P.7.5 Sociodemographic and health related profile of adults with Duchenne/Becker Muscular Dystrophy (DBMD): Data from the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet)

Improved respiratory and cardiac management along with glucocorticoid therapy have contributed to increasing survival of patients with DBMD.This has led to a growing population of adults with DBMD. The purpose of this population based study was to describe the sociodemographic profile and health related outcomes of individuals with DBMD followed in the MD STARnet. MD STARnet is a multi state population based surveillance system that collects data on all individuals with DBMD born since 1/1/1982. In addition to epidemiologic data, MD STARnet collects extensive sociodemograhic and clinical data related to natural history milestones and treatments and services received by individuals with DBMD. The cohort for this analysis was all individuals born from 1/1/1982 to 12/31/1992 and followed through 8/31/2011. Overall, 384 individuals were identified of which 32% have died [mean age 17.6 years (8.7–25.5)], and 20% were lost to follow up [mean age 15.3 (0.9–25.8)]. The remainder continue to be followed [mean age 21.5 (17–28.6)]. Most individuals were white, non-Hispanic (59%) and live with parent/s/family (98%), have post high school/college training (24%). The number of individuals and the average age (range) at which they crossed clinical milestones or required specific treatments were as follows: loss of ambulation [ N = 300, 11.7 years (6.9–24.50)], scoliosis surgery [ N = 128, 14.7 years (10.3–20.2)], night time ventilator support [ N = 158, 16.9 years (9.4–26.2)], cardiomyopathy (SF N = 188, 16.5 years (9–25.5)], PEG [ N = 58, 19.2 years (11.8–26.0)]. Forty nine percentage have been treated with corticosteroids. Our population based data support an emerging population of adults with DBMD. This sociodemographic and health related profile provides information for providers, payors and policy makers to help design the appropriate care models for the needs of adults with DBMD and their families.