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Updated: Jun 23, 2025

A Patient-Derived Xenograft Model for Venous Malformation
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Targeted therapy for capillary-venous malformations.

Lola Zerbib1,2, Sophia Ladraa1,2, Antoine Fraissenon2,3,4,5

  • 1Université Paris Cité, Paris, France.

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|June 16, 2024
PubMed
Summary
This summary is machine-generated.

Alpelisib effectively treats PIK3CA-related capillary venous malformations by inhibiting PI3Kα. This therapy improved lesions and prolonged survival in a mouse model and showed significant volume reduction in patients with PIK3CA or TEK mutations.

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Area of Science:

  • Genetics
  • Vascular Biology
  • Pharmacology

Background:

  • Sporadic venous malformations stem from PIK3CA or TEK mutations, causing significant health issues with no approved treatments.
  • These malformations lead to pain, bleeding, thrombosis, and potentially life-threatening complications.
  • Current therapies like surgery and sclerotherapy are often insufficient.

Purpose of the Study:

  • To develop and validate a PIK3CA-related capillary venous malformation mouse model.
  • To evaluate the efficacy of PI3Kα inhibitors, specifically alpelisib, in treating these malformations.
  • To assess alpelisib's safety and effectiveness in human patients with refractory venous malformations.

Main Methods:

  • Created a genetic mouse model mimicking human PIK3CA-related capillary venous malformations.
  • Compared alpelisib (PI3Kα inhibitor) with other drugs like rapamycin and miransertib in the mouse model.
  • Conducted a clinical trial treating 25 patients (including children) with PIK3CA or TEK mutations using alpelisib, assessing outcomes via MRI.

Main Results:

  • Alpelisib demonstrated efficacy in preventing, improving, and prolonging survival in the mouse model.
  • All 25 patients treated with alpelisib showed improvement, with reduced vascular malformation volume (33.4% for PIK3CA, 27.8% for TEK) over 6 months.
  • Clinical symptoms were attenuated, and previously intractable malformations responded to alpelisib therapy.

Conclusions:

  • PI3Kα inhibition with alpelisib is a promising therapeutic strategy for PIK3CA or TEK-related capillary venous malformations.
  • Alpelisib offers a viable treatment option for patients, including children, with severe or refractory venous malformations.
  • This study validates the therapeutic potential of targeting the PI3Kα pathway in vascular malformations.