Factors Influencing Health-Related Quality of Life of Patients with Spinocerebellar Ataxia
Niklas Weber, Maresa Buchholz, Anika Rädke, Jennifer Faber, Tanja Schmitz‐Hübsch, Heike Jacobi, Thomas Klockgether, Wolfgang Hoffmann, Bernhard Michalowsky, the EUROSCA study group, Sophie Tézenas du Montcel, Peter Bauer, Paola Giunti, Arron Cook, Robyn Labrum, Michael Parkinson, Alexandra Dürr, Alexis Brice, Perrine Charles, Cécilia Marelli, Caterina Mariotti, Lorenzo Nanetti, Marta Panzeri, Maria Rakowicz, Anna Sułek, Anna Sobańska, Lüdger Schöls, Holger Hengel, László Balikó, Béla Melegh, Alessandro Filla, Antonella Antenora, Jon Infante, José Berciano, Bart P.C. van de Warrenburg, Dagmar Timmann, Sandra Szymanski, Sylvia Boesch, Jun‐Suk Kang, Massimo Pandolfo, Jörg B. Schulz, Sonia Molho, Alhassane Diallo, the ESMI study group, Jeanette Hübener-Schmid, Magda M. Santana, Marcus Grobe‐Einsler, Berkan Koyak, Mafalda Raposo, Manuela Lima, Héctor García‐Moreno, Paola Giunti, Luís Pereira de Almeida, Bart van de Warrenburg, Judith van Gaalen, Dagmar Timmann, Andreas Thieme, Kathrin Reetz, Imis Dogan, Carlo Wilke, Lüdger Schöls, Olaf Rieß, Matthis Synofzik, Jeroen de Vries, Jon Infante, Oz Gulin, James M. Joers, Chiadikaobi Onyike, Michal Považan, Eva‐Maria Ratai, Jeremy D. Schmahmann
Abstract
Abstract Background Little is known about the progression of health-related quality of life (HRQoL) and predicting factors in spinocerebellar ataxia (SCA). Such knowledge is crucial to identify modifiable factors promoting everyday life with SCA and attenuating HRQoL decline. Objectives This study is to assess HRQoL progression and identify factors affecting SCA patients’ HRQoL. Methods Longitudinal data (three-year follow-up) of 310 SCA patients of the European SCA3/Machado-Joseph-Disease Initiative (ESMI) (2016-2022) and 525 SCA patients (SCA1, SCA2, SCA3 or SCA6) of the EUROSCA natural history study cohort (2006–2015) were assessed. Both large cohort studies share standardized assessments of clinical measures, SARA, INAS, PHQ-9, and HRQoL (EQ-5D-3L). The association between HRQoL and clinical measures was assessed by Spearman Correlation (r s ). Multivariable panel regression models were performed to evaluate the impact of patients’ socio-demographics, age of onset, SCA type and body mass index (BMI), and clinical measures on HRQoL progression. Results HRQoL significantly decreased over one (− 0.014, p = 0.095), two (− 0.028, p = 0.003), and three years (− 0.032, p = 0.002). Ataxia severity and mental health strongly correlated with HRQoL (r s SARA = − 0.589; r s PHQ-9 = − 0.507). HRQoL more intensively declined in male (ß = − 0.024, p = 0.038) patients with an earlier age of onset (ß = 0.002, p = 0.058). Higher progression of ataxia severity (ß = − 0.010, p ≤ 0.001), mental health problems (ß = − 0.012, p < 0.001), and higher BMI (ß = − 0.003, p = 0.029) caused more severe decline of patients’ HRQoL over time. Discussion In absence of curative treatments, stronger focus on mental health and weight influence could help clinical evaluation and accompany treatment improving SCA patients’ HRQoL, especially in male patients with early disease onset.