Background:Durvalumab (anti-PD-L1) in combination with gemcitabine and cisplatin has become the first-line treatment for patients with locally advanced, surgically unresectable, or metastatic biliary tract cancer, following the survival benefit demonstrated in the TOPAZ-1 phase III trial. This study presents real-world data from UK centres in patients who received early access to the regimen via AstraZeneca’s scheme. The aim was to assess the safety and efficacy of this treatment approach in routine clinical practice and compare it to outcomes reported in the TOPAZ-1 trial.Method:This retrospective study included patients with locally advanced, surgically unresectable, or metastatic biliary tract adenocarcinoma who received durvalumab in combination with gemcitabine and cisplatin. Data were collected across ten UK centres. The primary endpoint was progression-free survival (PFS), with secondary endpoints including overall survival (OS), overall response rate (ORR), and safety outcomes, encompassing both chemotherapy and immunotherapy-related adverse events (AEs).Results:A total of 134 patients treated between April 2022 and December 2023 were included. The median follow-up was 12.8 months (95% CI: 11–16.8). The median PFS was 8.83 months (95% CI: 5.73–11.7), closely aligning with the 7.2 months reported in TOPAZ-1 (95% CI: 6.7–7.4). The median OS was 12 months (95% CI: 10.7–13.9), slightly below the 12.8 months observed in TOPAZ-1 (95% CI: 11.1–14.0). The ORR was 29.1% (TOPAZ-1: 26.7%), and the disease control rate was 61.2%. In terms of safety, 64 patients (52.3%) experienced any-grade AEs, and 9 patients (6.8%) had grade 3–4 AEs, representing a lower toxicity profile than TOPAZ-1. Immunotherapy-related AEs occurred in 25 patients (18.7%), with grade 3–4 events in 3%.Conclusions:These real-world findings from UK cancer centres support the outcomes of the TOPAZ-1 trial, demonstrating comparable efficacy and a favourable safety profile for durvalumab combined with gemcitabine-cisplatin as first-line treatment for advanced biliary tract cancer.
背景：基于TOPAZ-1 III期试验证实的生存获益，度伐利尤单抗（抗PD-L1抗体）联合吉西他滨和顺铂已成为局部晚期、手术不可切除或转移性胆道癌患者的一线治疗方案。本研究通过阿斯利康早期用药计划，收集了英国多家中心接受该方案治疗患者的真实世界数据，旨在评估该治疗方案在常规临床实践中的安全性与有效性，并与TOPAZ-1试验结果进行比较。 方法：这项回顾性研究纳入了接受度伐利尤单抗联合吉西他滨和顺铂治疗的局部晚期、手术不可切除或转移性胆道腺癌患者，数据来源于英国十家医疗中心。主要终点为无进展生存期（PFS），次要终点包括总生存期（OS）、客观缓解率（ORR）及安全性指标（涵盖化疗与免疫治疗相关不良事件）。 结果：共纳入2022年4月至2023年12月期间接受治疗的134例患者。中位随访时间为12.8个月（95% CI：11-16.8）。中位PFS为8.83个月（95% CI：5.73-11.7），与TOPAZ-1试验报告的7.2个月（95% CI：6.7-7.4）高度吻合；中位OS为12个月（95% CI：10.7-13.9），略低于TOPAZ-1试验的12.8个月（95% CI：11.1-14.0）。ORR为29.1%（TOPAZ-1：26.7%），疾病控制率达61.2%。安全性方面，64例患者（52.3%）出现任意级别不良事件，9例患者（6.8%）发生3-4级不良事件，总体毒性低于TOPAZ-1试验。免疫治疗相关不良事件发生率为18.7%（25例），其中3-4级事件占3%。 结论：英国癌症中心的真实世界数据支持TOPAZ-1试验结果，证实度伐利尤单抗联合吉西他滨-顺铂方案在晚期胆道癌一线治疗中具有相当的疗效和良好的安全性。
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