Molecular Profiling-Selected Therapy for Treatment of Advanced Pancreaticobiliary Cancer: A Retrospective Multicenter Study

Abstract EN

This multicenter cohort study assessed the impact of molecular profiling (MP) on advanced pancreaticobiliary cancer (PBC).

The study included 30 patients treated with MP-guided therapy after failing ≥1 therapy for advanced PBC.

Treatment was considered as having benefit for the patient if the ratio between the longest progression-free survival (PFS) on MP-guided therapy and the PFS on the last therapy before MP was ≥1.3.

The null hypothesis was that ≤15% of patients gain such benefit.

Overall, ≥1 actionable (i.e., predictive of response to specific therapies) biomarker was identified/patient.

Immunohistochemistry (the most commonly used method for guiding treatment decisions) identified 1–6 (median: 4) actionable biomarkers per patient.

After MP, patients received 1–4 (median: 1) regimens/patient (most commonly, FOLFIRI/XELIRI).

In a decision-impact analysis, of the 27 patients for whom treatment decisions before MP were available, 74.1% experienced a treatment decision change in the first line after MP.

Twenty-four patients were evaluable for clinical outcome analysis; in 37.5%, the PFS ratio was ≥1.3.

In one-sided exact binomial test versus the null hypothesis, P = 0.0015; therefore, the null hypothesis was rejected.

In conclusion, our analysis demonstrated the feasibility, clinical decision impact, and potential clinical benefits of MP-guided therapy in advanced PBC.