Evidence map›Paper›PMID 40478255›Full record

Trial reportClinical and translational gastroenterology2025

Sirolimus for Colon Polyposis in PTEN Hamartoma Tumor Syndrome.

Peter P Stanich, Kebire Gofar, Maegan E Roberts, Hisham Hussan

Abstract readClinical Trial
In one paragraph

Trial report in Clinical and translational gastroenterology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Peter P StanichDivision of Gastroenterology, Hepatology & Nutrition, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.ORCID 0000-0002-6844-640
Kebire GofarDivision of Gastroenterology, Hepatology & Nutrition, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
Maegan E RobertsDivision of Human Genetics, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA.
Hisham HussanDivision of Gastroenterology and Hepatology, UC Davis Medical Center, Sacramento, California, USA .

Funding

PTEN Research, Pfizer
6 · The paper itself

Abstract

introductionPTEN 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.

methodsWe 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.

resultsFive 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.

Indexed as

Colonic PolypsHamartoma Syndrome, MultipleSirolimusAdultAgedColonColonoscopyFemaleHumansMaleMiddle AgedPTEN PhosphohydrolaseTreatment OutcomePTEN PhosphohydrolasePTEN protein, humanSirolimuscolon polypsCowden syndromepolyposissirolimus

Identifiers

PMID40478255
PMCPMC12377304

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.