Background: Treatment options for cholangiocarcinoma (CCA) are limited, with poor overall survival (OS). The impact of molecular alterations on clinical outcomes in real-world practice is poorly understood. Patients and methods: EvIDHence was a retrospective, descriptive, multicenter, international, chart review study investigating the natural disease course of patients with advanced/metastatic CCA who underwent mutant isocitrate dehydrogenase-1 (mIDH1) testing and initiated second-line treatment on or before 1 July 2019 until 31 December 2021, before commercial availability of mIDH1-targeted therapy. Outcomes included patient, disease, and treatment characteristics, biomarker testing and survival. Results: Sixty-three patients with CCA (31 mIDH1; 32 IDH1 wild-type) were included. There were no differences in patient and disease characteristics between mIDH1 and IDH1 wild-type groups. Biomarker testing was carried out in the context of research/clinical trials in 61.9% of patients. The most frequent first- and second-line systemic regimens were gemcitabine plus cisplatin and FOLFOX, respectively. Median OS from first-line treatment initiation was 20.8 [95% confidence interval (CI) 15.1-29.3] and 18.6 (95% CI 13.0-20.5) months for mIDH1 and IDH1 wild-type groups, respectively. Median OS from second-line treatment initiation was 9.7 (95% CI 5.5-11.2) and 8.5 (95% CI 6.5-11.3) months for patients with mIDH1 and IDH1 wild-type CCA, respectively. Conclusion: These real-world data highlight the poor prognosis of patients with CCA, regardless of IDH1 mutational status. Limitations included the small sample size and biased selection of patients still fit for second-line therapy.
The EvIDHence study: real-world treatment patterns and safety outcomes in mutant and wild-type IDH1 cholangiocarcinoma before availability of mIDH1 inhibitors
Rimassa, L.
2026-01-01
Abstract
Background: Treatment options for cholangiocarcinoma (CCA) are limited, with poor overall survival (OS). The impact of molecular alterations on clinical outcomes in real-world practice is poorly understood. Patients and methods: EvIDHence was a retrospective, descriptive, multicenter, international, chart review study investigating the natural disease course of patients with advanced/metastatic CCA who underwent mutant isocitrate dehydrogenase-1 (mIDH1) testing and initiated second-line treatment on or before 1 July 2019 until 31 December 2021, before commercial availability of mIDH1-targeted therapy. Outcomes included patient, disease, and treatment characteristics, biomarker testing and survival. Results: Sixty-three patients with CCA (31 mIDH1; 32 IDH1 wild-type) were included. There were no differences in patient and disease characteristics between mIDH1 and IDH1 wild-type groups. Biomarker testing was carried out in the context of research/clinical trials in 61.9% of patients. The most frequent first- and second-line systemic regimens were gemcitabine plus cisplatin and FOLFOX, respectively. Median OS from first-line treatment initiation was 20.8 [95% confidence interval (CI) 15.1-29.3] and 18.6 (95% CI 13.0-20.5) months for mIDH1 and IDH1 wild-type groups, respectively. Median OS from second-line treatment initiation was 9.7 (95% CI 5.5-11.2) and 8.5 (95% CI 6.5-11.3) months for patients with mIDH1 and IDH1 wild-type CCA, respectively. Conclusion: These real-world data highlight the poor prognosis of patients with CCA, regardless of IDH1 mutational status. Limitations included the small sample size and biased selection of patients still fit for second-line therapy.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
