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@ARTICLE{vanEimeren:1025640,
author = {van Eimeren, Thilo and Giehl, Kathrin and Reetz, Kathrin
and Sampaio, Cristina and Mestre, Tiago A.},
title = {{N}euroimaging biomarkers in {H}untington's disease:
{P}reparing for a new era of therapeutic development},
journal = {Parkinsonism $\&$ related disorders},
volume = {114},
issn = {1353-8020},
address = {Amsterdam [u.a.]},
publisher = {Elsevier Science},
reportid = {FZJ-2024-03029},
pages = {105488 -},
year = {2023},
abstract = {BackgroundA critical challenge for Huntington's disease
(HD) clinical trials in disease modification is the
definition of endpoints that can capture change when
clinical signs are subtle/non-existent. Reliable biomarkers
are therefore urgently needed to facilitate drug development
by allowing the enrichment of clinical trial populations and
providing measures of benefit that can support the
establishment of efficacy.MethodsBy systematically examining
the published literature on HD neuroimaging biomarker
studies, we sought to advance knowledge to guide the
validation of neuroimaging biomarkers. We started by
reviewing both cross-sectional and longitudinal studies and
then conducted an in-depth review to make quantitative
comparisons between biomarkers using data only from
longitudinal studies with samples sizes larger than ten
participants in PET studies or 30 participants in MRI
studies.ResultsFrom a total of 2202 publications initially
identified, we included 32 studies, 19 of which underwent
in-depth comparative review. The majority of included
studies used various MRI-based methods (manual to automatic)
to longitudinally assess either the volume of the putamen or
the caudate, which have been shown to undergo significant
structural change during HD natural
history.ConclusionDespite the impressively large number of
neuroimaging biomarker studies, only a small number of
adequately designed studies met our criteria. Among these
various biomarkers, MRI-based volumetric analyses of the
caudate and putamen are currently the best validated for use
in the disease phase before clinical motor diagnosis. A
biomarker that can be used to demonstrate a
disease-modifying effect is still missing.},
cin = {INM-2},
ddc = {610},
cid = {I:(DE-Juel1)INM-2-20090406},
pnm = {5252 - Brain Dysfunction and Plasticity (POF4-525) / 5253 -
Neuroimaging (POF4-525)},
pid = {G:(DE-HGF)POF4-5252 / G:(DE-HGF)POF4-5253},
typ = {PUB:(DE-HGF)16},
pubmed = {37407343},
UT = {WOS:001078735000001},
doi = {10.1016/j.parkreldis.2023.105488},
url = {https://juser.fz-juelich.de/record/1025640},
}