Ivosidenib in treatment of patients with cholangiocarcinoma with IDH1 mutation — real-world data from a multicenter study

Introduction. Cholangiocarcinoma, especially in the advanced stage, is a disease with poor prognosis despite significant progress in the treatment of cancer patients in recent years. Using new therapies, such as immunotherapy and molecular targeted therapy, may improve the prognosis in this group of patients. This publication presents a multicenter analysis of a Polish research group, including ivosidenib in the treatment of patients with cholangiocarcinoma with IDH1 mutation in the second-fourth lines in salvage access to drug therapy (RDTL, ratunkowy dostęp do technologii lekowych) . Material and methods. The study included patients with unresectable or metastatic CCA with an IDH1 mutation who started treatment with ivosidenib between 01/08/2023 and 31/07/2024 within the RDTL program at ten tertiary oncology centers in Poland. Results. The study included 13 patients with unresectable or metastatic cholangiocarcinoma with IDH1 mutation, who were treated with ivosidenib, including second-line patients — 2 patients (15%), third-line patients — 10 patients (77%), and fourth-line patients — 1 patient (8%). The estimated median PFS and OS rates from the start of ivosidenib treatment were 2.5 and 6.3 months, respectively, while the median OS rate from the start of systemic therapy for the study group was 20 months. The median duration of treatment with ivosidenib was 2.6 months (range 1.0–13.9 months), and 4 (31%) patients continued therapy. Grade 3 adverse events during ivosidenib therapy were observed in 3 (23%) patients. There were no grade 4 adverse events or deaths related to ivosidenib. Conclusions. The study confirmed the efficacy and safety of ivosidenib in patients with advanced cholangiocarcinoma with IDH1 mutation.