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.
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.
Authors: Allan M Glanzman; Michael P McDermott; Jacqueline Montes; William B Martens; Jean Flickinger; Susan Riley; Janet Quigley; Sally Dunaway; Jessica O'Hagen; Liyong Deng; Wendy K Chung; Rabi Tawil; Basil T Darras; Michele Yang; Douglas Sproule; Darryl C De Vivo; Petra Kaufmann; Richard S Finkel Journal: Pediatr Phys Ther Date: 2011 Impact factor: 3.049
Authors: Petra Kaufmann; Michael P McDermott; Basil T Darras; Richard S Finkel; Douglas M Sproule; Peter B Kang; Maryam Oskoui; Andrei Constantinescu; Clifton L Gooch; A Reghan Foley; Michele L Yang; Rabi Tawil; Wendy K Chung; William B Martens; Jacqueline Montes; Vanessa Battista; Jessica O'Hagen; Sally Dunaway; Jean Flickinger; Janet Quigley; Susan Riley; Allan M Glanzman; Maryjane Benton; Patricia A Ryan; Mark Punyanitya; Megan J Montgomery; Jonathan Marra; Benjamin Koo; Darryl C De Vivo Journal: Neurology Date: 2012-10-17 Impact factor: 9.910
Authors: Anna Mayhew; Stefan Cano; Elaine Scott; Michelle Eagle; Kate Bushby; Francesco Muntoni Journal: Dev Med Child Neurol Date: 2011-03-17 Impact factor: 5.449
Authors: Kristin J Krosschell; Michael Bosch; Leslie Nelson; Tina Duong; Linda P Lowes; Lindsay N Alfano; Danielle Benjamin; Terri B Carry; Ginger Devine; Carolyn Kelley; Rebecca Gadekan; Elizabeth C Malkus; Amy Pasternak; Stephanie Provance-Orr; Lynne Roemeiser-Logan; Alina Nicorici; Donata Trussell; Sally Dunaway Young; Jennifer R Fetterman; Jacqueline Montes; Penny J Powers; Rebecca Quinones; Janet Quigley; Christopher S Coffey; Jon W Yankey; Amy Bartlett; John T Kissel; Stephen J Kolb Journal: J Neuromuscul Dis Date: 2018
Authors: Danielle Ramsey; Mariacristina Scoto; Anna Mayhew; Marion Main; Elena S Mazzone; Jacqueline Montes; Roberto de Sanctis; Sally Dunaway Young; Rachel Salazar; Allan M Glanzman; Amy Pasternak; Janet Quigley; Elizabeth Mirek; Tina Duong; Richard Gee; Matthew Civitello; Gihan Tennekoon; Marika Pane; Maria Carmela Pera; Kate Bushby; John Day; Basil T Darras; Darryl De Vivo; Richard Finkel; Eugenio Mercuri; Francesco Muntoni Journal: PLoS One Date: 2017-02-21 Impact factor: 3.240
Authors: Heike Kölbel; Berthold P Hauffa; Stefan A Wudy; Anastasios Bouikidis; Adela Della Marina; Ulrike Schara Journal: PLoS One Date: 2017-03-09 Impact factor: 3.240