Rasch analysis of clinical outcome measures in spinal muscular atrophy
SJ. Cano, A. Mayhew, AM. Glanzman, KJ. Krosschell, KJ. Swoboda, M. Main, BF. Steffensen, C. Bérard, F. Girardot, CA. Payan, E. Mercuri, E. Mazzone, B. Elsheikh, J. Florence, LS. Hynan, ST. Iannaccone , LL. Nelson, S. Pandya, M. Rose, C. Scott, R. Sadjadi, MA. Yore, C. Joyce, JT Kissel; International Coordinating Committee for SMA Clinical Trials Rasch Task Force.
Muscle Nerve. 2014. 49(3):422-30.
Preliminary analysis of 9 motor scales in spinal muscular atrophy (SMA) using the Rasch methodology. Results showed that 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.
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.
Keywords: clinical trials, motor neuron disease, outcome measures, Rasch analysis, spinal muscular atrophy
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