LB: Gross motor delays in boys with Duchenne Muscular Dystrophy are seen in infancy

Topic:

Clinical Trials

Poster Number: T451

Author(s):

Natalie Rease, DPT, AWRI @ Nationwide Children’s Hospital, Anne Connolly, MD, AWRI @ Nationwide Children’s Hospital, Megan Iammarino, PT, DPT, AWRI @ Nationwide Children’s Hospital, Lindsay Pietruszewski, PT, DPT, University of California Los Angeles Center for Cerebral Palsy, Melissa Smith, PT, DPT, AWRI @ Nationwide Children’s Hospital, Jing Peng, PhD, The Ohio State Center for Biostatistics, Christopher Steiner, PT, DPT, AWRI @ Nationwide Children’s Hospital, Chang-Yong Tsao, MD, Nationwide Children's Hospital, Megan Waldrop, MD, AWRI @ Nationwide Children’s Hospital, Kevin Flanigan, MD, AWRI @ Nationwide Children’s Hospital, Shannon Chagat, NP, Nationwide Children’s Hospital, Alayne Meyer, MS, LGC, Nationwide Children’s Hospital, Jerry Mendell, MD, Nationwide Children's Hospital, Lindsay Alfano, DPT, PCS, AWRI @ Nationwide Children’s Hospital

Aim: Determine the prevalence of gross motor delay and document the trajectory of gross motor skill acquisition in boys with Duchenne muscular dystrophy using the Bayley Scales of Infant & Toddler Development, Third Edition.

Method: Ninety boys (2 months – 5 years 10 months at their first visit) were evaluated as part of their standard of care during regularly scheduled clinic visits.  This sample of convenience than allowed longitudinal assessments for 47 boys, with two to six follow up visits. This resulting in 128 total assessments.

Results: As expected, most boys with DMD demonstrated delays in gross motor skills across the age span when compared with normative controls.

Interpretation: A well-documented developmental natural history study could be used to determine if the rate of change in gross motor development following an intervention falls outside of expectations for DMD.