Abstract
Prospective short-term studies on effectiveness of non-steroidal anti-inflammatory drugs (NSAIDs) point towards a decrease in the number and size of polyps. Effectiveness and safety in the prevention of progression in familial polyposis with NSAIDs in long-term use, which is the prerequisite for therapeutic evaluation in prospective studies, is unknown. The total absolute observation period of 54 patients under sulindac was 399 patient years with a mean of 7.4 (2–19) years per patient. 36 patients (66.7%) showed a fast decrease of polyp burden, 8 (14.8%) were slow responders, and 9 (16.7%) had stable disease; one patient had a slow progression. Upper gastrointestinal (GI) polyp burden remained stable in 47% patients, increased in 31%, and improved in 22%. Advanced adenomas were found in 8 patients only within the first 5 years of chemoprevention, no patient developed desmoid disease, anamnestically evaluated on every follow-up. There were no life-threatening side-effects. Dosage and delivery pattern were essential for effectiveness. This study provides evidence that chemoprevention with sulindac is effective and safe and can, either alone or in combination with other drugs, become a long-term management option in cases of adenomatous polyposis. These results justify further long-term prospective chemoprevention studies to elaborate treatment protocols and guidelines.
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The datasets generated during and/or analyzed during the current study are available from the corresponding author upon reasonable request.
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Acknowledgements
We thank the endoscopists Wilhelm Höchter, Josef Weingart, Gerhard Feder, and Bernd Birkner.
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Study concept and design (EHF, TMN, VSL); acquisition of data (TMN, VSL, KH); analysis and interpretation of data (EHF, TMN, VSL, KH, MM); drafting of the manuscript (EHF, TMN, VSL, KH); critical revision of the manuscript for important intellectual content (MM, AL, ML).
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Yes, project no. 19-005, ethics committee of the Ludwig Maximilian University Munich, Germany (28.05.2019).
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10689_2022_292_MOESM1_ESM.jpg
Supplementary Fig. 1: Colonoscopy findings in a young patient (age 15 years at diagnosis) with APC pathogenic variant. (top = sigmoid and bottom = rectum). Left—colonoscopy findings at time of diagnosis. Center—colonoscopy findings after two years of sulindac therapy. Right—colonoscopy findings after five years. (JPG 138 kb)
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Neuhann, T.M., Haub, K., Steinke-Lange, V. et al. Long-term chemoprevention in patients with adenomatous polyposis coli: an observational study. Familial Cancer 21, 463–472 (2022). https://doi.org/10.1007/s10689-022-00292-2
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DOI: https://doi.org/10.1007/s10689-022-00292-2