Sociodemographic and Clinical Profile of a Cohort of Males with Becker Muscular Dystrophy



Poster Number: T302


Natalie Street, MS, CGC, Centers for Disease Control and Prevention, Hollie Clark, PhD, Centers for Disease Control and Prevention, Shannon Kilburn, MS, New York State Department of Health, Julia Finn, MS, Oak Ridge Institute for Science and Education, Centers for Disease Control and Prevention, Katherine Mathews, MD, FAAN, University of Iowa, Aida Soim, PhD, MD, New York State Department of Health, Pangaja Paramsothy, PhD, MPH, Centers for Disease Control and Prevention

We expanded upon previous studies from clinics or registries by using U.S. population-based surveillance data to describe sociodemographic and clinical characteristics of males with childhood-onset Becker muscular dystrophy (BMD).

Using data from five sites in the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet), we performed a descriptive analysis to determine sociodemographic features and median ages at which individuals with BMD reached clinical milestones and received interventions. We included males who were born and diagnosed with a dystrophinopathy during 1982-2011, had definite or probable BMD, and were aged ≥16 years at last clinic visit. Males were followed through 2011 (two sites) or 2016 (three sites). We examined vital status, race/ethnicity, and health insurance and calculated percentages and median ages for reaching clinical milestones (ambulation loss, impaired pulmonary or cardiac function) and initiating interventions (invasive/noninvasive ventilation, corticosteroids, musculoskeletal surgeries).

Among 74 males with BMD, median age at last clinic visit was 21 years (range: 16-35); 68% were Non-Hispanic White, and 95% were living. Forty-three percent had private health insurance, 28% had public, and 19% had both. Nearly one-quarter (23%) had ceased ambulation; median age at ambulation loss was 18 years (range: 13-31). Thirty-two percent had ever taken corticosteroids; median age at initiation was 10 years (range: 6-30). Fifteen percent ever had a forced vital capacity (FVC) value <50%; median age at first FVC<50% was 19 years (range: 14-28), and 11% had ever used ventilation. Thirty-seven percent ever had an abnormal echocardiogram; median age at first abnormal echocardiogram was 17 years (range: 10-32). Eleven males (15%) had tendon release or scoliosis surgery. Among males with BMD with a median age of 21 most were still ambulating; a minority had impaired pulmonary and cardiac function. These data can be used to help determine healthcare needs of this population.