LAUSR

Abstract Background Gemcitabine plus cisplatin has been the standard palliative chemotherapy for patients with advanced biliary tract cancer (aBTC). Recently, the survival benefit of combination chemotherapy with gemcitabine, cisplatin, and S-1 (GCS) was reported, and GCS is expected to be a new standard treatment. This study aimed to evaluate the efficacy and safety of GCS. Methods This retrospective study was conducted at a single Japanese institute from February 2012 to February 2019. Gemcitabine and cisplatin were administered intravenously at doses of 1,000 or 30 mg/m2, respectively, on day 1, and oral S-1 was administered daily at a dose of 80 mg/m2 on days 1-7 every 2 weeks. We evaluated overall survival (OS), progression-free survival (PFS), overall response and toxicity. Results 18 patients were enrolled in this study. These patients included with the following characteristics: median age (range),70 (47-75) years; male/female,12/6; ECOG PS 0/1/2, 12/5/1. Median OS and PFS were 12.3 M (95% CI, 6.6-17.1) and 9.3 M (95% CI, 5.1-12.3). Response rate was 28%, disease control rate was 89%. The grade 3-4 hematological toxicities were as follows: neutropenia (44%) and anemia (17%). The common grade 3-4 non-hematological toxicities were anorexia/nausea (11%) and biliary tract infection (6%). Discontinuation of treatment due to any toxicities was none. The proportion of patients who were treated with subsequent chemotherapies was in 28% of patients, and no patient was performed a conversion surgery. Conclusion We found that GCS was well tolerated and effective for patients with aBTC, and can be a novel treatment option as first-line treatment.