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@ARTICLE{Chavaz:305736,
author = {L. Chavaz and A. Bagchi and S. K. Dhanda and F. Toutain and
S. M. Pfister$^*$ and D. Sturm$^*$ and T. Pietsch and G. H.
Gielen and A. Waha and M. Clarke and C. Lu and M. Karremann
and M. Benesch and T. Perwein and G. Nussbaumer and C. Kramm
and M. Massimino and V. Biassoni and M. Vinci and A.
Mastronuzzi and D. van Vuurden and S. E. M. Veldhuijzen van
Zanten and A. Mackay and C. Jones and D. T. W. Jones$^*$ and
A. S. Guerreiro Stucklin and U. Tabori and C. Hawkins and S.
Ryall and A. Morales La Madrid and A. Lassaletta and S.
Bailey and D. Hargrave and J. Chiang and M. El-Ayadi and B.
M. Mançano and R. Manuel Reis and C. Hagel and H. Gorsi and
N. Silvestrini and A. Gilani and L. Papusha and P. Klimo and
X. Zhou and A. Gajjar and G. W. Robinson and A. O. von
Bueren},
title = {{A} {S}ystematic {S}tudy of {M}olecular {D}iagnosis,
{T}reatment and {P}rognosis in {I}nfant-{T}ype {H}emispheric
{G}lioma): {A}n {I}ndividual {P}atient {D}ata
{M}eta-analysis of 164 patients.},
journal = {Neuro-Oncology},
volume = {nn},
issn = {1522-8517},
address = {Oxford},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2025-02359},
pages = {nn},
year = {2025},
note = {epub},
abstract = {Due to the novelty and rarity of infant-type hemispheric
glioma (IHG), optimal treatment and factors determining
clinical outcomes are yet to be established.We curated a
series of 164 patients with IHG; 155 identified by
methodical literature search and nine additional patients
contributed by collaborators.All tumors were hemispheric,
diagnosed at a median age of 3.4 (0-52) months, and
frequently $(95\%)$ non-metastatic. 142 $(86.5\%)$ tumors
harbored fusions involving RTK genes (ALK [67/142, $47\%],$
NTRK1/2/3[32/142, $22.5\%],$ ROS1 [29/142, $20.4\%],$ MET
[13/142, $9.2\%],$ and ABL2 [1/142, $0.7\%]).$ $64\%,$
$20\%,$ and $8\%$ of patients were treated with surgery and
adjuvant chemotherapy, surgery-only, and surgery plus
targeted therapy, respectively. Five patients received
radiation. Three-year event-free survival (EFS) and overall
survival (OS) was $49.5\%$ [40.7-60.2] and $79.6\%$
[72.1-87.9], respectively. 22 patients succumbed to disease,
of which tumor progression (8/22, $36\%)$ and intra-cranial
hemorrhage (5/22, $23\%)$ were the most common causes.
Multivariate analysis showed that the factors most
associated with an increased risk of death were no treatment
except for surgery and presence of residual tumor after
definitive surgery. These findings present a challenging
dichotomy where surgery is both a serious risk factor for
early death and, when successful, a benefit.Together, these
findings show that IHG is a fusion driven tumor of the very
young that is survivable even after progression. While
optimal primary therapy for patients with IHG has yet to be
established, the findings of this meta-analysis suggest
treatment should focus on lowering surgical morbidity and
improving its success.},
keywords = {Pediatric-type diffuse high-grade glioma (pHGG) (Other) /
Receptor Tyrosine Kinase (RTK) (Other) / Tyrosine Kinase
Inhibitor (TKI) (Other) / high-grade glioma (HGG) (Other) /
infant-type hemispheric glioma (IHG) (Other)},
cin = {B062 / B360},
ddc = {610},
cid = {I:(DE-He78)B062-20160331 / 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:41206756},
doi = {10.1093/neuonc/noaf264},
url = {https://inrepo02.dkfz.de/record/305736},
}