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Clinical Report

Oral and Topical Sirolimus for Vascular Anomalies: A Multicentre Study and Review

Shira Sandbank, Vered Molho-Pessach, Adam Farkas, Aviv Barzilai, Shoshana Greenberger
DOI: 10.2340/00015555-3262

Preview of fully accepted paper, still not published in any volume

Abstract

Vascular anomalies (VAs) may be associated with significant morbidity and mortality. The aim of this study was to evaluate the efficacy and safety of sirolimus (rapamycin) in the treatment of children and young adults with complicated VAs. A retrospective chart was created that included 19 patients treated with sirolimus for complicated VAs. Concurrently, a search of the PubMed database for VA cases treated with sirolimus was conducted. Descriptive analysis was performed and the efficiency rate of sirolimus was calculated. This retrospective study included 19 patients, 17 of whom were treated with oral sirolimus and 2 with topical sirolimus. Clinical improvement occurred in 15 patients (79%). One patient experienced near-complete resolution. Only 2 patients showed poor response and discontinued treatment. The literature review analysed 150 cases of VA treated with sirolimus. Sirolimus was efficient in 85% of cases, including 5 cases of complete resolution. Sirolimus appears to be an effective and safe treatment for children and young adults with complicated VAs.

Significance

In recent years, sirolimus, an mTOR inhibitor, has become a new therapeutic option for patients with vascular anomalies that do not respond to other treatments. We report here a retrospective series of 19 young adults, children and neonates with complex vascular anomalies treated with sirolimus. Overall, clinical improvement was demonstrated in 15 patients (79%). In addition, an up-to-date literature review was performed and 150 cases of vascular anomalies treated with sirolimus were analysed. The results suggest that sirolimus is an effective and safe treatment. Further study is needed into the early use of sirolimus in cases of low-flow lesions, and overgrowth syndromes with low-flow components.

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