The efficacy of frontline platinum-based combination chemotherapy in advanced adenocarcinoma of the ampulla of Vater.

Adenocarcinoma arising from the ampulla of Vater is a rare neoplasm that accounts for only 0.2% of all gastrointestinal tract malignancies and has limited data regarding its frontline therapy. We investigated the treatment outcomes in patients with advanced adenocarcinoma of the ampulla of Vater receiving frontline cisplatin-based combination chemotherapy. We analyzed 29 patients with advanced adenocarcinoma of the ampulla of Vater who had been treated by frontline cisplatin-based combination chemotherapy between June 2003 and April 2008. The chemotherapeutic agent added to cisplatin was gemcitabine in 9 patients and fluorouracil (FU) in 20 patients (11; intravenous 5-FU and 9; oral 5-FU (capecitabine)). The median age of patients was 56 years (range, 36-78), and the median ECOG performance status was 1 (0-1). The confirmed overall response rate was 27.5%, and the disease control rate was 72.4%. In all patients, no complete responses and 8 partial responses were observed (overall response rate, 27.5%). Stable disease was observed in 13 patients (44.8%), and progressive disease in 5 patients (17.2%). The median time to progression (TTP) was 4.9 months (95% CI, 3.4-6.4), and the median overall survival (OS) was 12.5 months (95% CI, 10.6-14.4). There were no significant differences for TTP and OS according to the different chemotherapeutic agents added to cisplatin. Grade 3 or 4 hematologic toxicities included leukopenia in seven patients and thrombocytopenia in one patient. There were no grade 3 or 4 nonhematologic toxicities or treatment-related deaths. The cisplatin-based combination chemotherapy showed moderate activity and a favorable toxicity profile as a frontline treatment for patients with advanced adenocarcinoma of the ampulla of Vater.