%0 Journal Article
%A Karajannis, Matthias A
%A Legault, Geneviève
%A Fisher, Michael J
%A Milla, Sarah S
%A Cohen, Kenneth J
%A Wisoff, Jeffrey H
%A Harter, David H
%A Goldberg, Judith D
%A Hochman, Tsivia
%A Merkelson, Amanda
%A Bloom, Michael C
%A Sievert, Angela J
%A Resnick, Adam C
%A Dhall, Girish
%A Jones, David
%A Korshunov, Andrey
%A Pfister, Stefan
%A Eberhart, Charles G
%A Zagzag, David
%A Allen, Jeffrey C
%T Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas.
%J Neuro-Oncology
%V 16
%N 10
%@ 1523-5866
%C Oxford
%I Oxford Univ. Press
%M DKFZ-2017-02849
%P 1408 - 1416
%D 2014
%X Activation of the RAS-RAF-MEK-ERK signaling pathway is thought to be the key driver of pediatric low-grade astrocytoma (PLGA) growth. Sorafenib is a multikinase inhibitor targeting BRAF, VEGFR, PDGFR, and c-kit. This multicenter phase II study was conducted to determine the response rate to sorafenib in patients with recurrent or progressive PLGA.Key eligibility criteria included age ≥ 2 years, progressive PLGA evaluable on MRI, and at least one prior chemotherapy treatment. Sorafenib was administered twice daily at 200 mg/m(2)/dose (maximum of 400 mg/dose) in continuous 28-day cycles. MRI, including 3-dimensional volumetric tumor analysis, was performed every 12 weeks. BRAF molecular testing was performed on tumor tissue when available.Eleven patients, including 3 with neurofibromatosis type 1 (NF1), were evaluable for response; 5 tested positive for BRAF duplication. Nine patients (82
%K Antineoplastic Agents (NLM Chemicals)
%K Phenylurea Compounds (NLM Chemicals)
%K Protein Kinase Inhibitors (NLM Chemicals)
%K Niacinamide (NLM Chemicals)
%K sorafenib (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:24803676
%2 pmc:PMC4165419
%R 10.1093/neuonc/nou059
%U https://inrepo02.dkfz.de/record/126821