Population-based Cohort Study on Treatment and Overall Survival of Patients Clinically Diagnosed With T1 Ampullary...

To evaluate treatment outcomes, overall survival (OS), and prognostic factors for OS in patients diagnosed with T1 ampullary cancer.

Ampullary cancer is a rare gastrointestinal malignancy with limited data from large cohorts, especially regarding T1 disease.

Patients diagnosed with clinical (c) T1 ampullary cancer and patients with pathologic (p) T1 in the case of cTx were included from the Netherlands Cancer Registry (2014-2021). The primary endpoint was OS, analyzed using the Kaplan-Meier estimator. Multivariable Cox proportional hazards regression was used to identify OS predictors.

Overall, 244 patients with cT1 ampullary cancer were included, of whom 75% (n=184) underwent resection. Among these, 68% (n=125) were upstaged to a higher pathologically T classification (pT2:40%, pT3:22%, pT4:5%). Similarly, cN0 was upstaged to pN1 in 47% of patients (n=87). Next, 100 patients with pT1 and cTx ampullary cancer were included, making a total of 159 patients with pT1 tumor. Ninety-two percent (146/159) underwent pancreatoduodenectomy, while 8% (13/159) underwent endoscopic or local surgical resection. The 1- and 5-year OS for cT1N0 ampullary cancer were 72% and 36%, while for pT1N0 they were 94% and 75%. Independent poor prognostic factors for OS were pN1 classification (HR: 2.12; 95% CI: 1.15-3.94, P =0.017), pNx classification (ie, locally resected patients) (HR: 2.82; 95% CI: 1.22-6.55, P =0.016), and poorly differentiated tumors (HR: 4.05; 95% CI: 1.33-12.40, P =0.014).

In patients with cT1 ampullary cancer, more than two-thirds had a pathologically higher T classification, and almost half had a pathologically higher N classification. These findings suggest that pancreatoduodenectomy is recommended for cT1 ampullary cancer.