Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to management across a range of health-care specialties. In part 3 of this update of the DMD care considerations, we focus on primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Many primary care and emergency medicine clinicians are inexperienced at managing the complications of DMD. We provide a guide to the acute and chronic medical conditions that these first-line providers are likely to encounter. With prolonged survival, individuals with DMD face a unique set of challenges related to psychosocial issues and transitions of care. We discuss assessments and interventions that are designed to improve mental health and independence, functionality, and quality of life in critical domains of living, including health care, education, employment, interpersonal relationships, and intimacy.

The data suggest a need for improved awareness of palliative care and related services among families of young men with DMD.

POTENTIAL CONFLICT OF INTEREST: Within the past 12 months, Dr Gibbons was a consultant for Sarepta Therapeutics and attended an advisory board meeting; the other authors have indicated they have no potential conflicts of interest to disclose. Significant progress has been made in managing the medical needs of patients who have Duchenne muscular dystrophy (DMD). As a result, many patients live well into adulthood and have the potential to lead independent, productive lives. However, the challenge of coping with a chronic and progressive illness from childhood into young adulthood is formidable. Close monitoring and appropriate interventions focused on improving emotional and social functioning should be The guidelines or recommendations in this article are not American Academy of Pediatrics policy, and publication herein does not imply endorsement. In this article, we outline a comprehensive plan for the psychosocial management of patients with Duchenne muscular dystrophy (DMD) across the life span. In 2010, the Centers for Disease Control and Prevention sponsored the development of multidisciplinary management guidance for DMD, and in 2018, that guidance was updated. In the intervening years, a new emphasis was placed on studying and addressing the psychosocial issues that affect patients with DMD, driven in part by improved patient survival. Once viewed as ancillary to managing the significant medical needs of patients with DMD, it is now standard practice to integrate psychosocial management into the multidisciplinary management of the disease. It is also increasingly recognized that neurodevelopmental disorders in DMD occur at a higher rate than what was previously understood and that these disorders warrant early and intensive intervention. In this article, we expand on the content found in the 2018 DMD Care Considerations.

Introduction Care Considerations supported by the Centers for Disease Control and Prevention for the management of Duchenne muscular dystrophy were published in 2010, but there has been limited study of implementation in the United States. Methods A questionnaire collecting information about standard care practices and perceived barriers was piloted by 9 clinic directors of facilities within the Muscular Dystrophy Surveillance, Tracking and Research network. Results Six clinic directors completed the questionnaire; 1 adult-only clinic was excluded. Over 80% adherence was found for 30 of 55 recommendations examined. Greatest variability was for initiation of corticosteroids, bone health monitoring, type of pulmonary function testing, and psychosocial management. Barriers included unclear guidelines, inadequate time and funding, family-specific barriers and lack of empirical support for some recommendations. Discussion This pilot study showed implementation of the 2010 Care Considerations, except for recommendations based largely on expert consensus. Complete adherence requires more studies and active promotion.

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.