TY  - JOUR
AU  - Buchholz, Maresa
AU  - Pfaff, Michelle
AU  - Iskandar, Audrey
AU  - Reetz, Kathrin
AU  - Schulz, Jörg B
AU  - Grobe-Einsler, Marcus
AU  - Klockgether, Thomas
AU  - Michalowsky, Bernhard
TI  - Health-Related Quality of Life in Patients with Friedreich Ataxia Using Mobility Assistive Technologies: Limited Fit of the EQ-5D-3L Mobility Dimension.
JO  - Neurology and Therapy
VL  - 14
IS  - 1
SN  - 2193-6536
CY  - Heidelberg [u.a.]
PB  - Springer
M1  - DZNE-2025-00697
SP  - 379 - 398
PY  - 2025
AB  - Friedreich Ataxia (FA) is a multisystem neurodegenerative disease. Affected individuals rely on mobility assistive technologies (MAT) (e.g. wheelchairs) and require long-term treatments and care. To analyse the patients' health-related quality of life (HRQoL), the EuroQol 5 Dimension 3 Level survey (EQ-5D-3L)-a widely used and recommended generic measure-is used in clinical and in health economic studies. Concerns about using the instrument in mobility-impaired individuals who might have difficulties finding appropriate response options for mobility-related items led us to investigate how the 3L dimensions perform in patients with FA using or not using MAT.Using longitudinal data from 607 patients with FA of the EFACTS study (from baseline to the 3-year follow-up), we analysed the acceptability, distribution properties, validity, and responsiveness of the EQ-5D-3L, focusing on the mobility item. Analyses were stratified for patients without and with different MAT-usage.We identified that n = 177 patients used no MAT, n = 299 a wheelchair and n = 131 walking aids. The mobility item non-response was highest in wheelchair users (6.8
KW  - EQ-5D-3L (Other)
KW  - Friedreich Ataxia (Other)
KW  - Health-related quality of life (Other)
KW  - Mobility assistive technology (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:39738982
C2  - pmc:PMC11762039
DO  - DOI:10.1007/s40120-024-00694-7
UR  - https://pub.dzne.de/record/279169
ER  -