Digestive Molecular Clinical Oncology Research Unit, Università degli Studi di Verona, Verona, Italy.
Medical Oncology Unit, Santa Chiara Hospital, Trento, Italy.
Future Oncol. 2021 Feb;17(4):389-402. doi: 10.2217/fon-2020-0726. Epub 2020 Oct 9.
The prognosis of patients affected by cholangiocarcinoma is classically poor. Until recently, chemotherapeutic drugs were the only systemic treatment option available, leading to an overall survival lower than 1 year. In recent decades, different genetic alterations have been identified as playing a key role in the oncogenic signaling. A subgroup of intrahepatic cholangiocarcinoma is characterized by family mutations, more frequently represented by gene fusions of . Based on the results of FIGHT-202 trial, in April 2020 the US FDA approved the FGFR inhibitor pemigatinib in advanced previously treated cholangiocarcinoma patients with rearrangements, opening the way to targeted therapy in this disease. This review summarizes the body of evidence about the efficacy of pemigatinib in cholangiocarcinoma.
胆管癌患者的预后通常较差。直到最近，化疗药物一直是唯一可用的全身性治疗选择，导致总体生存率低于 1 年。近几十年来，不同的基因改变已被确定为致癌信号传导中的关键作用。肝内胆管癌的亚组特征是 家族突变，更常表现为 基因融合。基于 FIGHT-202 试验的结果，美国食品药品监督管理局于 2020 年 4 月批准了 FGFR 抑制剂 pemigatinib 用于先前接受治疗的伴有 重排的晚期胆管癌患者，为该疾病的靶向治疗开辟了道路。这篇综述总结了关于 pemigatinib 在胆管癌中的疗效的证据。
