Endoscopic papillectomy for ampullary lesions in patients with familial adenomatous polyposis compared with sporadic lesions: a propensity score-matched cohort

Abstract

Background: Familial-adenomatous-polyposis (FAP) is a rare inherited cancer predisposition syndrome. The treatment for FAP-related ampullary lesions (AL) is challenging and the role of endoscopic papillectomy (EP) is not elucidated yet. Data of FAP associated AL are limited and showed, at least in part, inconclusive results. We retrospectively analyzed the outcomes of EP in matched cohorts of FAP-related and sporadic ampullary lesions (SAL). Methods: The ESAP study included 1422 EPs. A propensity-score matching (nearest-neighbor-method) including age, gender, comorbidity, histologic subtype and size was performed. Main outcomes were complete resection (R0), technical success, complications and recurrence. Results: Propensity-score-based matching identified 202 patients (101 FAP, 101 SAL) with comparable baseline characteristics. FAP-patients were mainly asymptomatic (79.2% [95%CI 71.2-87.3] vs. 46.5% [95%CI 36.6-56.4]), p<0.001). The initial R0-rate was significantly lower in FAP-patients (63.4% [95%CI 53.8-72.9] vs. 83.2% [95%CI 75.8-90.6], p=0.001). After repeated (mean: 1.30 per patient) interventions, R0 was comparable (FAP 93.1% [95%CI 88.0-98.1] vs. SAL 97.0% [95%CI 93.7-100], p=0.194). The overall complication rate was 28.7%. Pancreatitis and bleeding were most common adverse events in both groups. Severe complications were very rare (3.5%). Twenty-one patients in the FAP-group (20.8% [95%CI 12.7-28.8]) and sixteen patients in the SAL-group (15.8% [95%CI 8.6-23.1], p=0.363) had a reccurence . Recurrences occurred later in FAP-patients (25 [95%CI 18.3-31.7] vs. 2 [95%CI CI 0.06-3.9] months). Conclusions: EP is safe and effective in FAP-related ampullary lesions Criteria for endoscopic resection of AL can be extended to FAP-patients. FAP-patients have a life-time risk to relapse even after complete resection and require long-time-surveillance.

Reply to Wang et al.

“…

We thank Wang et al for their thoughtful comments and thorough reading of our manuscript analyzing endoscopic papillectomy for patients with familial adenomatous polyposis (FAP) compared with those with sporadic lesions [1]. We agree that the initial reduced R0 rate was lower in the FAP group, although we thoroughly matched for age, sex, size, histology, and other predictors of treatment success.

…”

Updates on the Management of Ampullary Neoplastic Lesions

“…Conse-quently, long-term surveillance is necessary to closely monitor these patients. Recently, in a retrospective multicenter study involving 1422 endoscopic papillectomy procedures, a propensity score matching approach was employed [63]. The matching process considered factors such as age, sex, comorbidity, histologic subtype, and size [63].…”

“…Recently, in a retrospective multicenter study involving 1422 endoscopic papillectomy procedures, a propensity score matching approach was employed [63]. The matching process considered factors such as age, sex, comorbidity, histologic subtype, and size [63]. The purpose was to analyze main outcomes, including complete resection (R0), technical success, complications, and recurrence, within matched cohorts of FAP-related and sporadic ANLs [63].…”

“…The matching process considered factors such as age, sex, comorbidity, histologic subtype, and size [63]. The purpose was to analyze main outcomes, including complete resection (R0), technical success, complications, and recurrence, within matched cohorts of FAP-related and sporadic ANLs [63]. Among the FAP patients, the majority (79.2%; 95% CI = 71.2-87.3) were asymptomatic, which was significantly higher compared to the sporadic group (46.5%; 95% CI = 36.6-56.4; p < 0.001) [63].…”

Comments on “Endoscopic papillectomy for ampullary lesions in patients with familial adenomatous polyposis compared with sporadic lesions: a propensity score-matched cohort”

“…We read with great interest the article by Vu Trung et al [1] analyzing the efficacy and safety of endoscopic papillectomy for ampullary lesions in patients with familial adenomatous polyposis (FAP) compared with sporadic lesions. We agree that endoscopic papillectomy is useful for the resection of ampullary lesions in patients with FAP; however, we have some questions about the research.…”