Young Elizabeth P, O'Neill Allison F, Rangaswami Arun A
Department of Pediatrics, Division of Oncology, University of California San Francisco, San Francisco, CA 94158, USA.
Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston Children's Hospital and Harvard Medical School, Boston, MA 02215, USA.
Int J Mol Sci. 2025 Jan 31;26(3):1252. doi: 10.3390/ijms26031252.
Pediatric hepatocellular carcinoma (HCC) is a rare malignant liver tumor affecting children and adolescents and occurring either sporadically or in the context of underlying liver disease. In this review, we detail the epidemiology of pediatric HCC with a focus on predisposing factors including hepatic or systemic disease, genetic disorders, and familial cancer syndromes. We summarize existing research on the pathophysiology of pediatric HCC, including molecular mechanisms of oncogenesis, highlighting unique disease features differentiating pediatric HCC from adult HCC. We then survey the landscape of therapeutic options for pediatric HCC, including novel therapeutics. Lastly, we discuss the pathologic spectrum upon which pediatric HCC is postulated to exist, ranging from hepatoblastoma to HCC and including the hybrid entity hepatocellular neoplasm not otherwise specifed (HCN-NOS). In summary, we highlight the key clinical and molecular features of pediatric HCC that may inform future research and novel approaches to the clinical care of these patients.
小儿肝细胞癌(HCC)是一种罕见的恶性肝脏肿瘤,影响儿童和青少年,可散发性发生或在潜在肝脏疾病背景下出现。在本综述中,我们详细阐述小儿HCC的流行病学,重点关注易感因素,包括肝脏或全身性疾病、遗传疾病和家族性癌症综合征。我们总结了关于小儿HCC病理生理学的现有研究,包括肿瘤发生的分子机制,强调小儿HCC与成人HCC不同的独特疾病特征。然后,我们审视小儿HCC的治疗选择格局,包括新型疗法。最后,我们讨论小儿HCC据推测所存在的病理谱,范围从肝母细胞瘤到HCC,包括未另行指定的混合性实体肝细胞肿瘤(HCN-NOS)。总之,我们强调小儿HCC的关键临床和分子特征,这些特征可能为这些患者未来的研究和临床护理新方法提供信息。