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Sirolimus for Colon Polyposis in PTEN Hamartoma Tumor Syndrome
Peter P Stanich1, Kebire Gofar1, Maegan E Roberts2
1Division of Gastroenterology, Hepatology & Nutrition, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
Introduction:
PTEN hamartoma tumor syndrome (PHTS) is a rare condition with a high rate of colon polyposis. Sirolimus may reduce colorectal polyps in PHTS. We designed a trial to assess the safety and impact of sirolimus on colon polyp burden in PHTS.
Methods:
We performed an open-label trial of sirolimus for 1 year in adults with a PTEN pathogenic variant and colon polyposis. The primary outcome was change in polyp burden and staging.
Results:
Five participants were enrolled, with 2 completing the planned study course. Baseline colonoscopies showed a mean polyp burden of 107 (range 31-184), with multiple histologic types. For the 2 patients completing the study, polyp burden decreased.
Discussion:
Sirolimus has promise for reduction of colon polyp burden in PHTS, but side effects may limit usage. Future treatment trials in rare polyposis syndromes would benefit from the involvement of multiple centers to allow for improved recruitment.
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