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Published online: 2023-12-22

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Ivosidenib in the treatment of patients with IDH1 mutated cholangiocarcinoma

Agnieszka Chmielewska1, Tomasz Kubiatowski12


Metastatic cholangiocarcinoma (CCA) has poor prognosis. Chemotherapy in this indication demonstrated a limited benefit. Adding immunotherapy to standard chemotherapy is associated with an increase in median progressio.- free survival (mPFS) and increase in the response rate. An interesting therapeutic option for patients who have experienced progression during first line of treatment are targeted therapies. It is estimated that in over 50% of patients with CCA molecular tests based on next-generation sequencing (NGS) make it possible to identify genomic disorders, potentially enabling the use of targeted treatment. The most frequently reported disorders are fusions in the gene encoding fibroblast growth factor receptor 2 (FGFR2) and mutations in the IDH1 gene encoding isocitrate dehydrogenase (IDH). Ivosidenib is an oral reversible inhibitor of the abnormal form of IDH1 enzyme. The use of ivosidenib in patients with CCA and the IDH1 gene mutation after failure of previous therapy was evaluated in a phase III ClarIDHy study. The results of the trial confirmed its beneficial effect in terms of both PFS and overall survival (OS).

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