title: A proposed staging system and stage-specific interventions for familial adenomatous polyposis.
abstract: OBJECTIVE
  It is not possible to accurately count adenomas in many patients with familial adenomatous polyposis (FAP). Nevertheless, polyp counts are critical in evaluating each patient's response to interventions. However, the U.S. Food and Drug Administration no longer recognizes the decrease in polyp burden as a sufficient chemoprevention trial treatment endpoint requiring a measure of "clinical benefit." To develop endpoints for future industry-sponsored chemopreventive trials, the International Society for Gastrointestinal Hereditary Tumors (InSIGHT) developed an FAP staging and intervention classification scheme for lower-GI tract polyposis.
  
  
  METHODS
  Twenty-four colonoscopy or sigmoidoscopy videos were reviewed by 26 clinicians familiar with diagnosis and treatment of FAP. The reviewers independently assigned a stage to a case by using the proposed system and chose a stage-specific intervention for each case. Our endpoint was the degree of concordance among reviewers staging and intervention assessments.
  
  
  RESULTS
  The staging and intervention ratings of the 26 reviewers were highly concordant (ρ = 0.710; 95% credible interval, 0.651-0.759). Sixty-two percent of reviewers agreed on the FAP stage, and 90% of scores were within ±1 stage of the mode. Sixty percent of reviewers agreed on the intervention, and 86% chose an intervention within ±1 level of the mode.
  
  
  CONCLUSIONS
  The proposed FAP colon polyposis staging system and stage-specific intervention are based on a high degree of agreement on the part of experts in the review of individual cases of polyposis. Therefore, reliable and clinically relevant means for measuring trial outcomes can be developed. Outlier cases showing wide scatter in stage assignment call for individualized attention and may be inappropriate for enrollment in clinical trials for this reason.
authors.affiliation: Department of Gastroenterology, Hepatology, and Nutrition, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: Patrick M
authors.initials: PM
authors.lastname: Lynch
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: Jeffrey S
authors.initials: JS
authors.lastname: Morris
authors.affiliation: Department of Biostatistics, West Virginia University Health Sciences Center, Morgantown, West Virginia, USA.
authors.forename: Sijin
authors.initials: S
authors.lastname: Wen
authors.affiliation: Department of Gastroenterology, Hepatology, and Nutrition, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: Shailesh M
authors.initials: SM
authors.lastname: Advani
authors.affiliation: Department of Gastroenterology, Hepatology, and Nutrition, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: William
authors.initials: W
authors.lastname: Ross
authors.affiliation: Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: George J
authors.initials: GJ
authors.lastname: Chang
authors.affiliation: Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: Miguel
authors.initials: M
authors.lastname: Rodriguez-Bigas
authors.affiliation: Department of Gastroenterology, Hepatology, and Nutrition, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: Gottumukkala S
authors.initials: GS
authors.lastname: Raju
authors.affiliation: Department of Medical and Surgical Sciences, University of Bologna, Bologna, Italy.
authors.forename: Luigi
authors.initials: L
authors.lastname: Ricciardiello
authors.affiliation: Department of Digestive Tract and General Surgery, Saitama Medical University, Saitama, Japan.
authors.forename: Takeo
authors.initials: T
authors.lastname: Iwama
authors.affiliation: Division of Surgical Oncology, Hereditary Cancer Registry, Hospital Sirio Libanes, Sao Paulo, Brazil.
authors.forename: Benedito M
authors.initials: BM
authors.lastname: Rossi
authors.affiliation: Department of Gastroenterology, Institut de Malalties Digestives i Metabòliques, Hospital Clinic, Centro de Investigación Biomédica en Red de Enfermedades Hepáticas y Digestivas (CIBERehd), Institut d'Investigacions Biomèdiques August Pi I Sunyer (IDIBAPS), University of Barcelona, Barcelona, Spain.
authors.forename: Maria
authors.initials: M
authors.lastname: Pellise
authors.affiliation: Division of Internal Medicine, University of Michigan Health System, Ann Arbor, Michigan, USA.
authors.forename: Elena
authors.initials: E
authors.lastname: Stoffel
authors.affiliation: Section of Colon and Rectal Surgery, Washington University School of Medicine, St. Louis, Missouri, USA.
authors.forename: Paul E
authors.initials: PE
authors.lastname: Wise
authors.affiliation: Unit of Hereditary Digestive Tract Tumors, Fondazione IRCCS, Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Lucio
authors.initials: L
authors.lastname: Bertario
authors.affiliation: Wolfson Unit for Endoscopy, St. Marks Hospital, Harrow, Middlesex, United Kingdom.
authors.forename: Brian
authors.initials: B
authors.lastname: Saunders
authors.affiliation: Department of Internal Medicine, Huntsman Cancer Institute, University of Utah, Salt Lake City, Utah, USA.
authors.forename: Randall
authors.initials: R
authors.lastname: Burt
authors.affiliation: Department of Medical and Surgical Sciences, S. Orsola-Malpighi University Hospital, Bologna, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Belluzzi
authors.affiliation: Division of Gastroenterology, Department of Veterans Affairs Eastern Colorado Health Care System and University of Colorado School of Medicine, Denver, Colorado, USA.
authors.forename: Dennis
authors.initials: D
authors.lastname: Ahnen
authors.affiliation: Department of Surgery, Hyogo College of Medicine, Hyogo, Japan.
authors.forename: Nagahide
authors.initials: N
authors.lastname: Matsubara
authors.affiliation: The Danish Polyposis Register, Gastrointestinal Unit, Hvidovre University Hospital, Copenhagen, Denmark.
authors.forename: Steffen
authors.initials: S
authors.lastname: Bülow
authors.affiliation: The Danish Polyposis Register, Gastrointestinal Unit, Hvidovre University Hospital, Copenhagen, Denmark.
authors.forename: Niels
authors.initials: N
authors.lastname: Jespersen
authors.affiliation: The Polyposis Registry, St. Mark's Hospital, Harrow, Middlesex, United Kingdom.
authors.forename: Susan K
authors.initials: SK
authors.lastname: Clark
authors.affiliation: Division of Gastroenterology, Hepatology and Nutrition, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio, USA.
authors.forename: Steven H
authors.initials: SH
authors.lastname: Erdman
authors.affiliation: Gastroenterology and Nutrition Service, Memorial Sloan-Kettering Cancer Center, New York, New York, USA.
authors.forename: Arnold J
authors.initials: AJ
authors.lastname: Markowitz
authors.affiliation: Department of Surgical Gastroenterology, Aalborg Universitetshospital, Aalborg, Denmark.
authors.forename: Inge
authors.initials: I
authors.lastname: Bernstein
authors.affiliation: Department of Surgical Gastroenterology, Aalborg Universitetshospital, Aalborg, Denmark.
authors.forename: Niels
authors.initials: N
authors.lastname: De Haas
authors.affiliation: Division of Population Sciences, Division of Gastroenterology, Dana-Farber Cancer Institute, Brigham and Women's Hospital, and Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Sapna
authors.initials: S
authors.lastname: Syngal
authors.affiliation: Center for Hereditary Tumors, HELIOS Klinikum Wuppertal, University Witten-Herdecke, Wuppertal, Germany.
authors.forename: Gabriela
authors.initials: G
authors.lastname: Moeslein
mesh_terms: D011125:Adenomatous Polyposis Coli / Q000473:pathology* / Q000628:therapy; D003082:Colectomy; D003113:Colonoscopy; D003107:Colorectal Surgery; D032921:Consensus; D000069916:Endoscopic Mucosal Resection; D005260:Female; D000072102:Gastroenterologists; D006801:Humans; D008297:Male; D009367:Neoplasm Staging; D009378:Neoplasms, Multiple Primary / Q000473:pathology*; D012720:Severity of Illness Index; D012812:Sigmoidoscopy; D012460:Sulfasalazine; D014741:Video Recording
chemical_list: D012460:Sulfasalazine