Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care

Summary

The phosphatidylinositol 3-kinase (PI3Kinase)/Protein Kinase B (AKT)/mammalian target of rapamycin (mTor) pathway plays a role on the development and the venous/lymphatic vascular organisations. The investigators want to study the efficacy and the safety of Rapamycin, an mTor inhibitor.

Timeline

Start

2016-01-25

Primary completion

2025-04-01

Completion

2030-04-01

Publications

• Seront E, Van Damme A, Coulie J, Vikkula M, Boon LM. Personalized sirolimus regimen for vascular malformations: a retrospective analysis of VASE cohort. Orphanet J Rare Dis. 2025 Dec 10;21(1):62. doi: 10.1186/s13023-025-04151-y.
• Seront E, Van Damme A, Legrand C, Bisdorff-Bresson A, Orcel P, Funck-Brentano T, Sevestre MA, Dompmartin A, Quere I, Brouillard P, Revencu N, De Bortoli M, Hammer F, Clapuyt P, Dumitriu D, Vikkula M, Boon LM. Preliminary results of the European multicentric phase III trial regarding sirolimus in slow-flow vascular malformations. JCI Insight. 2023 Nov 8;8(21):e173095. doi: 10.1172/jci.insight.173095.

Drugs

Indications