BACKGROUND: Multiple system atrophy (MSA) has considerable effect on health-related quality of life (Hr-QoL). The aim of this study was to prospectively evaluate Hr-QoL by using the MSA health-related Quality of Life (MSA-QoL) scale. METHODS: Evaluation of 100 patients at baseline and after a mean follow-up of 11.5 months was performed. Assessment was made of potential associations with established markers of disease progression. Calculation was performed of sample-size estimates for various effect sizes. RESULTS: MSA-QoL scale scores were less responsive to change than Unified MSA Rating Scale (UMSARS) scores. Responsiveness was largely improved and reasonable sample-size estimates were obtained when limiting the analysis to items with significant change over time. CONCLUSIONS: The UMSARS remains the "gold standard" for disease-modifying/neuroprotection trials. An MSA-QoL Change Scale, based on the most responsive items, may become a valuable tool.
BACKGROUND:Multiple system atrophy (MSA) has considerable effect on health-related quality of life (Hr-QoL). The aim of this study was to prospectively evaluate Hr-QoL by using the MSA health-related Quality of Life (MSA-QoL) scale. METHODS: Evaluation of 100 patients at baseline and after a mean follow-up of 11.5 months was performed. Assessment was made of potential associations with established markers of disease progression. Calculation was performed of sample-size estimates for various effect sizes. RESULTS: MSA-QoL scale scores were less responsive to change than Unified MSA Rating Scale (UMSARS) scores. Responsiveness was largely improved and reasonable sample-size estimates were obtained when limiting the analysis to items with significant change over time. CONCLUSIONS: The UMSARS remains the "gold standard" for disease-modifying/neuroprotection trials. An MSA-QoL Change Scale, based on the most responsive items, may become a valuable tool.
Authors: Tatjana Pekmezović; Milica Ječmenica-Lukić; Igor Petrović; Vladana Špica; Aleksandra Tomić; Vladimir S Kostić Journal: J Neurol Date: 2015-06-13 Impact factor: 4.849