Efficacy and Safety of Sirolimus in the Management of pediatric Vascular Tumors and Malformations- A Prospective Study

Vascular anomalies represent a broad spectrum of disorders ranging from a tiny “birthmark” to life threatening entities. They are difficult to treat clinical entities causing significant morbidity and functional impairment. Currently available treatment modalities like sclerotherapy/ embolization/ surgical excision are seldom curative with high recurrence rates. Medical management can be a way forward in this regard. Sirolimus has shown promising results in the past studies as an effective drug in management of vascular anomalies leading to decrease in size and improvement in functional status of the patients. Thus our research hypothesis is that the use of sirolimus will improve clinical symptoms, reduce the size, and improve the quality of life in symptomatic patients with vascular anomalies in 70% of patients after 3 months of use. This will be a single arm trial to be conducted at Department of paediatrics, AIIMS Delhi for a period of 2 years from july 2024 to july 2026. The primary objective will be to assesss the efficacy of Sirolimus in decreasing the size of vascular anomalies. The secondary objectives will be to assess the efficacy of sirolimus in decreasing the signs and symptoms of vascular anomalies, in improving the quality of life of the patients; and to assess its safety during the treatment. Additionally, data regarding the distribution of types of vascular anomalies will be recorded.