Effectiveness and safety of sirolimus in the treatment of venous malformations: A meta-analysis of prospective studies.

Abstract

Background: Venous malformations are prevalent vascular anomalies. Recent clinical studies have explored the use of sirolimus for these conditions, particularly in patients who are not candidates for, or have shown limited response to, traditional treatments such as sclerotherapy and surgery. This meta-analysis systematically evaluates the efficacy and safety of sirolimus in treating venous malformations.

Methods: We conducted searches in PubMed, Cochrane Library, Web of Science, and the Cochrane Database of Systematic Reviews until August 10, 2024. The quality of the included prospective studies (including both randomized and single-arm pre-post trials) was assessed using the Cochrane risk of bias tool. Statistical analyses were performed with Review Manager (Version 5.4).

Results: Our analysis included eight prospective studies involving 74 patients. Primary outcomes measured changes in the volume and size of the malformations. Secondary outcomes assessed were functional disability scores, hemoglobin levels, coagulation indices, transfusion requirements, patient quality of life, and radiologic responses. Sirolimus demonstrated significant therapeutic benefits, with an odds ratio of 0.02 (95% confidence interval, 0.00-0.08) across six studies evaluating dichotomous variables. Results for continuous variables were consistent. Sirolimus showed safety in short-to medium-term use, with reversible mild to moderate side effects such as oral ulcers and liver function abnormalities. No severe adverse events (grade 3-5) were reported.

Conclusions: Sirolimus is effective and safe for treating venous malformations, especially in patients unresponsive to conventional therapies. Future studies should explore long-term effects, optimal dosages, and administration techniques.