Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, 40138 Bologna, Italy.
Medicina (Kaunas). 2021 May 8;57(5):458. doi: 10.3390/medicina57050458.
Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in approximately 50% of cases, and thus, molecularly targeted therapies have been actively explored in this setting. Among these, fibroblast growth factor receptor (FGFR) inhibitors have reported important results, as witnessed by the FDA approval of pemigatinib in previously treated metastatic CCA patients harboring FGFR2 fusion or other rearrangements. Herein, we provide an overview of available evidence on FGFR inhibitors in CCA, especially focusing on the development, pitfalls and challenges of emerging treatments in this setting.
尽管晚期疾病仍然与严峻的预后相关，但在过去的十年中，已经出现了几种新的胆管癌（CCA）患者治疗选择。事实上，CCA 已经成为一组异质性恶性肿瘤，大约 50%的病例存在潜在可用药的基因突变，因此，分子靶向治疗在这一领域得到了积极探索。其中，成纤维细胞生长因子受体（FGFR）抑制剂已经取得了重要的成果，FDA 批准了 pemigatinib 用于治疗先前治疗过的携带 FGFR2 融合或其他重排的转移性 CCA 患者。本文综述了 CCA 中 FGFR 抑制剂的现有证据，特别关注了这一领域新兴治疗方法的发展、陷阱和挑战。
