Ivosidenib in the treatment of IDH1-mutated cholangiocarcinoma –⁠ a review of current evidence and case report demonstrating durable disease control

Czech version

Authors: MUDr. Michal Eid, Ph.D. ^1,2; MUDr. Udmila Eidová, Ph.D. ^1,3
Authors‘ workplace: Interní hematologická a onkologická klinika LF MU a FN Brno ¹; Centrum precizní medicíny, FN Brno ²; Onkologická klinika 1. LF a VFN v Praze ³
Published in: Klin Onkol 2026; 39(5): 356-363
Category: Case Report
doi: https://doi.org/10.48095/ccko2026356

Overview

Background: Cholangiocarcinoma (CCA) is one of the malignancies in which molecular profiling has substantially expanded the therapeutic landscape and facilitated the implementation of precision oncology into routine clinical practice. Potentially actionable molecular alterations are identified in approximately 40% of patients, with mutations in the isocitrate dehydrogenase 1 (IDH1) gene representing one of the most common and best-characterized molecular alterations in CCA. Aim: The aim is to summarize current evidence regarding the efficacy and safety of ivosidenib in patients with advanced IDH1-mutated CCA and to present the case of a patient who achieved exceptionally durable disease control despite initially facing substantial barriers to treatment access. Results: In the phase III ClarIDHy trial, ivosidenib demonstrated statistically significant improvement in progression-free survival in patients with previously treated IDH1-mutated CCA. The final overall survival analysis, adjusted for crossover, also confirmed an overall survival benefit. These findings have subsequently been supported by multiple prospective and retrospective real-world studies demonstrating comparable efficacy, high disease control rates, and a favorable safety profile. The presented case illustrates durable disease control lasting more than 25 months during ivosidenib treatment while maintaining an excellent quality of life. Conclusion: Ivosidenib represents the standard treatment option in the second and subsequent lines of therapy for advanced CCA harboring an IDH1 R132 mutation. Optimal patient management requires early identification of this molecular alteration through comprehensive molecular profiling and removal of barriers that continue to limit timely access to targeted therapy for eligible patients.

Keywords:

cholangiocarcinoma – ivosidenib – NGS – precision oncology – IDH1

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