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, 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 & 12 others 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

Research output: Contribution to journalArticle

47 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)529-539
Number of pages11
JournalNeuromuscular Disorders
Volume23
Issue number7
DOIs
StatePublished - Jul 2013

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Duchenne Muscular Dystrophy
Muscular Dystrophies
Research
Language
Outcome Assessment (Health Care)
Child Development
Multicenter Studies
Population

Keywords

  • Bayley III Scales of Infant and Toddler Development
  • Duchenne Muscular Dystrophy
  • Hammersmith Functional Motor Scale Extended
  • Infant
  • North Star Ambulatory Assessment

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Genetics(clinical)
  • Neurology

Cite this

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy : Results from the Muscular Dystrophy Association DMD Clinical Research Network. / Connolly, Anne M.; Florence, Julaine M.; Cradock, Mary M.; Malkus, Elizabeth C.; Schierbecker, Jeanine R.; Siener, Catherine A.; Wulf, Charlie O.; Anand, Pallavi; Golumbek, Paul T.; Zaidman, Craig M.; Philip Miller, J.; Lowes, Linda P.; Alfano, Lindsay N.; Viollet-Callendret, Laurence; Flanigan, Kevin M.; Mendell, Jerry R.; McDonald, Craig M; Goude, Erica; Johnson, Linda; Nicorici, Alina; Karachunski, Peter I.; Day, John W.; Dalton, Joline C.; Farber, Janey M.; Buser, Karen K.; Darras, Basil T.; Kang, Peter B.; Riley, Susan O.; Shriber, Elizabeth; Parad, Rebecca; Bushby, Kate; Eagle, Michelle.

In: Neuromuscular Disorders, Vol. 23, No. 7, 07.2013, p. 529-539.

Research output: Contribution to journalArticle

Connolly, AM, Florence, JM, Cradock, MM, Malkus, EC, Schierbecker, JR, Siener, CA, Wulf, CO, Anand, P, Golumbek, PT, Zaidman, CM, Philip Miller, J, Lowes, LP, Alfano, LN, Viollet-Callendret, L, Flanigan, KM, Mendell, JR, McDonald, CM, Goude, E, Johnson, L, Nicorici, A, Karachunski, PI, Day, JW, Dalton, JC, Farber, JM, Buser, KK, Darras, BT, Kang, PB, Riley, SO, Shriber, E, Parad, R, Bushby, K & Eagle, M 2013, 'Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network', Neuromuscular Disorders, vol. 23, no. 7, pp. 529-539. https://doi.org/10.1016/j.nmd.2013.04.005
Connolly, Anne M. ; Florence, Julaine M. ; Cradock, Mary M. ; Malkus, Elizabeth C. ; Schierbecker, Jeanine R. ; Siener, Catherine A. ; Wulf, Charlie O. ; Anand, Pallavi ; Golumbek, Paul T. ; Zaidman, Craig M. ; Philip Miller, J. ; Lowes, Linda P. ; Alfano, Lindsay N. ; Viollet-Callendret, Laurence ; Flanigan, Kevin M. ; Mendell, Jerry R. ; McDonald, Craig M ; Goude, Erica ; Johnson, Linda ; Nicorici, Alina ; Karachunski, Peter I. ; Day, John W. ; Dalton, Joline C. ; Farber, Janey M. ; Buser, Karen K. ; Darras, Basil T. ; Kang, Peter B. ; Riley, Susan O. ; Shriber, Elizabeth ; Parad, Rebecca ; Bushby, Kate ; Eagle, Michelle. / Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy : Results from the Muscular Dystrophy Association DMD Clinical Research Network. In: Neuromuscular Disorders. 2013 ; Vol. 23, No. 7. pp. 529-539.
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