Department of Medical Oncology, St. James's Hospital, Dublin 8, Ireland.
Expert Rev Gastroenterol Hepatol. 2021 May;15(5):475-481. doi: 10.1080/17474124.2021.1915765. Epub 2021 May 18.
: Cholangiocarcinomas (CCAs) are associated with poor survival outcomes, with limited treatment options in the unresectable or metastatic setting. A precision medicine approach to cancer treatment has revealed new therapeutic options that provide an alternative to traditional chemotherapeutic strategies. Isocitrate dehydrogenase 1 () mutations are identified in approximately 10-15% of CCAs and may be targeted by ivosidenib, an oral selective inhibitor of mutant .: This review will discuss the pathogenesis of mutant CCA and the role of ivosidenib in patients with mutant CCA. Topics to be covered include the pharmacology, safety and clinical efficacy of ivosidenib in this patient population.: Ivosidenib represents a promising treatment option for patients with mutant CCA with a favorable side effect profile. Future studies will guide whether this targeted agent may be utilized in combination with other anticancer treatments to improve upon survival outcomes in advanced CCA.
胆管癌（CCA）患者的预后较差，在不可切除或转移性情况下治疗选择有限。癌症治疗的精准医疗方法揭示了新的治疗选择，为传统化疗策略提供了替代方案。约 10-15%的 CCA 存在异柠檬酸脱氢酶 1（IDH1）突变，可被ivosidenib 靶向，ivosidenib 是一种突变 IDH1 的口服选择性抑制剂。本综述将讨论突变型 CCA 的发病机制以及ivosidenib 在突变型 CCA 患者中的作用。涵盖的主题包括ivosidenib 在该患者人群中的药理学、安全性和临床疗效。Ivosidenib 为 IDH1 突变型 CCA 患者提供了一种有前景的治疗选择，具有良好的副作用特征。未来的研究将指导该靶向药物是否可以与其他抗癌治疗联合使用，以改善晚期 CCA 的生存结果。
