5 year study of 6 children each year with vascular anomalies who have severe complications failing to respond to the usual treatments will be offered a new use of an old medication- sirolimus

Why is this study important? Vascular anomalies comprise a diverse group of diagnoses. The majority of these conditions are quite rare and have not been widely studied. Some of these lesions can impair vital structures, be deforming or even becoming life-threatening. Many children with complicated vascular anomalies have already failed the conventional treatment and are at risk of significant morbidity and mortality. Currently sirolimus is in clinical trials in various centres of Europe and United States of America. Case studies are suggestive of optimistic outcome. By doing this prospective study, we will be able to assess the response and safety of this medication in our own patients which will definitely be a valuable contribution to the wider medical world to shine light on the management of this rare complex conditions . The protocol is designed as a prospective research study. It involves children in the age group of Zero to Eighteen years. We envisage approximately 6 children per year for 5 years who have complex vascular anomalies will be deemed eligible for treatment with sirolimus. This includes children who are ineligible for or refractory to conventional treatments as well as children who are suffering from rare subtypes of vascular anomaly for whom there is no universally accepted standard therapy. BACKGROUND INFORMATION Vascular anomalies include a heterogeneous group of disorders that are categorized as vascular tumors or vascular malformations. The standard treatment options include resection, sclerotherapy and cytostatic chemotherapy. Some of these anomalies can be very complicated causing disfigurement, chronic pain and organ dysfunction with significant morbidity and mortality. These complicated vascular anomalies are quite rare and have not been widely studied. These patients have either failed the conventional treatment or unable to tolerate the available treatment due to life threatening side effects. 4 Supporting References from the review of literature 1) Adrienne M, MarySue W etal. Sirolimus for the treatment of complicated vascular anomalies in children. Paediatr Blood Cancer 2011;57:1018-1024. 2) Denise M, Cameron C etal. Efficacy and safety of sirolimus in the treatment of complicated vascular anomalies. Pediatrics2016Feb 18:137(2):1-10. 3) Lackner H, Karastaneva A etal. Sirolimus for the treatment of children with various complicated vascular anomalies. Eur J Pediatr 2015 Dec; 174(12):1579-84. 4) Laurence M, Jennifer H etal. Rapamycin as novel treatment for refractory to standard care venous malformation. ISSVA 2016 Conference abst