Clinical Trials Study
Copyright ©The Author(s) 2025. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Jul 7, 2025; 31(25): 105677
Published online Jul 7, 2025. doi: 10.3748/wjg.v31.i25.105677
Efficacy and safety of sirolimus in the treatment of gastrointestinal angiodysplasias
Qi Sun, Jin-Cheng Wu, Xiao Chen, Dong-Hao Li, Bai-Rong Li, Nian-Jun Xiao, Xiao-Ying Wang, Xin-Zhuo Tu, Shou-Bin Ning, Tao Sun
Qi Sun, Jin-Cheng Wu, Xiao Chen, Dong-Hao Li, Bai-Rong Li, Nian-Jun Xiao, Xiao-Ying Wang, Shou-Bin Ning, Tao Sun, Department of Gastroenterology, Air Force Medical Center, Beijing 100142, China
Jin-Cheng Wu, Department of Gastroenterology, Puning People’s Hospital, Puning 515300, Guangdong Province, China
Xin-Zhuo Tu, Department of Pathology, Air Force Medical Center, Beijing 100142, China
Co-first authors: Qi Sun and Jin-Cheng Wu.
Co-corresponding authors: Shou-Bin Ning and Tao Sun.
Author contributions: Sun Q and Wu JC contributed equally to this work, they designed the study, collected patients’ medical records, analyzed the data, and drafted the manuscript; Chen X, Li DH and Li BR recruited the patient and conceived the study; Xiao NJ, Wang XY and Tu XZ collected patients’ medical records; Ning SB and Sun T designed the study, reviewed and edited the manuscript; All the authors read and approved the manuscript; Sun Q and Wu JC has made equal contributions to the writing of the manuscript, data collection, and analysis, and is thus designated as a co-first author; Ning SB and Sun T, as the corresponding author, provided crucial guidance and support in the research design, methodology, and interpretation of results, while also being responsible for all communication during the manuscript submission, peer review, and publication processes.
Supported by the Air Force Medical Center Youth Talent Program Project, No. 22YXQN034; Capital Health Development Research Special Project, No. 2020-4-5123; and Beijing Haidian District Health and Wellness Development Scientific Research Cultivation Program, No. HP2021-03-80803.
Institutional review board statement: The study was reviewed and approved by the Ethics Committee of the Air Force Medical Center (approval No. 2021-97-PJ01).
Clinical trial registration statement: The clinical trial for this study has been registered. Initially, there was a lack of awareness regarding the registration requirement; however, the registration process has since been completed and is currently pending approval. Compliance with all necessary guidelines has been ensured.
Informed consent statement: All study participants, or their legal guardian, provided informed written consent prior to study enrollment.
Conflict-of-interest statement: The authors declare that they have no conflict of interest.
CONSORT 2010 statement: The authors have read the CONSORT 2010 Statement, and the manuscript was prepared and revised according to the CONSORT 2010 Statement.
Data sharing statement: No additional data are available.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Tao Sun, MD, PhD, Department of Gastroenterology, Air Force Medical Center, No. 30 Fucheng Road, Haidian District, Beijing 100142, China. suntao8703@126.com
Received: February 5, 2025
Revised: April 13, 2025
Accepted: June 17, 2025
Published online: July 7, 2025
Processing time: 151 Days and 21 Hours
Abstract
BACKGROUND

Gastrointestinal angiodysplasias (GIAD) causes recurrent bleeding, and current treatments have limitations. Sirolimus, a mammalian target of rapamycin inhibitor, shows promise in inhibiting abnormal angiogenesis.

AIM

To evaluate the efficacy and safety of sirolimus in reducing bleeding episodes and improving clinical outcomes in patients with GIAD.

METHODS

We conducted a self-controlled study with 11 patients taking oral sirolimus. Retrospective data were collected prior to treatment, and prospective data were gathered during the study. Efficacy was assessed primarily by comparing bleeding episodes before and after sirolimus, with measurements at 3 and 6 months post-administration. The initial dose was 0.8 mg/m² once daily, adjusted to maintain trough blood concentrations between 5-10 ng/mL. Secondary outcomes included hemoglobin (Hb) levels, blood transfusion volume, and vascular lesions. Safety was monitored by tracking adverse reactions.

RESULTS

The average number of bleeding episodes decreased significantly from 2.09 ± 1.04 to 1.00 ± 0.75 in the 3 months preceding treatment, and from 3.80 ± 1.93 to 2.00 ± 1.63 in the 6 months preceding treatment. Sirolimus also increased Hb levels, reduced the need for transfusions, and decreased vascular lesions, improving clinical outcomes. All adverse effects were mild and resolved or improved within 1 week to 1 month without stopping sirolimus or needing lipid-lowering treatment.

CONCLUSION

Sirolimus reduced bleeding and transfusion needs while improving Hb levels in GIAD patients. Although these findings are encouraging, the limited sample size and lack of a control group warrant caution. Future controlled trials with larger populations are needed to validate sirolimus’s potential in GIAD.

Keywords: Gastrointestinal angiodysplasias; Sirolimus; Bleeding; Efficacy; Safety

Core Tip: This study indicates that sirolimus, a mammalian target of rapamycin inhibitor, effectively reduces bleeding episodes and improves hemoglobin levels in patients with gastrointestinal angiodysplasias (GIAD). By decreasing transfusions requirements and reducing vascular lesions, sirolimus shows promise as a novel therapeutic strategy. The treatment was generally well-tolerated, with mild and manageable side effects. While these findings are encouraging, future larger, controlled trials are needed to validate sirolimus’s role in managing GIAD.