ABSTRACT Background the main secondary objectives were overall survival (OS), disease control rate (DCR), overall response rate (ORR) and safety. As a pre‐planned exploratory objective, mPFS and OS of second‐line ivosidenib and FOLFOX/CAPOX were compared by means of inverse probability of treatment weights (IPTW)‐adjusted analysis. Results The study included 46 patients treated with Ivosidenib; 43.5% received ivosidenib as second line and 56.5% as ≥ third line. Median PFS and OS were 3.7 (95% CI, 2.2–36.5) and 11.5 months (95% CI, 9.5–36.5). DCR was 50.0%. Grade ≥ 3 adverse events occurred in 8.7% of patients. IPTW‐adjusted mPFS was 6.9 months with ivosidenib and 2.1 months with FOLFOX/CAPOX (HR: 0.36, 95% CI, 0.20–0.64, p = 0.0005), while the mOS was 15.9 and 9.0 months with ivosidenib and FOLFOX/CAPOX, respectively (HR: 0.47, 95% CI, 0.23–0.96, p = 0.0405). Conclusion This study suggests that ivosidenib is a valid option for patients affected by metastatic IDH1 mutant CCA after at least one line of standard treatment.
Niger et al. (Tue,) studied this question.