Introduction
Several therapeutics are in development for familial adenomatous polyposis (FAP) to reduce polyp burden, the incidence of cancer and prevent or postpone intestinal surgery. However, there is limited consensus on the optimal method for measuring intestinal disease activity in FAP.
Aims & Methods
We aimed to systematically review existing endoscopic severity indices for FAP.
We searched MEDLINE, EMBASE and the Cochrane Library from inception to February 2023 to identify studies that evaluated endoscopic disease activity and severity in children and adults with FAP.
Results
A total of 94 studies were included. We evaluated component items of scoring indices, such as polyp count, polyp size and histology, and the scoring indices for the upper- and lower gastrointestinal tract . Partial validation was observed for polyp count and size. The most common reported scoring index was the Spigelman classification system, which was used for assessing the severity of duodenal disease. A single study reported an almost perfect inter- and intra-observer agreement for this system. The InSIGHT polyposis staging system, which was used for assessing colorectal polyp burden has been partially validated. It showed substantial inter-observer agreement, however it lacked the analysis of the intra-observer agreement. The SPACE criteria is a novel system to identify high-risk gastric polyps and was assessed for the inter-observer agreement; however it showed a moderate level of inter-observer agreement.
Conclusion
There are no fully validated endoscopic disease severity indices for FAP. Development and validation of reliable and responsive endoscopic disease severity instrument will be useful for clinical care, research and to assess the impact of pharmacological therapies on polyp burden in FAP.
Disclosure
Hicham Bouchiba: nothing to disclose
Anna L. Silverman: nothing to disclose
Arthur S. Aelvoet: nothing to disclose
John K. MacDonald: nothing to disclose
Brian G. Feagan: BGF has received grant/research support from AbbVie Inc., Amgen Inc., AstraZeneca/MedImmune Ltd., Atlantic Pharmaceuticals Ltd., Boehringer-Ingelheim, Celgene Corporation, Celltech, Genentech Inc/Hoffmann-La Roche Ltd., Gilead Sciences Inc., GlaxoSmithKline (GSK), Janssen Research & Development LLC., Pfizer Inc., Receptos Inc./Celgene International, Sanofi, Santarus Inc., Takeda Development Center Americas Inc., Tillotts Pharma AG, and UCB; consulting fees from Abbott/AbbVie, Akebia Therapeutics, Allergan, Amgen, Applied Molecular Transport Inc., Aptevo Therapeutics, Astra Zeneca, Atlantic Pharma, Avir Pharma, Biogen Idec, BioMx Israel, Boehringer-Ingelheim, Bristol-Myers Squibb, Calypso Biotech, Celgene, Elan/Biogen, EnGene, Ferring Pharma, Roche/Genentech, Galapagos, GiCare Pharma, Gilead, Gossamer Pharma, GSK, Inception IBD Inc, JnJ/Janssen, Kyowa Kakko Kirin Co Ltd., Lexicon, Lilly, Lycera BioTech, Merck, Mesoblast Pharma, Millennium, Nestle, Nextbiotix, Novonordisk, Pfizer, Prometheus Therapeutics and Diagnostics, Progenity, Protagonist, Receptos, Salix Pharma, Shire, Sienna Biologics, Sigmoid Pharma, Sterna Biologicals, Synergy Pharma Inc., Takeda, Teva Pharma, TiGenix, Tillotts, UCB Pharma, Vertex Pharma, Vivelix Pharma, VHsquared Ltd., and Zyngenia; speakers bureau fees from Abbott/AbbVie, JnJ/Janssen, Lilly, Takeda, Tillotts, and UCB Pharma; is a scientific advisory board member for Abbott/AbbVie, Allergan, Amgen, Astra Zeneca, Atlantic Pharma, Avaxia Biologics Inc., Boehringer-Ingelheim, Bristol-Myers Squibb, Celgene, Centocor Inc., Elan/Biogen, Galapagos, Genentech/Roche, JnJ/Janssen, Merck, Nestle, Novartis, Novonordisk, Pfizer, Prometheus Laboratories, Protagonist, Salix Pharma, Sterna Biologicals, Takeda, Teva, TiGenix, Tillotts Pharma AG, and UCB Pharma; and is the Senior Scientific Officer of Alimentiv Inc. (formerly Robarts Clinical Trials, Inc.).
Vipul Jairath: has received has received consulting/advisory board fees from AbbVie, Alimentiv Inc, Arena pharmaceuticals, Asahi Kasei Pharma, Asieris, Astra Zeneca, Avoro Capital, Bristol Myers Squibb, Celltrion, Eli Lilly, Endpoint Health, Ferring, Flagship Pioneering, Fresenius Kabi, Galapagos, Gilde Healthcare, GlaxoSmithKline, Genentech, Gilead, Janssen, Merck, Metacrine, Mylan, Pandion, Pendopharm, Pfizer, Protagonist, Prometheus Biosciences, Reistone Biopharma, Roche, Sandoz, Second Genome, Sorriso pharmaceuticals, Takeda, Teva, Topivert, Ventyx, Vividion; speaker’s fees from, Abbvie, Ferring, Bristol Myers Squibb, Galapagos, Janssen Pfizer Shire, Takeda, Fresenius Kabi
CM: has received consulting fees from AbbVie, Alimentiv, Amgen, AVIR Pharma Inc, BioJAMP, Bristol Myers Squibb, Celltrion, Ferring, Fresenius Kabi, Janssen, McKesson, Mylan, Pendopharm, Pfizer, Prometheus Biosciences Inc., Roche, Sanofi, Takeda, Tillotts Pharma; speaker's fees from AbbVie, Amgen, AVIR Pharma Inc, Alimentiv, Bristol Myers Squibb, Ferring, Fresenius Kabi, Janssen, Organon, Pendopharm, Pfizer, Takeda; royalties from Springer Publishing; research support from Ferring, Pfizer.
Evelien Dekker: Endoscopic equipment on loan of FujiFilm. Research grant FujiFilm. Honoraria for consultancy from Olympus, Fujifilm, Ambu and InterVenn, and speakers' fees from Olympus, GI Supply, Norgine, IPSEN, PAION and FujiFilm.
Jewel Samadder: Consulting or Advisory Role: Janssen Research & Development, Cancer Prevention Pharmaceuticals, Recursion Pharmaceuticals