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Precision targeted-immunotherapy for BRAF V600E and MET-amplified biliary tract cancer: two case reports
Xin He1, Sidong Xie2, Changchang Jia3
1Department of Medical Oncology, The Third Affiliated Hospital of Sun Yat-sen University, Guangzhou 510630, China.
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.
Insights
Targeted therapies combined with immune checkpoint inhibitors show promise for advanced biliary tract cancer (BTC) patients with specific mutations. These personalized treatments led to significant tumor reduction and successful surgeries, improving outcomes.
Area of Science:
- Oncology
- Genetics
- Immunotherapy
Background:
- Biliary tract cancer (BTC) has a poor prognosis, with limited response rates to current treatments.
- Combining immunotherapy with chemotherapy offers survival benefits but remains suboptimal.
- Targeted therapies based on genetic mutations may enhance outcomes when used with immune checkpoint inhibitors.
Purpose of the Study:
- To assess the efficacy and feasibility of combining targeted therapy with immune checkpoint inhibitors in advanced BTC.
- To examine 2 cases of advanced BTC with distinct genetic alterations treated with this combined approach.
Main Methods:
- Case 1: A patient with BRAF V600E-mutated intrahepatic cholangiocarcinoma received dabrafenib, trametinib, and durvalumab.
- Case 2: A patient with MET-amplified gallbladder cancer was treated with tepotinib, durvalumab, and chemotherapy.
- Both patients underwent subsequent surgical resection.
Main Results:
- Case 1: Achieved significant tumor reduction, enabling complete surgical resection with a major pathological response.
- Case 2: Demonstrated metastases regression and successful tumor downstaging, with surgery resulting in no evidence of disease.
- The combined approach led to tumor reduction and improved resectability.
Conclusions:
- Combining targeted therapies with immune checkpoint inhibitors shows promising results for advanced BTC.
- Personalized treatment guided by genetic profiling may improve resectability and patient outcomes.
- Further research is warranted to validate these findings and explore broader applications.
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