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SAFETY AND EFFICACY OF CHEMOPREVENTION FOR FAMILIAL ADENOMATOUS POLYPOSIS: A SYSTEMATIC REVIEW AND META-ANALYSIS
Kaique Flávio Xavier Cardoso Filardi
4, Thaís Cabral de Melo Viana
2, Eric T. Nakamura
2, Amanda Park
3, Rodrigo Moisés d. Leite
1, Pedro Luiz Serrano Usón Junior
1, Francisco Tustumi
*11Sociedade Beneficente Israelita Brasileira Albert Einstein, Sao Paulo, São Paulo, Brazil; 2Universidade de Sao Paulo, Sao Paulo, Brazil; 3Centro Universitario Lusiada, Santos, São Paulo, Brazil; 4Beth Israel Deaconess Medical Center, Boston, MA
Introduction: Familial adenomatous polyposis (FAP) is a hereditary disorder resulting from mutations in the APC gene, significantly increasing the risk of colorectal cancer (CRC). This review explores the efficacy and safety of pharmacological therapies aimed at preventing cancer and precancerous conditions in individuals with FAP.
Methods: A systematic review was conducted following PRISMA guidelines (PROSPERO: CRD42024575526). Searches were performed in Embase, PubMed, and Cochrane databases to identify longitudinal studies evaluating chemopreventive drugs in FAP patients. Key outcomes assessed included cumulative polyp number, polyp size, and adverse events. Results were expressed as risk difference (RD) or mean difference (MD).
Results: Out of 366 search results, 18 controlled trials and one retrospective cohort study, comprising a total of 1008 patients, were included. On average, 47% of participants were female, with a mean age of 15.1 years (±22.3) and a mean follow-up duration of 15 months (±16). Among the included studies, 61.1% focused on colorectal polyps, 16.7% on rectal polyps, and 27.8% on duodenal polyps. Metformin, ursodeoxycholic acid, non-selective COX inhibitors, and COX-2-specific inhibitors were the main drugs evaluated. Pharmacological interventions significantly reduced polyp burden (MD: -1.03, 95% CI: -1.91, -0.16), notably with non-selective COX inhibitors (MD: -1.86, 95% CI: -3.69, -0.03) and metformin (MD: -1.06, 95% CI: -1.86, -0.27). A significant reduction in polyp number (MD: -0.56, 95% CI: -1.01, -0.12) was also observed, especially with non-selective COX inhibitors (MD: -0.85, 95% CI: -1.72, 0.01), along with reductions in polyp size (MD: -0.26, 95% CI: -0.48, -0.04). Pre and post-treatment comparisons indicated substantial reductions in polyp number (MD: -1.24, 95% CI: -1.71, -0.76) and size (MD: -1.76, 95% CI: -2.52, -1.01). Non-selective COX inhibitors had the most substantial impact, with MD of -1.56 (polyp number) and -2.42 (polyp size). Overall adverse events were low and showed similar results to those of placebo or no-drug groups (RD: -0.03, 95% CI: -0.15, 0.10). Chemoprevention did not increase the risk of headache, abdominal pain, diarrhea, nausea or vomiting, and bleeding. No significant differences in severe adverse events were observed.
Conclusions: These findings suggest that pharmacological interventions, particularly non-selective COX inhibitors, are associated with a reduction in polyp number, burden, and size, with a favorable safety profile.
Baseline characteristics of the included studies. Out of 366 search results, 18 controlled trials and one retrospective cohort study, comprising a total of 1008 patients, were included. On average, 47% of participants were female, with a mean age of 15.1 years (±22.3) and a mean follow-up duration of 15 months (±16). Among the included studies, 61.1% focused on colorectal polyps, 16.7% on rectal polyps, and 27.8% on duodenal polyps. Metformin, ursodeoxycholic acid, non-selective COX inhibitors, and COX-2-specific inhibitors were the main drugs evaluated.
Safety and efficacy outcomes categorized by the class of chemopreventive drugs. Results are presented as risk differences or mean differences (Hedges' g), along with 95% confidence intervals (95% CI). Statistical heterogeneity was assessed using the I
2 statistic.
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