Ivo the great.
IDH1 mutations are reported in 13% of patients with cholangiocarcinoma, a disease with a dismal prognosis. Ivosidenib is an oral targeted inhibitor of IDH1, and this impressive international effort actually pulled off a large phase 3 trial of this therapy specifically for patients with IDH1-mutated cholangio progressing on prior therapy. Among 187 patients, the half who received ivosidenib achieved double the median survival (10 months) than those who received placebo (5 months) when accounting for crossover. What’s more, serious treatment-related adverse events occurred in only 3 patients with no decline in quality of life. | Zhu, JAMA Oncol 2021