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@ARTICLE{Chapman:274243,
author = {R. J. Chapman and D. R. Ghasemi$^*$ and F. Andreiuolo and
V. Zschernack and A. Tauziede Espariat and F. R. Buttarelli
and F. Giangaspero and J. Grill and C. Haberler and S. M. L.
Paine and I. Scott and T. S. Jacques and M. Sill and S.
Pfister$^*$ and J.-P. Kilday and P. Leblond and M. Massimino
and H. Witt$^*$ and P. Modena and P. Varlet and T. Pietsch
and R. G. Grundy and K. Pajtler$^*$ and T. A. Ritzmann and
B. o. E. i. Childhood},
collaboration = {Adolescence},
title = {{O}ptimising biomarkers for accurate ependymoma diagnosis,
prognostication and stratification within {I}nternational
{C}linical {T}rials: {A} {BIOMECA} study.},
journal = {Neuro-Oncology},
volume = {25},
number = {10},
issn = {1522-8517},
address = {Oxford},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2023-00529},
pages = {1871-1882},
year = {2023},
note = {#LA:B062# / 2023 Oct 3;25(10):1871-1882},
abstract = {Accurate identification of brain tumour molecular subgroups
is increasingly important. We aimed to establish the most
accurate and reproducible ependymoma subgroup biomarker
detection techniques, across 147 cases from International
Society of Pediatric Oncology (SIOP) Ependymoma II trial
participants, enrolled in the pan-European 'Biomarkers of
Ependymoma in Children and Adolescents (BIOMECA)'
study.Across six European BIOMECA laboratories we evaluated
epigenetic profiling (DNA methylation array);
immunohistochemistry (IHC) for nuclear p65-RELA, H3K27me3,
and Tenascin-C; copy number analysis via FISH and MLPA (1q,
CDKN2A), and MIP and DNA methylation array (genome-wide copy
number evaluation); analysis of ZFTA- and YAP1-fusions by
RT-PCR and sequencing, Nanostring and break-apart FISH.DNA
Methylation profiling classified $65.3\%$ (n=96/147) of
cases as EPN-PFA and $15\%$ (n=22/147) as ST-ZFTA
fusion-positive. Immunohistochemical loss of H3K27me3 was a
reproducible and accurate surrogate marker for EPN-PFA
(sensitivity $99-100\%$ across three centres). IHC for
p65-RELA, FISH, and RNA-based analyses effectively
identified ZFTA- and YAP1- fused supratentorial ependymomas.
Detection of 1q gain using FISH exhibited only $57\%$
inter-centre concordance and low sensitivity and specificity
whilst MIP, MLPA and DNA methylation-based approaches
demonstrated greater accuracy.We confirm, in a prospective
trial cohort, that H3K27me3 immunohistochemistry is a robust
EPN-PFA biomarker. Tenascin-C should be abandoned as a PFA
marker. DNA methylation and MIP arrays are effective tools
for copy number analysis of 1q gain, 6q and CDKN2A loss
whilst FISH is inadequate. Fusion detection was successful,
but rare novel fusions need more extensive technologies.
Finally, we propose test sets to guide future diagnostic
approaches.},
keywords = {Ependymoma (Other) / biomarkers (Other) / brain tumours
(Other) / neuro-oncology (Other) / paediatric (Other)},
cin = {B062 / HD01},
ddc = {610},
cid = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:36916248},
doi = {10.1093/neuonc/noad055},
url = {https://inrepo02.dkfz.de/record/274243},
}