Ampullary Adenocarcinoma, Version 1.2023, NCCN Clinical Practice Guidelines in Oncology

Abstract

Ampullary cancers refer to tumors originating from the ampulla of Vater (the ampulla, the intraduodenal portion of the bile duct, and the intraduodenal portion of the pancreatic duct), while periampullary cancers may arise from locations encompassing the head of the pancreas, distal bile duct, duodenum, or ampulla of Vater. Ampullary cancers are rare gastrointestinal malignancies, and prognosis varies greatly based on factors such as patient age, TNM classification, differentiation grade, and treatment modality received. Systemic therapy is used in all stages of ampullary cancer, including neoadjuvant therapy, adjuvant therapy, and first-line or subsequent-line therapy for locally advanced, metastatic, and recurrent disease. Radiation therapy may be used in localized ampullary cancer, sometimes in combination with chemotherapy, but there is no high-level evidence to support its utility. Select tumors may be treated surgically. This article describes NCCN recommendations regarding management of ampullary adenocarcinoma.

Assessing the genomic feature of Chinese patients with ampullary adenocarcinoma: potential therapeutic targets

“…The Level 3 actionable GAs were primarily enriched in the DNA damage response (DDR) pathway ( ATM , BRCA1 /2) and PI3K pathway ( PIK3CA , TSC1 ), suggesting potential sensitivity to PARP inhibitors or PI3K inhibitors. Among the actionable GAs supported by Level 3 evidence, FGFR2 , BRCA1/2 , PALB2 , and ERBB2 have been recommended by the NCCN AMPAC guideline as markers for identifying potential candidates for matched anti-cancer therapy [ 22 ]. Additionally, all of these identified actionable GAs with Level 3 evidence align with the ESMO Scale for Clinical Actionability of molecular Targets (ESCAT) evidence tier III [ 23 ].…”

“…Although AMPAC only accounts for 0.2% of gastrointestinal malignancies, it carries significant clinical importance due to its pathological variations and associated prognosis. The 5-year overall survival rate for AMPAC patients who undergo resection ranges from 35 to 50%, with outcomes heavily influenced by various clinical and histological factors, particularly tumor stage and treatment [ 2 ]. Despite nearly 80% of AMPAC cases being resectable at diagnosis, approximately half of these cases may experience disease recurrence [ 3 ].…”

“…However, it remains unknown whether there are genomic differences between Eastern and Western patients, and little is known about the genomic features of Chinese patients with AMPAC. The National Comprehensive Cancer Network (NCCN) guidelines recommend genetic testing for AMPAC patients, including genes such as ALK , NRG1 , NTRK , ROS1 , FGFR2 , RET , BRAF , BRCA1/2 , KRAS , and PALB2 [ 2 ]. However, it is worth noting that this recommendation is directly adapted from that for pancreatic cancer, and the actual prevalence of these actionable alterations in AMPAC remains uncertain.…”

A Rare Case of Metastatic Ampullary Adenocarcinoma Following the Whipple Procedure

“…The intestinal subtype, associated with mutations in APC and PI3KCA, represents 25-35% of cases. The mixed subtype, exhibiting features of both the pancreatobiliary and intestinal subtypes, constitutes 10-20% of cases [5]. These statistics highlight the diversity of ampullary adenocarcinoma.…”

“…For the pancreaticobiliary subtype, clinicians often refer to the NCCN guidelines for pancreatic cancer or hepatobiliary cancers, which often recommend a gemcitabine-based regimen [6]. The potential utility of many regimens in individual patients must be carefully evaluated by the treating physicians based on interpretation of data and drug risk/benefit profile [5].…”