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Abstract Introduction This study assesses the effectiveness of durvalumab with platinum and gemcitabine for biliary tract cancers (BTC). It aims to confirm the Topaz-1 trial results in a real-world context and explore the link between BTC molecular profiles and patient outcomes. Methods A retrospective analysis was conducted on 102 BTC patients treated with durvalumab, platinum, and gemcitabine at five cancer centers in Austria and one in Germany from 2022 to 2024. Molecular profiling used targeted DNA and RNA assays. Clinical endpoints, including progression-free survival (PFS) and overall survival (OS), were assessed using log-rank tests and Cox regression, with correlations to second-line molecular-targeted therapies. Results Among 102 patients, 60.8% had intrahepatic cholangiocarcinoma. The treatment achieved a disease control rate of 71.57% and an overall response rate of 35.11%. Median PFS was 6.51 months, and OS was 13.61 months. Patients under 65 had significantly better OS. Alterations in Chromatin Remodeling or Homologous Recombination Repair genes were not predictive of survival benefit (HR 0.45; p=0.851 and HR 1.63; p=0.26, respectively). Patients with molecular-informed second-line therapy showed a trend towards survival benefit (HR 0.23, p=0.052). Conclusion This study confirms the phase 3 trial results of durvalumab with platinum and gemcitabine, providing a substantial real-world dataset with detailed molecular characterization. No specific patient subgroup showed a markedly better response to durvalumab based on conventional NGS panels. Further research is needed to explore the link between immunotherapy responses and molecular subgroups
Doleschal et al. (Tue,) studied this question.
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