ArticleBMJ open gastroenterology2020
Sirolimus for the treatment of polyposis of the rectal remnant and ileal pouch in four patients with familial adenomatous polyposis: a pilot study.
Article in BMJ open gastroenterology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03095703 (Sirolimus for the Treatment of Severe Intestinal Polyposis in Patients With Familial Adenomatous Polyposis), which is not on this map. Cited by 14 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
Sirolimus for the Treatment of Severe Intestinal Polyposis in Patients With Familial Adenomatous Polyposis (FAP): a Pilot Study
Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 19 citations in OpenAlex.
- Updated European guidelines for clinical management of familial adenomatous polyposis (FAP), MUTYH-associated polyposis (MAP), gastric adenocarcinoma, proximal polyposis of the stomach (GAPPS) and other rare adenomatous polyposis syndromes: a joint EHTG-ESCP revision.The British journal of surgery · 2024Guideline
- Trial
- Advances in chemoprevention of familial adenomatous polyposis.Frontiers in oncology · 2026Review
- Familial adenomatous polyposis: non-surgical management of large bowel disease: endoscopic and chemoprevention strategies.Familial cancer · 2025Review
- Familial adenomatous polyposis: non-surgical management of large bowel disease: endoscopic and chemoprevention strategies.Familial cancer · 2025Review
- Chemoprevention in Inherited Colorectal Cancer Syndromes.Clinics in colon and rectal surgery · 2024Review
- Endoscopic and chemopreventive management of familial adenomatous polyposis syndrome.Familial cancer · 2023Review
- Molecular Pathways of Carcinogenesis in Familial Adenomatous Polyposis.International journal of molecular sciences · 2023Review
- Updated Perspectives on the Diagnosis and Management of Familial Adenomatous Polyposis.The application of clinical genetics · 2023Review
- Chemoprevention in hereditary digestive neoplasia: A comprehensive review.Therapeutic advances in gastroenterology · 2023Review
- Hereditary Colorectal Cancer Syndromes: Molecular Genetics and Precision Medicine.Biomedicines · 2022Review
- Article
- The CHAMP-study: the CHemopreventive effect of lithium in familial AdenoMatous Polyposis; study protocol of a phase II trial.BMC gastroenterology · 2022Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveAfter prophylactic colectomy, adenomas continue to develop in the remaining intestine of patients with familial adenomatous polyposis (FAP). There is a lack of standard clinical recommendation for chemoprevention in patients with FAP. Because of promising in vivo studies, the aim of this pilot study was to investigate the safety of sirolimus and its effect on progression of intestinal adenomas.
designPatients with FAP with InSiGHT Polyposis Staging System 3 of the retained rectum or pouch received sirolimus for 6 months, dosed at plasma concentration levels of 5-8 µg/L. Primary outcomes were safety and change in marked polyp size. Secondary outcomes were change in number of polyps and effect on proliferation and apoptosis assessed by immunohistochemistry.
resultsEach of the included four patients reported 4 to 18 adverse events (toxicity grades 1-3). One patient prematurely terminated the study because of adverse events. Marked polyp size decreased in 16 (80%)/20 and remained the same in 4 (20%)/20 patients. The number of polyps decreased in all patients (MD -25.75, p=0.13). Three out of four patients showed substantial induction of apoptosis or inhibition of proliferation.
conclusionSix months of sirolimus treatment in four patients with FAP showed promising effects especially on the number of polyps in the rectal remnant and ileal pouch, although at the cost of numerous adverse events. TRIAL REGISTRATION NUMBER: ClinicalTrials.gov ID NCT03095703.
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Registered trials
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.