Clinical practice and implications of biomarker testing in biliary tract cancer: An observational study
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Le résumé fourni par la source
Background & Aims Biliary tract cancers (BTC) comprise a group of aggressive malignancies with limited therapeutic options. Owing to the high frequency of actionable genomic alterations (GA) and the availability of targeted therapies, molecular testing has gained increasing importance; however, its implementation in clinical practice varies across centers. The aim of our analysis was to provide a comprehensive real-world assessment of molecular testing, annotate the molecular landscape in BTC patients, and understand the prognostic and predictive implications of selected GA. Methods We retrospectively analyzed genomic and clinical information of 1521 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 24 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%) , BRAF V600E 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 mo vs 22.8 mo, 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 1521 BTC patients 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 NGS-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.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Clinical practice and implications of biomarker testing in biliary tract cancer: An observational study
- Date Crossref
- 01/01/2026
- Éditeur
- Elsevier BV
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Medizinische Hochschule Hannover pays non établi dans la noticeUniversité ou école supérieure
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Essen University Hospital pays non établi dans la noticeOrganisme public
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Heidelberg University Institute of Pathology pays non établi dans la noticeUniversité ou école supérieure
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University Hospital Heidelberg pays non établi dans la noticeÉtablissement de santé
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University Hospital Magdeburg Department of Gastroenterology pays non établi dans la noticeÉtablissement de santé
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Sigmund Freud Privatuniversität Wien pays non établi dans la noticeUniversité ou école supérieure
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Ludwig-Maximilians-Universität München pays non établi dans la noticeUniversité ou école supérieure
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University of Regensburg Institute of Pathology pays non établi dans la noticeUniversité ou école supérieure
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University Hospital Bonn pays non établi dans la noticeÉtablissement de santé
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Universitätsklinikum St. Pölten pays non établi dans la noticeÉtablissement de santé
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Karl Landsteiner University of Health Sciences Department of Internal Medicine 2 pays non établi dans la noticeUniversité ou école supérieure
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Technical University of Munich pays non établi dans la noticeUniversité ou école supérieure
Medizinische Hochschule Hannover, Essen University Hospital et Institute of Pathology — Heidelberg University, avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.