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肝母细胞瘤:从分子机制到治疗策略

Hepatoblastoma: From Molecular Mechanisms to Therapeutic Strategies.

作者信息

Fan Ling, Na Jintong, Shi Tieliu, Liao Yuan

机构信息

State Key Laboratory of Targeting Oncology, Guangxi Medical University, Nanning 530021, China.

National Center for International Research of Bio-Targeting Theranostics, Guangxi Medical University, Nanning 530021, China.

出版信息

Curr Oncol. 2025 Mar 4;32(3):149. doi: 10.3390/curroncol32030149.

Abstract

Hepatoblastoma (HB) is the most common malignant liver tumor in children under five years of age. Although globally rare, it accounts for a large proportion of liver cancer in children and has poor survival rates in high-risk and metastatic cases. This review discusses the molecular mechanisms, diagnostic methods, and therapeutic strategies of HB. Mutations in the CTNNB1 gene and the activation of the Wnt/β-catenin pathway are essential genetic factors. Furthermore, genetic syndromes like Beckwith-Wiedemann syndrome (BWS) and Familial Adenomatous Polyposis (FAP) considerably heighten the risk of associated conditions. Additionally, epigenetic mechanisms, such as DNA methylation and the influence of non-coding RNAs (ncRNAs), are pivotal drivers of tumor development. Diagnostics include serum biomarkers, immunohistochemistry (IHC), and imaging techniques. Standard treatments are chemotherapy, surgical resection, and liver transplantation (LT). Emerging therapies like immunotherapy and targeted treatments offer hope against chemotherapy resistance. Future research will prioritize personalized medicine, novel biomarkers, and molecular-targeted therapies to improve survival outcomes.

摘要

肝母细胞瘤(HB)是5岁以下儿童最常见的肝脏恶性肿瘤。尽管在全球范围内较为罕见,但它在儿童肝癌中占比很大,并且在高危和转移病例中的生存率较低。本综述讨论了HB的分子机制、诊断方法和治疗策略。CTNNB1基因的突变以及Wnt/β-连环蛋白通路的激活是重要的遗传因素。此外,诸如贝克威思-维德曼综合征(BWS)和家族性腺瘤性息肉病(FAP)等遗传综合征会显著增加相关疾病的风险。另外,表观遗传机制,如DNA甲基化和非编码RNA(ncRNA)的影响,是肿瘤发展的关键驱动因素。诊断方法包括血清生物标志物、免疫组织化学(IHC)和成像技术。标准治疗方法是化疗、手术切除和肝移植(LT)。免疫疗法和靶向治疗等新兴疗法为克服化疗耐药带来了希望。未来的研究将优先关注个性化医疗、新型生物标志物和分子靶向治疗,以改善生存结果。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22d2/11941340/df7e4167fb7d/curroncol-32-00149-g001.jpg

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