Genomic landscape of lymphatic malformations: a case series and response to the PI3Kα inhibitor alpelisib in an N-of-1 clinical trial

Background: Lymphatic malformations (LMs) often pose treatment challenges due to a large size or a critical location that could lead to disfigurement, and there are no standardized treatment approaches for either refractory or unresectable cases. Methods: We examined the genomic landscape of a patie...

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Main Authors: Montaser F Shaheen, Julie Y Tse, Ethan S Sokol, Margaret Masterson, Pranshu Bansal, Ian Rabinowitz, Christy A Tarleton, Andrey S Dobroff, Tracey L Smith, Thèrése J Bocklage, Brian K Mannakee, Ryan N Gutenkunst, Joyce Bischoff, Scott A Ness, Gregory M Riedlinger, Roman Groisberg, Renata Pasqualini, Shridar Ganesan, Wadih Arap
Format: Article
Language:English
Published: eLife Sciences Publications Ltd 2022-07-01
Series:eLife
Subjects:
Online Access:https://elifesciences.org/articles/74510