Ivosidenib for the treatment of isocitrate dehydrogenase-1 mutant cholangiocarcinoma

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.

Automated summary

Cholangiocarcinomas (CCAs) have poor survival outcomes with limited treatment options. Ivosidenib, a selective inhibitor of mutant IDH1, presents a promising treatment option for patients with IDH1 mutant CCA. Future studies may explore the use of this targeted agent in combination with other anticancer treatments to improve survival outcomes in advanced CCA.