Forschung & Innovation
Publikationen
Stage-Specific Health Status in Multiple System Atrophy
PMU Autor*in
Stefan Lorenzl
Alle Autor*innen
Igor Kuchin, Sibylle Puntscher, Gaby Sroczynski, Marjan Arvandi, Julia Santamaria Navarro, Georg Goebel, Anette Schrag, Fabian Leys, Bianca Caliò, Frank Jagusch, Jean-Pierre Ndayisaba, Maria Schneller, Klaus Seppi, Stefan Lorenzl, Stefan Kiechl, Richard Dodel, Werner Poewe, Florian Krismer, Petra Schwingenschuh, Alessandra Fanciulli, Uwe Siebert, Beate Jahn,
Fachzeitschrift
MOVEMENT DISORDERS
Kurzfassung
BACKGROUND: Multiple system atrophy (MSA) is a rare neurodegenerative disorder that severely impairs health-related quality of life (HRQoL). Disease progression is assessed using the Unified Multiple System Atrophy Rating Scale Part IV (UMSARS IV), which defines five stages. However, stage-specific HRQoL estimates, particularly health-utility values, are unavailable. These estimates are essential for health-economic evaluation of treatments and for supporting HRQoL as a clinical trial endpoint. Bayesian methods offer a practical approach for analyzing HRQoL in rare diseases.
OBJECTIVE: To estimate EQ-5D-based health-utility values across UMSARS IV stages in MSA.
METHODS: EQ-5D data from the European MSA Study Group cohort were analyzed. EQ-5D-3L responses were mapped to EQ-5D-5L health-utility values using the German value set, where a value of 1 represents perfect health and lower or negative values indicate worse health. Bayesian modeling estimated stage-specific health-utility values, reporting posterior means and 95% credible intervals (CrI). Sensitivity analyses included alternative model specifications and imputations of missing UMSARS IV stages.
RESULTS: Stage-specific EQ-5D-5L health-utility values were 0.672 (95% CrI 0.628-0.719) for UMSARS IV Stage 1, 0.606 (95% CrI 0.523-0.679) for Stage 2, 0.517 (95% CrI 0.416-0.615) for Stage 3, 0.262 (95% CrI 0.158-0.364) for Stage 4, and 0.084 (95% CrI -0.045 to 0.217) for Stage 5. Bayesian models demonstrated good fit, capturing variability within stages and across countries. Sensitivity analyses confirmed robustness, with health-utility values reflecting expected MSA progression.
CONCLUSIONS: This study is the first to provide stage-specific health-utility values across all UMSARS IV stages, enabling health-economic evaluations of emerging interventions and supporting HRQoL as a clinical trial endpoint. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.