The aim of the study is to evaluate the efficacy and safety of sirolimus (oral form), to decrease the volume and symptoms due to superficial arteriovenous malformations (AVM).
Sirolimus has properties that reduce the activity of the immune system (immunosuppressant), to fight against the proliferation of cancer cells (anti- tumor) and also reduce the proliferation of blood vessels (anti -vascular). Sirolimus is primarily used in transplant patients to prevent organ transplant rejection. Many animal and laboratory studies were carried out and demonstrate in particular the activity of sirolimus on vessels. It is this anti- vascular effect that could help treat arteriovenous malformations.
Patients (adults, adolescents and children older than 2 years), with arteriovenous malformation stage II + III or IV (according to Schöbinger's classification) : active or quiescent, marked or not by hemorrhagic phenomena.
Patients (parents for minors) must sign a consent form established after clear information risks and expected benefits of the study.
Patients (major and minor of childbearing age) must have effective contraception during the study period and continuing until 12 weeks after the end of treatment
Negative pregnancy blood test for women of childbearing age.
patients with transplanted organ or who received a hematopoietic stem cell
patient with congenital immunodeficiency
Patients implanted with chronic active infection associated with hepatitis B , hepatitis C or HIV
Pregnant or nursing woman.
Single group
Sirolimus
DrugFor patients with swallowing problems, and for children under 6 years and / or who have an inability to swallow tablets, the 1mg/ml solution form should be used.
Treatment efficacy at M12
The efficacy of treatment is a composite criteria based on: * The proportion of patients with no evolution of the AVM during the study period, * The proportion of patients with a reduction in tumor volume of the AVM at least 30% of CT Angiography (CTA) criteria during the first year of the study (comparison of the volume of the AVM a year versus pre-inclusion).
Treatment efficacy at M3
Treatment efficacy at M6
Treatment efficacy at M9
Treatment tolerability
Number and description of serious advent events
Click on the lead sponsor to view all of their trials.
