Integrated response analysis of pediatric low-grade gliomas during and after targeted therapy treatment.

ABSTRACT:

Background

Pediatric low-grade gliomas (pLGGs) are the most common central nervous system tumor in children, characterized by RAS/MAPK pathway driver alterations. Genomic advances have facilitated the use of molecular targeted therapies, however, their long-term impact on tumor behavior remains critically unanswered.

Methods

We performed an IRB-approved, retrospective chart and imaging review of pLGGs treated with off-label targeted therapy at Dana-Farber/Boston Children's from 2010 to 2020. Response analysis was performed for BRAFV600E and BRAF fusion/duplication-driven pLGG subsets.

Results

Fifty-five patients were identified (dabrafenib n = 15, everolimus n = 26, trametinib n = 11, and vemurafenib n = 3). Median duration of targeted ther

SUBMITTER: Tsai JW 

PROVIDER: S-EPMC10011805 | biostudies-literature | 2023 Jan-Dec

REPOSITORIES: biostudies-literature