Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life
Status:
Active, not recruiting
Trial end date:
2023-03-01
Target enrollment:
Participant gender:
Summary
Congenital vascular anomalies are uncommon and belong to the group of rare diseases.These
vascular malformations can cause serious complications including obstruction of vital organs
and their function, recurrent infection and significantly reduced quality of life of persons
affected.Treatment options range from conservative to surgical extirpation or intralesional
embolisation/sclerosis. Unfortunately, this is often not enough. Many patients still have
complaints like severe pain and invalidation due to the lymphatic or venous malformation
making a normal functional life impossible. Recent case reports mention the positive effects
of refractory patients with Sirolimus. Sirolimus, also known as rapamycin, is currently the
only FDA-approved mammalian target of rapamycin (mTOR) inhibitor.