Precision targeted-immunotherapy for BRAF V600E and MET-amplified biliary tract cancer: two case reports.

BACKGROUND

Biliary tract cancer (BTC), as a relatively rare tumor, carries a poor prognosis. While immunotherapy combined with chemotherapy can extend survival, response rates remain low. Targeted therapies tailored to specific genetic mutations may improve outcomes when paired with immune checkpoint inhibitors. This report examines 2 cases of advanced BTC with distinct genetic alterations, treated with targeted therapy and immune checkpoint inhibitors, to assess efficacy and feasibility.

CASE PRESENTATION: A 45-year-old patient with a BRAF V600E-mutated intrahepatic cholangiocarcinoma received dabrafenib, trametinib, and durvalumab. This led to significant tumor reduction, enabling complete surgical resection with clear margins. Postoperative analysis showed a major pathological response. A 39-year-old patient with MET-amplified gallbladder cancer was treated with tepotinib, durvalumab, and chemotherapy, resulting in metastases regression and successful tumor downstaging. Surgery achieved no evidence of disease.

CONCLUSIONS

Combining targeted therapies with immune checkpoint inhibitors showed promising results in 2 patients with advanced BTC driven by specific genetic mutations. Significant tumor reduction and successful surgeries suggest this approach may improve resectability and outcomes. These cases highlight the potential of personalized treatment guided by genetic profiling. Further research is needed to confirm these findings and explore broader applications for this strategy.