Article
Targeted treatment of severe vascular malformations harboring PIK3CA and TEK mutations with alpelisib is highly effective with limited toxicity.
Scientific reports - 28 Jun 2023
Sterba Martin, Pokorna Petra, Faberova Renata, Pinkova Blanka, Skotakova Jarmila, Seehofnerova Anna, Blatny Jan, Janigova Lucia, Koskova Olga, Palova Hana, Mahdal Michal, Pazourek Lukas, Jabandziev Petr, Slaby Ondrej, Mudry Peter, Sterba Jaroslav
Abstract excerpt
This was a prospective cohort study of eighteen patients with large and debilitating vascular malformations with one or more major systemic complications. In all patients, we discovered activating alterations in either TEK or PIK3CA. Based on these findings, targeted treatment using the PI3K inhibitor alpelisib was started with regular check-ups, therapy duration varied from 6 to 31 months. In all patients,...
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