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@ARTICLE{Liu:167588,
author = {A. P. Y. Liu and B. K. Li and E. Pfaff$^*$ and B. Gudenas
and A. Vasiljevic and B. A. Orr and C. Dufour and M. Snuderl
and M. A. Karajannis and M. K. Rosenblum and E. I. Hwang and
H.-K. Ng and J. R. Hansford and A. Szathmari and C.
Faure-Conter and T. E. Merchant and M. Levine and N. Bouvier
and K. von Hoff and M. Mynarek and S. Rutkowski and F.
Sahm$^*$ and M. Kool$^*$ and C. Hawkins and A. Onar-Thomas
and G. W. Robinson and A. Gajjar and S. M. Pfister$^*$ and
E. Bouffet and P. A. Northcott and D. T. W. Jones$^*$ and A.
Huang},
title = {{C}linical and molecular heterogeneity of pineal
parenchymal tumors: a consensus study.},
journal = {Acta neuropathologica},
volume = {141},
number = {5},
issn = {1432-0533},
address = {Heidelberg},
publisher = {Springer},
reportid = {DKFZ-2021-00448},
pages = {771-785},
year = {2021},
note = {2021 May;141(5):771-785 #LA:B360#},
abstract = {Recent genomic studies have shed light on the biology and
inter-tumoral heterogeneity underlying pineal parenchymal
tumors, in particular pineoblastomas (PBs) and pineal
parenchymal tumors of intermediate differentiation (PPTIDs).
Previous reports, however, had modest sample sizes and
lacked the power to integrate molecular and clinical
findings. The different proposed molecular group structures
also highlighted a need to reach consensus on a robust and
relevant classification system. We performed a meta-analysis
on 221 patients with molecularly characterized PBs and
PPTIDs. DNA methylation profiles were analyzed through
complementary bioinformatic approaches and molecular
subgrouping was harmonized. Demographic, clinical, and
genomic features of patients and samples from these pineal
tumor groups were annotated. Four clinically and
biologically relevant consensus PB groups were defined:
PB-miRNA1 (n = 96), PB-miRNA2 (n = 23), PB-MYC/FOXR2 (n =
34), and PB-RB1 (n = 25). A final molecularly distinct
group, designated PPTID (n = 43), comprised histological
PPTID and PBs. Genomic and transcriptomic profiling allowed
the characterization of oncogenic drivers for individual
tumor groups, specifically, alterations in the microRNA
processing pathway in PB-miRNA1/2, MYC amplification and
FOXR2 overexpression in PB-MYC/FOXR2, RB1 alteration in
PB-RB1, and KBTBD4 insertion in PPTID. Age at diagnosis, sex
predilection, and metastatic status varied significantly
among tumor groups. While patients with PB-miRNA2 and PPTID
had superior outcome, survival was intermediate for patients
with PB-miRNA1, and dismal for those with PB-MYC/FOXR2 or
PB-RB1. Reduced-dose CSI was adequate for patients with
average-risk, PB-miRNA1/2 disease. We systematically
interrogated the clinical and molecular heterogeneity within
pineal parenchymal tumors and proposed a consensus
nomenclature for disease groups, laying the groundwork for
future studies as well as routine use in tumor diagnostic
classification and clinical trial stratification.},
keywords = {Consensus (Other) / DNA methylation profiling (Other) /
Molecular groups (Other) / Pineal parenchymal tumors of
intermediate differentiation (Other) / Pineoblastoma (Other)
/ Risk-stratification (Other)},
cin = {B360 / B300 / HD01 / B062},
ddc = {610},
cid = {I:(DE-He78)B360-20160331 / I:(DE-He78)B300-20160331 /
I:(DE-He78)HD01-20160331 / I:(DE-He78)B062-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:33619588},
doi = {10.1007/s00401-021-02284-5},
url = {https://inrepo02.dkfz.de/record/167588},
}