Abstract Background Cholangiocarcinoma (CCA) is the second most common liver malignancy after hepatocellular carcinoma and is difficult to treat with high rates of metastasis. The isocitrate dehydrogenase‐1 (IDH1) inhibitor ivosidenib has emerged as a promising second‐line therapy for CCA patients with IDH1 mutations. Methods We conducted a systematic review and meta‐analysis to examine ivosidenib's impact on patient survival in advanced‐stage CCA patients, following the Preferred Reporting Items for Systematic Reviews and Meta‐analyses guidelines. Results A total of 317 patients across four studies were analysed in this review, with 256 ivosidenib‐treated patients and 61 placebo‐treated patients. Almost all patients have intrahepatic CCA. Ivosidenib was found to improve advanced CCA patient outcomes in median progression‐free survival (PFS) (Range: 2.7‐4.4 months), median overall survival (OS) (Range: 10.3–15 months), objective response rate (ORR) (Range: 2.4%–18.2%), and DCR (Range: 50.0%–63.6%), compared to outcomes in placebo‐treated advanced CCA (PFS = 1.4 months, OS = 7.5 months, ORR = 0% and DCR = 27.9%). Using individual‐level data and a random effects model, patient survival was calculated with a hazard ratio of 0.29 (95% confidence interval 0.21–0.42, p = 5.6 × 10 − 12). Common adverse events include nausea, vomiting, and abdominal pain, but QT prolongation was observed in between 5.5% and 9.1% of patients. Conclusion Our analysis suggests that pooled ivosidenib‐treated patients with advanced CCA possess better PFS and OS compared to placebo‐treated patients. However, studies combining ivosidenib with checkpoint blockade immunotherapy drugs or other targeted therapies have yet to be conducted.
Wong et al. (Wed,) studied this question.
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