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@ARTICLE{Quattrone:271711,
author = {Quattrone, Andrea and Franzmeier, Nicolai and Huppertz,
Hans-Jürgen and Klietz, Martin and Roemer, Sebastian N and
Boxer, Adam L and Levin, Johannes and Höglinger, Günter U},
collaboration = {AL-108-231 Investigators, the Tauros MRI Investigators, the
PASSPORT Study Group, the DESCRIBE-PSP Group},
othercontributors = {Williams, David and Lafontaine, Anne Louise and Marras,
Connie and Jog, Mandar and Panisset, Michael and Lang,
Anthony and Parker, Lesley and Stewart, Alistair J and
Corvol, Jean-Christophe and Azulay, Jean-Philippe and
Couratier, Philippe and Mollenhauer, Brit and Lorenzl,
Stefan and Ludolph, Albert and Benecke, Reiner and
Höglinger, Günter and Lipp, Axel and Reichmann, Heinz and
Woitalla, Dirk and Chan, Dennis and Zermansky, Adam and
Burn, David and Lees, Andrew and Boxer, Adam and Miller,
Bruce L and Lobach, Iryna V and Roberson, Erik and Honig,
Lawrence and Zamrini, Edward and Pahwa, Rajesh and Bordelon,
Yvette and Driver-Dunkley, Erika and Lessig, Stephanie and
Lew, Mark and Womack, Kyle and Boeve, Brad and Ferrara,
Joseph and Hillis, Argyle and Kaufer, Daniel and Kumar,
Rajeev and Xie, Tao and Gunzler, Steven and Zesiewicz,
Theresa and Dayalu, Praveen and Golbe, Lawrence and
Grossman, Murray and Jancovic, Joseph and McGinnis, Scott
and Santiago, Anthony and Tuite, Paul and Isaacson, Stuart
and Leegwater-Kim, Julie and Litvan, Irene and Grossman,
Murray and Knopman, David S and Miller, Bruce L and
Schneider, Lon S and Doody, Rachelle S and Golbe, Lawrence I
and Roberson, Erik D and Koestler, Mary and Jack, Clifford R
and Van Deerlin, Viviana and Randolph, Christopher and
Lobach, Iryna V and Gozes, Illana and Whitaker, Steve and
Hirman, Joe and Gold, Michael and Morimoto, Bruce H and
Gómez, J. C. and Tijero, B. and Villoria, R. and de
Yebenes, J García and Sendón, J L Lopez and Tolosa, E. and
Buongiorno, M. T. and Bargalló, N. and Burguera, J. A. and
Martinez, I. and Ruiz-Martínez, J. and Villanua, J. and
Vivancos, F. and Ybot, I. and Aguilar, M. and Dolz, J. L.
and Boada, M. and Lafuente, A. and Tejero, M. A. and
López-Lozano, J. J. and Mata, M. and Kupsch, A. and Lipp,
A. and Höllerhage, M. and Oertel, W. H. and Respondek,
Gesine and Stamelou, M. and Knake, S. and Berg, D. and
Maetzler, W. and Srulijes, K. K. and Gröger, A. and
Ludolph, Albert and Kassubek, Jan and Steiger, M. and Tyler,
K. and Burn, D. J. and Morris, L. and Lees, A. and Ling, H.
and Strycharczuk, L. and Aiba, Ikuko and Antonini, Angelo
and Apetauerova, Diana and Azulay, Jean-Philippe and
Martinez, Ernest Balaguer and Bang, Jee and Barone, Paolo
and Barrett, Matthew and Bega, Danny and Berg, Daniela and
Corrales, Koldo Berganzo and Bordelon, Yvette and Boxer,
Adam L and Brandt, Daniel Moritz and Brueggemann, Norbert
and Castelnovo, Giovanni and Ceravolo, Roberto and Chuang,
Rosalind and Chung, Sun Ju and Church, Alistair and Corvol,
Jean-Christophe and Cudia, Paola and Dale, Marian and
Defebvre, Luc and Drapier, Sophie and Driver-Dunckley, Erika
D and Ebersbach, Georg and Egger, Karla M and Ellenbogen,
Aaron and Eusebio, Alexandre and Evans, Andrew H and
Fedorova, Natalia and Finger, Elizabeth and Foubert-Samier,
Alexandra and Ghosh, Boyd and Golbe, Lawrence and Perez,
Francisco Grandas and Grossman, Murray and Hall, Deborah and
Hamada, Kyoko and Hasegawa, Kazuko and Hoeglinger, Guenter
and Honig, Lawrence and Houghton, David and Huang, Xuemei
and Isaacson, Stuart and Koh, Seong-Beom and Bojarski, Jaime
Kulisevsky and Lang, Anthony E and Leigh, Peter Nigel and
Litvan, Irene and Lozano, Juan Jose Lopez and Moreno, Jose
Luis Lopez-Sendon and Ludolph, Albert and Piudo, Ma Rosario
Luquin and Torres, Irene Martinez and McFarland, Nikolaus
and Meissner, Wassilios and Mestre, Tiago and Rivera, Pablo
Mir and Molho, Eric and Mollenhauer, Britt and Morris, Huw R
and Murata, Miho and Obi, Tomokazu and Magne, Fabienne Ory
and O'Suilleabhain, Padraig and Pahwa, Rajesh and Pantelyat,
Alexander and Pavese, Nicola and Pokhabov, Dmitry and
Prudlo, Johannes and Rodriguez-Porcel, Federico and Rowe,
James and Savitt, Joseph and Schnitzler, Alfons and Schulz,
Joerg B and Seppi, Klaus and Shah, Binit and Shill, Holly
and Shprecher, David and Steiger, Malcolm and Takahashi,
Yuji and Takigawa, Hiroshi and Tartaglia, Carmela and
Toenges, Lars and Truong, Daniel and Tse, Winona and Tuite,
Paul and Volc, Dieter and Wills, Anne-Marie A and Woitalla,
Dirk and Xie, Tao and Yuasa, Tatsuhiko and Zauber, Sarah
Elizabeth and Zesiewicz, Theresa and Peters, Oliver and
Ersözlü, Ersin and Priller, Josef and Spruth, Eike Jakob
and Klockgether, Thomas and Vogt, Ina Rosemarie and Spottke,
Annika and Kimmich, Okka and Dinter, Elisabeth and Brandt,
Daniel Moritz and Flöel, Agnes and Benennen, Bitte and
Bürger, Katharina and Janowitz, Daniel and Katzdobler,
Sabrina and Jäck, Alexander and Düzel, Emrah and Glanz,
Wenzel and Teipel, Stefan and Kilimann, Ingo and Prudlo,
Johannes and Synofzik, Matthis and Beichert, Lukas and
Hoffmann, Daniel Christopher and Lüsebrink-Rindsland, Jann
Falk Silvester and Klockgether, Thomas and Höglinger,
Günter},
title = {{M}agnetic {R}esonance {I}maging {M}easures to {T}rack
{A}trophy {P}rogression in {P}rogressive {S}upranuclear
{P}alsy in {C}linical {T}rials.},
journal = {Movement disorders},
volume = {39},
number = {8},
issn = {0885-3185},
address = {New York, NY},
publisher = {Wiley},
reportid = {DZNE-2024-01063},
pages = {1329 - 1342},
year = {2024},
abstract = {Several magnetic resonance imaging (MRI) measures have been
suggested as progression biomarkers in progressive
supranuclear palsy (PSP), and some PSP staging systems have
been recently proposed.Comparing structural MRI measures and
staging systems in tracking atrophy progression in PSP and
estimating the sample size to use them as endpoints in
clinical trials.Progressive supranuclear palsy-Richardson's
syndrome (PSP-RS) patients with one-year-follow-up
longitudinal brain MRI were selected from the placebo arms
of international trials (NCT03068468, NCT01110720,
NCT01049399) and the DescribePSP cohort. The discovery
cohort included patients from the NCT03068468 trial; the
validation cohort included patients from other sources.
Multisite age-matched healthy controls (HC) were included
for comparison. Several MRI measures were compared:
automated atlas-based volumetry (44 regions), automated
planimetric measures of brainstem regions, and four
previously described staging systems, applied to volumetric
data.Of 508 participants, 226 PSP patients including
discovery (n = 121) and validation (n = 105) cohorts, and
251 HC were included. In PSP patients, the annualized
percentage change of brainstem and midbrain volume, and a
combined index including midbrain, frontal lobe, and third
ventricle volume change, were the progression biomarkers
with the highest effect size in both cohorts (discovery:
>1.6; validation cohort: >1.3). These measures required the
lowest sample sizes (n < 100) to detect $30\%$ atrophy
progression, compared with other volumetric/planimetric
measures and staging systems.This evidence may inform the
selection of imaging endpoints to assess the treatment
efficacy in reducing brain atrophy rate in PSP clinical
trials, with automated atlas-based volumetry requiring
smaller sample size than staging systems and planimetry to
observe significant treatment effects. © 2024 The
Author(s). Movement Disorders published by Wiley Periodicals
LLC on behalf of International Parkinson and Movement
Disorder Society.},
keywords = {Aged / Female / Humans / Male / Middle Aged / Atrophy:
pathology / Brain: diagnostic imaging / Brain: pathology /
Cohort Studies / Disease Progression / Magnetic Resonance
Imaging: methods / Supranuclear Palsy, Progressive:
diagnostic imaging / Supranuclear Palsy, Progressive:
pathology / Randomized Controlled Trials as Topic /
atlas‐based volumetry (Other) / clinical trials (Other) /
progression (Other) / progressive supranuclear palsy (Other)
/ staging system (Other)},
cin = {Clinical Research (Munich) / AG Levin},
ddc = {610},
cid = {I:(DE-2719)1111015 / I:(DE-2719)1111016},
pnm = {353 - Clinical and Health Care Research (POF4-353)},
pid = {G:(DE-HGF)POF4-353},
experiment = {EXP:(DE-2719)DESCRIBE-PSP-20160101},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:38825840},
doi = {10.1002/mds.29866},
url = {https://pub.dzne.de/record/271711},
}
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