Survey Explores Needs of Patients With DMD, BMD Transitioning to Adulthood

Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition to adult life, according to a study published in PLOS One.

DMD is an inherited neuromuscular disorder that is incurable and progressively impacts a patient's functional independence.² This affects quality of life for both patients and their caregivers.

Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy or Becker muscular dystrophy. | Image credit: Nana_studio - stock.adobe.com

The cross-sectional study explored the transition needs of patients with DMD or BMD in a cohort of 42 males aged 16 to 35 years.¹ The average age was 24.3 years, and 66.6% of participants in the study made full use of assistive mobility devices. The mean age in the DMD group was 26.43, and the mean age in the BMD group was 23.45 years.

The survey included 5 assessment domains: health care, education and employment, housing and transportation, activities of daily living, and independent life and autonomy. In each domain, patients reported whether they need help, partially completed, or fully completed various items.

In the health care domain, 45% of patients reported needing help with various aspects. Notably, 1 item that fewer participants reported fully completing was consultation regarding palliative care (33.3%). Most patients had fully completed the tasks of involvement in the decision-making process (66.6%), familiarization with general practitioner (71.4%), and conversations on anticipated changes in health (69%).

A smaller proportion of patients (23.8%) reported fully completing the tasks of approaching employment or educational support groups in the education and employment domain. Less than half (40.4%) fully completed the task of initiation of planning living options after studying.

Regarding the domain of housing and transportation plans, only a small percentage of patients (23.8%) completed the task of consultation with a social worker regarding eligibility for residential assistance. For 2 items—performing an assessment of the accessibility of the family home and discussing mobility using the family car—most patients reported fully completing these tasks (78.5% and 80.9%, respectively).

In the activities of daily living domain, 35.7% of patients reported fully completing 2 items: receiving instruction on hiring aid and receiving consultation on assistive technology. Regarding the independent life and autonomy domain, high proportions of patients (between 69.0% and 90.4%) reported fully completing 6 of 12 items.

The researchers also assessed the relationships between transition domains and age, number of siblings, mobility ability, and respiratory function. The only statistically significant correlations were between number of siblings and all of the domains, except independent life and autonomy and education and employment.

“The study findings reinforce the impact of DMD and BMD on the transition to adult roles,” the authors wrote. “More specifically, the survey data suggest that activities of daily living—mainly hiring an aide and consulting on accessories and assistive technology—is an important area of need. Another domain with a high percentage of participants needing help was education and employment, especially in approaching support groups and initiating planning of living options after education.”

There was a lack of connection between age and most of the domains, which suggests a need for long-term assistance regardless of a patient’s age, the authors noted. Independent life and autonomy was the domain associated with the least difficulty, although there were aspects that patients found challenging. These included knowledge on actions to promote relationships and whom to turn to for professional help regarding intimacy.

“The current study also yielded some unique findings, such as the null association of the level of motor and respiratory disability on transition outcomes, and the significance of sociodemographic factors like the number of siblings,” the authors concluded. “These results may be relevant for professionals aiming to develop training programs to facilitate the transition of people with DMD or BMD to new adult roles and responsibilities.”

References

1. Barak S, Ackerman Laufer S, Gudinsky Elyashiv M, Rubinstein-Shatz S, Davidson R, Kaplan T. Navigating adulthood: exploring the transition needs of adolescents and young adults affected by Duchenne or Becker muscular dystrophy. PLoS One. 2025;20(1):e0317006. doi:10.1371/journal.pone.0317006

2. Uttley L, Carlton J, Woods HB, Brazier J. A review of quality of life themes in Duchenne muscular dystrophy for patients and carers. Health Qual Life Outcomes. 2018;16(1):237. doi:10.1186/s12955-018-1062-0