Response to trametinib treatment in progressive pediatric low-grade glioma patients.

ABSTRACT:

Introduction

A hallmark of pediatric low-grade glioma (pLGG) is aberrant signaling of the mitogen activated protein kinase (MAPK) pathway. Hence, inhibition of MAPK signaling using small molecule inhibitors such as MEK inhibitors (MEKi) may be a promising strategy.

Methods

In this multi-center retrospective centrally reviewed study, we analyzed 18 patients treated with the MEKi trametinib for progressive pLGG as an individual treatment decision between 2015 and 2019. We have investigated radiological response as per central radiology review, molecular classification and investigator observed toxicity.

Results

We observed 6 partial responses (PR), 2 minor responses (MR), and 10 stable diseases (SD) as best overall responses. Disease control rate (DCR) was 100% under th

SUBMITTER: Selt F 

PROVIDER: S-EPMC7609413 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature