Evaluating Sirolimus Rapamune Oral Treatment for Severe Arteriovenous Malformations in Children and Adults

What this Trial Is About

Researchers are evaluating the efficacy and safety of oral sirolimus in reducing the volume and symptoms of superficial arteriovenous malformations AVM in patients aged 2 years and older. This phase 2 study explores sirolimus, which has immunosuppressant, anti-tumor, and anti-vascular properties. The drugs potential to inhibit blood vessel growth may help treat AVMs, which currently have poorly understood mechanisms. This research is sponsored by Centre Hospitalier Universitaire, Amiens. Participants will receive sirolimus orally, starting at 2 mg per day with doses adjusted weekly over four weeks to reach and maintain a blood level of 8 to 12 ngml. If side effects occur but do not require stopping treatment, the dose may be lowered while keeping a minimum blood level of 3 ngml. Tablets are preferred, but a liquid form is available for children under 6 years or those with swallowing difficulties. To reduce early side effects, patients will take a corticosteroid, prednisolone, at 0.5 mgkg daily for the first week. During the 12-month treatment, participants will be monitored for treatment effectiveness and tolerability, with evaluations at 3, 6, 9, and 12 months. Quality of life assessments will be done before treatment and after one year. Researchers will observe changes in AVM volume and symptoms while tracking side effects. The total study duration for each participant is one year, with regular clinical assessments and blood tests to ensure safety and dose management.

Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations