TY  - JOUR
AU  - Selt, Florian
AU  - van Tilburg, Cornelis M
AU  - Bison, Brigitte
AU  - Sievers, Philipp
AU  - Harting, Inga
AU  - Ecker, Jonas
AU  - Pajtler, Kristian W
AU  - Sahm, Felix
AU  - Bahr, Annabelle
AU  - Simon, Michèle
AU  - Jones, David T W
AU  - Well, Lennart
AU  - Mautner, Victor-Felix
AU  - Capper, David
AU  - Hernáiz Driever, Pablo
AU  - Gnekow, Astrid
AU  - Pfister, Stefan M
AU  - Witt, Olaf
AU  - Milde, Till
TI  - Response to trametinib treatment in progressive pediatric low-grade glioma patients.
JO  - Journal of neuro-oncology
VL  - 149
IS  - 3
SN  - 1573-7373
CY  - Dordrecht [u.a.]
PB  - Springer Science + Business Media B.V
M1  - DKFZ-2020-02154
SP  - 499-510
PY  - 2020
N1  - #EA:B310#LA:B310# 2020 Sep;149(3):499-510
AB  - 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.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.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
LB  - PUB:(DE-HGF)16
C6  - pmid:33026636
DO  - DOI:10.1007/s11060-020-03640-3
UR  - https://inrepo02.dkfz.de/record/163930
ER  -