pediatric hepatocellular neoplasms


 Hepatocellular neoplasms are the most common primary liver malignancies in children, with hepatoblastoma (HB) accounting for more than two-thirds of all cases. HB is a histologically heterogeneous embryonal tumor often recapitulating liver developmental stages, more commonly diagnosed during the first three years of life. HB classification has been historically challenged by its rarity and the lack of a systematic review of diagnostic tumor specimens. Recent international initiatives resulted in the development of international datasets (CHIC) and muti-consortia therapeutic trials (PHITT) that required a new pediatric liver tumors consensus classification. Meanwhile, genomic profiling is quickly advancing our understanding of HB and its biological heterogeneity, and allowing for the identification of biomarkers and biological subgroups of potential clinical relevance. Preliminary biological annotation of the International Consensus Classification, including diagnostic and prognostically relevant tumor subtypes, is ongoing. Molecular profiling of high-risk tumors, such as those in the HCN-NOS provisional category, is revealing histological and molecular features which are important to recognize to correctly diagnose and risk-stratify this group of patients. Current initiatives, including those led by clinical consortia participating in the International Pediatric Liver Tumors Trial (PHITT), are validating potential diagnostic and risk-stratifying biomarkers and engaged in exploratory studies, such genomic tumor profiling and investigation of novel therapeutic strategies, to be considered in upcoming trials.
 Citation Format: Dolores (Lola) Lopez-Terrada. Advances in the pathogenesis and classification of pediatric hepatocellular neoplasms [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 IA28.