Introduction
Randomized controlled trials are the gold standard for efficacy and safety evidence in oncology. However, with restricted eligibility and often limited population diversity, they can lack generalisability, particularly in rare cancers. The TOPAZ-1 randomised trial demonstrated improved survival with first line gemcitabine, cisplatin and durvalumab compared to chemotherapy alone in advanced biliary tract cancer [1]. The aim of this study is to evaluate the efficacy and safety of this regimen in a real-world Australian cohort.
Methods
This is a retrospective study of first line gemcitabine, cisplatin and durvalumab at multiple Australian sites. Data on demographics, treatment, toxicities and survival were collected.
Results
From February 2022 to January 2024, 17 patients commenced treatment at Prince of Wales and St George Hospitals, Sydney. Median age was 60-years (range 41-77 years). 71% had intrahepatic cholangiocarcinoma. 15 (88%) had metastatic disease at baseline. 4 patients had prior resection and 3 had received prior adjuvant chemotherapy. At data cut off on 31/01/2024, median follow up was 9.3 months (range 3.3-23.3), 2 patients continue on treatment and 6 patients had died. There were 4 Partial and 1 Complete response [ORR 29%]. Median DOR was 5.5 months. The median PFS was 4.1 months (95% CI 3.0-6.2). Median OS has not been reached. 9 (53%) patients had at least 1 toxicity, mostly grade 1-2, 2 grade 3 anaemia and 1 grade 3 neutropaenia. There were no high-grade immune related toxicities. Data from additional sites in Victoria and Western Australia will be presented at COSA ASM.
Conclusions
Real-world evidence is an important adjunct to the evidence from randomised controlled trials. The preliminary results from this study have demonstrated similar ORR and DOR but lower median PFS of 4.1 months, compared to TOPAZ-1 (median PFS 7.2 months (95% CI 6.7-7.4)), with manageable toxicity. Longer follow up is required.