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@ARTICLE{vandenBent:303036,
author = {M. J. van den Bent and T. F. Cloughesy and B. M. Ellingson
and J. E. Allen and S. C. Blackman and M. W. Kieran and J.
C. Tonn$^*$ and P. Karschnia and M. Wijnenga and J. Pallud
and R. J. Young and R. Y. Huang and I. K. Mellinghoff and J.
de Groot and A. van der Hoorn and G. Youssef and S. L.
Hervey-Jumper and M. A. Vogelbaum and S. M. Chang and P. Y.
Wen},
title = {{T}he {U}se of {M}inor {R}esponse, {V}olumetric
{A}ssessment and {G}rowth {R}ate {K}inetics as {E}ndpoints
in {G}rade 1-3 {G}lioma {C}linical {T}rials: a {RANO}
perspective.},
journal = {Neuro-Oncology},
volume = {nn},
issn = {1522-8517},
address = {Oxford},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2025-01483},
pages = {nn},
year = {2025},
note = {epub},
abstract = {The RANO criteria for response to treatment in glioma make
a distinction between enhancing and unenhancing tumors,
which reflects assessment of a different biological process
underlying the MRI findings in enhancing and non-enhancing
tumors. The recent regulatory approval of tovorafenib has
also considered the patients with minor responses $(25\%$ -
$50\%$ decrease) based on the 2D measurement on T2/FLAIR MR
imaging, while incorporating other measures of patient
benefit. The INDIGO placebo controlled trial led to the
regulatory approval of vorasidenib based on the increase in
PFS in the active treatment arm, as measured by blinded
independent radiology review using the 2D RANO criteria.
With that, more insight in the regulatory requirements for
approval in low grade tumors has been obtained, but other
issues in assessing outcome in studies on low grade
enhancing tumors remain. These include the added value of
volumetric assessment instead of 2D assessments, and
assessment of tumor growth rate as an early marker of
outcome. This manuscript gives an overview of the current
perspective on and challenges of assessing outcome in trials
on unenhancing glioma, with both regulatory and clinical
perspectives.},
keywords = {Minor Response (Other) / RANO (Other) / Tumor Growth rate
(Other) / unenhancing tumors (Other) / volumetric (3D)
assessment (Other)},
cin = {MU01},
ddc = {610},
cid = {I:(DE-He78)MU01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40692474},
doi = {10.1093/neuonc/noaf173},
url = {https://inrepo02.dkfz.de/record/303036},
}