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

TL;DR

This study tested a chemotherapy regimen for biliary tract cancer with positive lymph nodes and found it safe and potentially effective, with a 60% curative resection rate.

Contribution

The study introduces a new neoadjuvant chemotherapy regimen (GCS) for resectable biliary tract cancer with FDG-PET-positive lymph nodes.

Findings

Neoadjuvant GCS chemotherapy achieved a 60% curative resection rate with acceptable toxicity.

Radiological and pathological complete response rates for FDG-PET-positive lymph nodes were 24% and 28%, respectively.

The 1-year survival rate was 75% among the 25 enrolled patients.

Abstract

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).

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).

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%.

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).

Ogiso and colleagues conducted a multicenter phase II trial evaluating neoadjuvant gemcitabine, cisplatin, and S‐1 for resectable biliary tract cancer with FDG‐PET‐positive lymph node metastasis. The regimen was safe and feasible, achieved a 60% curative resection rate with acceptable toxicity, and showed encouraging survival, supporting further evaluation in randomized phase III trials.

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