Connie Marras1, Kelly A Mills2, Shirley Eberly3, David Oakes3, Kelvin L Chou4, Matthew Halverson5, Sotirios A Parashos6, Christopher G Tarolli7, Jin-Shei Lai8, Cindy J Nowinsky9, Oksana Suchowersky10, Eric S Farbman11, Lisa M Shulman12, Tanya Simuni13. 1. The Edmond J Safra Program in Parkinson's Research and the Morton and Gloria Shulman Movement Disorders Centre, Toronto Western Hospital, University of Toronto, Toronto, Ontario, Canada. 2. Johns Hopkins Parkinson's Disease and Movement Disorders Center, Johns Hopkins University, Baltimore, Maryland, USA. 3. Department of Biostatistics and Computational Biology, University of Rochester Medical Center, Rochester, New York, USA. 4. Departments of Neurology and Neurosurgery, University of Michigan Medical School, Ann Arbor, Michigan, USA. 5. University of Utah, Salt Lake City, Utah, USA. 6. Struthers Parkinson's Center, Park Nicollet Health Services, Golden Valley, Minnesota, USA. 7. Department of Neurology, University of Rochester, Rochester, New York, USA. 8. Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA. 9. Departments of Medical Social Sciences and Neurology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA. 10. Department of Medicine (Neurology), Medical Genetics and Pediatrics, University of Alberta, Edmonton, Alberta, Canada. 11. Department of Neurology, Roseman University of Health Sciences, Las Vegas, Nevada, USA. 12. Department of Neurology, University of Maryland School of Medicine, Baltimore, Maryland, USA. 13. Department of Neurology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA.
Abstract
BACKGROUND: The Quality of Life in Neurological Disorders (Neuro-QoL) is a publicly available health-related quality-of-life measurement system. OBJECTIVE: The aim of this study was to evaluate the utility of Neuro-QoL item banks as outcome measures for clinical trials in Parkinson's disease. METHODS: An analysis of Neuro-QoL responsiveness to change and construct validity was performed in a multicenter clinical trial cohort. RESULTS: Among 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. CONCLUSIONS: More sensitive tools will be required to serve as an outcome measure in early Parkinson's disease.
BACKGROUND: The Quality of Life in Neurological Disorders (Neuro-QoL) is a publicly available health-related quality-of-life measurement system. OBJECTIVE: The aim of this study was to evaluate the utility of Neuro-QoL item banks as outcome measures for clinical trials in Parkinson's disease. METHODS: An analysis of Neuro-QoL responsiveness to change and construct validity was performed in a multicenter clinical trial cohort. RESULTS: Among 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. CONCLUSIONS: More sensitive tools will be required to serve as an outcome measure in early Parkinson's disease.
Authors: Lisa M Shulman; Ann L Gruber-Baldini; Karen E Anderson; Christopher G Vaughan; Stephen G Reich; Paul S Fishman; William J Weiner Journal: Mov Disord Date: 2008-04-30 Impact factor: 10.338
Authors: Christopher G Goetz; Barbara C Tilley; Stephanie R Shaftman; Glenn T Stebbins; Stanley Fahn; Pablo Martinez-Martin; Werner Poewe; Cristina Sampaio; Matthew B Stern; Richard Dodel; Bruno Dubois; Robert Holloway; Joseph Jankovic; Jaime Kulisevsky; Anthony E Lang; Andrew Lees; Sue Leurgans; Peter A LeWitt; David Nyenhuis; C Warren Olanow; Olivier Rascol; Anette Schrag; Jeanne A Teresi; Jacobus J van Hilten; Nancy LaPelle Journal: Mov Disord Date: 2008-11-15 Impact factor: 10.338