Molecular tumor board-guided personalized therapies in biliary tract cancer: a real-world analysis

Introduction: Biliary tract cancer (BTC) represents a major cause of cancer-related mortality worldwide, with a 5-year survival rate of less than 20%. Notably, distinct BTC subtypes, particularly intrahepatic cholangiocarcinoma, exhibit up to 40% actionable targets. This underscores the importance of an early referral to a Molecular Tumor Board (MTB) for the development of individualized, molecular guided therapeutic strategies. This retrospective study aims to evaluate the impact of MTB inclusion, recommended targeted therapies and their subsequent implementation in patients with BTC in a real-world setting.

Methods: We retrospectively analysed clinical and molecular data from 66 patients with BTC who were presented at the MTB of the Comprehensive Cancer Center Freiburg (MTB-FR) between 2019 and 2024. Patient and disease characteristics, molecular profiling results (including whole exome sequencing and targeted next-generation sequencing), MTB therapeutic recommendations, and subsequent treatment implementation were assessed. Clinical outcomes were evaluated relative to prior therapies using the progression-free survival (PFS) ratio.

Results: The majority of patients (98%) were referred to the MTB-FR with advanced-stage disease, having received a median of one prior systemic therapy (range 0–6). Among 66 patient discussions, 56 targeted therapy recommendations were made, of which 18 were implemented. Most recommended regimens involved combinatorial approaches, particularly the combination of immune checkpoint inhibitors with tyrosine kinase inhibitors. Among patients receiving MTB-recommended therapies, four achieved a partial response, four maintained stable disease, and eight exhibited disease progression. In two cases, therapeutic outcomes could not be assessed due to treatment-related toxicity or death. Notably, 55% of evaluable patients achieved a PFS ratio greater than 1.3 compared to their previous therapy, indicating a clinical benefit from MTB-guided interventions.

Conclusion: Implementation of MTB-recommended personalized therapies was associated with clinical benefit in 55% of BTC patients. A major limitation remains the advanced disease stage at the time of MTB presentation, which precluded therapy implementation in approximately one-third of cases. These findings underscore the necessity of early molecular profiling and timely referral to an MTB to increase the feasibility of targeted therapy application and improve outcomes in BTC.

Informationen zum Einsatz von KI: Parts of this text were translated and phrased with the assistance of artificial intelligence

 

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Publication History

Article published online:
04 September 2025