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Updated: Jul 16, 2025
Treatment of Liver Metastases Using an Internal Target Volume Method for Stereotactic Body Radiotherapy
Published on: May 8, 2018
Real world evidence reveals improved survival outcomes in biliary tract cancer through molecular matched targeted
Bernhard Doleschal1, Hossein Taghizadeh2, Gerald Webersinke3
1Department of Internal Medicine I for Hematology with Stem Cell Transplantation, Hemostaseology, and Medical Oncology, Ordensklinikum Linz, Seilerstaette 4, 4010, Linz, Austria. bernhard.doleschal@ordensklinikum.at.
Abstract:
Biliary tract cancers are rare cancers with poor prognosis due to a lack of therapeutic options, especially after the failure of first-line systemic treatment. Targeted treatments for this clinical situation are promising and have entered clinical practice. We aimed to describe the overall survival of matched targeted treatment after first-line treatment in patients with biliary tract cancers in an Austrian real-world multicenter cohort. We performed a multicenter retrospective chart review of patients with biliary tract cancer between September 2015 and January 2022. Data, including comprehensive molecular characteristics-next generation sequencing (NGS) and immunohistochemistry (IHC), clinical history, surgical procedures, ablative treatments, patient history, and systemic chemotherapy, were extracted from the records of the participating institutions. Targeted treatment was matched according to the ESMO scale for the clinical actionability of molecular targets (ESCAT). We identified 159 patients with the available molecular characteristics. A total of 79 patients underwent second-line treatment. Of these, 36 patients received matched targeted treatment beyond the first-line and were compared with 43 patients treated with cytotoxic chemotherapy in terms of efficacy outcomes. For Tier I/II alterations, we observed a progression free survival ratio (PFStargeted/PFSpre-chemotherapy) of 1.86, p = 0.059. The overall survival for patients receiving at least two lines of systemic treatment significantly favored the targeted approach, with an overall survival of 22.3 months (95% CI 14.7-29.3) vs. 17.5 months (95% CI 1.7-19.8; p = 0.048). Our results underscore the value of targeted treatment approaches based on extended molecular characterization of biliary tract cancer to improve clinical outcomes.
Insights
Targeted therapies improve survival in biliary tract cancers after initial treatment failure. Molecular profiling guides effective matched treatments, showing superior overall survival compared to chemotherapy in a real-world Austrian cohort.
Area of Science:
- Oncology
- Genetics
- Clinical Medicine
Background:
- Biliary tract cancers (BTC) have a poor prognosis and limited treatment options post-first-line therapy.
- Targeted treatments offer a promising avenue for managing advanced BTC.
- Real-world data is crucial for evaluating treatment efficacy in diverse patient populations.
Purpose of the Study:
- To evaluate the overall survival of patients with biliary tract cancer receiving matched targeted therapy after first-line treatment.
- To compare the efficacy of targeted treatments versus cytotoxic chemotherapy in a real-world Austrian multicenter cohort.
- To assess the impact of molecular profiling on treatment outcomes in BTC.
Main Methods:
- Retrospective chart review of 159 biliary tract cancer patients (Sept 2015 - Jan 2022).
- Comprehensive molecular profiling using next-generation sequencing (NGS) and immunohistochemistry (IHC).
- Targeted treatments were selected based on ESMO Scale for Clinical Actionability of Molecular Targets (ESCAT); 36 patients received targeted therapy, 43 received chemotherapy for second-line treatment.
Main Results:
- Patients receiving targeted therapy after first-line treatment showed significantly improved overall survival (22.3 months) compared to those receiving cytotoxic chemotherapy (17.5 months; p=0.048).
- A trend towards improved progression-free survival was observed for targeted therapy in Tier I/II alterations (PFS ratio 1.86, p=0.059).
- Molecular characterization enabled the identification of actionable targets for personalized treatment strategies.
Conclusions:
- Matched targeted treatment based on comprehensive molecular characterization significantly improves overall survival in biliary tract cancer patients post-first-line therapy.
- Extended molecular profiling is valuable for guiding effective treatment decisions in advanced BTC.
- Real-world evidence supports the integration of targeted therapies into the management of biliary tract cancers.
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