Real-world Analysis of Treatment Patterns, Clinical Outcomes, and Molecular Profiling in Advanced Biliary Tract...

Area of Science:

• Medical Oncology
• Genomics
• Precision Medicine

Background:

• Limited real-world data exists on immunotherapy and targeted agents for advanced biliary tract cancer (BTC).
• Treatment patterns, outcomes, and molecular profiles in advanced BTC are evolving.

Purpose of the Study:

• To evaluate treatment patterns and outcomes in advanced BTC.
• To analyze molecular characteristics of advanced BTC.
• To assess the efficacy of immunotherapy and targeted agents in advanced BTC.

Main Methods:

• Retrospective analysis of advanced BTC patients receiving palliative systemic therapy.
• Genomic profiling using next-generation sequencing (NGS).
• Actionable alterations defined by ESMO Scale for Clinical Actionability of molecular Targets (ESCAT) tier I.

Main Results:

• 85 patients analyzed; 65.9% received immunotherapy, 44.7% received first-line chemoimmunotherapy.
• NGS identified actionable genomic alterations (ESCAT tier I) in 8 patients, including ERBB2 amplification and IDH1 mutations.
• Matched targeted therapy in the second-line setting significantly improved progression-free survival (PFS) and overall survival (OS) compared to conventional chemotherapy (median PFS: 9.7 vs. 2.1 months; median OS: 27.7 vs. 11.0 months).

Conclusions:

• Integration of NGS and matched targeted therapy significantly improved survival outcomes in selected advanced BTC patients.
• Precision medicine strategies are crucial for improving outcomes in advanced BTC.
• Genomic profiling aids in identifying patients who benefit from targeted therapies.