Emerging Systemic Medical Therapies: A New Treatment Paradigm for Complex Vascular Malformations

Learning Objectives: • Review known molecular pathways in the development of vascular malformations.
• Describe available and emerging systemic medical therapies that target molecular pathways.
• Discuss how these medications fit into the treatment paradigm along with minimally invasive procedures.
 

 

 

 


Background: Vascular malformations are congenital anomalies that arise due to sporadic genetic mutations and can be slow-flow, such as venous and lymphatic malformations, or high-flow, such as arteriovenous malformations. They can cause significant pain, disfigurement, and functional impairments.

Currently, first-line treatment for most lesions is with minimally invasive procedures such as embolization and sclerotherapy. Increasing understanding of the molecular pathways underlying vascular anomalies is resulting in the use and development of targeted systemic medical therapies.
 

 

 

 

Clinical Findings/Procedure Details: This exhibit will review the molecular pathways currently known to cause the development of vascular malformations. Well-established medications that target these pathways, such as sirolimus for slow-flow and bevacizumab for high-flow malformations, will be outlined.

Newer medications targeting the PI3K and RAS pathways that have emerged recently or are under research, such as alpelisib, trametinib, and miransertib, will be discussed. In light of current evidence, the indications, effectiveness, and safety profiles of these medications will be highlighted.

Integration with established minimally invasive therapies such as embolization and sclerotherapy will also be examined. The table summarizes systemic therapies for vascular malformations.
 

 

 

 

Conclusion and/or Teaching Points: Advances in molecular research have ushered in targeted medications for vascular malformations, providing new tools for managing complex lesions. To achieve the best clinical outcomes, interventional radiologists treating vascular anomalies should be well-informed about the changing landscape of treatments, including emerging systemic therapies.