Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study

RCT (n=185) reported improved progression-free survival with ivosidenib, a small-molecule targeted inhibitor of mutated IDH1, compared with placebo, (median 2.7 vs 1.4 months; HR 0.37; 95% CI 0.25–0.54; p<0.0001) in patients with IDH1-mutant cholangiocarcinoma

SPS commentary:

A commentary notes that ancillary studies are eagerly awaited, which will increase knowledge and identify biomarkers and resistance mechanisms mediating IDH inhibition. Future developments investigating IDH inhibitors in biliary tract carcinoma are exploring combinations of IDH inhibitors with chemotherapy in a first-line, metastatic setting or their use as maintenance therapy after a chemotherapy induction treatment. It adds that this current study reinforces the need for genetic testing for all patients with biliary tract carcinoma, especially intrahepatic cholangiocarcinoma, to identify candidates for personalised treatment.


The Lancet Oncology

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