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@ARTICLE{vanTilburg:131762,
author = {C. M. van Tilburg$^*$ and F. Selt$^*$ and F. Sahm$^*$ and
H. Bächli and S. Pfister$^*$ and O. Witt$^*$ and T.
Milde$^*$},
title = {{R}esponse in a child with a {BRAF} {V}600{E} mutated
desmoplastic infantile astrocytoma upon retreatment with
vemurafenib.},
journal = {Pediatric blood $\&$ cancer},
volume = {65},
number = {3},
issn = {1545-5009},
address = {New York, NY},
publisher = {Wiley},
reportid = {DKFZ-2018-00061},
pages = {e26893 -},
year = {2018},
abstract = {Infants with low-grade glioma (LGG) and diencephalic
syndrome have a poor outcome. The patient described here had
a desmoplastic infantile astrocytoma harboring a BRAF V600E
mutation. After relapse following initial standard
chemotherapy treatment, he was successfully treated with the
BRAF V600E inhibitor vemurafenib at the age of 3 years 11
months and 5 years 0 months. A rapid response was observed
on both occasions. This illustrates the possibility of
continuous oncogenic addiction and the therapeutic potential
of BRAF V600E inhibitor monotherapy in LGG, even in very
young severely compromised children. BRAF V600E inhibition
in LGG and possible (re-)treatment regimens are briefly
discussed.},
cin = {L101 / G340 / G380 / B062},
ddc = {610},
cid = {I:(DE-He78)L101-20160331 / I:(DE-He78)G340-20160331 /
I:(DE-He78)G380-20160331 / I:(DE-He78)B062-20160331},
pnm = {317 - Translational cancer research (POF3-317)},
pid = {G:(DE-HGF)POF3-317},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:29230924},
doi = {10.1002/pbc.26893},
url = {https://inrepo02.dkfz.de/record/131762},
}