Achieving life milestones in Duchenne/Becker muscular dystrophy: A retrospective analysis
AbstractObjective:To understand the milestones achieved in the transition from childhood to adulthood for patients with Duchenne and Becker Muscular Dystrophies (DMD/BMD).Methods:We performed a retrospective chart review on patients ≥ 15 years of age with a clinical diagnosis of DMD/BMD who received care from January 1, 2008 to 2018 at the University of Kansas Medical Center and the University of Rochester Medical Centers. Participants were identified using local MDA-funded clinic lists, neuromuscular research databases, and electronic medical record review. Data was abstracted using a uniform template on education, employment, community resources, relationships, and end of life discussions, and is presented as mean, median or frequency with associated 95% confidence interval (CI).Results:109 patients were identified: patients ranged in age from 15 to 56 years with a median of 24, and covered a 5-state region and Ontario, Canada. Seventy-eight percent of patients had DMD, and were, on average, 8.5 years younger than BMD patients. Over half (56.9%, 95% CI 47.6, 66.2) were high school graduates or beyond. Sixteen percent did not have their highest level of education documented. Only 20.0% had an occupation (95% CI 12.7, 27.7), most frequently in education and administrative support (34%). The majority were still living with parents (80.7%, 95% CI, 73.3, 88.1). A minority reported having end of life discussions (17.4%, 95% CI 10.3, 24.6).Conclusion:Psychosocial elements reflecting the transition to adulthood are inconsistently reported in clinical documentation. A prospective study will further elucidate this transition.