Efficacy and Safety of Mammalian Target of Rapamycin Inhibitors in Vascular Anomalies: A Systematic Review

Nadal, Marion
Giraudeau, Bruno
Tavernier, Elsa
Jonville-Bera, Annie-Pierre
Lorette, Gérard
Maruani, Annabel

Open link

Publication date

January 2016

DOI

10.2340/00015555-2300

ISSN

0001-5555

Citation count (estimate)

Abstract

Mammalian target of rapamycin (mTOR) inhibitors are a promising new treatment in vascular anomalies, but no published randomized controlled trials are available. The aim of this systematic review of all reported cases was to assess the efficacy and safety of mTOR inhibitors in all vascular anomalies, except cancers, in children and adults. In November 2014 MEDLINE, CENTRAL, LILACS and EMBASE were searched for studies of mTOR inhibitors in any vascular condition, except for malignant lesions, in humans. Fourteen publications and 9 posters, with data on 25 and 59 patients, respectively, all <18 years old were included. Of these patients, 35.7% (n=30) had vascular tumours, and 64.3% (n=54) had malformations. Sirolimus was the most frequent mTO...