Role Attainment in Emerging Adulthood: Subjective Evaluation by Male Adolescents and Adults with Duchenne and Becker Muscular Dystrophy

Article type: Research Article

Authors: Peay, Holly L.^{a; *} | Do, Barbara T.^b | Khosla, Neil^c | Paramsothy, Pangaja^d | Erickson, Stephen W.^a | Lamb, Molly M.^e | Whitehead, Nedra^f | Fox, Deborah J.^g | Pandya, Shree^h | Kinnett, Kathiⁱ | Wolff, Jodi^{j; k} | Howard, James F.^{l; #}

Affiliations: [a] Genomics, Bioinformatics, and Translational Research Center, RTI International, Research Triangle Park, NC, USA | [b] Clinical Research Network Center, RTI International, Research Triangle Park, NC, USA | [c] Oak Ridge Institute for Science and Education (ORISE) Fellowship, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA | [d] National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta GA, USA | [e] Department of Epidemiology, Colorado School of Public Health, University of Colorado Anschutz Medical Campus, Aurora, CO, USA | [f] Applied Public Health Research Center, RTI International, Research Triangle Park, NC, USA | [g] New York State Department of Health, Albany, NY, USA | [h] School of Medicine and Dentistry, University of Rochester, Rochester, NY, USA | [i] Parent Project Muscular Dystrophy, Hackensack, NJ, USA | [j] Santhera Pharmaceuticals, Burlington, MA, USA | [k] Stride Bio, Research Triangle Park, NC, USA | [1] Department of Neurology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA

Correspondence: [*] Correspondence to: Holly Peay, PhD, RTI International, 3040 E Cornwallis Rd, Research Triangle Park NC 27613, USA. Tel.: +1 919 485 7734; E-mail: hpeay@rti.org.

Note: [#] On behalf of the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet).

Abstract: Background:Youth with Duchenne and Becker muscular dystrophy (DBMD) experience challenges in attaining adult roles, which may impact quality of life. New interventions and treatments may facilitate adult role attainment through improved function. Historical data on adult role attainment is important to assess the impact of new interventions on teens and young adults with DBMD. This study assesses medical knowledge, independence and employment, and relationships among adolescents and young adults with DBMD. Methods:This study uses data from a 2013 Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) survey on adult transition. Males with DBMD aged 16–30 years were included. Results:Sixty-five of 258 eligible males participated; we report results on 60 participants with an MD STARnet case definition of DMD or BMD. Individuals with BMD reported higher rates than those with DMD of frequently staying home without supervision (50% BMD; 14% DMD), independently performing daily physical needs (93% BMD; 7% DMD) and being employed full or part time (33% BMD; 4% DMD). Most participants understood medication and physical therapy goals; less than half indicated being often or always responsible for scheduling DMBD-related management and refilling medications. Most had not been in a romantic relationship but reported desiring such relationships. Conclusions:Our data reinforce the impact of DMD (and to a lesser extent, BMD) on transition to adult roles. These results provide an important historical comparator for teen and adult patients who are trying new interventions and therapies. Such data are important for assessing the quality-of-life impact of new treatments and to inform support and training programs for people with DBMD as they transition to new adult roles and responsibilities.

Keywords: Muscular dystrophies, transition to adult care, transfer from pediatric to adult care, self-care, adolescent

DOI: 10.3233/JND-210709

Journal: Journal of Neuromuscular Diseases, vol. 9, no. 3, pp. 447-456, 2022