ABSTRACT

Introduction: 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 (IDH1) mutations are identified in approximately 10–15% of CCAs and may be targeted by ivosidenib, an oral selective inhibitor of mutant IDH1.

Areas covered: This review will discuss the pathogenesis of IDH1 mutant CCA and the role of ivosidenib in patients with IDH1 mutant CCA. Topics to be covered include the pharmacology, safety and clinical efficacy of ivosidenib in this patient population.

Expert opinion: Ivosidenib represents a promising treatment option for patients with IDH1 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.

摘要

简介：胆管癌 (CCA) 与较差的生存结果相关，在不可切除或转移的情况下治疗选择有限。癌症治疗的精准医学方法揭示了新的治疗选择，为传统的化疗策略提供了替代方案。异柠檬酸脱氢酶 1 ( IDH1 ) 突变在大约 10-15% 的 CCA 中被发现，并且可能被 ivosidenib（一种突变IDH1的口服选择性抑制剂）靶向。

涵盖领域：本综述将讨论IDH1突变 CCA的发病机制以及 ivosidenib 在IDH1突变 CCA患者中的作用。涵盖的主题包括 ivosidenib 在该患者群体中的药理学、安全性和临床疗效。

专家意见： Ivosidenib 是IDH1突变 CCA患者的一种有前景的治疗选择，具有良好的副作用。未来的研究将指导这种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。