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One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development

Lookup NU author(s): Dr Michelle Eagle, Emerita Professor Katherine Bushby


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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.

Publication metadata

Author(s): 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, MDA DMD Clinical Research Network

Publication type: Article

Publication status: Published

Journal: Pediatric Neurology

Year: 2014

Volume: 50

Issue: 6

Pages: 557-563

Print publication date: 01/06/2014

Online publication date: 15/02/2014

Acceptance date: 10/02/2014

ISSN (print): 0887-8994

ISSN (electronic): 1873-5150

Publisher: Elsevier


DOI: 10.1016/j.pediatrneurol.2014.02.006


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Funder referenceFunder name
NIH Roadmap for Medical Research
Muscular Dystrophy Association DMD-center
UL1 RR024992National Center for Research Resources (NCRR), a component of the National Institutes of Health (NIH)