Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling.
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.
Parent Project Muscular Dystrophy, 401 Hackensack Ave 9th Floor, Hackensack, NJ, 07601, USA. holly@parentprojectmd.org.|Department of Clinical Genetics, Leiden University Medical Centre, Leiden, The Netherlands. holly@parentprojectmd.org.
Holly Landrum
HL
Peay
Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
I L
IL
Hollin
Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
J F P
JF
Bridges
D000203:Activities of Daily Living / Q000145:classification / Q000523:psychology; D000328:Adult; D001007:Anxiety / Q000175:diagnosis* / Q000523:psychology*; D002648:Child; D002675:Child, Preschool; D004185:Disability Evaluation; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D051346:Mobility Limitation; D020388:Muscular Dystrophy, Duchenne / Q000145:classification* / Q000175:diagnosis / Q000235:genetics* / Q000523:psychology; D016487:Parenting / Q000523:psychology; D010290:Parents / Q000523:psychology*; D011379:Prognosis; D011594:Psychometrics; D015203:Reproducibility of Results; D018434:Sickness Impact Profile
Best-worst scaling; Disease impact; Duchenne muscular dystrophy; Worry