Real-World Evidence Study Reveals Improved Survival in Biliary Tract Cancer Through Molecular Targeted Treatments

A new study from Bernhard Doleschal, MD, and colleagues aimed to assess the efficacy of targeted treatments for biliary tract cancer (BTC). What they found suggested that molecular profiling in combination with individualized targeted treatments could significantly improve survival outcomes for BTC patients. The study included patients with histologically proven BTC diagnosis who had already undergone molecular profiling of their tumor tissue. The physicians' primarily goal was to assess the overall survival of patients, as well as evaluate patients' response rate and time from progression, failure of first-line treatment, death, or last follow-up.

Dr Doleschal demonstrated that advanced molecular characterization of BTC can potentially double the overall survival of patients when compared with sequential chemotherapy. Specifically, patients who received ESCAT I-IV or NCT m1-m4 alterations matched to targeted drugs compared with those treated with chemotherapy demonstrated improved survival outcomes. The modified progression-free survival ratio (PFS/PFS previous) for ESCAT I and II tiers met the threshold used in precision oncology to determine the benefit of matched targeted treatment, demonstrating the potential of this precision approach.

"Our results underscore the value of comprehensive molecular testing for BTC and reinforce the statement of the ESMO guidelines that every patient with BTC should undergo molecular characterization," wrote the authors.

The study also notes some key limitations. MSI and TMB testing were not performed routinely in every patient and the extent of the NGS panel differed across patients. Although these discrepancies reflect the diagnostic advances in molecular techniques over time and the different availability of diagnostic methods across various centers, it also raises the issue of the importance of standardization for more consistency in patient treatment and outcomes.

In conclusion, the study underscores the importance of comprehensive molecular profiling for BTC and reinforces the move toward precision oncology as a robust and promising therapeutic option. Despite certain limitations and challenges, adopting a molecular-informed treatment approach could lead to significant improvement in survival outcomes for patients with this rare type of cancer. Dr Doleschal and his colleagues recommended further research to study resistance mechanisms to targeted agents and strategies to overcome these limitations for a more effective and optimized treatment approach.

Source:
Doleschal B, Taghizadeh H, Webersinke G, et al. Real world evidence reveals improved survival outcomes in biliary tract cancer through molecular matched targeted treatment. Sci Rep. 2023 Sep 18;13(1):15421. doi:10.1038/s41598-023-42083-4