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Clinical Report

Efficacy and Tolerance of Sirolimus (Rapamycin) for Extracranial Arteriovenous Malformations in Children and Adults

Romain Gabeff, Olivia Boccara, Véronique Soupre, Gérard Lorette, Christine Bodemer, Denis Herbreteau, Elsa Tavernier, Annabel Maruani
DOI: 10.2340/00015555-3273

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Managing extracranial arteriovenous malformations is challenging. Sirolimus (rapamycin) is increasingly being used when surgery and embolization are not advised. Because of its anti-angiogenic properties here we report all extracranial arteriovenous malformation cases treated with sirolimus in 2 French tertiary centers for vascular anomalies. The outcomes were efficacy (complete, partial, no response) based on arteriovenous malformation volume and necrosis/hemorrhage and side effects. We retrospectively included 10 patients (7 children). The sirolimus dose ranged from 0.6 to 3.5 mg/m2. Median (interquartile range [IQR]) treatment time was 24.5 (4.5; 35) months. Five patients showed no response and 5 showed partial response at a median (IQR) of 3 (1; 5) months followed in 2 cases by therapeutic resistance (i.e., progressive disease after 9 and 24 months of treatment). The most frequent side effect was mouth ulcers. This study shows poor efficacy of sirolimus for treating extracranial arteriovenous malformations.


Managing extracranial arteriovenous malformations is challenging. Sirolimus (rapamycin) is increasingly being used in all kinds of vascular anomalies. In this study we report the largest series of arteriovenous malformations treated with sirolimus which included 10 patients (7 children). Results suggest a poor efficacy of sirolimus for this condition with only partial response in 50% of patients which was transient and lasted several months in 2 of 5 patients with further worsening.

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