Ivosidenib for IDH1 Mutant Cholangiocarcinoma: A Narrative Review.

Cholangiocarcinoma is an uncommon gastrointestinal neoplasm characterized by the abnormal proliferation of cholangiocytes within the biliary duct. This type of malignancy can be subdivided into three major classes: intrahepatic cholangiocarcinoma (iCCA), perihilar cholangiocarcinoma (pCCA), and distal cholangiocarcinoma (dCCA). Based on the results of various clinical trials, ivosidenib was approved for acute myeloid leukemia harboring the IDH1 mutation. It has also been shown that ivosidenib was effective in patients with IDH1 mutated cholangiocarcinoma. In this article, we briefly review the genomics and prognosis of cholangiocarcinoma with a special focus on ivosidenib and the mechanisms by which its approval was met.