Targeted treatment of severe vascular malformations harboring PIK3CA and TEK mutations with alpelisib is highly effective with limited toxicity

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 inhibit...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10307862/
https://www.ncbi.nlm.nih.gov/pubmed/37380669
http://dx.doi.org/10.1038/s41598-023-37468-4

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