In a groundbreaking development, the interim analysis of the large multicenter Phase III VASE (Vascular Anomaly-Sirolimus Europe) trial, using sirolimus (Rapamune from Pfizer), to address slow-flow vascular malformations has been published in JCI Insight. This marks a significant milestone in the ongoing study and provides insight into the potential efficacy of this treatment.
This study is led by the research team of Prof. Laurence M. BOON and Prof. Miikka VIKKULA (members of the VASCERN Vascular Anomalies Working Group) at the Cliniques universitaires Saint-Luc- Université Catholique de Louvain. It aims to evaluate the efficacy of sirolimus for the treatment of slow-flow vascular malformations and the associated safety profile in children and adults.
The published results are promising, showing that 70-80% of patients benefit from sirolimus treatment. This groundbreaking information opens up new possibilities for the treatment of slow-flow vascular malformations and offers hope to individuals affected by this condition.
While the interim analysis provides valuable insights, the study is far from concluded. With all 250 patients now enrolled, the research team is preparing for the next phase of the study, which promises even more comprehensive data and a longer follow-up period. This continued investigation will contribute significantly to our understanding of sirolimus and its potential impact on vascular malformations.
For more details and to access the published interim analysis, please click here. Stay tuned for further updates as the VASE trial progresses towards its final results.
Reference: 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. PMID: 37937645.