Neoadjuvant Chemotherapy With Gemcitabine/Cisplatin/S‐1 for Resectable Biliary Tract Cancer With FDG ‐ PET ‐Positive Lymph Node Metastasis ( KHBO1201 ): A Multicenter Phase II Trial

Purpose To evaluate the safety and efficacy of neoadjuvant gemcitabine, cisplatin, and S‐1 (GCS) chemotherapy for resectable biliary tract cancer (BTC) and FDG‐PET‐positive lymph nodes in a multicenter phase II study (KHBO1201). Methods Patients with resectable BTC (intrahepatic/extrahepatic bile duct, gallbladder, or ampullary cancers) and FDG‐PET‐positive lymph nodes received GCS chemotherapy: gemcitabine/cisplatin on Day 1 and oral S‐1 for 7 days, repeated every 2 weeks for 3–6 cycles. Surgery was planned 4–8 weeks later if the tumor was deemed resectable. The primary endpoint was the curative resection rate. Secondary endpoints were the completion rate, radiological response, radiological/pathological complete response (CR) of FDG‐PET‐positive lymph nodes, and 1‐year survival (UMIN000009831). Results Twenty‐five patients were enrolled. Twenty‐three (92%) completed GCS without treatment‐related deaths; grade 3 biliary infection occurred in 8.0%. Curative resection was achieved in 60% with a morbidity rate of 40%. The radiological response rate was 13%, and the radiological and pathological CR rates of FDG‐PET‐positive lymph nodes were 24% and 28%, respectively. The 1‐year survival rate was 75%. Conclusions Neoadjuvant GCS chemotherapy is safe, feasible, and potentially effective for resectable BTC with FDG‐PET‐positive lymph nodes. A randomized phase III trial (JCOG1920) is underway to compare neoadjuvant GCS chemotherapy with upfront surgery (jRCTs031200388).