Cholangiocarcinoma is a rare, aggressive malignancy. Standard-of-care treatments for inoperable disease is gemcitabine plus cisplatin. However, no standard second-line treatments are available. IDH1 is mutated in up to 25% of cholangiocarcinoma patients. Ivosidenib is approved for the treatment of IDH1-mutated acute myeloid leukemia and is being investigated in a phase 1 trial in patients with IDH1-mutated solid tumors. The results of the cholangiocarcinoma were reported. According to the trial, 73 patients with IDH1-mutated cholangiocarcinoma were enrolled and received ivosidenib. Median progression free survival (PFS) was 3.8 months (95% CI; 3.6-7.3), 6-month PFS was 40.1% (28.4-51.6) and 12-month PFS was 21.8% (12.3-33.0). Common grade 3 or worse adverse events were ascites (5%) and anaemia (4%); the only treatment-related grade 3 or worse event reported was fatigue, which was reported in two patients (3%).The data suggest that ivosidenib could be a potential treatment option for IDH1-mutated cholangiocarcinoma.
Lancet Gastroenterol Hepatol. 2019 Jul 9. pii: S2468-1253(19)30189-X.