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@ARTICLE{Iskandar:277976,
author = {Iskandar, Audrey and Buchholz, Maresa and Blotenberg, Iris
and Schmitz-Hübsch, Tanja and Faber, Jennifer and Jacobi,
Heike and Xie, Feng and Grobe-Einsler, Marcus and
Klockgether, Thomas and Michalowsky, Bernhard and ESMI},
collaboration = {groups, EUROSCA Study},
othercontributors = {Reetz, Kathrin and Raponso, Mafalda and Gonzales, Carlos
and Koyak, Berkan and Oender, Demet and de Almeida, Luís
Pereira and Silva, Patrick and Ribeiro, Joana Afonso and
Thieme, Andreas and Erdlenbruch, Friedrich and de Vries,
Jeroen and Onyike, Chiadikaobi and Giunti, Paola and
Garcia-Moreno, Hector and Öz, Gülin and Bischoff, Almut
and van de Warrenburg, Bart and van Gaalen, Judith and
Infante, Jon and Manrique, Leire and Schöls, Ludger and
Riess, Olaf and du Montcel, Sophie Tezenas and Bauer, Peter
and Giunti, Paola and Cook, Arron and Labrum, Robyn and
Parkinson, Michael H and Durr, Alexandra and Brice, Alexis
and Charles, Perrine and Marelli, Cecilia and Mariotti,
Caterina and Nanetti, Lorenzo and Panzeri, Marta and
Rakowicz, Maria and Sulek, Anna and Sobanska, Anna and
Hengel, Holger and Baliko, Laszlo and Melegh, Bela and
Filla, Alessandro and Antenora, Antonella and Berciano,
José and Timmann, Dagmar and Szymanski, Sandra and Boesch,
Sylvia and Kang, Jun-Suk and Padolfo, Massimo and Schulz,
Jörg B and Molho, Sonia and Diallo, Alhassane},
title = {{L}ongitudinal description of health-related quality of
life and depressive symptoms in poly{Q} spinocerebellar
ataxia patients.},
journal = {Journal of neurology},
volume = {272},
number = {5},
issn = {0367-004X},
address = {[Darmstadt]},
publisher = {Steinkopff},
reportid = {DZNE-2025-00508},
pages = {323},
year = {2025},
abstract = {Due to limited treatment options, managing symptoms has
dominated care for Spinocerebellar Ataxia (SCA). Little
attention has been given to health-related quality of life
(HRQoL) and depressive symptoms experienced by patients
across disease duration.To investigate the course of HRQoL
and the severity of depressive symptoms in SCA from disease
onset to 26 years after onset and identify influencing
factors.We analyzed data from two longitudinal SCA cohorts,
the EUROSCA (European Spinocerebellar Ataxia Registry) and
ESMI study (European Spinocerebellar Ataxia Type
3/Machado-Joseph Disease Initiative). Multilevel
mixed-effects models were employed to demonstrate the course
of HRQoL and depressive symptoms severity to investigate the
role of disease progression with disease duration as a
predictor of interest, along with time-varying clinical
variables and time-fixed covariates.Seven hundred seventy
four participants (Mage = 50.8 ± 13.4; $48.6\%$ female)
were included. HRQoL consistently decreased throughout
disease duration across all SCA subtypes, but the decline
was smallest in SCA6. The decrease in HRQoL was explained by
ataxia and depression severity and driven by increasing
problems with self-care, usual activities and mobility.
Depressive symptoms significantly increased in SCA2 and 3
only, with a trend toward slight improvement in SCA6.The
trend direction of HRQoL and its significant association
with the severity of ataxia symptoms align with the
literature. The rapid worsening of self-care problems, the
differential associations between depression and HRQoL
sub-dimensions in different SCA subtypes, and the
unexplainable resilience may warrant a deeper look at
patient-specific intra- and interpersonal factors.},
keywords = {Humans / Female / Male / Quality of Life: psychology /
Middle Aged / Spinocerebellar Ataxias: psychology /
Spinocerebellar Ataxias: complications / Spinocerebellar
Ataxias: genetics / Longitudinal Studies / Adult /
Depression: etiology / Depression: psychology / Aged /
Disease Progression / Severity of Illness Index / Registries
/ Peptides: genetics / Ataxia (Other) / Genetic disorder
(Other) / Health-related quality of life (Other) /
Patient-centered care (Other) / Psychologic well-being
(Other) / Rare disease (Other) / polyglutamine (NLM
Chemicals) / Peptides (NLM Chemicals)},
cin = {AG Michalowsky / AG Hoffmann / AG Thyrian / Clinical
Research (Bonn) / Patient Studies (Bonn)},
ddc = {610},
cid = {I:(DE-2719)5000067 / I:(DE-2719)1510600 /
I:(DE-2719)1510800 / I:(DE-2719)1011001 /
I:(DE-2719)1011101},
pnm = {353 - Clinical and Health Care Research (POF4-353)},
pid = {G:(DE-HGF)POF4-353},
experiment = {EXP:(DE-2719)ESMI-20140101},
typ = {PUB:(DE-HGF)16},
pmc = {pmc:PMC11982069},
pubmed = {pmid:40202598},
doi = {10.1007/s00415-025-13024-0},
url = {https://pub.dzne.de/record/277976},
}