Klippel-Trenaunay-Weber Syndrome Medication

Updated: Apr 11, 2022
  • Author: Camila K Janniger, MD; Chief Editor: Dirk M Elston, MD  more...
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Medication

Medication Summary

Alpelisib (Vijoice) is the first drug approved for patients aged 2 years and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy.

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PI3K Inhibitors

Class Summary

Gain-of-function mutations in the gene encoding the catalytic alpha-subunit of phosphatidylinositol-3 kinase (PIK3CA) lead to activation of PI3K-alpha and Akt-signaling cellular transformation. Many, but not all patients with KTS have been found to have PIK3CA gene mutations. [38]  

Alpelisib (Vijoice)

Indicated for patients aged 2 years and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy.

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