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@ARTICLE{Ritzmann:178484,
author = {T. A. Ritzmann and R. J. Chapman and J.-P. Kilday and N.
Thorp and P. Modena and R. A. Dineen and D. Macarthur and C.
Mallucci and T. Jaspan and K. Pajtler$^*$ and M. Giagnacovo
and T. S. Jacques and S. M. L. Paine and D. W. Ellison and
E. Bouffet and R. G. Grundy},
collaboration = {B. consortium},
title = {{SIOP} {E}pendymoma {I}: {F}inal results, long term
follow-up and molecular analysis of the trial cohort: {A}
{BIOMECA} {C}onsortium {S}tudy.},
journal = {Neuro-Oncology},
volume = {24},
number = {6},
issn = {1522-8517},
address = {Oxford},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2022-00102},
pages = {936-948},
year = {2022},
note = {2022 Jun 1;24(6):936-948},
abstract = {SIOP Ependymoma I was a non-randomised trial assessing
event free and overall survival (EFS/OS) of non-metastatic
intracranial ependymoma in children aged 3 to 21 years
treated with a staged management strategy. A further aim was
to assess the response rate (RR) of subtotally resected
(STR) ependymoma to vincristine, etoposide and
cyclophosphamide (VEC). We report final results with 12-year
follow-up and post hoc analyses of recently described
biomarkers.74 participants were eligible. Children with
gross total resection (GTR) received radiotherapy, whilst
those with STR received VEC before radiotherapy. DNA
methylation, 1q, hTERT, ReLA, Tenascin-C, H3K27me3 and pAKT
status were evaluated.Five- and ten-year EFS was $49.5\%$
and $46.7\%,$ OS was $69.3\%$ and $60.5\%.$ GTR was achieved
in 33/74 $(44.6\%)$ and associated with improved EFS
(p=0.003, HR=2.6, $95\%$ confidence interval (CI) 1.4-5.1).
Grade 3 tumours were associated with worse OS (p=0.005,
HR=2.8, $95\%CI$ 1.3-5.8). 1q gain and hTERT expression were
associated with poorer EFS (p=0.003, HR=2.70, $95\%CI$
1.49-6.10 and p=0.014, HR=5.8, $95\%CI$ 1.2-28) and H3K27me3
loss with worse OS (p=0.003, HR=4.6, $95\%CI$ 1.5-13.2).
Methylation profiles showed expected patterns. 12
participants with STR did not receive chemotherapy; a
protocol violation. However, best chemotherapy RR was
$65.5\%$ (19/29, $95\%CI$ 45.7-82.1), exceeding the
prespecified $45\%.Participants$ with totally resected
ependymoma had the best outcomes. RR of STR to VEC exceeded
the pre-specified efficacy criterion. However, cases of
inaccurate stratification highlighted the need for rapid
central review. 1q gain, H3K27me3 loss and hTERT expression
were all associated with poorer survival outcomes.},
keywords = {Chemotherapy (Other) / Ependymoma (Other) / Radiotherapy
(Other) / Recurrence (Other) / Resection (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:35018471},
doi = {10.1093/neuonc/noac012},
url = {https://inrepo02.dkfz.de/record/178484},
}