Real-world outcomes with ivosidenib in mIDH1 cholangiocarcinoma

In this video, Dr Amy de Haar-Holleman discusses a real-world chart review study presented at ESMO GI 2026, evaluating ivosidenib as second-line treatment in patients with advanced or metastatic IDH1-mutated cholangiocarcinoma.¹ Ivosidenib had previously shown progression-free and overall survival benefit in the phase III ClarIDHy trial, with a manageable safety profile. This study aimed to assess whether these results are reflected in routine clinical practice. 

The analysis included 55 patients from the United States who received second-line ivosidenib monotherapy after progression on first-line chemotherapy. Most patients had intrahepatic cholangiocarcinoma and were in relatively good clinical condition at treatment initiation. The most common reasons for selecting ivosidenib were its efficacy profile, guideline recommendations and safety/tolerability profile. 

Clinical outcomes were consistent with the benefit observed in ClarIDHy. Based on physician assessment, 27.3% of patients achieved a partial response and 60.0% had stable disease, resulting in disease control in 87.3% of patients. Median overall survival was 18.5 months, and the estimated 12-month overall survival rate was 74.2%. At the time of the chart review, nearly half of patients were still receiving ivosidenib. 

The safety profile was favourable. Most patients did not experience grade ≥3 adverse events, and no adverse events led to treatment discontinuation. Dr de Haar-Holleman notes that, although the retrospective design and small sample size require cautious interpretation, these real-world data support ivosidenib as a valuable treatment option for patients with previously treated IDH1-mutated cholangiocarcinoma.