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@ARTICLE{Selt:130542,
author = {F. Selt$^*$ and A. Deiß$^*$ and A. Korshunov$^*$ and D.
Capper$^*$ and H. Witt$^*$ and C. M. van Tilburg$^*$ and D.
Jones$^*$ and R. Witt$^*$ and F. Sahm$^*$ and D. Reuss$^*$
and C. Kölsche$^*$ and J. Ecker$^*$ and I. Oehme$^*$ and T.
Hielscher$^*$ and A. von Deimling$^*$ and A. E. Kulozik and
S. Pfister$^*$ and O. Witt$^*$ and T. Milde$^*$},
title = {{P}ediatric {T}argeted {T}herapy: {C}linical {F}easibility
of {P}ersonalized {D}iagnostics in {C}hildren with
{R}elapsed and {P}rogressive {T}umors.},
journal = {Brain pathology},
volume = {26},
number = {4},
issn = {1015-6305},
address = {Oxford},
publisher = {Wiley-Blackwell},
reportid = {DKFZ-2017-05621},
pages = {506 - 516},
year = {2016},
abstract = {The 'pediatric targeted therapy' (PTT) program aims to
identify the presence and activity of druggable targets and
evaluate the clinical benefit of a personalized treatment
approach in relapsed or progressive tumors on an individual
basis. 10 markers (HDAC2, HR23B, p-AKT, p-ERK, p-S6, p-EGFR,
PDGFR-alpha/beta, p53 and BRAFV600E) were analyzed by
immunohistochemistry. Pediatric patients with tumors
independent of the histological diagnosis, with relapse or
progression after treatment according to standard protocols
were included. N = 61/145 $(42\%)$ cases were eligible
for analysis between 2009 and 2013, the most common entities
being brain tumors. Immunohistochemical stainings were
evaluated by the H-Score (0-300). In $93\%$ of the cases
potentially actionable targets were identified. The
expressed or activated pathways were histone deacetylase
(HDACs; $83.0\%$ of cases positive), EGFR $(87.2\%),$ PDGFR
$(75.9\%),$ p53 $(50.0\%),$ MAPK/ERK $(43.3\%)$ and
PI3K/mTOR $(36.1\%).$ Follow-up revealed partial or full
implementation of PTT results in treatment decision-making
in $41\%$ of the cases. Prolonged disease stabilization
responses in single cases were noticed, however, response
rates did not differ from cases treated with other
modalities. Further studies evaluating the feasibility and
clinical benefit of personalized diagnostic approaches using
paraffin material are warranted.},
cin = {G340 / G380 / B062 / C060 / G040 / L101},
ddc = {610},
cid = {I:(DE-He78)G340-20160331 / I:(DE-He78)G380-20160331 /
I:(DE-He78)B062-20160331 / I:(DE-He78)C060-20160331 /
I:(DE-He78)G040-20160331 / I:(DE-He78)L101-20160331},
pnm = {317 - Translational cancer research (POF3-317)},
pid = {G:(DE-HGF)POF3-317},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:26445087},
doi = {10.1111/bpa.12326},
url = {https://inrepo02.dkfz.de/record/130542},
}