A proposed staging system and stage-specific interventions for familial adenomatous polyposis

Patrick M. Lynch, Jeffrey S. Morris, Sijin Wen, Shailesh M. Advani, William Ross, George J. Chang, Miguel Rodriguez-Bigas, Gottumukkala S. Raju, Luigi Ricciardiello, Takeo Iwama, Benedito M. Rossi, Maria Pellise, Elena Stoffel, Paul E. Wise, Lucio Bertario, Brian Saunders, Randall Burt, Andrea Belluzzi, Dennis Ahnen, Nagahide Matsubara & 9 others Steffen Bülow, Niels Jespersen, Susan K. Clark, Steven H. Erdman, Arnold J. Markowitz, Inge Bernstein, Niels De Haas, Sapna Syngal, Gabriela Moeslein

Research output: Contribution to journalArticle

15 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)115-125.e4
JournalGastrointestinal Endoscopy
Volume84
Issue number1
DOIs
Publication statusPublished - Jul 1 2016

Fingerprint

Adenomatous Polyposis Coli
Polyps
Lower Gastrointestinal Tract
Sigmoidoscopy
Chemoprevention
United States Food and Drug Administration
Colonoscopy
Adenoma
Industry
Clinical Trials
Therapeutics
Neoplasms

ASJC Scopus subject areas

  • Gastroenterology
  • Radiology Nuclear Medicine and imaging

Cite this

Lynch, P. M., Morris, J. S., Wen, S., Advani, S. M., Ross, W., Chang, G. J., ... Moeslein, G. (2016). A proposed staging system and stage-specific interventions for familial adenomatous polyposis. Gastrointestinal Endoscopy, 84(1), 115-125.e4. https://doi.org/10.1016/j.gie.2015.12.029

A proposed staging system and stage-specific interventions for familial adenomatous polyposis. / Lynch, Patrick M.; Morris, Jeffrey S.; Wen, Sijin; Advani, Shailesh M.; Ross, William; Chang, George J.; Rodriguez-Bigas, Miguel; Raju, Gottumukkala S.; Ricciardiello, Luigi; Iwama, Takeo; Rossi, Benedito M.; Pellise, Maria; Stoffel, Elena; Wise, Paul E.; Bertario, Lucio; Saunders, Brian; Burt, Randall; Belluzzi, Andrea; Ahnen, Dennis; Matsubara, Nagahide; Bülow, Steffen; Jespersen, Niels; Clark, Susan K.; Erdman, Steven H.; Markowitz, Arnold J.; Bernstein, Inge; De Haas, Niels; Syngal, Sapna; Moeslein, Gabriela.

In: Gastrointestinal Endoscopy, Vol. 84, No. 1, 01.07.2016, p. 115-125.e4.

Research output: Contribution to journalArticle

Lynch, PM, Morris, JS, Wen, S, Advani, SM, Ross, W, Chang, GJ, Rodriguez-Bigas, M, Raju, GS, Ricciardiello, L, Iwama, T, Rossi, BM, Pellise, M, Stoffel, E, Wise, PE, Bertario, L, Saunders, B, Burt, R, Belluzzi, A, Ahnen, D, Matsubara, N, Bülow, S, Jespersen, N, Clark, SK, Erdman, SH, Markowitz, AJ, Bernstein, I, De Haas, N, Syngal, S & Moeslein, G 2016, 'A proposed staging system and stage-specific interventions for familial adenomatous polyposis', Gastrointestinal Endoscopy, vol. 84, no. 1, pp. 115-125.e4. https://doi.org/10.1016/j.gie.2015.12.029
Lynch, Patrick M. ; Morris, Jeffrey S. ; Wen, Sijin ; Advani, Shailesh M. ; Ross, William ; Chang, George J. ; Rodriguez-Bigas, Miguel ; Raju, Gottumukkala S. ; Ricciardiello, Luigi ; Iwama, Takeo ; Rossi, Benedito M. ; Pellise, Maria ; Stoffel, Elena ; Wise, Paul E. ; Bertario, Lucio ; Saunders, Brian ; Burt, Randall ; Belluzzi, Andrea ; Ahnen, Dennis ; Matsubara, Nagahide ; Bülow, Steffen ; Jespersen, Niels ; Clark, Susan K. ; Erdman, Steven H. ; Markowitz, Arnold J. ; Bernstein, Inge ; De Haas, Niels ; Syngal, Sapna ; Moeslein, Gabriela. / A proposed staging system and stage-specific interventions for familial adenomatous polyposis. In: Gastrointestinal Endoscopy. 2016 ; Vol. 84, No. 1. pp. 115-125.e4.
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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

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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.

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