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

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 im...

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Main Authors: Welland Sabrina (Author) , Zöller, Ann-Kristin (Author) , Mavroeidi, Ilektra A. (Author) , Tomczak, Aurelie (Author) , Müller, Christian (Author) , Yawen, Dong (Author) , Zhang, Danmei (Author) , Keil, Felix (Author) , Pangerl, Maria (Author) , Zhou, Taotao (Author) , Taghizadeh, Hossein (Author) , Lange, Sebastian (Author) , Kinzler, Maximilian N. (Author) , Shmanko, Kataryna (Author) , Barsch, Maryam (Author) , Zimpel, Carolin (Author) , Djanani, Angela (Author) , Schulze-Bergkamen, Henning (Author) , Keyl, Julius (Author) , Lüke, Florian (Author) , Wirth, Thomas (Author) , Dill, Michael T. (Author) , Longerich, Thomas (Author) , Petschnak, Sophia (Author) , Marquardt, Jens U. (Author) , Quante, Michael (Author) , Weinmann, Arndt (Author) , Walter, Dirk (Author) , Pfarr, Nicole (Author) , Prager, Gerald (Author) , Doleschal, Bernhard (Author) , Gonzalez-Carmona, Maria A. (Author) , Günther, Rainer (Author) , Scheiter, Alexander (Author) , Böck, Stefan (Author) , Bartels, Stephan (Author) , Gruenberger, Thomas (Author) , Venerito, Marino (Author) , Springfeld, Christoph (Author) , Kasper, Stefan (Author) , Saborowski, Anna (Author) , Vogel, Arndt (Author)
Format: Article (Journal)
Language:English
Published: January 2026
In: JHEP reports
Year: 2026, Volume: 8, Issue: 1, Pages: 1-15
ISSN:2589-5559
DOI:10.1016/j.jhepr.2025.101635
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1016/j.jhepr.2025.101635
Verlag, kostenfrei, Volltext: https://www.sciencedirect.com/science/article/pii/S2589555925003179
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Author Notes:Sabrina Welland, Ann-Kristin Zöller, Ilektra A. Mavroeidi, Aurelie Tomczak, Christian Müller, Dong Yawen, Danmei Zhang, Felix Keil, Maria Pangerl, Taotao Zhou, Hossein Taghizadeh, Sebastian Lange, Maximilian N. Kinzler, Kataryna Shmanko, Maryam Barsch, Carolin Zimpel, Angela Djanani, Henning Schulze-Bergkamen, Julius Keyl, Florian Lüke, Thomas Wirth, Michael Dill, Thomas Longerich, Sophia Petschnak, Jens U. Marquardt, Michael Quante, Arndt Weinmann, Dirk Walter, Nicole Pfarr, Gerald Prager, Bernhard Doleschal, Maria A. Gonzalez-Carmona, Rainer Günther, Alexander Scheiter, Stefan Böck, Stephan Bartels, Thomas Gruenberger, Marino Venerito, Christoph Springfeld, Stefan Kasper, Anna Saborowski, Arndt Vogel
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Summary: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. - Impact and implications - Genomic profiling is recommended in patients with biliary tract cancers (BTC) but lacks harmonization across platforms and centers. By retrospectively analyzing genomic and clinical information from 1,521 patients with BTC diagnosed and treated at 18 centers in Germany and Austria, we provide real-world insights into the implementation of molecular profiling in BTC, highlighting variability in next generation sequencing-based testing and its impact on the detection of genomic alterations. Standardized molecular testing strategies will be key to enable the integration of more consistent and comparable genomic datasets across studies. Further, by elucidating the prognostic relevance of individual genomic alterations, our insights carry significant implications for interpreting single-arm clinical trials within genomically stratified patient cohorts and underscore the importance of randomized studies to delineate the benefit of targeted therapies.
Item Description:Online verfügbar: 4. November 2025, Artikelversion: 4. Dezember 2025
Gesehen am 20.02.2026
Physical Description:Online Resource
ISSN:2589-5559
DOI:10.1016/j.jhepr.2025.101635

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