Zhu AX, Macarulla T, Javle MM, et al. - Researchers investigated overall survival outcomes among patients with chemotherapy-refractory cholangiocarcinoma with IDH1 mutation in correlation with receiving treatment with ivosidenib vs placebo.

• In this multicenter, randomized, double-blind, placebo-controlled, clinical phase 3 trial, 187 previously treated patients with advanced cholangiocarcinoma with IDH1 mutation were included.

• In cases with disease progression as determined by radiographic findings, crossover from placebo to ivosidenib was permitted.

• Numerically improved overall survival benefits were recorded among those receiving ivosidenib treatment vs placebo, despite a high rate of crossover.

• Based on the combined efficacy data and tolerable safety profile, and corroborating quality of life data, researchers support ivosidenib as clinically beneficial when compared with placebo in cholangiocarcinoma with IDH1 mutation.