One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

Anne M. Connolly, Julaine M. Florence, Mary M. Cradock, Michelle Eagle, Kevin M. Flanigan, Craig M McDonald, Peter I. Karachunski, Basil T. Darras, Kate Bushby, Elizabeth C. Malkus, Paul T. Golumbek, Craig M. Zaidman, J. Philip Miller, Jerry R. Mendell

Research output: Contribution to journalArticle

16 Citations (Scopus)

Abstract

Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.

Original languageEnglish (US)
Pages (from-to)557-563
Number of pages7
JournalPediatric Neurology
Volume50
Issue number6
DOIs
StatePublished - 2014

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Duchenne Muscular Dystrophy
Child Development
Outcome Assessment (Health Care)
Clinical Trials
Parturition

Keywords

  • Bayley-III
  • clinical trial outcomes
  • Duchenne muscular dystrophy
  • infant development

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Developmental Neuroscience
  • Neurology

Cite this

One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development. / Connolly, Anne M.; Florence, Julaine M.; Cradock, Mary M.; Eagle, Michelle; Flanigan, Kevin M.; McDonald, Craig M; Karachunski, Peter I.; Darras, Basil T.; Bushby, Kate; Malkus, Elizabeth C.; Golumbek, Paul T.; Zaidman, Craig M.; Miller, J. Philip; Mendell, Jerry R.

In: Pediatric Neurology, Vol. 50, No. 6, 2014, p. 557-563.

Research output: Contribution to journalArticle

Connolly, AM, Florence, JM, Cradock, MM, Eagle, M, Flanigan, KM, McDonald, CM, Karachunski, PI, Darras, BT, Bushby, K, Malkus, EC, Golumbek, PT, Zaidman, CM, Miller, JP & Mendell, JR 2014, 'One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development', Pediatric Neurology, vol. 50, no. 6, pp. 557-563. https://doi.org/10.1016/j.pediatrneurol.2014.02.006
Connolly, Anne M. ; Florence, Julaine M. ; Cradock, Mary M. ; Eagle, Michelle ; Flanigan, Kevin M. ; McDonald, Craig M ; Karachunski, Peter I. ; Darras, Basil T. ; Bushby, Kate ; Malkus, Elizabeth C. ; Golumbek, Paul T. ; Zaidman, Craig M. ; Miller, J. Philip ; Mendell, Jerry R. / One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development. In: Pediatric Neurology. 2014 ; Vol. 50, No. 6. pp. 557-563.
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abstract = "Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.",
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T1 - One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

AU - Connolly, Anne M.

AU - Florence, Julaine M.

AU - Cradock, Mary M.

AU - Eagle, Michelle

AU - Flanigan, Kevin M.

AU - McDonald, Craig M

AU - Karachunski, Peter I.

AU - Darras, Basil T.

AU - Bushby, Kate

AU - Malkus, Elizabeth C.

AU - Golumbek, Paul T.

AU - Zaidman, Craig M.

AU - Miller, J. Philip

AU - Mendell, Jerry R.

PY - 2014

Y1 - 2014

N2 - Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.

AB - Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.

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