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@ARTICLE{Suwala:165986,
author = {A. K. Suwala$^*$ and D. Stichel$^*$ and D. Schrimpf$^*$ and
M. Kloor$^*$ and A. K. Wefers$^*$ and A. Reinhardt$^*$ and
S. L. N. Maas and C. P. Kratz and L. Schweizer$^*$ and M.
Hasselblatt and M. Snuderl and M. S. J. Abedalthagafi and Z.
Abdullaev and C. M. Monoranu and M. Bergmann and A. Pekrun
and C. Freyschlag and E. Aronica and C. M. Kramm and F.
Hinz$^*$ and P. Sievers$^*$ and A. Korshunov$^*$ and M.
Kool$^*$ and S. M. Pfister$^*$ and D. Sturm$^*$ and D. T. W.
Jones$^*$ and W. Wick$^*$ and A. Unterberg and C. Hartmann
and A. Dodgshun and U. Tabori and P. Wesseling and F.
Sahm$^*$ and A. von Deimling$^*$ and D. Reuß$^*$},
title = {{P}rimary mismatch repair deficient {IDH}-mutant
astrocytoma ({PMMRDIA}) is a distinct type with a poor
prognosis.},
journal = {Acta neuropathologica},
volume = {141},
number = {1},
issn = {1432-0533},
address = {Heidelberg},
publisher = {Springer},
reportid = {DKFZ-2020-02525},
pages = {85-100},
year = {2021},
note = {#EA:B300#LA:B300#2021 Jan;141(1):85-100},
abstract = {Diffuse IDH-mutant astrocytoma mostly occurs in adults and
carries a favorable prognosis compared to IDH-wildtype
malignant gliomas. Acquired mismatch repair deficiency is
known to occur in recurrent IDH-mutant gliomas as resistance
mechanism towards alkylating chemotherapy. In this
multi-institutional study, we report a novel epigenetic
group of 32 IDH-mutant gliomas with proven or suspected
hereditary mismatch repair deficiency. None of the tumors
exhibited a combined 1p/19q deletion. These primary mismatch
repair-deficient IDH-mutant astrocytomas (PMMRDIA) were
histologically high-grade and were mainly found in children,
adolescents and young adults (median age 14 years).
Mismatch repair deficiency syndromes (Lynch or
Constitutional Mismatch Repair Deficiency Syndrom (CMMRD))
were clinically diagnosed and/or germline mutations in DNA
mismatch repair genes (MLH1, MSH6, MSH2) were found in all
cases, except one case with a family and personal history of
colon cancer and another case with MSH6-deficiency available
only as recurrent tumor. Loss of at least one of the
mismatch repair proteins was detected via
immunohistochemistry in all, but one case analyzed. Tumors
displayed a hypermutant genotype and microsatellite
instability was present in more than half of the sequenced
cases. Integrated somatic mutational and chromosomal copy
number analyses showed frequent inactivation of TP53, RB1
and activation of RTK/PI3K/AKT pathways. In contrast to the
majority of IDH-mutant gliomas, more than $60\%$ of the
samples in our cohort presented with an unmethylated MGMT
promoter. While the rate of immuno-histochemical ATRX loss
was reduced, variants of unknown significance were more
frequently detected possibly indicating a higher frequency
of ATRX inactivation by protein malfunction. Compared to
reference cohorts of other IDH-mutant gliomas, primary
mismatch repair-deficient IDH-mutant astrocytomas have by
far the worst clinical outcome with a median survival of
only 15 months irrespective of histological or molecular
features. The findings reveal a so far unknown entity of
IDH-mutant astrocytoma with high prognostic relevance.
Diagnosis can be established by aligning with the
characteristic DNA methylation profile, by
DNA-sequencing-based proof of mismatch repair deficiency or
immunohistochemically demonstrating loss-of-mismatch repair
proteins.},
cin = {B300 / HD01 / F210 / BE01 / B062 / B360 / B320},
ddc = {610},
cid = {I:(DE-He78)B300-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)F210-20160331 / I:(DE-He78)BE01-20160331 /
I:(DE-He78)B062-20160331 / I:(DE-He78)B360-20160331 /
I:(DE-He78)B320-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:33216206},
doi = {10.1007/s00401-020-02243-6},
url = {https://inrepo02.dkfz.de/record/165986},
}