TY  - JOUR
AU  - Karajannis, Matthias A
AU  - Legault, Geneviève
AU  - Fisher, Michael J
AU  - Milla, Sarah S
AU  - Cohen, Kenneth J
AU  - Wisoff, Jeffrey H
AU  - Harter, David H
AU  - Goldberg, Judith D
AU  - Hochman, Tsivia
AU  - Merkelson, Amanda
AU  - Bloom, Michael C
AU  - Sievert, Angela J
AU  - Resnick, Adam C
AU  - Dhall, Girish
AU  - Jones, David
AU  - Korshunov, Andrey
AU  - Pfister, Stefan
AU  - Eberhart, Charles G
AU  - Zagzag, David
AU  - Allen, Jeffrey C
TI  - Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas.
JO  - Neuro-Oncology
VL  - 16
IS  - 10
SN  - 1523-5866
CY  - Oxford
PB  - Oxford Univ. Press
M1  - DKFZ-2017-02849
SP  - 1408 - 1416
PY  - 2014
AB  - 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
KW  - Antineoplastic Agents (NLM Chemicals)
KW  - Phenylurea Compounds (NLM Chemicals)
KW  - Protein Kinase Inhibitors (NLM Chemicals)
KW  - Niacinamide (NLM Chemicals)
KW  - sorafenib (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:24803676
C2  - pmc:PMC4165419
DO  - DOI:10.1093/neuonc/nou059
UR  - https://inrepo02.dkfz.de/record/126821
ER  -