TY - JOUR
T1 - A proposed staging system and stage-specific interventions for familial adenomatous polyposis
AU - Lynch, Patrick M.
AU - Morris, Jeffrey S.
AU - Wen, Sijin
AU - Advani, Shailesh M.
AU - Ross, William
AU - Chang, George J.
AU - Rodriguez-Bigas, Miguel
AU - Raju, Gottumukkala S.
AU - Ricciardiello, Luigi
AU - Iwama, Takeo
AU - Rossi, Benedito M.
AU - Pellise, Maria
AU - Stoffel, Elena
AU - Wise, Paul E.
AU - Bertario, Lucio
AU - Saunders, Brian
AU - Burt, Randall
AU - Belluzzi, Andrea
AU - Ahnen, Dennis
AU - Matsubara, Nagahide
AU - Bülow, Steffen
AU - Jespersen, Niels
AU - Clark, Susan K.
AU - Erdman, Steven H.
AU - Markowitz, Arnold J.
AU - Bernstein, Inge
AU - De Haas, Niels
AU - Syngal, Sapna
AU - Moeslein, Gabriela
N1 - Publisher Copyright:
© 2016 American Society for Gastrointestinal Endoscopy.
PY - 2016/7/1
Y1 - 2016/7/1
N2 - Background and Aims 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.
AB - Background and Aims 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.
UR - http://www.scopus.com/inward/record.url?scp=84961114343&partnerID=8YFLogxK
U2 - 10.1016/j.gie.2015.12.029
DO - 10.1016/j.gie.2015.12.029
M3 - Article
C2 - 26769407
AN - SCOPUS:84961114343
SN - 0016-5107
VL - 84
SP - 115-125.e4
JO - Gastrointestinal endoscopy
JF - Gastrointestinal endoscopy
IS - 1
ER -