Sociodemographic and Clinical Profile of Adult Males with Duchenne Muscular Dystrophy


Topic:

Other

Poster Number: P107

Author(s):

Manju Jayaram, MPH, New York State Department of Health, Hollie Clark, PhD, Centers for Disease Control and Prevention, Atlanta, GA, Shiny Thomas, New York State Department of Health, Albany, NY, Christina Westfield, South Carolina Department of Public Health, Columbia, SC, Emma Ciafaloni, MD, University of Rochester, Katherine Mathews, MD, University of Iowa, Shannon Kilburn, MS, New York State Department of Health, Albany, NY, Aida Soim, PhD, MD, New York State Department of Health, Albany, NY, Muscular Dystrophy Surveillance, Tracking, and Research Network), N/A

Introduction: Advancements in clinical care have enabled many individuals with Duchenne Muscular Dystrophy (DMD) to live longer into adulthood, highlighting the need for studies to characterize this emerging population.

Objective: To provide an updated sociodemographic and clinical profile of adult (≥18 years) males with DMD using population-based surveillance data from the Muscular Dystrophy Surveillance Tracking and Research network (MD STARnet).

Methods: Previously, MD STARnet data were used to describe sociodemographic and clinical characteristics of adult males with DMD, born since 01/01/1982, who had healthcare visits through 2011. The present study adds healthcare visit data for 2012-2016. We provide an updated profile for 173 adult males within the 3 sites (Colorado, Iowa, New York) and describe sociodemographic characteristics using the latest data, and clinical characteristics based on first recorded diagnoses and interventions.

Results: As of 12/31/2016, nearly 53% of individuals were alive, 10% were lost to follow up, and 38% were deceased (median age at death=22.6 years). Over half (65%) were non-Hispanic White. At last clinic visit, the cohort had a median age of 22.5 years. Nearly 58% had public health insurance, 6% had private, and 32% had both. Nearly 6% had not completed high school, 72% had completed high school and/or further education, and in 22% data were unavailable. Most had been diagnosed with cardiomyopathy (74%). Fractures and neurobehavioral issues each were recorded in nearly 60%. Most (79%) were on non-invasive ventilation, and most (84%) were taking cardiac medications. Corticosteroids were ever used by 67% but by only 8.7% at time of last clinic visit or death.

Discussion: Our multi-site study results provide a benchmark for understanding DMD characteristics in adult males, and for examining the impact of evolving DMD care.