Clinical practice and implications of biomarker testing in biliary tract cancer: An observational study

Welland, Sabrina and Zoller, Ann-Kristin and Mavroeidi, Ilektra A. and Tomczak, Aurelie and Muller, Christian and Yawen, Dong and Zhang, Danmei and Keil, Felix and Pangerl, Maria and Zhou, Taotao and Taghizadeh, Hossein and Lange, Sebastian and Kinzler, Maximilian N. and Shmanko, Kataryna and Barsch, Maryam and Zimpel, Carolin and Djanani, Angela and Schulze-Bergkamen, Henning and Keyl, Julius and Lüke, Florian and Wirth, Thomas and Dill, Michael and Longerich, Thomas and Petschnak, Sophia and Marquardt, Jens U. and Quante, Michael and Weinmann, Arndt and Walter, Dirk and Pfarr, Nicole and Prager, Gerald and Doleschal, Bernhard and Gonzalez-Carmona, Maria A. and Gunther, Rainer and Scheiter, Alexander and Bock, Stefan and Bartels, Stephan and Gruenberger, Thomas and Venerito, Marino and Springfeld, Christoph and Kasper, Stefan and Saborowski, Anna and Vogel, Arndt (2026) Clinical practice and implications of biomarker testing in biliary tract cancer: An observational study. JHEP REPORTS, 8 (1): 101635. ISSN , 2589-5559

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Abstract

Background & Aims: Biliary tract cancers (BTC) are aggressive malignancies with limited treatment options. Owing to the high frequency of actionable genomic alterations (GA) and the availability of targeted therapies, molecular testing has become increasingly important; however, its clinical implementation remains inconsistent. This study aimed to evaluate real-world molecular testing practices, characterize the BTC molecular landscape, and assess the prognostic and predictive relevance of selected GA. Methods: We retrospectively analyzed genomic and clinical data from 1,521 patients treated at 18 centers in Germany and Austria. A side-by-side comparison of clinical grade reports generated on two different sequencing platforms was performed for 90 patients. Results: Twenty-four different NGS panels were used across 18 centers. A comparative analysis highlighted the significant variability in reports used to inform therapeutic decisions in clinical practice. Although there were substantial differences in the number of GA covered, the broader panels identified a similar number of actionable GA, indicating that key therapeutic targets are sufficiently represented. Integration with clinical data suggested that certain GA, such as HER2 amplifications (3%), BRAFV600E mutations (2%), and FGFR2 alterations (14%), may have prognostic significance beyond their predictive value. Patients with actionable alterations (610, 40%) that were treated accordingly (n = 204, 13%) had prolonged overall survival (31.8 months vs. 22.8 months, p <0.01). Conclusion: Standardized biomarker testing is crucial for effective integration of targeted therapies in the management of BTC. Our findings reinforce the value of targeted treatments and underscore the predictive and prognostic significance of selected GA.

Item Type: Article
Uncontrolled Keywords: HER2; panel sequencing; molecular targeted therapy; precision oncology; next-generation sequencing; biopsy
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie)
Medicine > Lehrstuhl für Pathologie
Depositing User: Dr. Gernot Deinzer
Date Deposited: 02 Apr 2026 04:32
Last Modified: 02 Apr 2026 04:32
URI: https://pred.uni-regensburg.de/id/eprint/67720

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