A new analysis published in Developmental Medicine & Child Neurology reveals that changes in height and weight over time may be closely tied to motor function outcomes in young boys with Duchenne muscular dystrophy (DMD). These findings could have implications for treatment discussions and weight management strategies, researchers say.
The study followed 194 ambulatory boys aged 4 to 7 years across 32 sites in five countries. The boys were randomly assigned to one of three glucocorticoid treatment regimens: daily prednisone, intermittent prednisone or deflazacort.
The researchers tracked height, weight and body mass index (BMI) over several years and examined how these related to performance on functional outcome measures, including walking, running and motor assessment tests.
The results showed that boys who were taller at the start of glucocorticoid therapy experienced slower subsequent growth, while older children gained weight more rapidly once on treatment. The type and schedule of steroid therapy influenced height and weight trajectories, though BMI patterns didn’t change much between groups.
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Importantly, declines in height and weight scores over time were associated with poorer performance on motor assessment tests. These correlations were weak within the first three years of treatment but became more pronounced after five years, around the age when many boys with DMD begin to lose the ability to walk.
The researchers say that the findings reinforce the importance of closely monitoring growth alongside motor function in children with DMD. They also suggest that weight management should be an integral part of care, since unfavorable trajectories appear linked to worse outcomes in mobility and independence.
“Our findings highlight factors affecting height, weight, and BMI z-scores, and their impact on COA [clinical outcome assessments] performance,” the study’s authors concluded. “This emphasizes the importance of monitoring weight changes and understanding how baseline traits influence growth and motor function.”
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