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@ARTICLE{Deng:136677,
author = {M. Y. Deng$^*$ and M. Sill$^*$ and J. Chiang and J.
Schittenhelm and M. Ebinger and M. U. Schuhmann and C.-M.
Monoranu and T. Milde$^*$ and A. Wittmann$^*$ and C.
Hartmann and C. Sommer and W. Paulus and J. Gärtner and W.
Brück and T. Rüdiger and A. Leipold and Z. Jaunmuktane and
S. Brandner and F. Giangaspero and P. Nozza and J. Mora and
A. Morales la Madrid and O. Cruz Martinez and J. R. Hansford
and T. Pietsch and A. Tietze and P. Hernáiz-Driever and I.
Stoler and D. Capper and A. Korshunov$^*$ and D. W. Ellison
and A. von Deimling$^*$ and S. Pfister$^*$ and F. Sahm$^*$
and D. Jones$^*$},
title = {{M}olecularly defined diffuse leptomeningeal glioneuronal
tumor ({DLGNT}) comprises two subgroups with distinct
clinical and genetic features.},
journal = {Acta neuropathologica},
volume = {136},
number = {2},
issn = {1432-0533},
address = {Berlin},
publisher = {Springer},
reportid = {DKFZ-2018-01145},
pages = {239 - 253},
year = {2018},
abstract = {Diffuse leptomeningeal glioneuronal tumors (DLGNT)
represent rare CNS neoplasms which have been included in the
2016 update of the WHO classification. The wide spectrum of
histopathological and radiological features can make this
enigmatic tumor entity difficult to diagnose. In recent
years, large-scale genomic and epigenomic analyses have
afforded insight into key genetic alterations occurring in
multiple types of brain tumors and provide unbiased,
complementary tools to improve diagnostic accuracy. Through
genome-wide DNA methylation screening of > 25,000
tumors, we discovered a molecularly distinct class
comprising 30 tumors, mostly diagnosed histologically as
DLGNTs. Copy-number profiles derived from the methylation
arrays revealed unifying characteristics, including loss of
chromosomal arm 1p in all cases. Furthermore, this molecular
DLGNT class can be subdivided into two subgroups [DLGNT
methylation class (MC)-1 and DLGNT methylation class
(MC)-2], with all DLGNT-MC-2 additionally displaying a gain
of chromosomal arm 1q. Co-deletion of 1p/19q, commonly seen
in IDH-mutant oligodendroglioma, was frequently observed in
DLGNT, especially in DLGNT-MC-1 cases. Both subgroups also
had recurrent genetic alterations leading to an aberrant
MAPK/ERK pathway, with KIAA1549:BRAF fusion being the most
frequent event. Other alterations included fusions of
NTRK1/2/3 and TRIM33:RAF1, adding up to an MAPK/ERK pathway
activation identified in $80\%$ of cases. In the DLGNT-MC-1
group, age at diagnosis was significantly lower (median 5 vs
14 years, p < 0.01) and clinical course less aggressive
(5-year OS 100, vs $43\%$ in DLGNT-MC-2). Our study proposes
an additional molecular layer to the current
histopathological classification of DLGNT, of particular use
for cases without typical morphological or radiological
characteristics, such as diffuse growth and radiologic
leptomeningeal dissemination. Recurrent 1p deletion and
MAPK/ERK pathway activation represent diagnostic biomarkers
and therapeutic targets, respectively-laying the foundation
for future clinical trials with, e.g., MEK inhibitors that
may improve the clinical outcome of patients with DLGNT.},
cin = {B360 / B062 / G340 / L101 / G380},
ddc = {610},
cid = {I:(DE-He78)B360-20160331 / I:(DE-He78)B062-20160331 /
I:(DE-He78)G340-20160331 / I:(DE-He78)L101-20160331 /
I:(DE-He78)G380-20160331},
pnm = {312 - Functional and structural genomics (POF3-312)},
pid = {G:(DE-HGF)POF3-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:29766299},
doi = {10.1007/s00401-018-1865-4},
url = {https://inrepo02.dkfz.de/record/136677},
}