Post-hoc analysis of patients with advanced ampullary cancer in the ABC01, -02, and -03 clinical trials

Ampullary carcinoma (AC) is a rare malignancy often classified within biliary tract cancers (BTC) bu without dedicated treatment guidelines. Post-hoc analysis of the British phase 2 and 3 ABC-01, 02-, and -03 trials found that among 534 BTC trial participants, 28 (5.24%) had AC. The median age was 64 years and 75% were male. Most patients (89%) had metastatic disease at baseline. Among 17 patients receiving cisplatin plus gemcitabine, median follow-up was 10.2 months. The objective response rate was 23.5% and disease control was achieved in 58.8%. Estimated median progression-free survival was 7.98 months (95% CI 6.86-8.44) and median overall survival was 11.76 months (5.94-14.88), comparable to outcomes in other BTCs. No reliable prognostic or predictive factors for ORR, PFS, or OS were identified. Molecular profiling revealed potentially actionable alterations, including HER2 amplification and KRAS mutations. The authors conclude that the analysis provides a reference dataset for advanced AC treated with cisplatin and gemcitabine.