Health-Related Quality of Life in Patients with Spinocerebellar Ataxia: a Validation Study of the EQ-5D-3L

• DOI: 10.1007/s12311-023-01597-3
• Publication Date: 2023-09-15T09:06:00Z
• Authors: Maresa Buchholz; Niklas Weber; Anika Rädke; Jennifer Faber; Tanja Schmitz-Hübsch; Heike Jacobi; Feng Xie; Thomas Klockgether; Bernhard Michalowsky; ; Sophie Tezenas du...; Peter Bauer; Paola Giunti; Arron Cook; Robyn Labrum; Michael H. Parkinson; Alexandra Durr; Alexis Brice; Perrine Charles; Cecilia Marelli; Caterina Mariotti; Lorenzo Nanetti; Marta Panzeri; Maria Rakowicz; Anna Sulek; Anna Sobanska; Ludger Schöls; Holger Hengel; Laszlo Baliko; Bela Melegh; Alessandro Filla; Antonella Antenora; Jon Infante; José Berciano; Bart P. van de Wa...; Dagmar Timmann; Sandra Szymanski; Sylvia Boesch; Jun-Suk Kang; Massimo Pandolfo; Jörg B. Schulz; Sonia Molho; Alhassane Diallo; ; Jeanette Hübener-...; Magda Santana; Marcus Grobe-Einsler; Berkan Koyak; Mafalda Raposo; Manuela Lima; Hector Garcia-Moreno; Paola Giunti; Luís Pereira de A...; Bart van de Warre...; Judith van Gaalen; Dagmar Timmann; Andreas Thieme; Kathrin Reetz; Imis Dogan; Carlo Wilke; Ludger Schöls; Olaf Riess; Matthis Synofzik; Jeroen de Vries; Jon Infante; Oz Gulin; James Joers; Chiadikaobi Onyike; Michal Povazan; Eva-Maria Ratai; Jeremy Schmahmann
• Keywords: EQ-5D; Convergent validity

Abstract Although health-related quality of life (HRQoL) has developed into a crucial outcome parameter in clinical research, evidence the EQ-5D-3L validation performance is lacking patients with spinocerebellar ataxia (SCA) types 1, 2, 3, and 6. The objective this study to assess acceptability, validity, reliability, responsiveness EQ-5D-3L. For n = 842 predominantly European SCA two longitudinal cohort studies, EQ-5D-3L, PHQ-9 (Patient Health Questionnaire), ataxia-specific assessments (SARA: Scale for Assessment Rating Ataxia; ADL: activities daily living as part Friedreich’s Ataxia Scale; INAS: Inventory Non-Ataxia Signs) were assessed at baseline multiple annual follow-ups. was evaluated regarding distribution properties, convergent known-groups test-retest effect size measures analyze health changes. non-item response low (EQ-5D-3L index: 0.8%; EQ-VAS: 3.4%). Ceiling effects occurred 9.9% (EQ-5D-3L) 3.0% (EQ-VAS) mean index 0.65 ± 0.21. In total, validity showed moderate strong Spearman’s rho ( r s > 0.3) coefficients comparing EQ-VAS PHQ-9, SARA, ADL, INAS. could discriminate between groups age, Intra-class correlation ICC : 0.95/EQ-VAS 0.88) Kappa statistics (range 0.44 0.93 items) indicated tolerable reliability. shows small (effect < 0.3–0.59) changes severity. analysis confirms an acceptable, reliable, valid, responsive recommended patients, measuring HRQoL adequately, besides well-established instruments.