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.