Advanced biliary tract cancer (BTC) remains an aggressive disease with limited therapeutic options and historically poor survival outcomes. Findings from the phase 3 TOPAZ-1 study (NCT03875235) established durvalumab plus gemcitabine and cisplatin (GemCis) as the standard of care for patients with advanced BTC and a leading first-line regimen. Although multiple real-world studies have validated the TOPAZ-1 findings across diverse populations, data specifically characterizing outcomes in US real-world practice using claims data remain limited. This study, presented at ASCO GI 2026, addresses that gap by describing the characteristics, clinical outcomes, and treatment patterns of patients with advanced BTC treated with durvalumab plus GemCis or GemCis alone in the United States, providing real-world insights into these facets of advanced BTC treatment.
The study evaluated real-world clinical outcomes using Optum’s deidentified Market Clarity claims database. Adult patients with de novo or recurrent advanced BTC were included if they initiated first-line durvalumab plus GemCis on or after NCCN guideline inclusion on March 11, 2022, or GemCis on or after January 1, 2019, to February 28, 2025. The primary objective of this study was to characterize baseline demographics and clinical characteristics of patients receiving durvalumab plus GemCis or GemCis. Exploratory objectives included estimating real-world time to next treatment (rwTTNT) and overall survival (rwOS) using Kaplan–Meier methods without direct cohort comparisons.
In total, 636 patients were included in the study: 210 treated with durvalumab plus GemCis and 426 treated with GemCis, including 367 patients who received GemCis prior to March 2022. Baseline characteristics were generally well balanced across cohorts. Most patients in both cohorts had de novo disease (~86%-87%) and intrahepatic cholangiocarcinoma (76.2% vs 68.5%). The median age was 67 years in the durvalumab plus GemCis cohort and 63 years in the GemCis cohort, and the mean Charlson comorbidity index score was 2.0 in both groups. Median time from treatment initiation to end of follow-up was 6.9 months in the durvalumab plus GemCis cohort and 8.7 months in the GemCis cohort. At the last follow-up, 26.2% of patients on durvalumab plus GemCis and 7.7% on GemCis remained on first-line therapy, whereas 30.0% and 46.2% of those discontinuing durvalumab plus GemCis or GemCis, respectively, transitioned to second-line therapy.
Patients treated with durvalumab plus GemCis experienced longer median rwTTNT compared with those receiving GemCis alone (10.3 months [95% CI, 8.0-15.8] vs 7.5 months [95% CI, 6.6-8.5]). Median rwOS was also longer with durvalumab plus GemCis at 14.6 months (95% CI, 12.9-19.0) compared with 11.9 months (95% CI, 10.3-14.5) for GemCis. Kaplan–Meier estimates at 24 months showed higher rwTTNT (0.32 vs 0.19) and rwOS (0.38 vs 0.28) rates for the durvalumab plus GemCis cohort compared with GemCis alone. These real-world outcomes are consistent with the survival improvement observed in the TOPAZ-1 trial.
This study represents the first US-based analysis using comprehensive claims data to describe real-world outcomes associated with durvalumab plus GemCis or GemCis alone in patients with advanced BTC. While inherent limitations of retrospective claims analyses apply, and there were no direct comparisons between cohorts, the findings support the clinical utility of durvalumab plus GemCis as a meaningful first-line treatment option in routine practice.
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