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Updated: Mar 10, 2026
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A Patient-Derived Xenograft Model for Venous Malformation
Published on: June 15, 2020
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Current and emerging pharmacotherapies for treating vascular malformations
Emmanuel Seront1, An Van Damme2, Julien Coulie3,4
1Institut Roi Albert II, Department of Medical Oncology, Cliniques Universitaires St Luc, University of Louvain, Brussels, Belgium.
Expert Review of Clinical Pharmacology
|March 8, 2026
Summary
Targeted therapies offer new hope for chronic vascular malformations by addressing underlying pathway dysregulation. This review explores emerging treatments like mTOR and PI3K inhibitors for improved patient outcomes.
Area of Science:
- Molecular genetics
- Pharmacology
- Vascular biology
Background:
- Vascular malformations are chronic, progressive disorders with limited treatment options.
- Advances in molecular genetics reveal pathway alterations driving these conditions.
- Mechanism-based pharmacologic treatments are now possible.
Purpose of the Study:
- To review current and emerging targeted therapies for vascular malformations.
- To discuss clinical evidence, limitations, and practical considerations for specific inhibitors.
- To explore future strategies for balancing efficacy and toxicity.
Main Methods:
- Narrative review based on a structured PubMed/MEDLINE search up to December 2025.
- Prioritization of original translational studies, prospective trials, and large clinical series.
- Focus on PI3K-AKT-mTOR and RAS-MAPK-ERK pathway dysregulation.
Main Results:
- Targeted therapies, including mTOR, PI3K, MEK, and anti-angiogenic inhibitors, show promise.
- Repurposing of targeted anticancer drugs is supported by pathway understanding.
- Emerging combination and intermittent strategies aim to optimize treatment.
Conclusions:
- Targeted therapies are transitioning care from procedural to biology-guided approaches.
- Standardized molecular testing is crucial for future progress.
- Optimizing treatment duration is key for durable disease control and acceptable toxicity.
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