First targeted therapy for cholangiocarcinoma shows clinical benefit in phase III trial
“The ClarIDHy study demonstrates for the first time the feasibility and clinical benefit of targeting a molecularly defined subgroup in cholangiocarcinoma. It shows that targeting mutated IDH1 with ivosidenib significantly improves progression-free survival and gives a favourable trend in overall survival in patients with advanced IDH1-mutated cholangiocarcinoma,”
“The findings mean all patients with cholangiocarcinoma should be tested for IDH-1 mutation. Tumour mutation profiling should be a new standard for the care for patients with this heterogeneous tumour type,”
The study randomised 185 patients with advanced cholangiocarcinoma and IDH1 mutations to ivosidenib or matched placebo. Patients could crossover from placebo to ivosidenib when their disease progressed.
Median progression-free survival was 2.7 months for patients treated with ivosidenib compared to 1.4 months with placebo (hazard ratio [HR] 0.37; 95% confidence interval [CI]: 0.25, 0.54, p<0.001). The median progression-free survival rate at six months was 32.0% with ivosidenib, while no patients randomised to placebo were free from progression at this timepoint.
Results showed a favourable trend in overall survival with ivosidenib. Median overall survival was 10.8 months for ivosidenib and 9.7 months for placebo (HR 0.69, one-sided p=0.06). Adjusting the overall survival results to take account of 57% of placebo patients crossing over to ivosidenib gave an adjusted overall survival of 6 months for placebo, which was significantly shorter than with ivosidenib (HR 0.46, p=0.0008).
Ivosidenib was generally well tolerated, with Grade 3 or higher adverse events reported in 46% of patients on the targeted agent and 36% of those on placebo. There were no treatment-related deaths.
https://www.eurekalert.org/pub_releases/2019-09/esfm-ftt092419.php
Konkurrent?