Ampullary cancers are rare, accounting for only 0.2% of gastrointestinal cancers and approximately 7% of all periampullary cancers. Curative surgery is possible in approximately 50% of ampullary cancer compared to that of less than 10% in pancreatic adenocarcinoma. Despite the high rate of potentially curative resection, the majority of patients with ampullary carcinomas will eventually succumb to recurrent disease Given the rarity of this disease, there is absence of randomized clinical trials focused on ampullary carcinomas and treatment recommendations are mainly derived from results of adjuvant clinical trials conducted in pancreatico-biliary cancers where ampullary cancers may represent a sub-group of patients.
Although there is no clear guidance in regards to adjuvant therapy, patients with resected ampullary carcinomas and stage IB or higher often receive concurrent chemotherapy with radiation. Most studies did not suggest the presence of a survival benefit from adjuvant chemoradiation, except perhaps for a subset of patients with adverse risk factors (T stage, lymph node involvement, histologic grade) that may potentially benefit from chemoradiation. The role of adjuvant chemotherapy in ampulla or vater cancer demonstrated a trend toward improving overall survival favoring the chemotherapy group versus observation.
Despite advances in cancer research, ampullary cancer remain very challenging with a clear absence of an evidence-based standard of adjuvant treatment paradigm. Given the rarity of the disease, it is very unlikely to perform well-powered randomized controlled clinical trials to understand further the role of the various modalities or establish the role of new therapies. Perhaps, as understanding of the molecular and genetic origins of cancer will improve, more targeted therapeutic strategies will help us achieve a better outcome for patients with ampullary cancer.