Content » Preview

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

Preview of a paper in the copy-editing process

This article has been accepted for publication in Acta Dermato-Venereologica and is currently being edited and typeset. Readers should note that article shown below have been fully refereed, but have not been through the copy-editing and proof correction process. Only Abstract is possible to read. When this process is finalized the complete paper will be able to find.

Abstract

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.

Significance

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.

Supplementary content

Comments

Not logged in! You need to login/create an account to comment on articles. Click here to login/create an account.