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@ARTICLE{Tonn:212544,
author = {S. Tonn and A. Korshunov$^*$ and D. Obrecht and M. Sill$^*$
and M. Spohn and K. von Hoff and T. Milde$^*$ and T. Pietsch
and T. Goschzik and B. Bison and B.-O. Juhnke and N. Struve
and D. Sturm$^*$ and F. Sahm$^*$ and M. Bockmayr and C.
Friedrich and A. O. von Bueren and N. U. Gerber and M.
Benesch and D. Jones$^*$ and M. Kool$^*$ and A. K. Wefers
and U. Schüller and S. Pfister$^*$ and S. Rutkowski and M.
Mynarek},
title = {{R}isk prediction in early childhood {SHH} medulloblastoma
treated with radiation-avoiding chemotherapy: {E}vidence for
more than two subgroups.},
journal = {Neuro-Oncology},
volume = {25},
number = {8},
issn = {1522-8517},
address = {Oxford},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2023-00229},
pages = {1518-1529},
year = {2023},
note = {2023 Aug 3;25(8):1518-1529},
abstract = {The prognostic impact of clinical risk factors and DNA
methylation patterns in sonic hedgehog (SHH)-activated early
childhood desmoplastic/nodular medulloblastoma (DMB) or
medulloblastoma with extensive nodularity (MBEN) were
evaluated to better identify patients at risk for
relapse.Hundred-forty-four patients with DMB (n=99) or MBEN
(n=45) aged <5 years and treated with radiation-sparing
approaches, including intraventricular methotrexate in 132
patients, were evaluated.Patients with DMB had less
favorable 5-year progression-free survival than MBEN
(5y-PFS, $71\%$ [DMB] vs $93\%$ [MBEN]). Patients' age >3
years was associated with more unfavorable 5y-PFS $(47\%$
[>3 years] vs $85\%$ [<1 year] vs $84\%$ [1-3 years]). DNA
methylation profiles available (n=78) were reclassified
according to the 2021 WHO classification into SHH-1 (n=39),
SHH-2 (n=38), and SHH-3 (n=1). Hierarchical clustering
delineated two subgroups among SHH-2: SHH-2a (n=19) and
SHH-2b (n=19). Patients with SHH-2b medulloblastoma were
older, predominantly displayed DMB histology, and were more
often located in the cerebellar hemispheres. Chromosome 9q
losses were more frequent in SHH-2b, while few chromosomal
alterations were observed in SHH-2a. SHH-2b medulloblastoma
carried a significantly increased relapse risk (5y-PFS:
$58\%$ [SHH-2b] vs $83\%$ [SHH-1] vs $95\%$ [SHH-2a]).
Subclassification of SHH-2 with key clinical and cytogenetic
characteristics was confirmed using two independent cohorts
(total n=188). Gene mutation analysis revealed a correlation
of SHH-2a with SMO mutations.These data suggest further
heterogeneity within early childhood SHH-DMB/MBEN: SHH-2
splits into a very low-risk group SHH-2a enriched for MBEN
histology and SMO mutations, and SHH-2b comprising older DMB
patients with higher risk of relapse.},
keywords = {DNA methylation profiling (Other) / Medulloblastoma (Other)
/ Risk prediction (Other) / Sonic hedgehog-activated
(Other)},
cin = {B300 / HD01 / B062 / B310 / B360},
ddc = {610},
cid = {I:(DE-He78)B300-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)B062-20160331 / I:(DE-He78)B310-20160331 /
I:(DE-He78)B360-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:36715306},
doi = {10.1093/neuonc/noad027},
url = {https://inrepo02.dkfz.de/record/212544},
}