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@ARTICLE{Quattrone:282561,
author = {Quattrone, Andrea and Franzmeier, Nicolai and Huppertz,
Hans-Jürgen and Seneca, Nicholas and Petzold, Gabor C and
Spottke, Annika and Levin, Johannes and Prudlo, Johannes and
Düzel, Emrah and Höglinger, Günter U},
collaboration = {PASSPORT Study Group, the AL-108-231 Investigators, the
Arise Investigators, the Tauros MRI Investigators, the
DESCRIBE-PSP group},
othercontributors = {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
Eggert, 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 Stamelou, Maria 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 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
Bor-Delon, 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 Ahmed, Anwar and Aiba, Ikuko and
Albanese, Alberto and Bertram, Kelly and Bordelon, Yvette
and Bower, James and Brosch, Jared and Claassen, Daniel and
Colosimo, Carlo and Corvol, Jean-Christophe and Cudia, Paola
and Daniele, Antonio and Defebvre, Luc and Driver-Dunckley,
Erika and Duquette, Antoine and Eleopra, Roberto and
Eusebio, Alexandre and Fung, Victor and Geldmacher, David
and Golbe, Lawrence and Grandas, Francisco and Hall, Deborah
and Hatano, Taku and Höglinger, Günter and Honig, Lawrence
and Hui, Jennifer and Kerwin, Diana and Kikuchi, Akio and
Kimber, Thomas and Kimura, Takashi and Kumar, Rajeev and
Litvan, Irene and Ljubenkov, Peter and Lorenzl, Stefan and
Ludolph, Albert and Mari, Zoltan and McFarland, Nikolaus and
Meissner, Wassilios and Rivera, Pablo Mir and Mochizuki,
Hidek and Morgan, John and Munhoz, Renato and Nishikawa,
Noriko and O'Sullivan, John and Oeda, Tomoko and Oizumi,
Hideki and Onodera, Osamu and Ory-Magne, Fabienne and
Peckham, Elizabeth and Postuma, Ronald and Quattrone, Aldo
and Quinn, Joseph and Ruggieri, Stefano and Sarna, Justyna
and Schulz, Paul E and Slevin, John and Tagliati, Michele
and Wile, Daryl and Wszolek, Zbigniew and Xie, Tao and
Zesiewicz, Theresa and Gómez, Juan C and Tijero, Beatriz
and Villoria, Rafael and de Yebenes, Justo García and
Sendón, Jose L and Tolosa, Eduardo and Buongiorno, Maria T
and Bargalló, Nuria and Burguera, Juan A and Martinez,
Iñiguez and Ruiz-Martínez, Javier and Villanua, Jorge and
Vivancos, Francisco and Ybot, Isabel and Aguilar, Miquel and
Dolz, Josep L and Boada, Mercè and Lafuente, Asuncion and
Tejero, Miguel A and López-Lozano, Juan J and Mata, Marina
and Kupsch, Andreas and Lipp, Axel and Höllerhage, Matthias
and Oertel, Wolfgang H and Respondek, Gesine and Stamelou,
Maria and Knake, Susanne and Berg, Daniela and Maetzler,
Walter and Srulijes, Karin K and Gröger, Adriane and
Ludolph, Albert and Kassubek, Jan and Steiger, Malcom and
Tyler, Kevin and Burn, David J and Morris, Cristopher and
Lees, Andrew and Ling, Helen and Strycharczuk, Lisa and
Altenstein, Slawek and Bähr, Mathias and Barkhoff, Miriam
and Bartels, Claudia and Michael, Bartl and Beschorner, Rudi
and Karls, Eberhard and Beyle, Aline and Boecker, Henning
and Brandt, Daniel Moritz and Brockmann, Kathrin and
Brosseron, Frederic and Burow, Lena and Catak, Cihan and
Cetindag, Arda Can and Cosma, Nicoleta Carmen and Daamen,
Marcel and Dashti, Eman and de Jonge, Sylvia and Dechent,
Peter and Dichgans, Martin and Dinter, Elisabeth and
Dobisch, Laura and Dörr, Angelika and Donix, Markus and
Senden, Louisa Droste Zu and Drzezga, Alexander and Düzel,
Emrah and Dyrba, Martin and Ehrlich, Marie and Endres,
Matthias and Engels, Tanja and Ersözlü, Ersin and Ewers,
Michael and Faber, Jennifer and Falkenburger, Björn and
Fenski, Frederike and Fließbach, Klaus and Flöel, Agnes
and Freiesleben, Silka Dawn and Fries, Franca and Frommann,
Ingo and Gasser, Thomas and Glanz, Wenzel and Görß, Doreen
and Grieger-Klose, Doreen and Grobe-Einsler, Marcus and
Grond, Martin and Gürsel, Selim Üstün and Halle, Annett
and Hansen, Niels and Hartmann, Deike and Hassoun, Lina and
Haussmann, Robert and Hellmann-Regen, Julian David Nicolai
and Hennes, Guido and Hermann, Wiebke and Hermann, Andreas
and Herms, Jochen and Herrmann, Gabi and Hetzer, Stefan and
Hinderer, Petra and Höglinger, Günther U and Horvath,
Ildiko and Hujer, Nicole and Incesoy, Enise and Jäck,
Alexander and Janowitz, Daniel and Jessen, Frank and Jost,
Lorraine and Kalbhen, Pascal and Kassubek, Jan and
Katzdobler, Sabrina and Kilimann, Ingo and Kimmich, Okka and
Kleinadam, Luca and Klockgether, Thomas and Kobeleva, Xenia
and Krause, Patricia and Kuder-Buletta, Elke and Kühn,
Andrea and Kurz, Carolin and Lange, Catharina and Lappe,
Chris and Laske, Christoph and Levin, Johannes and
Schneider, Sandra Lichte and Lingor, Paul and Löhle,
Matthias and Ludolph, Albert and Lüsebrink-Rindsland, Jann
Falk Silvester and McCormick, Cornelia and Mengel, David and
Metzger, Coraline and Munk, Matthias and Neumann, Manuela
and Oender, Demet and Perneczky, Robert and Peters, Oliver
and Petzold, Gabor C and Pfaff, Henrike and Polcher,
Alexandra and Preis, Lukas and Priller, Josef and Prudlo,
Johannes and Pürner, Dominik and Purrer, Veronika and
Rauchmann, Boris Stephan and Ramirez, Alfredo and Raum,
Heike and Röske, Sandra and Ruß, Christin and Sabik, Petr
and Sagik, Yilmaz and Scheffler, Klaus and Schmidt, Monika
and Schneider, Anja and Schneider, Luisa-Sophie and Schöls,
Ludger and Schott, Björn and Schulz, Heike and Schulze,
Franziska and Sodenkamp, Sebastian and Spottke, Annika and
Spruth, Eike Jakob and Stark, Melina and Synofzik, Matthis
and Sulzer, Patricia and Teipel, Stefan and Vogt, Ina
Rosemarie and Wagner, Michael and Weidinger, Endy and Wilke,
Carlo and Wiltfang, Jens and Wolfsgruber, Steffen and
Wüllner, Ullrich and Yakupov, Renat and Zech, Heike and
Zerr, Inga and Ziegler, Gabriel and Zollver, Adelgunde},
title = {{B}rain {A}trophy {D}oes {N}ot {P}redict {C}linical
{P}rogression in {P}rogressive {S}upranuclear {P}alsy.},
journal = {Movement disorders},
volume = {40},
number = {11},
issn = {0885-3185},
address = {New York, NY},
publisher = {Wiley},
reportid = {DZNE-2025-01324},
pages = {2517 - 2530},
year = {2025},
abstract = {Clinical progression rate is the typical primary endpoint
measure in progressive supranuclear palsy (PSP) clinical
trials.This longitudinal multicohort study investigated
whether baseline clinical severity and regional brain
atrophy could predict clinical progression in
PSP-Richardson's syndrome (PSP-RS).PSP-RS patients (n = 309)
from the placebo arms of clinical trials (NCT03068468,
NCT01110720, NCT02985879, NCT01049399) and DescribePSP
cohort were included. We investigated associations of
baseline clinical and volumetric magnetic resonance imaging
(MRI) data with 1-year longitudinal PSP rating scale (PSPRS)
change. Machine learning (ML) models were tested to predict
individual clinical trajectories.PSP-RS patients showed a
mean PSPRS score increase of 10.3 points/yr. The frontal
lobe volume showed the strongest association with subsequent
clinical progression (β: -0.34, P < 0.001). However, ML
models did not accurately predict individual progression
rates (R2 <0.15).Baseline clinical severity and brain
atrophy could not predict individual clinical progression,
suggesting no need for MRI-based stratification of patients
in future PSP trials. © 2025 The Author(s). Movement
Disorders published by Wiley Periodicals LLC on behalf of
International Parkinson and Movement Disorder Society.},
keywords = {Humans / Supranuclear Palsy, Progressive: pathology /
Supranuclear Palsy, Progressive: diagnostic imaging /
Disease Progression / Atrophy: pathology / Male / Female /
Aged / Magnetic Resonance Imaging / Brain: pathology /
Brain: diagnostic imaging / Middle Aged / Longitudinal
Studies / Severity of Illness Index / Machine Learning /
atlas‐based volumetry (Other) / clinical trials (Other) /
outcome (Other) / progression (Other) / progressive
supranuclear palsy (Other)},
cin = {Clinical Research (Munich) / AG Petzold / AG Spottke /
Clinical Research Platform (CRP) / AG Levin / AG Düzel},
ddc = {610},
cid = {I:(DE-2719)1111015 / I:(DE-2719)1013020 /
I:(DE-2719)1011103 / I:(DE-2719)1011401 / I:(DE-2719)1111016
/ I:(DE-2719)5000006},
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:40884249},
pmc = {pmc:PMC12661634},
doi = {10.1002/mds.70026},
url = {https://pub.dzne.de/record/282561},
}
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