Longitudinal Change in Quality of Life in Neurological Disorders Measures Over 3 Years in Patients With Early Parkinson's Disease

BackgroundThe Quality of Life in Neurological Disorders (Neuro- QoL) is a publicly available health- related quality- of- life measurement system.ObjectiveThe aim of this study was to evaluate the utility of Neuro- QoL item banks as outcome measures for clinical trials in Parkinson’s disease.MethodsAn analysis of Neuro- QoL responsiveness to change and construct validity was performed in a multicenter clinical trial cohort.ResultsAmong 310 participants over 3- years, changes in five of eight Neuro- QoL domains were significant (P <- 0.05) but very modest. The largest effect sizes were seen in the cognition and mobility domains (0.35- 0.39). The largest effect size for change over the year in which levodopa was initiated was - 0.19 for lower extremity function- mobility. For a similarly designed clinical trial, estimated sample size required to demonstrate a 50% reduction in worsening ranged from 420 to more than 1000 participants per group.ConclusionsMore sensitive tools will be required to serve as an outcome measure in early Parkinson’s disease. © 2021 International Parkinson and Movement Disorder SocietyPeer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/169296/1/mds28641.pdfhttp://deepblue.lib.umich.edu/bitstream/2027.42/169296/2/mds28641_am.pd

Longitudinal Change in Quality of Life in Neurological Disorders Measures Over 3- Years in Patients with Early Parkinson’s Disease

BackgroundThe Quality of Life in Neurological Disorders (Neuro- QoL) is a publicly available health- related quality- of- life measurement system.ObjectiveThe aim of this study was to evaluate the utility of Neuro- QoL item banks as outcome measures for clinical trials in Parkinson’s disease.MethodsAn analysis of Neuro- QoL responsiveness to change and construct validity was performed in a multicenter clinical trial cohort.ResultsAmong 310 participants over 3- years, changes in five of eight Neuro- QoL domains were significant (P <- 0.05) but very modest. The largest effect sizes were seen in the cognition and mobility domains (0.35- 0.39). The largest effect size for change over the year in which levodopa was initiated was - 0.19 for lower extremity function- mobility. For a similarly designed clinical trial, estimated sample size required to demonstrate a 50% reduction in worsening ranged from 420 to more than 1000 participants per group.ConclusionsMore sensitive tools will be required to serve as an outcome measure in early Parkinson’s disease. © 2021 International Parkinson and Movement Disorder SocietyPeer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/169296/1/mds28641.pdfhttp://deepblue.lib.umich.edu/bitstream/2027.42/169296/2/mds28641_am.pd