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Targeted therapies for vascular malformations.

Gavin Kane1, Israel Fernandez-Pineda1

  • 1Children's Health Ireland, Dublin, Ireland.

Frontiers in Medicine
|September 18, 2024
PubMed
Summary

Targeted therapies for vascular malformations are advancing, offering new hope for pediatric patients. Further research is needed to optimize treatment duration, discontinuation, and combination strategies.

Area of Science:

  • Vascular biology
  • Translational medicine
  • Genetics

Background:

  • Vascular malformations (VMs) are congenital conditions.
  • Advances in genetic identification are driving targeted therapy development.
  • Translational research bridges basic science and clinical practice for VMs.

Purpose of the Study:

  • To review recent studies on targeted therapies for VMs.
  • To focus on the pediatric population.
  • To identify future research needs.

Main Methods:

  • Mini-review of published literature.
  • Focus on studies involving genetic mutations in VMs.
  • Emphasis on pediatric cases.

Main Results:

Keywords:
malformationpediatrictargetedtherapyvascular

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  • Emerging targeted therapies show promise for VM treatment.
  • Genetic insights are enabling personalized treatment approaches.
  • Paediatric applications are a key focus.

Conclusions:

  • Targeted therapies represent a significant advancement in VM treatment.
  • Further research is crucial for optimizing treatment protocols.
  • Future studies should address treatment duration, discontinuation, combination therapies, and long-term effects.