A Personalized Surveillance and Intervention Protocol for Patients With Familial Adenomatous Polyposis That Have Undergone (Procto)Colectomy

The purpose of this study is to determine the efficacy and safety of a personalised surveillance and intervention protocol for patients with familial adenomatous polyposis (FAP) that have undergone (procto)colectomy.

Study Overview

• Status: Recruiting
• Conditions: Familial Adenomatous Polyposis
• Intervention / Treatment: Procedure: Personalized surveillance and intervention protocol

Detailed Description

Familial adenomatous polyposis (FAP) is characterized by formation of up to hundreds to thousands of polyps throughout the entire colon and rectum. When left untreated, nearly all patients with FAP develop colorectal cancer at a median age of 35-45 years. To prevent colorectal cancer in patients with FAP, prophylactic colorectal surgery is performed. The preferred surgical procedures for FAP are a restorative proctocolectomy with ileal pouch-anal anastomosis (IPAA) or a subtotal colectomy with ileorectal anastomosis (IRA) or ileosigmoidal anastomosis (ISA).

After both types of prophylactic colorectal surgery, subtotal colectomy with IRA/ISA or proctocolectomy with IPAA, patients will require life-long surveillance because disease progression and development of new adenomas in retained rectum, pouch or residual rectal cuff will occur.

The 10-years risk of developing one or more adenomas in the rectum after IRA is 100% compared to 33% in the pouch after IPAA. The risk of developing rectal cancer after IRA was found to be 9% and 11% in two large studies with a median follow-up of 12.8 and 15 years, respectively. One study showed that the 10-years risk of developing a carcinoma in the pouch was 1%. As patients are usually operated at a young age, and nowadays have a long life-expectancy, the actual cumulative life-time risk will presumably be higher.

The recently published ESGE (European Society of Gastrointestinal Endoscopy) polyposis guideline recommends a one to two yearly endoscopic surveillance interval after prophylactic colorectal surgery in FAP, both for patients that underwent IRA/ISA and IPAA, with removal of all polyps >5mm. This recommendation is based on expert-opinion, since no studies have been reported comparing the efficacy and safety of different surveillance intervals. No advices are provided on which patients will benefit from which surveillance interval.

With the proposed study, the investigators aim to provide evidence for personalized endoscopic surveillance for patients with FAP that have undergone (procto)colectomy with construction of an IRA/ISA or IPAA with the goal to prevent development of advanced neoplasia (AN) by endoscopically removing lesions before they progress to AN.

• Study Type: Observational
• Enrollment (Anticipated): 1000

Contacts and Locations

Study Locations

• Netherlands
  • Noord-Holland
    • Amsterdam, Noord-Holland, Netherlands, 1105AZ
      • Recruiting
      • Academic Medical Centre
      • Contact: Evelien Dekker, MD, PhD; Phone Number: 0031205661260; Email: e.dekker@amc.uva.nl
      • Sub-Investigator: Arthur Aelvoet, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

All patients with FAP treated at one of the participating centres.

Description

Inclusion Criteria:

• Diagnosis of FAP, at least one of following: genetic diagnosis (proven APC germline mutation) and/or clinical diagnosis (>100 colorectal adenomas in combination with a positive family history of FAP)
• Have undergone prophylactic (procto)colectomy with IRA/ISA or IPAA
• Age 18 years or older

Exclusion Criteria:

• Not able to remove all polyps with an indication for removal during (multiple) clearing endoscopies
• Cancer at baseline endoscopy
• Need for surgery

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Personalized surveillance and intervention protocol	Procedure: Personalized surveillance and intervention protocol; This study uses one arm. Participants will undergo endoscopic surveillance with intervals between 6 months and 2 years, depending on severity of polyposis and performed endoscopic interventions.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Advanced neoplasia	Incidence of advanced neoplasia (advanced adenoma and cancer). An advanced adenoma is defined as size ≥ 10mm and/or high-grade dysplasia. This surveillance and intervention protocol will be considered successful when the incidence of advanced neoplasia is less than 5% after a study period of 5 years.	Up to 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Characteristics polyps	Incidence and characteristics of polyps detected/removed in patients with IRA/ISA and IPAA	Up to 5 years
Radicality of different endoscopic intervention techniques	Rate of radical endoscopic interventions	Up to 5 years
Feasibility endoscopic interventions	Incidence of lesions not amenable to endoscopic removal	Up to 5 years
Surgical interventions	Incidence of surgical interventions	Up to 5 years
Surveillance burden	Surveillance burden (number of endoscopies per patient)	Up to 5 years
Complications	Incidence of endoscopy related complications	Up to 5 years

Collaborators and Investigators

• Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Collaborators: IRCCS Azienda Ospedaliero-Universitaria di Bologna; Hospital Clinic of Barcelona; St Mark's Hospital Foundation; Hôpital Edouard Herriot; The Netherlands Cancer Institute; Leiden University Medical Center; Maria Sklodowska-Curie National Research Institute of Oncology; Hospital General Universitario de Alicante; University Medical Center Mainz

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 24, 2020
• Primary Completion (ANTICIPATED): November 1, 2025
• Study Completion (ANTICIPATED): November 1, 2025

Study Registration Dates

• First Submitted: November 24, 2020
• First Submitted That Met QC Criteria: December 16, 2020
• First Posted (ACTUAL): December 21, 2020

Study Record Updates

• Last Update Posted (ACTUAL): December 21, 2020
• Last Update Submitted That Met QC Criteria: December 16, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Keywords: Gastrointestinal neoplasia; Endoscopic surveillance; Endoscopic interventions; Personalised care
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Gastrointestinal Neoplasms; Digestive System Neoplasms; Gastrointestinal Diseases; Genetic Diseases, Inborn; Pharyngeal Neoplasms; Otorhinolaryngologic Neoplasms; Head and Neck Neoplasms; Nasopharyngeal Diseases; Pharyngeal Diseases; Stomatognathic Diseases; Otorhinolaryngologic Diseases; Colonic Diseases; Intestinal Diseases; Intestinal Neoplasms; Rectal Diseases; Neoplastic Syndromes, Hereditary; Adenomatous Polyps; Adenoma; Intestinal Polyposis; Nasopharyngeal Neoplasms; Colorectal Neoplasms; Adenomatous Polyposis Coli
• Other Study ID Numbers: W20_182

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No