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MINI REVIEW article

Front. Med.
Sec. Hematology
Volume 11 - 2024 | doi: 10.3389/fmed.2024.1446046
This article is part of the Research Topic Vascular Malformations: Advancements, Debates, and Consensus View all 4 articles

Targeted Therapies for Vascular Malformations

Provisionally accepted
  • Children’s Health Ireland, Dublin, Ireland

The final, formatted version of the article will be published soon.

    Targeted medical therapies for the treatment of vascular malformations is an exciting and evolving area of research. As the identification of specific causative genetic mutations involved in vascular malformations becomes more accessible and inexpensive, the development of targeted therapies to address these genetic anomalies becomes all the more enticing. It is an excellent example of the potential of translational research where basic science discoveries are translated to clinical practice from 'bench to bedside'. In this mini-review we aim to synopsise some of the recent studies published in this area with specific focus on the paediatric population. We also aim to highlight the growing demand for future research in the field to elucidate further the optimum duration of treatments, strategies for discontinuation, potential for combination of therapies and the effects of prolonged use of these medications.PIK3CA-Phosphatidylinositol-4,5-Bisphosphate 3-Kinase Catalytic Subunit Alpha TEK-TEK Receptor Tyrosine Kinase (also called TIE2 gene) KRAS= Kristen Rat Sarcoma Viral oncogene BRAF= v-raf murine sarcoma viral oncogene homolog B1 NRAS= neuroblastoma RAS viral oncogene homolog. CLOVES-Congenital lipomatous overgrowth, vascular malformations, epidermal nevi and skeletal anomalies

    Keywords: vascular, Malformation, targeted, therapy, pediatric

    Received: 08 Jun 2024; Accepted: 12 Aug 2024.

    Copyright: © 2024 Kane and Fernandez-Pineda. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Israel Fernandez-Pineda, Children’s Health Ireland, Dublin, Ireland

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.