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.