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Clinical Response to PI3K-α Inhibition in a Cohort of Children and Adults With PIK3CA-Related Overgrowth Spectrum Disorders

OBJECTIVE: The goal of this report is to describe, through a series of 5 cases, the clinical response and safety of alpelisib (BYL719) use in children and adults with PIK3CA-related overgrowth spectrum (PROS) disorders at our center. METHODS: We reviewed clinical records of 5 patients from October 2...

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Detalles Bibliográficos
Autores principales: Raghavendran, Prashant, Albers, Sharon E., Phillips, James D., Zarnegar-Lumley, Sara, Borst, Alexandra J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9592062/
https://www.ncbi.nlm.nih.gov/pubmed/36284525
http://dx.doi.org/10.1097/jova.0000000000000038