肝纤维板层癌治疗的当前进展
Current Advances in the Treatment of Fibrolamellar Carcinoma of Liver.
作者信息
Gummadi Jyotsna, Wang Xin, Xie Changqing
机构信息
Department of Medicine, MedStar Franklin Square Medical Center, Baltimore, MD, 21237, USA.
Thoracic and GI Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, 20814, USA.
出版信息
J Hepatocell Carcinoma. 2023 May 15;10:745-752. doi: 10.2147/JHC.S406902. eCollection 2023.
Abstract
Fibrolamellar carcinoma (FLC) of the liver is a rare type of liver cancer that is prevalent in children and young adults, often less than 40 years old. The etiology is unclear. It presents without underlying liver disease with distinctive histological features such as fibrous collagen bands surrounding the tumor cells. Fusion protein DNAJB1-PRKACA is found in most of the cases. The prognosis of FLC is poor. Even though curative treatment option is surgery for a certain patient population, other treatment modalities including radiation, chemotherapy are currently being used without significant improvement of overall survival. Recently, targeted therapy and immunotherapy have been studied which may provide survival advantage in the future. This review sought to compile data from clinical trials and case reports/series to outline the current state of FLC treatment.
摘要
肝纤维板层癌（FLC）是一种罕见的肝癌类型，在儿童和年轻人中较为常见，通常年龄小于40岁。病因尚不清楚。它在无潜在肝脏疾病的情况下出现，具有独特的组织学特征，如肿瘤细胞周围有纤维胶原带。大多数病例中可发现融合蛋白DNAJB1-PRKACA。FLC的预后较差。尽管对于特定患者群体，治愈性治疗选择是手术，但目前正在使用包括放疗、化疗在内的其他治疗方式，总体生存率并无显著改善。最近，靶向治疗和免疫治疗已在研究中，未来可能会带来生存优势。本综述旨在汇总临床试验和病例报告/系列的数据，以概述FLC治疗的现状。
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2
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