Abstract
BACKGROUND: Ampullary carcinoma (AC) is a rare and severe gastrointestinal cancer with a disease recurrence rate of around 40% after curative-intent surgery and for which the main prognostic factors and adjuvant treatment decision remain a matter of debate. PATIENTS AND METHODS: The FFCD-AC cohort is a French nationwide prospective cohort, which included patients with non-metastatic resected AC. The primary objective of this study was to describe prognostic factors associated with disease-free survival (DFS) and overall survival (OS) after pancreaticoduodenectomy (PD) so as to propose a user-friendly score to better estimate the risk of recurrence. The secondary objective was to study the benefit of adjuvant therapy in terms of DFS and OS. RESULTS: Three hundred and seventy patients with resected AC were included. Median follow-up was 40.6 months. Median age was 68.5 years (32.0-87.0 years), 53.8% of patients were male and 56.1%/37.4%/6.5% had an Eastern Cooperative Oncology Group performance status 0/1/2, respectively. Pathological subtype was intestinal/pancreatobiliary/mixed-undetermined in 29.5%/40.5%/30.0% of patients, respectively. Adjuvant chemotherapy was carried out in 61% of patients. In multivariable analysis, stage III tumor [hazard ratio (HR) 2.86, (95% confidence interval {95% CI}: 1.89-4.17), P < 0.0001], high tumor grade [HR 2.51, (95% CI: 1.42-4.43), P = 0.002] and non-intestinal subtype [HR 1.58, (95% CI: 1.00-2.49), P = 0.052] were associated with shorter DFS. A score based on these three parameters divided patients into low (n = 83), intermediate (n = 133) and high risk (n = 96) with median DFS not reached (NR)/73.1/15.2 months and a median OS NR/86.1/38.2 months, respectively. After propensity score matching, adjuvant chemotherapy was associated with longer DFS [HR 0.57, (95% CI: 0.45-0.72), P < 0.0001] in the cohort. CONCLUSION: Our integrated score based on three easy-to-collect items-lymph node invasion, tumor grade and non-intestinal subtypes-seems highly prognostic in resected AC and needs to be confirmed in an external validation dataset to help adjuvant treatment decision making.