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. 2013 Jul;23(7):529-39.
doi: 10.1016/j.nmd.2013.04.005. Epub 2013 May 28.

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

Anne M Connolly  1 , Julaine M Florence, Mary M Cradock, Elizabeth C Malkus, Jeanine R Schierbecker, Catherine A Siener, Charlie O Wulf, Pallavi Anand, Paul T Golumbek, Craig M Zaidman, J Philip Miller, Linda P Lowes, Lindsay N Alfano, Laurence Viollet-Callendret, Kevin M Flanigan, Jerry R Mendell, Craig M McDonald, Erica Goude, Linda Johnson, Alina Nicorici, Peter I Karachunski, John W Day, Joline C Dalton, Janey M Farber, Karen K Buser, Basil T Darras, Peter B Kang, Susan O Riley, Elizabeth Shriber, Rebecca Parad, Kate Bushby, Michelle Eagle; MDA DMD Clinical Research Network
Affiliations

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network

Anne M Connolly et al. Neuromuscul Disord. 2013 Jul.

Abstract

Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III Scales of Infant and Toddler Development (Bayley III) have been validated in developing children and those with developmental disorders but have not been studied in DMD. Expanded Hammersmith Functional Motor Scale (HFMSE) and North Star Ambulatory Assessment (NSAA) may also be useful in this young DMD population. Clinical evaluators from the MDA-DMD Clinical Research Network were trained in these assessment tools. Infants and boys with DMD (n = 24; 1.9 ± 0.7 years) were assessed. The mean Bayley III motor composite score was low (82.8 ± 8; p ≤ .0001) (normal = 100 ± 15). Mean gross motor and fine motor function scaled scores were low (both p ≤ .0001). The mean cognitive comprehensive (p=.0002), receptive language (p ≤ .0001), and expressive language (p = .0001) were also low compared to normal children. Age was negatively associated with Bayley III gross motor (r = -0.44; p = .02) but not with fine motor, cognitive, or language scores. HFMSE (n=23) showed a mean score of 31 ± 13. NSAA (n = 18 boys; 2.2 ± 0.4 years) showed a mean score of 12 ± 5. Outcome assessments of young boys with DMD are feasible and in this multicenter study were best demonstrated using the Bayley III.

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Figures

Figure 1
Figure 1
Figure 1A. Distribution of Bayley-III Cognitive Composite scores of DMD boys compared to normal children. There was a shift of the distribution to the left (t = −4.36, p=.0001). Figure 1B. Distribution of Bayley-III Language Composite scores of DMD boys compared to normal children(n=24). There was a shift of the distribution to the left (t=−4.42, p<.0001). Figure 1C. Distribution of Bayley-III Motor Composite scores of DMD boys compared to normal children(n=23). There was a shift of the distribution to the left (t =−7.60, p=<.0001).
Figure 1
Figure 1
Figure 1A. Distribution of Bayley-III Cognitive Composite scores of DMD boys compared to normal children. There was a shift of the distribution to the left (t = −4.36, p=.0001). Figure 1B. Distribution of Bayley-III Language Composite scores of DMD boys compared to normal children(n=24). There was a shift of the distribution to the left (t=−4.42, p<.0001). Figure 1C. Distribution of Bayley-III Motor Composite scores of DMD boys compared to normal children(n=23). There was a shift of the distribution to the left (t =−7.60, p=<.0001).
Figure 1
Figure 1
Figure 1A. Distribution of Bayley-III Cognitive Composite scores of DMD boys compared to normal children. There was a shift of the distribution to the left (t = −4.36, p=.0001). Figure 1B. Distribution of Bayley-III Language Composite scores of DMD boys compared to normal children(n=24). There was a shift of the distribution to the left (t=−4.42, p<.0001). Figure 1C. Distribution of Bayley-III Motor Composite scores of DMD boys compared to normal children(n=23). There was a shift of the distribution to the left (t =−7.60, p=<.0001).
Figure 2
Figure 2
Figure 2A. Bayley-III Gross Motor Scaled Scores versus Age (n=24). There was a negative correlation with age (r = −0.45; p=.03) demonstrating motor function relative to typically developing children is low. Figure 2B. Hammersmith Functional Motor Extended versus Age (n=23). There was a positive correlation with age (r = 0.63 p=.001) showing absolute motor gains are demonstrated across time. Figure 2C. North Star Ambulatory Assessment (n=18). There was not a significant correlation with age (r = 0.26; p=.3).
Figure 2
Figure 2
Figure 2A. Bayley-III Gross Motor Scaled Scores versus Age (n=24). There was a negative correlation with age (r = −0.45; p=.03) demonstrating motor function relative to typically developing children is low. Figure 2B. Hammersmith Functional Motor Extended versus Age (n=23). There was a positive correlation with age (r = 0.63 p=.001) showing absolute motor gains are demonstrated across time. Figure 2C. North Star Ambulatory Assessment (n=18). There was not a significant correlation with age (r = 0.26; p=.3).
Figure 2
Figure 2
Figure 2A. Bayley-III Gross Motor Scaled Scores versus Age (n=24). There was a negative correlation with age (r = −0.45; p=.03) demonstrating motor function relative to typically developing children is low. Figure 2B. Hammersmith Functional Motor Extended versus Age (n=23). There was a positive correlation with age (r = 0.63 p=.001) showing absolute motor gains are demonstrated across time. Figure 2C. North Star Ambulatory Assessment (n=18). There was not a significant correlation with age (r = 0.26; p=.3).
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