Sociodemographic and Clinical Profile of Adult Males With Duchenne Muscular Dystrophy

INTRODUCTION/AIMS: Due to improvements in clinical care, individuals with Duchenne muscular dystrophy (DMD) are living into adulthood, but little has been published about adults with DMD. We describe key characteristics of adults with DMD using US population-based surveillance data. METHODS: Muscular Dystrophy Surveillance Tracking and Research network (MD STARnet) data were used to describe sociodemographic and clinical characteristics of living and deceased adult males with DMD, born since January 1, 1982, who were ≥ 18 years old at last healthcare visit, using healthcare visit data through 2016. Frequencies and percentages were used to describe the distribution of characteristics, and ages at key milestones were described using medians and interquartile ranges. RESULTS: As of December 31, 2016, of 173 adult males, 61.8% were living. At their last visit, median age was 22.5 years and 35.3% had completed some college. Over 75% were on noninvasive ventilation, over 70% had cardiomyopathy, and 83.8% were prescribed cardiac medications. Corticosteroids were used by 67.1%, including 8.7% as of last clinic visit/death. Public insurance was more common among deceased (66.7%) than living (52.3%). Cough assist was used by 82.2% of living versus 57.6% of deceased individuals. DISCUSSION: Except for cough assist and insurance type, sociodemographic and clinical characteristics were similar between the living and deceased. Low continuation of corticosteroids into adulthood needs interpretation in the context of the study's timeframe. These results can assist clinicians in anticipating the care needs of adults with DMD.