Compound muscle action potential and motor function in children with spinal muscular atrophy |
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| Authors: | Aga Lewelt MD Kristin J. Krosschell PT Charles Scott PhD Ai Sakonju MD John T. Kissel MD Thomas O. Crawford MD Gyula Acsadi MD PhD Guy D'anjou MD Bakri Elsheikh MD Sandra P. Reyna MD Mary K. Schroth MD Jo Anne Maczulski OT Gregory J. Stoddard MPH Elie Elovic MD Kathryn J. Swoboda MD |
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| Affiliation: | 1. Division of Physical Medicine and Rehabilitation, University of Utah School of Medicine, 30 North 1900 East, Salt Lake City, Utah 84132, USA;2. Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA;3. CBS Squared, Inc., Fort Washington, Pennsylvania, USA;4. Department of Neurology, University of Utah School of Medicine, Salt Lake City, Utah, USA;5. Department of Neurology, Ohio State University Medical Center, Columbus, Ohio, USA;6. Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA;7. Department of Neurology, Wayne State University School of Medicine, Detroit, Michigan, USA;8. Division of Pediatric Neurology, H?pital Sainte‐Justine Montréal, Montréal, Québec, Canada;9. Department of Pediatrics, University of Wisconsin School of Medicine, Madison, Wisconsin, USA;10. Pediatric Occupational Therapy Services, Chicago, Illinois, USA;11. Department of Internal Medicine, University of Utah School of Medicine, Salt Lake City, Utah, USA |
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| Abstract: | Reliable outcome measures that reflect the underlying disease process and correlate with motor function in children with SMA are needed for clinical trials. Maximum ulnar compound muscle action potential (CMAP) data were collected at two visits over a 4–6‐week period in children with SMA types II and III, 2–17 years of age, at four academic centers. Primary functional outcome measures included the Modified Hammersmith Functional Motor Scale (MHFMS) and MHFMS‐Extend. CMAP negative peak amplitude and area showed excellent discrimination between the ambulatory and non‐ambulatory SMA cohorts (ROC = 0.88). CMAP had excellent test–retest reliability (ICC = 0.96–0.97, n = 64) and moderate to strong correlation with the MHFMS and MHFMS‐Extend (r = 0.61–0.73, n = 68, P < 0.001). Maximum ulnar CMAP amplitude and area is a feasible, valid, and reliable outcome measure for use in pediatric multicenter clinical trials in SMA. CMAP correlates well with motor function and has potential value as a relevant surrogate for disease status. Muscle Nerve, 2010 |
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| Keywords: | compound muscle action potential motor function outcome measures pediatrics spinal muscular atrophy |
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