Rasch analysis of clinical outcome measures in spinal muscular atrophy

Stefan J. Cano, Anna Mayhew, Allan M. Glanzman, Kristin J. Krosschell, Kathryn J. Swoboda, Marion Main, Birgit F. Steffensen, Carole Bérard, Françoise Girardot, Christine A.M. Payan, Eugenio Maria Mercuri, Elena Mazzone, Bakri Elsheikh, Julaine Florence, Linda S. Hynan, Susan T. Iannaccone, Leslie L. Nelson, Shree Pandya, Michael Rose, Charles ScottReza Sadjadi, Mackensie A. Yore, Cynthia Joyce, John T. Kissel

Risultato della ricerca: Contributo in rivistaArticolo in rivista

40 Citazioni (Scopus)


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.
Lingua originaleEnglish
pagine (da-a)422-430
Numero di pagine9
Stato di pubblicazionePubblicato - 2014


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


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