Neurobehavioral Concerns Among Males with Dystrophinopathy Using Population-Based Surveillance Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network

Abstract

Objective To describe the occurrence of selected neurobehavioral concerns among males with a dystrophinopathy and explore associations with corticosteroid or supportive device use. Method Medical record abstraction of neurobehavioral concerns was conducted for 857 affected males from 765 families, born since 1982 and followed through 2011, and enrolled in the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network. Cumulative probabilities for attention deficit/hyperactivity disorder (ADHD), behavior problems, and depressed mood were calculated from Kaplan-Meier estimates for the subsample of oldest affected males (n=765). Hazard ratios (HRs) and 95% confidence intervals (95%CIs) for corticosteroid and supportive device use were estimated from Cox regression models with time-dependent covariates. Results Of the 857 affected males, 375 (44%) had at least one of the three selected neurobehavioral concerns; a similar percentage (45%) was found among the 765 oldest affected males. The estimated cumulative probabilities among these oldest affected males were 23% for ADHD, 43% for behavior problems, and 51% for depressed mood. Corticosteroid (HR=2.35, 95%CI=1.75,3.16) and mobility device (HR=1.53, 95%CI=1.06,2.21) use were associated with behavior problems. Use of a mobility device (HR=3.53, 95%CI=2.13,5.85), but not corticosteroids, was associated with depressed mood. ADHD was not significantly associated with corticosteroid or mobility device use. Respiratory assist device use was not examined due to low numbers of users prior to onset of neurobehavioral concerns. Conclusion Selected neurobehavioral concerns were common among males with a dystrophinopathy. Reported associations highlight the importance of increased monitoring of neurobehavioral concerns as interventions are implemented and disease progresses.

The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy

“…The spectrum of cognitive issues in DMD include a downward shift in intelligence, deficits in executive function and working memory, speech delay, learning difficulties, and social anxiety problems . There is also an increased incidence of neurodevelopmental disorders such as obsessive‐compulsive disorder (OCD), attention‐deficit hyperactivity disorder (ADHD), and autism spectrum disorder (ASD) ADHD is twice more common and ASD is four times more common in DMD than in the general population . Likewise, speech delay is 10 times more common in DMD …”

Secondary Conditions Among Males With Duchenne or Becker Muscular Dystrophy

“…6 Previous studies have typically focused on a single or few conditions that could be considered secondary to a diagnosis of Duchenne muscular dystrophy. 7 –25 Collectively, these studies suggest a higher than expected occurrence of conditions affecting cognition, behavior, gastrointestinal, and genitourinary functioning.…”

Needs management in families affected by childhood-onset dystrophinopathies

“…depression and anxiety) and externalizing (e.g. aggression, hyperactivity) neurobehavioral problems have been highlighted in several recent publications 39–41 and have been identified for older individuals with DMD as an area of importance that needs further attention. 21 The failure to meet adequately the mental health needs of these individuals might be due to a lack of recognition and referral on the part of medical professionals, but might also reflect the national need for mental health service providers.…”