Current and emerging pharmacotherapies for treating vascular malformations
(2026) Expert Review of Clinical Pharmacology — p. 1-14 (2026)
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- Abstract
- Introduction: Vascular malformations are chronic, often progressive disorders for which conventional procedures frequently provide incomplete control. Advances in molecular genetics have revealed recurrent pathway alterations that now enable mechanism-based pharmacologic treatment. Areas covered: This narrative review summarizes current and emerging targeted therapies for vascular malformations, including mTOR, PI3K, MEK, anti-angiogenic, and genotype-specific inhibitors. Vascular malformations – capillary, lymphatic, venous, and arteriovenous – are increasingly recognized as disorders driven by dysregulation of the PI3K – AKT – mTOR and RAS – MAPK – ERK pathways, supporting rational repurposing of targeted anticancer drugs. We discuss clinical evidence, limitations, and practical considerations for sirolimus, PI3K inhibitors, thalidomide, MEK inhibitors, and KRAS- directed agents, as well as emerging combination and intermittent strategies to balance efficacy and toxicity. The review is based on a structured PubMed/MEDLINE search up to December 2025, prioritizing original translational studies, prospective trials, and large clinical series. Expert opinion: Targeted therapies are shifting management from procedure-centered to biology- guided care. Future progress depends on standardized molecular testing, patient-centered outcomes, and optimized treatment duration to achieve durable disease control with acceptable long-term toxicity.
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Citations
Seront, E., Van Damme, A., Coulie, J., Boon, L., & Vikkula, M. (2026). Current and emerging pharmacotherapies for treating vascular malformations. Expert Review of Clinical Pharmacology, 1-14. https://doi.org/10.1080/17512433.2026.2641807 (Original work published 2026)