Role of Molecular Tumor Board in Pancreatic and Biliary Tract Tumors

PURPOSE The use of molecularly guided treatment options (MGTOs) according to molecular tumor board (MTB) recommendations demonstrated improved clinical outcomes for patients with cancer. Still, limited data are available about the clinical value of discussing patients specifically affected by pancreatic cancer (PC) and biliary tract cancer (BTC) in a real-world MTB scenario. MATERIALS AND METHODS We retrospectively retrieved patients with advanced PC and BTC who were discussed at the European Institute of Oncology's MTB between August 2019 and December 2024. We investigated the clinical outcomes associated with MTB-endorsed MGTOs compared with those of patients treated with standard treatment (ST), including those treated with ST despite receiving MGTO recommendations (unmatched cases) and those not showing actionable biomarkers (nonactionable cases). RESULTS Of 75 patients (n = 28 with PC, n = 47 with BTC) discussed at the MTB, MGTOs were recommended for 65.3% of cases, with n = 31 (35.5%) having follow-up data. Patients treated with MGTOs (n = 11) showed longer real-world median progression-free survival (mPFS) than those treated with ST (n = 20; mPFS 13.4 v 5.0 months; hazard ratio HR, 0.37 95% CI, 0.13 to 1.05; P = .063), with a clinical benefit rate of 58.9% (95% CI, 34.6 to 99.9) versus 34.4% (95% CI, 18.2 to 65.3), respectively. The benefit was consistent compared with unmatched cases (n = 14, HR, 0.35 95% CI, 0.12 to 1.00; P = .05) and nonactionable cases (n = 6, HR, 0.47 95% CI, 0.12 to 1.84; P = .27). No overall survival difference was observed between the two groups (restricted mean survival time ratio MGTO/ST 0.85 95% CI, 0.60 to 1.19; P = .36). CONCLUSION Our findings support the clinical value of MTB-informed decision-making in PC and BTC, offering additional therapeutic options and prolonged PFS in selected patients.