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Rasch analysis of clinical outcome measures in spinal muscular atrophy

Authors:	Stefan J Cano Anna Mayhew Allan M Glanzman Kristin J Krosschell Kathryn J Swoboda Marion Main Birgit F Steffensen Carole Brard Franoise Girardot Christine AM Payan Eugenio Mercuri Elena Mazzone Bakri Elsheikh Julaine Florence Linda S Hynan Susan T Iannaccone Leslie L Nelson Shree Pandya Michael Rose Charles Scott Reza Sadjadi Mackensie A Yore Cynthia Joyce John T Kissel

Institution:	1. Clinical Neurology Research Group, Room N13 ITTC Building, Peninsula College of Medicine and Dentistry, , Plymouth, United Kingdom;2. Newcastle Muscle Team, Institute of Human Genetics, International Centre for Life, , Newcastle, United Kingdom;3. Department of Physical Therapy, The Children's Hospital of Philadelphia, , Philadelphia, Pennsylvania, USA;4. Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine Northwestern University, , Chicago, Illinois, USA;5. University of Utah School of Medicine, , Salt Lake City, Utah, USA;6. Great Ormond Street Children's Hospital for the Dubowitz Neuromuscular Center, , London, England;7. Rehabiliterings Center for Muskelsvind, , Aarhus, Denmark;8. Centre Hospitalier, , Lyon Sud, France;9. Department de Pharmacologie Clinique, H?pital Pitié‐Salpêtrière, , Paris, France;10. Department of Paediatric Neurology, Neuropsichiatra Infantile, , Rome, Italy;11. Division of Neuromuscular Medicine, Department of Neurology, The Ohio State University, , Columbus, Ohio, USA;12. Neuromuscular Division;13. Department of Neurology, Washington University School of Medicine, , St. Louis, Missouri, USA;14. Departments of Clinical Sciences (Biostatistics) and Psychiatry, University of Texas Southwestern Medical Center, , Dallas, Texas, USA;15. Pediatrics and Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, , Dallas, Texas, USA;16. Department of Physical Therapy, University of Texas Southwestern Medical Center, , Dallas, Texas, USA;17. Department of Neurology, University of Rochester School of Medicine and Dentistry, , Rochester, New York, USA;18. Department of Neurology, King's College Hospital, , Denmark Hill, London, United Kingdom;19. CBS Squared, Inc, Fort Washington, PA for Project Cure, , USA;20. Department of Neurology, University of Virginia, , Charlottesville, Virginia, USA;21. Spinal Muscular Atrophy Foundation, , New York, New York, USA;22. Departments of Neurology and Pediatrics, The Ohio State University/Nationwide Children's Hospital, , Columbus, Ohio, 43210 USA

Abstract:	Introduction: Trial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA). Methods: Data from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index. Results: Each scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum. Conclusions: The sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges. Muscle Nerve 49 :422–430, 2014

Keywords:	clinical trials motor neuron disease outcome measures Rasch analysis spinal muscular atrophy

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