A new study published in Therapeutic Advances in Medical Oncology confirmed the results of previous real-world data on the use of ivosidenib in patients with cholangiocarcinoma (CCA) harboring isocitrate dehydrogenase 1 (IDH1) mutations.

“Real-world evidence on larger numbers of patients is needed to confirm our findings,” the authors wrote.

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“Moreover, a deeper knowledge about the molecular and genomic profile would help to understand possible resistance mechanisms, which could underlie, thus suggesting possible new therapeutic strategies for this setting of patients,” they added.

The team led by Andrea Casadei-Gardini, MD, from Vita-Salute University San Raffaele in Milan, Italy, recently published the first data on using ivosidenib in a real-world setting.

The present study provided updated survival results from 11 patients treated with the drug as second-line treatment and third-line treatment for advanced CCA.

They reported that the median progression-free survival of the patients was 4.4 months from the start of the treatment to a median follow-up of 13.7  months. The median overall survival was 15  months, and the disease control rate of the treatment was 63%. A total of 2 patients achieved a partial response, the researchers said. 

Regarding adverse events, 18% of patients experienced at least 1 treatment-related adverse event, but none were grade 3 or more. The most frequent grade 2 adverse events were irregular heart rhythm and hypomagnesemia. 

In most patients (72.7%), the most common coaltered genes were BAP1, TP53, CDKN2B,and CDKN2A.

Ivosidenib, marketed under the brand name Tibsovo®, is a first-in-class targeted IDH1 inhibitor indicated for the treatment of adult patients with CCA carrying a susceptible IDH1 mutation. 

The treatment was approved by the US Food and Drug Administration based on the results of a phase 3 clinical trial called ClarIDHy.


Rimini M, Burgio V, Antonuzzo L, et al. Updated survival outcomes with ivosidenib in patients with previously treated IDH1-mutated intrahepatic-cholangiocarcinoma: an Italian real-world experience. Ther Adv Med Oncol. Published online July 12, 2023. doi:10.1177/17588359231171574