Real-world outcomes of gemcitabine-cisplatin plus durvalumab or pembrolizumab in advanced BTC: K-HBP Cancer Alliance study

초록

Background Following the positive results of TOPAZ-1 and KEYNOTE-966, gemcitabine plus cisplatin (GemCis) combined with durvalumab or pembrolizumab has been established as a global standard first-line regimen for advanced biliary tract cancer (BTC). Because patients treated in routine practice may differ from those enrolled in prospective trials, real-world evaluation of these regimens is warranted. Method The K-HBP Cancer Alliance comprises 11 tertiary referral hospitals in Korea. This multicenter retrospective study included patients who received first-line GemCis plus durvalumab or pembrolizumab for unresectable or metastatic BTC. Efficacy endpoints included overall response rate (ORR), progression-free survival (PFS), and overall survival (OS). Results Between November 2022 and May 2025, 534 patients with BTC (211 [40%] intrahepatic cholangiocarcinoma [iCCA], 212 [40%] extrahepatic cholangiocarcinoma [eCCA], and 111 [20%] gallbladder cancer [GBC]) received GemCis plus durvalumab or pembrolizumab. Median age was 67 years (range, 29-89); 58% were male; 95% had ECOG performance status 0-1; and 81o/o had recurrent or metastatic disease. Median follow-up was 12.0 months (95% Cl, 10.3-13.5). ORR was 20% (95% Cl, 17-24%). Median PFS and OS were 6.6 months (95% Cl, 6.1-7.2) and 14.3 months (95% Cl, 12.9-15.8), respectively. One-year PFS and OS rates were 26% and 58%, respectively. Primary tumor site was not associated with PFS or OS. Elevated CA 19-9 (> median) was associated with inferior PFS (HR, 1.35; 95% Cl, 1.08-1.70; p = 0.009) and OS (HR, 1.53; 95% Cl, 1.13-2.03; p = 0.006). Conclusion In this large multicenter real-world cohort, GemCis plus durvalumab or pembrolizumab demonstrated efficacy outcomes consistent with prospective trials.