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Updated: Apr 5, 2026
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling
Holly Landrum Peay1,2, I L Hollin3, J F P Bridges3
1Parent Project Muscular Dystrophy, 401 Hackensack Ave 9th Floor, Hackensack, NJ, 07601, USA. holly@parentprojectmd.org.
Insights
Parents of children with Duchenne muscular dystrophy (DMD) prioritize worries about weakness progression and care access. Interventions should address the emotional toll of caring for a child with this progressive, fatal disease.
Area of Science:
- Pediatric rare diseases
- Clinical psychology
- Patient-reported outcomes
Background:
- Duchenne muscular dystrophy (DMD) is a severe pediatric disorder impacting families.
- Understanding parental concerns is crucial for effective clinical support.
- Existing assessment methods may not fully capture parental priorities.
Purpose of the Study:
- To identify and prioritize parental worries related to Duchenne muscular dystrophy.
- To compare these priorities between parents of ambulatory and non-ambulatory children.
- To demonstrate the utility of Best-Worst Scaling (BWS) in assessing parental concerns.
Main Methods:
- A community-engaged approach identified 16 short-term worries in DMD.
- Best-Worst Scaling (BWS) was employed with 119 parents to rank worries.
- Independent-sample t-tests analyzed differences based on child's ambulatory status.
Main Results:
- The highest parental priorities were weakness progression and consistent care access.
- Parents of ambulatory children prioritized avoiding missed treatments and parental adequacy more than those with non-ambulatory children.
- Parents of non-ambulatory children were more concerned about their child feeling like a burden.
Conclusions:
- Caregiver interventions must address the emotional burden of DMD, irrespective of disease stage.
- BWS provides a clinically relevant and accessible method for prioritizing patient and caregiver concerns.
- This approach offers an alternative to traditional rating scales for assessing disease impact.
Abstract:
Duchenne muscular dystrophy (DMD) is a progressive, fatal pediatric disorder with significant burden on parents. Assessing disease impact can inform clinical interventions. Best-worst scaling (BWS) was used to elicit parental priorities among 16 short-term, DMD-related worries identified through community engagement. Respondents viewed 16 subsets of worries, identified using a balanced, incomplete block design, and identified the most and least worrying items. Priorities were assessed using best-worst scores (spanning +1 to -1) representing the relative number of times items were endorsed as most and least worrying. Independent-sample t-tests compared prioritization of parents with ambulatory and non-ambulatory children. Participants (n = 119) most prioritized worries about weakness progression (BW score = 0.64) and getting the right care over time (BW = 0.25). Compared to parents of non-ambulatory children, parents of ambulatory children more highly prioritized missing treatments (BW = 0.31 vs. 0.13, p < 0.001) and being a good enough parent (BW = 0.06 vs. -0.08, p = 0.010), and less prioritized child feeling like a burden (BW = -0.24 vs. -0.07, p < 0.001). Regardless of child's disease stage, caregiver interventions should address the emotional impact of caring for a child with a progressive, fatal disease. We demonstrate an accessible, clinically-relevant approach to prioritize disease impact using BWS, which offers an alternative to the use of traditional rating/ranking scales.
