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Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling

  • Original Research
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Journal of Genetic Counseling

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.

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Acknowledgments

We appreciate the leadership and commitment of the Parent Project Muscular Dystrophy oversight committee: Pat Furlong, Brian Denger, Sharon Hesterlee, and Kathleen Kinnett. We are indebted to the stakeholder informants, parents who participated in the cognitive interviews, and caregivers who completed the survey. We acknowledge Aad Tibben’s review and comments and Hadar Scharff’s study assistance.

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Authors and Affiliations

  1. Parent Project Muscular Dystrophy, 401 Hackensack Ave 9th Floor, Hackensack, NJ, 07601, USA

    Holly Landrum Peay

  2. Department of Clinical Genetics, Leiden University Medical Centre, Leiden, The Netherlands

    Holly Landrum Peay

  3. Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA

    I. L. Hollin & J. F. P. Bridges

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  1. Holly Landrum Peay
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  2. I. L. Hollin
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  3. J. F. P. Bridges
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Correspondence to Holly Landrum Peay.

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Conflict of Interest This study was funded by Parent Project Muscular Dystrophy (PPMD). Holly Landrum Peay was an employee of PPMD during the study development, data collection, and manuscript writing, and John FP Bridges was hired as a consultant by PPMD for this project. Ilene L. Hollin has no conflicts to disclose.

Human Studies and Informed Consent All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2000 (5). Informed consent was obtained from all patients for being included in the study.

Animal Studies No animal studies were carried out by the authors for this article.

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Peay, H.L., Hollin, I.L. & Bridges, J.F.P. Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling. J Genet Counsel 25, 305–313 (2016). https://doi.org/10.1007/s10897-015-9872-2

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  • DOI: https://doi.org/10.1007/s10897-015-9872-2

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