Sirolimus (rapamycin) for slow-flow malformations in children: the observational-phase randomized clinical PERFORMUS trial

A Maruani, E Tavernier, O Boccara… - JAMA …, 2021 - jamanetwork.com
Importance Sirolimus is increasingly being used to treat various vascular anomalies,
although evidence of its efficacy is lacking. Objective To assess the efficacy and safety of
sirolimus for children with slow-flow vascular malformations to better delineate the
indications for treatment. Design, Setting and Participants This multicenter, open-label,
observational-phase randomized clinical trial included 59 children aged 6 to 18 years with a
slow-flow vascular malformation who were recruited between September 28, 2015, and …